Among the health department’s efforts is an expedited investigational new drug pilot program that would leverage collaborations with U.S. research institutions to reduce early trial timelines by as much as 12 months.
The FDA and U.S. health department are doubling down on their efforts to secure clinical trials leadership over China, announcing an “unprecedented” initiative that would reduce the time from drug identification to Phase 1 trials and shorten early trial timelines by as much as a year.
“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,” Health Secretary Robert F. Kennedy Jr. said in a prepared statement on Monday.
To this end, the FDA is putting out a call for public comments on an expedited investigational new drug (IND) pilot program “to accelerate and modernize early and late-stage clinical development.” The proposed pilot would “leverage America’s world-class research institutions as collaborative partners to shorten the time from drug identification to first-in-human study,” use a rolling IND submission platform to create a more flexible and adaptable pre-IND process, “and increase the quality of IND submissions, with the goal of minimizing the need for clinical holds,” according to the program’s webpage.
In February, the FDA officially pivoted from requiring two well-controlled and adequately registrational trials to one. In its press release on Monday, the agency said it has issued draft guidance clarifying that “in many cases, one high-quality late-stage clinical trial with confirmatory evidence will generally be sufficient to provide substantial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.”
The multi-department effort also includes the NIH’s National Center for Advancing Translational Sciences (NCATS) “building on lessons learned from pioneering work that led to the first fully personalized CRISPR-based gene-editing treatment” to accelerate the development of future therapies for patients with rare diseases, HHS said. That first-ever treatment was given last May to Baby KJ Muldoon, who had a rare and potentially fatal genetic condition called CPS1 deficiency.
And the National Cancer Institute is working with cancer centers, researchers and other stakeholders to streamline clinical trial activation and improve enrollment in cancer studies “that have contributed to major advances in patient care,” HHS said.
These new initiatives are part of a broader push to expedite novel medicines to the market that began under former FDA Commissioner Marty Makary. In June 2025, the agency launched the Commissioner’s National Priority Voucher (CNPV) program with the idea of shortening the drug review timeline from 10–12 months to just 1–2 months for companies whose products are “aligned with U.S. national priorities.” These include addressing unmet public health need and increasing domestic drug manufacturing.
So far, seven drugs have won FDA approval under this pathway, including Eli Lilly’s GLP-1 pill Foundayo and a hearing loss gene therapy from Regeneron. The CNPV program has also come under fire, however, for a perceived lack of transparency, particularly around candidate selection, as well as interference from the agency’s senior leaders. Earlier this month, representatives from patient, industry and trade groups called for a temporary pause to the program, citing these issues.
The FDA under Makary also sought to reduce the industry’s reliance on and the agency’s requirement for animal testing. Last April, the agency announced a plan to phase out these requirements for monoclonal antibodies and other therapies in favor of AI and human organoid lab models. Makary was forced out of his position on May 12.
“America led the world in medical innovation before,” Kennedy said in his statement regarding Monday’s clinical trials initiative. “We will lead again.”
