Industry asks FDA for clearer rules on when one trial suffices for drug approval

Pharmaceutical companies and industry trade groups have asked the U.S. Food and Drug Administration for greater clarity on when a single pivotal trial, paired with confirmatory evidence, can support approval of a new drug, according to Endpoints News.

The publication reports that the push comes as sponsors seek more concrete detail from the agency about the circumstances under which this single-trial pathway applies, rather than the traditional requirement of two adequate and well-controlled studies. Industry groups want examples and specific criteria laid out, so companies can better plan development programs and avoid ambiguity about what evidence the FDA will accept.

Endpoints News frames the request as part of an ongoing dialogue between industry and regulators over the standards governing drug approval, with sponsors seeking predictability in how the FDA applies its existing flexibility.

The FDA has previously indicated it can accept a single pivotal trial in certain situations, particularly when supported by strong confirmatory data, but the industry’s request suggests current guidance leaves room for interpretation that companies would like resolved.

No specific timeline for an FDA response is indicated in the report. The outcome of this exchange could influence how sponsors design trials and structure evidence packages when seeking approval for new therapies going forward.