Broomfield, Jonathan http://orcid.org/0000-0003-1846-2150
Hill, M.
Chandler, F.
Crowther, M. J.
Godfrey, J.
Guglieri, M.
Hastie, J.
Larkindale, J.
Mumby-Croft, J.
Reuben, E.
Woodcock, F.
Abrams, K. R.
,
Funding for this research was provided by:
Duchenne UK
Article History
Accepted: 18 October 2023
First Online: 29 November 2023
Declarations
:
: This study was funded by Duchenne UK.
: Micki Hill has worked for GSK since April 2022. Keith Abrams is a partner and director of Visible Analytics Limited, a health technology assessment consultancy company; a National Institute for Health and Care Research (NIHR) Senior Investigator Emeritus (NF-SI-0512-10159); and a member of the National Institute for Health and Care Excellence (NICE) Diagnostics Advisory Committee. Jane Larkindale has worked for PepGen Inc. since April 2022. There are no other conflicts of interest for authors contributing to this manuscript.
: D-RSC data access applications can be made via ExternalRef removed. Access to the mortality data set and the simulated individual patient data obtained from the elicitation exercise in this study are available on request from jb781@leicester.ac.uk.
: All studies within the D-RSC dataset are fully de-identified and compliant to the terms and conditions specified by the data owners, with more information at ExternalRef removed. Further analysis of the D-RSC dataset and methods of the elicitation exercise were reviewed and approved by the University of Leicester ethics committee (ref number: 32556-jb781-Ls:healthsciences).
: All participants were provided with an information sheet and required to provide informed consent before completing the elicitation exercise.
: Participants in the online elicitation exercise were presented with a comprehensive information sheet, and by taking part, gave consent for the results of the exercise to be published anonymously.
: A multi-state model was fitted in R using the <i>msm</i> package, allowing for piecewise-constant transition intensities, with details of the analysis described in the main body of text.
: EC and FC conceived the study; EC acquired the funding; KRA and MH designed the study; JG provided project administration; JL supervised the conduct of the study and data collection; JL and MH managed the data, including quality control; KRA, MH, MG, JB and MJC provided statistical advice on study design and analysed the data; KRA, JB, JM-C and FW drafted the manuscript; FC, JH and MG conducted the writing review; and JB is responsible for the whole paper.