The U.S. Food and Drug Administration (FDA) is preparing to revise its drug approval process, planning to require only one pivotal clinical trial instead of the traditional two before considering new drugs for approval. The news was first reported by STAT.
“You can achieve the same statistical power with one trial as you would with two,” FDA commissioner Marty Makary told STAT, which broke the news on Thursday.
The proposed change is not yet final but is expected to be completed within three to six months. The announcement has already led to significant reactions within the pharmaceutical industry and was reportedly a factor in the sudden retirement of Richard Pazdur, who recently became head of the FDA’s Center for Drug Evaluation and Research.
Biopharma consultant David Alderman expressed strong support for the move. “Makary just dropped a regulatory acceleration warhead in the middle of every portfolio model in the industry,” Alderman wrote in a LinkedIn post. He added that lowering the data requirements for approval could save companies years in planning and billions in costs.
Analysts at Truist noted that while the FDA has become more flexible—especially regarding rare diseases or conditions with unmet needs—larger disease indications with multiple existing treatments have typically required stronger evidence. They stated, “While the FDA has increasingly shown flexibility in recent years, particularly for rare diseases and areas of significant unmet need, indications with large, heterogeneous populations and multiple existing therapies have historically required stronger evidence.”
Despite concerns about potential long-term risks from lower approval thresholds, Truist analysts observed that many companies awaiting FDA decisions could benefit if this policy is adopted. For example, Biogen may be able to seek approval based on current positive Phase III data for dapirolizumab pegol in systemic lupus erythematosus. Similarly, Cytokinetics might gain approval for ulcamten—currently being tested in a Phase II heart failure study—with only one Phase III trial rather than multiple studies.
However, questions remain about how broadly this policy would apply since the FDA already allows single-trial approvals under certain circumstances. It is also unclear when two trials might still be required or how this change will impact companies aiming for global launches since other countries often demand more extensive trial data.
