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.
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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.”
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