Patients with serious or immediately life-threatening diseases who don’t qualify for clinical trials and have exhausted all treatment options can ask for FDA approval for access to experimental drugs, known as compassionate use.
While exact numbers are hard to come by, studies, drugmaker actions and expert insights suggest that getting access to experimental drugs for rare disease patients is more difficult than for common illnesses, Kaiser Health News reported.
Drugmakers are often unwilling to provide their drugs for compassionate use to rare disease patients before clinical trials are done for fear of an adverse event jeopardizing FDA approval of the drug.
“There’s not as much investment in rare diseases, so an [adverse] event could frighten the already limited number of potential investors,” Lisa Kearns, a researcher in the ethics division of New York University’s medical school, told Kaiser Health News.
It’s also hard to study drugs designed for rare diseases, as the patient population is small and often diverse, according to Kaiser Health News.
Drugmakers base their decision on whether to approve a drug for compassionate use on a variety of factors, commonly on evidence that patients can tolerate the drug and that the benefit outweighs the risk.
But Jess Rabourn, CEO of WideTrial, which helps drugmakers run compassionate use programs, told Kaiser Health News: “This idea that you have to wait until the research is done is baloney. We’re talking about patients who are going to die if they’re told to wait.”
Evidence also shows that granting compassionate use very rarely disrupts drug approval, Kaiser Health News reported.
Drugs that weren’t made available for compassionate use until clinical trials were completed in 2020 include Evrysdi for spinal muscular atrophy; Enspryng, for an autoimmune disease called neuromyelitis optica spectrum disorder; and Viltepso, for certain patients with Duchenne muscular dystrophy, Kaiser Health News reported.
A spokesperson for Roche, which makes Evrysdi and Enspryng, told Kaiser Health News the decision not to make the drugs available through compassionate use was due to its company policy that it doesn’t set up expanded access programs for any drugs until results are available from a phase 3 clinical trial.
Drugs for more common diseases are given to many more patients through compassionate use programs. Last year, Novartis gave more than 7,000 patients worldwide early access to cancer drugs, according to Kaiser Health News.
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