Words by Isabel O’Brien
The FDA has announced the elimination of Risk Evaluation and Mitigation Strategies (REMS) for gene therapies used to treat certain blood cancers, potentially simplifying how patients access these treatments in the future.
The new ruling is applicable to BCMA- and CD19-therapies that are used to treat diseases such as multiple myeloma and specific types of leukaemia. REMS is a safety programme that the FDA can require for medicines with serious safety concerns to help ensure the benefits of the medication outweigh its risks.
The US regulator stated that it believes the safety and effectiveness of these immunotherapies can be assured without a REMS, following a period of clinical experience and monitoring. The agency also noted that removing REMS would reduce the administrative burden on providers treating patients in clinics.
“The FDA has taken the bold step to remove the Risk Evaluation and Mitigation Strategy requirement from giving CAR T therapies. REMS is a useful safety system, but re-evaluation over time helps inform whether a REMS is still needed to ensure that the benefits of a product outweigh its risks,” said Dr Vinay Prasad, Chief Medical and Scientific Officer and Director, Center for Biologics Evaluation and Research, FDA.
Dr Richard Pazdur, Oncology Center of Excellence Director, FDA, added: “Physicians and institutions now have greater experience identifying and managing toxicities with the currently approved CAR T products. This approach will potentially facilitate patient access to these treatments while continuing to prioritise safety.”
The FDA confirmed that safety monitoring will continue through adverse event reporting and mandatory post-marketing observational studies, including long-term follow-up for up to 15 years after product administration. The agency added that it remains committed to ongoing oversight and will inform the public of any new safety information as it arises.
