FDA Drops Two-Study Requirement for Drug Approvals to Speed Access

The Food and Drug Administration plans to drop its longtime standard of requiring two rigorous studies to win approval for new drugs, the latest change from officials vowing to speed up the availability of certain medical products. Going forward, the FDA's "default position" will be to require one study for new drugs and other novel health products, FDA Commissioner Dr. Marty Makary and a top deputy wrote in a New England Journal of Medicine piece published Wednesday. The announcement is the latest example of Makary and his team changing longstanding FDA standards and procedures with the stated goal of slashing bureaucracy and accelerating the availability of new medicines.

"We want to challenge the assumption that it takes 10 to 12 years for a drug to come to market," Makary said during a press conference with reporters. "We believe it can be done faster without cutting any corners on safety. We'd like to modernize the agency with technology, while maintaining our gold-standard thresholds for approving drugs, devices, food, cosmetics, and tobacco."

The FDA officials predicted the shift would lead to "a surge in drug development." In their piece published Wednesday, Makary and his deputy state that dropping the two-trial requirement reflects modern advances that have made drug research "increasingly precise and scientific." "In this setting, overreliance on two trials no longer makes sense," they write. "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 two-study standard for drugs dates to the early 1960s, when Congress passed a law requiring the FDA to review data from "adequate and well-controlled investigations," before clearing new medications. For decades, the agency interpreted that requirement as meaning at least two studies, preferably with a large number of patients and significant follow-up time. The reason for requiring the second study was to confirm that the first trial's results weren't a fluke and could be reproduced.

But beginning in the 1990s, the FDA increasingly began accepting single studies for the approval of treatments for rare or fatal diseases that companies often struggle to test in large numbers of patients. Over the last five years, roughly 60% of first-of-a-kind drugs approved each year have been cleared based on a single study. The shift reflects laws passed by Congress that directed regulators to be more flexible when reviewing drugs for serious or hard-to-treat conditions.

The FDA's former drug director said the change makes sense and reflects the FDA's decades-long move toward relying on one trial, combined with supporting evidence, for various life-threatening diseases, including cancer. "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," said the former director, who led the FDA's drug center for about 20 years before retiring in 2024.

The new policy announced Wednesday will mainly impact drugs for common diseases that previously weren't eligible for reduced testing standards. "It's not the cancers and the rare diseases that will be affected by this," the former director noted. "The agency has been approving those on a single trial already."

Since arriving at the agency last April, Makary has launched a series of directives that he says will shorten FDA reviews, including mandating the use of artificial intelligence by staffers and offering one-month drug assessments for new medications that serve "national interests." He noted that the FDA approved 67 medications last year, more than the 5-year average (65), the 10-year average (64), and the 15-year average (60).

One of the agency's big priorities is to reduce the idle time, use technology, and prioritize drug applications, he said, touting the pilot National Priority Voucher Program, which addresses those issues. "We are giving vouchers to drugs that can meet an unmet public health need, companies that are making steps towards affordability -- to summarize, I would say drugs that are game-changing drugs," Makary said. "Speed is a high priority for all therapies not just rare diseases. Last year we created the National Priority Voucher Program which can accelerate the review [of drugs] from 10 to 12 months to two months," Makary added. He noted that about a third of the recipients of the voucher program are for rare diseases.

One drug being evaluated for possible approval under the program is a gene therapy known as DB-OTO for a congenital form of deafness. "When you see an amazing set of results, as was reported in the New England Journal of Medicine, that is the type of game-changing finding that we want to see delivered to patients rapidly, without cutting any corners on safety," he noted.

The latest approach from FDA leadership contrasts with the agency's recent actions on vaccines, gene therapies and other treatments. Last week, the FDA's vaccine division refused to accept Moderna's application for a new mRNA flu shot, saying its clinical trial was insufficient. Then on Wednesday the agency reversed course, saying it would review the vaccine after Moderna agreed to conduct an additional study in older people. Separately, the deputy has rejected a string of experimental gene therapies and biotech drugs, citing the need for additional studies or more definitive evidence.

The former drug director said the drug industry will have to wait and see whether the FDA's approach to promising experimental therapies changes. "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."