Health utilities and quality-adjusted life years for patients with amyotrophic lateral sclerosis receiving <i>reldesemtiv</i> or placebo in FORTITUDE-ALS.


Gebrehiwet, Paulos, Meng, Lisa, Rudnicki, Stacy A, Sarocco, Phil, Wei, Jenny, Wolff, Andrew A, Butzner, Michael, Chiò, Adriano, Andrews, Jinsy A, Genge, Angela et al (show 5 more authors) (2023) Health utilities and quality-adjusted life years for patients with amyotrophic lateral sclerosis receiving <i>reldesemtiv</i> or placebo in FORTITUDE-ALS. Journal of medical economics, 26 (1). pp. 488-493.

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Abstract

<h4>Aims</h4>To estimate the health utilities and quality-adjusted life years (QALYs) in patients with amyotrophic lateral sclerosis (ALS) receiving <i>reldesemtiv</i> versus placebo in FORTITUDE-ALS.<h4>Materials and methods</h4>We performed a post hoc analysis of clinical trial data from FORTITUDE-ALS (NCT03160898). This Phase IIb, double-blind, randomized, dose-ranging, placebo-controlled, parallel-group, 12-week trial evaluated <i>reldesemtiv</i> in patients with ALS. Health utilities from the five-level version of the EuroQol five-dimensional questionnaire (EQ-5D-5L) were estimated using ALS Functional Rating Scale-Revised (ALSFRS-R) scores collected during the trial. QALYs were estimated using the area under the curve method.<h4>Results</h4>The full analysis set consisted of 456 patients (<i>reldesemtiv n</i> = 342, placebo <i>n</i> = 114), who received at least one dose of the double-blind study drug, and had ALSFRS-R assessed at baseline and at least one post-baseline assessment. The difference in EQ-5D-5L utility least-squares (LS) mean change from baseline to week 12 for <i>reldesemtiv</i> versus placebo, adjusted for baseline values, was statistically significant (0.03, 95% confidence interval [CI]: 0.01, 0.05; <i>p</i> = .0008). The incremental QALY of <i>reldesemtiv</i> versus placebo adjusted for baseline utility values showed a modest, but statistically significant, difference (0.004, 95% CI: 0.001, 0.007; <i>p</i> = .0058).<h4>Conclusions</h4>This post hoc analysis of FORTITUDE-ALS suggests that <i>reldesemtiv</i> showed a modest but significant benefit in health utilities and QALYs compared with placebo. Future long-term studies that include direct collection of EQ-5D-5L data will be needed to confirm our findings.<h4>Clinicaltrials.gov identifier</h4>NCT03160898.

Item Type: Article
Uncontrolled Keywords: Randomized clinical trial, amyotrophic lateral sclerosis, ALS, reldesemtiv, EQ-5D-5L, health utilities
Divisions: Faculty of Health and Life Sciences
Faculty of Health and Life Sciences > Institute of Systems, Molecular and Integrative Biology
Depositing User: Symplectic Admin
Date Deposited: 20 Apr 2023 08:22
Last Modified: 31 May 2023 20:16
DOI: 10.1080/13696998.2023.2192588
Open Access URL: https://doi.org/10.1080/13696998.2023.2192588
Related URLs:
URI: https://livrepository.liverpool.ac.uk/id/eprint/3169795
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