Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis.

ABSTRACT:

Objectives

Caring for individuals with spinal muscular atrophy (SMA), a rare genetic disorder, poses tremendous challenges for the economy and healthcare system. This study evaluated the cost-utility of onasemnogene abeparvovec-xioi gene therapy and risdiplam for SMA in Thailand.

Methods

A Markov model was used to analyze the lifetime costs and outcomes of these treatments compared with standard of care for symptomatic SMA types 1 and 2-3. SMA type 1 patients were treated with one of either onasemnogene or risdiplam, while SMA types 2-3 patients received risdiplam. Data on disease progression and medical costs were sourced from hospital databases, while treatment efficacy was based on clinical trials. Interviews with patients and caregivers provided data on non-medical costs

SUBMITTER: Khuntha S 

PROVIDER: S-EPMC11811457 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature