Abstract

Background: Mavacamten treats obstructive hypertrophic cardiomyopathy in adults, improving symptoms and functional capacity. Its complex management process involves frequent echocardiograms, dose adjustments based on Valsalva left ventricular outflow tract (VLVOT) gradient changes, and screening for drug interactions. The high cost of mavacamten poses a financial burden to patients, and the management process lacks adequate simplification. Real-world workflow descriptions are needed for successful patient outcomes. Methods: We retrospectively analyzed patients prescribed mavacamten from May 2022 to May 2023 through a multidisciplinary workflow. Patients were referred to a pharmacist after an initial clinic visit with a cardiologist. Pharmacists counseled patients, enrolled them in the online Risk Evaluation and Mitigation Strategies program, and facilitated enrollment in the manufacturer's patient support program for cost reduction options and prior authorization. Echocardiogram dates were determined using a calculator created by the authors and coordinated with the echocardiography staff. Pharmacists adjusted doses based on finalized echocardiogram reports. Outcomes included ejection fraction (EF), LVOT, and VLVOT which were analyzed using a generalized linear mixed model controlling for time. Additional outcomes were out-of-pocket cost, time to approval, and time to first dispense. Results: Among 34 patients considered, 21 received mavacamten. Reasons for non-receipt included cost (6 patients), unwillingness to follow-up (4 patients), drug interactions (1 patient), absence of mavacamten on insurance formulary (1 patient), and awaiting insurance approval (1 patient). Patients on mavacamten received therapy for 2-48 weeks. They experienced significant decreases in average LVOT (P<0.001) and VLVOT (P<0.001) over time. Average EF showed a non-significant decrease (P=0.097), with one patient recovering from a decline below 50% upon discontinuing mavacamten. Conclusion: Mavacamten faces barriers such as echocardiogram follow-up, complex dosing, drug interaction screening, and cost. However, the described workflow effectively addresses many of these barriers, serving as a template for mavacamten management.

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