Download celgene-corporate

Living Longer
Better and Healthier Lives
Celgene Key Facts
M edical Innovation =
V + A + L + U + E
Increased societal
VALUE
Every 1 percent reduction
in United States cancer
related deaths = $500 billion
in societal value.1 Since 1990,
cancer patients in the U.S.
have enjoyed 50 million addi­
tional years of life and gener­
ated $4.1 trillion in additional
economic activity. 2
ADVANCING the quality of life
Greater
Patients are not just surviving;
they are thriving—spending
less time in hospitals,3 more
time with their families, able to
continue with jobs and careers.
New medicines account for 73
percent of the increase in life
expectancy.4 Between 1990–
2014, the number of cancer
survivors more than doubled—
from six million to 14.5 million.5,6
LONGEVITY
UNENCUMBERED access
ECONOMIC impact
Between 1990 and 2010, the
average time spent in the
hospital by cancer patients
declined by 70 percent, sav­
ing $250 billion, that is directly
attributable to access to medi­
cal innovation.7
The overall economic impact
of the biopharmaceutical
sector on the U.S. economy
totals about $790 billion on
an annual basis when direct,
indirect and induced effects
are considered.8
K ey Facts 2015
CELGENE IS COMMITTED TO IMPROVING THE
LIVES OF PATIENTS WORLDWIDE
•M
ore than 46,000 patients have been helped by Celgene’s
Patient Support® Program.9
• Celgene’s broad and deep pipeline currently has 50 programs
in pre-clinical development, 25 treatments in clinical trials and
15 pivotal Phase III programs underway.10
• Celgene Annual Reports 2010–2014 reflect average research
and development (R&D) investment of 32.29 percent of total
revenue based on U.S. GAAP.11
• Celgene’s R&D expenditure per employee is approximately
$265,000.12
KEY HEALTHCARE STATISTICS
•A
dvances in medical innovation have yielded large societal
gains. There has been a 96 percent decrease in deaths per
100,000 people and a 62 percent increase in life expectancy
since 1900.13
• For every $1 spent on innovative medicines, total healthcare
spending in the U.S. is reduced by $6.20.14
• Only 10 percent of U.S. healthcare spending is dedicated to
biopharmaceuticals, with new targeted cancer-related therapies
accounting for approximately one percent. That percentage
has remained constant for decades and is expected to remain
the same through the next decade.15,16,17
• Innovative medicines accounted for 73 percent of the increase
in life expectancy between 2000 and 2009.18,19
• Every 1 percent increase in prescriptions filled among Medicare
beneficiaries lowers Medicare spending for other health services
by 0.2 percent.20
ACCESS AND ADHERENCE
According to a 2011 Journal of the American Medical Associ­ation
(JAMA) study, improved access and adherence to medicines
through Part D saves Medicare about $1,200 per year in hospital,
nursing home and other costs for each individual who previously
lacked comprehensive prescription drug coverage. This reduction
achieved about $13 billion in overall savings during the first full
year of Part D.21
MEDICAL INNOVATION IN CANCER
•O
ver the last two decades, there has been a 22 percent
decrease in cancer-related deaths in the U.S.22
• As of 2014, the U.S. five-year relative survival rate for all
cancers diagnosed between 2003 and 2009 is 68 percent.23
• U.S. cancer survivorship has more than doubled, with nearly
14.5 million cancer survivors alive today.24
• For decades, treating cancer has remained just five percent
of the total U.S. healthcare expenditure.25,26,27,28,29
• Eighty-three percent of survival gains since 1975 in cancer are
attributable to new medicines.30
• The chances that a cancer patient will live five years or more
has increased by 39 percent since 1975.31
• Survival rates for childhood cancers have increased 58 percent
over the last several decades.32
COST OF DEVELOPING NEW THERAPIES
According to the Tufts Center for the Study of Drug Development,
the average cost to develop one new approved therapy more
than tripled between the late 1990s and 2014, costing up to
$2.6 billion.33
MEDICAL INNOVATION IN INFLAMMATION AND
IMMUNOLOGY (I&I)
•A
first-of-its-kind multinational survey examined the impact of
psoriasis and psoriatic arthritis on patients’ lives. The survey
revealed several areas that warrant further attention and action,
including high rates of under-treatment, a mismatch between
patient/physician assessment of the disease and the desire for
new treatment options.34
• Based on the same survey, half of psoriasis and psoriatic
arthritis patients found their current traditional or biologic treatments burdensome.35
• The estimated economic burden for psoriasis, psoriatic arthritis,
Chron’s disease and rheumatoid arthritis is approximately $51
billion.36,37,38,39,40
• There are more than 80 medicines in development for immune
disorders like psoriasis, psoriatic arthritis, rheumatoid arthritis
and Chron’s disease.41,42
Living Longer, Better and Healthier Lives
Sustaining a
Virtuous Cycle of
Medical Innovation
focus is on building long-term strategic partnerships with
these groups that provide important insights to advance
research and clinical trial design, access, medical innovation, patient policies and education.
It extends to our research partners who work with us to
build and expand next generation capabilities, including
new treatment combinations that have never been tried
before. Our history of perseverance in the name of scientific discovery, even when the outcome was unclear and
Medical innovation is a virtuous
cycle. A cycle in which each
action creates the building blocks
for next-generation therapies,
improved healthcare, longer life
and economic growth.
the benefit uncertain, along with our willingness to demonstrate the courage to think differently, act boldly and set
high aspirations, has created a reputation that positions
Celgene as a partner of choice for global thought leaders
in science and medicine.
Of great importance is the strength of our business
model, a unique model that positions us in a leadership
role in the fields of hematology, oncology, and now,
forging ahead with our paradigm-shifting therapies for
immune-inflammatory diseases. Our belief in the potential
At Celgene, we take our role in the healthcare ecosystem
of this new business is so strong that we decided to build
very seriously, and we strive to embody our position as a
an entirely new franchise that can benefit patients world-
leader in medical innovation, pursuing transformational
wide with immune-mediated diseases.
science that will translate into life-enhancing medicines.
We believe that being a leader in healthcare means that
Our ultimate goal is to change the course of human health
we drive in new scientific solutions, innovative medicines,
while promising to always put patients first. We do this by
patient and health economic outcomes and social/economic
protecting and nurturing an environment, inside Celgene
benefits. It means that we lead not just within our own
and across the larger healthcare ecosystem, where inno-
industry, but across the entire healthcare ecosystem.
vation can flourish. This is paramount to us because
patients are at the center of our mission. Patients want to
This profile—Living Longer, Better and Healthier Lives—
live longer, better and healthier lives. Patients want the
provides an overview of how Celgene’s core commitment
strength to do what matters most to them. Patients want
to patients supports the virtuous cycle of medical innova-
to be active participants in the lives of their loved ones
tion across many spheres. In Chapter 1, we explore how
and communities.
our work has resulted in patients living longer, better and
healthier lives through breakthroughs in blood cancers,
Biopharmaceutical leadership is inherently expressed in
solid tumors and inflammation and immunology. Chapter
the virtuous cycle: through medical innovation that has
2 focuses on the impact of Celgene’s commitment to
increased U.S. life expectancy from 68 years in 1950
reducing the burden of disease through improved quality
to 79 years in 2010;43 through research that generates a
of life, better outcomes, reduced hospitalizations and
seven­fold savings in healthcare expenditures; through
increased productivity, as seen through the eyes of patients.
biopharmaceutical investment that is more than ten times
The role of medical innovation in boosting economic pros-
the amount of R&D per employee than manufacturing
perity, societal benefit and the healthcare ecosystem
industries overall;44 and through patient access support
is highlighted in Chapter 3. Celgene’s immensely deep
programs. The Celgene Patient Support Program has
and diverse pipeline of high-potential compounds across
impacted over 50,000 patients since 2007.45
hematology, oncology, inflammation and immunology and
The virtuous cycle also extends to helping patients advo-
cellular therapeutics comprises the topic of turning science
cate for their needs. Celgene works with nearly 150 patient
into reality for patients in Chapter 4. And in Chapter 5, we
advocacy groups worldwide that are dedicated to support-
discuss the future of medical innovation and our role in
ing and advocating for patients and their families. Our
reaching toward a world free from cancer.
1
Contents
Celgene Contents
Innovation leads to the discovery and development of therapies that save and extend lives46 —the primary goal of any
healthcare program. And by doing that, innovation helps keep healthcare costs down and drives economic growth.47,48,49
Thanks to medical innovation patients are living longer, healthier, more productive lives, a benefit that extends across
the entire healthcare ecosystem to society as a whole. Simply put, the longer one lives a healthy life, the more time that
1
2
3
4
5
person has to be productive and contribute to their families and society.
LIVING LONGER, BETTER AND HEALTHIER LIVES, page 4
Commitment to Patients + Medical Innovation = Social Value
• Changing the Course of Human Health
• The Value of Medical Innovation
BETTER HEALTHCARE, BETTER OUTCOMES, page 18
Longevity, Quality of Life, Reduced Hospitalizations & Added Productivity
Patient Profiles:
• Multiple Myeloma
• Psoriasis
• Myelodysplastic Syndromes (MDS)
• Psoriatic Arthritis
• Acute Lymphoblastic Leukemia (ALL)
PROGRESS AND PROSPERITY, page 30
Medical Innovation Reduces Costs and Saves Lives
• Creating Societal Value
• More Than 50 Million Life Years Saved
• Accelerating Medical Innovation in Immune Disorders
TURNING SCIENCE INTO REALITY, page 38
Greater Investment in R&D Leads to Better Health and Longer Life
•
•
•
•
•
Pipeline of Possibilities
Hematology
Oncology
Inflammation and Immunology
Cellular Therapeutics
TRANSLATING SCIENCE INTO TRANSFORMATIONAL
MEDICINES, page 52
Working Toward a World Free from Cancer
•
•
•
•
A Tribute to Patients
Nurturing Innovation
The Value of Sharing: Project DataSphere
Toward a World Free from Cancer
2
Living Longer, Better and Healthier Lives
Letter from the Chairman and CEO
What will it take for America to continue as the world’s leader in medical
innovation? A stronger and more sustainable ecosystem of innovation.
It Takes an Ecosystem:
Alzheimer’s and other forms of dementia in the U.S. will
double by 2050.59 Without new medical innovations that
change the course of these diseases, the burden of these
conditions will overwhelm health systems and threaten
economic growth.
The challenges to U.S. leadership in medical innovation
are clear. According to a study published by the New
England Journal of Medicine, Asian investments in biomedical research and development have increased 51
percent while U.S. R&D expenditures have fallen nine
percent.50 That’s a $12-billion decline.
Curing diseases like these will require substantial investments of time and money. Developing the average new
drug takes over a decade, and accounting for failures,
costs more than one to two billion dollars.60 The risk of
failure is high. Hundreds of companies are working on
new cancer treatments. Only a fraction of those are likely
to produce new breakthroughs for patients.
Those numbers should serve as a wake-up call to America’s
leaders in both the public and private sectors. The federal
government, academia, patient advocacy groups and private sector research-driven companies are all essential
parts of our nation’s innovation ecosystem. All need to be
healthy and successful for the ecosystem to thrive. Suc­
cessful collaboration among all these sectors of the ecosystem will deliver new cures for patients, reduce the
burden on healthcare and grow the economy.
The odds are long, but the returns in longer, healthier lives
and stronger economic growth are astronomical. Each
one percent reduction in U.S. cancer-related deaths delivers $500 billion in value to society.61 Delaying the onset
of Alzheimer’s by just five years could reduce healthcare
expenditures by almost $600 billion over the next 20 years.
The impact of biomedical research is striking. Consider
cancer.
The biomedical research ecosystem is closer than ever to
achieving those goals. Globally, research-based biopharmaceutical companies spend $135 billion every year on
research and development.62 Biopharmaceutical company
scientists are investigating nearly 1,000 treatments that
could transform life-threatening cancers into chronic, manageable conditions. Another 100 are being developed as
potential therapies for Alzheimer’s.
Biopharmaceutical breakthroughs have contributed to a
22-percent drop in American cancer deaths over the last
two decades.51 The number of cancer survivors has doubled
in that time—from 6.3 million to 14.5 million.52 Since 1990,
cancer patients have enjoyed 50 million additional years
of life and generated $4.1 trillion in additional economic
activity,53 with 83 percent of life expectancy gains attributable to new treatments.54
But converting those research initiatives into new therapies
—and keeping the research pipeline flowing with promising new cures—requires more than science. It requires
collaborative, integrated solutions.
Just a generation ago, an HIV/AIDS diagnosis was tantamount to a death sentence. Since innovative treatments
were introduced in the 1990s, the disease’s death rate
has dropped by approximately 80 percent.55 Today, in
most cases HIV/AIDS is being successfully managed with
innovative medicines as a long-term chronic condition.
And it requires policies that ensure the health of our nation’s
ecosystem of innovation: increased support for the National
Institutes of Health’s vital basic research mission, a strong,
science-based Food and Drug Administration, and the
sustainable market-based access and reimbursement for
innovative medicines today that is necessary to incentivize
the long-term, high-risk investment needed for new medical breakthroughs in the future.
But patients aren’t the only ones who have benefited from
the biomedical research ecosystem. So has the economy.
Research-driven biopharmaceutical firms employ more
than 810,000 people directly and support a total of 3.4
million jobs across the country.56
For the sake of patients—and the broader economy—we
must work together to help make this happen.
All that economic activity generates tax revenue. In 2010,
the government collected $3.8 billion on the activity related
to the Human Genome Project. With just that one year’s
incremental tax revenue resulting from the Human Genome,
the government recouped 97 percent of its 13-year investment in the Project.57
But there are still many more health challenges to solve.
Over the next 20 years, the number of new cancer cases
is projected to increase by more than 50 percent, according to the World Health Organization.58 The incidence of
Robert J. Hugin is Chairman and Chief Executive Officer, Celgene Corporation.
3
Celgene Chapter 1
Living Longer, Better and H ealthier Lives
Commitment to Patients + Medical Innovation = Social Value
4
Living Longer, Better and Healthier Lives
Changing the Course of
Human Health
At Celgene we are changing the course of human health
patients, but a company that serves the changing model
through bold pursuits in science and a promise to
of healthcare and the needs of society, with a purpose to
always put patients first. The results are evident; we
change the course of health through medical innovation.
are delivering what matters most: a meaningful BLOOD CANCERS: We have developed first-in-class oral
difference in the lives of patients worldwide. We
immuno-therapeutics for blood cancers with increased
discover, develop and deliver innovative medicines
overall survivability and improved side-effect profiles. Our
that are helping patients live longer and better lives,
immuno-therapeutics such as REVLIMID ® (lenalidomide)
reducing the burden on healthcare systems and help-
and POMALYST® (pomalidomide)/IMNOVID ® inhibit cell
ing economies grow through greater productivity.
proliferation, promote anti-angiogenesis (cutting off the
In recent years we have seen expanded approvals for
blood supply to the cancer) and enhance the induction of
our flagship therapeutics based on unique mechanisms
apoptosis (cell death) in multiple myeloma cells.1,2
of action that differentiate them from other approved med­
Immunomodulatory properties include activation of T-cells
ications. Along with our partners, we are involved in a
and natural killer (NK) cells, increased numbers of NKT
deep and diverse platform of transformational science
cells, and inhibition of pro-inflammatory cytokines (e.g.,
including immunotherapies, epigenetics, protein homeo­
TNF-alpha and IL-6).
stasis, cancer metabolism, genomics, proteomics, kinase
The newest member of our immuno-therapeutics family
inhibitors, cellular therapies and beyond. As of the end
POMALYST, IMNOVID in Europe, recently won interna­
of 2014, our broad and deep pipeline of 22 programs in
tional approvals for patients who do not respond or who
clinical develop­ment touches roughly 28,000 patients
have stopped responding to other available regimens.
across 50 indications. More than 100 clinical trials are
In the U.S., our flagship therapy REVLIMID has received
under our development and we support more than 500
expanded indications since its initial approvals. As a
investigator-initiated trials using approved therapies or
result, REVLIMID ® is now approved in the U.S. in a investigational compounds from Celgene. These are the
number of indications, including: for patients with hallmarks of a biopharmaceutical leader that not only serves
multiple myeloma, for certain patients with MDS del5q* REVLIMID Prescribing Information
POMALYST Prescribing Information
“Patients are voting with their feet.” That is exactly how the founding physician of
a leading advocacy organization described patients’ response to the introduction of Celgene’s oral
blood cancer medications. Patients wanted the efficacy of our regimens, of course. But they also
liked the freedom and convenience of taking a pill with a glass of water at home instead of spending
hours in an intravenous infusion center, or weeks in a hospital after an invasive stem cell transplant.
*for patients with low- or intermediate-1-risk MDS with a deletion 5q chromosomal abnormality (MDS del5q)
5
Celgene Chapter 1
and mantle cell lymphoma (MCL).* In 2015, REVLIMID
INFLAMMATION AND IMMUNOLOGY: In 2014 we
became the first oral treatment in 50 years approved for all
launched our novel, oral anti-inflammatory agent OTEZLA®
patients with multiple myeloma. In Europe, REVLIMID is
(apremilast) for patients with psoriasis and psoriatic
arthritis. OTEZLA inhibits the enzyme PDE4 to modulate
indicated for patients with newly diagnosed elderly
non-transplant eligible multiple myeloma and MDS del5q.
inflammatory responses that lead to skin and joint condi­
3
tions.6 This novel therapeutic is changing the treatment
REVLIMID Prescribing Information
paradigm based on unique science and compelling clinical
efficacy and safety data from six Phase III programs—
SOLID TUMORS: ABRAXANE® (nab-paclitaxel) is a
again with strong efficacy and the convenience of a pill
nanotechnology agent that is currently the only nanomedi­
administered at home.
cine approved in oncology. Current indications include
In addition to approvals in the U.S. and Europe for psoriasis
metatstatic breast cancer, non-small cell lung cancer and
and psoriatic arthritis, ongoing studies are addressing
metastatic pancreatic cancer in the U.S., Europe and other
unmet medical needs for patients with ulcerative colitis,
markets around the world. It contains albumin-bound
ankylosing spondylitis, Behçet’s disease and more.7
paclitaxel nanoparticles and is manufactured using pat­
OTEZLA Prescribing Information
ented nab ® technology. ABRAXANE is formulated with
albumin, a human protein, and is free of solvents. The
protein coating utilizes the tumor’s own affinity for protein
to attract the ABRAXANE and pull it inside where it may
OTEZLA offers a valuable
treatment option for a spectrum of
plaque psoriasis patients—patients who
are treatment-naive as well as patients
who are treatment-experienced, including those previously treated with biologic agents or conventional systemic
agents.8 The FDA and EMA approvals
of OTEZLA for moderate to severe psoriasis and psoriatic arthritis reflects
Celgene’s commitment to extending the
reach of our research and science in
an effort to improve the lives of people
worldwide living with chronic inflammatory diseases.”
stop cancer cells from dividing and induce cell-death.
ABRAXANE Prescribing Information
We have been able to show
that the addition of ABRAXANE to conventional treatment with gemcitabine
provides substantial benefits in overall
survival,4 with manageable side effects.5
My perception is that this approval is
going to mandate a change in the way
that we treat patients, offering a new
option with a good efficacy and safety
profile.”
Josep Tabernero, MD, Head of the Medical Oncology
Department, Vall d’Hebron Institute of Oncology Barcelona,
Spain, primary investigator for the MPACT trial
Scott Smith, President, Inflammation and Immunology,
Celgene Corporation
*for patients with relapsed/refractory mantle cell lymphoma (MCL) after they have received at least two prior therapies, one of which included bortezomib.
6
Living Longer, Better and Healthier Lives
Multiple Pathways
Continuing
Advances in
Precision Medicine
A vast array of research projects is underway developing the right therapeutic approach, for the right
patient, at the right time in a certain stage of their disease that advances the approach that works
best for the patient and delivers greatest value to the healthcare system. Precision medicine is one of
the hallmarks of new cancer research because each type of cancer may be a specific sub-type that
does not respond to one-size-fits-all treatment.9 But what if multiple myeloma actually has multiple
personalities, that is, diverse genetic patterns within the same cancer samples in the same patients?
“So myeloma is sneaky: it can evolve over time. Multiple pathways within the myeloma cell
are involved.”10
Brian G.M. Durie, M.D., chairman and co-founder of the International Myeloma Foundation (IMF)
wrote about this phenomenon in April 2012. He says this is why combinations of medicines can be
so effective, adding: “Some therapies, such as IMiDs (e.g., REVLIMID ®), we know are multifunctional.
This means they have effects upon multiple pathways, and that may be a major aspect of the benefit
of these agents.” Dr. Durie first published a paper about this as far back as 1985. His conclusion?
“ … Although the idea of personalized medicine is instantly appealing, it may be that broader
strategies to include a majority of patients will prove to be both simpler and more effective.”11
Dr. Durie is now leading an international effort through the IMF to evaluate the efficacy of specific
regimens on a molecular level for patients with varying genetic subtypes.
www.BSRI.myeloma.org
7
Celgene Chapter 1
The Value of
Medical Innovation
Celgene’s unwavering focus on medical innovation underlies our position in
a healthcare ecosystem that has delivered longer, healthier lives to patients.
Innovative medicines help reduce hospitalizations and allow patients to return
to work and families,12 increasing their productivity and financial con­tributions to
society.13 The convenience of our oncology and immune-inflammatory therapies
reduce the time for treatment, improving patients’ quality of life.
New Medicines Are Now the Largest Contributor to Improvements In Life Expectancy
40%
73%
1986–2000
New therapies accounted for
40% of the increase in life
expectancy
2000–2009
New therapies accounted for
73% of the increase in life
expectancy14
LONGER LIFE THROUGH BETTER MEDICINES
Advances in Treatment Have Yielded Gains in Life Expectancy(19)
Advances in Treatment Have Yielded Gains in Life Expectancy19
Deaths per
100,000 people
>96%
Decrease
500 475
450
400
350
300
250
200
150
100 48
50
0
1900
First Large-Scale Adoption
of Water Chlorination
1900
78
1920
Pertussis
Vaccine
1920
Diphtheria and
Sulfa Drugs
TB Vaccines
(first antibiotic)
1940
1960
Penicillin First Used
as a Treatment
Meningococcal
Disease Vaccine
1940
Yellow Fever
Vaccine
1980
1960
Polio
Vaccine
1980
Measles
Vaccine
8
80
75
70
65
60
55
20 50
45
40
2000
Life expectancy
>62%
Increase
Hepatitis A
Vaccine
2000
Influenza Azidothymidine
Vaccine (first HIV treatment)
2010
Living Longer, Better and Healthier Lives
Medical innovation has changed the course of human health. Over the period of a century, innovation has steadily
increased life expectancy, most notably as the result of biopharmaceutical advancements.15 Antibiotics, vaccines and
therapies targeting the immune system have had a profound impact on longevity.16
Progress Against Disease Extending Lives
CANCER: Celgene is a leader in developing and providing
access to the biopharmaceutical breakthroughs that in part
have contributed to a 22-percent drop in American cancer
22%
deaths over the last two decades.17 Since 1990, cancer
Drop in
American Cancer
Deaths
patients have enjoyed more than 50 million additional lifeyears attributed largely to next-generation life-enhancing
cancer therapeutics.18
MILESTONES IN THE PROGRESS AGAINST CANCER
20,21,22,23
Milestones
in Cancer
the U.S.
MilestonesininWar
the against
ProgressCancer
against
H
N
KEY
Advances in drug screening
Events with national Impact
Advances in cancer therapeutics
Model
development
1930
Nitrogen mustard
in lymphomas
Cure of ALL &
Hodgkin’s disease
H N
F
O
First monoclonal
Vinca alkaloids
antibody approved
Cure of
Antitumor
Genome
testicular
antibiotics
sequenced
cancer
5-Fluorouracil
Antifolates
1940
O
1960
Methotrexate in
choriocarcinoma
1980
2000
NCI investment in
molecular biology
National Cancer Act
Personalized medicine
9
Immunomodulators,
epigenetics, nanotechnology
2010
Molecular Tyrosine
kinase inhibitors
profiling
Adjuvant chemotherapy
Cancer linked to oncogenes
Target-specific screens
2050
200 years of major
advances in the history
of cancer treatment
Celgene Chapter 1
Slowing and Preventing Disease
Progression
At Celgene, we believe we can change the course of
IMMUNE-INFLAMMATORY DISEASES: Our expanding
human health by focusing on the cause as opposed to
focus on inflammation and immunology, built upon the
the symptoms of disease. Therapies such as our immuno-
ongoing research of scientists at Celgene which now therapeutics franchise go way beyond addressing the
features an emerging pipeline of disease-altering immuno­
symptoms of disease. Using a combination of actions,
modulatory compounds, kinase inhibitors, RNA antisense
these therapies both attack cancer cells and bolster the
and cellular therapies24
body’s innate defense system to fight diseases such as
MULTIPLE MYELOMA: The American Cancer Society
cancer and immune-mediated diseases. Our hematology
estimates about 27,000 new cases of multiple myeloma
portfolio includes powerful epigenetic therapies that reac­
will be diagnosed in 2015.25 Myeloma is a cancer of plasma
tivate genes that suppress tumors and regulate cell growth.
cells in the bone marrow. It affects the immune system and
This mechanism of action has allowed us to make great
can cause painful and debilitating bone damage. Today it
strides in diseases such as myelodysplastic syndromes
can be treated with targeted therapies that aim at specific
and T-cell lymphomas. Our pursuits in solid tumors are
features of the cancer cells to provide more tolerable
a natural extension of our learnings and the success we
treatments. Celgene’s oral immuno-therapeutics have
have achieved in hematology.
changed the treatment paradigm in multiple myeloma by
enabling healthcare providers to treat their patients in the
convenience of their home by taking a pill with a glass of
water—minimally disruptive to work or family life. Moreover,
Changing the course of human
health through bold pursuits in science
is at the core of our purpose and our
people are bound to each other by a
palpable desire to change patient lives
on an individual level. They share a set
of traits that set them apart, traits that
embody passion, spirit, commitment,
curiosity and the ambition to create true
and lasting value for patients, healthcare
and the economy. It is a promise to support, nurture and create medical innovation, to think differently, to transform,
to be bold, to be agile and to
persevere.”
between 2000 and 2009 there has been a 73 percent
increase in the number of myeloma survivors from 47,000
to more than 80,000 survivors alive today.26
Relative Survival Rate for Multiple Myeloma
Patients Soars with Innovative Therapies27,28
5-Year Relative Survival Rates (%) based on year of diagnosis
Projected 5-year OS 2014*
66
66%
62
58
54
50
44.9
46
42
38
34
30
27.5
25.8
24.6
26
27.2
27.2
32.3
31.6
29.2
35.5
10
14
20
9
2
-2
00
20
03
8
-2
00
-1
99
19
99
5
-1
99
Year of diagnosis
19
96
2
9
-1
99
19
93
19
90
6
-1
98
-1
98
19
87
3
-1
98
19
84
7
-1
98
19
81
19
78
-1
97
19
75
0
22
Mark Alles, President and Chief Operating Officer,
Celgene Corporation
Living Longer, Better and Healthier Lives
Celgene’s Oral
Immuno-Therapeutics
Contribute to the Value
of Medical Innovation
I was deemed a ‘high risk’ patient when I was first diagnosed with
multiple myeloma, a bone marrow cancer, on St. Patrick’s Day in March of
2009. Fortunately, I responded very well to a new oral cancer treatment regimen
that I took at home. I have been on a maintenance dose of this treatment for four
years with no perceptible signs of the cancer. So what does my story say about
the value of innovative new therapies? I feel good, I’m able to work teaching
college level business economics, and I’m not alone.”
Bob Tufts, patient, professor and former Major League Baseball player
I was given two years to live unless I had a bone marrow (stem cell)
transplant when I was diagnosed with multiple myeloma back in 2001. They said a
transplant might give me five years, but during that time I’d need a year to recover
and recuperate. I said that’s only four years and that didn’t make sense to me.
Instead, I agreed to take part in a
clinical trial of a completely new
approach to my disease—an oral
pill that would allow me to keep
working and being with my family.
That turned out to be REVLIMID®.
Fourteen years later I’m still taking REVLIMID once a day, every
day. I still run a business and I
employ 12 people. To me that
makes economic sense.”
Terry Barter, entrepreneur and family man (See Terry’s story in Chapter 2)
11
REVLIMID Prescribing Information
Celgene Chapter 1
Myeloma: Then and Now
Then: In 1999, the journal American Family Physician reported, “Chemotherapy with melphalan-prednisone is the
standard treatment for multiple myeloma. Other treatment modalities include polychemotherapy and bone marrow
transplantation. Only 50 to 60 percent of patients respond to therapy. The aggregate median survival for all stages of
multiple myeloma is three years.”29
Today: The introduction of novel cancer therapies was advanced through unique mechanisms of action beginning
with the first use of THALOMID® (thalidomide) in 1999, followed by Velcade®, REVLIMID®, Kyprolis® and most recently
POMALYST®/IMNOVID®. The five-year survival rate is projected to increase to 66 percent.30
Even higher risk patients—elderly patients not eligible for transplant—now have a 70 percent three-year relative survival
rate when treated with continuous dosing of REVLIMID, a new approach to therapy.31
These are median figures; some patients don’t respond, while many patients survive even longer. In fact, one of the first
patients to ever take REVLIMID for myeloma still takes it every day nearly 14 years later.
1844
1947
1958
1983
First
documented
case
Urethane,
cola-syrup placebo
worked better
Melphalan
Autologous
transplantation
2006
2013
REVLIMID®
POMALYST®
32
1840
1940
1844 - 1845
Early 1950’s
1962
2002
2012
Rhubarb and
orange peel,
Steel and
quinine
Radiation was the only
treatment for MM
mainly for bone pain
associated with MM
Corticosteroids
VELCADE®
KYPROLIS®
2000
46,865 Myeloma
survivors
REVLIMID Prescribing Information
POMALYST Prescribing Information
THALOMID Prescribing Information
2006
THALOMID®
2009
81,089 Myeloma
survivors
History has proven that medical innovation
is transforming rare diseases.
Approximately one-third of all medicines approved in the past five years have been designated
as “orphan drugs,” a term used to designate medicines that treat rare diseases.
EURORDIS (The European Organization for Rare Diseases) a non-profit alliance of organizations and individuals in the
field of rare diseases, awarded Celgene the prestigious EURORDIS Company Award for excellence in the field of rare
diseases. The award, presented just in advance of Rare Diseases Day, February 28, 2013, recognized Celgene’s
established track record in the area of orphan diseases. With 17 orphan drug designations and four approved orphan
drug indications granted by the EMA, Celgene holds a leading position among companies developing therapies for rare
diseases. As part of these efforts, there is a clear commitment from the company to continue extensive research in the
area of rare diseases for patients with unmet medical needs, which is the foundation on which the company was built.
12
Living Longer, Better and Healthier Lives
Improving the Quality of Life
Psoriasis is an auto-immune disease that manifests as raised scaly lesions on the skin. It affects about 125 million
people worldwide and has a significant impact on the lives of those who are affected.33 One survey found that 75 percent
of patients believe the skin condition has a moderate-to-large negative impact on their quality of life, leading to changes
in their work and other activities. Other studies have found that approximately one-quarter of people with psoriasis suffer
from depression.
The MAPP survey, Multinational Assessment of
Psoriasis, and Psoriatic Arthritis (PsA) of 3,426
Putting OTEZLA to Work
patients from seven countries34
About half of patients found their current treatments,
whether traditional or biologic, burdensome.
85%
of those surveyed
reported a need for
better therapies than
PSORIASIS & PsA THERAPIES
TRADITIONAL ORAL
MEDICATIONS
BIOLOGIC
MEDICATIONS
the traditional or biologic
treatments.
47%
of patients had
not seen a healthcare
provider in the last 12
months for their psoriasis.
46% of patients said
Plaque psoriasis
ABOUT
OTEZLA is an oral immunomodulatory compound that
FOUND CURRENT
fine tunes the body’s immune response by modulating
THERAPIES
a network of pro- and anti-inflammatory mediators.
BURDENSOME
Specifically, OTEZLA is a targeted inhibitor of an enzyme
called phosphodiesterase 4, or PDE4, to regulate inflam-
that the treatment was worse than the disease itself.
mation.36 This novel mechanism of action may provide
To learn more, please see MAPP study
http://arthritisbroadcastnetwork.org/2014/04/results-psoriaticarthritis-psoriasis-survey/
advantages over existing agents.
Approximately one out of three patients with plaque
psoriasis taking OTEZLA saw 75 percent clearer skin
OTEZLA® (apremilast), Celgene’s oral, selective inhibitor of
at 16 weeks.37
phosphodiesterase 4 (PDE4), is approved by the U.S. Food
and Drug Administration (FDA) for the treatment of patients
with moderate to severe plaque psoriasis who are candi­
dates for phototherapy or systemic therapy and for the
treatment of adults with active psoriatic arthritis.35 It does
not take time from work, school or social life for treatment
at the doctor’s office, and does not require preparation or
follow-up tests.
OTEZLA Prescribing Information
13
Access
Celgene Chapter 1
Helping U.S. patients gain access to the treatments they
need is just one way Celgene’s people are committed
to improving the lives of patients.
At Celgene, we believe that patients have the opportunity
to take advantage of significant advances in medicine
that may help them live longer, better and healthier lives.
We have a health insurance
failure for innovative medicines. If you
get cancer today, your copays can prevent you from accessing the medicines
you desperately need. That’s a failure
of health insurance.”
Accordingly, we work to help ensure access to the clinical
benefits of our innovative therapies.
Celgene Patient Support® is a U.S. service created in 2006
and provides patients a dedicated, central point of contact,
to assist with access to Celgene medications. Since 2007,
Celgene Patient Support has helped more than 46,000
patients, exhausting options to help patients get the information and support they need.38
Tomas Philipson, Ph.D., Professor, University of Chicago
Barriers to Access
Patients who face cost-sharing challenges often fail to take
$105 billion in avoidable healthcare costs in 2012, which
medicines correctly, skipping doses or taking less to save
is about four percent of the nation’s healthcare spending.
money. When medicine is not taken as prescribed, patients
While a variety of factors contribute to this figure, high
often fail to receive the full benefit of the treatment. Poor
cost-sharing is one barrier to responsible use of effective
medication adherence not only threatens health outcomes,
therapies.41
but it is estimated to cost the health care system between
Specialty Tiers: One of the ways some health insur­
$100 billion and $300 billion annually.39
ance plans impose high copays is the use of “specialty
For further information, read a recent article published
tiers.” These categories reimburse less and require patients
in the Journal of Risk Management and Healthcare
to shoulder more of the cost of these life-changing medi­
Policy. To access, visit http://www.ncbi.nlm.nih.gov/pmc/
cations on their own. Placing cancer medications on these
articles/PMC3934668/
tiers may require patients to pay up to 50 percent of the
cost of their cancer therapies.42
In addition to working with individual patients, Celgene
works on a policy level to help remove obstacles to access
created by twists and turns in insurance coverage. Those
Insurers have historically used tiered
obstacles include:
formularies to encourage enrollees to select
High Copays: While 98 percent of prescriptions are
generic or preferred brand-name drugs instead
filled for less than $70 in copays (the amount not covered
of higher-cost alternatives. But if plans place
by insurance) the remaining two percent of prescriptions
all HIV drugs in the highest cost-sharing tier,
account for 30 percent of all costs paid by patients.40 This
high cost-sharing is associated mostly with new treatment
enrollees with HIV will incur high costs regard-
options for diseases such as cancer, multiple sclerosis
less of which drugs they take. This effect sug-
and HIV.
gests that the goal of this approach—which we
According to a report commissioned by the Leukemia &
call “adverse tiering”—is not to influence enroll-
Lymphoma Society (LLS), patients who struggle to pay
ees’ drug utilization but rather to deter certain
high copays or coinsurance fees may end up forgoing
people from enrolling in the first place.
treatment. This medication non-adherence resulted in
New England Journal of Medicine, January 29, 2015 43
14
Living Longer, Better and Healthier Lives
Fail First: Step therapy, also called Fail First policies,
are practices that generally require the least expensive
Affordable access to life-saving
therapies and continued support through
pro-patient and pro-innovation policies
are essential to improving patient lives,
healthcare and the economy.”
drug be prescribed to a patient first, even if that patient’s
physician believes a different therapy would medically work
best. According to the U.S. Pain Foundation, “This prac­
tice has the potential to result in serious negative conse­
quences for consumers and the public health system. By
limiting the array of medication options, both physicians
Peter J. Pitts, President and Co-founder, Center for Medicine in
the Public Interest
and consumers are forced to compromise their treatment
decisions in a way that is dangerous, time consuming
Oral Cancer Drug Parity: Oral medications
and more expensive in the long-term.” 47 A Pfizer study of
offer medical and monetary value to patients that range
patients required to use “Fail First” for hypertension treat­
from efficacy to ease of use. That means oral therapies
ment found that it led to increased hospital and emergency
can help lower the total cost of treatment. Taking a pill
room visits and, in the end, the Fail First patients cost more
at home is convenient and less expensive to administer.
money to the insurers and to the healthcare system.48
44
Patients and caregivers do not have to travel to the doc­
tor’s office every week and take time away from work or
family while waiting for an injection or IV infusion.
Fail First and step therapy
policies often require patients—human
beings—to suffer: with pain, with side
effects and with the fear that they will
never get the treatment they need.
These policies allow insurance companies to prey on and profit most from
those who suffer with ailments that
make it difficult for them to fight back.”
On April 14, 2009, The New York Times reported that
insurance coverage for cancer medicines has not
caught up with the advantages of oral treatments.45 Six
years later, that is all too often still very much the case.
According to the Patients Equal Access Coalition (PEAC),
this can mean, “Patients who rely on self-injectables or
pills find themselves spending as much as $50,000 a year
out of pocket because they receive the drugs from a phar­
macy rather than in a doctor’s office.” 46
Matthew Titone, New York State Assembly Member,
Huffington Post, June 4, 2012
15
Celgene Chapter 1
Removing Barriers to Treatment
Doctors, not insurance companies, should determine what is best for each patient.
State Legislation Leads the Way for Patient Access
Washington
Montana
North Dakota
Oregon
Idaho
Vermont
Minnesota
Wisconsin
South Dakota
Michigan
Wyoming
Nebraska
Nevada
Utah
California
Arizona
Pennsylvania
Iowa
Illinois Indiana
Colorado
New Mexico
Kansas
Oklahoma
Missouri
Ohio
Texas
West
Virginia
Kentucky
New Jersey
Delaware
Maryland
Washington, D.C.
South
Carolina
Arkansas
Louisiana
Virginia
New Hampshire
Massachusetts
Rhode Island
Connecticut
North Carolina
Tennessee
Alabama
Hawaii
New York
Maine
Oral Cancer Drug Parity
Georgia
Oral Cancer Drug Parity &
Specialty Tiers/Cap the Copay
Mississippi
Nothing Enacted
Florida
Alaska
TAKE ACTION NOW—Contact Your Elected Official to Support Patient Access to Medical Innovation
merican College of Rheumatology—Legislative Action Center:
A
http://www.rheumatology.org/actioncenter/
merican Society of Hematology—Oral Cancer Drug Parity:
A
http://www.hematology.org/Advocacy/Campaigns/667.aspx
Arthritis Foundation—Specialty Tiers/Cap the Copay:
http://www.arthritis.org/advocate/our-policy-priorities/pass-the-patients-access-to-treatments-act.php
International Myeloma Foundation—Oral Anticancer Treatment Access Legislation:
http://cqrcengage.com/myeloma/access
Leukemia & Lymphoma Society—Legislative Action Center:
https://salsa4.salsalabs.com/o/50878//p/dia/action3/common/public/
National Organization for Rare Diseases—Policy Issues:
https://rarediseases.org/advocate/policy-priorities/issues/
Patient Services, Inc.—Legislation: https://www.patientservicesinc.org/Advocacy/Legislation
16
Living Longer, Better and Healthier Lives
States Step In: Over the past several years, policy­
• The average plan member is expected to see very little
change in their annual healthcare spending upon imple­
mentation of any of the proposed benefit design changes.
makers in 39 states and Washington, D.C. have voted yes
for pro-patient policies that would improve patient access
to life-changing medical innovations.49 These states now
address the problem of inequitable coverage by requiring
health plans to equalize the patients’ out-of-pocket costs
between oral and IV therapies.
or the silver, gold and platinum plans, premiums are
•F
not expected to increase beyond 0.5%, or can be limited
to a 0.5% increase through minimal increases in physi­
cian visit copays. All of these plans remain compliant
under Affordable Care Act (ACA) actuarial value (AV)
requirements.
Capping the Copays: Similar pro-patient legislation
is pending at the federal level and is now being enacted in
six states—with more to come—enforcing health plans to
cap the devastating high copayments imposed on patients,
which makes it impossible for them to afford their medi­
cines. High cost-sharing and specialty tiers are key mech­
anisms used by health insurers that require patients to
pay more for their health care out of their pockets. These
practices discriminate against patients with serious dis­
eases and undermine the concept of health insurance.50
•F
or the bronze plans, added changes in benefit design
are required to keep the plan premium within 0.5% of the
original premium, including increases to the deductible
and out-of-pocket maximum. One bronze plan required
minimal changes to remain compliant under ACA AV
requirements.49
Oral Cancer Drug Parity: Thirty-nine states
have enacted laws requiring insurance companies to
reimburse all cancer medications at the same rate regard­
less of whether they are oral, injections or IV, with active
campaigns in eight more states.50
The Affordable Care Act limits what most individuals must
pay out of pocket for prescription medications to $6,600
(in 2015). The family limit is $13,200.51 However, that may
be too high for many patients, especially those who may
have to pay their entire deductible on their first visit to
the pharmacy.52
Will that raise insurance costs for everyone? Milliman
calculates equal coverage for oral and IV medications
would cost “well below” one dollar per person per month
on most policies.
According to a March 2015 analysis conducted by financial
consulting firm Milliman, potential policy changes can limit
a member’s out-of-pocket expenditures for prescription
therapies. The study confirms that such changes can
result in dramatic cost savings for the individual policy
holder. The benefit design changes result in decreased
total cost sharing (including both medical services and
prescription medicines) for members.
Fail First: No state bans step therapy/fail first protocols.
Connecticut, Kentucky, Louisiana, Maryland, Mississippi
and Washington have enacted laws to limit step therapy/
fail first policies by health insurance companies. Similar
legislation is likely to be considered in California, Maine,
Florida, Missouri, Illinois and Massachusetts in 2015.
Some states have laws in place with a narrow application
(e.g., Texas requires plans to notify the insured before
enacting step therapy protocol, Utah deals only with pain
medications and Arkansas deals only with mental health).
• Members with high spending on specialty drugs are
expected to see a significant reduction in annual health­
care spending upon implementation of any of the benefit
design changes.
Caethe Goetz
moved the packed Sacramento hearing room to silence when she began
to speak. A former Marine from the Vietnam era, Caethe was diagnosed with multiple
myeloma and her insurance did not fully cover the new oral medication her doctor pre­
scribed. Instead of taking a pill, she had to go to the doctor’s office twice a week for her
infusions, and that meant she could not work full time. This proud ex-Marine maxed-out her
credit cards and had to move back in with her elderly mother. So Caethe went to the state
legislature in California because it was time for a change.
Caethe Goetz lost her battle with myeloma in November 2012, before she could see she
had won her legislative battle. California passed oral drug parity in 2013. To read more
about her story and the connection between exposures to herbicides and her myeloma
read: http://www.cpeo.org/lists/military/2011/msg00329.html
17
Celgene Chapter 2
Better H ealthcare, Better Outcomes
18
Living Longer, Better and Healthier Lives
Celgene Therapies, Transforming
TREATMENT FOR PATIENTS
At Celgene, we understand what matters most to patients
Our patients are special to us and we share their triumphs,
with life-threatening illnesses: a longer, healthier life. We
and their challenges, with them.
are committed to discovering, developing and delivering
To understand the total impact of cancer, it’s important to
life-enhancing therapies that turn debilitating and deadly
meet the people who live courageously with the disease
diseases into long-term manageable ones enabling
every day. Here are their stories.
patients to live longer, better and more productive lives.
For patients, the hope to actively participate in life is
Five Year Survival Has Increased 39%
Across Cancers1
precious and our purpose at Celgene honors that goal.
A longer, healthier life is also a benefit to everyone in a
100%
90%
80%
70%
60%
5-year survival 50%
40%
rate for
30%
selected
20%
cancers,
10%
1975-2006
0%
/B
ng
Lu
/R
1975
ro
ec
nc
tu
hu
s
m
r
ta
os
most effective, and which ones can be further developed
Pr
Br
ea
st
te
C
C
an
researchers learn which therapies and pathways are
an
ce
ce
r
a whole. Patients living longer, healthier lives may help
on
across the healthcare ecosystem to society as
83%
of survival gains
in cancer are
attributable to
new treatments
ol
network of people whose lives they touch. It extends
C
patient’s life; family, friends, colleagues and the broader
2006
for future therapeutic improvements. Hospitalizations and
extraordinary measures may become less frequent, freeing up medical professionals to serve more people in
need. And, of equal societal value, patients living longer,
According to Project Innovation, “From the first chemo­
healthier lives brings economic benefits.
Celgene Color Palette
PMS 557
PMS 7466
therapy in 1949 to the newest targeted treatments,
PMS 7441
PMS 646
PMS 143
PMS CG10
disease-altering medicines have transformed cancer
At Celgene, our efforts are making that goal a reality for
care and led to more people surviving cancer than ever
the tens of thousands of patients who rely on our thera­
before. Scientific discoveries have netted new and
pies. In the late 1990s, Celgene took a bold move to pur­
better ways to prevent, detect and diagnose and treat
sue immunotherapies to treat cancer. What was learned
more than 200 types of cancer. Overall cancer deaths
from this initiative gave rise to the development of new
have dropped 20 percent since 1991 while five-year
classes of innovative cancer medicines principally for
survival rates now average 68.5 percent, up from 48.7
blood cancers. The ability to construct chemotherapy into
percent in 1975. As a result, the population of cancer
nano-particles of protein is changing the course of
survivors has grown from three million in 1971 to almost
how we treat many solid tumors.
14 million today.”2
Part of our culture at Celgene is to stay close to our
patients, to remember the people behind the therapies.
19
Celgene Chapter 2
And Benjamin
Makes Eleven!
Terry Barter measures his life in grandchildren. Four­
Patient: Terry Barter
Condition: Multiple Myeloma
at the start of 2015, all because of what some might
teen years ago, when he was diagnosed with the blood
cancer multiple myeloma, he had three grandchildren.
Given the outlook for patients at the time, he didn’t know
if he’d live to see any more. But today Terry has lived to
see eight more grandchildren, including Benjamin, born
consider to be a daring decision.
His doctor, Kenneth Anderson, M.D. of the Dana-Farber
Cancer Institute in Boston recalls, “Terry was our hero.
He’s the very first person to receive this oral medica­
tion, REVLIMID ®.”
REVLIMID Prescribing Information
20
Living Longer, Better and Healthier Lives
medication that would not disrupt his life. He did not know
how long that life would be—after all, this would be a
very new approach to treating myeloma. But he decided
to give it a try.
Fourteen years after he was diagnosed with multiple
myeloma, Terry Barter is alive and well, working and
spending time with his grandchildren, so he knows full
well what value is when it comes to treating disease.
However, there is one unintended consequence—one
that Terry gladly accepts.
“If cancer was going to cut my life short, I wanted a medi­
cine that would improve my quality of life and give me the
When he was first diagnosed, Terry and Diane accepted
most out of my remaining time to spend with my wife
the prognosis that Terry had just a little time left, so they
Diane,” said Terry.
decided to live it up. They planned to spend their money,
because there was no need for long-term savings. They
“When I was diagnosed they wanted me to have a bone
took an extended trip to Italy, and when they came back,
marrow transplant. They said if I had it, I might live for
Terry bought himself a Corvette.
another five years, but with the transplant I’d be out of
work for about a year. I said that’s only four years of
REVLIMID changed their lives and their plans.
really living. I said no.”
“And then we thought, oh no, he’s going to be OK,” says
So he opted to enter a clinical trial to try REVLIMID even
Diane. “So we had to settle down, pay our bills and Terry
though it was still brand new. Terry knew it was an oral
had to sell the Corvette.”
®
Today, Terry is focused on the impact his treatment has
REVLIMID, the brand name for lenalidomide, represented
on others, including the network of people who extend
a next-generation approach to treating blood cancers.
beyond himself and his family.
REVLIMID belongs to Celgene’s class of cancer drugs
called IMiDs ® compounds, which are small molecule,
“I own a business and I employ 12 people. I had the busi­
oral therapies that act on the microenvironment of the
ness long before I had multiple myeloma. But if it weren’t
tumor cell through multiple mechanisms of action. These
for this new approach to treatment, I don’t know where
mechanisms of action have not been fully characterized,
those jobs or those people would be.”
but it is thought that REVLIMID works through dual effects
And for Diane? “We’re looking ahead, planning for the
that lead to multiple myeloma cell death and inhibit tumor
future, which we were afraid to do before. We have this
regrowth. Specifically, REVLMID is thought to have the
new baby in our lives, another son who is about to be
following actions:
married. It’s really fun.”
• Blocks the ability of tumor cells to replicate
Happily, Terry has lived long enough and well enough to
• Directly kills tumor cells by pushing them into apoptosis—
pay for his children’s college and watch them start their
a cellular suicide pathway
own families and careers.
• Blocks cytokines (small cellular chemical messengers)
He’s paid for his children’s college and watched them
in immune cells that induce potentially damaging
start their own families and careers. And yes, he’s bought
inflammation
himself a new Corvette. This one he’ll keep.
• Activates and stimulates parts of the immune system
REVLIMID Prescribing Information
known as T cells and natural killer (NK) cells, which
attack tumor cells and battle the cancer
21
Celgene Chapter 2
A Portrait of
Confidence
Patient: Susan Freeman
Condition: Psoriasis
Susan Freeman takes a pill with a glass of water, rolls up
Susan had a similar experience with some of the newer
her sleeves and heads outdoors to get to work on one of
medical options that preceded OTEZLA.
her favorite pastimes, photography. Taking pictures isn’t
“The previous drug was an injectable. I could only have
new for Susan. But rolling up her sleeves to go outside
it administered at the doctor’s office. They had to order it
is just a small example of a huge change in her life—a
from the manufacturer. It had to be kept in a refrigerator,
change due to the pill she takes called OTEZLA®.
so there were all these other ramifications in terms of
taking it.”
Susan has psoriasis, a skin disorder that produces red,
scaly patches. At first for Susan it was just on her arms
Then she entered a clinical trial for a new investigational
and legs; then it spread. Susan says, from the time she
treatment, at the time known just by its scientific name,
was a teenager she stayed covered up to hide her psoria­
apremilast. At first, she was on a placebo and saw no
sis. It affected her activities, perhaps her relationships.
improvement. Then when she was switched to apremilast
her skin began to clear.
“My skin ruled my life, in terms of my clothing, not going
to the beach and not wearing short sleeves.”
“It’s like fabulous. I could be walking around in short
About 10 years ago, the unsightly red patches reached
sleeves. I could be wearing a skirt. I wouldn’t feel that
her face, and covering up became more difficult. UV light
I needed to cover up. I am very excited. I’m thrilled.”
rays from a special box in her doctor’s office worked, but
Now, camera in hand, she can wander through the city’s
it was a hassle.
gardens. She can stop and smell the tulips without being
embarrassed.
“It was effective, but I had to go over to the hospital three
days a week before I went to work. I just got to a point
where I couldn’t stand doing it anymore.”
OTEZLA Prescribing Information
22
Living Longer, Better and Healthier Lives
People with Psoriasis Often Shunned by Others*4
OTEZLA® is an immunomodulatory compound. It fine
tunes the body’s immune response by modulating a net­
49%
45%
42% w
ould not want to kiss or hug a person
work of pro- and anti-inflammatory mediators. Specifically,
with psoriasis
OTEZLA is a targeted inhibitor of an enzyme called PDE4.
would not want to share a swimming pool
OTEZLA Prescribing Information
with someone with psoriasis
would not eat food prepared by someone who had psoriasis
*Study surveyed 5,029 people from France, Germany, Spain and the UK
23
Celgene Chapter 2
A Pleasant Surprise
Patient: Fiona Pirilla
Condition: Myelodysplastic Syndromes (MDS)
Fiona Pirilla, who lives in the United Kingdom, was diag­
Prior to my diagnosis, I had been very active, particularly
nosed with MDS in 1999 when she was just 33 years old.
playing basketball. I had played basketball for England,
This is her story in her own words:
and actually came to the USA between 1984–1988 on a
basketball scholarship at the U.S. International University
“I was first diagnosed after a particularly bad chest infec­
in San Diego, where I obtained my BA degree in sociology.
tion that wouldn’t seem to go away. After many visits to
When I returned to the UK I continued to play for a National
my doctor and with consultants at the hospital, I was
League club, and also England, before I eventually had to
eventually diagnosed with MDS deletion 5q (a chromo­
retire from the sport. Running up and down a basketball
somal abnormality). At that time there were no real phar­
court became very difficult because I couldn’t breathe prop­
maceutical treatments available and it was decided that
erly, was very fatigued and often susceptible to infections.
it was too risky for me to consider a bone marrow trans­
plant as I didn’t have any related donors. That is a story
By 2006, my hemoglobin had dropped to the level where
in itself that we’ll get to later. In the meantime my health
I needed to have blood transfusions. Fortunately at
deteriorated to the point where my hemoglobin levels
around the same time, the opportunity for me to partici­
dropped continuously making me very anemic.
pate in a clinical trial for REVLIMID ® came about via
REVLIMID Summary of Product Characteristics
Kings College Hospital in London. I was able to start the
trial in September 2006 and continued until December
2007. I had a fantastic response to the drug, with my
hemoglobin levels shooting right back up again, no short­
ness of breath, no feeling constantly tired all the time.
MDS are a group of diverse bone marrow disorders
Unfortunately after the clinical trial finished, I was unable
often referred to as a “bone marrow failure disorder”
to have access to the drug, and had to wait until 2010
and classified as a “blood cancer.” MDS affects the pro­
before I was finally given access to it again, as my condi­
duction and function of blood cells—red blood cells,
tion had again deteriorated. I’ve been back on REVLIMID
white blood cells or platelets. Patients often require
since January 2010, and again it has had a really good
blood transfusions that can lead to iron overload, and
effect on my hemoglobin.
the condition can progress to AML (acute myeloid leu­
FEELING FIT AND HEALTHY AGAIN
kemia), a serious form of leukemia that has a median
REVLIMID has had a dramatic effect on my quality of life.
survival of less than one year. The incidence of MDS
I work as a health and safety trainer in the UK which
and AML is underestimated, conservatively believed to
requires me to stand up and give presentations all day
affect tens of thousands annually.5
and also drive all around the country, meaning very long
working days sometimes. The difference now is amazing.
I feel “normal” with my blood counts being very healthy.
24
Living Longer, Better and Healthier Lives
Although I was too old really to start playing competitive
1999/2000, that wasn’t considered to be a good enough
basketball again, I was able to return to “normal” levels
match, although it could have been today. But luckily for
of exercise and have now renewed my gym membership!
me I was able to discover a whole new family!
As I write this at age 49, my future is far from certain
I continue to remain the secretary and one of the
but I do know that my quality of life has been drastically
directors of the MDS UK Patient Support Group—
improved.
http://www.mdspatientsupport.org.uk/—and I’m really proud
of the work we have done over the years in raising aware­
A PLEASANT SURPRISE
ness of MDS in the UK. I hope the awareness of the
When doctors wanted to discover if I had any potential
massive need for bone marrow donors continues to grow.”
bone marrow donors, it raised an unexpected situation
for me. I had been adopted from birth and had therefore
POST SCRIPT
had no previous family medical history. To cut a very long
In July 2013 REVLIMID ® was approved in Europe by the
story short, I was then able to contact my birth mother
EMA for use in certain MDS patients who have the dele­
(who actually only lived about 17 miles away from me).
tion 5q chromosomal abnormality. In August 2014 reim­
I do have a half-brother and half-sister who had the same
bursement for this indication was approved in the UK.6
biological mother as me but not the same father. Back in
REVLIMID Summary of Product Characteristics
25
Celgene Chapter 2
“OTEZLA Gave
Me Back My Life.”
®
Those are the words spoken by Theresa Dishner when
she finally found relief from her crippling psoriatic
arthritis. It had been a very long way from just a year
earlier when she said, “I have no more hope.”
Theresa’s long and painful journey actually begins in
the 1970s when she was diagnosed with psoriasis.
Patient: Theresa Dishner
Condition: Psoriatic Arthritis
The condition made her hands so rough from chapping
that they would bleed. Thirty years later her condition
got worse—a lot worse. Theresa began to experience
OTEZLA Prescribing Information
pain and swelling, first in her fingers then throughout
her body. She was a high school teacher and had to sit
26
Living Longer, Better and Healthier Lives
on a stool in front of her classes because it was too pain­
ful to get up and down out of a chair.
At first doctors blamed the salt in her diet. She thought
the culprit might be rheumatoid arthritis. Finally a doctor
diagnosed her with a condition she had never heard of,
psoriatic arthritis, a swelling of the joints related to her
psoriasis. She went through the usual list of treatments for
joint pain and swelling: non-steroidal anti-inflammatories
(NSAIDS), the powerful drug methotrexate and the leading
biologics. The NSAIDS had serious side effects and the
methotrexate initially worked but gradually decreased in
efficacy. She had to stop biologics when she developed
further complications.
Soon afterwards, Theresa was confined to a wheelchair
and she had to retire from teaching. She could not help
with her newborn twin grandsons. She couldn’t even get
up the stairs to her bedroom. Doctors told her they had
run out of options and she remembers the anguish she
felt. “That was the lowest point of my life.”
MARCH 2014: A LIFE-CHANGING OPTION
In March 2014, the FDA approved OTEZLA® for psoriatic
from the window of a beach house while her family frol­
arthritis. Theresa’s doctor agreed this new therapeutic
icked on the sand. A year later, and only months after
would be a good alternative and started her on the ther­
starting her new treatment, she returned to the beach a
apy. Her skin improved within just two weeks after she
new person. Instead of watching from afar, she woke up,
started taking it, and her pain began to subside. Two
took her morning dose of OTEZLA and joined her family
months later, Theresa put aside her wheelchair and she
all the way to the water’s edge.
7
began to live without experiencing serious side effects.
Today she goes shopping, climbs stairs and plays with
her grandchildren. A special shout-out to the Celgene
Product Support Team that helped get Theresa her first
Psoriatic arthritis is a form of arthritis that affects approx­
starter-pack of OTEZLA quickly, even though it was
imately a third of the people who have psoriasis.8 Joint
newly approved—another example of the Celgene
pain, stiffness and swelling are the main symptoms of
culture in action.
psoriatic arthritis. It can affect any part of the body,
The prior year, while on vacation with her family but
including fingertips and spine, and can range from
confined to her wheelchair, Theresa watched sadly
relatively mild to severe.
OTEZLA Prescribing Information
27
Celgene Chapter 2
“The Boy Who
Launched a
Thousand Cures”
Patient: Quentin Murray
Condition: Acute Lymphoblastic
Leukemia
Quentin Murray, just seven years old back in 2010,
squirmed on the set of TV station Fox 8 Louisiana, as
his mother Mary Webb told the reporter about Quentin’s
battle against acute lymphoblastic leukemia (ALL). It was
a battle that Quentin won, teetering at the very edge of
medical potential and scientific imagination.
Three years earlier Mary Webb was pregnant with their
second child when her happy, active Quentin, then just
four years old, began having strange aches and pains.
He was in too much pain for summer camp; too much
pain to be ring bearer at his uncle’s wedding; he just
a potential cell therapy, to combat leukemia and related
wasn’t himself. Doctor after doctor couldn’t figure out
diseases. Their challenge was the number of stem cells
what was wrong with Quentin. Some told Mary she was
that could be harvested from cord blood was relatively
paranoid because of her pregnancy. Ironically, that preg­
small. So Celgene Cellular Therapeutics’ (CCT) Dr. Robert
nancy was what would save Quentin.
Hariri decided to go a step further. Dr. Hariri saw the
ALL is the most common childhood cancer.9 In the 1960s,
placenta as a rich source of stem cells.
the five-year survival rate was less than 10 percent.10 By
But would Quentin’s new sister Jory be a genetic match?
the time Quentin was diagnosed, the survival rate was
Again, doctors said there was only a 25 to 30 percent
improving dramatically, but in Quentin’s case his doctor
chance that cord blood and placenta cells from Jory’s
said specific genetic factors meant, with traditional che­
birth could be used for Quentin. When Jory was born, the
motherapy, his chances of survival were only 35 percent.
hospital harvested both her cord blood and blood from the
Cancer specialists had been using blood taken from the
placenta. It was a match!
umbilical cord of newborns as a source of stem cells,
28
Living Longer, Better and Healthier Lives
FIRST-OF-ITS-KIND TRANSPLANT
POST SCRIPT
The stem cells were separated from the blood, and on
Today that little boy is not so little. Quentin is 12 years old
March 28, 2008, Quentin received the first-ever com­
and celebrating the seventh anniversary of his transplant.
bined cord blood/placenta blood transplant.
He loves basketball and plays trombone in his school’s
marching band. Recently he marched in the Mardi Gras
“It was done at birth with no risk to myself and no risk to
Parade in New Orleans, where he lives.
the child,” says Mary Webb. “It’s the afterbirth—tissue
that would typically be discarded. But we would have
Mary Webb wrote a book about her son’s battle with leu­
been throwing away my son’s chance at survival.”
kemia. She calls it, The Summer of Superheroes and the
Making of Iron Boy, because, she says, the title speaks
That transplant advanced the development of placenta-
volumes about how resilient Quentin was, standing at the
derived stem cells which have been further studied in
frontier of medical innovation.
several clinical trials. But Mary Webb says, “It all began
with the little boy who launched a thousand cures.”
29
Celgene Chapter 3
3
P rogress and P rosperity
Medical Innovation Reduces Costs and Saves Lives
30
Living Longer, Better and Healthier Lives
Failure
Is Not an Option
for Patients, the Healthcare System and the Economy.
Medical Innovation Reduces Healthcare
Spending While Increasing Patient Health
and Survival8
The single most important element in the success of the
global healthcare system is medical innovation.
Medical innovation leads to the discovery and devel­
opment of therapies that save and extend lives1—the
primary goal of healthcare. And by doing that, innovation
REDUCTIONS
DECREASE
REDUCTIONS
Medical
Spending
Hospital
Expenditure
Physician OfficeVisit Expenditures
helps keep healthcare costs down while driving economic
growth higher.
Innovative
Medicines
Thanks to medical innovation, patients are living longer,
Healthcare Spending
$1 SPENT
healthier, more productive lives,2,3 a benefit that extends
$6.20 SAVED
For every dollar spent on innovative medicines,
total healthcare spending is reduced by $6.20
across the entire healthcare ecosystem to society as a
whole.4 Simply put, the longer one lives, the more time
At Celgene specifically, we are expanding our innovative
they have to be productive and contribute to society.
therapies to nearly 100 countries worldwide. Our portfolio
In 1990, there were about six million cancer survivors
of approved therapies, now encompassing OTEZLA®,
in the U.S. Today there are about 14.5 million.5 Back then,
ABRAXANE®, POMALYST®/IMNOVID ®, REVLIMID ®,
60 percent of cancer patients could expect to live five
THALOMID ®, VIDAZA® (azacitidine) and ISTODAX®
years or more. Today, that number is nearly 70 percent.6
(romidepsin), provides life-changing benefits to patients.
Over the past 50 years in the U.S., that increasing life
expectancy has translated into improvements in real GDP
of over 10 percent.7
The Bureau of Economic Analysis
(BEA) has developed a Health Care Satellite
Account from multiple government and academic sources to “improve our understanding
of health care spending trends.” The first
release shows that from 2000 to 2010, health
services grew at an annual rate of 4.6 percent,
while the prescription therapy com­ponent grew
at less than one percent. Other analyses showed
slightly different numbers, but the relative growth
rates remain consistent.9
Driving future significant impact are the more than 6,800
medicines in clinical development around the world.
In fact, according to a recent report, 70 percent of treat­
ments in the pipeline have the potential to be first-inclass therapies.
ABRAXANE Prescribing Information
OTEZLA Prescribing Information
POMALYST Prescribing Information
REVLIMID Prescribing Information
THALOMID Prescribing Information
VIDAZA Prescribing Information
31
Celgene Chapter 3
Medical Innovation Reduces Costs and
Saves Lives
IMPACT ON TREATMENT
INNOVATION THAT BENEFITS
ALL OF US
For more than 50 years, the total cost of treating cancer
Thanks in part to medicines:
given the accelerated rate of ground-breaking advances in
• The U.S. HIV/AIDS death rate is down 83 percent and
cancer treatment. The cancer treatment model is an exam­
has remained approximately five percent of the total U.S.
healthcare expenditure. This fact is even more impressive
HIV/AIDS is now a chronic, manageable condition10,11
ple of proven sustainability in healthcare today.22,23,24,25
• U.S. cancer death rates are down 20 percent from their
Cost of cancer treatment is a small and
stable
portion
of total
personal
Cost of Cancer
Treatment
is a Small
Portion healthcare
26,27,28,29Expenditure
of Total Personal Healthcare
expenditures
peak and the five-year relative survival rate is up to
68 percent12,13
• Since 1975, childhood cancer five-year relative survival
7
rates are up 58 percent,14 lung cancer five-year survival
6
rates are up 69 percent, prostate cancer five-year sur­
5
vival rates are up 66 percent, colon cancer five-year sur­
4
vival rates are up 73 percent and breast cancer five-year
3
survival rates are up 83 percent
2
15,16,17,18,19
% of Total Personal Healthcare Expenditure
6
5
5.7
4.8
4.1
4.5
4.7
4.7
1995
2004
4.4
1
0
1963
1972
1980
1985
1990
2005
2010
What about the other side of the healthcare coin, the everpresent issue of cost: cost of treatment, of hospitalizations
Unfortunately, while overall spending on cancer treatment
and of productivity?
has remained relatively constant, the portion that cancer
patients being served by innovative therapies must pay
To understand the total impact of cancer, it is important
directly has increased substantially due to insurance reim­
to see the cost picture clearly. Despite concerns we may
bursement challenges including specialty tiers, inconsis­
read and complaints we may hear, the benefits of medical
tent oral parity laws and fail first practices (see Chapter 1).30
innovation clearly outweigh the spending.20
Patients should not have to choose between paying for
For example, consider blood cancers: Clinical data are
treatment and maintaining their day-to-day life. Yet many
showing us that now, more than ever, we are increasing
are forced to do so because of their insurance coverage
survival rates for patients and lessening the financial
design, and in some cases patients decide to skip treatment
burden on the healthcare system.21
because they cannot afford the copay or coinsurance.31
Celgene Patient Support® is a free service that has been helping patients for years. When patients enroll in Celgene
Patient Support®, they’ll be assigned a dedicated Specialist who will work closely with them and their doctor’s office. For
more information, please call Celgene Patient Support® at 1-800-931-8691 or visit the Celgene Patient Support® website.
32
Living Longer, Better and Healthier Lives
On average, consumers pay more than 20 percent of their
If consistent use of prescription drugs decreases overall
prescription drug spending out-of-pocket, compared to
healthcare spending, and oncology drug coverage constitutes
four percent for in-patient and seven percent of out-patient
less than one percent of that spend,34 wouldn’t it make more
hospital care.32
sense to cover a higher percentage of consumer costs for
the greater, proven benefits such therapies provide?
Finally, unique to the business of creating newer and
better therapeutics, after a limited period of time, bio­
CREATING SOCIETAL VALUE
pharmaceutical innovations are gifted to society forever
Innovative prescription therapies can save lives and
through generics that facilitate broad and low-cost access
save money.35
to the greatest medical innovations in the world. Today,
The National Patient Advocate Foundation (NPAF) said it
nearly nine out of 10 U.S. prescriptions are filled with
succinctly. “Sicker patients require more expensive care.”
generics.
The average cost of a hospital stay in the U.S. is nearly
$10,000, more than $12,000 for patients over 45 years old.
The NPAF says these costs create “an unacceptable finan­
The failure to … measure
value has slowed innovation, led to
ill-advised cost containment, and
encouraged micromanagement of
physicians’ practices, which imposes
substantial costs of its own … . If value
improves, patients, payers, providers,
and suppliers can all benefit while the
economic sustainability of the health
care system increases.”33
cial burden” on patients and on the healthcare system.36
At Celgene, we help reduce that burden.
Between 1990 and 2010, the average time spent in the
hospital by cancer patients declined by 70 percent. If
there had been no decline, hospital costs would have
been about $250 billion more over the same time period.37
According to an economic analysis from the Center for
Medicine in the Public Interest, this saving is directly
attributable to medical innovation—including oral medi­
cations that can be administered at home.
Michael E. Porter, Ph.D., Harvard Business School
Bringing Healthcare Costs into Focus 38,39,40,41,42,43
Spending on cancer, relative to total healthcare spending, has remained constant over the past half century
National
treatment
expenditures
(in billions)
Total
personal
healthcare
spending
(billions)
National cancer treatment expenditures, 1963 - 2012
$2,500
$2,000
$1,500
$1,000
$500
Cancer treatment
spending (billions)
$0
1893
1917
1947
1956-66
1971
1988
33
2014
Celgene Chapter 3
Medicare Part D
Part D has been a real success:
1) O
ver 90% of beneficiaries are satisfied with their
Medicare Part D coverage.
Part of the Solution for Better Access,
Improving Lives
he Congressional Budget Office (CBO) estimates that
2) T
total Part D costs are $348 billion less than original
estimates.
“When Part D began, it was surrounded by a good deal of
controversy because of the vigorous debates in Congress
over its passage and the very close margin of the final
votes. Today, Part D is viewed as part of the fabric of the
Medicare program. In a relatively short time, this program
has become so widely accepted that I don’t think anyone
can imagine a Medicare that didn’t offer coverage of prescription medications. I’ve had the opportunity to sit down
with so many men and women who have told me how
they used to cut pills in half to make their prescriptions
last longer, or how they simply didn’t fill what their doctor prescribed. Now, though, they have a program that
allows them to protect their health without putting them
in financial jeopardy. The joy and relief on their faces is
something I will never forget.”
—M ary Grealy, President, Healthcare Leadership Council
(HLC), September 22, 2011.
3) P
remiums, including those for 2015, have been holding
steady.
Improved Access and Adherence to
Medicines through Medicare Part D
Results in Overall Cost Savings
MEDICARE SAVINGS THROUGH PART D
Improved access and
adherence to medicines
SAVED $1,200 / year
this reduction
achieved...
In hospital, nursing home, and other
costs for each individual who
previously lacked comprehensive
prescription drug coverage
Medicare Part D continues to be a success for beneficia­
ries. The patient-focused program is just under 10 years
old, yet it covers more than 35 million people, or roughly
2
∕3 of all Medicare beneficiaries.
$13
BILLION
In overall savings during
first full
year of Part D
A 2014 study by the National Bureau of Economic Research
shows that Medicare Part D coverage created an eight
percent decrease in hospital admissions for seniors and
A report recently released by the Congressional Budget
Office (CBO) noted that Part D spending in 2013 was
nearly 50 percent less than expected when the program
began in 2006. In 2013, Part D spending was $50 billion
compared to the projected $99 billion. Not only was
spending in 2013 less than anticipated, but the CBO
also continues to reduce its 10-year baseline forecasts
for Part D spending. For 2014 alone, it has been reduced
by $56 billion. The report also noted “the competitive
structure of Part D gives plan sponsors significant incen­
tives to hold down spending.”
hospital-cost savings of roughly $1.5 billion. A recent
study in the Journal of the American Medical Association
found that the implementation of Part D created a $1,200
decrease in nondrug medical spending by patients with
prior limited drug coverage. This translated into roughly
$13.4 billion in healthcare savings during Part D’s first year.
Private market competition within the plan is working. The
Medicare Trustees estimate savings from drug rebates
of between 20-30 percent, secured by plans negotiating
directly with the biopharmaceutical industry without gov­
Following the CBO report, the Centers for Medicare and
Medicaid Services announced estimated 2015 premiums
for the Part D program would remain stable. It is esti­
mated that the average premium for Medicare Part D
plans will be $32 per month in 2015. Low average monthly
premiums make it possible for Part D to continue provid­
ing patients with affordable access to their medicines.
Beneficiary satisfaction with Medicare Part D remains
high and the program continues to be a model for suc­
cess, emphasizing the value of access to medical innova­
tion in patients’ lives.
ernment intervention.
This is value, plain and simple. And it spans patients,
taxpayers and the healthcare system itself. It may have
taken decades to solve the puzzle of fully integrating
prescription therapy into the picture of senior health, but
the results have been proven to be successful. For a company like Celgene, this is another indication that our
focus on patients is a value shared across society.
34
Living Longer, Better and Healthier Lives
More Than 50 Million Life Years Saved
Survival time for patients with cancer has been increased
consumer consumption of goods and societal prosperity,
by more than 50 million additional years of life since 1990.
the value reaches more than $4 trillion.46
44
Some patients have gained extra months, while some have
The U.S. invests substantial public and private resources
survived years even decades longer than expected. The
in maintaining and improving the health of its population.47
progress has been made possible by investing in new
Even with substantial public expenditures, the social ben-
treatments, new pharmaceuticals and research to under-
efits from the greater investment in medical knowledge
stand the mechanisms underlying cancer and how they
change from person to person.45
may far outstrip costs, so that the current investment in
The benefits to patients and their families can be counted
cent decrease in cancer mortality could be worth about
in birthdays, anniversaries, weddings and other life events
$500 billion.48
medical innovation too low. Imagine, just a modest 1 per-
that would have been missed if it weren’t for medical innovation. But in just economic terms, such as productivity,
Living Longer, Better and Healthier Benefits Society
Living Longer, Better and Healthier Benefits Society49
A
1%
REDUCTION
Improved
quality of life
Worth
Stimulated
the economy*
$500 Billion**
Maximized life
expectancy
in CANCER related
DEATHS in the U.S.
*Extended survival contributes to economic stimulus by affording people more time to purchase and enjoy leisure activities
**To current and future Americans
INNOVATION IN PERSPECTIVE50
1960, all medicines have represented approximately 10 percent of total U.S. healthcare
10% Since
spending
decades, the cost of treating cancer specifically has remained around five percent of the total
5% For
U.S. healthcare expenditure
on new targeted cancer medicines represents less than one percent of overall healthcare
1% Spending
spending
For further information, please see the National Cancer Institute’s Cancer Trends Progress. To access, visit:
http://www.progressreport.cancer.gov/sites/default/files/archive/report2005.pdf
35
Celgene Chapter 3
Immune Disorders Impact Productivity
Saving and extending lives is not the only way medical
innovation creates societal value. The limitations and dis­
abilities caused by immune disorders have a significant
Over the last half century,
improvements in health have been
as valuable as all other sources of
economic growth and productivity
combined.”
effect on productivity.51,52,53,54
Nearly half of the people with rheumatoid arthritis and
40 percent of patients with psoriatic arthritis have workrelated disabilities.55,56 A recent survey found that 74
percent of inflammatory bowel disease and rheumatoid
Kevin Murphy, Ph.D., and Robert Topel, Ph.D., University of
Chicago economists
arthritis patients have taken time off in the past year due
to their condition.57,58 See Theresa’s story in Chapter 2.
But medical innovation cannot stop there. Eighty-five
Of course, this takes an emotional toll on patients with
percent of patients with psoriasis and psoriatic arthritis
immune disorders and their families, but in just dollars
say they need a better therapy.63 Nearly half of psoriatic
and cents, these conditions cost society. According to
patients have stopped their treatment with biologics com­
recent reports, the estimated economic burden of Crohn’s
pletely. Patients who discontinue therapy cost an average
disease, psoriatic arthritis, psoriasis and rheumatoid
of $22,000 per year in lost productivity per person. To
arthritis is approximately $51 billion.59,60,61
address this need, Celgene supports patient edu­cation
Medical innovation can make a difference. According to
to encourage treatment and earlier diagnosis. Celgene
the Journal of Rheumatology, one group of patients with
has also been at the forefront of important new research.
psoriatic arthritis had a 68 percent increase in productivity
Recent findings identified important signaling
after one year of treatment.62
molecules within immune cells. One such molecule,
phosphodiesterase-4 (PDE4), is a key intracellular
enzyme involved in modulating immune cell activity in
36
Living Longer, Better and Healthier Lives
psoriasis. Modulating PDE4 is an important mechanism
of action for OTEZLA®.
The discovery of OTEZLA dates back 15 years to the
company’s own laboratories where the molecule was first
synthesized by Celgene scientists. They saw its potential
in the laboratory almost immediately and multiple teams
at Celgene worked together to guide it through critical
milestones. They selected the best indications to harness
its potential, took it through clinical trials and onto approv­
als. The development of OTEZLA illustrates Celgene’s
success as a fully-integrated, global biopharmaceutical
company that translates unique scientific discoveries into
disease-altering therapies.
In 2014 and early 2015, OTEZLA was approved in the
U.S. and Europe for psoriasis and psoriatic arthritis. It is
already having an impact. Twenty percent of patients
switched to OTEZLA from a biologic. And dermatologists
across the board are prescribing it, including those who
prescribe biologics, those who prescribe oral systemic,
and those who prescribe mostly topical treatments.
OTEZLA Prescribing Information
Surveyed Patients with Psoriasis and Psoriatic Arthritis64:
WORK DAYS MISSED
1 - 5 DAYS
6 - 10 DAYS
>10 DAYS
62%
6.6%
31%
in a typical month due to their desease
37
Celgene Chapter 4
T urning Science into R eality
Greater Investments in R&D Leads to Better Health and Longer Life
38
Living Longer, Better and Healthier Lives
Investment Yields
Important
Outcomes
Biopharmaceutical companies invest heavily in research and development, investing roughly ten
times more per employee than other industries.1 In 2012, biopharmaceutical companies globally spent
$135 billion on research and development. 2 Celgene spent, on average, more than 30% of the
Company’s revenue, or $1.6 billion.
New medicines have transformed the trajectory of many diseases. Survival rates are increasing for certain cancers,3 HIV/
AIDS4 and diabetes,5 to name a few. Many deadly diseases are on the cusp of being transformed into conditions that can
be managed long-term and that means less time is spent in the hospital;6 more time is spent being productive at work or
with loved ones.7,8
More People Are Surviving as More New Therapies are Developed
More People Are Surviving as More New Therapies Are Developed9
200
180
160
140
120
100
80
60
40
20
0
Number of cancer therapies available
from 1995 through 2014
16
179
14
Cancer survivors
(millions)
11.7
12
8
67
6
4
3.0
2
4.0
1995
9.8
10
101
14.5
2001
2007
0
2015
1971
2001
2007
2014
NEW CANCER THERAPEUTICS DO MORE, FOR LESS
A 2009 study from Cornell University researchers found that the true cost of cancer pharmaceuticals is 30 percent less
than a decade ago once longevity and quality of life are considered. The research report says new therapeutics are
boosting survival rates nearly 100 percent from a decade ago along with a dramatic drop in complications. Patients taking
these new medications are living longer and significantly improving their quality of life. The study draws on the experiences
of thousands colon cancer patients and their doctors’ treatment decisions to cast new light on old assumptions about
how to evaluate the value of new medications.10 http://www.nber.org/papers/w15174.
39
Celgene Chapter 4
PIPELINE OF POSSIBILITIES
Investing in and preparing for a future that is ultimately
The more than 5,400 medicines in the global biopharma-
pose to change the course of human health through bold
ceutical pipeline offer great hope for continued advances
in the years ahead.11 Using 2013 as a benchmark, one-
free from disease is an imperative that defines our purpursuits in science, and a promise to always put patients
first. The successes we continue to deliver are based
third of the 27 new molecular entities approved by the
on fundamental, sound and exciting science. That sci-
Food and Drug Administration (FDA) represent first-inclass medicines, meaning they use new or unique mechanisms of action. A third of these new entities also
address rare diseases adding to the tremendous promise
in the drug development pipeline.12
ence is advancing, and we are advancing along with it,
toward the fully-emerging potential of the revolution in
molecular biology.
Our early clinical stage programs are accelerating across
hematologic, oncologic and immune-inflammatory diseases (I&I). Our early development program is an ambitious value-creating undertaking with life-enhancing
We believe the pipeline is
INFLAMMATION &
IMMUNOLOGY
the
lifeblood of our Company. We
OTEZLA
Psoriatic arthritis
recognize
the successes that we
Psoriasis
Ankylosing spondylitis
Rheumatoid
arthritisin 2014 and will have in
have
had
Behçet’s
Atopic dermatitis
2015
are rooted in the significant
Ulcerative colitis
Mongersen (GED-0301)
investments
that were made a decade
Crohn’s disease
Sotatercept (ACE-011)
Renal
anemia
or
more
ago to commit to making
Pomalidomide
Systemic sclerosis
those
new technologies available to
CC-220
Lupus
patients
today. We believe it is only
Other I&I indications
CC-292 (BTKi)
Rheumatoid arthritis
through
disruptive technologies with
PDA-002 (placenial stem cells)
Peripheral arterial disease/
transformational
impact on patients
Diabetic foot ulcer
that it is possible to produce a great
value proposition for payers, patients
and physicians.”
Phase I
potential programs underway internally as well as with
high-value partners—collaborations that extend our
Phase II
®
Approval
Phase III
capabilities and
maximize opportunities to benefit
patients, public health and the global economy.
One of the most important recent additions to the Celgene
portfolio is GED-0301 that we acquired in early 2014. It is
an example of the cutting-edge technology of anti-sense
that blocks the expression of genes that cause certain
disease. This has the potential to transform the treatment
of Crohn’s disease, affecting millions of people worldwide
from young children to senior citizens, as explained in
detail in the immune-inflammatory section later in this
chapter.13
Additional areas of research, growth and expansion
include new opportunities in the field of epigenetics, such
as our HDAC inhibitors; our first internal candidate in the
field of biologics; key partnerships in the area of protein
homeostasis, the manufacture and control of proteins
Robert J. Hugin, Chairman and Chief Executive Officer,
Celgene Corporation
JP Morgan Healthcare Conference, January 2015
by cellular mechanisms; and bispecific monoclonal antibodies and targeting the processes that lead to cancer
stem cell resistance.
Celgene Hematology and Oncology Discovery Pipeline
HEMATOLOGY &
ONCOLOGY R&D
Discovery
Lead Op
Preclinical
Phase I
Discovery
40
Lead Op
Preclinical
Phase I
CC-223 ( TORKi)
CC-122 ( CELMoD)
Marizomib IV (PI)
ACY-1215 ( HDACi)
AG-120 (IDH-1)
EZ-5676 (DOT1L)
CC-90003 ( ERKi)
CC-90002 (anti-CD-47)
CELMoD AML
CAR-T
OMP-305B83
RPS03
Early Targets
INFLAMMATION &
IMMUNOLOGY R&D
PDA-001
ACY-1215 ( HDACi)
AG-120 (IDH-1)
EZ-5676 (DOT1L)
CC-90003 ( ERKi)
CC-90002 (anti-CD-47)
CELMoD AML
CAR-T
OMP-305B83
RPS03
Living Longer, Better and Healthier Lives
Early Targets
Celgene Inflammation and Immunology Discovery Pipeline
INFLAMMATION &
IMMUNOLOGY R&D
Discovery
Lead Op
Preclinical
Phase I
PDA-001
CC-90001 (JNK-1)
CC-90005 (PKC )
TAPA
BTKi 2.0
ARRY (TYK2)
Early Targets
>
>
EPIGENETICS IN ACTION
These changes in gene expression (turning a gene on)
and gene silencing (turning a gene off), which do not
change the underlying DNA sequence, are collectively
referred to as epigenetics. Some of these epigenetic
changes may be benign as in the caterpillar and the butterfly. But when they allow cells to multiply uncontrollably,
the result can be cancer!14
When the caterpillar changes into a butterfly, its genome—its basic genetic sequence—does not change.
We have important partnerships with Agios in the area of
Some of the specifics include CC-220, our first-in-class
cancer metabolism, applied specifically to the significant
immuno-modulatory therapeutic for inflammatory disease.
unmet medical need treating acute myeloid leukemia
We are designing registration trials for CC-122, our next-
(AML), with Acceleron for its novel approaches to anemia
generation immuno-modulatory for hematologic and solid
and beta-thalassemia, and we are working with partners
tumors, and for CC-292, our BTK inhibitor—a unique target
to assess the potential of the new area of CAR-T cells
for the treatment of B-cell diseases with many possible
that have generated so much attention.
indications in both oncology and I&I. Because these biological functions are complementary, we are also looking
In 2015, we expect to initiate eight new phase I studies
at using these compounds in combinations and possibly
with the majority of those coming from new molecules.
with kinase inhibitors or CAR-T cells.
In our early and mid-stage pipeline we plan to accelerate
12 programs over the next two years.
Working with combinations places us at the leading edge of medical innovation.
We remain committed to, above all, following the science to see where it leads.
41
Celgene Chapter 4
Trust, Patience, Discovery:
The Role of Partnership
At Celgene, we believe that partnerships are as critical to
medical innovation as in-house research. In some areas,
these partnerships allow us to explore new mechanisms
and expand the usefulness of existing ones.
As Tom Daniel, President, Celgene Research and Early
Development, explains, “We are continuously scanning
the landscape for emerging companies, far-sighted academic researchers and novel, innovative projects. We look
for categorical diversification and strategic coherence—a
disruptive technology that offers an anchor point for future
research efforts, a new therapeutic class of molecules, a
next-generation capa­bility. We consider how well their
work complements
and enhances the direction of our own future programs.
Finally, we apply careful scrutiny to test the value and the
utility of the products of their work, therapeutic candidates
and technologies.”
In 2014 alone, Celgene established more than 10 innovative collaborations with some of the best emerging science
companies in the world. We also extended a number of
existing collaborations in support of our long-standing
approach to follow scientific discoveries to see where they lead.
Celgene’s reputation in the research community as a partner of choice is something we are proud of; we’re known
to be willing to take measured risks to advance science in
the interest of science. What’s behind this reputation is
our approach—an approach that focuses on trust,
patience and the quest for discovery.
This is just the first step. The next focuses on the criteria
used to judge fit—for Celgene and for the collaborators
themselves. “We are selective about the people we work
with, looking for accomplished drug hunters and technology developers with great track records, strong leadership
and attitudes that are a cultural fit with our organization,”
Daniel notes. “We pay close attention to their objectives,
and where possible, align our mutual views of the opportunities. Perhaps the most critical element is our ongoing
support of each collaboration with internal experts who
contribute high added value to the collaborators and who
assure ongoing excellent communication.”
This is the most exciting time
in the history of medicine. If we can
make some radical changes to accommodate the enormous opportunities,
there will be better health at lower
costs for many generations to come.”15
Celgene acknowledges that letting the science come to
life is the ultimate goal. By working with partners to dig
deep into new and existing mechanisms, we are able
to get closer to the heart of what triggers and regulates
disease. The company is working on its first immunotherapy drugs directed toward inflammation, expanding
its epigenetic program to make tumors more susceptible
to treatment and advancing new treatment combinations
that have never been tested before.
Eric Topol, M.D., The Creative Destruction of Medicine author
Ultimately, such partnerships build and expand an already
rich pipeline, diversified by timing and by portfolio candidates, for the benefit of patients. By being willing to take
measured risks and invest in promising collaborations,
Celgene is creating benefit for patients now and in the future.
42
Living Longer, Better and Healthier Lives
Hematology—The REVolution
“REVLIMID® Approval for Multiple
Myeloma Can Be Life-Changing for
Patients.”
The International Myeloma Foundation, 2006
In the mid-2000’s, REVLIMID, the brand name for lenalid-
Six months later, June 29, 2006, Celgene reported
omide, represented a new approach to treating blood
REVLIMID plus dexamethasone was approved by the
cancers. It was created in Celgene laboratories as an
FDA for patients with multiple myeloma who had relapsed
immuno-therapeutic with multiple mechanisms to fight
after a prior treatment.
hematologic cancers, and used principally with a synthetic steroid called dexamethasone.
In December 2005, REVLIMID plus dexamethasone was
first approved by the FDA for certain patients with a malig-
REVLIMID is a vital addition to
the treatments we use for myeloma.”
nant bone marrow condition called MDS, who have a
chromosome abnormality called deletion 5q. A press
release from the FDA stated, “In clinical trials, patients
Brian G.M. Durie, M.D., Hematologist and Chairman, International
Myeloma Foundation
treated with REVLIMID no longer needed transfusions,
with most patients becoming independent of transfusion
within three months. The transfusion-free period lasted
for an average of 44 weeks.”16
REVLIMID Prescribing Information
43
Celgene Chapter 4
A year later, June 19, 2007, REVLIMID® plus dexamethasone was approved by the European Union for the same
indication—patients who have been previously treated for
The approval of REVLIMID as
an option for use in all patients with
multiple myeloma represents a new
paradigm in the management of this
disease. We now have clinical evidence
demonstrating that starting and keeping newly diagnosed multiple myeloma
patients on REVLIMID significantly
improves progression-free survival.”
multiple myeloma. Similar approvals came in Switzerland
and Australia. In Canada, REVLIMID was also approved
for MDS patients with deletion 5q.
SURPRISE FINDING FOR STEROIDS USED WITH
REVLIMID: LESS MAY BE MORE
In June 2007, there was a dramatic finding for the
REVLIMID/dexamethasone combination. Many patients
don’t like the mood swings when taking dexamethasone,
Kenneth Anderson, MD, Director, Jerome Lipper Multiple Myeloma
Center, Dana-Farber/Brigham and Women’s Cancer Center
so Michael Katz, then a 16-year myeloma survivor, and
co-chair of the Patient Representatives Committee of the
Eastern Cooperative Oncology Group (ECOG), proposed
a study of a new approach. The study was to determine
Continuing Beyond Myeloma
if patients could do just as well with lower doses of “dex”
when used with REVLIMID.
In June 2013, the FDA approved REVLIMID for mantle
cell lymphoma on strong data from a phase II study.21 Also
THE ANSWER WAS A HUGE SURPRISE.
Patients with low-dose dexamethasone
and REVLIMID actually did better!17
in June 2013, the EMA approved REVLIMID for high-risk,
transfusion dependent MDS patients in Europe with
deletion 5q.22
Survival
Trends
in Hematology
Survival Trends
in Hematology
Although officially approved in the U.S. and Europe only
for patients who had relapsed after an initial treatment,
100
data published in the New England Journal of Medicine in
September 2014 confirmed previous findings, namely that
5-Year Relative
Survival Rates (%)23
88%
90
82
REVLIMID plus low-dose dexamethasone improves survival and other measures of efficacy in newly diagnosed
80
multiple myeloma patients who are not eligible for a stem
70
cell transplant.
18
Hodgkin
Lymphoma
71%
72
59%
60
REVLIMID for All Lines
of Myeloma
50
Non-Hodgkin
Lymphoma
51
45
45%
47
40
In February 2015, the FDA approved REVLIMID for the
broadest possible range of myeloma treatments including
30
newly diagnosed patients and continuous treatment,
demonstrating expanded value by advancing the course
20
Leukemia
34
Myeloma
29
25
of treatment for patients.19 The European Union followed
10
soon after with approvals for frontline REVLIMID treatment
for elderly patients not eligible for transplant.20
0
REVLIMID Prescribing Information
44
1975-1977
1990-1992
2003-2009
Living Longer, Better and Healthier Lives
REVLIMID® is leading the revolution in hematology as a standard of
care in multiple diseases. Patients with myeloma, MDS and mantle cell lymphoma
now have access to this innovative, oral therapy. REVLIMID has also become a
model for developing other successful treatments like POMALYST®/IMNOVID®
and newer hematology compounds still in our early pipeline.”
Jacqualyn A. Fouse, Ph.D., President, Global Hematology and Oncology, Celgene Corporation
Celgene Hematology Pipeline24
HEMATOLOGY
Phase I
Phase II
Phase III
Approval
REVLIMID ®
MM
MDS deletion 5q
MDS non-deletion 5q
CLL
T-Cell leukemia
NHL-MCL
NHL-DLBCL
NHL-FL
NHL-iNHL
VIDAZA®
MDS
AML
ISTODAX ®
CTCL, PTCL
POMALYST®/IMNOVID ®
MM
CC-486 (oral aza)
MDS
AML
Sotatercept (ACE-011)
MDS
beta-thalassemia
Luspatercept (ACE-536)
MDS
beta-thalassemia
AG-221
Heme malignancies
AG-120
Heme malignancies
Our hematology franchise continues beyond REVLIMID
• Agios with AG-221, its IDH2 inhibitor that targets
and POMALYST/IMNOVID. CC-486, our oral epigenetic
mutated enzymes for use in advanced hematologic
agent, often referred to as oral azacitidine, continues
malignancies and some solid tumors for our oncology
enrolling its phase III programs in MDS and AML. In
franchise.
Europe VIDAZA continues to advance with results from
AML-001 showing improvement inPhase
survival
for older
I
ONCOLOGY
25
patients
ABRAXANE ®with acute myeloid leukemia.
• And Acceleron with sotatercept and luspatercept, its
Phase II
TGF-beta superfamily
growth factor regulators
for both
Phase III
Approval
MDS and beta-thalassemia.
Breast
NSCLC
Clinical
Pancreatic trials also continue through collaborations with our
Adjuvant pancreatic
partners
including:
Triple negative breast
VTX-2337
•Ovarian
Acetylon
cancer with ACY-1215, its epigenetic HDAC inhibitor
Squamous cell carcinoma
in combinations
with REVLIMID and with POMALYST/
CC-486
(oral aza)
Solid tumors
IMNOVID
for
previously
treated multiple myeloma.
Demcizumab
NSCLC
REVLIMID
Prescribing Information
Pancreatic
POMALYST
Prescribing Information
Ovarian
VIDAZA
AG-221 Prescribing Information
Solid tumors
ISTODAX
Prescribing Information
45
Celgene Chapter 4
Oncology
What do tumors like to eat, and how
can we trick the tumor into believing
we’re their Friend?
Albumin shell
Paclitaxel
ABRAXANE® (nab-paclitaxel) is a nanotechnology-based
Celgene is working with multiple partners to further evalu-
therapy that is currently the only nanomedicine approved in
ate the potential of these approaches to help improve out-
oncology. With approvals for advanced breast, lung and
comes for patients.27
26
pancreatic cancers in more than 50 countries, ABRAXANE
ABRAXANE also allows solvent-free administration of
remains an essential chemotherapy for treatment success
chemotherapy that would have otherwise limited solubility
in challenging cancers. There are more than 100 studies
in the blood stream. The chemotherapy in standard for-
of ABRAXANE, across poor prognosis cancers, such as
mulation is prepared in Cremophor or ethanol for admin­
triple negative breast cancer, pancreatic cancer and
istration. According to a study in the Annals of Oncology,
squamous lung cancer. In addition, Celgene continues to
the Cremophor vehicle itself has substantial toxicity. This
leverage the unique attributes of ABRAXANE as a back-
includes fatal hyper­sensitivity reactions. The ABRAXANE
bone chemotherapy in combination with immunothera-
nanotechnology eliminates the need for pretreatments and
pies. Given its efficacy and lack of steroid premedication,
for solvents.28
ABRAXANE Prescribing Information
46
Living Longer, Better and Healthier Lives
In 2012, Celgene reported that the FDA approved ABRAXANE® for
the first-line treatment of locally advanced or metastatic non-small
cell lung cancer (NSCLC), in combination with carboplatin, in patients
who are not candidates for curative surgery or radiation therapy. This
approval marked the second indication for ABRAXANE in the U.S.;
it was first approved in 2005 for the treatment of metastatic breast
cancer after failure of combination chemotherapy.29
At LUNGevity our
goal is to extend patients’ lives
and improve the quality of their
lives, and ABRAXANE has the
potential to impact both.”
Andrea Stern Ferris, president, LUNGevity Foundation
Lung & Bronchus Cancer30
20
19
5-Year Relative Survival Rates (%)
Based on Year of Diagnosis
17.5%
18
17
16
15
14
13
12.2
13.1
12.8
13.6
13
12.8
14.3
14.6
15.3
12
11
10
*Y-axis skips from 0 -10, and then continues in intervals of 1 from there on
0
1975-77
1978-80
1981-83
1984-86
1987-89
1990-92
1993-95
1996-98
1999-2002
2003-09
ABRAXANE Prescribing Information
In metastatic pancreatic cancer, ABRAXANE in combination
with gemcitabine is the first and only taxane-based therapy
proven to extend survival. In 2013, the Translational Genomics
Research Institute (TGen) and Scottsdale Healthcare in
Arizona noted the following: The MPACT study of
ABRAXANE plus gemcitabine versus gemcitabine alone
improved one-year median survival rates by 59 percent,
increasing one-year survival from less than a quarter of the
patients (22 percent) to more than a third (35 percent) compared to the standard of care.31 In pancreatic cancer,
ABRAXANE is approved by the FDA for the first-line treatment of patients with metastatic adenocarcinoma of the pancreas, in combination with gemcitabine.
Here we have a therapeutic
that increases survival by a statisti­
20
cally significant and clinically relevant
quantity. In pancreatic cancer, ABRAXANE
15 plus gemcitabine is a significant step
forward. So it’s a good option for patients.”
Manuel Hidalgo, M.D., Ph.D., director of the clinical research pro-
10 gram at CNIO, Centro Nacional de Investigaciones Oncológicas
in Madrid, Spain
Pancreatic Cancer32
7
5-Year Relative Survival Rates (%)
Based on Year of Diagnosis
6.4%
6
5.3
5
4.3
3.5
4
3
2.4
2.8
2.8
2.9
1978-80
1981-83
1984-86
3.9
4.4
2
1
0
1975-77
1987-89
1990-92
47
1993-95
1996-98
1999-2002
2003-09
Celgene Chapter 4
In 2014 at the San Antonio Breast Cancer Symposium, the German Breast Group (GBG) announced that ABRAXANE®
presented significant benefit for patients with early high risk breast cancer when compared to conventional paclitaxel.
These findings were from the GeparSepto clinical trial, which was sponsored by GBG and conducted together with the
German AGO-B study group. The trial involved over 1,200 patients, which is currently the largest randomized phase III
study ever completed with nab-paclitaxel and the first trial completed in high risk early breast cancer.33 In breast cancer,
ABRAXANE is approved by the FDA for the treatment of metastatic breast cancer (MBC) after failure of combination
chemotherapy for metastatic disease or relapse within six months of adjuvant chemotherapy. Prior therapy should have
included an anthracycline unless clinically contraindicated.
HEMATOLOGY
REVLIMID
Breast
Cancer34
MM
Phase I
Phase II
Approval
Phase III
®
MDS deletion 5q
MDS non-deletion 5q
100
5-Year Relative
CLL
Based
T-Cell leukemia
95
NHL-MCL
Survival Rates (%)
on Year of Diagnosis
NHL-DLBCL
NHL-FL
85
NHL-iNHL
VIDAZA®
80
MDS
AML
®
ISTODAX
80
CTCL, PTCL
74.8
POMALYST®/IMNOVID ®
75
MM
CC-486 (oral aza)
MDS
70
AML
Sotatercept (ACE-011)
*Y-axis skips
MDS
beta-thalassemia
Luspatercept (ACE-536)
0
MDS
1975-77
beta-thalassemia
AG-221
Heme malignancies
AG-120
Heme malignancies
85.2
84
74.4
76.1
86.3
88.2
89.9
90.3%
78.9
from 0-70, and then continues in intervals of 5 from there on
1978-80
1981-83
1984-86
1987-89
1990-92
1993-95
1996-98
1999-2002
2003-09
Beyond the current approvals in metastatic breast cancer, non-small-cell lung cancer and metastatic pancreatic cancer,
ABRAXANE is a pipeline in itself, with trials in combination with new immuno-oncologic agents such as PD-1 and PDL-1
100inhibitors, trials with locally advanced pancreatic cancer, a broad clinical program in non-small-cell lung cancer, additional
trials in triple negative breast cancer, and more.
80
Celgene Oncology Pipeline35
ONCOLOGY
Phase I
Phase II
ABRAXANE ®
Breast
NSCLC
Pancreatic
Adjuvant pancreatic
Triple negative breast
VTX-2337
Ovarian cancer
Squamous cell carcinoma
CC-486 (oral aza)
Solid tumors
Demcizumab
NSCLC
Pancreatic
Ovarian
AG-221
Solid tumors
60
ABRAXANE Prescribing Information
48
Phase III
Approval
Living Longer, Better and Healthier Lives
Inflammation and Immunology
Celgene I&I Pipeline36
INFLAMMATION &
IMMUNOLOGY
Phase I
Phase II
Phase III
Approval
OTEZLA ®
Psoriatic arthritis
Psoriasis
Ankylosing spondylitis
Rheumatoid arthritis
Behçet’s
Atopic dermatitis
Ulcerative colitis
Mongersen (GED-0301)
Crohn’s disease
Sotatercept (ACE-011)
Renal anemia
Pomalidomide
Systemic sclerosis
CC-220
Lupus
Other I&I indications
CC-292 (BTKi)
Rheumatoid arthritis
PDA-002 (placenial stem cells)
Peripheral arterial disease/
Diabetic foot ulcer
Ozanimod
Relapsing multiple sclerosis (RMS)
Ulcerative colitis
Crohn’s disease
Now, with the global launch of OTEZLA® underway, Celgene
moves beyond groundbreaking work in hematology and
oncology to create new therapeutic potential for the millions
of patients with immune-inflammatory disorders in the U.S.
and around the world.
The therapeutic area of Inflammation and Immunology (I&I)
Since
2009, the International Federation of
HEMATOLOGY &
Discovery
Lead Op
ONCOLOGY
R&D Associations (IFPA)
Psoriasis
and psoriasis
CC-223 ( TORKi)
CC-122 ( CELMoD)
associations
worldwide have worked hard to
Marizomib IV (PI)
put
psoriasis
on the agenda of the World Health
ACY-1215
( HDACi)
AG-120 (IDH-1)
Organization
(WHO).
During the 133rd meeting
EZ-5676 (DOT1L)
CC-90003 ( ERKi)
of
the(anti-CD-47)
WHO Executive Board, a resolution on
CC-90002
CELMoD AML
psoriasis
was proposed and discussed, leading
CAR-T
OMP-305B83
to
unanimous adoption by the WHO Executive
RPS03
Board.
This resulted in the resolution being
Early Targets
put before the 67th World Health Assembly in
May 2014, where it was also approved.
INFLAMMATION &
IMMUNOLOGY R&D
Discovery
is new to Celgene, but the creation of OTEZLA dates
Preclinical
Phase I
back 15 years to our own laboratories. Celgene’s scientific
teams nurtured apremilast, eventually named OTEZLA,
through preclinical testing, clinical trials and on to regulatory approvals and commercial availability. Today, patients
with psoriasis and psoriatic arthritis can access the clinical
benefits of OTEZLA in more than 30 countries.37
Supporting our belief that patients with autoimmune and
inflammatory disorders were underserved, the reaction
to OTEZLA has been clear and strong. Since launching
in psoriatic arthritis in 2014, OTEZLA has obtained the
highest initial weekly
of any launch
Preclinical prescription volume
Phase I
Lead Op
For more information and to read the resolution visit
PDA-001
http://www.ifpa-pso.org/web/page.aspx?refid=273.
CC-90001
(JNK-1)
CC-90005 (PKC )
TAPA
BTKi 2.0
ARRY (TYK2)
in the I&I space in recent years. In psoriasis, the number
of dermatology prescribers of OTEZLA quadrupled in just
the first three months after its U.S. launch.
OTEZLA Prescribing Information
Early Targets
49
Celgene Chapter 4
Beyond these large areas of unmet medical need,
GED-0301 targets mRNA for the protein Smad7. High
Celgene remains committed to expanding the OTEZLA®
levels of Smad7 interfere with anti-inflammatory pathways
brand in terms of indications and geography. We are
(TGF-beta 1) in the digestive tract, leading to increased
advancing the program in ankylosing spondylitis and initi-
inflammation.39
ating phase III studies of OTEZLA in atypical dermatitis
In April 2014, Celgene acquired this late-stage compound
and ulcerative colitis. We are also pursuing an indication
targeting Crohn’s disease and other gastrointestinal
in Behçet’s disease, a rare, chronic, auto-inflammatory
disorders.
disorder that leads to damage to blood vessels throughout the body. Turkey, the country with the most significant
prevalence of Behçet’s, accepted the phase II trial results
for regulatory filing because they recognize the potential
GED-0301 is a potentially
transformative therapy that demonstrated striking clinical activity in
a Phase II trial for Crohn’s disease.
It strengthens our expanding pipeline
of novel therapies intended to address
significant unmet medical need in
immune-mediated diseases.”
value OTEZLA could bring to their people.
This impressive acceleration of approvals and demand for
the clinical benefits of this medical innovation would seem
to be enough for the I&I franchise. But it is not.
True to the Celgene purpose to pursue transformational
science that will translate into life-enhancing medicines,
we’ve followed the science to Dublin, Ireland and GED0301. This is a first-in-class oral anti-sense therapeutic
being studied for the treatment of Crohn’s disease, an
immune-mediated, chronic inflammatory condition of the
Scott Smith, President, Inflammation and Immunology,
Celgene Corporation
gastrointestinal tract, with potential further applications in
ulcerative colitis.
Celgene’s CC-220 (cereblon modulator), a next-generation
Antisense Oligonucleotide Therapy
compound targeting inflammatory diseases, which has
phase II trials underway in lupus, a disease that affects
five million people worldwide,40 with further plans for Double-stranded DNA
clinical trials in systemic sclerosis and sarcoidosis.
Sotatercept (ACE-011) represents a potential new
approach to treating for renal anemia. The success of
OTEZLA and the potential of our new therapies highlight
the promising future of our newest franchise.
Celgene Cellular Therapeutics, CCT, is Celgene’s cellular
therapies research division, focused on the therapeutic
use of stem cells derived from human placentas and
Gene
Single-stranded mRNA
carries instructions to
make a protein
umbilical cord blood. Stem cell-based therapies repre­
Single-stranded ANTISENSE
oligonucleotide, locks onto
the mRNA to inactivate it
sent an important new option in the treatment of currently
untreatable diseases.
Stem cells from umbilical cord blood and from the placenta, which is discarded as medical waste, are abun­
Antisense therapies target molecules called messenger
dant and ethically uncontroversial. Celgene has multiple
RNA (mRNA). The mRNA carries information from each
studies including those that demonstrate that these cells
section of the DNA to create a protein. Anti-sense tech­
have the potential to repair or regenerate a wide range of
nology creates a complementary or mirror image of the
damaged or disease-affected tissues.
mRNA that binds to it and inactivates its ability to function.38
OTEZLA Prescribing Information
50
Living Longer, Better and Healthier Lives
Celgene Cellular Therapeutics
Exploring Novel Areas of Research
Celgene Cellular Therapeutics Pipeline41
CELLULAR THERAPIES
Phase I
Phase II
Phase III
Filing/Approval
Post-Reg/Approval
BIOVANCE® (human amniotic
membrane allograft)
Wound management
extracellular matrix
Wound management
PDA-001
Crohn’s disease
PDA-002
Peripheral artery disease
/Diabetic foot ulcers
In 2006, CCT obtained the first U.S. patent for methodology
•A
second product, PDA-002, is in trials for peripheral
to recover a variety of cells from the human placenta. This
artery disease and diabetic foot ulcers—painful sores
was the first of many CCT stem cell patents, and Celgene
that do not heal.
maintains a dominant intellectual property estate in this
•T
he human placental extracellular matrix (ECM), the
increasingly important therapeutic area.
non-cellular part of tissue that provides structural support to cells, is a highly versatile biomaterial that can be
LifebankUSA is Celgene’s tissue banking facility that
®
formulated and configured for a number of therapeutic
allows families to store placental cells and cord blood
applications.
so they can be used to harness the potential of stem cell-
• BIOVANCE® is a topically-applied wound covering
based therapies if needed in the future.
produced from the human amniotic membrane and
In 2008, the first human patient was treated with a combi-
partnered for distribution.
nation of cord blood and placenta-derived stem cells for
ALL (acute lymphoblastic leukemia). See Quentin Murray’s
story in Chapter 2.
Celgene scientists also discovered specific cell surface
All these advancements rep­
resent the various ways CCT is giving
birth to a wide range of new ideas
from nature’s own sources of healing
tissues.”
properties that allow pharmaceuticalized placenta-derived
stem cells to be infused therapeutically without the need
for tissue matching.
Since that time, CCT has developed a number of products
with potential therapeutic applications:
• PDA-001 is the pharmaceuticalized product derived
Perry A. Karsen, Chief Executive Officer, Celgene
Cellular Therapeutics
from placental stem cells. It is currently in clinical trials
for Crohn’s disease.
51
Celgene Chapter 5
5
Translating Science into Transformational M edicines
Working Toward a World Free from Cancer
52
Living Longer, Better and Healthier Lives
Creating the Future Through
Medical I nnovation
The future of cancers and other profound, debilitating diseases is being shaped today by:
• Research-stage science that will translate into exciting new life-enhancing therapies, changing the shape and
course of disease
• Public policy, access and reimbursement decisions that will allow all patients who need treatment to receive it
• Patient-focused models that will support the whole person and not just the illness.
The progress toward better outcomes and longer lives is reflected in the annual cancer death rate dropping by
an average 0.5 percent every year between 2002 and 2011.1 At the current pace of innovation, the number of
life-years lost to cancer is expected to decrease by 43 percent within the next 30 years.2
The Promise of Medical
Innovation = Fewer Lives Lost 3
Cancer Patients Want a Say
in Treatment Decisions 4
•
•
•
•
80
169.3 Million
Patients and Their Families
Physicians
Public Payers/Insurers
Other
78%
72%
70
43%
Reduction
60
50
41%
40
96 Million
28%
30
20
10
0
Life-years Lost to Cancer Worldwide
Who Decides?
Who Pays?
The quality of those life-years is expected to increase
choice of medicines to treatment models to broad and
measurably as well, as patients enjoy medicines that are
unencumbered access to essential new scientific devel-
better targeted and easier to tolerate, including new cancer
opments. A 2012 survey entitled, “New Insight into Public
medicines that leverage the body’s own innate immune
Perceptions of Cancer” found that patients, well above
system to attack select cancer cells and stop them in
physicians and other interested parties, wanted a clear
their tracks.
decision making role in their treatment, placing them as
5
the primary determinant of what happens in their fight
In the midst of such profound change is the ever-growing
against disease.7
voice of the patient, an important and necessary voice.
Patients want to be more involved in their care,6 from
53
Celgene Chapter 5
A Tribute to
Patients
In fact, she was able to celebrate her 49th birthday and
enjoy another year and a half of life. The gift she gave of
herself to support the trial paid off.
I pity the next person who asks
me if investment in medical innovation
is affordable for our society. Because
of medical advances I just celebrated a
birthday I never thought I would have.”
The story of medical
innovation begins and
ends with patients.
Lynne B. Jacoby—September 9, 1964–October 6, 2013
Lynne passed away 18 months after her initial diagnosis.
Her story, however, is triumphant. She, like the countless
patients who fight for more every day, gave us deeper
knowledge about her disease and, more importantly,
about the power of the human spirit, and a reminder that
we’re all in this together.
We thank the patients who volunteer for trials, giving freely
of their time to advance treatments for themselves and,
in some cases, for people they will never meet. We thank
the patients who advocate for themselves and others,
ensuring that the investments made in research, programs
and treatment models truly serve those in need. We thank
people like Lynne Jacoby.
“For someone who is given a diagnosis like me and told
that their life would be measured in weeks—the fact is,
everybody’s life is measured in weeks, and we have to do
whatever we can to make it as many weeks as possible.”
Lynne was diagnosed at age 48 with Stage 4 pancreatic
cancer. Her doctor told her that she had about three
months of life left. The advice was to go home, get her
things in order and focus on making her remaining days
comfortable.
That did not sit well with Lynne.
Instead, she offered her time and body to a trial for
ABRAXANE®. She knew she wouldn’t live forever, but she
knew she could not spend her time just waiting for the
end. As Lynne said, “I could accept the diagnosis but
I could not accept the prognosis.”
Within two months the tumors on her liver shrank tremendously. Soon afterward, the tumors on her pancreas followed suit. And the difficulties that accompanied her
disease—the night sweats, indigestion, stomach pains,
neck and back pains, and elevated white-blood cell
counts—subsided. She lived far beyond three months.
The cost of new cancer drugs represents less than one percent
of the value these therapies yielded in mortality reduction.”
ABRAXANE Prescribing Information
Frank Lichtenberg, Ph.D., Columbia Business School
54
Living Longer, Better and Healthier Lives
Nurturing
innovation
During the 1970s, the four largest European countries were
responsible for 55 percent of new medicines produced by
major nations, while the U.S. held a 31 percent share. But
over the decade from 2001 to 2010, the U.S. share jumped
to 57 percent, while France, Germany, Switzerland, and
the United Kingdom saw their share of new medicines
plummet 33 percent.8
Researchers are pioneering sophisticated new techniques
The future of transformational medicines also lies in how open a society is in terms of
in the areas of molecular diagnostics, computational tools,
targeted therapies and overall healthcare practices that
allowing innovation to flourish. Smart, up-to-date regula-
will also tame cancers to one day transform them into
tory processes that match the maturity of the science,
manageable conditions. Leveraging epigenetics, genom-
legal operating environments that honor the intellectual
ics, proteomics, Big Data, and more, today’s members of
rights of innovators and reimbursement models that give
the healthcare ecosystem are emboldened in their pursuit
patients more options rather than less are all factors in
of making debilitating diseases and conditions a thing of
how receptive and productive a society is in its support
the past. Here is an example of where science and policy
of patients and their medical benefit.
can overcome hurdles to work together, transforming
forward-thinking ideas into medical solutions that will help
patients live longer, better and healthier lives.
55
Celgene Chapter 5
The Value of Sharing
SEEKING INNOVATIVE TREATMENTS FOR
NEGLECTED DISEASES OF THE DEVELOPING WORLD
At Celgene, we believe patients should have the opportu-
CGH is screening our diverse chemical library for activity
nity, regardless of their location or financial resources, to
in neglected diseases affecting the developing world
benefit from advances in prevention, diagnosis and treat­
including sleeping sickness (human African trypanosomia-
ment of disease. Celgene Global Health (CGH), founded
sis), an epidemic in 36 African countries;10 Chagas disease,
PMS 143
PMS CG10
in 2009, collaborates with partners around the globe in
endemic in 21 countries in Latin America;11 leishmaniasis,
finding innovative solutions for healthcare challenges in
occurring in 98 countries with 350 million people at risk;12
the developing world. This work is based on our belief
lymphatic filariasis, affecting more than one billion people;
that innovative therapies and healthcare partnerships are
along with malaria; tuberculosis and viral hemorrhagic
essential components of long-term progress and prosper-
fevers.
PMS 557
PMS 7466
PMS 7441
PMS 646
ity around the globe.9
For more information please visit:
https://www.celgene.com/content/uploads/2014/12/celgene2014-crr.pdf
56
Living Longer, Better and Healthier Lives
PROJECT DATA SPHERE®
For decades, life sciences companies, research institu-
Project Data Sphere, which is the brainchild of the CEO
tions, hospitals and independent researchers have been
Roundtable on Cancer’s Life Sciences Consortium (LSC),
searching for answers to cancer’s most complex questions.
is a singular platform where the entire global cancer com-
For decades, they have done this work alone or in small
munity can share, integrate and analyze historical patient
collaborations. The patients who freely participated in their
level, comparator arm data from academic and industry
research hoped their experiences could lead to break-
phase III cancer trials (individual information is de-identified
throughs—for themselves or for future cancer patients who
before sharing). It is open to researchers affiliated with life
would benefit from their sacrifice. But what could happen if
science companies, hospitals, research institutions and
trial information could be shared freely and without borders?
independent researchers, at no cost. By sharing informa-
What could happen if the pace of innovation moved faster?
tion on an individual patient level, everyone participating
in the platform can gain new insight into cancer research,
In 2014, Celgene co-led an effort that included Sanofi,
supporting the goal of sharing an ever increasing, ever
AstraZeneca, Bayer, Pfizer, Johnson & Johnson and
diverse and ever improving set of information for the bene-
Memorial Sloan-Kettering Cancer Center in the launch of
fit of cancer patients now and in the future. Celgene sits
Project Data Sphere—a first-of-its-kind initiative to create a
on the Board of Directors of LSC.
global, research-sharing platform. By taking a leadership
role in this collective approach to research, Celgene con-
The LSC understood that cancer is a global disease, and it
tributes to the goal of creating lasting value for patients
needs global cooperation, global transparency and global
in terms of insight, innovation and speed. As Bob Hugin,
innovation to meet it head-on. Collective action is a powerful
Celgene CEO, noted at Project Data Sphere’s inauguration,
response, powerful for research and, ultimately, a powerful
“ … Project Data Sphere has the potential to accelerate the
way to find solutions for patients.13
speed with which clinical trials are conducted, improve the
efficiency of trial designs and assist with the development
of data standards applicable to all cancer types.”
For more information about Project Data Sphere, visit our website at: https://projectdatasphere.org
Celgene Global Health Pipeline
Discovery
CELGENE GLOBAL
HEALTH PIPELINE
Hit ID
Lead Identification
Development
Lead Optimization
New Chemical Entities in Celgene’s Library
Visceral Leishmaniasis
Chagas/HAT
Malaria
Helminths
Wolbachia
Tuberculosis
CC-11050
Tuberculosis
Erythema nodosum leprosum
Immune reconstitution
inflammatory syndrome
CC-11050/CC-5048
Hemorrhagic Fevers (Junin, Lassa,
Ebola, Rift Valley)
Lenalidomide
Human Immunodeficiency Virus
Pomalidomide
Kaposi Sarcoma
57
DC
Nonclinical
Phase I
Phase II
Celgene Chapter 5
Toward a World
Free from CANCER
CANCER
58
Living Longer, Better and Healthier Lives
We need to continue the momentum we have
started and work together to change the course of human
health for patients, health care, our economy and future
generations.”
Robert Hariri, Ph.D., Vice-chairman and Co-founder, Human Longevity, Inc.
THE GOAL IS TO SAVE THE PATIENT, NOT JUST KILL THE CANCER.
“It’s realistic to think we will have a world free from the
Despite these advances, cases and deaths continue to
impact of cancer,” said Amy Abernethy, MD, Ph.D., a
rise globally from 12.7 million in 2008 to over 14.1 million
medical oncologist at Duke Medicine in North Carolina.
cancer cases in 2012, according to the World Health
Organization (WHO).17 That’s an increase of 11 percent.
She was one of the experts at a recent forum on cancer
The World Cancer Research Fund International estimates
research—experts who believe we might be getting closer
that this number could increase to 24 million by 2035.18
to a new reality: a WORLD FREE FROM CANCER.
With statistics likes these, it’s understandable why so
Medical innovation has already made significant progress
many people are afraid of cancer, and why they some-
against cancer. Today, the five-year relative survival rate
times feel hopeless upon diagnosis.
for all cancers is 68 percent, up from 49 percent in the
However, as the experts believe, a WORLD FREE FROM
1970s.14 Over the next decade, the number of people who
CANCER is not only possible but also obtainable. Advances
have survived five or more years after cancer diagnosis
are starting to turn cancer into a chronic disease, allowing
is expected to increase by 37 percent to over 11.9 million
patients to live longer, better and healthier lives. These
in the U.S. according to a report published in Cancer
patients, and the growing number of patients to follow in
Epidemiology, Biomarkers and Prevention.15
the years to come, will be known as cancer survivors, not
The changing tide of cancer survival is already evident in
cancer victims; people who set and achieve new goals in
multiple myeloma. In the U.S. alone, survivorship increased
life after diagnosis. Although not all of them will actually be
by 73 percent in the first decade of this century.16
cured of the disease, they will have a better chance to live
full lives with the right treatments.
To learn more, read a recent Scientific
American Worldview publication,
Cancer: The March on Malignancy.
To access please visit:
www.nature.com/nature/outlook/cancer
59
Celgene Chapter 5
Let Us Begin
To achieve a WORLD FREE FROM CANCER, we need continued innovation and collaboration
from patients, advocates, healthcare providers, academia, innovators, payers, policymakers
and regulators. Public-private partnerships provide an option to help researchers share
resources and data in a collaborative manner. In addition, by simplifying the accelerated
approval pathway for new therapies, patients could get access to new, effective medicines
faster than ever before.
Intervene Sooner Louis J. DeGennaro, Ph.D., president and CEO of the Leukemia
& Lymphoma Society (LLS) says, “Currently, healthcare is in the mode of thinking about
treating cancer after it happens, but we should be challenging ourselves in the near term
to put more resources into ways to prevent cancer.”
Diagnose Faster Lee Hood, M.D., Ph.D., president of the Institute for Systems
Biology, has an initiative that looks at the starting point where health begins to transform
into disease so we can learn how and why this happens in time to intervene.
Treat Smarter But once the cancer does escape and begins to do its damage,
smarter targeted treatments must be the order of the day.
In some cases, that has started to happen already. Inno­vative oral medications can be
administered at home reducing costly hospitalizations.19 More tolerable treatments can be
taken continuously so the cancer has less of a chance to regroup and re-emerge.20
60
Living Longer, Better and Healthier Lives
Miles Beyond Myeloma
With a single pill, a patient with terminal cancer maintains his status as a
marathon machine.
Wearing a yellow visor, Don Wright slipped between other runners on his way to the finish line. On that sunny December
day in 2012 in Honolulu, he completed his goal of running a marathon in each of the 50 states, having participated in
70 marathons overall. Few 71-year-olds could accomplish that, especially nearly a decade after being diagnosed with
multiple myeloma. Wright beat the typical five-year survival average, surpassing it by two times so far.
He is able to do all that running because a once-a-day pill keeps his myeloma at bay. “It’s so easy to take,” he says, “and
I don’t have any side effects.” In fact, Wright, now 74 years old, feels so good that even a marathon in every state was not
enough for him.
“We’re going to go to 100 marathons,” he says. “When we get there, we’ll go for 101.”
61
Celgene Chapter 5
Celgene’s Leadership Position
Celgene is positioned to play a leading role in the transformation of cancer to a chronic,
manageable disease. We can do this in many ways, such as proposing patient-friendly policies that may help to help reduce health care costs and spur innovation while preserving the overall high quality of our healthcare ecosystem. These recommendations are a call to action for us all, to fight for:
• Faster ways to approve new therapies with a goal of saving more lives sooner.
• Adequate funding for our incubators of basic research, such as the National Institutes
of Health.
• A regulatory paradigm that recognizes the nature of 21st century science, including the
creation of “innovation spaces” for regulators.
• Putting cost in context. “While the cost of bringing cutting-edge cancer treatments to
the public is a subject of endless debate, to the patient facing a devastating illness,
results are priceless,” says Tomas J. Philipson, Ph.D., The University of Chicago.
• A reimbursement paradigm that acknowledges broad and unencumbered access to
new medicines generates greater longevity; improved quality of life; reduced cost of
managing disease and increased productivity which is good for our economy.
• And, above all, bringing the voice of the patient to the forefront through comprehensive, transparent and consistent processes to integrate patient input into healthcare
decision making.
In just 30 years, medical innovation decreased the
number of HIV-related deaths in the U.S. by 90 percent. Imagine
that by the year 2050, if we work together, we could proclaim
the same for patients with cancer.”
Robert Goldberg, Ph.D., Health Economist, Center for Medicine in the Public Interest
At Celgene, reaching for this WORLD FREE FROM CANCER is what drives us every day.
62
Living Longer, Better and Healthier Lives
We are a company of people committed to delivering
on aspirational objectives that meaningfully improve the
lives of patients in need. We are continuing to build a company where every employee’s best work is supported and
celebrated for the impact it has on patients’ lives.”
Robert J. Hugin, Chairman and Chief Executive Officer, Celgene Corporation
63
Living Longer, Better and Healthier Lives
References
1. Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.
2. E stimate derived from the National Bureau of Economic Research. Sun EC, Jena AB, Lakdawalla DN, et al. An Economic Evaluation of the War on Cancer. NBER Working Paper No. 15574.
Issued in December 2009. Available at http://www.nber.org/papers/w15574. Accessed April, 2015. Methodology discussed at http://valueofinnovation.org/about-the-clock.html and
http://imgsrv.wben.com/image/wben2/UserFiles/File/Philipson%20FF.pdf. Lichtenberg, FR. Current estimates based on “Has Medical Innovation Reduced Cancer Mortality?” National
Bureau of Economic Research. Revised October, 2013. Accessed April, 2015.
3. A merican Cancer Society. Cancer treatment & survivorship facts & figures 2014–2015. Available at
http://www.cancer.org/acs/groups/content/@research/documents/document/acspc-042801.pdf. Accessed April, 2015.
4. Lichtenberg FR. NBER Working Paper No. 18235. Pharmaceutical innovation and longevity growth in 30 developing and high-income countries, 2000–2009. Available at
http://www.nber.org/papers/w18235. Accessed April, 2015. Lichtenberg FR. NBER Working Paper No. 9754. The impact of new drug launches on longevity: evidence from longitudinal
disease-level data from 52 countries, 1982–2001. Available at http://www.nber.org/papers/w9754. Accessed April, 2015.
5.Centers for Medicine in the Public Interest. The medical value of innovation. Available at http://valueofinnovation.org. Accessed April, 2015.
6. A merican Cancer Society. Cancer treatment & survivorship facts & figures 2014–2015. Available at
http://www.cancer.org/acs/groups/content/@research/documents/document/acspc-042801.pdf. Accessed April, 2015.
7. M Y LIFE IS WORTH IT. MY LIFE IS WORTH IT Calls for Balanced View of the Value of Advanced Therapeutics for Cancer Patients at Global Hematology Conference. Available at
http://www.businesswire.com/news/home/20141203005328/en/LIFE-WORTH-Calls-Balanced-View-Advanced-Therapeutics#.VTavpFxEgzI. Accessed April, 2015
8. P harmaceutical Research and Manufacturers of America. The biopharmaceutical industry helps strengthen the U.S. economy, Available at http://www.phrma.org/economic-impact.
Accessed April, 2015.
9. Celgene data on file.
10. Celgene data on file.
11. Celgene data on file.
12. Celgene data on file.
13. T he Hamilton Project at the Brookings Institution. Available at http://www.hamiltonproject.org/multimedia/charts/deaths_from_major_infectious_disease/. Accessed April, 2015.
14. Lichtenberg F. Benefits and Costs of Newer Drugs: An Update. National Bureau of Economic Research, Working Paper No. 8996. June 2007. Available at
http://www.nber.org/papers/w8996. Accessed April, 2015.
15. IMS Institute. Innovation in cancer care and implications for health systems, Global oncology trend report. Available at
http://www.imshealth.com/portal/site/imshealth/menuitem.762a961826aad98f53c753c71ad8c22a/?vgnextoid=f8d4df7a5e8b5410VgnVC. Accessed April, 2015.
16. Centers for Medicare and Medicaid Services. National Health Expenditure Data. Available at https://www.cms.gov/. Accessed April, 2015.
17. Cuckler GA, Sisko AM, Keehan SP, et al. National health expenditure projections, 2012–22: slow growth until coverage expands and economy improves. Health Aff (Millwood)
2013;32(10):1820-1831.
18. Lichtenberg FR. NBER Working Paper No. 18235. Pharmaceutical innovation and longevity growth in 30 developing and high-income countries, 2000–2009. Available at
http://www.nber.org/papers/w18235. Accessed April, 2015.
19. Lichtenberg FR. NBER Working Paper No. 9754. The impact of new drug launches on longevity: evidence from longitudinal disease-level data from 52 countries, 1982–2001. Available at
http://www.nber.org/papers/w9754. Accessed April, 2015.
20. Congressional Budget Office. Offsetting Effects of Prescription Drug Use on Medicare’s Spending for Medical Services. Report, November 29, 2012. Available at
http://www.cbo.gov/publication/43741. Accessed April, 2015.
21. McWilliams, J. Michael. Implementation of Medicare Part D and Nondrug Medical Spending for Elderly Adults With Limited Prior Drug Coverage. JAMA. 2011;306(4):402-409.
doi:10.1001/jama.2011.1026.
22. A merican Cancer Society. Cancer Facts & Figures, 2014. Available at http://www.cancer.org/acs/groups/content/@research/documents/webcontent/acspc-042151.pdf. Accessed April, 2015.
23. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2011. Table 2.8, All Cancer Sites (Invasive), 5-Year Relative
and Period Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/csr/1975_2011/results_single/sect_02_table.08.pdf. Accessed April, 2015.
24. A merican Cancer Society. Cancer Treatment and Survivorship Facts & Figures 2014–2015. Available at
http://www.cancer.org/acs/groups/content/@research/documents/document/acspc-042801.pdf. Accessed April, 2015.
25. Pharmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed April, 2015.
26. N ational Cancer Institute. Cancer Trends Progress Report—2005 Update. Table L1: National Cancer Treatment Expenditures in Billions of Dollars (1963–2004). Available at
http://www.progressreport.cancer.gov/sites/default/files/archive/report2005.pdf. Accessed April, 2015.
27. C
utler DM. Are we finally winning the war on cancer? J Econ Persp 2008:22(4);3-26.
28. Mariotto AB, Yabroff KR, Shao Y, et al. Projections of the cost of cancer care in the United States: 2010–2020. J Natl Cancer Inst 2011;103(2):117-128.
enters for Disease Control and Prevention. Health, United States, 2012. Table 111. Gross domestic product, national health expenditures, per capita amounts, percent distribution, and
29. C
average annual percent change: United States, selected years 1960–2011. Available at http://www.cdc.gov/nchs/data/hus/2012/111.pdf. Accessed April, 2015.
30. P harmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed April, 2015.
31. Pharmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed April, 2015.
32. Pharmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed April, 2015.
33. Tufts Center for the Study of Drug Development. Cost to develop and win marketing approval for a new drug is $2.6 billion. Available at
http://csdd.tufts.edu/news/complete_story/pr_tufts_csdd_2014_cost_study. Accessed April, 2015.
34. L ebwohl MG, Bachelez H, Barker J, et al. Patient perspectives in the management of psoriasis: Results from the population-based Multinational Assessment of Psoriasis and Psoriatic
Arthritis Survey. J Am Acad Dermatol. 2014;70(5):871-881.e30.
35. Lebwohl MG, Bachelez H, Barker J, et al. Patient perspectives in the management of psoriasis: Results from the population-based Multinational Assessment of Psoriasis and Psoriatic
Arthritis Survey. J Am Acad Dermatol. 2014;70(5):871-881.e30.
36. A merican Autoimmune Related Diseases Association (AARDA). The Cost Burden of Autoimmune Disease: The Latest Front in the War on Healthcare Spending. Available at
http://www.diabetesed.net/page/_files/autoimmune-diseases.pdf. Accessed April, 2015.
37. Cortesi, P, Scalone, L, D’Angiolella, L, et al. Systematic Literature Review on Economic Implications and Pharmacoeconomic Issues of Psoriatic Arthritis. Clinical and Experimental
Rheumatology. 2012; 30(73): S126-S131.
38. Boonen. A., Socioeconomic Consequences of Ankylosing Spondylitis. Clinical and Experimental Rheumatology. 2002; 20(6Suppl 28): S23-6.
64
Living Longer, Better and Healthier Lives
39. Dean L, Jones G, MacDonald A, et al. Global Prevalence of ankylosing spondylitis. Rheumatology. 2014; 53: 650-657.
40. United States Census Bureau. U.S. and World Population Clock. Available at https://www.census.gov/popclock/. Accessed April, 2015.
41. National Psoriasis Foundation. Drug Pipeline. Available at http://services.psoriasis.org/drug-pipeline/index.php. Accessed April, 2015.
42. Pharmaceutical Research and Manufacturers of America (PhRMA). Medicines in Development: Arthritis. 2014. Available at
http://www.phrma.org/sites/default/files/pdf/2014-meds-in-dev-arthritis.pdf. Accessed April, 2015.
43. United Nations Development Program. World Population Prospects The 2010 Revision. Volume 1: Comprehensive Tables. Available at
http://esa.un.org/wpp/Documentation/pdf/WPP2010_Volume-I_Comprehensive-Tables.pdf. Accessed April, 2015. U.S. Food and Drug Administration. Centers for Disease Control and
Prevention and National Center for Health Statistics. Health, United States, 2011: With Special Feature on Socioeconomic Status and Health. Available at http://www.cdc.gov/nchs/index.htm.
Accessed April, 2015. Prentice T. Health, History and Hard Choices: Funding Dilemmas in a Fast-Changing World. Nonprofit and Voluntary Sector Quarterly. 2008: Supplement to vol 37;
no 1: 63S-75S.
44. P harmaceutical Research and Manufacturers of America. PhRMA Chart Pack: Biophar­m aceuticals in Perspective. Spring 2013. Version 3.0. Chapter 6, Slide 75. Available at
http://www.phrma.org/sites/default/files/pdf/CHART%20PACK_online%20version_13APR04_forweb.pdf. Accessed April, 2015.
45. Celgene data on file.
46. Lichtenberg FR. NBER Working Paper No. 18235. Pharmaceutical innovation and longevity growth in 30 developing and high-income countries, 2000–2009. Available at
http://www.nber.org/papers/w18235. Accessed April, 2015. Lichtenberg FR. NBER Working Paper No. 9754. The impact of new drug launches on longevity: evidence from longitudinal
disease-level data from 52 countries, 1982–2001. Available at http://www.nber.org/papers/w9754. Accessed April, 2015.
47. Lichtenberg F. Benefits and Costs of Newer Drugs: An Update. National Bureau of Economic Research, Working Paper No. 8996. June 2007. Available at http://www.nber.org/papers/w8996.
Accessed April, 2015.
48. Pharmaceutical Research and Manufacturers of America (PhRMA). 2013 Profile: Biopharmaceutical Research Industry. Figure 9: The Ripple Effect of High-Value Biopharmaceutical Jobs.
Available at http://www.phrma.org/sites/default/files/pdf/PhRMA%20Profile%202013.pdf. Accessed April, 2015.
49. Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.
50. Justin Chakma, J, Sun, GH, Steinberg JD, et al. Asia’s Ascent—Global Trends in Biomedical R&D Expenditures. N Engl J Med. 2014; 370:3-6January 2, 2014DOI: 10.1056/NEJMp1311068.
51. Pharmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed April, 2015.
52. A merican Cancer Society. Cancer Treatment & Survivorship Facts & Figures 2014–2015. Available at
http://www.cancer.org/acs/groups/content/@research/documents/document/acspc-042801.pdf. Accessed April, 2015.
53. E stimate derived from the National Bureau of Economic Research. Sun EC, Jena AB, Lakdawalla DN, et al. An Economic Evaluation of the War on Cancer. NBER Working Paper No. 15574.
Issued in December 2009. Available at http://www.nber.org/papers/w15574. Accessed April, 2015. Methodology discussed at http://valueofinnovation.org/about-the-clock.html and
http://imgsrv.wben.com/image/wben2/UserFiles/File/Philipson%20FF.pdf. Lichtenberg, FR. Current estimates based on “Has Medical Innovation Reduced Cancer Mortality?” National
Bureau of Economic Research. Revised October, 2013.
54. Pharmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed April, 2015.
55. Duggan MG, Evans WN. Estimating the impact of medical innovation: A case study of HIV antiretroviral treatments. National Bureau of Economic Research. Working Paper 11109. Issued in
February 2005. Available at http://www.nber.org/digest/apr05/w11109.html. Accessed April, 2015. National Center for Health Statistics. “Health, United States, 2010: with Special Feature
on Death and Dying, Table 35.” Hyattsville, MD: NCHS, 2011. Available at www.cdc.gov/nchs/data/hus/hus10.pdf#045. Accessed April, 2015.
56. Pharmaceutical Research and Manufacturers of America (PhRMA). 2013 Profile: Biopharmaceutical Research Industry. Figure 9: The Ripple Effect of High-Value Biopharmaceutical Jobs.
Available at http://www.phrma.org/sites/default/files/pdf/PhRMA%20Profile%202013.pdf. Accessed April, 2015.
57. Tripp S and Grueber M. Economic impact of the human genome project. Available at http://battelle.org/docs/default-document-library/economic_impact_of_the_human_genome_project.pdf.
Accessed April, 2015.
58. Stewart BW and Wild CP. World Cancer Report 2014. World Health Organization. ISBN 978-92-832-0429-9.
59. Alzheimer’s Association. 2015 Alzheimer’s disease facts and figures. Available at https://www.alz.org/facts/downloads/facts_figures_2015.pdf. Accessed April, 2015.
60. Drug Discovery and Development: Understanding the R&D Process. Available at www.innovation.org. Accessed April, 2015.Congressional Budget Office, Research and Development in
the Pharmaceutical Industry, 2006. PhRMA. 2013 Profile Biopharmaceutical Research Industry. Available at http://www.phrma.org/sites/default/files/pdf/PhRMA%20Profile%202013.pdf.
Accessed April, 2015. Tufts Center for the Study of Drug Development. Cost to develop and win marketing approval for a new drug is $2.6 billion. Available at
http://csdd.tufts.edu/news/complete_story/pr_tufts_csdd_2014_cost_study. Accessed April, 2015.
61. Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.
62. Falconi M. Novartis chairman stresses need for R&D investment. The Wall Street Journal. Published March 22, 2014. Available at
http://www.wsj.com/articles/SB10001424052702303802104579453092985730948. Accessed April, 2015.
Chapter 1
1. REVLIMID Full Prescribing Information. Available at https://www.celgene.com/content/uploads/revlimid_full_prescribing_info.pdf. Accessed April, 2015.
2. POMALYST Full Prescribing Information. Available at http://www.pomalyst.com/wp-content/uploads/2013/08/prescribing_information.pdf. Accessed April, 2105.
3. European Medicines Agency. Annex 1 summary of product characteristics. Available at
http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000717/WC500056018.pdf. Accessed April, 2015.
4. Goldstein D et al. nab-Paclitaxel Plus Gemcitabine for Metastatic Pancreatic Cancer: Long-Term Survival From a Phase III Trial. J Natl Cancer Inst. (2015);107(2) pii: dju413. doi: 10.1093/
jnci/dju413. Print 2015 Feb.
5. Von Hoff DD et al. N Engl J Med. 2013;369:1691-1703.
6. OTEZLA Full Prescribing Information. Available at http://www.otezla.com/otezla-prescribing-information.pdf. Accessed April, 2015.
7. Celgene Q3 2014 Conference Call presentation. October 23, 2014. Available at
http://files.shareholder.com/downloads/AMDA-262QUJ/72150623x0x788116/4A1E9DC8-404E-4342-92F3-4EE84B851FC5/Q3_2014_EPS_Deck_-_FINAL_-_22Oct14.pdf. Accessed
April, 2015.
8. OTEZLA Full Prescribing Information. Available at http://www.otezla.com/otezla-prescribing-information.pdf. Accessed April, 2105.
9. Rutgers Cancer Institute of New Jersey. What is Precision Medicine?. Available at http://www.cinj.org/precision-medicine. Accessed, April 2015.
10. International Myeloma Foundation. The promise and challenges of “Personalized Medicine” for myeloma. Available at
http://myeloma.org/MtEntryPage.action?source=/imf_blogs/myeloma_voices/2012/04/the-promise-and-challenges-of-personalized-medicine-for-myeloma.html. Accessed April, 2015.
11. International Myeloma Foundation. The promise and challenges of “Personalized Medicine” for myeloma. Available at
http://myeloma.org/MtEntryPage.action?source=/imf_blogs/myeloma_voices/2012/04/the-promise-and-challenges-of-personalized-medicine-for-myeloma.html. Accessed April, 2015.
12. A merican Cancer Society. Cancer Treatment & Survivorship Facts & Figures 2014–2015. Available at
http://www.cancer.org/acs/groups/content/@research/documents/document/acspc-042801.pdf. Accessed April, 2015.
65
Living Longer, Better and Healthier Lives
13. E stimate derived from the National Bureau of Economic Research. Sun EC, Jena AB, Lakdawalla DN, et al. An Economic Evaluation of the War on Cancer. NBER Working Paper No. 15574.
Issued in December 2009. Available at http://www.nber.org/papers/w15574. Accessed April, 2015. Methodology discussed at http://valueofinnovation.org/about-the-clock.html and
http://imgsrv.wben.com/image/wben2/UserFiles/File/Philipson%20FF.pdf. Lichtenberg, FR. Current estimates based on “Has Medical Innovation Reduced Cancer Mortality?” National
Bureau of Economic Research. Revised October, 2013. Accessed April, 2015.
14. L ichtenberg FR. NBER Working Paper No. 18235. Pharmaceutical innovation and longevity growth in 30 developing and high-income countries, 2000–2009. Available at
http://www.nber.org/papers/w18235. Accessed April, 2015. Lichtenberg FR. NBER Working Paper No. 9754. The impact of new drug launches on longevity: evidence from longitudinal
disease-level data from 52 countries, 1982–2001. Available at http://www.nber.org/papers/w9754. Accessed April, 2015.
15. United Nations Development Program. World Population Prospects The 2010 Revision. Volume 1: Comprehensive Tables. Available at
http://esa.un.org/wpp/Documentation/pdf/WPP2010_Volume-I_Comprehensive-Tables.pdf. Accessed April, 2015. U.S. Food and Drug Administration. Centers for Disease Control and
Prevention and National Center for Health Statistics. Health, United States, 2011: With Special Feature on Socioeconomic Status and Health. Available at http://www.cdc.gov/nchs/index.htm.
Accessed April, 2015. Prentice T. Health, History and Hard Choices: Funding Dilemmas in a Fast-Changing World. Nonprofit and Voluntary Sector Quarterly. 2008: Supplement to vol 37;
no 1: 63S-75S.
16. The Hamilton Project at the Brookings Institution. Available at http://www.hamiltonproject.org/multimedia/charts/deaths_from_major_infectious_disease/. Accessed April, 2015.
17. A merican Cancer Society. Cancer Facts & Figures, 2014. Available at http://www.cancer.org/acs/groups/content/@research/documents/webcontent/acspc-042151.pdf. Accessed April, 2015.
18. Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.
19. The Hamilton Project at the Brookings Institution. Available at http://www.hamiltonproject.org/multimedia/charts/deaths_from_major_infectious_disease/. Accessed April, 2015.
20. D eVita VT , Chu E. A History of Cancer Chemotherapy. Cancer Res 2008;68:8643-8653. Accessed April, 2015. ©2008 by American Association for Cancer Research. U.S. Food and Drug
Administration, Therapeutic Biologic Applications (BLA). Available at
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/TherapeuticBiologicApplications/ucm080591.htm. Accessed April, 2015.
21. Kaittanis C, Santra S, Manuel PJ. Emerging nanotechnology-based strategies for the identification of microbial pathogenesis. Advanced Drug Delivery Reviews 62:408-423, 2010.
22. Filippini G1, Del Giovane C, Vacchi L. Immunomodulators and immunosuppressants for multiple sclerosis: a network meta-analysis. Cochrane Database Syst Rev. 6:CD008933, 2013 Jun 6.
23. U.S. Food and Drug Administration, Epigenetics and Select Biomarkers of Organ Toxicity. Available at
http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/ToxicologicalResearch/UCM419140.pdf. Accessed April, 2015.
24. Celgene Q3 2014 Conference Call presentation. October 23, 2014. Available at
http://files.shareholder.com/downloads/AMDA-262QUJ/72150623x0x788116/4A1E9DC8-404E-4342-92F3-4EE84B851FC5/Q3_2014_EPS_Deck_-_FINAL_-_22Oct14.pdf. Accessed
April, 2015.
25. A merican Cancer Society. What are the key statistics about multiple myeloma? Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-key-statistics.
Accessed April, 2015.
26. National Cancer Institute, Surveillance Epidemiology and End Results (SEER). Available at http://seer.cancer.gov/archive/csr/1975_2010. Accessed April, 2015.
27. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 18.8: Myeloma, 5-Year Relative and Period
Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_18_myeloma.pdf. Accessed April, 2015.
28. B ergsagel, P. Where We Were, Where We Are, Where We Are Going: Progress in Multiple Myeloma. ASCO 2014 Educational Book. Available at
http://meetinglibrary.asco.org/content/114000199-144. Accessed April, 2015.
29. Sadovsky R. Multiple myeloma: recognition and management. Am Fam Physician;59(7):1885-94.
30.National Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 18.8: Myeloma, 5-Year Relative and Period
Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_18_myeloma.pdf. Accessed April, 2015.
Bergsagel, P. Where We Were, Where We Are, Where We Are Going: Progress in Multiple Myeloma. ASCO 2014 Educational Book. Available at
http://meetinglibrary.asco.org/content/114000199-144. Accessed April, 2015.
31.Porter DL et al. Randomized, phase II dose optimization study of chimeric antigen receptor modified T cells directed against CD19 (CT019) in patients with relapsed, refractory CLL. Paper
and poster presented the 55th Annual Meeting and Exposition of the American Society of Hematology; 2013 Dec 7–10; New Orleans, LA.
32. Kyle, Robert. Multiple myeloma. Blood. 2008;111(6)
33. National Psoriasis Foundation. Statistics. Available at http://www.psoriasis.org/research/science-of-psoriasis/statistics. Accessed April, 2015.
34. Lebwohl MG, Bachelez H, Barker J et al. Patient perspectives in the management of psoriasis: Results from the population-based Multinational Assessment of Psoriasis and Psoriatic
Arthritis Survey. J Am Acad Dermatol. 2014;70(5):871-881.e30.
35. OTEZLA Full Prescribing Information. Available at http://www.otezla.com/otezla-prescribing-information.pdf. Accessed April, 2015.
36. OTEZLA Full Prescribing Information. Available at http://www.otezla.com/otezla-prescribing-information.pdf. Accessed April, 2015.
37. OTEZLA Full Prescribing Information. Available at http://www.otezla.com/otezla-prescribing-information.pdf. Accessed April, 2015.
38. Celgene data on file.
39. Iuga, A., McGuire, M. Adherence and health care Costs. Risk Manag Healthc Policy. 2014; 7: 35-44
40. Protecting patients from higher cost sharing. Available at https://www.celgene.com/protecting-patients-from-higher-cost-sharing/. Accessed April, 2015.
41. M
illiman, Inc. 2014 Individual Exchange Policies in Four States: An Early Look for Patients with Blood Cancer. Available at
http://us.milliman.com/uploadedFiles/insight/2014/four-states-individual-exchange-policies.pdf. Accessed April, 2015.
42 Milliman, Inc. 2014 Individual Exchange Policies in Four States: An Early Look for Patients with Blood Cancer. Available at
http://us.milliman.com/uploadedFiles/insight/2014/four-states-individual-exchange-policies.pdf. Accessed April, 2015.
43. Jacobs DB and Sommers BD. Using Drugs to Discriminate—Adverse Selection in the Insurance Marketplace. N Engl J Med. 2015; 372:399-402. January 29, 2015. DOI: 10.1056/
NEJMp1411376.
44. Project Innovation. Delivery of Innovation to Cancer Patients: Access, Communication and Coordination. Available at
https://projectinnovation.org/NPAF_Backgrounder_on_Access_Communication.pdf. Accessed April, 2015.
45. Pollack, A. As pills treat cancer, insurance lags behind. The New York Times. Published April 14, 2009. Available at http://www.nytimes.com/2009/04/15/business/15pill.html?_r=.
Accessed April, 2015.
46. Patients Equal Access Coalition. The patients equal access coalition applauds a new bill to improve access to oral cancer drugs (2013). Available at http://myeloma.org/pdfs/peac_05-02-13.pdf.
Accessed April, 2015.
47. U.S. Pain Foundation. What is step therapy/fail first. Available at http://uspainfoundation.org/advocacy-fail-first-step-therapy.html. Accessed April, 2015.
48. ABC news. Patients irate with insurers’ ‘fail first’ policy (2009, April 22). Available at http://abcnews.go.com/Health/PainManagement/story?id=7395636&page=3. Accessed April, 2015.
49. Patients Equal Access Coalition. Oral Chemotherapy Access Legislative Landscape—March 2015. Available at http://peac.myeloma.org/oral-chemo-access-map/. Accessed April, 2015.
50. Cap the Copy. About the Bills. Available at http://www.capthecopay.org/about-the-bills/. Accessed April, 2015.
51. HealthCare.gov. Out-of-pocket maximum/limit. Available at https://www.healthcare.gov/glossary/out-of-pocket-maximum-limit/. Accessed April, 2015.
52. S choonveld E. Impact of ACA on the Dinner-for-Three Dynamic. Clin Ther. 2015; 37(4); pp 733-746. Available at
http://www.clinicaltherapeutics.com/article/S0149-2918(15)00079-X/fulltext#s0030. Accessed May, 2015.
66
Living Longer, Better and Healthier Lives
53. Milliman, Inc. 2014 Individual Exchange Policies in Four States: An Early Look for Patients with Blood Cancer. Available at
http://us.milliman.com/uploadedFiles/insight/2014/four-states-individual-exchange-policies.pdf. Accessed April, 2015.
54. Patients Equal Access Coalition. Oral Chemotherapy Access Legislative Landscape—March 2015. Available at http://peac.myeloma.org/oral-chemo-access-map/. Accessed April, 2015.
55. Congress.gov. H.R.1801—Cancer Drug Coverage Parity Act of 2013. Available at https://www.congress.gov/bill/113th-congress/house-bill/1801. Accessed April, 2015.
56. Congress.gov. S.1879—Cancer Treatment Parity Act of 2013. Available at https://www.congress.gov/bill/113th-congress/senate-bill/1879. Accessed April, 2015.
Chapter 2
1. Pharmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed January, 2015.
2. Project Innovation. Medical Innovation Throughout History. Available at https://projectinnovation.org/timeline.php. Accessed April, 2015.
3. REVLIMID Full Prescribing Information. Available at https://www.celgene.com/content/uploads/revlimid_full_prescribing_info.pdf. Accessed April, 2015.
4. BBC News. People with psoriasis ‘shunned.’ Available at http://news.bbc.co.uk/2/hi/health/3958617.stm. Accessed April, 2015.
5. http://www.mds-foundation.org/pdf/handbook-english.pdf
6. REVLIMID. Summary of Product Characteristics. Available at http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000717/WC500056018.pdf.
Accessed April, 2015.
7. OTEZLA Full Prescribing Information. Available at http://www.otezla.com/otezla-prescribing-information.pdf. Accessed April, 2015.
8. National Psoriasis Foundation. Psoriatic Arthritis. Available at http://www.psoriasis.org/psoriatic-arthritis. Accessed April, 2015.
9. Medline Plus. Acute lymphoblastic leukemia (ALL). Available at http://www.nlm.nih.gov/medlineplus/ency/article/000541.htm. Accessed April, 2015.
10. A merican Cancer Society. Childhood Leukemia Survival Rates Improve Significantly. Available at http://www.cancer.org/cancer/news/childhood-leukemia-survival-rates-improve-significantly.
Accessed April, 2015.
Chapter 3
1. The Hamilton Project at the Brookings Institution. Available at http://www.hamiltonproject.org/multimedia/charts/deaths_from_major_infectious_disease/. Accessed April, 2015.
2. The Hamilton Project at the Brookings Institution. Available at http://www.hamiltonproject.org/multimedia/charts/deaths_from_major_infectious_disease/. Accessed April, 2015.
3.Lichtenberg F. Benefits and Costs of Newer Drugs: An Update. National Bureau of Economic Research, Working Paper No. 8996. June 2007. Available at http://www.nber.org/papers/w8996.
Accessed January, 2015.
4. Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.
5. A merican Cancer Society. Cancer Treatment and Survivorship Facts & Figures 2014–2015. http://www.cancer.org/acs/groups/content/@research/documents/document/acspc-042801.pdf.
Accessed April, 2015.
6. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2011. Table 2.8, All Cancer Sites (Invasive), 5-Year Relative
and Period Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/csr/1975_2011/results_single/sect_02_table.08.pdf. Accessed October, 2014.
7. A rias E. United States Life Tables, 2000. Table 12: Estimated life expectancy at birth in years, by race and sex: Death-registration States, 1900–28, and United States, 1929–2000. Natl
Vital Stat Rep 2002;51(3):1-38. Murphy SL, Xu J, Kochanek KD. Deaths: Final Data for 2010. Table 8: Life expectancy at birth, by race, Hispanic origin, race for non-Hispanic population,
and sex: United States, 1940, 1950, 1960, 1970, and 1975–2010. Natl Vital Stat Rep 2013;61(4):1-117. Hoyert DL, Xu J. Deaths: Preliminary Data for 2011. Table 6: Expectation of life at
selected ages, by race, Hispanic origin, race for non-Hispanic population, and sex: United States, final 2010 and preliminary 2011. Natl Vital Stat Rep 2012;61(6):1-51. Federal Reserve
Bank of St. Louis; FRED Economic Data. Real GDP per Capita in the United States. Available at http://research.stlouisfed.org/fred2/series/USARGDPC. Accessed April, 2015.
8. Lichtenberg F. Benefits and Costs of Newer Drugs: An Update. National Bureau of Economic Research, Working Paper No. 8996. June 2007. Available at http://www.nber.org/papers/w8996.
Accessed April, 2015.
9. Dunn A, Rittmueller R, Whitmire B. Introducing the new BEA health care satellite account (January 2015). Available at
http://www.bea.gov/scb/pdf/2015/01%20January/0115_bea_health_care_satellite_account.pdf. Accessed April, 2015.
10. Duggan MG, Evans WN. Estimating the impact of medical innovation: A case study of HIV antiretroviral treatments. National Bureau of Economic Research. Working Paper 11109. Issued in
February 2005. Available at http://www.nber.org/digest/apr05/w11109.html. Accessed April, 2015. National Center for Health Statistics. Health, United States, 2010: with Special Feature
on Death and Dying, Table 35. Hyattsville, MD: NCHS, 2011. Available at www.cdc.gov/nchs/data/hus/hus10.pdf#045. Accessed April, 2015.
11. U
.S. Department of Health & Human Services. Newly diagnosed: What you need to know. Available at
https://www.aids.gov/hiv-aids-basics/just-diagnosed-with-hiv-aids/overview/newly-diagnosed/. Accessed April, 2015.
12. A merican Cancer Society. Cancer Facts & Figures, 2014. Available at
http://www.cancer.org/acs/groups/content/@research/documents/webcontent/acspc-042151.pdf. Accessed April, 2015.
13. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2011. Table 2.8, All Cancer Sites (Invasive), 5-Year Relative
and Period Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/csr/1975_2011/results_single/sect_02_table.08.pdf. Accessed April, 2015.
14. P harmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed April, 2015.
15. Pharmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed January, 2015.
16. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 15.12: Cancer of the Lung and Bronchus
(Invasive), 5-Year Relative and Period Survival (Percent) by Race, Sex, Diagnosis Year, Stage and Age. Available at
http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_15_lung_bronchus.pdf. Accessed April, 2015.
17. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 15.12: Cancer of the Colon and Rectum, 5-Year
Relative and Period Survival (Percent) by Race, Sex, Diagnosis Year, Stage and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_06_colon_rectum.pdf.
Accessed October, 2015.
18. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 15.12: Cancer of the Prostate, 5-Year Relative
and Period Survival (Percent) by Race, Sex, Diagnosis Year, Stage and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_23_prostate.pdf. Accessed
April, 2015.
19. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 4.14: Cancer of the Female Breast (Invasive),
5-Year Relative and Period Survival (Percent) by Race, Diagnosis Year, Stage and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_04_breast.pdf.
Accessed October, 2014.
20. Lichtenberg F. Benefits and Costs of Newer Drugs: An Update. National Bureau of Economic Research, Working Paper No. 8996. June 2007. Available at http://www.nber.org/papers/w8996.
Accessed April, 2015.
67
Living Longer, Better and Healthier Lives
21. T he University of Texas MD Anderson Cancer Center. Leukemia Fact Sheet. Available at
http://www.mdanderson.org/patient-and-cancer-information/cancer-information/cancer-types/leukemia/index.html. Accessed April, 2015. Yin W, Penrod JR, Maclean JR, et al. Value of
survival gains in chronic myeloid leukemia. Am J Manag Care 2012;18 (11 Suppl):S257-64. LeBas J. Findings could alter chronic myelogenous leukemia treatment: Phase II trials show
newer drugs yield better responses than matinib. OncoLog 2010;55(8):4-5. Available at http://www2.mdanderson.org/depts/oncolog/articles/10/8-aug/8-10-2.html. Accessed April, 2015.
22. N ational Cancer Institute. Cancer Trends Progress Report—2005 Update. Table L1: National Cancer Treatment Expenditures in Billions of Dollars (1963–2004). Available at
http://www.progressreport.cancer.gov/sites/default/files/archive/report2005.pdf. Accessed April, 2015.
23. Cutler DM. Are we finally winning the war on cancer? J Econ Persp 2008:22(4);3-26.
24. M ariotto AB, Yabroff KR, Shao Y, et al. Projections of the cost of cancer care in the United States: 2010–2020. J Natl Cancer Inst 2011;103(2):117-128.
25. Centers for Disease Control and Prevention. Health, United States, 2012. Table 111. Gross domestic product, national health expenditures, per capita amounts, percent distribution,
and average annual percent change: United States, selected years 1960–2011. Available at http://www.cdc.gov/nchs/data/hus/2012/111.pdf. Accessed April, 2015.
26. N ational Cancer Institute. Cancer Trends Progress Report—2005 Update. Table L1: National Cancer Treatment Expenditures in Billions of Dollars (1963–2004). Available at
http://www.progressreport.cancer.gov/sites/default/files/archive/report2005.pdf. Accessed April, 2015.
27. Cutler DM. Are we finally winning the war on cancer? J Econ Persp 2008:22(4);3-26.
28. M ariotto AB, Yabroff KR, Shao Y, et al. Projections of the cost of cancer care in the United States: 2010–2020. J Natl Cancer Inst 2011;103(2):117-128.
29. Centers for Disease Control and Prevention. Health, United States, 2012. Table 111. Gross domestic product, national health expenditures, per capita amounts, percent distribution, and
average annual percent change: United States, selected years 1960–2011. Available at http://www.cdc.gov/nchs/data/hus/2012/111.pdf. Accessed April, 2015.
30. Milliman, Inc. 2014 Individual Exchange Policies in Four States: An Early Look for Patients with Blood Cancer. Available at
http://us.milliman.com/uploadedFiles/insight/2014/four-states-individual-exchange-policies.pdf. Accessed April, 2015.
31. M
illiman, Inc. 2014 Individual Exchange Policies in Four States: An Early Look for Patients with Blood Cancer. Available at
http://us.milliman.com/uploadedFiles/insight/2014/four-states-individual-exchange-policies.pdf. Accessed April, 2015.
32. Pharmaceutical Research and Manufacturers of America. PhRMA chart pack: cancer medicines value in context. Spring 2014. Chapter 1, slide 4. Available at
http://www.phrma.org/sites/default/files/pdf/cancer-chart-pack-5-22-14.pdf. Accessed April, 2015.
33. Porter ME. What is value in health care? N Engl J Med; 363:2477-2481.
34. IMS Institute. Innovation in cancer care and implications for health systems. Global oncology trend report. Available at
http://www.imshealth.com/vgn-ext-templating/v/index.jsp?vgnextoid=f8d4df7a5e8b5410VgnVCM10000076192ca2RCRD. Accessed April, 2015. Centers for Medicare and Medicaid
Services. National Health Expenditure Data. Available at https://www.cms.gov/. Accessed April, 2015. Cuckler GA,
Sisko AM, Keehan SP, et al. National health expenditure projections, 2012–22: slow growth until coverage expands and economy improves. Health Aff (Millwood) 2013;32(10):1820-1831.
35. Lichtenberg, F. Benefits and Costs of Newer Drugs: An Update. National Bureau of Economic Research, Working Paper No. 8996. June 2007. Available at http://www.nber.org/papers/w8996.
Accessed April, 2015.
36. P funtner A, Wier L, Steiner C. Costs for hospital stays in the United States, 2011 (December 2013). Available at
http://www.hcup-us.ahrq.gov/reports/statbriefs/sb168-Hospital-Costs-United-States-2011.jsp. Accessed April, 2015.
37. M y Life is Worth It. MY LIFE IS WORTH IT Calls for Balanced View of the Value of Advanced Therapeutics for Cancer Patients at Global Hematology Conference. Available at
http://www.businesswire.com/news/home/20141203005328/en/LIFE-WORTH-Calls-Balanced-View-Advanced-Therapeutics#.VTavpFxEgzI. Accessed April, 2015.
38. N ational Cancer Institute. Cancer Trends Progress Report—2005 Update. Table L1: National Cancer Treatment Expenditures in Billions of Dollars (1963–2004). Available at
http://www.progressreport.cancer.gov/sites/default/files/archive/report2005.pdf. Accessed April, 2015.
39. Cutler DM. Are we finally winning the war on cancer? J Econ Persp 2008:22(4);3-26.
40. Mariotto AB, Yabroff KR, Shao Y, et al. Projections of the cost of cancer care in the United States: 2010–2020. J Natl Cancer Inst 2011;103(2):117-128.
enters for Disease Control and Prevention. Health, United States, 2012. Table 111. Gross domestic product, national health expenditures, per capita amounts, percent distribution, and
41. C
average annual percent change: United States, selected years 1960–2011. Available at http://www.cdc.gov/nchs/data/hus/2012/111.pdf. Accessed April, 2015.
42. Centers for Medicare & Medicaid Services. Available at
http://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-and-Reports/NationalHealthExpendData/Downloads/tables.pdf. Accessed April, 2015.
43. Centers for Medicare and Medicaid Services. National Health Expenditures; Aggregate and Per Capita Amounts, Annual Percent Change and Percent Distribution: Selected Calendar Years
1960–2012. Available at
http://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-and-Reports/NationalHealthExpendData/Downloads/tables.pdf. Accessed April, 2015.
44. E stimate derived from the National Bureau of Economic Research. Sun EC, Jena AB, Lakdawalla DN, et al. An Economic Evaluation of the War on Cancer. NBER Working Paper No. 15574.
Issued in December 2009. Available at http://www.nber.org/papers/w15574. Accessed April, 2015. Methodology discussed at http://valueofinnovation.org/about-the-clock.html and
http://imgsrv.wben.com/image/wben2/UserFiles/File/Philipson%20FF.pdf. Lichtenberg, FR. Current estimates based on “Has Medical Innovation Reduced Cancer Mortality?” National
Bureau of Economic Research. Revised October, 2013. Accessed April, 2015.
45. Centers for Disease Control and Prevention and National Center for Health Statistics. Health, United States, 2011. Table 22: Life expectancy at birth, at age 65, and at age 75, by sex, race,
and Hispanic origin: United States, selected years 1900–2010. Available at http://www.cdc.gov/nchs/data/hus/2011/022.pdf. Accessed April, 2015. Centers for Disease Control and
Prevention and National Center for Health Statistics. Health, United States, 2011. Table 24: Age-adjusted death rates for selected causes of death, by sex, race, and Hispanic origin:
United States, selected years 1950–2010. Available at http://www.cdc.gov/nchs/data/hus/2011/024.pdf. Accessed April, 2015.
46. E stimate derived from the National Bureau of Economic Research. Sun EC, Jena AB, Lakdawalla DN, et al. An Economic Evaluation of the War on Cancer. NBER Working Paper No. 15574.
Issued in December 2009. Available at http://www.nber.org/papers/w15574. Accessed April, 2015. Methodology discussed at http://valueofinnovation.org/about-the-clock.html and
http://imgsrv.wben.com/image/wben2/UserFiles/File/Philipson%20FF.pdf. Lichtenberg, FR. Current estimates based on “Has Medical Innovation Reduced Cancer Mortality?” National
Bureau of Economic Research. Revised October, 2013. Accessed April, 2015.
47. McKinsey. Accounting for the Cost of U.S. Health Care: A New Look at Why Americans Spend More. Available at
http://www.mckinsey.com/insights/health_systems_and_services/accounting_for_the_cost_of_us_health_care. Accessed April, 2015.
48. Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.
49. Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.
50. IMS Institute. Innovation in cancer care and implications for health systems. Global oncology trend report. Available at
http://www.imshealth.com/vgn-ext-templating/v/index.jsp?vgnextoid=f8d4df7a5e8b5410VgnVCM10000076192ca2RCRD. Accessed April, 2015. Centers for Medicare and Medicaid
Services. National Health Expenditure Data. Available at https://www.cms.gov/. Accessed April, 2015. Cuckler GA,
Sisko AM, Keehan SP, et al. National health expenditure projections, 2012–22: slow growth until coverage expands and economy improves. Health Aff (Millwood) 2013;32(10):1820-1831.
51. A rmstrong, A, Schupp, C, Wu, J, et al. Quality of Life and Work Productivity Impairment Among Psoriasis Patients: Findings from the National Psoriasis Foundation Survey Data 2003–
2011. PLoS One. 2012; 7(12): e52935.
68
Living Longer, Better and Healthier Lives
52. European Federation of Crohn’s and Ulcerative Colitis Associations (EFCCA). The True Impact of IBD: A European Crohn’s and Ulcerative Colitis Patient Life: IMPACT Survey 2010–2011.
Available at http://efcca-solutions.net/media/jointhefight/ImpactReport.pdf. Accessed April, 2015.
53. Moral R, Rillo O, Casalla L, et al. Work Productivity in Rheumatoid Arthritis: Relationship with Clinical and Radiological Features. Arthritis. 2012; Article ID 137635.
54. Kennedy M, Papneja A, Thavaneswaran A, et al. Prevalence and Predictors of Reduced Work Productivity in Patients with Psoriatic Arthritis. Clinical and Experimental Rheumatology. 2014;
32(3): 342-8.
55. Verstappen S, Watson K, Lunt M, et al. Working Status in Patients with Rheumatoid Arthritis, Ankylosing Spondylitis and Psoriatic Arthritis: Results from the British Society for
Rheumatology Biologics Register. Rheumatology. 2010; 49(8): 1570‐1577.
56. Tillett W, de‐Vries C, McHugh N. Work Disability in Psoriatic Arthritis: A Systematic Review. Rheumatology. 2012; 51(2): 275‐283.
57 European Federation of Crohn’s and Ulcerative Colitis Associations (EFCCA). The True Impact of IBD: A European Crohn’s and Ulcerative Colitis Patient Life: IMPACT Survey 2010–2011.
Available at http://efcca-solutions.net/media/jointhefight/ImpactReport.pdf. Accessed April, 2015.
58. Moral, R., Rillo, O., Casalla, L., et al. Work Productivity in Rheumatoid Arthritis: Relationship with Clinical and Radiological Features. Arthritis. 2012; Article ID 137635.
59. A merican Autoimmune Related Diseases Association (AARDA). The Cost Burden of Autoimmune Disease: The Latest Front in the War on Healthcare Spending. Available at
http://www.diabetesed.net/page/_files/autoimmune-diseases.pdf. Accessed April, 2015.
60. Cortesi, P., Scalone, L., D’Angiolella, L., et al. Systematic Literature Review on Economic Implications and Pharmacoeconomic Issues of Psoriatic Arthritis. Clinical and Experimental
Rheumatology. 2012; 30(73): S126-S131.
61. United States Census Bureau. U.S. and World Population Clock. Available at https://www.census.gov/popclock/. Accessed April, 2015.
62. K avanaugh A, Antoni C, Mease P, et al. Effect of infliximab therapy on employment, time lost from work, and productivity in patients with psoriatic arthritis. J Rheumatol. 2006; 33(11):
2254–2259.
63. Lebwohl M, Bachelez H, Barker J, et al. Patient Perspectives in the Management of Psoriasis: Results from the Population-Based Multinational Assessment of Psoriasis and Psoriatic
Arthritis. J Am Acad of Dermatol. 2014; 70(5): 871-881.e30
64. A rmstrong, A., Schupp, C., Wu, J., et al. Quality of Life and Work Productivity Impairment Among Psoriasis Patients: Findings from the National Psoriasis Foundation Survey Data 2003–
2011. PLoS One. 2012; 7(12): e52935.
Chapter 4
1. P harmaceutical Research and Manufacturers of America. PhRMA Chart Pack: Biopharmaceuticals in Perspective. Spring 2013. Version 3.0. Chapter 6, Slide 75. Available at
http://www.phrma.org/sites/default/files/pdf/CHART%20PACK_online%20version_13APR04_forweb.pdf. Accessed October, 2014.
2. Falconi M. Novartis chairman stresses need for R&D investment. The Wall Street Journal. Published March 22, 2014. Available at
http://www.wsj.com/articles/SB10001424052702303802104579453092985730948. Accessed April, 2015.
3. Centers for Disease Control and Prevention (CDC). Health, United States, 2012. Table 28: Death Rates for Malignant Neoplasms, by Sex, Race, Hispanic origin, and Age: United States,
selected years 1950–2010. Available at http://www.cdc.gov/nchs/hus/contents2012.htm#028. Accessed March, 2015. National Cancer Institute, Surveillance, Epidemiology, and End
Results (SEER) Program. 5-Year Relative Survival by Year Dx, By Cancer Site, All Ages, All Races, Both Sexes, 1975–2006. Available at
http://seer.cancer.gov/archive/csr/1975_2010/browse_csr.php?sectionSEL=2&pageSEL=sect_02_table.09.html. Accessed March, 2015.
4. Duggan MG, Evans WN. Estimating the Impact of Medical Innovation: A Case Study of HIV Antiretroviral Treatments. National Bureau of Economic Research. Working Paper 11109. Issued
in February 2005. Available at http://www.nber.org/digest/apr05/w11109.html. Accessed October, 2014. National Center for Health Statistics. “Health, United States, 2010: with Special
Feature on Death and Dying, table 35.” Hyattsville, MD: NCHS, 2011. Available at www.cdc.gov/nchs/data/hus/hus10.pdf#045. Accessed September, 2014
5. Institute for Health Metrics and Evaluation. Life Expectancy Increases Globally as Death toll Falls From Major Diseases. Available at
http://www.healthdata.org/news-release/life-expectancy-increases-globally-death-toll-falls-major-diseases. Accessed April, 2015.
6. A merican Cancer Society. Cancer Treatment & Survivorship Facts & Figures 2014–2015. Available at
http://www.cancer.org/acs/groups/content/@research/documents/document/acspc-042801.pdf. Accessed April, 2015.
7. T he Hamilton Project at the Brookings Institution. Available at http://www.hamiltonproject.org/multimedia/charts/deaths_from_major_infectious_disease/. Accessed April, 2015.
8. Lichtenberg F. Benefits and Costs of Newer Drugs: An Update. National Bureau of Economic Research, Working Paper No. 8996. June 2007. Available at http://www.nber.org/papers/w8996.
Accessed January, 2015.
9. Cancer Treatment and Survivorship Facts & Figures. Available at http://www.cancer.org/acs/groups/content/@research/documents/document/acspc-042801.pdf. Accessed April, 2015.
United States Centers for Disease Control and Prevention. Cancer Survivorship—United States, 1971–2001. Available at http://www.cdc.gov/mmwr/preview/mmwrhtml/mm5324a3.htm.
Accessed April, 2015. Center for Disease Control and Prevention. Cancer Survivors. United States, 2007. Center Watch. Available at
http://www.cdc.gov/mmwr/preview/mmwrhtml/mm6009a1.htm. Accessed April, 2015. Cancer Watch. FDA Approved Drugs by Therapeutic Area. Available at
https://www.centerwatch.com/drug-information/fda-approved-drugs/therapeutic-area/12/oncology. Accessed April, 2015.
10. Lucarelli C and Nicholson S. A Quality-Adjusted Price Index for Colorectal Cancer Drugs: The National Bureau of Economics; NBER Working Paper No. 15174; July 2009.
11. A nalysis Group. Innovation in the Biopharmaceutical Pipeline: A Multidimensional View (January 2013). Available at
http://www.analysisgroup.com/uploadedFiles/Publishing/Articles/2012_Innovation_in_the_Biopharmaceutical_Pipeline.pdf. Accessed April, 2015.
12. U.S. Food and Drug Administration. Novel New Drugs 2013 summary. Available at http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DrugInnovation/UCM381803.pdf.
Accessed April, 2015.
13. Monteleone G, Neurath M, Ardizzone S et al. Mongersen, an Oral SMAD7 Antisense Oligonucleotide, and Crohn’s Disease. N Engl J Med 2015; 372:1104-1113.
14. Yu D, Waterland R and Zhang P. Targeted p16 Ink4a Epimutation causes tumorigenesis and Reduces Survival in Mice. J Clin Invest 2014; 124(9):3708-3712.
15. Topol E. The Creative Destruction of Medicine: How the Digital Revolution Will Create Better Health Care. 2013. Basic books. ISBN: 978-0465061839.
16. FDA News Release. FDA Approves New Treatment for Myelodysplastic Syndrome. Available at http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/2005/ucm108546.htm.
Accessed April, 2015.
17. R ajkumar S, Jacobus S, Callander N, et al. Lenalidomode Plus High-Dose Dexamethasone Versus Lenalidomide Plus Low-Dose Dexamethasone as Initial Therapy for Newly Diagnosed
Multiple Myeloma: An Open-Label Randomized Controlled Trial. Lancet Oncol 2010; 11(1):29-37.
18. B enboubker L, Dimopoulos M, Dispenzieri A, et al. Lenalidomide and Dexamethasone in Transplant-Ineligible Patients with Myeloma. N Engl J Med 2014; 371:906-917.
19. REVLIMID Full Prescribing Information. Available at https://www.celgene.com/content/uploads/revlimid_full_prescribing_info.pdf. Accessed April, 2015.
20. Revlimid. Summary of Product Characteristics. Available at http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000717/WC500056018.pdf.
Accessed April, 2015.
21. REVLIMID Full Prescribing Information. Available at https://www.celgene.com/content/uploads/revlimid_full_prescribing_info.pdf. Accessed April, 2015.
22. Revlimid. Summary of Product Characteristics. Available at http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000717/WC500056018.pdf.
Accessed April, 2015.
69
Living Longer, Better and Healthier Lives
23. N
ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 18.8: Myeloma, 5-Year Relative and Period
Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_18_myeloma.pdf. Accessed April, 2015.
National Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 18.8: Hodgkin Lymphoma, 5-Year Relative and
Period Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_09_hodgkins.pdf. Accessed April, 2015.
National Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 19.8: Non-Hodgkin Lymphoma, 5-Year Relative
and Period Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_19_nhl.pdf. Accessed April, 2015.
National Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 19.8: Leukemia, 5-Year Relative and Period
Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_13_leukemia.pdf. Accessed April, 2015.
24. Celgene data on file.
25. Döhner H et al. Overall Survival in Older Patients with Newly Diagnosed Acute Myeloid Leukemia (AML) with >30% Bone Marrow Blasts Treated with Azacitidine by Cytogenetic Risk
Status: Results of the AZA-AML-001 Study. Presented at the 56th American Society of Hematology Meeting and Exposition; 2014 Dec 6–9; San Francisco, CA.
26. A BRAXANE Full Prescribing Information. Available at http://www.abraxane.com/downloads/Abraxane_PrescribingInformation.pdf. Accessed April, 2015.
27. C
elgene data on file.
28. Green M, Manikhas G, Orlov S, et al. Abraxane, a Novel Cremophor-Free, Albumin-Bound Particle form of Paclitaxel for the Treatment of Advanced Non-Small-Cell Lung Cancer.
Ann Oncol 2006; 17(8):1263-1268.
29. A BRAXANE Full Prescribing Information. Available at http://www.abraxane.com/downloads/Abraxane_PrescribingInformation.pdf. Accessed April, 2015.
30. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 15.12: Cancer of the Lung and Bronchus
(Invasive), 5-Year Relative and Period Survival (Percent) by Race, Sex, Diagnosis Year, Stage and Age. Available at
http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_15_lung_bronchus.pdf. Accessed April, 2015.
31. Von Hoff DD, Ervin T, Arena FP, et al. Increased Survival in Pancreatic Cancer with Nab-Paclitaxel Plus Gemcitabine. N Engl J Med; 368(18):1691-703.
32. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 22.8: Cancer of the Pancreas (Invasive), 5-Year
Relative and Period Survival (Percent) by Race, Sex, Diagnosis Year, Stage and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_22_pancreas.pdf.
Accessed April, 2015.
33. Untch, M., Jackisch, C., Schneeweiß, A., Conrad, B., et al. A Randomized Phase III Trial Comparing Neoadjuvant Chemotherapy with Weekly Nanoparticle-Based Paclitaxel with
Solventbased Paclitaxel Followed by Anthracyline/Cyclophosphamide for Patients with Early Breast Cancer (GeparSepto); GBG 69. S2-07.
34. N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2010. Table 4.14: Cancer of the Female Breast (Invasive),
5-Year Relative and Period Survival (Percent) by Race, Diagnosis Year, Stage and Age. Available at http://seer.cancer.gov/archive/csr/1975_2010/results_merged/sect_04_breast.pdf.
Accessed April, 2015.
35. Celgene data on file.
36. C
elgene data on file.
37. Celgene data on file.
38. Wagner R.W. and Flanagan, W.M. (1997) Antisense Technology and Prospects for Therapy of Viral Infections and Cancer. Mol. Med. Today, 3, 31–38.
39. Celgene. Phase II Data for Celgene’s Investigational Oral GED-0301 for Patients with Active Crohn’s Disease Published in New England Journal of Medicine. Available at
http://ir.celgene.com/releasedetail.cfm?releaseid=902361. Accessed April, 2015.
40. Lupus Foundation of America. Statistics on Lupus. Available at http://www.lupus.org/about/statistics-on-lupus. Accessed April, 2015.
41. Celgene data on file.
Chapter 5
1. A merican Cancer Society. Annual Report to the Nation: Cancer Death Rates Still Dropping. Available at
http://www.cancer.org/cancer/news/news/annualreport-to-the-nation-cancer-death-rates-still-dropping. Accessed April, 2015.
2. E stimate and projection of reduction of 96 million life years lost to cancer projects linear decline based on previous decade long change. Soerjomataram I, Lortet-Tieulent J, Parkin DM,
et al. Global burden of cancer in 2008: a systematic analysis of disability-adjusted life-years in 12 world regions. Lancet 2012;380(9856):1840-1850.
3. E stimate and Projection of Reduction of 96 Million Life Years Lost to Cancer Projects Linear Decline Based on Previous Decade Long Change. Soerjomataram I., Lortet-Tieulent, J., Parkin,
D, et al. Global Burden of Cancer in 2008: A Systematic Analysis of Disability-Adjusted Life-Years in 12 World Regions. Lancet. 2012;380(9856):1840-1850.
4. R amers-Verhoeven, C., Geipel, G., Howie, M. New Insights into Public Perceptions of Cancer. Ecancer. 2013. 7. doi: 10.3332/ecancer.2013.349.
5. Murphy KM and Topel RH. The Value of Health and Longevity. J Political Econ 2006: 114; 5; 871-904.
6. Adams D. Patients Today Want To Be More Involved In Care. American Medical News. Published October 22, 2007. Available at:
http://www.amednews.com/article/20071022/profession/310229955/7/. Accessed April, 2015.
7. R amers-Verhoeven C, Geipel G, and Howie M. New Insights into Public Perceptions of Cancer. Published online September 10, 2013. Available at:
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3766630/. Accessed April, 2015.
8. Milken Institute. The Global Biomedical Industry: Preserving U.S. Leadership. Published September 22, 2011. Available at: http://www.milkeninstitute.org/publications/view/476.
Accessed April, 2015.
9. Celgene data on file.
10. Hide G. History of Sleeping Sickness in East Africa. Clin Microbiol Rev. 1999; 12(1):112-125.
11. D
rugs for Neglected Diseases Initiative. Chagas Disease Global View. Available at: http://www.dndi.org/diseases-projects/diseases/chagas.html. Accessed April, 2015.
12 Drugs for Neglected Diseases Initiative. Leishmaniasis Global View. Available at: http://www.dndi.org/diseases-projects/diseases/vl.html. Accessed April, 2015.
13 Project Data Sphere. Available at https://www.projectdatasphere.org/projectdatasphere/html/home.html. Accessed April, 2015.
14 N ational Cancer Institute, Surveillance, Epidemiology, and End Results (SEER) Program. SEER Cancer Statistics Review, 1975–2011. Table 2.8, All Cancer Sites (Invasive), 5-Year Relative
and Period Survival (Percent) by Race, Sex, Diagnosis Year and Age. Available at http://seer.cancer.gov/csr/1975_2011/results_single/sect_02_table.08.pdf. Accessed October, 2014.
15. De Moor J, Mariotto A, Parry C, et al. Cancer Survivors in the United States: Prevalence across the Survivorship Trajectory and Implications for Care. Cancer Epidemiol Biomarkers Prev
2013; 22: 561.
16. N ational Cancer Institute. Surveillance Epidemiology and End Results (SEER). Available at http://seer.cancer.gov/archive/csr/1975_2010. Accessed April, 2015.
17. S tewart, B.W., Wild, C.P., editors (2014). World Cancer Report 2014. Lyon, France: International Agency for Research on Cancer.
18. World Cancer Research Fund International. Worldwide Data. Available at: http://www.wcrf.org/int/cancer-facts-figures/worldwide-data. Accessed April, 2015.
19. K handelwal, N., Dunacan, I., Ahmed, T., et al. Impact of Clinical Oral Chemotherapy Program on Wastage and Hospitalizations. J Oncol Prac 2011; 7(3s):e25s-e29s.
20. Continuous Lenalidomide and Low-dose Dexamethasone Demonstrates a Significant PFS and OS Advantage in Transplant Ineligible NDMM Patients—The FIRST: MM020/IFM0701 Trial.
Available at http://web.oncoletter.ch/files/cto_layout/Kongressdateien/WCLC13/Facon%20New.pdf. Accessed April, 2015.
70
Five Pillars of Celgene
Responsibility
Celgene Responsibility, which incorporates Patients & Communities, Safety, Governance, Global Health, and Environment
& Sustainability, defines who we are and ensures that we continue to provide and maximize opportunities for our patients,
our partners, our employees and our planet.
Patients & Communities:
Developing products and providing support that make a positive impact on
patients, communities and the world.
Commitment to Safety:
Pioneering the standard for patient and employee safety.
Governance:
Proactively implementing responsible business principles
and practices.
Global Health:
Delivering our promise to put patients first through the pursuit of innovative
solutions to healthcare challenges in low-income settings.
Environment & Sustainability:
Promoting environmentally responsible, safe and sustainable business
practices in our day-to-day operations.
Learn more: https://www.celgene.com/responsibility/
Celgene Corporation
86 Morris Avenue
Summit, NJ 07901
+1-908-673-9000
Celgene International
Switzerland—Boudry
International Headquarters
Route de Perreux 1 Boudry
CH-2017 Switzerland
+41 32 729 85 00
Twitter
LinkedIn
Google Plus
YouTube
Pinterest