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美国Nefecon与免疫球蛋白A肾病(IgAN)患者最佳支持治疗的成本效益分析。

Cost-Effectiveness Analysis of Nefecon versus Best Supportive Care for People with Immunoglobulin A Nephropathy (IgAN) in the United States.

作者信息

Ramjee Lauren, Vurgun Nesrin, Ngai Christopher, Patel Mit, Tremblay Gabriel

机构信息

Health Economics & Outcomes Research (HEOR), Cytel, Inc, Waltham, MA, USA.

Market Access, Calliditas NA Enterprises, New York, NY, USA.

出版信息

Clinicoecon Outcomes Res. 2023 Mar 29;15:213-226. doi: 10.2147/CEOR.S389456. eCollection 2023.

Abstract

PURPOSE

To estimate the cost-effectiveness of Nefecon in addition to the best supportive care (BSC) vs BSC in a hypothetical cohort of commercially insured adult patients with primary immunoglobulin A nephropathy (IgAN) from a United States (US) societal perspective.

METHODS

A lifetime horizon, semi-Markov model was developed that consisted of nine health states: chronic kidney disease (CKD) stage 1, 2, 3a, 3b, 4, end-stage renal disease (ESRD) with dialysis, ESRD without dialysis, post-kidney transplant, and death. Health state occupancy was estimated from individual patient-level data from the Phase 3 randomized controlled trial NefIgArd Part A (NCT03643965). Additional scenarios evaluated the impact of varying the time horizon, discounting, costs included, rounds of treatment, and the method used to calculate transition probabilities.

RESULTS

In the deterministic base case analysis over a lifetime horizon, Nefecon plus BSC (hereafter Nefecon) had an incremental cost of $3,810 vs BSC. Nefecon resulted in a mean survival gain of 0.247 quality-adjusted life years (QALYs), 0.195 life years (LYs), and 0.244 equal value life years (evLYs) vs BSC alone - this resulted in incremental cost-effectiveness ratios (ICERs) of $15,428 per QALY, $19,502 per LY, and $15,611 per evLY gained. Probabilistic sensitivity analyses estimated that with willingness to pay thresholds of $100,000, $150,000, and $250,000 per QALY gained, Nefecon would be cost-effective over BSC in 66.70%, 75.02%, and 86.82% of cases, respectively. In the scenario analysis, Nefecon remained cost-effective with 4 rounds of treatment.

CONCLUSION

Nefecon was associated with LY and QALY gains vs BSC, with an incremental cost of $3,810. Based on these values, with a willingness to pay threshold of $100,000 per QALY gained, Nefecon was found to be a cost-effective treatment for US adults with primary IgAN.

摘要

目的

从美国社会角度评估在最佳支持治疗(BSC)基础上加用奈非那酮(Nefecon)与单纯BSC相比,对一组假设的商业保险成年原发性免疫球蛋白A肾病(IgAN)患者的成本效益。

方法

建立了一个终身视角的半马尔可夫模型,该模型包含九个健康状态:慢性肾脏病(CKD)1期、2期、3a期、3b期、4期、接受透析的终末期肾病(ESRD)、未接受透析的ESRD、肾移植后以及死亡。健康状态占用情况根据3期随机对照试验NefIgArd A部分(NCT03643965)的个体患者层面数据进行估计。其他情景分析评估了改变时间范围、贴现、所包含的成本、治疗轮次以及用于计算转移概率的方法的影响。

结果

在终身视角的确定性基础病例分析中,与单纯BSC相比,Nefecon加BSC(以下简称Nefecon)的增量成本为3810美元。与单纯BSC相比,Nefecon导致平均生存获益为0.247个质量调整生命年(QALY)、0.195个生命年(LY)和0.24个等效生命年(evLY)——这导致每获得一个QALY的增量成本效益比(ICER)为15428美元、每获得一个LY为19502美元、每获得一个evLY为15611美元。概率敏感性分析估计,对于每获得一个QALY的支付意愿阈值分别为100000美元、150000美元和250000美元时,Nefecon在66.70%、75.02%和86.82%的病例中比BSC更具成本效益。在情景分析中,Nefecon在进行4轮治疗时仍具有成本效益。

结论

与单纯BSC相比,Nefecon可带来LY和QALY获益,增量成本为3810美元。基于这些数值,对于每获得一个QALY的支付意愿阈值为100000美元时,Nefecon被发现是治疗美国成年原发性IgAN患者的一种具有成本效益的治疗方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/515b/10067365/9aab58f9d78e/CEOR-15-213-g0001.jpg

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