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Abstract
Introduction Preference-based measures (PBMs) of health-related quality of life (HRQoL) are used to generate quality-adjusted life years, which are necessary for cost-effectiveness evaluations of health interventions via cost–utility analysis. These measures of health can be generic (ie, pandiagnostic) or condition specific. No condition-specific PBM of HRQoL in Duchenne muscular dystrophy (DMD) exists, yet there are concerns that standard generic measures lack the specificity to assess aspects of HRQoL that are especially important to people with DMD. This study has been designed to produce a condition-specific PBM of HRQoL in DMD.
Methods and analysis This mixed-methods study proceeds through three stages. In the first stage (concept elicitation), semistructured interviews will be conducted with boys and men diagnosed with DMD, and analysed with framework to produce a draft health state descriptive system for HRQoL in DMD. In the second stage (refining the descriptive system), patients, clinicians and primary caregivers of people with DMD will assess the face validity of the descriptive system. This will be followed by a quantitative survey on a larger sample of patients, which will be analysed with psychometric analyses to produce a refined descriptive system. In the third stage (valuation and econometric modelling), an online discrete choice experiment with duration will be administered to a general public sample to generate utility values for the new measure.
Ethics and dissemination This study has received ethical approval from the National Health Service (REC reference: 18/SW/0055). The primary output of this research will be a condition-specific PBM (or ‘bolt-on’ to an existing generic PBM) in people with DMD and an associated value set. Results will be disseminated through international conferences and open-access journals.
- duchenne muscular dystrophy
- patient reported outcome measures
- psychometrics
- qualitative research
- quality of life
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Footnotes
Contributors JC, DR and JEB conceived the study. PAP, JC and DR contributed to the design of the study. PAP wrote the manuscript. All authors contributed to and approved the manuscript.
Funding This work was supported by Duchenne UK under the Project Hercules funding stream.
Competing interests None declared.
Ethics approval This study has been ethically reviewed and received Health Research Authority approval and a favourable ethical opinion from the NHS South West—Central Bristol Research Ethics Committee (REC reference: 18/SW/0055) on 14 March 2018.
Provenance and peer review Not commissioned; externally peer reviewed.
Patient consent for publication Not required.