Developing a Natural History Model for Duchenne Muscular Dystrophy

Jonathan Broomfield (), M. Hill, F. Chandler, M. J. Crowther, J. Godfrey, M. Guglieri, J. Hastie, J. Larkindale, J. Mumby-Croft, E. Reuben, F. Woodcock and K. R. Abrams
Additional contact information
Jonathan Broomfield: University of Leicester
M. Hill: GlaxoSmithKline
F. Chandler: Sanofi
M. J. Crowther: Karolinska Institute
J. Godfrey: JG Zebra Consulting
M. Guglieri: Newcastle University and Newcastle Hospitals NHS Foundation Trust
J. Hastie: Sanofi
J. Larkindale: Critical Path Institute
J. Mumby-Croft: Source Health Economics
E. Reuben: Duchenne UK
F. Woodcock: Source Health Economics
K. R. Abrams: University of Warwick

PharmacoEconomics - Open, 2024, vol. 8, issue 1, No 7, 79-89

Abstract: 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. This was supplemented with expert input obtained via an elicitation exercise, and a systematic literature review and meta-analysis of mortality data. Results The NHM includes ambulatory, transfer and non-ambulatory phases, which capture loss of ambulation, ability to weight bear and upper body and respiratory function, respectively. The NHM estimates patients spend approximately 9.5 years in ambulatory states, 1.5 years in the transfer state and the remainder of their lives in non-ambulatory states. Median predicted survival is 34.8 years (95% CI 34.1–35.8). Conclusion The model includes a detailed disease pathway for DMD, including the clinically and economically important transfer state. The NHM may be used to estimate the current trajectory of DMD in economic evaluations of new treatments, facilitating inclusion of a lifetime time horizon, and will help identify areas for further research.

Date: 2024
References: View complete reference list from CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s41669-023-00450-x Abstract (text/html)

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:spr:pharmo:v:8:y:2024:i:1:d:10.1007_s41669-023-00450-x

Ordering information: This journal article can be ordered from
http://www.springer.com/adis/journal/41669

DOI: 10.1007/s41669-023-00450-x

Access Statistics for this article

PharmacoEconomics - Open is currently edited by Timothy Wrightson and Christopher Carswell

More articles in PharmacoEconomics - Open from Springer
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().