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OTP advises sponsors how to submit prior clinical and real‑world data and notes possible tailoring of long‑term follow‑up
Summary
Dr. Emily Wires explained how prior clinical data, natural history and real‑world evidence can inform trial design and analysis, described submission mechanics for INDs/BLAs, and said FDA remains open to case‑by‑case tailoring of long‑term follow‑up requirements.
Dr. Emily Wires said prior clinical data can inform dose selection, monitoring plans and clinical pharmacology assessments for proposed trials and that sponsors planning to use natural history studies or real‑world evidence should flag those proposals so FDA can evaluate whether the data are fit‑for‑purpose. She advised that when sponsors propose real‑world evidence in a submission they should explicitly state so in the cover letter to facilitate internal routing.
On long‑term follow up, Wires said it remains a critical component of gene therapy development but that FDA will consider tailoring monitoring requirements where supported by evidence, noting any proposed deviations must be discussed with FDA and supported by robust justification. She also walked through how prior knowledge may be submitted in INDs (directly or cross‑referenced with authorization) and that BLAs require direct submission of applicable reference information per the CFR. "The comment period for this draft guidance closes 09/01/2026," Wires said.
