Health Quality Ontario, Toronto, Canada.
Department of Epidemiology, Biostatistics, and Occupational Health, McGill University, Montreal, Canada.
Int J Technol Assess Health Care. 2019;35(4):291-297. doi: 10.1017/S0266462319000576. Epub 2019 Jul 24.
Traditional decision rules have limitations when a new technology is less effective and less costly than a comparator. We propose a new probabilistic decision framework to examine non-inferiority in effectiveness and net monetary benefit (NMB) simultaneously. We illustrate this framework using the example of repetitive transcranial magnetic stimulation (rTMS) and electroconvulsive therapy (ECT) for treatment-resistant depression.
We modeled the quality-adjusted life-years (QALYs) associated with the new intervention (rTMS), an active control (ECT), and a placebo control, and we estimated the fraction of effectiveness preserved by the new intervention through probabilistic sensitivity analysis (PSA). We then assessed the probability of cost-effectiveness using a traditional cost-effectiveness acceptability curve (CEAC) and our new decision-making framework. In our new framework, we considered the new intervention cost-effective in each simulation of the PSA if it preserved at least 75 percent of the effectiveness of the active control (thus demonstrating non-inferiority) and had a positive NMB at a given willingness-to-pay threshold (WTP).
rTMS was less effective (i.e., associated with fewer QALYs) and less costly than ECT. The traditional CEAC approach showed that the probabilities of rTMS being cost-effective were 100 percent, 39 percent, and 14 percent at WTPs of $0, $50,000, and $100,000 per QALY gained, respectively. In the new decision framework, the probabilities of rTMS being cost-effective were reduced to 23 percent, 21 percent, and 13 percent at WTPs of $0, $50,000, and $100,000 per QALY, respectively.
This new framework provides a different perspective for decision making with considerations of both non-inferiority and WTP thresholds.
当新技术的效果不如对照疗法，且成本更低时，传统决策规则存在局限性。我们提出了一种新的概率决策框架，同时考察有效性和净货币收益（NMB）的非劣效性。我们使用重复经颅磁刺激（rTMS）和电惊厥疗法（ECT）治疗难治性抑郁症的例子来说明这一框架。
我们构建了与新干预措施（rTMS）、活性对照（ECT）和安慰剂对照相关的质量调整生命年（QALYs）模型，并通过概率敏感性分析（PSA）估计新干预措施保留的有效性分数。然后，我们使用传统的成本效益接受曲线（CEAC）和新的决策框架来评估成本效益的概率。在我们的新框架中，如果新干预措施保留了至少 75%的活性对照的有效性（从而证明了非劣效性），并且在给定的支付意愿阈值（WTP）上具有正的 NMB，则认为新干预措施在 PSA 的每次模拟中都是成本有效的。
rTMS 的效果（即，与较少的 QALYs 相关）不如 ECT，且成本更低。传统的 CEAC 方法表明，rTMS 在 WTP 分别为 0、50000 美元和 100000 美元/QALY 时，其成本效益的概率分别为 100%、39%和 14%。在新的决策框架中，rTMS 在 WTP 分别为 0、50000 美元和 100000 美元/QALY 时，其成本效益的概率分别降低至 23%、21%和 13%。
该新框架提供了一种不同的决策视角，同时考虑了非劣效性和 WTP 阈值。
