What's New in FDA's Latest Cell and Gene Therapy Guidance

Healthcare & Life Sciences attorneys Michael Werner and Sara Klock co-authored a Law360 article analyzing the U.S. Food and Drug Administration's (FDA) new draft guidance on using various agency programs to expedite development and review of cell and gene therapies and other regenerative medicine products. They explain how the guidance updates regenerative medicine advanced therapy (RMAT) designation criteria and processes, encourages flexible trial designs (including use of natural history data and multisite data sharing for clinical trials) and emphasizes enhanced safety monitoring with both short- and long-term assessments, potentially leveraging digital health technologies. The article also highlights that chemistry, manufacturing and controls (CMC) expectations remain unchanged despite accelerated timelines and cautions that manufacturing changes could jeopardize RMAT status. It notes opportunities to use real‑world evidence for the expedited review pathways and broader FDA initiatives to advance therapies for unmet needs, advising sponsors to engage early with the Office of Therapeutic Products and to consider submitting comments before the guidance is finalized.

The attorneys also published a Holland & Knight alert on this topic.

READ: What's New in FDA's Latest Cell and Gene Therapy Guidance