FDA Announces Shift to Single-Trial Standard for Drug Approvals Under New Commissioner Makary
核心洞察
The FDA will change its default requirement from two clinical trials to one pivotal trial for drug and medical device approvals, with the new policy expected to roll out in three to six months.
FDA Commissioner Marty Makary stated that one appropriately designed trial can achieve the same statistical power as two trials, making the approval process more practical and efficient.
In 2024, 66% of the 50 novel drugs approved already used only one pivotal trial, representing a significant increase from 38% in 2019, with orphan drugs (搜索) comprising 70% of single-trial approvals.
The Food and Drug Administration will shift to requiring one clinical study as the default standard for drug and medical product approvals, marking a significant departure from the traditional two-trial requirement, FDA Commissioner Marty Makary announced this week.
The new policy, which Makary described as "more practical," is designed to accelerate the drug review and approval process while maintaining scientific rigor. "You can achieve the same statistical power with one trial as you would with two trials when it's designed and controlled appropriately," Makary told STAT by phone.
Growing Trend Toward Single-Trial Approvals
The policy change formalizes a trend already underway in drug approvals. According to an AgencyIQ analysis, 66% of the 50 novel drugs approved in 2024 relied on just one pivotal trial, a substantial increase from 38% in 2019. The previous high was 68% in 2021.
Orphan drugs (搜索), which treat rare diseases affecting 200,000 or fewer people, represented 70% of the drugs approved last year based on a single trial. Oncology (搜索) led all therapeutic categories with 14 single-trial approvals, according to AgencyIQ data.
Economic Impact on Drug Development
The shift addresses significant cost pressures in pharmaceutical development. Clinical trials cost an average of $117 million and represent the most expensive component of drug development, accounting for 68% of R&D spending, according to an Eastern Research Group study for the Department of Health and Human Services conducted in October 2024.
While the FDA has historically required two trials to provide additional assurance of safety and efficacy, the agency has become increasingly flexible in recent years, with many drugmakers already submitting single pivotal trials for approval consideration.
Implementation Timeline and Concerns
The new one-trial policy is expected to be implemented within three to six months. However, the change has raised concerns among some drug reviewers and regulatory officials. Richard Pazdur (搜索), the FDA's top drug regulator who announced his sudden retirement this week, and other reviewers worry the new policy could weaken the agency's ability to request additional evidence when needed.
Makary emphasized that the FDA will still require two trials in certain cases where additional evidence is deemed necessary, but the default standard will shift to one appropriately designed and controlled study.
Mixed Signals on Vaccine Approvals
The policy change comes amid conflicting signals from the FDA regarding different types of medical products. A leaked memo from FDA vaccine chief Vinay Prasad suggests the agency may move in the opposite direction for vaccine approvals, potentially requiring randomized controlled trials instead of the current practice of approving vaccines like pneumonia and seasonal flu shots (搜索) based on antibody data.
This divergent approach highlights the complexity of balancing expedited approvals with maintaining public confidence in regulatory standards, particularly in an already challenging vaccine market environment.