Development of a cost-effectiveness analysis model for Duchenne muscular dystrophy utilizing the national registry in Japan.

ABSTRACT: Cost-effectiveness analysis models for Duchenne muscular dystrophy (DMD)-a rare muscular disease-have been developed based on data from a limited patient population, such as clinical trials. Thus, this study aimed to construct a more robust cost-effectiveness analysis model based on real-world evidence. The model was constructed using the Registry of Muscular Dystrophy (Remudy) database, the national registry of muscular diseases in Japan. Parameters for transition probability and drug cost were estimated based on this registry, and a quality-of-life (QOL) survey was conducted on Remudy-listed patients for utility. A Markov model was adopted using motor functions as outcomes. Age-specific transition probabilities were estimated by fitting a Weibull distribution to Remudy data of 730 patien

SUBMITTER: Okada T 

PROVIDER: S-EPMC12827352 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature