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Published on: January 12, 2019
Assessing the Appropriateness of the EQ-5D for Duchenne Muscular Dystrophy: A Patient-Centered Study
Norah L Crossnohere1,2, Ryan Fischer3, Andrew Lloyd4
1Department of Biomedical Informatics, Ohio State University Wexner Medical Center, Columbus, OH.
Insights
The EQ-5D is appropriate for assessing health status in Duchenne muscular dystrophy patients and caregivers, despite concerns about its sensitivity and interpretation. This generic measure offers valuable insights into patient well-being.
Area of Science:
- Neurology
- Health Economics
- Patient-Reported Outcomes
Background:
- Duchenne muscular dystrophy (DMD) is a rare pediatric neuromuscular disorder.
- Recent DMD drug approvals highlight the need for economic evaluations in rare disease pricing and reimbursement.
- The appropriateness of the EQ-5D, a generic health utility measure, for DMD patients and caregivers requires assessment.
Purpose of the Study:
- To evaluate the suitability of the EQ-5D for measuring health status in individuals affected by Duchenne muscular dystrophy.
- To address stakeholder concerns regarding the EQ-5D's ability to capture meaningful differences, correlate with disease-specific measures, reflect real health status, exhibit face validity, be accurately interpreted, and impose a low burden.
Main Methods:
- An international, cross-sectional survey of adult DMD patients and their caregivers was conducted.
- Participants self- or proxy-reported health status using the EQ-5D-3L and EQ-VAS.
- Concerns were evaluated by comparing EQ-5D scores to condition-specific functional measures and qualitative questions.
Main Results:
- 263 participants completed the survey (74% response rate); 24% were adult patients.
- EQ-5D index scores were significantly higher in ambulatory patients (0.60) versus nonambulatory patients (0.30).
- EQ-5D scores negatively correlated with upper limb impairment (r = 0.61). 74% agreed EQ-5D measured real health status; 86% found it easy to understand.
Conclusions:
- The EQ-5D demonstrates appropriateness for assessing health status in Duchenne muscular dystrophy, contrary to some stakeholder concerns.
- While not always more sensitive than disease-specific measures, the EQ-5D provides valuable health status information in DMD.
- The study supports the utility of the EQ-5D in economic evaluations for Duchenne muscular dystrophy.
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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