FDA plans to make one study the default for some new drug approvals
The FDA plans to make one study the default for some new drug approvals instead of two robust clinical trials. The shift could have its biggest effect on drugs for common ailments.
The Food and Drug Administration is preparing to amend its longstanding approval process for new drugs by eliminating the requirement for two robust clinical trials. In a recent article published in the New England Journal of Medicine, FDA Commissioner Marty Makary and his deputy Vinay Prasad indicated that the FDA’s new “default position” will shift to requiring only one study for the approval of new drugs and other innovative health products. They argued that modern methodologies provide alternative avenues to verify the efficacy of new drugs, thereby justifying the reduction in trial requirements.
The move is part of a broader strategy to dismantle bureaucratic hurdles and streamline the drug approval process. The FDA officials anticipate that this change will catalyze a “surge in drug development.”
The requirement for two rigorous studies has been a standard since the early 1960s, when federal law mandated that the FDA review data from “adequate and well-controlled investigations” prior to approving new medications. For many years, the agency interpreted this as necessitating at least two studies, with a strong emphasis on large patient cohorts and extended follow-up periods. The rationale behind the second trial was to ensure that the findings from the first trial were not accidental and could be reliably duplicated.
Since the 1990s, the FDA has progressively accepted single trials for drugs targeting rare or fatal conditions, aligning with legislative directives to adopt a more flexible regulatory approach for serious diseases. The recent announcement is significant because its primary impact will be felt in the approval of drugs for common ailments, rather than those for rare diseases or cancers, which have already benefitted from a more lenient standard.
The latest directive may contrast with the FDA’s decisions regarding vaccines and gene therapies. The agency rejected Moderna’s submission for a new mRNA flu vaccine, citing inadequate clinical trial data; it subsequently agreed to review the application after the company committed to performing an additional study focused on older populations. Several experimental gene therapies and biotech drugs have also been turned down with requests for more comprehensive studies.
The FDA is moving to clarify its new stance on therapies, but uncertainty regarding the application of these policies persists. Implementation will be critical, and uncertainty remains about whether the FDA’s approach to promising therapies will truly change.