Risk evaluation and mitigation strategies for approved oncology drug and non-cellular biologic products from 2008 to 2025: An FDA analysis.

Abstract

e23029 Background: The Food and Drug Administration Amendments Act of 2007 (FDAAA) granted FDA the authority to require Risk Evaluation and Mitigation Systems (REMS), when necessary, to help ensure that the benefits of an otherwise effective therapy outweigh serious risks. We sought to review and summarize FDA approved REMS for oncology drug and non-cellular biologic products. Methods: We examined FDA’s databases for REMS required for FDA oncology drug and non-cellular biologic products approved from January 1, 2008, to December 31, 2025. We characterized approved REMS for all products as well as for novel products (defined as new molecular entities and original biologics) approved in 3 successive 6-year periods: 2008-2013; 2014-2019; and 2020-2025. Results: Our analysis showed that 27 REMS were required for products under FDA Oncology’s purview. Of these, 56% were for products with hematologic malignancy indications; 56% required ≥ 1 element to assure safe use (ETASU); and 52% are still active. Of these 27 REMS, 20 were for novel products approved after FDAAA enactment, corresponding to 9% of all novel oncology products approved since 2008 (vs. 10% for novel non-oncology products). There were proportionally more REMS required for novel oncology products approved in 2008-2013 ( n = 8 [18%]) than in 2014-2019 ( n = 6 [8%]) or in 2020-2025 ( n = 6 [7%]). Conclusions: The majority of approved non-cellular oncologic (and related) products in the U.S have not required REMS to ensure that their respective benefits outweigh their risks, and most recent novel oncology drugs were approved without a REMS. However, when REMS were required, they often included ETASUs. Since REMS with ETASU have the potential to increase burden on the healthcare system and/or adversely impact patient access, REMS for oncology products are continuously being re-evaluated to ensure their ongoing relevance to, and adequacy for, the mitigation of product-specific risks to ensure that their potential benefit(s) outweigh serious risks to the American public. Characteristics of REMS for FDA-approved oncology drug and non-cellular biologic products since FDAAA enactment. REMS for all oncology products( N =27) * REMS for novel oncology products2008-2013( N =8) REMS for novel oncology products2014-2019( N =6) REMS for novel oncology products2020-2025( N =6) Accelerated Approval, n (%) 10 (40) ** 3 (38) 3 (50) 4 (67) REMS at Initial approval, n (%) 20 (95) *** 7 (88) 6 (100) 6 (100) Hematological malignancies, n (%) 15 (56) 2 (25) 4 (67) 6 (100) REMS with ≥ 1 ETASU, n (%) 15 (56) 4 (50) 2 (33) 6 (100) Currently active REMS, n (%) 14 (52) 1 (13) 3 (50) 6 (100) *Includes REMS for 6 products already approved prior to 2008 and 1 non-novel product approved in 2023; **Denominator excluded 2 products approved prior to accelerated approval legislation passed in 1991; ***Excludes REMS for 6 products already approved prior to 2008.