The Food and Drug Administration and the U.S. Department of Health and Human Services announced on June 22 a new initiative aimed at strengthening American leadership in clinical trials by reducing the time from drug identification to Phase 1 studies and shortening early trial timelines by up to a year.
Health Secretary Robert F. Kennedy Jr. said in a prepared statement, “America should be the best place in the world to develop new medicines, yet we have built a system that drives too much clinical research overseas.”
As part of this effort, the FDA is seeking public comments on an expedited investigational new drug pilot program designed to modernize early and late-stage clinical development. The proposed pilot would use America’s research institutions as collaborative partners, introduce a rolling IND submission platform for more flexibility, and aim to increase submission quality while minimizing clinical holds.
In February, the FDA shifted its policy from requiring two well-controlled registrational trials for approval to generally accepting one high-quality late-stage trial with confirmatory evidence. The agency said draft guidance clarifies that, “in many cases, one high-quality late-stage clinical trial with confirmatory evidence will generally be sufficient to provide substantial evidence of effectiveness in support of a drug approval.” Chief regulators Marty Makary and Vinay Prasad said applications should include confirmative evidence such as mechanistic data or findings from related indications or animal models.
The multi-department effort also involves the National Institutes of Health’s National Center for Advancing Translational Sciences building on work that led to the first fully personalized CRISPR-based gene-editing treatment last May for Baby KJ Muldoon with CPS1 deficiency. The National Cancer Institute is working with cancer centers and researchers to streamline trial activation and improve enrollment in cancer studies.
These actions follow previous initiatives under former FDA Commissioner Marty Makary, including launching the Commissioner’s National Priority Voucher program intended to reduce review times for drugs aligned with national priorities. Seven drugs have been approved through this pathway so far, but concerns about transparency have led some groups to call for a temporary pause in the program. The FDA has also begun phasing out animal testing requirements for certain therapies in favor of AI models.
