Patients with life-threatening diagnoses and limited treatment options have long struggled with the question of who will pay when they attempt to obtain experimental, unapproved treatments.
Drug manufacturers have long been tasked with financing so-called compassionate use, despite the fact that many of them are unable or unwilling to provide their medications free of charge to terminally ill patients.
Lou Gehrig’s disease sufferers have discovered an extraordinary remedy after years of petitioning Congress: making the federal government pay.
According to a recent law, the National Institutes of Health will start spending roughly $25 million to sign up people for open access, or “compassionate use,” programmes of unapproved medications. Patients will have access to a sugar-based injection called trehalose in the initial phase, which was disclosed on Friday.
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The programme only accepts people who are ineligible for regular drug trials. To learn more about the therapy and their underlying condition, amyotrophic lateral sclerosis, or ALS, data on their progress must be gathered.
The project blurs the distinction between treatment and research, and it forces the NIH to pay for experimental treatments in studies that might only produce sparse data. While it provides ALS sufferers with a crucial new alternative, it also raises the potential that scarce federal funds might eventually be used for more experimental treatments for 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 numerous other specialists welcome the new strategy as an inventive response to the problems faced by ALS patients, who normally live three to five years after experiencing their first symptoms. The illness kills the nerve cells required for walking, talking, and eventually breathing.
According to researchers, up to 90% of ALS patients are ineligible for conventional clinical trials, usually because their disease has advanced too far to show significant therapy benefits. Even qualified patients have to fight for access.
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Most businesses in their industry, according to ALS sufferers, are small startups unable to cover such expenses. Drug manufacturers may refuse access for a variety of additional reasons, such as worries that unforeseen safety issues would harm their prospects of receiving clearance.
The great majority of the NIH’s $45 billion funding is allocated to early-stage research aimed at determining the causes, treatments, and prospective remedies for diseases.
One crucial component of the new approach, along with numerous biological indicators of ALS, is tracking drug safety. But because patients won’t be matched to a placebo group, the gold-standard method for doing medical research, it is unclear that the programme will be able to determine whether the treatments are genuinely helping.
(With inputs from agencies)
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