The U.S. Food and Drug Administration has signaled a major policy shift that would reduce the number of confirmatory clinical trials required for new drug approval from a minimum of two to just one. The move reflects the agency's intent to prioritize the quality of clinical trial design over quantity, aiming to improve efficiency in drug development.
According to the Korea Biotechnology Industry Organization on the 23rd, FDA Commissioner Makary and Center for Biologics Evaluation and Research (CBER) Director Fassad outlined the new direction in a recent contribution to the New England Journal of Medicine. They stated that a single well-controlled clinical trial would serve as the basic requirement for drug approval going forward.
The measure focuses on strengthening evaluation of clinical trial design rather than simple deregulation. The agency determined that poorly designed control groups or flawed statistical methodology could lead to erroneous conclusions regardless of how many trials are conducted. FDA is expected to make design precision—including use of contemporaneous control groups, effect size, primary endpoint selection, statistical power, and biological correlation—the key criteria for approval decisions rather than the number of trials.
FDA noted it has already granted approvals based on single clinical trials in certain fields. In oncology, numerous approvals have been based on single trials. The agency also cited cases where drugs were approved based on one pivotal trial combined with confirmatory evidence in specific disease areas.
Companies stand to benefit from reduced development costs and shorter timelines. According to FDA, a single confirmatory clinical trial costs between $30 million and $150 million, while drug development averages over seven years. Reducing trial requirements could lower participant numbers and shorten timelines, easing the financial burden on companies. Whether cost savings will translate to lower drug prices remains uncertain.
A Korea Biotechnology Industry Organization official said, "If this policy is implemented, companies will see significant development cost savings from reduced participant numbers and shorter timelines."