FDA Reforms Evidence Standards to Accelerate Drug Approvals

New proposals from the FDA aim to make drug development more efficient by accepting limited data for certain treatments.

Daniel Beaird

Feb. 25, 2026

Key Highlights

The FDA will now often accept a single strong clinical trial for common medications, supported by additional data.
New frameworks allow approval of rare or personalized therapies based on small patient groups and biological plausibility.
These changes aim to accelerate access to treatments without compromising safety or effectiveness.

The FDA is rethinking its traditional evidence requirements, aiming to streamline the drug approval process and get treatments to patients faster. For common medications, the agency will now often accept a single strong clinical trial, supplemented by supporting data, instead of the long-standing requirement of two independent studies. The move aims to streamline the approval timeline without compromising confidence in a drug’s safety or effectiveness.

For rare or highly individualized therapies like gene-editing treatments, large trials are often impractical. To address this, the FDA has proposed a new framework that allows approval based on data from very small patient groups, as long as the therapy has a plausible biological mechanism and targets the root cause of the disease. Public comments on the proposal will be accepted before it is finalized.

The two announcements, issued just a week apart, highlight a broader push to reshape how the FDA evaluates clinical evidence. They also mark the latest in a series of shifts to long-standing FDA norms and standards, many of which have been introduced outside of the traditional federal rulemaking processes typically used to revise agency policy.