The U.S. FDA announced actions to accelerate and modernize clinical research across the full continuum of drug development —from the Investigational New Drug (IND) phase to late-stage pivotal trials. The FDA’s work is outlined in Operation TrialBlazer, a U.S. Department of Health and Human Services (HHS) initiative.
The actions are divided into:
1. Early stage actions
The FDA is committed to ensuring the US remains the global standard for pharmaceutical innovation and regulatory rigor for the benefit of American patients and innovators. The agency is eliminating unnecessary regulatory burden, clarifying phase-appropriate requirements, and building partnerships with government, academic medical centers and the private sector.
Early stage actions include the following. Click on each link for further information.
- Expedited Investigational New Drug Pilot Program Request for Information
- Phase 1 IND Navigator Webpage
- Phase 1 IND Chemistry, Manufacturing, and Controls (CMC) Webpage
- Phase 1 Contact Center – Complementing the Phase 1 webpage is a Phase 1 Contact Center, reachable at 240-276-9358 or Phase1Questions@fda.hhs.gov.
- Quantitative Systems Pharmacology-Based Dose Selection for Minimum Anticipated Biological Effect Level in First-in-Human Trials – Draft Guidance
2. Late-Stage Trial Actions
The FDA is enhancing its guidance for industry to facilitate greater efficiency further along the drug development continuum.
The following guidance is provided:
- Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products – Revised Draft Guidance
- Master Protocols for Drug and Biological Product Development – Revised Draft Guidance
This HHS roadmap is a coordinated response to a genuine and growing
competitive challenge with real consequences for American patients, industry,
and national security. This roadmap addresses that challenge across six
interconnected areas:
- Modernizing regulatory requirement.
- Improving transparency for regulated entities.
- Encouraging the adoption of more efficient practices.
- Ensuring federal funding dollars are spent on adequately powered and designed trials.
- Better utilizing existing data sources and technologies for regulatory and data generation purposes.
- Improving patient access to clinical trials and removing disincentives
- Preventing healthcare workers from being involved in the conduct of research
All of this work is an ongoing, iterative process: The FDA will continue to collaborate, engage stakeholders, and innovate to ensure our guidance remains relevant, science-based, and responsive to the realities of modern drug development.
Further information is also available on the HHS website