SC3 The Drivers of Health State Utilities in Duchenne Muscular Dystrophy (DMD): A Study Using the Health Utilities Index Mark 3 (HUI3)

Abstract Background Preferences for health states for Duchenne muscular dystrophy (DMD) are necessary to assess costs and benefits of novel therapies. Because DMD progression begins in childhood, the impact of DMD on health-related quality-of-life (HRQoL) affects preferences of both DMD patients and their families. The objective of this review was to synthesize published evidence for health state utility from the DMD patient and caregiver perspectives. Methods A systematic review was performed using MEDLINE and Embase, according to best practices. Data were extracted from studies reporting DMD patient or caregiver utilities; these included study and patient characteristics, health states considered, and utility estimates. Quality appraisal of studies was performed. Results From 888 abstracts, eight publications describing five studies were identified. DMD utility estimates were from preference-based measures presented stratified by ambulatory status, ventilation, and age. Patient (or patient–proxy) utility estimates ranged from 0.75 (early ambulatory DMD) to 0.05 (day-and-night ventilation). Caregiver utilities ranged from 0.87 (for caregivers of adults with DMD) to 0.71 (for caregivers of predominantly childhood patients). Both patient and caregiver utilities trended lower with higher disease severity. Variability in utilities was observed based on instrument, respondent type, and country. Utility estimates for health states within non-ambulatory DMD are under reported; nor were utilities for DMD-related health states such as scoliosis or preserved upper limb function identified. Conclusion Published health state utilities document the substantial HRQoL impacts of DMD, particularly with disease progression. Additional research in patient utilities for additional health states, particularly in non-ambulatory DMD patients, is warranted.

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PRO86 HEALTH STATE UTILITY VALUES FOR STAGES OF FUNCTIONAL DECLINE IN DUCHENNE MUSCULAR DYSTROPHY: AN INTERNATIONAL STUDY

Value in Health ◽

10.1016/j.jval.2020.04.1307 ◽

2020 ◽

Vol 23 ◽

pp. S344

Author(s):

N. Crossnohere ◽

R. Fischer ◽

J. Bridges

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Functional Decline ◽

International Study ◽

Health State Utility ◽

Health State ◽

Utility Values ◽

Health State Utility Values

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Erratum to ‘Health state values for the health utilities index mark 2 descriptive system: results from a UK valuation survey’Health Economics 2005;14(3): 231–244

Health Economics ◽

10.1002/hec.1136 ◽

2006 ◽

Vol 15 (7) ◽

pp. 761-761

Author(s):

Christopher McCabe ◽

Katherine Stevens ◽

Jennifer Roberts ◽

John Brazier

Keyword(s):

Health State ◽

Health Utilities Index ◽

Health Utilities ◽

Descriptive System

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Producing a preference-based quality of life measure for people with Duchenne muscular dystrophy: a mixed-methods study protocol

BMJ Open ◽

10.1136/bmjopen-2018-023685 ◽

2019 ◽

Vol 9 (3) ◽

pp. e023685 ◽

Cited By ~ 1

Author(s):

Philip A Powell ◽

Jill Carlton ◽

Donna Rowen ◽

John E Brazier

Keyword(s):

Quality Of Life ◽

Mixed Methods ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Mixed Methods Study ◽

Health State ◽

Open Access Journals ◽

Life Years ◽

Descriptive System

IntroductionPreference-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 analysisThis 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 disseminationThis 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.

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Assessing the Appropriateness of the EQ-5D for Duchenne Muscular Dystrophy: A Patient-Centered Study

Medical Decision Making ◽

10.1177/0272989x20978390 ◽

2021 ◽

Vol 41 (2) ◽

pp. 209-221

Author(s):

Norah L. Crossnohere ◽

Ryan Fischer ◽

Andrew Lloyd ◽

Lisa A. Prosser ◽

John F. P. Bridges

Keyword(s):

Health Status ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

The United States ◽

Neuromuscular Disorder ◽

Face Validity ◽

Health State ◽

Patient Centered ◽

Cross Sectional ◽

Eq Vas

Background Duchenne muscular dystrophy is a rare degenerative neuromuscular disorder with pediatric onset. Recent approvals in Duchenne have placed attention on the economic evaluation in pricing and reimbursement decisions across a range of rare conditions. We sought to assess the appropriateness of the EQ-5D, a common measure of generic health state utility, for use among patients and caregivers affected by Duchenne. Methods An international, cross-sectional sample of adults with Duchenne and caregivers reported patient health status using self- or proxy-reported EQ-5D-3L. Appropriateness was assessed across 6 domains of concern raised by stakeholders in Duchenne. These concerns were that the EQ-5D/EQ-VAS would not capture meaningful differences in health status, correlate with disease-specific measures, reflect real health status, exhibit face validity, be accurately interpreted, and be low burden. We evaluated these concerns by comparing EQ-5D index score and EQ-VAS scores to other condition-specific functional measures and open- and closed-ended questions. Results In total, 263 participants (74% response) completed the survey, 24% of whom were adult patients. Most participants lived in the United States or United Kingdom (58%). Patient age ranged from 1 to 48 y. EQ-5D index was higher in ambulatory than nonambulatory patients (0.60 v. 0.30, P < 0.001) and was negatively correlated with upper limb impairment ( r = 0.61, P < 0.001). Three-quarters of respondents agreed that EQ-5D measured real health status (74%). Most respondents interpreted EQ-VAS anchors of best and worst imaginable health as full health (61%) and death/near death (58%). Respondents indicated the EQ-5D was easy to understand (86%) and answer (71%). Conclusions Contrary to anecdotal concerns, we found support for the appropriateness of EQ-5D to assess health status in Duchenne. While other measures may be more sensitive to specific outcomes in Duchenne, there may be some value in results using the EQ-5D measure.

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