FDA Seeks to Spur Trial Innovation With Two New Guidances

The FDA might be willing to extrapolate clinical data for targeted therapies even if sponsors can’t recruit patients with a specific genetic variant of the disease being treated, the agency says in the first of two new guidance documents.

Sponsors may get some leeway from the agency if they can show that a proposed treatment worked in clinical trials for molecularly similar diseases, or if they can show through computer modeling, close analyses of the molecular structures of disease variants, evidence from other drugs in the same pharmacological class, or some combination of these approaches, the FDA says in a final guidance on rare genetic varieties of diseases.

The agency also issued draft guidance on how sponsors should measure and use minimal residual disease as a biomarker in blood cancer trials. Both documents are part of an effort to encourage innovation in clinical trials.