Norah L Crossnohere1,2, Ryan Fischer3, Andrew Lloyd4, Lisa A Prosser5, John F P Bridges1,2. 1. Department of Biomedical Informatics, Ohio State University Wexner Medical Center, Columbus, OH. 2. Department of Health, Behavior and Society, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA. 3. Parent Project Muscular Dystrophy, Hackensack, NJ, USA. 4. Acaster Lloyd Consulting Ltd, London, UK. 5. University of Michigan Medical School, Ann Arbor, MI, USA.
Abstract
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.
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.
Entities:
Keywords:
cost utility analysis; health technology assessment; quality of life; rare diseases; utilities
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