The FDA plans to drop its long-standing requirement that drugmakers conduct two rigorous studies to secure approval for new drugs and is also pushing to expand over-the-counter access to medications.
Going forward, the FDA’s “default position” will be to require one study for new drugs and other novel health products, FDA Commissioner Marty Makary, MD, and Vinay Prasad, MD, wrote in a New England Journal of Medicine article published Feb. 18. The two-study standard dates to the early 1960s, when Congress required the agency to review data from “adequate and well-controlled investigations.” Over the last five years, roughly 60% of first-of-a-kind drugs approved each year have been cleared based on a single study.
Dr. Makary also told CNBC in a Feb. 18 report that “everything should be over the counter” unless a drug is unsafe, addictive or requires monitoring. He said the FDA is going through “the proper regulatory processes” to update over-the-counter monographs — rulebooks that determine which drugs can be sold without a prescription — and is looking at prescription drugs such as nausea medications and vaginal estrogen.
Congress passed legislation in November to streamline the regulatory process for prescription-to-over-the-counter transitions, including full, conditional and partial “switch” pathways.
Some industry groups pushed back on the FDA’s plans, arguing that most over-the-counter drugs are not covered by insurance and that pricing considerations may not factor into FDA regulatory decision-making, according to CNBC.
Janet Woodcock, MD, who led the agency’s drug center for about 20 years before retiring in 2024, told CNBC the single-study shift reflects a broader move toward more flexible approvals and will mainly affect drugs for common diseases that previously were not eligible for reduced testing standards.
