The US Department of Health and Human Services announced a coordinated, department-wide effort to strengthen the country's position in clinical research and accelerate development of new treatments, according to an HHS press release. A companion HHS roadmap, Operation TrialBlazer, outlines the initiative.
The roadmap frames the effort as a response to increasing global competition in clinical research. According to the roadmap, China's share of global Phase 1 trials surpassed that of the US for the first time in 2021, and by 2024 China had registered more than 7,100 clinical trials, accounting for 39% of the global total and surpassing the US in total trial volume. The roadmap also cites a 2025 figure showing that global companies spent more than $137 billion licensing China-based assets and references a projection that, if current trends continue, Chinese-developed drugs could account for 35% of FDA approvals by 2040.
The roadmap attributes part of the gap to differences in clinical trial timelines. It notes that Australia's Clinical Trial Notification System allows trials to begin fewer than 70 days after a final protocol is submitted, with regulatory approval granted in as little as 21 to 28 days and sites activated within 6 to 12 weeks. China, following regulatory reforms, now moves from discovery to an investigational new drug (IND) application 50% to 70% faster than the rest of the world, according to the roadmap, and requires IND applicants to commit to starting their trial within 12 weeks of submission. By contrast, the roadmap states that the average time between a US sponsor's pre-IND meeting request and IND submission is on average 380 days, with a range approaching 700 days, and that Institutional Review Board (IRB) approval and contract negotiation can add up to 13 additional months before a single patient is enrolled.
The initiative spans multiple HHS agencies. According to the release, the Food and Drug Administration (FDA) is seeking public comments on a proposed pilot program intended to shorten the time from drug identification to first-in-human Phase 1 trials. HHS said FDA is also clarifying regulatory expectations for sponsors that could reduce early trial timelines by 6 to 12 months.
The roadmap identifies the effort as the Expedited-IND Acceleration Pilot, which would establish a network of "Qualified Research Institutions," including academic medical centers, health care networks, contract research organizations, and regulatory advisors, to review components of sponsors' IND submissions alongside a new FDA rolling-review platform. Sponsors would retain ownership of their IND applications, and FDA would retain full regulatory authority over the review process.
The roadmap also describes proposed changes to nonclinical and manufacturing requirements. It states that the FDA is describing a risk-based approach to chemistry, manufacturing, and controls data, noting that some sponsors currently submit more than 6 months of stability data when agency guidance recommends data sufficient to support the proposed duration of the Phase 1 study. FDA is also exploring circumstances in which a single animal species, or non-animal methods, may be sufficient for toxicology evaluation rather than the historical use of two animal species. Separately, the roadmap cites an HHS study finding that 45% of protocol amendments were somewhat or completely avoidable and references an HHS estimate that reducing unnecessary amendments could lower the overall cost of bringing a drug to market by up to 22%.
On trial startup and enrollment, the roadmap states that the FDA is considering rulemaking to require the use of a single IRB model for multisite cooperative studies, aligning FDA regulations with existing federal policy under the Common Rule and NIH policy. The roadmap also cites a 2024 study finding that fewer than 5% of patients with cancer receive treatment through a clinical trial despite more than 70% reporting willingness to participate.
The FDA has launched a Phase 1 Clinical Trial Contact Center to answer sponsor questions in real time and plans to create a centralized online resource outlining phase-appropriate IND requirements. The FDA has also issued draft guidance stating that, in many cases, one high-quality late-stage trial with confirmatory evidence will generally suffice as substantial evidence of effectiveness for drug approval. The roadmap also highlights separate draft guidance on master protocols, which allow multiple substudies to operate under a single overarching clinical trial framework.
The National Institutes of Health (NIH) is increasing support for well-powered clinical trials while promoting responsible use of artificial intelligence, human cell-based models, real-world data, and practical trial tools intended to move therapies to patients more efficiently. Within NIH, the National Center for Advancing Translational Sciences is building on earlier work that produced the first fully personalized CRISPR-based gene-editing treatment, with the goal of accelerating development of future therapies for patients with rare diseases.
The National Cancer Institute is collaborating with cancer centers, researchers, and other stakeholders to streamline trial activation and improve enrollment in cancer studies. The roadmap adds that NIH plans to expand use of SMART IRB, a national reliance platform for multisite studies, and update its 1998 Data and Safety Monitoring Policy.
The Office of the National Coordinator for Health Information Technology is exploring ways to connect patients to clinical trials through electronic health records during routine care. The roadmap notes that ONC-certified electronic health records are used by approximately 99% of hospitals and 90% of providers and states that ONC may propose integrating certified systems with the ClinicalTrials.gov database to support eligibility screening during clinical care.
The Advanced Research Projects Agency for Health (ARPA-H) has launched several programs relevant to the initiative. Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) and Computational ADME-Tox and Physiology Analysis for Safer Therapeutics (CATALYST) are developing approaches intended to test multiple therapies or disease targets within more flexible development structures and to use artificial intelligence and machine learning to predict safety, optimize dosing, and improve trial efficiency before patient enrollment begins.
The roadmap also describes two additional ARPA-H programs, ENGINE and UNICORN, which focus on improving consistency, scalability, and predictability in manufacturing and clinical evaluation of advanced therapies, including cell and gene therapies.
Separately, the HHS Office of Inspector General is publishing a request for information seeking public input on whether safe harbor regulations under the federal anti-kickback statute, or civil monetary penalty exceptions related to beneficiary remuneration for trial participation, should be modified.
HHS said it will continue working with industry, academic medical centers, contract research organizations, patient groups, health care providers, and other stakeholders to identify and remove barriers to clinical research. The roadmap states that HHS plans to hold a public roundtable series and open a feedback docket to gather additional stakeholder input on IND processes, IRB reform, clinical trial initiation, and participant payment policy.
"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," HHS Secretary Robert F. Kennedy, Jr., said in the release. "Under President Trump's leadership, HHS is launching a coordinated department-wide effort to restore America's leadership in clinical research, remove unnecessary barriers, and bring more clinical research and investment back to the United States."
Sources: HHS Press Office