An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). Issue 1 (1st January 2021)

• Record Type: Journal Article
• Authors: Dean, Rebecca; Jensen, Ivar; Cyr, Phil; Miller, Beckley; Maru, Benit; Sproule, Douglas M.; Feltner, Douglas E.; Wiesner, Thomas; Malone, Daniel C.; Bischof, Matthias; Toro, Walter; Dabbous, Omar
• Is Part Of: Journal of market access & health policy. Volume 9:Issue 1(2021)
• Publication Date: 2021-01-01
• Subjects: Cost-effectiveness analysis -- gene therapy -- spinal muscular atrophy -- onasemnogene abeparvovec -- cost-utility analysis -- nusinersen; Medical policy -- Periodicals; Medical policy -- Economic aspects -- Periodicals; International trade -- Environmental aspects -- Periodicals; Health Policy -- economics; Technology Assessment, Biomedical; Marketing of Health Services; International trade -- Environmental aspects; Medical policy; Periodicals; Periodicals; Fulltext; Internet Resources; Periodicals; 362.1
• DOI: 10.1080/20016689.2021.1889841
• Languages: English
• ISSNs: 2001-6689

Abstract

ABSTRACT: Background : Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results. Objective : To compare the manufacturer's proprietary CUA model to the model published by the Institute for Clinical and Economic Review (ICER), and to update the manufacturer's model with long-term follow-up data and some key ICER assumptions. Study design : We updated a recent CUA evaluating value for money in cost per incremental Quality-adjusted Life Year (QALY) of onasemnogene abeparvovec versus nusinersen (Spinraza®) or best supportive care (BSC) in symptomatic SMA1 patients, and compared it to the ICER model. Setting/Perspective : USA/Commercial payer Participants : Children aged <2 years with SMA1. Interventions : Onasemnogene abeparvovec, a single-dose gene replacement therapy, versus nusinersen, an antisense oligonucleotide, versus BSC. Main outcome measure : Incremental-cost effectiveness ratio and value-based price using traditional thresholds for general medicines in the US. Results : Updated survival (undiscounted) predicted by the model was 37.60 years for onasemnogene abeparvovec compared to 12.10 years for nusinersen and 7.27 years for BSC. Updated quality-adjusted survival using ICER's utility scores and discounted at 3% were 13.33, 2.85, and 1.15 discounted QALYs for onasemnogene abeparvovec, nusinersen, and BSC, respectively. Using estimated net prices, the discounted lifetime cost/patient was $3.93 M for onasemnogene abeparvovec, $4.60 M for nusinersen, and $1.96 M for BSC. The incremental cost per QALY gained for onasemnogene abeparvovec was dominant against nusinersen and $161, 648 against BSC. These results broadly align with the results of the ICER model, which predicted a cost per QALY gained of $139, 000 compared with nusinersen, and $243, 000 compared with BSC (assuming a placeholder price of $2 M for onasemnogene abeparvovec), differences in methodology notwithstanding. Exploratory analyses in presymptomatic patients were similar. Conclusion : This updated CUA model is similar to ICER analyses comparing onasemnogene abeparvovec with nusinersen in the symptomatic and presymptomatic SMA populations. At a list price of $2.125 M, onasemnogene abeparvovec is cost-effective compared to nusinersen for SMA1 patients treated before age 2 years. When compared to BSC, cost per QALY of onasemnogene abeparvovec is higher than commonly used thresholds for therapies in the USA ($150, 000 per QALY).