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Rowen D, Powell P, Mukuria C, et al. Deriving a Preference-Based Measure for People With Duchenne Muscular Dystrophy From the DMD-QoL. Value Health. 2021;24(10):1499-1510doi: 10.1016/j.jval.2021.03.007.
Rowen, D., Powell, P., Mukuria, C., Carlton, J., Norman, R., & Brazier, J. (2021). Deriving a Preference-Based Measure for People With Duchenne Muscular Dystrophy From the DMD-QoL. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research, 24(10), 1499-1510. https://doi.org/10.1016/j.jval.2021.03.007
Rowen, Donna, et al. "Deriving a Preference-Based Measure for People With Duchenne Muscular Dystrophy From the DMD-QoL." Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research vol. 24,10 (2021): 1499-1510. doi: https://doi.org/10.1016/j.jval.2021.03.007
Rowen D, Powell P, Mukuria C, Carlton J, Norman R, Brazier J. Deriving a Preference-Based Measure for People With Duchenne Muscular Dystrophy From the DMD-QoL. Value Health. 2021 Oct;24(10):1499-1510. doi: 10.1016/j.jval.2021.03.007. Epub 2021 May 06. PMID: 34593174.
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Value Health. 2021 Oct;24(10):1499-1510. doi: 10.1016/j.jval.2021.03.007. Epub 2021 May 06.

Deriving a Preference-Based Measure for People With Duchenne Muscular Dystrophy From the DMD-QoL.

Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research

Donna Rowen, Philip Powell, Clara Mukuria, Jill Carlton, Richard Norman, John Brazier

Affiliations

  1. School of Health and Related Research (ScHARR), University of Sheffield, England, UK. Electronic address: [email protected].
  2. School of Health and Related Research (ScHARR), University of Sheffield, England, UK.
  3. School of Public Health, Curtin University, Perth, Australia.

PMID: 34593174 DOI: 10.1016/j.jval.2021.03.007

Abstract

OBJECTIVES: This study generates a preference-based measure for capturing the quality of life of people with Duchenne muscular dystrophy (DMD) from a new measure of quality of life, DMD-QoL.

METHODS: A health state classification system was derived from the DMD-QoL based on psychometric performance of items, factor analysis, and item response theory analysis. Preferences for health states described by the classification system were elicited using an online discrete choice experiment survey with life years as an additional attribute, from members of the UK general population (n = 1043). Discrete choice experiment data was modeled using a conditional fixed-effects logit model and utility estimates were directly anchored on the 1 to 0 full health-dead scale.

RESULTS: The health state classification system has 8 dimensions: mobility, difficulty using hands, difficulty breathing, pain, tiredness, worry, participation, and feeling good about yourself. The standard model had mostly statistically significant coefficients and reflected the instrument's monotonic structure. However, 2 dimensions had inconsistent coefficients (where utility increased as health worsened) and a consistent model was estimated that merged adjacent inconsistent severity levels. The best state defined by the classification system has a value of 1 and the worst state has a value of -0.559.

CONCLUSION: The modeled results enable DMD-QoL-8D utility values to be generated using DMD-QoL or DMD-QoL-8D data to generate QALYs for people with DMD. QALYs can then be used to inform economic models of the cost-effectiveness of interventions in DMD. Future research comparing the psychometric performance of DMD-QoL-8D to existing generic preference-based measures, including EQ-5D-5L, is recommended.

Copyright © 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.

Keywords: DMD-QoL; Duchenne muscular dystrophy; QALYs; condition-specific preference based measure; utilities

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