Pharma groups urge FDA to clarify criteria for approving drugs based on a single pivotal trial

Pharmaceutical companies and industry associations are asking the U.S. Food and Drug Administration for more detailed guidance on when a single pivotal clinical trial, supported by additional confirmatory evidence, can be sufficient for drug approval. They want the draft guidance to include concrete examples and clearer criteria.

The request reflects concerns that the current draft leaves sponsors uncertain about the evidentiary standards required for a one-trial pathway. Clearer rules could help companies plan development programs and reduce regulatory risk.

Industry groups say that greater clarity would benefit both innovators and patients by streamlining the approval process while maintaining safety standards.