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. 2023 May 26;11(1):2216518.
doi: 10.1080/20016689.2023.2216518. eCollection 2023.

Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States

Alexa C Klimchak  1 , Lauren E Sedita  1 , Louise R Rodino-Klapac  1 , Jerry R Mendell  2   3 , Craig M McDonald  4 , Katherine L Gooch  5 , Daniel C Malone  6
Affiliations

Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States

Alexa C Klimchak et al. J Mark Access Health Policy. .

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 #$5M, 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; Duchenne muscular dystrophy; SRP-9001; delandistrogene moxeparvovec; gene therapy.

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Conflict of interest statement

Alexa C. Klimchak, Lauren E. Sedita, Louise R. Rodino-Klapac, and Katherine L. Gooch are employees of Sarepta Therapeutics, Inc., and may own stock/options in the company. Jerry R. Mendell has reported receiving grants from Parent Project Muscular Dystrophy; receiving personal fees from Sarepta Therapeutics, Inc., and Nationwide Children’s Hospital outside the submitted work; and holding a pending patent to micro-dystrophin cassette for gene therapy and an issued patent to rAAV.SGCA delivery isolated limb infusion. Craig M. McDonald served as a consultant on clinical trials of DMD for Astellas, Avidity Biosciences, Capricor Therapeutics, Catabasis, Edgewise Therapeutics, Entrada Therapeutics, Epirium Bio (formerly Cardero Therapeutics), FibroGen, Italfarmaco, Pfizer, PTC Therapeutics, Roche, Santhera Pharmaceuticals, and Sarepta Therapeutics, Inc. He reports honoraria for presentations from PTC Therapeutics, Sarepta Therapeutics, Inc., Solid Biosciences, Santhera Pharmaceuticals, Capricor Therapeutics, and Catabasis. He has received compensation for participation in advisory boards from PTC Therapeutics, Sarepta Therapeutics, Inc., Avidity Biosciences, Edgewise Therapeutics, and Santhera Pharmaceuticals. He has received research support for clinical trials from Capricor Therapeutics, Catabasis, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals, and Sarepta Therapeutics, Inc., and reports grants from the U.S. Department of Defense, U.S. National Institutes of Health (NINDS), Parent Project Muscular Dystrophy, and U.S. National Institute on Disability, Independent Living, and Rehabilitation Research (NIDILRR). Daniel C. Malone has served as a consultant to Sarepta Therapeutics, Inc.

Figures

Figure 1.
Figure 1.
Model structure. Loss of the ability to stand from supine in under 5 seconds signified the transition from the early ambulatory to late ambulatory state as that is associated with progressive mobility decline [49,50]. LoA was defined in this model as the inability to ambulate 10 meters [49] and signified the transition from the late ambulatory to early non-ambulatory state. Progression to a Brooke score above 4 (loss of unweighted hand-to-mouth function) signified the transition from the early non-ambulatory to late non-ambulatory state. LoA, loss of ambulation.
Figure 2.
Figure 2.
Cost-effectiveness acceptability curve for scenarios A and B. Scenario A includes a CEA with QALYs and a placeholder treatment cost of $1,038,093. Scenario B includes a CEA with evLYG and a placeholder treatment cost of $1,485,635. Both scenarios assume 3.0% discounting of costs and benefits.
See this image and copyright information in PMC

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