The FDA has launched an initiative to develop a new regulatory framework for rare disease drugs and biologics, as only about 5% of rare conditions have an FDA-approved treatment.
The Rare Disease Framework Initiative will consider the nuances of each disease, patient needs, product type and evidence that can realistically be generated in small patient populations, according to an Oct. 14 FDA release.
The framework will seek greater consistency and predictability in regulatory decisions and incorporate patient input and external expertise. The FDA also plans to apply risk-based approaches to chemistry, manufacturing and controls review and pre-approval inspections and promote novel scientific and statistical approaches.
The agency will hold an Oct. 28 public meeting to gather feedback. Sector-specific roundtables, requests for information and internal FDA feedback sessions will follow.
The initiative follows the FDA’s February draft guidance on a “plausible mechanism” pathway, which would allow approval of individualized gene-editing and RNA therapies for ultra-rare diseases when randomized trials are not feasible. Recent rare disease approvals include apitegromab, the first muscle-targeted therapy for spinal muscular atrophy in patients on SMN2-targeted treatment.
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