Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment

Abstract Objective To examine patient/caregiver preference for key attributes of treatments spinal muscular atrophy (SMA). Background In the rapidly evolving SMA treatment landscape, it is critically important to understand how potential may impact choices. Design/methods A discrete choice experiment survey was developed based on qualitative interviews. Patients with (≥ 18 years) and caregivers patients were recruited through a U.S. patient organization. Respondents made choices in each 12 sets hypothetical treatments. The relative importance five characteristics compared (measured by regression coefficients [RC] conditional logit models): (1) improvement or stabilization motor function, (2) breathing (3) indication all ages pediatric only, (4) route administration [repeated intrathecal (IT) injections, one-time intravenous (IV) infusion, daily oral delivery] (5) harm (mild, moderate, serious/life threatening). Results Patient ranged from less than 1 67 years (n = 101, 65 self-reported 36 caregiver-reported) 64 female. Total subtypes included: type 21), 2 48), 3 29), other 3). Prior surgery reported 47 patients. Nusinersen onasemnogene abeparvovec-xioi use 59 10 patients, respectively. Improvement function highly valued [RC: 0.65, 95% confidence interval (CI): 0.47–0.83 RC: 0.79, CI: 0.60–0.98, respectively]. Oral medication infusion strongly preferred over repeated IT injections (RC: 0.80, 0.60–0.98 0.51, 0.30–0.73, respectively). least an age-restricted label/approved (≤ age) − 1.28, 1.47 1.09). Cross-attributes trade-off decision suggested lower willingness high-risk therapy despite additional efficacy gain. For some there be trade off gains change medication. Conclusions Improvements motor/breathing broad indication, minimal risk attributes. Treatment decisions should clinical context tailored needs.