Authors: Tsuraya Iswanto, Mariam Saba, Victoria Phan
Background: Resmetirom, a selective agonist of THR-β in the liver, received FDA accelerated approval in March 2024 for treatment of metabolic dysfunction-associated steatohepatitis (MASH) with stage F2-F3 fibrosis. Patients prescribed on resmetirom often have comorbidities of hypertension, dyslipidemia, and diabetes. As a limited distribution drug, resmetirom is dispensed only through select specialty pharmacies. Due to its recent approval, adherence and side effect data are limited, creating an opportunity for retrospective data analysis to identify therapy-related barriers and guide interventions.
Methods: This will be a retrospective data analysis (from March 2025 to April 2026) involving patients who were prescribed with resmetirom, have a diagnosis of MASH, and had an initial fill and refills from a community-based specialty pharmacy. Patients will be included in this study if they are prescribed resmetirom and have a documented prescription refill record for at least 5 months. Patients will be excluded from the study if they have been on resmetirom for less than 6 months. Adherence will be analyzed using Proportion of Days Covered (PDC) scores. Patients must have had at least 6 total fills (or 180 days) of therapy for PDC scores to be calculated. The pharmacy dispensing system data with dates of the initial fill and refills will be used for PDC scores. PDC scores will be categorized into two adherence groups: PDC scores will be categorized into two adherence groups:
> 98% and < 98%. Therapy outcomes will be assessed by self-reported health status shared by the patient during 6-month therapy reassessments. From pharmacy reassessments calls, the following data will be collected: 1. Data including patient reported side effects 2. Perceived effectiveness of resmetirom in managing MASH treatment 3. Missed doses 4. Challenges while taking resmetirom related to drug therapy • For drug interactions, the pharmacist will identify patients who are on medications that require pharmacist intervention due to major interactions with resmetirom (Statins, CYP2C8 inhibitors, or other hepatotoxic medications).
Results (preliminary results): Overall adherence was high, with a mean PDC of 98.17% and 65.3% of patients classified as highly adherent (≥98%). Patients with ≥98% adherence demonstrated a higher proportion of positive perception compared to those with lower adherence. Adherence showed a non-significant trend towards improved positive perception (OR = 1.54, p = 0.47) and reduced side effects (OR = 0.52, p = 1.00). Confidence intervals were wide, indicating high uncertainty. No statistically significant associations were observed.
Conclusions (reached to date): Adherence to resmetirom therapy was high, and patient-perceived effectiveness was positive. Higher adherence (≥98%) showed trends toward more positive perception (OR 1.54) and fewer side effects (OR 0.52), however were not statistically significant. Limited variability and small sample size likely reduced power to detect associations. These findings can help specialty pharmacists identify strategies that can be implemented to improve therapy outcomes by identifying potential barriers to continuing therapy.
E-mail of resident:
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