Download CSL Behring Key Issues Dialogue – Hereditary Angioedema in the US

Key Issues Dialogue: HAE in the US
Featuring Anthony Castaldo, Dr. Bruce Zuraw, Dr. Marc Riedl,
Dr. H. Henry Li, Janet Long, Dr. Garrett Bergman
and Robert Lefebvre
Biotherapies for Life™
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Key Issues Dialogue: HAE in the US
Featuring Anthony Castaldo, Dr. Bruce Zuraw, Dr. Marc Riedl,
Dr. H. Henry Li, Janet Long, Dr. Garrett Bergman and Robert Lefebvre
This discussion is sponsored by
Diagnosis—and Misdiagnosis
CSL Behring for patients,
caregivers and healthcare
providers in the hereditary
angioedema community.
D R . G A R R E T T B E R G M A N : Hereditary angioedema is increasing in recognition
and visibility, but it is often misdiagnosed. Why is this?
D R . H E N R Y L I : There are several reasons for the misdiagnosis. Number one,
hereditary angioedema (HAE) is uncommon. Many physicians, even allergists who are
the primary specialists taking care of this disease, may not see a single case in their
whole career. That’s the first hurdle. Number two, while C1 esterase inhibitor has been
available for more than 30 years in Europe, until now there have not been a lot of
treatment options in the US. Many patients had not been actively seeking medical help
because physicians did not have effective treatment to offer to them. Therefore, the
disease is not adequately recognized and properly diagnosed.
D R . M A R C R I E D L : Actually angioedema as a symptom is not uncommon. However,
the vast majority of cases with symptoms of angioedema are not due to hereditary
angioedema. As Henry said, it’s an uncommon condition. So while physicians are used
to seeing angioedema, they’re not used to thinking of this rare disease. Often they’ll
consider it a histamine-mediated condition and treat it with antihistamines and maybe
steroids, which we know don’t work in hereditary angioedema.
D R . B R U C E Z U R A W : The problem is that physicians often do not recognize C1
esterase inhibitor deficiency or hereditary angioedema. But the fact of the matter is for
the average physician and non-specialist out there, there’s a very simple screening test
that will largely answer the question. It’s inexpensive. It’s easy to obtain. So if we just
simply thought of the problem as misdiagnosis, we would solve this. But another
problem is the long gap that can occur—often many years—between symptom
and diagnosis.
J A N E T L O N G : HAE is difficult to put into a neat box. The location of swelling and
severity of pain associated with HAE has a broad range that contributes to delaying
diagnosis.
G A R R E T T: Are people with hereditary angioedema ever accused of faking the
illness when their attack affects only their internal organs?
DR. H. HENRY LI
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B R U C E : All the time. Some of the patients who have come to me have come
through psychiatry where they were thought to have some type of functional rather
than physiologic complaint. No doubt the emotional stressors in life affect the disease
symptoms, but we know now that there’s a molecular and biochemical basis for
this disease.
A N T H O N Y C A S TA L D O : In addition, we often hear that patients with acute
abdominal pain are labeled as drug seekers because physicians at the ER or urgent
care facility can’t figure out the cause of the symptoms.
G A R R E T T: In those emergency room circumstances, do patients sometimes have
unnecessary surgery?
J A N E T: That was my own experience. I went to a hospital with an abdominal attack
that was extremely severe and underwent an exploratory laparotomy, which revealed
nothing abnormal.
M A R C : Unfortunately that’s a common story—patients having one or multiple
surgical procedures and the physicians finding nothing wrong. Not only do the
patients undergo unnecessary procedures, but they are also misdiagnosed with other
conditions. Quite commonly patients are told that they have multiple food allergies or
multiple medication allergies and that’s the reason that they’re experiencing these
symptoms of peripheral or intestinal angioedema. So there’s a pattern where patients
are continually misdiagnosed, which causes other complications in their medical care
and their lives.
H E N R Y: Often patients rate the pain of the abdominal attack as an 8 or 9 on a scale
DR. BRUCE ZURAW
of zero to 10. That’s very disabling and very severe. Without any peripheral swelling,
angioedema is hard to correctly diagnose. It is not uncommon those attacks with
severe abdominal pain are misdiagnosed or handled as an abdominal emergency.
B R U C E : I would say it’s the norm rather than the exception to have patients getting
unnecessary surgery.
A N T H O N Y: Data we have gathered from members of the US HAE Association
confirms a high incidence of unnecessary surgery and points to the urgent need for
creating greater awareness of HAE among physicians as well as patients who have
symptoms that include swelling and abdominal pain.
Bringing New Therapies to Market—and Patient Advocacy
G A R R E T T: Appropriate diagnosis and treatment can certainly reduce the effect
that this condition has on people’s day-to-day lives. With that, what were some of
the barriers and difficulties industry faced bringing C1 esterase inhibitor to market
in the US?
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R O B E R T L E F E B V R E : Working with the FDA, there are many steps that must be
taken to bring such products to market. As with other rare diseases, it takes time to
identify patients for clinical studies and more time to observe the effects of treatment.
B R U C E : I think it’s also important to recognize that when we understood that
the basis of this disease was a deficiency of C1 inhibitor and when the capability
of purifying C1 inhibitor had reached the point where we could make it in
pharmaceutically appropriate amounts, it was introduced in Europe. The problem in
the United States was that introduction coincided with the outbreak of HIV/AIDS and
so there was a tremendous problem with introducing a new blood-based product
onto the market. It also took a certain amount of time and experience for people to
feel comfortable, and that we could reassure them, that this product has minimal
risk of transmitting viruses.
G A R R E T T: After many years of having no specific treatments available, the patient
with hereditary angioedema has seen marketing approval of new products and the
expectation of others soon to become available. What’s been the role of the HAE
patient organization in advocating for the availability of specific new therapies?
A N T H O N Y: The US HAE Association has a decade-long history of advocating for
patients suffering from HAE. HAE was a catastrophic unmet medical need in the
United States until new products for attack prevention and treatment received FDA
approval, so we focused the bulk of our attention on helping patients cope with their
disease, encouraging pharmaceutical companies to license their products in the United
States, and recruiting patients once clinical trials began.
ROBERT LEFEBVRE
We are fortunate to have a group of brilliant and dedicated physician-researchers—
several of whom are with us here today—who share our commitment to ensuring
that the US HAE patient community has access to all therapies. Clinical trials were
successful because of the physician community’s dedication to doing whatever
possible to help their patients live better lives.
B R U C E : The patient organization played an absolutely crucial role in dealing with
both the pharmaceutical companies as well as the physician community. It’s probably
true for most rare diseases that without the involvement of a group like the Hereditary
Angioedema Association (HAEA), it’s much harder to make progress.
J A N E T: We’re not stopping at this juncture simply because there are products on the
market now. As an association we’re also moving forward with a scientific registry for
future research. We’ve gone from zero to 150 miles an hour in the last year and we’re
going to keep moving forward.
B O B : The HAEA has played a crucial role in helping bring new therapies to the US
market. They have also worked to ensure that HAE patients have timely and accurate
information about products and support services they need. CSL Behring and the
HAEA are truly aligned in our goals to improve the lives of patients with HAE.
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I applaud Tony and the association for their dedication to patients with HAE and how
they have never wavered from keeping the patient at the center of all their efforts.
A N T H O N Y: The US HAE Association is a patient services and research organization
run by HAE patients. Currently we have seven highly motivated patient services representatives who are in touch with HAE patients on a day-to-day basis, and provide a
wide range of services and unbiased information. They are either are patients
themselves or the parents of patients.
I think it is important that I make clear that the US HAE Association is product-neutral.
We do not get involved in recommending or evaluating specific medicines or their
suitability for a patient. Our role is to provide HAE patients with unbiased information
to help them work with their physicians to craft a suitable treatment plan.
In addition to our patient services activities, we are committed to driving HAE research.
We have established a patient-driven scientific registry that is becoming a robust
repository of biomedical and clinical data we are confident will open up possibilities
for research.
M A R C : I don’t think one can overstate the impact the HAEA organization has had.
Without it, I don’t believe the progress that’s been made would have been possible.
A message I give to all my patients regardless of the condition they have is that they
are their own most powerful advocates for their best care.
A N T H O N Y: Let’s not forget the importance of the physician-researchers who have
helped us. We have a network of highly motivated compassionate physicians who
have been a guiding force in helping the patient group make sure that its strategies
and programs are aligned with the best outcomes for our patients. Our strategy all
along has been to identify both patients and key physicians and we’ve been doing
that systematically over the 10 year period that we’ve existed.
J A N E T: We currently have about 3,700 patients in our database. But some of those
patients probably have family members who simply haven’t taken the time to register
with us, so we figure that the number of patients we communicate with directly or
through their own family members would reach about 7,000.
The Role of Alternative Therapies
ANTHONY CASTALDO
G A R R E T T: Until a few years ago, the mainstay of treatment was anabolic
steroids. Now that there are specific therapies available to treat acute attacks of
HAE and reduce the number of attacks, what is the role of alternative therapy,
such as anabolic steroids, today?
H E N R Y: Based on the experience of our colleagues in Europe, where they have had
C1 esterase inhibitor available for more than 30 years, androgen derivatives still have a
role in prevention and in reducing the frequency and severity of HAE attacks. This is
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especially true for patients using a relatively low dose and not experiencing significant
adverse reaction. For these patients, I don’t think there is a need to stop androgen
derivatives. Having said that, the androgen derivatives are contraindicated for pregnant
women. It does require additional precaution when prescribing for young women,
teenage girls and young children. With those in mind, I believe androgen derivatives
continue to play a role in HAE treatment, especially for those who have been using
androgen derivatives and see benefits without serious side effects.
G A R R E T T: Would the role be prevention rather than treatment of an acute attack?
B R U C E : Yes, and that’s always been true. We do sometimes see people misusing
androgens to treat acute attacks, where they really have no role.
A N T H O N Y: We are ecstatic that HAE patients finally have a choice of therapies. It’s up
to physicians, who are well informed about the side effects and benefits of the range of
treatments, to work with the patient and tailor an optimal therapeutic approach.
M A R C : When we didn’t have specific drugs that were effective, androgens were very
useful. Certainly a number of women, who often suffer difficult virilizing side effects,
had to use androgens because they were at great risk without them and there were
no treatment options. Now that we have other effective treatments, we have the
ability to look at the problem differently. So the question becomes, how do we
maximize the benefit and minimize adverse effects in an individual patient?
Reimbursement for New Therapies
G A R R E T T: In some circumstances patients whose physicians have prescribed one
of the newer treatments have been denied by their insurance companies, who said
Danazol is good enough.
H E N R Y: There are many layers to this. First, a lot of insurance companies do not have
the new medicines on their formulary yet, so they automatically reject the claim. It’s a
matter of time to educate the insurance companies and to let them know that we have
a new drug available that is the standard of care in Europe and currently approved by
FDA for treating HAE. Second, despite the use of Danazol or other androgen derivatives,
many patients still have breakthrough swelling attacks. Without an effective drug
for their acute attacks, many of them require emergency department visits, or even
hospitalization. Third, Danazol has significant adverse reactions for many patients.
DR. H. HENRY LI
B R U C E : It’s incumbent on us as physicians and clinical investigators now to continue
to look at the real world use of these drugs and publish our findings. We’ve been
using androgens for 40 years and are now in the process of trying to figure out how
treatment should change. We need a published record of what the pros and cons are.
I think we’re going to see more and more consensus documents and clinical studies
surveys that will help us learn how to maximize benefits and at what cost.
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M A R C : This highlights the importance of physicians being strong advocates for their
patients, and specialists or experienced physicians in hereditary angioedema fighting
this battle to get our patients the best possible care. This is a scenario we see in many
conditions when new therapies become available that will benefit specific patients.
However, there’s pushback by some payors to promote the most inexpensive treatment,
even if that’s not in the best interest of the patient. That’s why we have to take
the time to document the risks of treatments and document the benefits of newer
treatments and use that information to individualize the therapy for each patient.
A N T H O N Y: I’ve been quite surprised, but we’re seeing very good success in patient
reimbursement for newer medicines. Even in cases when there was an appeal, patients
have won and received reimbursement.
The reality of the situation is that the newer HAE medicines are expensive and many
patients can’t afford to pay co-pays and other expenses, even if they do have insurance.
So in the rare disease realm, it is vital to have an infrastructure that provides patients
with co-pay assistance. Each one of the pharmaceutical companies assured us there
would be a very robust insurance reimbursement infrastructure, or assistance, and that
there would be medicine available for those who were uninsured or couldn’t afford it.
We are quite happy that each company has lived up to its promise.
B O B : CSL Behring has a long heritage of advocating for patient access to care. This
DR. MARC RIEDL
encompasses access to both physicians and product when and where they need it.
Therapies patients cannot afford to pay for do them no good. We understand how
intimidating it can be for a patient to navigate through the insurance landscape. That’s
why we have created programs to ensure that patients can have a partner to assist
them every step of the way and to coordinate these efforts with the HAEA patient
service representatives so patients are aware of exactly where they are in the process
and to advocate for them. CSL Behring also understands that some patients may have
inadequate insurance coverage or no coverage at all. That is why we have generously
supported Patient Services Incorporated to provide co-pay and premium assistance to
HAE patients. We also have a Patient Assistance Program to support the short-term
product needs of patients while we find permanent insurance solutions for them.
We have dedicated staff who are experts in navigating the insurance system
and advocating for patients. These individuals are relentless in pursuing solutions to
these challenges.
J A N E T: Our association has also worked hard to educate our patients on the
reimbursement process and how to navigate through it, now that there is medication
available to them.
B R U C E : I’m also involved in fights over where patients get their treatment. The treat-
ment of hereditary angioedema is best done at home early in an attack, before it gets
severe. But there’s a lot of resistance right now among health care delivery systems
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and physicians to allowing patients to self-administer the new treatments at home.
Hopefully through a process of education, publication and advocacy, we’ll manage to
push the treatment more and more towards home.
A N T H O N Y: The HAE Association has always been a staunch advocate for the home
infusion option. I have seen the direct benefits of this treatment approach. My daughter
happens to have an extremely severe case of HAE. When she participated in a clinical
trial in the 2001 timeframe, we had to travel to a medical facility two or three times a
week to get treatment for acute HAE attacks. By the time we got my daughter to the
medical facility, her abdominal pain was so severe that pain medicine had to be given
immediately. She continued to miss school and experience an inordinate amount of
pain and suffering.
In the fall of 2002, we began purchasing medicine from overseas sources under the
aegis of the FDA’s Personal Importation Guideline and my daughter was able to
self-infuse at home. She quickly figured out how to use the medicine to both prevent
attacks and treat breakthrough attacks. Having home access and being able to
self-infuse totally transformed my daughter’s life. She stopped missing school and
her entire demeanor and outlook on life became more positive—it was the most
remarkable change I have ever witnessed.
G A R R E T T: How can you demonstrate that early intervention, at the first sign of
prodromal symptoms, actually prevented the clinical symptoms, such as swelling,
from occurring in the first place? If they don’t occur, how do you know that they
would have?
M A R C : That is a difficult question because not all patients have prodromes. As
DR MARC RIEDL
for how well do prodromal symptoms really predict an actual attack, the positive
predictive value of these non-specific symptoms has never been looked at. Even so,
in my practice I have patients who can very accurately predict their attacks before
they have visible swelling based on the prodromes. So I’m a strong advocate for selftreatment by patients because I think they’re the best judge of when an attack is
going to occur and when treatment is necessary.
Ensuring Adequate Supply
G A R R E T T: Bob, what does CSL Behring do now to ensure that patients have a
constant supply of product available?
B O B : It’s interesting that whereas at one time pharmaceutical manufacturers and
biotechnology companies were expected to find a solution and bring product or therapy
to the marketplace—and that was all—now companies need to go beyond that. Now
they also need to ensure that there’s access, that there’s a sufficient amount of product
available for the life of that patient’s therapy, and any assistance that’s needed in
terms of education and reimbursement support has to be part of the package.
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But CSL Behring has been doing this for a long time. That is the business we’re in.
Everything we do is about rare and serious disorders. Everything we do in terms of
where we make our products, the plasma we collect and the plasma that we ensure
gets turned into therapeutic proteins, is centered on the idea that we maintain supply
and patients can access the product. And we offer programs to assure supply and
reimbursement assistance for those who need it.
Increasing Awareness and Education
G A R R E T T: How can we address the need for increased awareness of hereditary
angioedema so that physicians as well as affected patients are more likely to
recognize it?
M A R C : I think everyone here has been involved in those efforts on a number of levels.
Pharmaceutical companies have given grants for educational programs sponsored by
the HAE Association, particularly for the patient population. As physician specialists,
we’ve also been involved in trying to get the word out to both health care providers
and to patients who are experiencing symptoms that might be consistent with HAE.
Most HAE patients are cared for by allergists/immunologists, so over the last few years
there has been a tremendous effort to educate these specialists. The bigger challenge
is to reach internists, family physicians, pediatricians, dermatologists and emergency
room physicians. It is a rare condition, but these groups are going to see these patients
on the front lines. So they are vital to making a diagnosis or referring the patient to
the proper specialist.
J A N E T: May I just add gastroenterologists to that list because of the abdominal
symptoms patients often present?
B R U C E : While the allergy community is trying to make sure that we’re up to speed
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and that we’re educating other physicians and are available for referrals, we have the
patient association speaking to patients, directing them to physicians who know about
hereditary angioedema. I think we really do a much better job when we approach it
from both sides.
J A N E T: For increasing awareness and recognition of the disease, the Internet has
been an amazing tool for us. When patients or someone who may suspect they have
symptoms of HAE reads information on our Website, they may then decide to get in
touch with the association. We put them in touch with a patient services representative,
who refers them to a physician.
A N T H O N Y: The other key group we’d like to target is emergency room physicians.
They can be a great source of referrals to knowledgeable physicians.
M A R C : In many emergency rooms, especially in large cities, there is a protocol
when a patient comes in with angioedema. It usually consists of antihistamines and
corticosteroids and maybe epinephrine, which are medications that are not very
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effective if at all in HAE. In some cases they may be harmful to an HAE patient over
time. We need to get ER physicians to think about this and put it on the radar.
B R U C E : Two quick comments here. First is a terminology issue. These days people
are using acquired angioedema to refer to a disease that I prefer be called acquired C1
esterase inhibitor deficiency, as opposed to idiopathic angioedema, which is really
what emergency rooms see more of. So we have to be careful when we talk about
acquired angioedema these days. And secondly, while I agree completely that the
emergency room is a key target, I think it’s almost impossible to get them not to use
their algorithm-driven treatment. But, if they’ve given these drugs and they don’t
work, that should trigger the question, what else could this be? If we can encourage
them to consider ordering a C4 level, or referring the patient at that point, we’ll get
where we’re trying to go.
A N T H O N Y: One way of attacking this issue would be to supplement the protocol
with a few key screening questions. For example, does your stomach hurt, do you get
a rash, and does anybody else in your family experience these symptoms?
H E N R Y: Again I believe a key element is to let the patients to be their own advocates.
Patients usually know to which emergency room they go. One of my patients set up
an appointment herself with the director of the emergency department of a local
hospital to discuss the protocol of treatment for her HAE attacks.
A N T H O N Y: It would be great if the ER physician recognized HAE. But our goal in
the US Hereditary Angioedema Association is to advocate for patients being treated
for HAE in settings outside of the emergency room, hopefully at home. We know from
experience that the earlier in the attack you get treated, the better your outcome.
M A R C : There is a small European study with C1 inhibitor therapy showing that
home therapy is superior to an emergency room visit in terms of the duration of
symptoms and the overall outcome. The more we can document and study that
issue, the better off we’ll be in terms of optimizing care.
Where is Research Headed?
G A R R E T T: What are the future research wishes of the HAE community? What
can be done here at CSL Behring to improve or innovate the treatment and care
of patients with HAE?
DR. BRUCE ZURAW
A N T H O N Y: One very important avenue of research for patients would be in finding
alternative ways to deliver IV medicines. There are some patients who don’t have good
veins, are needle phobic or just can’t get the knack of tapping into a vein. So anything
that could be done to help deliver medicine would be very beneficial for patients.
J A N E T: From the Association’s point of view, we’re excited to have our scientific
registry in place, with Dr. Zuraw’s help as our medical advisor. I’m sure he has a
number of research interests that he would like to address.
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B R U C E : There’s truly a lot that we don’t know about hereditary angioedema that
I think the registry will allow us to learn. We need to know more about the natural
history of this disease. We need to know who to treat with what type of drug and
what’s the best way to maximize the effectiveness of the treatment. We’d like to
know more about the side effects of alternative drugs. The registry is a key way in
a rare disease to collect enough clinical information that can be mined so we can
move forward.
M A R C : Looking at it as a scientific issue, I couldn’t agree more about the registry.
One of the hallmarks of HAE is its variability in severity, frequency and triggers, but we
have very little understanding about what causes that variability. The best way to try to
grapple with those questions is to have a registry where we can look at those factors
in large groups of patients. But the other area of need is for on-the-ground practical
research on the most effective way to deliver treatment to patients. What types of
treatment or delivery models give the best quality of life for patients? Is it having
patients go somewhere for treatment? Is it infusing themselves? And how do you give
access to these treatment options in a way that will be cost-effective and give the
greatest benefit to the largest group of patients?
B R U C E : In developing the registry, we have tried right from the outset to build into
it fields to look at the pharmaco-economic impact of these drugs, the overall direct
and indirect economic cost of hereditary angioedema, and quality of life parameters
over time. Those are going to be critical as we face a future in which everything will
have to be justified as being not only effective but also cost-effective.
B O B : At CSL we are constantly looking for ways to improve patients’ lives by
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improving the products we bring to market. Our expertise in rare, chronic diseases
helps us focus on product improvements that truly improve patients’ lives, such as
easier routes of administration and enhanced delivery devices. We will work closely
with the HAEA as we pursue these future enhancements.
Legislative Reforms
G A R R E T T: We talked today briefly about access issues that HAE patients are
facing due to reimbursement and health care policy. What would you like to see
in any reform legislation that may progress in Washington?
J A N E T: The first one that comes to my mind is insurance caps on individual or
lifetime expenditures. Because, for me as a patient, even with my insurance, there
will be a large co-pay and payment for the rest of my life. Certainly any rare disease
medication will come with a cost. But we’d like patients to be able to afford it for their
lifetime because this is a disease where we will need to be treated for our entire lives.
A N T H O N Y: The legislation is being phased in over time and we will evaluate its
impact as regulations are written and the various provisions of the law are implemented.
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However, the way we’re approaching this now is to look first to the pharmaceutical
companies. We are not going to be bashful about making sure that the pharmaceutical
companies providing new medicines maintain a robust infrastructure that guides
patients and their advocates in all aspects of insurance reimbursement. That’s our
focus right now. And so far we’ve seen some excellent programs that make sure that
patients who are prescribed these medicines get the appropriate assistance, expertise
and advocacy when it comes to insurance reimbursement.
B O B : In addition to insurance support, we also have a proactive public policy
function that advocates for laws and regulations to protect patient access to care.
CSL Behring believes that great strides have been made in HAE therapy but that there
is still a long way to go in insuring proper, timely diagnosis and access to appropriate
care. We are committed to working with everyone in the HAE community to make
this a reality.
Closing Points
G A R R E T T: Does anyone have a closing comment or reflection on what we
discussed today?
M A R C : I’d like to follow up on one of the comments made earlier about trying to
get providers to think about HAE as a diagnosis when patients aren’t responding to
the usual cocktail of antihistamine, steroids, epinephrine. In our current health care
system, which is very fragmented and full of overcrowded emergency rooms, often
the physicians don’t actually know whether that treatment worked or not. Patients
are often given this treatment and rushed out. The only person who really knows is
the patient. Unfortunately the dots are never connected, so the patient is treated
repeatedly with ineffective therapy. That’s another challenge in terms of educating
physicians and patients.
B R U C E : One additional point. We talked about abdominal attacks and unnecessary
surgeries. But I’d also like to mention along with the risk of laryngeal attacks, the risk
of getting the cocktail and then being kicked out of the emergency room. Basically
they’re being told, this will take care of your problem, we’re busy and you’re out of
here. I know there have been deaths due to that exact scenario.
ANTHONY CASTALDO
J A N E T: I’d also like to note that every HAE attack is important, not only abdominal
or laryngeal attacks. A peripheral swelling of the hand is important to someone who
is a pianist, and a foot attack is critical to someone who’s a salesperson and needs
to be on their feet. Of course the laryngeal attack is the one that we really need to
emphasize, but we need to see the attack in the context of the person’s whole social
and economic lifestyle.
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M A R C : One of the interesting effects of new therapies being available is that, in my
practice, I see patients who have been suffering in silence now coming in seeking care.
They’re interested in this new treatment. When I ask “How many attacks are you
having?”, they may say, “Well I have a couple of attacks a month.” But because there
has been no effective treatment, these patients were sitting at home with swelling and
not seeking treatment. So I personally am becoming increasingly aware that many
patients having these attacks have been marginalized before.
A N T H O N Y: We are delighted that there are new products available for HAE
patients, but the sad fact is that HAE patients are still dying from laryngeal attacks.
The HAE Association is looking forward to working with our physician advisors and
pharmaceutical companies to ensure that patients and their doctors understand
the importance of establishing an HAE treatment plan that includes explicit
instructions and a protocol for dealing with emergency situations.
B O B : I’d like to add a note here about caregivers and family members and their
importance to the patient. In a child’s case it’s obvious, but in many other adult
patient cases, the people they live with are vitally involved in making sure that the
patient is empowered.
M A R C : Yes, and when we talk about home treatment, that becomes really important.
With an IV drug it’s not hard to envision circumstances where the patient is unable
to do their own infusion because of swelling or because of anxiety or incapacitation.
Going forward I think you have to have either a caregiver who’s educated and
empowered or, if there isn’t one, you have to have a clinic or an emergency room
as a back-up plan, a Plan B, in case self-treatment somehow is impossible.
J A N E T: This is a long-term condition, so there is an ongoing impact on a family
ROBERT LEFEBVRE
when one person is chronically ill. We’ve started to look into how we can address that
for our patients. What are the resources that they may be able to use when a mother,
child or spouse has HAE? If you’re the person caring for someone with HAE in
emergencies and find that stressful, how can we help?
G A R R E T T: Bob, do you have any closing thoughts?
B O B : I think with the discussion today we have more clarity on the needs that are out
there. At CSL Behring we are committed not only to bringing effective therapeutics to
people who need them, but also to ensuring that patients are able to access them.
Without access and without appropriate education, we wouldn’t be doing everything
we could. We’re making investments for the future—in terms of our manufacturing
and our research and efforts—to make sure that we can respond to the needs of
patients and that they have access to the therapies that work for them.
K E Y
I S S U E S
D I A L O G U E :
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T H E
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About the Participants
Dr. Garrett E. Bergman
Robert D. Lefebvre
Senior Director, Medical Affairs, US
Commercial Operations, CSL Behring
Vice President and General Manager,
Dr. Garrett Bergman provides scientific
and medical support to the commercial
operations division of the company. A
pediatrician by training, Dr. Bergman
enjoyed a productive clinical and academic
career for 15 years at the Medical College
of Pennsylvania, leaving to enter the
biopharmaceutical industry. He has
worked in both large pharmaceutical and
small start-up biotherapies companies for
more than 20 years, primarily in Clinical
Research and Development.
Bob Lefebvre joined CSL Behring in
2006 as Vice President, General
Manager, US Commercial Operations.
He has led the successful launches of
plasma therapies to treat people with
rare and serious diseases and works
closely with patient communities to
focus on improvements to quality of
life. Prior to joining the company, he
was Vice President, Global Marketing,
MDS Pharma Services, a top five
contract research organization.
Previously, he was head of the
Oncology/Virology Business Unit at
Bristol-Myers Squibb. Bob also spent
a number of years in sales and
marketing management roles with
Sandoz Canada. He holds a degree
in Human Anatomical Sciences from
McGill University.
Anthony J. Castaldo
DR. GARRETT BERGMAN
President, United States Hereditary
Angioedema Association (HAEA)
The HAEA is a patient services and
research organization that represents
approximately 7,000 Americans afflicted
with C1 inhibitor deficiency disease.
He is also the President of Hereditary
Angioedema International—the umbrella
organization representing the world’s
HAE patient groups. Mr. Castaldo serves
as the Assistant Inspector General at the
Board of Governors of Federal Reserve
System in Washington, D.C. He has
also taught graduate level information
technology courses as an adjunct
Professor and has six HAE-related
publication credits.
US Commercial Operations, CSL Behring
Dr. H. Henry Li
Director of Immunology, Institute for
Asthma and Allergy
H. Henry Li, MD, PhD, is board certified in
Allergy and Immunology. Dr. Li received
his Allergy and Clinical Immunology
training from Johns Hopkins Hospital,
where he remains as a Medical Faculty.
He is the Director of Immunology of the
Institute for Asthma and Allergy in Chevy
Chase and Wheaton, Maryland. Dr. Li
received his medical degree from the
Tianjin Medical College. He received his
K E Y
PhD in Medical Microbiology and
Immunology from the University of Texas
Medical Branch at Galveston, Texas,
where he was awarded the McLaughlin
Fellowship for excellence in research. Dr.
Li has published many papers in allergy
and immunology peer-reviewed journals,
book chapters, and participated in many
clinical trials. His major areas of interests
are urticaria and angioedema, atopic
dermatitis, food allergies, asthma and
immunotherapy.
Janet Long
Executive Vice President, US HAEA
Ms. Long comes from a varied career
path, having served first as a hospital
chaplain, Executive Director of an
Ecumenical Council of Churches and
legal researcher before joining the US
HAEA as Vice President.
Ms. Long’s story of her 40-year struggle
to be finally diagnosed with HAE has been
featured in television and print media.
Most recently, she has assisted in the
creation of a patient-driven research
project—the US HAEA Scientific Registry
—which will forward new HAE research
and, eventually, a cure.
Ms. Long is the mother of three wonderful daughters, one of whom also has
HAE. Her passion is working toward a
better life for all HAE patients.
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D I A L O G U E :
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Dr. Marc Riedl
Assistant Professor of Medicine and
Section Head of Clinical Immunology and
Allergy, Department of Medicine, UCLA
David Geffen School of Medicine
Dr. Riedl received his medical degree
from the University of Chicago Pritzker
School of Medicine and completed his
Internal Medicine training at Washington
University/Barnes-Jewish Hospital,
St. Louis. He completed a fellowship in
Allergy/Immunology at UCLA, where he
also pursued advanced work in Clinical
Pharmacology and received a Master’s
degree in Clinical Research. Dr. Riedl is
a diplomate of the American Board of
Internal Medicine, the American Board
of Allergy and Immunology, and the
American Board of Clinical Pharmacology.
At UCLA, he directs the Clinical
Immunology and Allergy Consultation
Service, and leads an active clinical
research program funded by federal,
state, and industry sponsors.
Dr. Bruce Zuraw
Professor of Medicine, University of
California, San Diego and Staff Physician,
San Diego Veteran’s Administration
Medical Center
Dr. Zuraw is board certified in Internal
Medicine and Allergy/Immunology. He
did his Allergy/Immunology clinical and
research fellowship training at the Scripps
Clinic and Research Foundation, following
which he joined the faculty at the Scripps
Research Institute. His research interests
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include the molecular and cellular biology
of C1inhibitor deficiency as well as
the regulation of inflammatory gene
expression during airway inflammation. In
2004, Dr. Zuraw moved to the University
of California, San Diego where he is
currently the Head of the Allergy &
Immunology section as well as Training
Program Director. He sees allergy patients
in clinic, and runs a research laboratory.
About CSL Behring
CSL Behring is a leader in the plasma
protein therapeutics industry. Committed
to saving lives and improving the quality
of life for people with rare and serious
diseases, the company manufactures and
markets a range of plasma-derived and
recombinant therapies worldwide.
CSL Behring therapies are indicated for
the treatment of coagulation disorders
including hemophilia and von Willebrand
disease, primary immune deficiencies,
hereditary angioedema and inherited
respiratory disease. The company’s
products are also used in cardiac surgery,
organ transplantation, burn treatment
and to prevent hemolytic diseases in
newborns.
CSL Behring operates one of the world’s
largest plasma collection networks, CSL
Plasma. CSL Behring is a subsidiary of
CSL Limited, a biopharmaceutical
company headquartered in Melbourne,
Australia. For more information, visit
www.cslbehring.com.
www.CSLBehring.com
CSL Behring is a subsidiary of CSL Limited
© 2010 CSL Behring LLC