Developing a Natural History Model for Duchenne Muscular Dystrophy

• DOI: 10.1007/s41669-023-00450-x
• Publication Date: 2023-11-29T16:32:44Z
• Authors: Jonathan Broomfield; M. Hill; F. Chandler; M. J. Crowther; J. Godfrey; M. Guglieri; J. Hastie; J. Larkindale; J. Mumby-Croft; E. Reuben; F. Woodcock; K. R. Abrams
• Keywords: Original Research Article; 3. Good health

The aim of this study was to pool multiple data sets build a patient-centric, data-informed, natural history model (NHM) for Duchenne muscular dystrophy (DMD) estimate disease trajectory across patient lifetime under current standard care in future economic evaluations. conducted as part Project HERCULES, multi-stakeholder collaboration develop tools support health technology assessments new treatments DMD. Health states were informed by review NHMs DMD and input from clinicians, patients caregivers, defined using common outcomes clinical trials real-world practice. primary source informing the NHM Critical Path Institute Regulatory Science Consortium (D-RSC) database. This supplemented with expert obtained via an elicitation exercise, systematic literature meta-analysis mortality data. includes ambulatory, transfer non-ambulatory phases, which capture loss ambulation, ability weight bear upper body respiratory function, respectively. estimates spend approximately 9.5 years ambulatory states, 1.5 state remainder their lives states. Median predicted survival is 34.8 (95% CI 34.1–35.8). detailed pathway DMD, including clinically economically important state. may be used evaluations treatments, facilitating inclusion time horizon, will help identify areas further research.