Framework for the Regulation of Regenerative Medicine Products
The U.S. Food and Drug Administration has published four final guidance documents that are part of a comprehensive policy framework to address how the agency plans to support and expedite the development of regenerative medicine products, including human cells, tissues, and cellular and tissue-based products (HCT/Ps). These guidance documents build upon FDA’s risk-based, flexible regulatory framework, and underscore the agency’s commitment to help bring new and innovative treatment options to patients.
The first two final guidance documents are:
- Regulatory Considerations for Human Cell, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use
- Same Surgical Procedure Exception: Questions and Answers Regarding the Scope of the Exception
The final guidance on minimal manipulation and homologous use are intended to provide clarity in the determination of whether HCT/Ps are subject to FDA’s premarket review requirements. The final guidance on the same surgical procedure exception is intended to provide clarity as to whether an establishment may qualify for an exception from the requirements under Part 1271 by meeting the exception in 21 CFR 1271.15(b).
The FDA also published two final guidances intended to aid in the effort to bring innovative, safe, and effective products to patients as efficiently as possible:
- Expedited Programs for Regenerative Medicine Therapies for Serious Conditions
- Evaluation of Devices Used with Regenerative Medicine Advanced Therapies
The final guidance on expedited programs describes several programs, such as Fast Track designation and Breakthrough Therapy designation, that are available to sponsors of regenerative medicine therapies, and information about the requirements for, and benefits of, the new RMAT designation program that was created by the 21st Century Cures Act. The final device guidance, which FDA is publishing as required by section 3034 of the 21st Century Cures Act, provides the agency’s current thinking about concepts related to the evaluation of devices used in the recovery, isolation and delivery of RMATs.