The FDA wants drugmakers to run one large clinical trial instead of two before seeking approval, and it is making that case explicitly as a tool for competing with China.
Commissioner Marty Makary and Center for Biologics Evaluation and Research (CBER) director Vinay Prasad outlined the shift in a New England Journal of Medicine perspective last month: going forward, the FDA's default position will be to require one pivotal study for new drugs and other novel health products, according to the Associated Press. The change is framed not as patient-advocacy language, the agency's traditional rationale for acceleration, but as industrial strategy. "We are in a race against China," the authors wrote.
The two-study standard dates to the early 1960s, when Congress passed a law requiring the FDA to review data from adequate and well-controlled investigations. The agency now argues that modern drug research has grown precise enough that one rigorous trial, designed with input from the FDA itself, can generate the evidence needed for approval. Over the last five years, roughly 60 percent of first-of-a-kind drugs approved each year were already cleared based on a single study, per AP. The new policy makes that tendency the rule rather than the exception.
China's trajectory is what prompted the reframe. Data from Global Data and Morgan Stanley shows that China now conducts more clinical trials than the United States, accounts for nearly a third of new global drug approvals, and is on pace to reach 35 percent of FDA approvals by 2040, CNBC reported. China grew its share of clinical research to 39 percent in 2023, beating the U.S. and European Union in patient recruitment and development timelines, BioSpace reported. A McKinsey analysis, cited by BioSpace, found that China has managed to speed the timeline from early discovery to an investigational new drug (IND) application by 50 to 70 percent compared to U.S. benchmarks.
Makary cited three structural bottlenecks keeping the U.S. noncompetitive in early trials: hospital contracting, ethical reviews and approvals, and the IND application process itself, which he called "clunky" and "leaving us noncompetitive with countries that are moving a lot faster," per CNBC. The agency plans to reduce the amount of data required to start a trial, cutting out most parts that are not essential, Endpoints News reported.
Former FDA commissioner Scott Gottlieb offered a narrower diagnosis of China's advantage in remarks reported by Fierce Biotech: because launching first-in-human clinical trials is simpler in China, Chinese biotech companies get a competitive advantage by more quickly obtaining information on the most promising compounds.
Janet Woodcock, the former FDA commissioner, said the one-trial shift makes sense but noted it will mainly affect drugs for common diseases, not rare diseases or cancers, which the agency has been approving on a single trial basis for years already. "It's not the cancers and the rare diseases that will be affected by this," she told AP. "The agency has been approving those on a single trial already."
The timing is complicated by internal turbulence at the agency. One of the FDA's main review offices, the Center for Drug Evaluation and Research (CDER), is currently on its fifth director since January 2025. Tracy Beth Høeg, the current acting director, is not an agency veteran. The FDA's other big drug evaluation office, CBER, has seen its director Vinay Prasad twice step down over the course of a year, BioPharma Dive reported. Meanwhile, the FDA oncology review staff had previously been about 100 people and is now headed below 60, per CNBC, and accelerated approvals fell from 20 in 2024 to 9 in 2025.
What to watch next is whether the FDA can execute the acceleration without a stable review staff, and whether the one-trial default actually reduces development time or simply shifts the risk of failure earlier in the process. China's competitive position is not primarily a regulatory problem. It is also a hospital-contracting and institutional-capacity problem, and changing the trial count does not automatically fix either one.
The drug industry has spent decades building development playbooks around the two-study requirement. One pivotal trial as the default rewrites that playbook and puts more weight on trial design, FDA alignment, and luck. Whether that trade is worth it depends on whether the drugs that reach approval faster are actually the right ones.
† Add footnote: "Source-reported; not independently verified." Or seek independent corroboration of the McKinsey finding.
† Add footnote: "Source-reported; not independently verified." Or seek independent corroboration of the McKinsey finding.
