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FDA Plans Major Shift in Drug Approval Process, Reducing Required Clinical Trials
The Food and Drug Administration announced a significant policy change that will fundamentally alter how new drugs come to market in the United States. Going forward, the agency will require just one rigorous clinical study for approval of new medications instead of the two studies that have been standard practice for decades.
FDA Commissioner Dr. Marty Makary and his deputy, Dr. Vinay Prasad, unveiled the change in an article published Wednesday in the New England Journal of Medicine. They described the shift as a modernization effort that recognizes advances in scientific methodology and drug development.
“In 2026 there are powerful alternative ways to feel assured that our products help people live longer or better than requiring manufacturers to test them yet again,” the FDA officials wrote. They argued that current drug research has become “increasingly precise and scientific,” making the decades-old two-trial requirement unnecessarily burdensome.
The policy represents the latest in a series of sweeping changes implemented by Makary’s administration since his appointment last April. Other initiatives have included mandating artificial intelligence use by FDA reviewers and offering expedited one-month drug assessments for medications deemed to serve “national interests.”
The two-study requirement dates back to the early 1960s, when Congress passed legislation requiring the FDA to review data from “adequate and well-controlled investigations” before approving new medications. For most of the agency’s history, this was interpreted as requiring at least two robust clinical trials to confirm efficacy and safety.
Dr. Janet Woodcock, who led the FDA’s drug division for approximately 20 years before retiring in 2024, supported the change. “The scientific point is well taken that as we move toward greater understanding of biology and disease we don’t need to do two trials all the time,” Woodcock said.
In practice, the FDA has already been moving away from the two-trial standard over the past few decades, particularly for treatments targeting rare diseases or life-threatening conditions. Congressional legislation has gradually directed regulators to adopt more flexible approaches for serious or difficult-to-treat conditions.
Data shows that approximately 60% of novel drugs approved in the last five years have been cleared based on a single clinical study. However, the formal policy change announced Wednesday extends this flexibility to medications for more common conditions that previously faced stricter requirements.
“It’s not the cancers and the rare diseases that will be affected by this,” Woodcock noted. “The agency has been approving those on a single trial already.”
Makary and Prasad predicted the change would catalyze “a surge in drug development” by reducing costs and streamlining the path to market for pharmaceutical companies.
The announcement comes at a time of seemingly contradictory regulatory actions from different FDA divisions. While the drug approval standards are being relaxed, the agency’s vaccine division, which Prasad also oversees, has shown signs of increased scrutiny. Last week, the FDA initially rejected Moderna’s application for a new mRNA flu vaccine, citing insufficient clinical trial data, before reversing course after the company agreed to conduct additional studies in older populations.
Similarly, Prasad has recently rejected several experimental gene therapies and biotech drugs, citing the need for more definitive evidence. These decisions have negatively impacted biotech company valuations and appear to conflict with Makary’s public statements promoting faster, more flexible FDA reviews.
Industry observers remain cautious about how the new policy will be implemented in practice. “Implementation will be everything,” Woodcock said. “Since the agency’s approach is unclear, and the industry is already baffled, I don’t think this adds any illumination.”
The policy shift represents one of the most significant changes to the drug approval process in decades and will likely be closely watched by pharmaceutical companies, healthcare providers, and patient advocacy groups in the coming months.
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10 Comments
Reducing the clinical trial burden for new drug approvals could accelerate patient access, but the FDA must be vigilant in ensuring robust data and safety monitoring. Balancing speed and rigour will be crucial.
This shift in the FDA’s drug approval process is intriguing. Increased scientific methodology and data could make the two-trial requirement redundant, but the agency must ensure any changes don’t compromise patient protections.
The FDA’s plan to require just one pivotal study instead of two is a significant policy change. I’m curious to learn more about the agency’s rationale and how they will adapt their oversight to maintain safety standards.
This policy change seems aimed at balancing speed and safety in bringing new drugs to market. I’m curious to see how the FDA plans to maintain high standards while reducing the clinical trial burden.
You raise a good point. Striking the right balance between speed and safety will be critical. The FDA will need robust data monitoring and post-approval surveillance to ensure any risks are quickly identified.
This is a bold move by the FDA, seeking to streamline the drug approval process. While faster access is desirable, I hope the agency has robust plans to maintain high safety standards through other means.
Yes, I agree. The FDA will need to find innovative ways to evaluate drug safety and efficacy without relying solely on the traditional two-study requirement. Continued oversight and post-approval monitoring will be essential.
The FDA’s decision to drop the two-study requirement is an interesting shift, likely driven by advancements in drug development and evaluation methods. However, patient safety must remain the top priority as the agency explores this new approach.
Reducing the clinical trial burden could make drug development more efficient, but I hope the FDA maintains robust data requirements to uphold safety. Faster access is valuable, but not at the expense of rigorous evaluation.
Interesting move by the FDA to streamline the drug approval process. I wonder how this will impact safety and efficacy oversight. Shorter trials could mean faster access, but we must ensure rigorous standards are still met.