[This corrects the article DOI: 10.1016/j.ajpc.2020.100010.].

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.

Objective

National estimates of atherosclerotic cardiovascular disease (ASCVD) in the United States (US) are scarce, especially for patients grouped by cardiovascular risk, lipid-lowering therapy use, and low-density lipoprotein cholesterol (LDL-C) levels. The objective of this study was to estimate the size of the ASCVD population, including the subgroup at very high risk for recurrent events as defined by the 2018 Multi-Society Cholesterol Guidelines.

Methods

Patient-level data from the Truven MarketScan Research Database were used and extrapolated to approximate national figures based on known national demographic and ASCVD prevalence numbers. Demographic and clinical characteristics, including LDL-C levels and lipid-lowering therapy use, were captured.

Results

The extrapolated prevalence of ASCVD in 2014 was 18.3 million, of whom 690,524 had an acute coronary syndrome event in the past year. An estimated 41.4% of patients with ASCVD had diabetes, 44.9% had polyvascular disease, and 23.8% had multiple cardiovascular events. A third of those with ASCVD were estimated to be at very high risk for subsequent events per the 2018 Multi-Society Cholesterol Guidelines. Of those with ASCVD, 74.2% were estimated to have an LDL-C level of ≥70 ​md/dL, and more than half of these patients were neither on statins nor ezetimibe. Only 9.2% of patients with ASCVD and LDL-C ≥70 ​mg/dL were on a high-intensity statin.

Conclusions

The underutilization of lipid-lowering therapies in general, and in particular the relatively low usage of high-intensity statins among patients with uncontrolled LDL-C (including those at very high risk), suggests that eligible patients for proprotein convertase subtilisin/kexin type 9 inhibitor therapy may not be as numerous as previously estimated.