Sodium phenylbutyrate–taurursodiol access, adherence and adverse event in patients with amyotrophic lateral sclerosis: Experience at one center in the United States

Abstract

Introduction/AimsSodium phenylbutyrate–taurursodiol (PB‐TURSO) was recently approved for treating amyotrophic lateral sclerosis (ALS). Third‐party payors' coverage policies are evolving, and adverse events are just being fully assessed. The goals of this study were to evaluate patients' experiences in obtaining and continuing PB‐TURSO and assess adverse events and medication adherence.MethodsMedical records of 109 ALS patients who were considered PB‐TURSO candidates by the treating physician at a tertiary ALS clinic from October 2022 to May 2023 were reviewed. Data was recorded for demographics, clinical, and insurance information. A survey was e‐mailed to patients asking about out‐of‐pocket expenses for PB‐TURSO, financial assistance, medication start and (if applicable) stop dates, and reasons for discontinuation.ResultsInsurance information was available for 91 patients [57 males (62%); mean age 64.8 years (range 25.7–88)]. Of 79 who applied for insurance approval, 71 (90%) were approved; however, 19 required 1–3 appeals. Among 73 patients with available data about medication status, 54 started PB‐TURSO and 19 did not, most commonly due to personal choice or out‐of‐pocket expenses. About 44% of patients (24/54) stopped taking PB‐TURSO, primarily due to adverse events. Monthly out‐of‐pocket expenses varied from $0 to $3500 and 36 patients qualified for financial assistance. Administrative and nursing staff devoted 7.2 hours/week to the insurance authorization process.DiscussionMost patients received insurance approval for PB‐TURSO, but one‐fourth required appeals. Some out‐of‐pocket costs were very high. Investment of staff time was substantial. These findings have implications for insurance coverage of, and adherence to, future ALS treatments.