Guidance on Long-Term Follow-Up Clinical Studies for Chimeric Antigen Receptor T-Cell (CAR-T) Therapy Products Officially Released as a Group Standard

On September 27, after multiple rounds of discussion and revision by experts, the "Guidelines for Long-term Follow-up Clinical Studies of Chimeric Antigen Receptor T-cell (CAR-T) Therapy Products" (T/SHPPA 023-2023) group standard was officially released on the National Group Standard Information Platform and will be officially implemented starting from October 25, 2023. This standard was proposed by the Shanghai Center for Adverse Drug Reaction and Medical Device Monitoring (hereinafter referred to as the "Monitoring Center"), with drafting organizations including the Shanghai Center for Adverse Drug Reaction and Medical Device Monitoring, Fosun Kite Biotechnology Co., Ltd., JW Therapeutics, and the Shanghai Pharmaceutical Industry Association.

In recent years, with the increasing number of patients receiving CAR-T cell therapy, the need for long-term safety monitoring in patient follow-up has become more prominent. In 2021, the CDE released the "Technical Guidelines for Long-term Follow-up Clinical Studies of Gene Therapy Products," providing technical guidance for conducting long-term follow-up clinical studies for such products. However, in practice, post-marketing long-term follow-up of CAR-T treatment products still faces many challenges, such as the large number of CAR-T treatment centers, high patient mobility, a high rate of loss to follow-up over the 15-year period, and insufficient experience in the specific design and implementation management of follow-up studies.

This standard is the first group standard drafted and released by the Monitoring Center, which focuses on the long-term follow-up of CAR-T treatment products after their market launch. It fills a gap in China's industry and provides an operational guidance document for the design and implementation management of long-term follow-up clinical studies for autologous CAR-T cell therapy product marketing authorization holders and related parties. This will help ensure that holders promptly collect signals of delayed adverse reactions, identify and mitigate risks, guide the standardized development of the industry, and safeguard patient medication safety.