Developing a Natural History Model for Duchenne Muscular Dystrophy.

ABSTRACT:

Background

The aim of this study was to pool multiple data sets to build a patient-centric, data-informed, natural history model (NHM) for Duchenne muscular dystrophy (DMD) to estimate disease trajectory across patient lifetime under current standard of care in future economic evaluations. The study was conducted as part of Project HERCULES, a multi-stakeholder collaboration to develop tools to support health technology assessments of new treatments for DMD.

Methods

Health states were informed by a review of NHMs for DMD and input from clinicians, patients and caregivers, and defined using common outcomes in clinical trials and real-world practice. The primary source informing the NHM was the Critical Path Institute Duchenne Regulatory Science Consortium (D-RSC) database. Th

SUBMITTER: Broomfield J 

PROVIDER: S-EPMC10781931 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature