The ISSCR Responds to FDA’s Draft Guidance on Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations
On 24 November 2025, the ISSCR submitted comments on the Food and Drug Administration's draft guidance for Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations. ISSCR appreciates FDA’s willingness to consider alternative trial designs for cellular and gene therapy products. To support this effort, ISSCR requests additional guidance on managing manufacturing changes within adaptive or master protocols; decentralized trials for small populations; long-term follow-up; using surrogate endpoints and biomarkers; and trial design selection.