Nonprofit Foundation Rakes in Cystic Fibrosis Drug Revenues
Wednesday, 07 October 2015 00:00 By Chris Wegemer, Truthout | Op-Ed
Rather than push for lower medication costs for patients, the Cystic
Fibrosis Foundation has an incentive to keep prices high. (Photo: Money and
Pills via Shutterstock)
The Food and Drug Administration (FDA) recently approved a drug aimed at
treating an underlying mechanism of the fatal disease cystic fibrosis. At
first glance, this would seem to be an optimistic victory for those who
suffer from the condition and a validation of an effective system for
medical advancement. A deeper look tells a different story.
With a price tag of over $250,000 per year, the drug is understandably
controversial, as is the "venture philanthropy" partnership between a
nonprofit organization and pharmaceutical company that was responsible for
the creation and promotion of the drug. But it is not the biotech firm,
Vertex Pharmaceuticals, which faces scrutiny. Drug companies relentlessly
pursue ten-figure profit margins, so it was unsurprising news that Vertex
planned to pay its executives massive bonuses with revenue from the
high-priced drug, or that the new medication was being leveraged to make the
company profitable for the first time in its 26-year history. Rather, it is
the Cystic Fibrosis Foundation, the nonprofit organization supposedly
representing patients with the rare genetic defect, which is being called
into question.
Despite the astronomical price tag, benefits of the new drug, Orkambi, are
modest at best. The Cystic Fibrosis Foundation has been zealously pushing
the medication because of the $3.3 billion windfall it stands to gain from
it. It is unclear how this money will be spent by the foundation; it has no
obligation to spend it in the interest of the patients, and has indicated
that none of it will be used to help patients pay for the drug. Rather than
push for costs of medications to be lower, the foundation has an incentive
to keep costs high to increase its return on investments. The cost of
specialty drugs across the entire pharmaceutical industry is on a
spectacular rise.
The president of the Cystic Fibrosis Foundation, Robert Beall, insisted that
the organization's strategy represents the best interest of those who suffer
from the disease. According to Beall, "No one's profiting here; we're going
to make the investment back to the patients," primarily by continuing to
develop new drugs. While he admits that Orkambi is "too expensive" and he is
"very concerned" about patients being excluded from treatments because of
high costs, he still believes that the recent advances from Vertex "have
been among the most significant milestones in the journey toward a permanent
cure."
Receiving annual donations totaling over $130 million, the Cystic Fibrosis
Foundation was ranked as the 43rd-largest nonprofit in the United States in
2013 (prior to the foundation's $3.3 billion payout). It is now the largest
disease-focused charity in the nation, ahead of the American Cancer Society,
American Heart Association and American Diabetes Foundation. Representing
less than .001 percent of the global population, cystic fibrosis (a
primarily white European disease) is easily the most heavily funded rare
disease in the world.
THE CYSTIC FIBROSIS FOUNDATION HAS A VERTICALLY INTEGRATED MONOPOLY ON EVERY
ASPECT OF THE DISEASE IN THE US.
The genetic condition responsible for cystic fibrosis was discovered in
1989. Despite billions of dollars and more than two and a half decades of
research conducted on behalf of the Cystic Fibrosis Foundation, only three
medications addressing mechanisms unique to the disease have been developed
(including Orkambi and a closely related drug from Vertex, Kalydeco). In
contrast, other rare genetic diseases have seen tremendous improvements in
treatments with a small fraction of the resources. The Cystic Fibrosis
Foundation advertises several medications in various stages of research that
it is helping deliver to patients; the vast majority of these are simply
repackaged and repurposed drugs already widely available (including
ibuprofen, salt water and even Viagra). The only drug in development that
has shown effectiveness in addressing the underlying genetic defect of the
disease was not supported by the Cystic Fibrosis Foundation, but the
comparatively tiny UK Cystic Fibrosis Gene Therapy Consortium.
The Cystic Fibrosis Foundation has a vertically integrated monopoly on every
aspect of the disease in the United States. It sanctions fundraising events
and local awareness-building chapters. It dominates outreach to patients,
facilitates generous media exposure and touts successes of large
fundraisers. It approves - and even ranks - care centers and physicians
across the country in the only network of cystic fibrosis health-care
providers. It runs a research subsidiary, which directs the largest array of
cystic fibrosis research projects and clinical trials in the world. The
resulting standardization and lack of competition has bred a stagnant
environment and an entrenched business mindset that silences new
perspectives.
As a cystic fibrosis patient myself, I am indebted to researchers and
advocates. An extensive routine of treatments has been necessary for my
survival. Equally important has been a critical perspective that questions
my own lifestyle choices and seeks to understand best practices of medical
care. After 30 years of being actively engaged in developments in the field,
this healthy skepticism has extended to the larger cystic fibrosis industry,
where such a self-reflective attitude seems to be lacking.
One of my earliest memories was of my family crying tears of joy while
watching a story on the evening news announcing the discovery of the faulty
gene responsible for cystic fibrosis. Since then, a cure has perpetually
been advertised as just around the corner. For decades, parents who
desperately wished their children to be healthy couldn't help but to succumb
to believing this fiction. All of us - patients, parents and donors - must
have the courage to ask hard questions and demand more. Until we do,
breakthroughs will continue to be scarce and promises of an imminent cure
will remain empty.
The Cystic Fibrosis Foundation is far and away the largest nonprofit
organization dedicated to a rare disease and its actions dictate the terms
of the entire field. The 30 million people in the United States who are
afflicted with rare diseases deserve a more effective model for the
improvement of their well-being and the pursuit of a cure.
Copyright, Truthout. May not be reprinted without permission.
CHRIS WEGEMER
Chris Wegemer is a student at the University of California, Santa Barbara.
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Nonprofit Foundation Rakes in Cystic Fibrosis Drug Revenues
Wednesday, 07 October 2015 00:00 By Chris Wegemer, Truthout | Op-Ed
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. Rather than push for lower medication costs for patients, the
Cystic Fibrosis Foundation has an incentive to keep prices high. (Photo:
Money and Pills via Shutterstock)
. The Food and Drug Administration (FDA) recently approved a drug
aimed at treating an underlying mechanism of the fatal disease cystic
fibrosis. At first glance, this would seem to be an optimistic victory for
those who suffer from the condition and a validation of an effective system
for medical advancement. A deeper look tells a different story.
With a price tag of over $250,000 per year, the drug is understandably
controversial, as is the "venture philanthropy" partnership between a
nonprofit organization and pharmaceutical company that was responsible for
the creation and promotion of the drug. But it is not the biotech firm,
Vertex Pharmaceuticals, which faces scrutiny. Drug companies relentlessly
pursue ten-figure profit margins, so it was unsurprising news that Vertex
planned to pay its executives massive bonuses with revenue from the
high-priced drug, or that the new medication was being leveraged to make the
company profitable for the first time in its 26-year history. Rather, it is
the Cystic Fibrosis Foundation, the nonprofit organization supposedly
representing patients with the rare genetic defect, which is being called
into question.
Despite the astronomical price tag, benefits of the new drug, Orkambi, are
modest at best. The Cystic Fibrosis Foundation has been zealously pushing
the medication because of the $3.3 billion windfall it stands to gain from
it. It is unclear how this money will be spent by the foundation; it has no
obligation to spend it in the interest of the patients, and has indicated
that none of it will be used to help patients pay for the drug. Rather than
push for costs of medications to be lower, the foundation has an incentive
to keep costs high to increase its return on investments. The cost of
specialty drugs across the entire pharmaceutical industry is on a
spectacular rise.
The president of the Cystic Fibrosis Foundation, Robert Beall, insisted that
the organization's strategy represents the best interest of those who suffer
from the disease. According to Beall, "No one's profiting here; we're going
to make the investment back to the patients," primarily by continuing to
develop new drugs. While he admits that Orkambi is "too expensive" and he is
"very concerned" about patients being excluded from treatments because of
high costs, he still believes that the recent advances from Vertex "have
been among the most significant milestones in the journey toward a permanent
cure."
Receiving annual donations totaling over $130 million, the Cystic Fibrosis
Foundation was ranked as the 43rd-largest nonprofit in the United States in
2013 (prior to the foundation's $3.3 billion payout). It is now the largest
disease-focused charity in the nation, ahead of the American Cancer Society,
American Heart Association and American Diabetes Foundation. Representing
less than .001 percent of the global population, cystic fibrosis (a
primarily white European disease) is easily the most heavily funded rare
disease in the world.
The Cystic Fibrosis Foundation has a vertically integrated monopoly on every
aspect of the disease in the US.
The genetic condition responsible for cystic fibrosis was discovered in
1989. Despite billions of dollars and more than two and a half decades of
research conducted on behalf of the Cystic Fibrosis Foundation, only three
medications addressing mechanisms unique to the disease have been developed
(including Orkambi and a closely related drug from Vertex, Kalydeco). In
contrast, other rare genetic diseases have seen tremendous improvements in
treatments with a small fraction of the resources. The Cystic Fibrosis
Foundation advertises several medications in various stages of research that
it is helping deliver to patients; the vast majority of these are simply
repackaged and repurposed drugs already widely available (including
ibuprofen, salt water and even Viagra). The only drug in development that
has shown effectiveness in addressing the underlying genetic defect of the
disease was not supported by the Cystic Fibrosis Foundation, but the
comparatively tiny UK Cystic Fibrosis Gene Therapy Consortium.
The Cystic Fibrosis Foundation has a vertically integrated monopoly on every
aspect of the disease in the United States. It sanctions fundraising events
and local awareness-building chapters. It dominates outreach to patients,
facilitates generous media exposure and touts successes of large
fundraisers. It approves - and even ranks - care centers and physicians
across the country in the only network of cystic fibrosis health-care
providers. It runs a research subsidiary, which directs the largest array of
cystic fibrosis research projects and clinical trials in the world. The
resulting standardization and lack of competition has bred a stagnant
environment and an entrenched business mindset that silences new
perspectives.
As a cystic fibrosis patient myself, I am indebted to researchers and
advocates. An extensive routine of treatments has been necessary for my
survival. Equally important has been a critical perspective that questions
my own lifestyle choices and seeks to understand best practices of medical
care. After 30 years of being actively engaged in developments in the field,
this healthy skepticism has extended to the larger cystic fibrosis industry,
where such a self-reflective attitude seems to be lacking.
One of my earliest memories was of my family crying tears of joy while
watching a story on the evening news announcing the discovery of the faulty
gene responsible for cystic fibrosis. Since then, a cure has perpetually
been advertised as just around the corner. For decades, parents who
desperately wished their children to be healthy couldn't help but to succumb
to believing this fiction. All of us - patients, parents and donors - must
have the courage to ask hard questions and demand more. Until we do,
breakthroughs will continue to be scarce and promises of an imminent cure
will remain empty.
The Cystic Fibrosis Foundation is far and away the largest nonprofit
organization dedicated to a rare disease and its actions dictate the terms
of the entire field. The 30 million people in the United States who are
afflicted with rare diseases deserve a more effective model for the
improvement of their well-being and the pursuit of a cure.
Copyright, Truthout. May not be reprinted without permission.
Chris Wegemer
Chris Wegemer is a student at the University of California, Santa Barbara.
Related Stories
Big Pharma Lobbies Hard to End India's Distribution of Affordable Generic
Drugs
By Mike Ludwig, Truthout | ReportBig Pharma Is Killing Us
By The Daily Take Team, The Thom Hartmann Program | Op-EdHow Much of Big
Pharma's Massive Profits Are Used to Influence Politicians?
By Mike Ludwig, Truthout | Report
Show Comments