Patients with rare diseases may no longer have to wait years for gene therapies to clear the Food and Drug Administration. The agency issued draft guidance on Monday outlining a new “plausible mechanism” framework that would allow for the approval of targeted treatments for ultra-rare diseases without requiring large-scale randomized clinical trials — which can be impossible to run when patient numbers are low. [Nice News]
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Under the proposal, the FDA would consider approving a treatment when there’s solid scientific reasoning for why and how it should work, in hopes of creating tailored, cutting-edge therapies for those who have long had few or no options.
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The policy was partly inspired by an infant treated at Children’s Hospital of Philadelphia last year who became the world’s first patient to receive a personalized CRISPR-based gene therapy for his rare metabolic disease. The same approach, supporters say, could be adapted for other, similar conditions.
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“We realized we can do this over and over again, individualizing the therapy for many patients,” Dr. Kiran Musunuru, a professor at the University of Pennsylvania who helped develop the baby’s treatment, said at a briefing this week, per NPR. “It will allow doctors to treat many, many patients.”

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