Download Managing Participant and Patient Expectations

The Evolving Role of Patients in
the R&D Ecosystem
February 27, 2014
Margaret Anderson
Executive Director, FasterCures
About FasterCures
We are an action tank that works to
improve and speed up the time it takes
to get important new medicines from
discovery to patients.
Our name. Our mission.
Bringing a new discovery from lab to market
is a long, expensive and risky road
What is venture philanthropy?
Find Big Ideas in Small Spaces
• Loose affinity network of 56
nonprofit disease research
foundations
• Created to tackle the challenges
that cut across diseases through
innovative partnerships
• Connected through TRAIN
Central Station, an open-source
web platform:
www. fastercures.org/train
TRAIN Inventory
Thought leaders – including those in the venture capital community and media –
have turned to this inventory as a resource to provide them with an in-depth
understanding of current trends and practices in the venture philanthropy space.
TRAIN groups’ business models catalyzing
progress; draw attention, recognition
“…patient groups with
an entrepreneurial bent
have become the drug
industry’s
new power brokers.”
- Forbes
…Frustrated with the sluggishness, or
nonexistence, of medical research…a small band
of wealthy parents whose children have serious
illnesses are spending millions of dollars to fund
drug development...The principles they apply in
their jobs -- managing complicated tasks,
making investments and expecting positive
results -- translate to their new endeavors.
09/07/11
When Nonprofits Drive For-Profits
“At a time when so many companies
are starving for cash to advance their
best drug R&D ideas, many nonprofit
foundations know they can make an
impact by cleverly applying their own
blend of money, networks of patients,
and sincere advocacy.”
Increasingly powerful force in earlystage research
“They required us to lay out the
vision for the pre-clinical and
early clinical development… Most
importantly, they were able to
view the program with a critical
eye and provide meaningful
feedback.”
Although the funding models may
differ from organization to
organization, virtually everyone
agrees that the ties between
biotech industry and nonprofit
patient-centered organizations
will continue to strengthen.
WSJ 9-chapter multimedia special captures
the spirit of patient-driven R&D
Venture Philanthropy in Medical Research
Fund novel,
high-risk
research
Have strong
scientific
expertise
Bridge
disciplines,
institutions,
& ideas
Trusted by
and have
access to
patients
Patient Foundations are TRUSTED
by and have ACCESS to patients
Fund novel,
high-risk
research
Strong
scientific
expertise
Bridge
discipline,
institutions
& ideas
Trusted by
and have
access to
patients
• Strategic players in R&D in their disease areas
• Creating assets and capabilities that can be used to develop
evidence for regulatory approval and to demonstrate value to
payers.
•
•
•
•
clinical care and research networks
patient registries and tissue repositories
social networks
in some cases, pharmacy and care management services
• Form a unified advocacy front on key public policy issues
affecting the pace of research.
Foundations as “de-riskers”
 Developing pre-clinical tools
 Targeting research to support translation
 Funding mechanisms that bring in industry
 Managing academic science
 Access to patient community
 New indications for existing drugs
 High-throughput screening of promising compounds
 Access to scientific expertise
 Advocating with FDA
 Early-stage investors and/or “talent scouts”
“Not your grandmother’s charity”
• Make both investigator-initiated and directed grants
• Manage grants more actively to maximize efficiency and chances of
success
• Have internal scientific and/or industry expertise
• Provide access to patient resources via registries, tissue banks,
clinical care/research networks
• Mine academic grant portfolios for promising discoveries ready for
translation
• Provide resources and relationships to aid in translation to clinical
research and hand-off to industry
• Consistency and focus to push research projects along
Venture philanthropy impact
• Approval of 6 new
multiple myeloma
drugs
• World’s first and
largest myeloma
tissue bank
• Full sequencing of the
myeloma genome
• Opened 45 clinical
trials, accelerating 6
treatments to Phase III
• Personalized medicine
initiative includes
landmark trial that will
closely monitor 1,000
patients over 5 years-
• Funded $400 million in
research since 2000,
90% for translational
research
• Funded testing of over
100 therapeutic
targets for Parkinson’s
Disease
• Creating and sharing
research tools
• Leading the
development of a PD
biomarker
• Partnership with Elan
Corp. gave them first
look at promising
projects
• >$62M to 415 drug
research programs in
academia and
biotechnology in 18
countries
• >$8.5M to 40
companies with an
average investment of
$250K, including 4
successful start-ups
through tech transfer
• Funded programs
attracted hundreds of
millions of dollars in
follow-on funding
from government
grants to IPOs
• Funded more than $1
billion in research since
2005
• Actively supports more
than 22 new
treatments in
development – 10 FDAapproved
• Partners include
Vertex, Pfizer, PTC
Therapeutics, Genzyme
• 40-year-old patient
registry
• 74 accredited care
centers conduct trials
Honest Brokers for Cures
…Frustrated with the sluggishness, or nonexistence, of
medical research…a small band of wealthy parents
whose children have serious illnesses are spending
millions of dollars to fund drug development...The
principles they apply in their jobs -- managing
complicated tasks, making investments and expecting
positive results -- translate to their new endeavors.
09/07/11
•
•
•
•
In-house scientific expertise
Smart business strategies
Innovative financing models
Leadership & accountability
Patient Advocacy’s Impact
At the corner of…
Impatient Patients:
POWERFUL PARTNERS
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