FDA Outlines Process To Speed Up Rare Disease Therapy Approvals

The U.S. Food and Drug Administration on Thursday introduced the Rare Disease Evidence Principles (RDEP) to provide greater speed and predictability in the review of therapies intended to treat rare diseases with very small patient populations.

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Through the RDEP process, sponsors will receive clearer guidance on the types of evidence that can be used to demonstrate substantial evidence of effectiveness.

Also Read: What’s Going On With Sarepta, Capricor And Other Gene Therapy Stocks On Wednesday?

Approval under the process may be based on one adequate and well-controlled study plus robust confirmatory evidence, which may include:

• Strong mechanistic or biomarker evidence
• Evidence from relevant non-clinical models
• Clinical pharmacodynamic data
• Case reports, expanded access data, or natural history studies

Sponsor requests for review under the RDEP process are separate from and independent of requests for orphan-drug designation under section 526 of the Federal Food, Drug & Cosmetic (FD&C) Act. A drug that is reviewed under the RDEP process does not necessarily qualify as a drug that has an orphan drug designation.

William Blair on Wednesday wrote, “While we are encouraged that the FDA is continuing to exhibit flexibility in evaluating gene therapies for rare diseases, it remains unclear what tangible impact the framework will have on the development process and approval timeline.”

Several gene therapies in development are already utilizing single-arm trials for registration, including Neurogene Inc.’s NGNE NGN-401, Rocket Pharmaceutical Inc.’s RCKT RP-A501, Lexeo Therapeutics, Inc.'s LXEO LX2006, Ultragenyx Pharmaceutical Inc.'s RARE UX111 and DTX401 and uniQure NV’s QURE AMT-130.

William Blair’s analyst in August said Dr. Vinay Prasad's return as the head of the FDA’s Center for Biologics Evaluation and Research could reignite some volatility for the cell and gene therapy spaces. Dr. Prasad has openly criticized the biopharmaceutical industry and the FDA for using accelerated approvals broadly based on surrogate endpoints.

The companies developing therapeutics for rare diseases and relying more heavily on interim clinical data instead of surrogate biomarkers for accelerated approval are expected to fare better under Dr. Prasad's return.

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