1237 Risk Evaluation and Mitigation Strategies: From Burden to Benefit

Abstract Introduction Risk Evaluation and Mitigation Strategies (REMS) are safety programs required by FDA to ensure benefits outweigh risks of certain medications. Currently, 69 medications have REMS, including oxybates due to their boxed warning for CNS depression, abuse, and misuse. Oxybate REMS ensure safe and appropriate use, secure handling preventing diversion or unintended exposure, and provide infrastructure to document and communicate concerning behaviors. For unfamiliar patients and clinicians unfamiliar, oxybate REMS enrollment and requirements may be perceived as daunting, and possibly hinder oxybate use and restrict patient access to potentially transformative therapy. Methods Tasks for patient enrollment and provider and pharmacy enrollment/certification in LUMRYZ REMS and ongoing activities ensuring safe use, appropriate prescribing, and mitigating abuse, misuse, and diversion of extended-release sodium oxybate (SXB) were described. Results To enroll and become certified in LUMRYZ REMS, prescribers and patients provide basic contact/professional licensure information on an online or single-page paper form. Online enrollment enables potential same-day, often within minutes, prescriber certification. The office-contact portal allows for online initiation of patient enrollment and management of tasks and communication. Prescribers/patients attest to completion of counseling/training, understanding risks, proper handling, safe use, and storage of SXB; keeping medications/medical history updated; following assessment/evaluation protocols; and prescriber reporting of serious adverse events and signs of abuse/misuse/diversion. Through 30September2025, 3507 prescribers were enrolled and certified and 7981 patients were enrolled (disenrolled: prescriber, n=1; patients, n=5). At certified pharmacies (n=4), pharmacists confirm eligibility per the REMS and all Elements To Assure Safe Use (ETASU) are met (eg, patient counseling checklist includes drug-drug interaction and comorbidities check; risk management for abuse/misuse/diversion; check for active/overlapping oxybate prescriptions). Before dispensing or shipping prescriptions, pharmacists verify and document ETASU within the REMS, contacting the patient or prescriber as needed. To further protect patients and prescribers, the sponsor actively oversees the REMS to identify and address noncompliance; conduct REMS Knowledge Assessments of enrolled entities; and submit annual REMS data reports to FDA. Conclusion Understanding REMS requirements for oxybates, recognizing the responsibilities of communicating with REMS-certified pharmacists, and educating patients about the REMS are essential to reducing barriers to access and ensuring safe, compliant oxybate use. Support (if any) Avadel Pharmaceuticals