Objective: China has proactively adopted regulatory science (RS) to advance its regulatory capacity for advanced therapy medicinal products (ATMPs) but the reporting is scarce. The study aimed to identify the core actions in advancing RS and summarize the recent development of ATMPs in China.
Method: A mixed approach of documentary analysis of government policies, regulations and official information about ATMPs regulation in China, and a scoping review of the clinical registration, marketing application and approval of ATMPs from the Center for Drug Evaluation of NMPA (CDE) was employed.
Results: The first Regulatory Science Action Plan (RSAP) in China, first launched in 2019 and later updated in 2021, repeatedly emphasized on developing new regulatory tools, methods and standards for the technical evaluation and regulatory approaches of cell and gene therapy products. The scope spanned across quality control, non-clinical research, clinical study design, clinical outcome evaluation, as well as the use of real world data to improve regulation quality and efficiency. Since the RSAP, the review and regulatory systems and standards for ATMPs are constantly reviewed, specific consultation papers on different ATMPs (such as CAR-T (n=1), stem cell (n=2), immune cell (n=2), and gene therapy (n=1)) have been circulated and 9 technical guidelines specific to cell and gene therapies have been issued (such as CAR-T (n=1), immune cell (n=2), gene therapy (n=5), and oncolytic virus (n=1)). According to CDE, the number of ATMPs currently registered at the CDE is 151 (CAR-T (n=56), oncolytic virus (n=30), stem cell (n=24), gene therapy (n=22), other cell therapies (n=19)), of which 115 have been registered since RSAP. Most ATMPs under investigation are cancer-related (n=107), with CD19 (n=43), BCMA (n=8) and CSF2 (n=7) being the most common targets in clinical studies. As of 2003, the number of clinical trial applications for ATMPs as a new drug was 265 (antineoplastic drugs=199, immune system drugs=46), of which the number after RSAP was 173.
Conclusion: Advancing RS helps promote the advancement of the regulatory and technical evaluation systems of ATMPs in China, facilitating scientific regulation and regulatory standardization. The development of ATMP is still at an early stage. Strategically adopting RS to promote the successful clinical translation of ATMP innovation may also benefit reaching consensus across different drug regulatory authorities (DRAs).

Advancing regulatory science for developing advanced therapy medicinal products in China

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