Patient Perceptions of and Experiences with Risk Evaluation and Mitigation Strategies

Risk Evaluation and Mitigation Strategy (REMS) programs mandated by the US Food and Drug Administration involve special interventions such as education or documentation of laboratory testing to ensure that the benefits of certain drugs outweigh their risks. To understand patients’ and caregivers’ perception of and experiences with REMS programs with elements to assure safe use, we conducted semi‐structured interviews from March 2022 to July 2023 with 135 patients or caregivers of patients who had used alemtuzumab (Lemtrada), ambrisentan (Letairis), clozapine (Clozaril), isotretinoin (Accutane), lenalidomide (Revlimid), pegvaliase (Palynziq), or sodium oxybate (Xyrem/Xywav). Participants were recruited through national and regional patient volunteer registries, social media, and patient support groups. Interviews focused on patients’ and caregivers’ knowledge about the REMS program and REMS‐related side effects, treatment initiation, medication access and adherence, pandemic policies, and overall perspectives. Among 135 participants, five key themes emerged: (1) Participants were knowledgeable about REMS‐associated drug risks and requirements; (2) some participants had difficulty finding prescribers, particularly for clozapine; (3) administrative burdens related to REMS implementation were a source of frustration, with satisfaction higher when care coordination support was provided; (4) emotional and logistical challenges were identified with pregnancy‐ and clozapine‐related testing; and (5) participants desired similar engagement around risks not covered in REMS materials. Optimizing system‐level implementation can help improve REMS implementation.