Regenerative medicine regulatory policies: A systematic review and international comparison

Background: A small number of regenerative medicines (RMs) have received market authorization (MA) worldwide, relative to the large number of clinical trials currently being conducted. Regulatory issues constitute one major challenge for the MA of RMs.

Objective: This study aimed to systematically review the regulation of RMs internationally, to identify the regulatory pathways for approved RMs, and to detail expedited programs to stimulate MA process.

Methods: Official websites of regulatory authorities in 9 countries (United States (US), Japan, South Korea, Australia, Canada, New Zealand, Singapore, China, and India) and the European Union (EU) were systematically browsed, and was complemented by a systematic literature review in Medline and Embase database.

Results: Specific RM legislation/frameworks were available in the EU, US, Japan, South Korea and Australia. A risk-based approach exempting eligible RMs from MA regulations were adopted in the EU and 6 countries. All investigated regions have established accelerated review or approval programs to facilitate the MA of RMs. 55 RMs have received MA in 9 countries and the EU. Twenty-three RMs received Priority Medicine designation, 32 RMs received Regenerative Medicine Advanced Therapy designation, and 11 RMs received SAKIGAKE (fore-runner initiative) designation.

Conclusion: Regulators have adopted proactive strategies to facilitate RM approval. However, addressing the discrepancies in regulatory requirements internationally remains challenging.