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WASHINGTON (AP) — When patients with a deadly diagnosis and few treatment options have tried to get unapproved, experimental drugs, they have long faced a dilemma: Who will pay?
Responsibility
for funding so-called compassionate use has always fallen to drugmakers, though
many are unwilling or unable to make their drugs available for free to dying
patients.
After years
of lobbying Congress, patients with the debilitating illness known as Lou
Gehrig’s disease have found an unprecedented solution: make the federal
government pay.
Under a
recent law, the National Institutes of Health will begin spending about $25
million to enroll patients in compassionate use — also called open access —
programs of unapproved drugs. The first step, announced Friday, will give
patients access to a sugar-based injection called trehalose, that is thought to
help nerve cells clear toxic proteins.
Only
patients who can’t get into conventional drug trials are eligible for the
program. And their progress must be tracked to gather data about the treatment
and their underlying disease, amyotrophic lateral sclerosis, or ALS.
The
initiative blurs the line between treatment and research, and it puts the NIH
in the position of paying for unapproved drugs in studies that may yield
limited data. While it offers a critical new option for ALS patients, it also
raises the possibility that limited federal dollars could eventually be tapped
for more unproven treatments in other diseases.
“We don’t typically expect the government to
pay for things until we know they work,” said Holly Fernandez Lynch, a
University of Pennsylvania bioethicist. “But the system we have in this country
relies on drug companies to develop our drugs, and private companies are not in
the business of providing their products for free.”
Fernandez
Lynch and many other experts support the new approach as an innovative solution
to the challenges facing ALS patients, who typically survive three to five
years after initial symptoms. The disease destroys nerve cells needed to walk,
talk and — eventually — breathe.
Up to 90% of
ALS patients are ineligible for traditional clinical trials, according to researchers,
typically because their disease has progressed too far to show major treatment
benefits. Even eligible patients must compete for access. One recent analysis
counted 2,000 trial openings in the U.S. for 25,000 people living with ALS.
Patients
aren’t the only beneficiaries of the NIH program. The government funding
essentially replaces costs previously borne by drugmakers.
One
long-time patient advocate sees a troubling precedent in that financial shift.
“My sense is that it’s the companies’
responsibility, not the taxpayers’, to pick up the cost for expanded access
programs,” said Gregg Gonsalves, a Yale University researcher who has
informally advised ALS patients on expanded access. “But the companies have
stonewalled patients for years, so as a last resort they went to Congress.”
During the
1980s and 1990s, Gonsalves and other HIV activists were instrumental in pushing
drugmakers to provide early access to experimental medications.
ALS patients
say most companies in their field are tiny startups that can’t afford such
costs. Drugmakers have other reasons to deny access, including concerns that
unexpected safety problems could hurt their approval chances.
The NIH
spends the vast majority of its $45 billion budget on early-stage research
focused on identifying the root causes, treatments and potential cures for
diseases.
Tracking
drug safety is one key aspect of the new program, along with various biological
measures of ALS. But the initiative is unlikely to detect whether the drugs are
actually working, because patients won’t be compared to a placebo group, the
gold-standard approach to medical research.
“Unless the
drug is a miracle drug, it’s unlikely you would see efficacy in this type of
research,” said Dr. Walter Koroshetz, of the National Institute of Neurological
Disorders and Stroke.
The
initiative is part of broader legislation pushed through Congress last year by
patient advocates, including I AM ALS, a nonprofit co-founded by two former
Obama White House staffers.
“I’m five
years in so I can’t qualify for any clinical trials,” said Brian Wallach, who
launched I AM ALS with his wife after being diagnosed in 2017. “I hope to be
eligible for the expanded access pathway.”
He describes
NIH’s new program as a “pilot” that will be reviewed by federal inspectors, as
required by the new law.
Wallach
spent several years working on the legislation with congressional staffers. It
passed the House last year by a 423-3 vote, a rare display of bipartisanship
that also underscored the group’s political clout.
The
far-reaching bill requires the Food and Drug Administration to develop a plan
to accelerate drug development and form new partnerships to study
neurodegenerative diseases.
The
legislation grew out of patients’ deep frustration with access to experimental
therapies, including a stem cell treatment from the tiny drugmaker Brainstorm
Cell Therapeutics.
After the
company’s 200-patient study failed to show positive results in 2020, Brainstorm
allowed a handful of patients to continue receiving injections under expanded
access. But company executives said a larger program was infeasible, given that
Brainstorm has no revenue.
“We used
millions of dollars for our small expanded access program,” said Mary Kay
Turner, a company executive. “So we did the maximum we could, but it was just a
tiny sliver.”
Brainstorm
plans to submit its drug for FDA approval, despite a rare public statement from
the agency last year that company data “do not support the proposed clinical
benefit.” That followed thousands of calls and messages to the agency from ALS
patients.
While many
treatments may prove ineffective, Dr. Richard Bedlack of Duke University says
getting patients into expanded access programs is still preferable to the
current situation, in which they often seek out untested remedies on their own.
“Historically
their only option was to go on the internet and try to buy these supplements or
alternative therapies,” said Bedlack, who consults for several drugmakers.
It remains
to be seen how many patients the NIH will enroll under its $25 million grant.
The original law called for $100 million in funding over four years. House
lawmakers have budgeted $80 million in spending bills for the next fiscal year,
though those have not yet passed the Senate.
For now,
NIH’s Koroshetz notes that the expanded access studies will be more expensive
than other NIH trials because the government is bearing the cost of making and distributing
the drugs.
He conceded:
“It’s a little different from our usual grants, where we don’t pay the companies
at all for the drug.”
Follow Matthew
Perrone on Twitter: @AP_FDAwriter
The
Associated Press Health and Science Department receives support from the Howard
Hughes Medical Institute’s Department of Science Education. The AP is solely
responsible for all content.