FDA Says Only One Pivotal Clinical Trial Now Required for New Drug Approval

On February 19, 2026, FDA Commissioner Mark Makary announced that drug sponsors will need to submit data from only a single pivotal trial to support approval of a new drug product, marking a radical departure from FDAs former two-trial standard. FDA’s stated goal of the policy is to accelerate patient access to new therapies.

The Commissioner first publicly previewed this major change in December 2025, stating that “the same statistical power [of the two trial standard] can be achieved with one appropriately designed and well-controlled trial.” In early 2026, however, FDA leadership indicated that some type of additional confirmatory evidence will be expected in addition to the data generated from one trial.

The proposed reduction in controlled clinical trial evidence required to support market applications for new drugs has sent shockwaves through the industry and beyond, from patient groups to doctors, regulators, sponsors, and other stakeholders. In December 2025, Dr. Richard Pazdur, the FDA’s longtime oncology regulatory leader, resigned from the Agency. Reports indicate that deep concerns about the FDA’s changing regulatory direction, including the proposal to rely on a single pivotal trial, were among the factors contributing to his departure.

As of late February 2026, it remains too early to gauge the ultimate impact of Commissioner Makary’s announcement. Important questions related to the proposed change remain unresolved. For example, how will regulators within European Medicines Agency (EMA) member states view U.S. drug approvals supported by more limited clinical datasets? And from a CMC perspective, how will established regulatory expectations be maintained if smaller clinical development programs result in substantially fewer registration and stability lots?

Check CMC Regulatory Services’ News & Views Blog throughout 2026 as we track this development and discuss its potential impact on CMC regulatory issues.