Rare diseases: FDA proposes new system to therapy approvals

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The FDA proposed a new pathway to expedite the approval of customized treatments for rare diseases, addressing industry concerns about profitability.
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This innovative framework aims to facilitate the development of therapies that have only been tested in a limited number of patients.
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FDA Commissioner Marty Makary emphasized the need to remove barriers and promote scientific advances for rare disease treatments.
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The new pathway, named plausible mechanism, requires evidence that therapies target specific genetic or biological abnormalities.
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The FDA will accept public comments on the draft guidance for 60 days before finalizing the new approval process.