MSR21 Cost-Effectiveness Analysis of Treatments for Nonsense Mutation Duchenne Muscular Dystrophy: Expert Validation of Model Face Validity

نویسندگان

چکیده

To assess the face validity of a decision-analytic model evaluating cost-utility ataluren for treatment nonsense mutation Duchenne muscular dystrophy (nmDMD). This was Delphi panel comprising physicians with first-hand experience nmDMD. Consensus investigated key parameters, including expected benefit early treatment, mortality, informal caregiving, and patient health status quality life (assessed using Health Utility Index [HUI]) four disease stages: (1) ambulatory (patient age: 10 years), (2) non-ambulatory, not yet requiring ventilation support, (3) at time initiation night-time (4) full-time support. Nine experts from five countries participated in study. obtained all questions after three rounds (except two HUI-questions concerning hand function [dexterity]). Panelists agreed that starting 2 vs. 5 years age would be to delay loss ambulation by an additional years, support 3 respectively. On average, 4%, 13%, 33% patients reaching stages (1), (2), were die these stages, expectancy stage years. All panelists caregivers provided day-to-day care/support nmDMD across stages. HUI-derived utilities 1.0000 on top best supportive care (BSC) 0.7337 BSC alone. Corresponding estimates 0.3179 0.2672, 0.1643 0.0913, -0.0732 -0.1163. study confirms cost-effectiveness provides further evidence benefits ataluren.

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ژورنال

عنوان ژورنال: Value in Health

سال: 2022

ISSN: ['1098-3015', '1524-4733']

DOI: https://doi.org/10.1016/j.jval.2022.04.1228