Enrique Seoane-Vazquez, Ph.D., has been awarded a two-year, multi–Principal Investigator grant for his project, “Advanced Therapy Medicinal Products: Clinical and Patient-Centered Outcomes.”

The project will conduct the most comprehensive evaluation to date of Advanced Therapy Medicinal Products (ATMPs) between 2010 and 2025, and is funded by Arnold Ventures. The study will assess:

  • Safety, efficacy, and clinical outcomes at the time of FDA approval,
  • Post-marketing requirements and real-world evidence,
  • Strengths and limitations of accelerated approval pathways, including the Regenerative Medicine Advanced Therapy (RMAT) designation, and
  • Economic and population-level impact of ATMPs

ATMPs, including gene therapies, cell therapies, and tissue-engineered products, provide life-changing new treatment options for patients with severe and life-threatening diseases. Since the FDA approved the first cell therapy in 2010, sipuleucel-T, 37 ATMPs have been authorized in the U.S., including 26 gene therapies and 11 cell and tissue-based products. The pace of approvals continues to accelerate, with nine ATMPs approved in 2024 alone.

“Advanced therapies hold extraordinary promise, but their rapid development has outpaced our understanding of their long-term safety, actual performance, and economic impact,” says Seoane-Vazquez. “This project is a critical opportunity to evaluate the evidence behind select treatments and strengthen the guidelines that outline their use.”

While these therapies hold tremendous promise, many are approved based on limited clinical evidence from small, single-arm, or non-randomized trials. These constraints, which are often driven by the rarity and severity of targeted conditions, raise important questions about evidence quality, long-term safety, and the strength of the regulatory framework guiding ATMP development.