Studies on the Comparison of the Expedited Programs for Regenerative Medicine Therapies among the United States, the European Union and the Republic of Korea
10.24304/kjcp.2025.35.1.45
- Author:
ChaeEun KO
1
;
Jangik I. LEE
Author Information
1. College of Pharmacy, Seoul National University, Seoul 08826, Republic of Korea
- Publication Type:Original Article
- From:Korean Journal of Clinical Pharmacy
2025;35(1):45-64
- CountryRepublic of Korea
- Language:English
-
Abstract:
Background:The authors performed gap analyses on the expedited programs associated with regenerative medicine therapies(RMTs), offered by Food and Drug Administration (FDA), European Medicines Agency (EMA) and Ministry of Food and Drug Safety (MFDS). ethods: The regulations and guidelines on expedited programs (e.g., Regenerative Medicine Advanced Therapy [RMAT] designation) associated with RMTs, and regulatory reviews on approved RMTs were retrieved from the websites of FDA, EMA and MFDS. Based on analyses of the retrieved materials, gap analyses were performed on the qualifying criteria, features and operational frameworks. Included were the comparisons of the expedited programs granted for tisagenlecleucel among the agencies.
Results:FDA offers RMAT, Fast Track, Breakthrough Therapy, Priority Review designations, and Accelerated Approval. EMA’s programs include PRIority MEdicines (PRIME), Accelerated Assessment, Exceptional Circumstances, and Conditional Marketing Authorisation. MFDS implements a single Fast-Track Processing program. RMAT designation is specific to RMTs and can be utilized with FDA’s other expedited programs. PRIME applies to all medicinal products and can be used with EMA’s other programs.Fast-Track Processing program is specific to RMTs but combines all expedited features into a single track. Tisagenlecleucel received RMAT, Priority Review and Accelerated Approval for follicular lymphoma from FDA, PRIME for acute lymphocytic leukemia from EMA, and Fast-Track Processing for follicular lymphoma from MFDS.
Conclusion:FDA offers the RMAT designation exclusive to expediting the development, review and approval of RMTs in addition to other expedited programs, whereas EMA does RMT approvals under expedited drug approval programs. MFDS implements an exclusive, single-track program for RMTs.
