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
HEOR, Precision Xtract, Boston, MA, USA.
Medical Consulting, SSI Strategy, London, UK.
J Mark Access Health Policy. 2021 Feb 28;9(1):1889841. doi: 10.1080/20016689.2021.1889841.
: 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. : 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. : 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. : USA/Commercial payer : Children aged <2 years with SMA1. : Onasemnogene abeparvovec, a single-dose gene replacement therapy, versus nusinersen, an antisense oligonucleotide, versus BSC. : Incremental-cost effectiveness ratio and value-based price using traditional thresholds for general medicines in the US. : 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. : 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).
近期针对1型脊髓性肌萎缩症（SMA1）的onasemnogene abeparvovec（Zolgensma®，原名AVXS - 101）的成本效用分析（CUA）模型在关键假设和结果上存在差异。
为了将制造商的专有CUA模型与临床和经济评论研究所（ICER）发布的模型进行比较，并用长期随访数据和ICER的一些关键假设更新制造商的模型。
我们更新了一个近期的CUA，该CUA评估了在有症状的SMA1患者中，onasemnogene abeparvovec相对于诺西那生钠（Spinraza®）或最佳支持治疗（BSC）的每增加一个质量调整生命年（QALY）的成本效益，并将其与ICER模型进行比较。
美国/商业医保支付方
年龄小于2岁的SMA1患儿
单剂量基因替代疗法onasemnogene abeparvovec、反义寡核苷酸诺西那生钠和最佳支持治疗
使用美国普通药物的传统阈值计算增量成本效益比和基于价值的价格
模型预测的更新后（未贴现）生存期，onasemnogene abeparvovec为37.60年，诺西那生钠为12.10年，最佳支持治疗为7.27年。使用ICER的效用评分并按3%贴现后更新的质量调整生存期，onasemnogene abeparvovec、诺西那生钠和最佳支持治疗分别为13.33、2.85和1.15个贴现QALY。使用估计的净价格，每位患者的贴现终身成本，onasemnogene abeparvovec为393万美元，诺西那生钠为460万美元，最佳支持治疗为196万美元。onasemnogene abeparvovec每获得一个QALY的增量成本相对于诺西那生钠具有优势，相对于最佳支持治疗为161,648美元。这些结果与ICER模型的结果大致一致，ICER模型预测与诺西那生钠相比每获得一个QALY的成本为139,000美元，与最佳支持治疗相比为243,000美元（假设onasemnogene abeparvovec的占位价格为200万美元），尽管方法存在差异。对症状前患者的探索性分析结果相似。
这个更新后的CUA模型与ICER在有症状和症状前SMA人群中比较onasemnogene abeparvovec和诺西那生钠的分析相似。对于2岁前接受治疗的SMA1患者，以212.5万美元的标价，onasemnogene abeparvovec与诺西那生钠相比具有成本效益。与最佳支持治疗相比，onasemnogene abeparvovec每QALY的成本高于美国治疗常用的阈值（每QALY 15万美元）。
