|
 During the televised address, Trump acknowledged in the audience
Megan Crowley, a college student who has Pompe disease - a rare
muscle disorder that can be treated with an expensive enzyme
replacement therapy.
"But our slow and burdensome approval process at the Food and Drug
Administration keeps too many advances, like the one that saved
Megan's life, from reaching those in need," the president said. "If
we slash the restraints, not just at the FDA but across our
Government, then we will be blessed with far more miracles like
Megan."
Trump has in the past excoriated drugmakers for charging
"astronomical" prices, while acknowledging how "complicated" the
nation's healthcare system is.
Representatives of the Trump administration could not immediately be
reached for comment on Wednesday on whether the president believes
the treatment price for Pompe disease is fair.
Trump's comments on Tuesday night highlight an issue central to the
industry and investors - the potential for deregulation of the FDA,
the agency charged with approving pharmaceutical products.
"I think Trump used this example somewhat cynically as a way of
trying to advance the deregulatory agenda," said Dr. Aaron
Kesselheim, a researcher at Harvard Medical School. "The FDA's
standards actually promote innovation because patients don't want
just any old drug - they want drugs that work."
Megan's father, John Crowley, now chief executive at Amicus
Therapeutics Inc, started a biotech company in 1998 to seek a
treatment for Pompe disease.
In 2006, the FDA - after a nine-month review - approved infused
enzyme replacement therapy Myozyme for patients born with the
condition. A similar drug, Lumizyme, was approved in 2010 for
late-onset Pompe disease and is now used regardless of a patient's
age.
The average annual cost of treatment is $298,000, according to
Paris-based Sanofi SA, which now owns the drugs.
[to top of second column] |
Under the Orphan Drug Act of 1983, drugs for rare diseases - defined
as those that treat fewer than 200,000 patients - are given a range
of incentives including seven years of marketing exclusivity.
"Pharmaceutical companies know they get a free pass for very
expensive drugs for orphan diseases, whereas they don't for
mainstream diseases," said Joel Hay, professor of pharmaceutical
economics and policy at the University of Southern California.
The National Organization for Rare Disorders (NORD) applauded
Trump's recognition, but disagreed with the idea that the regulatory
process needs an overhaul.
"Our patients deserve the same quality therapies as everyone else,
and to weaken the standards will only threaten our population with
unsafe, ineffective therapies," NORD said in a statement on
Wednesday.
Pompe disease, affecting 5,000 to 10,000 people worldwide, prevents
the body from making enough of an enzyme used by heart and muscle
cells to convert a form of sugar called glycogen into energy. When
glycogen builds up in these cells it can lead to disability and
death.
NORD estimates that around 30 million Americans suffer from 7,000
rare diseases.
(Reporting by Deena Beasley in Los Angeles; Editing by Matthew
Lewis)
[© 2017 Thomson Reuters. All rights
reserved.] Copyright 2017 Reuters. All rights reserved. This material may not be published,
broadcast, rewritten or redistributed.
 |
