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‘Patient-backed law’ to fund NIH experimental ALS drugs!

Patients with life-threatening conditions and few available treatments have long wrestled with the issue of who will pay when they try to access experimental, unapproved medications. Despite the fact that many of them are unable or unwilling to give their products free of charge to terminally ill patients, drug manufacturers have long been required to pay for so-called compassionate usage. After spending years petitioning Congress, those who have Lou Gehrig’s disease have found an astonishing cure: making the federal government pay.

A recent law mandates that the National Institutes of Health will begin allocating about $25 million toward enrolling patients in open access, or ‘compassionate use,’ programmes for unapproved pharmaceuticals. In the beginning, patients will have access to an injectable made of sugar called trehalose, which was announced on Friday.

Only those who are not qualified for conventional drug trials are admitted to the programme. Data on their progress must be gathered in order to understand more about the therapy and their underlying disease, amyotrophic lateral sclerosis, or ALS. The endeavour conflates treatment with research, and it pushes the NIH to fund novel therapies in studies that might only yield scant data. It offers ALS patients a vital new option, but it also raises the possibility that limited federal monies could later be utilised for more experimental treatments for other illnesses.

‘We don’t usually expect the government to pay for things until we know they work,’ said Holly Fernandez Lynch, a bioethicist at the University of Pennsylvania. ‘ However, the system we have in this nation depends on drug corporations to provide our medications, and private companies are not in the business of delivering their products for free’. Fernandez Lynch and countless other experts applaud the new tactic as a creative solution to the issues presented by ALS patients, who typically live three to five years after developing their initial symptoms. The disease destroys the nerve cells necessary for walking, talking, and eventually breathing.

Up to 90% of ALS patients, according to doctors, are not eligible for standard clinical trials since their condition has typically progressed too far for meaningful therapeutic effects. Even eligible patients must struggle to gain access. According to ALS patients, the majority of companies in their sector are small startups unable to pay such costs. For a number of additional reasons, including concerns that unanticipated safety issues may hurt their chances of gaining clearance, drug manufacturers may refuse access.

Early-stage research aimed at identifying the causes, therapies, and potential treatments for diseases receives the vast majority of the $45 billion in funding provided to the NIH. Monitoring drug safety is a key element of the new strategy, coupled with a number of biological ALS indicators. The programme may not be able to tell whether the medicines are actually working because patients won’t be matched to a placebo group, which is the gold-standard approach in medical research.


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