The U.S. Department of Health and Human Services (HHS) has launched Operation Trailblazer, aiming to enhance clinical research in the U.S., according to an announcement.

The goal of this action is to stay ahead on the development of lifesaving treatments for patients.

Key points:

• The Food and Drug Administration (FDA) is working on launching a pilot program to expedite the drug identification to human Phase 1 clinical trial process. They are seeking feedback on the program proposal. The agency has published draft guidance stating that in several instances, only one “high-quality late-stage clinical trial with confirmatory evidence will generally be sufficient to provide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 National Institutes of Health (NIH) will focus on maintaining clinical trial integrity while promoting efficiency, responsible AI use, human cell models, and real-world data.
• The National Center for Advancing Translational Sciences (NCATS) will advance CRISPR therapies, implementing lessons learned from previous research.
• The National Cancer Institute (NCI), along with cancer centers, researchers, and stakeholders, will optimize cancer clinical trial activation and enrollment.
• The Office of the National Coordinator for Health Information Technology (ONC) is looking to leverage electronic health records to more effectively match patients and clinical trials and expand participation.
• The Advanced Research Projects Agency for Health (ARPA-H) has initiated Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) and Computational ADME-Tox and Physiology Analysis for Safer Therapeutics (CATALYST).
• The HHS Office of Inspector General is asking for public insights on if anything needs to be added or changed regarding anti-kickbacks and clinical trial participation, keeping in line with Federal regulations.