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Volume 11
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10.1080/20016689.2023.2216518
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Journal of Market Access & Health Policy (JMAHP) is published by MDPI from Volume 12 Issue 1 (2024). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with Taylor & Francis.
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Article

Assessing the Value of Delandistrogene Moxeparvovec (SRP-9001) Gene Therapy in Patients with Duchenne Muscular Dystrophy in the United States †

by
Alexa C. Klimchak
1,
Lauren E. Sedita
1,
Louise R. Rodino-Klapac
6,
Jerry R. Mendell
2,3,
Craig M. McDonald
4,
Katherine L. Gooch
1,‡ and
Daniel C. Malone
5,*,‡
1
Global HEOR, RWE & Analytics, Sarepta Therapeutics, Inc, Cambridge, MA, USA
2
Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, Columbus, OH, USA
3
Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA
4
Department of Pediatrics, University of California Davis School of Medicine, Davis, CA, USA
5
College of Pharmacy, University of Utah, Salt Lake City, UT 84112, USA
6
Research & Development, Sarepta Therapeutics, Inc, Cambridge, MA, USA
*
Author to whom correspondence should be addressed.
†
This article has been republished with minor changes. These changes do not impact the academic content of the article.
‡
Senior authors contributed equally.
J. Mark. Access Health Policy 2023, 11(1), 2216518; https://doi.org/10.1080/20016689.2023.2216518
Submission received: 14 March 2023 / Revised: 16 May 2023 / Accepted: 17 May 2023 / Published: 26 May 2023

Abstract

Background: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene therapy that may delay progression of Duchenne muscular dystrophy (DMD), a severe, rare neuromuscular disease caused by DMD gene mutations. Early cost-effectiveness analyses are important to help contextualize the value of gene therapies for reimbursement decision making. Objective: To determine the potential value of delandistrogene moxeparvovec using a cost-effectiveness analysis. Study design: A simulation calculated lifetime costs and equal value of life years gained (evLYG). Inputs included extrapolated clinical trial results and published utilities/costs. As a market price for delandistrogene moxeparvovec has not been established, threshold analyses established maximum treatment costs as they align with value, including varying willingness-to-pay up to $500,000, accounting for severity/rarity. Setting: USA, healthcare system perspective. Patients: Boys with DMD. Intervention: Delandistrogene moxeparvovec plus standard of care (SoC; corticosteroids) versus SoC alone. Main outcome measure: Maximum treatment costs at a given willingness-to-pay threshold. Results: Delandistrogene moxeparvovec added 10.30 discounted (26.40 undiscounted) evLYs. The maximum treatment cost was approximately $5 M, assuming $500,000/evLYG. Varying the benefit discount rate to account for the single administration increased the estimated value to >$5 M, assuming $500,000/evLYG. Conclusion: In this early economic model, delandistrogene moxeparvovec increases evLYs versus SoC and begins to inform its potential value from a healthcare perspective.
Keywords: cost-effectiveness analysis; delandistrogene moxeparvovec; Duchenne muscular dystrophy; gene therapy; SRP-9001 cost-effectiveness analysis; delandistrogene moxeparvovec; Duchenne muscular dystrophy; gene therapy; SRP-9001

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MDPI and ACS Style

Klimchak, A.C.; Sedita, L.E.; Rodino-Klapac, L.R.; Mendell, J.R.; McDonald, C.M.; Gooch, K.L.; Malone, D.C. Assessing the Value of Delandistrogene Moxeparvovec (SRP-9001) Gene Therapy in Patients with Duchenne Muscular Dystrophy in the United States †. J. Mark. Access Health Policy 2023, 11, 2216518. https://doi.org/10.1080/20016689.2023.2216518

AMA Style

Klimchak AC, Sedita LE, Rodino-Klapac LR, Mendell JR, McDonald CM, Gooch KL, Malone DC. Assessing the Value of Delandistrogene Moxeparvovec (SRP-9001) Gene Therapy in Patients with Duchenne Muscular Dystrophy in the United States †. Journal of Market Access & Health Policy. 2023; 11(1):2216518. https://doi.org/10.1080/20016689.2023.2216518

Chicago/Turabian Style

Klimchak, Alexa C., Lauren E. Sedita, Louise R. Rodino-Klapac, Jerry R. Mendell, Craig M. McDonald, Katherine L. Gooch, and Daniel C. Malone. 2023. "Assessing the Value of Delandistrogene Moxeparvovec (SRP-9001) Gene Therapy in Patients with Duchenne Muscular Dystrophy in the United States †" Journal of Market Access & Health Policy 11, no. 1: 2216518. https://doi.org/10.1080/20016689.2023.2216518

APA Style

Klimchak, A. C., Sedita, L. E., Rodino-Klapac, L. R., Mendell, J. R., McDonald, C. M., Gooch, K. L., & Malone, D. C. (2023). Assessing the Value of Delandistrogene Moxeparvovec (SRP-9001) Gene Therapy in Patients with Duchenne Muscular Dystrophy in the United States †. Journal of Market Access & Health Policy, 11(1), 2216518. https://doi.org/10.1080/20016689.2023.2216518

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