The news: Pharma and biotech companies will soon be able to seek drug approvals with just one clinical trial instead of the standard two under upcoming policy changes from FDA chief Marty Makary, per STAT.

• The new one pivotal trial requirement that Makary deemed “more practical” is meant to help speed up reviews and approvals.
• Some drug reviewers, along with Richard Pazdur, the top drug regulator who suddenly announced his retirement this week, are concerned the new policy will weaken the agency’s ability to request additional evidence, STAT reported.
• The one-trial policy is expected to roll out in three to six months.

Why it matters: One-trial reviews are already used when running two would be too difficult or not feasible, but the new policy changes the standard for most drug and medical devices.

• 66% of the 50 novel drugs approved in 2024 only used one pivotal trial, per an AgencyIQ analysis in February.
• In 2019, just 38% were based on a single study, with a previous high of 68% in 2021.
• 70% of the drugs approved last year on the basis of a single trial were orphan drugs which are therapies for a rare disease that affects 200,000 or fewer people. Oncology represented the largest category, with 14 approvals, according to AgencyIQ.
• Clinical trials cost an average of $117 million and are the most expensive part of drug development, accounting for 68% of R&D spending, according to an Eastern Research Group study for HHS in October 2024.

However, a leaked memo by FDA vaccine chief Vinay Prasad last week points in the opposite direction for vaccine approvals. The memo suggests the agency will shift to requiring randomized controlled trials for vaccine approvals. Currently, vaccines like pneumonia and seasonal flu shots are approved based on antibody data.

Implications for pharma and biotech companies: The FDA appears to be sending mixed signals. While some drugmakers may benefit from the shift to a one-study standard by cutting both costs and timelines, vaccine manufacturers are facing pressure to produce more evidence of effectiveness. Two independent trials have long helped reinforce safety and efficacy—and bolster public confidence—but raising the bar specifically for vaccines risks doing the opposite. It could undermine trust in an established approval system and make an already tough vaccine market even less attractive for manufacturers.

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