TY - JOUR
T1 - Health utilities and quality-adjusted life years for patients with amyotrophic lateral sclerosis receiving reldesemtiv or placebo in FORTITUDE-ALS
AU - Gebrehiwet, Paulos
AU - Meng, Lisa
AU - Rudnicki, Stacy A.
AU - Sarocco, Phil
AU - Wei, Jenny
AU - Wolff, Andrew A.
AU - Butzner, Michael
AU - Chiò, Adriano
AU - Andrews, Jinsy A.
AU - Genge, Angela
AU - Hughes, Dyfrig A.
AU - Jackson, Carlayne E.
AU - Lechtzin, Noah
AU - Miller, Timothy M.
AU - Shefner, Jeremy M.
N1 - Funding Information:
We wish to thank the participants of FORTITUDE-ALS and their families for their contributions to this clinical trial, the investigators of FORTITUDE-ALS, the FORTITUDE-ALS Study Group, and members of the Data Monitoring Committee and Steering Committee. The authors acknowledge Emily Chu (Evidence Scientific Solutions, Horsham, UK), and Richard Fay (Evidence Scientific Solutions, Philadelphia, PA, USA), for editorial assistance, which was funded by Cytokinetics, Incorporated.
Publisher Copyright:
© 2023 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2023
Y1 - 2023
N2 - Aims: To estimate the health utilities and quality-adjusted life years (QALYs) in patients with amyotrophic lateral sclerosis (ALS) receiving reldesemtiv versus placebo in FORTITUDE-ALS. Materials and methods: 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 reldesemtiv 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. Results: The full analysis set consisted of 456 patients (reldesemtiv n = 342, placebo n = 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 reldesemtiv versus placebo, adjusted for baseline values, was statistically significant (0.03, 95% confidence interval [CI]: 0.01, 0.05; p =.0008). The incremental QALY of reldesemtiv versus placebo adjusted for baseline utility values showed a modest, but statistically significant, difference (0.004, 95% CI: 0.001, 0.007; p =.0058). Conclusions: This post hoc analysis of FORTITUDE-ALS suggests that reldesemtiv 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. ClinicalTrials.gov identifier: NCT03160898.
AB - Aims: To estimate the health utilities and quality-adjusted life years (QALYs) in patients with amyotrophic lateral sclerosis (ALS) receiving reldesemtiv versus placebo in FORTITUDE-ALS. Materials and methods: 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 reldesemtiv 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. Results: The full analysis set consisted of 456 patients (reldesemtiv n = 342, placebo n = 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 reldesemtiv versus placebo, adjusted for baseline values, was statistically significant (0.03, 95% confidence interval [CI]: 0.01, 0.05; p =.0008). The incremental QALY of reldesemtiv versus placebo adjusted for baseline utility values showed a modest, but statistically significant, difference (0.004, 95% CI: 0.001, 0.007; p =.0058). Conclusions: This post hoc analysis of FORTITUDE-ALS suggests that reldesemtiv 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. ClinicalTrials.gov identifier: NCT03160898.
KW - ALS
KW - EQ-5D-5L
KW - Randomized clinical trial
KW - amyotrophic lateral sclerosis
KW - health utilities
KW - reldesemtiv
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U2 - 10.1080/13696998.2023.2192588
DO - 10.1080/13696998.2023.2192588
M3 - Article
C2 - 36930042
AN - SCOPUS:85151575449
SN - 1369-6998
VL - 26
SP - 488
EP - 493
JO - Journal of Medical Economics
JF - Journal of Medical Economics
IS - 1
ER -