Trends in Erythropoiesis‐stimulating agent use and blood transfusions for chemotherapy‐induced anemia throughout FDA 's risk evaluation and mitigation strategy lifecycle

Purpose Erythropoiesis‐stimulating agents (ESAs), indicated for treating some patients with chemotherapy‐induced anemia (CIA), may increase the risk of tumor progression and mortality. FDA required a Risk Evaluation and Mitigation Strategy (REMS) to mitigate these risks. We assessed REMS impact on ESA administration and red blood cell (RBC) transfusion as surrogate metrics for REMS effectiveness. Methods Retrospective cohort study including data from January 1, 2006 to December 31, 2018 for beneficiaries ≥65 years enrolled in Centers for Medicare & Medicaid Services (CMS) Medicare Parts A/B with a cancer diagnosis; patients with other indications for ESA use were excluded. Study time was divided into five periods demarcated by issuance of CMS National Coverage Determination (NCD) (Pre‐NCD, Pre‐REMS) and REMS milestones (Grace Period, REMS, post‐REMS). Study outcomes were monthly proportion of chemotherapy episodes (CTEs) with concomitant ESA administration, with post‐CTE ESA administration, and with RBC transfusions. Results Of 1 778 855 beneficiaries treated with CT, 308742 received concomitant ESA for CIA. The proportion of CTEs with concomitant and post‐CTE ESA administration decreased Pre‐REMS (9.0 percentage points [pp] and 3.5 pp, respectively). There were no significant post‐REMS changes in the proportion of CTEs with concomitant (0.0 pp) and post‐CTE ESA administration (0.1 pp). Fluctuation in RBC transfusions was <4 pp throughout the study period. Conclusions Medicare beneficiaries showed a substantive decrease in ESA administration after NCD, with minimal impact by the REMS and its removal. Small changes in RBC transfusion over the study period were likely due to a national secular trend.