FDA Speeds Up Approvals by Defaulting to One Clinical Trial
- Feb 20
- 2 min read
In a landmark shift that ends decades of regulatory precedent, the Food and Drug Administration (FDA) has announced it will abandon its traditional two-study requirement for new drug approvals, defaulting instead to a single pivotal study.
The sweeping policy change, detailed in The New England Journal of Medicine by FDA Commissioner Dr. Marty Makary and top deputy Dr. Vinay Prasad, aims to slash bureaucracy and accelerate patient access to novel therapies. Moving forward, the agency will base market authorization on just one clinical trial that is adequate and well-controlled, provided it is backed by "confirmatory evidence". This supportive evidence can include mechanistic data, findings from animal models, or real-world evidence.
Makary and Prasad characterized the historic two-trial standard—which stems from legislation passed in the early 1960s—as an outdated "dogma". They argue that modern drug research is "increasingly precise and scientific," rendering an overreliance on two trials unnecessary when biomarkers and secondary outcomes can tell a complete biological story. The agency leaders predict this streamlined approach will trigger a "surge in drug development".
While the FDA has increasingly accepted single studies for rare diseases and cancers over the past five years, this new default policy will largely alter the approval landscape for common diseases.
However, the change has sparked sharp debate. Dr. Janet Woodcock, the FDA's former drug director, acknowledged the scientific rationale but warned that the pharmaceutical industry remains "baffled" by the unclear approach, noting that "implementation will be everything".
Other experts have voiced stronger concerns. Health policy consultant Steven Grossman argued the shift undermines patient safety, emphasizing that second studies are crucial for catching safety and efficacy issues that a successful first study might miss. The policy has also caused internal friction; Dr. Richard Pazdur, the highly respected former head of the FDA's Oncology Center of Excellence, reportedly retired late last year after being pressured by leadership to sign off on the reduced trial requirements.
Interestingly, this relaxed standard for therapeutics contrasts sharply with the agency's recent strictness regarding vaccines and gene therapies. Under Prasad's leadership, the FDA's vaccine division recently rejected several experimental treatments and initially refused to review Moderna’s new mRNA flu shot due to a perceived need for additional, definitive studies.
As the FDA navigates this new era, drug manufacturers and patient advocates alike will be watching closely to see how the agency balances its desire for rapid innovation with the fundamental need for public safety.
🔖 Sources
Keywords: FDA Approvals One Clinical Trial
FDA Approvals One Clinical Trial
Comments