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This policy change aligns with President Donald Trump’s broader push to reduce bureaucracy and cut red tape across federal agencies. Since taking leadership at the FDA last April, Makary has rolled out multiple initiatives designed to accelerate reviews and modernize procedures.
Among those initiatives is a requirement that agency staff incorporate artificial intelligence into the review process. Makary has also offered expedited, one-month reviews for drugs that serve what he calls “national interests.”
Supporters argue that these reforms could unleash a new wave of innovation and investment in American drug development.
Makary and Prasad predict the new framework will result in “a surge in drug development,” potentially bringing life-saving treatments to patients faster than ever before.
The two-trial rule traces back to the early 1960s. After safety scandals rocked the pharmaceutical industry, Congress passed legislation requiring data from “adequate and well-controlled investigations” before new drugs could be approved. Over time, regulators interpreted that to mean at least two major studies.
The logic was simple: if one trial showed promising results, a second trial would confirm the findings and ensure they were not statistical flukes.
However, that rigid approach began to soften in the 1990s. Regulators increasingly allowed single studies for drugs targeting rare or life-threatening diseases where recruiting large patient populations proved difficult.
Over the past five years, nearly 60 percent of first-of-their-kind drugs have already been approved based on just one study. The change announced this week formalizes what had become a growing trend.
Dr. Janet Woodcock, the FDA’s longtime drug director who retired in 2024, said the scientific reasoning behind the shift is sound.
“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.
Importantly, Woodcock noted that many cancer and rare disease treatments were already being approved under single-trial standards. The real impact of this policy could be felt in treatments for more common conditions.
“It’s not the cancers and the rare diseases that will be affected by this,” she noted. “The agency has been approving those on a single trial already.”
Still, not everyone in the biotech world is celebrating just yet.
Recent decisions by the FDA’s vaccine division — also led by Prasad — have raised questions about consistency. The agency initially rejected Moderna’s application for a new mRNA flu shot, citing insufficient clinical data. Days later, regulators reversed course after the company agreed to conduct an additional study in older adults.
At the same time, Prasad has turned away several experimental gene therapies and biotech products, demanding stronger evidence before approval. Those actions have unsettled biotech investors and led to market volatility.
Woodcock acknowledged that industry leaders remain uncertain about how aggressively the new policy will be implemented.
“Implementation will be everything,” she said. “Since the agency’s approach is unclear, and the industry is already baffled, I don’t think this adds any illumination.”
The coming months will determine whether the FDA’s bold new direction leads to a renaissance in American medical innovation — or sparks renewed debate over safety standards.
One thing is certain: Washington has just rewritten a rulebook that has shaped drug approvals for more than half a century.
