Cost-Effectiveness of SacubitrilValsartan in Patients Who Have Heart Failure With Reduced Ejection Fraction
Cost-Effectiveness of SacubitrilValsartan in Patients Who Have Heart Failure With Reduced Ejection Fraction
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DOI：
10.7326/l17-0044
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发表时间：
2017-04
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影响因子：
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通讯作者：
Alexander Sandhu;Daniel Ollendorf;Richard Chapman;Steven Pearson;Paul Heidenreich
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作者：
Alexander Sandhu;Daniel Ollendorf;Richard Chapman;Steven Pearson;Paul Heidenreich
回复：我们同意King博士及其同事关于已发表的沙库比曲缬沙坦多重成本-效果分析的重要性（1，2）。这些发现的一致性支持了使用PARADIGM-HF试验结果设计的这些模型的内部有效性（3），但并没有加强这些结果的外部有效性或这些估计值与真实世界成本效益相匹配的可能性。鉴于这种联合治疗是新颖的，数据不足以估计真实世界的有效性或适应症漂移。然而，我们认为，在采用过程的早期评估治疗的价值是重要的，因为这些分析可以塑造治疗的采用，为购买谈判提供信息（例如，通过加州技术评估论坛的证据报告），并指导进一步研究需求的方向。在我们的分析中，我们改变了敏感性分析中的不确定性的重要领域，并发现这些变化并没有实质性地影响成本效益。然而，我们同意，来自随机临床试验的估计可能无法准确估计治疗的真实价值。试验和现实环境中的成本效益差异可能继发于患者选择和其他治疗实践的差异以及长期有效性的变化。尽管我们强调我们的研究结果仅适用于与PARADIGM-HF试验相匹配的队列，但适应症漂移是一个潜在问题。我们同意，分析新疗法的长期有效性和价值至关重要。目前，在收养期间继续监测效力的适当机制仍在进行中。我们希望，随着更多数据的可用，改进的数据收集和临床与经济数据之间的持续整合将加强我们修订经济评估的能力。
IN RESPONSE: We agree with Dr. King and colleagues about the importance of the multiple cost-effectiveness analyses of sacubitrilvalsartan that have been published (1, 2). The consistency in these findings supports the internal validity of these models that were designed using the PARADIGM-HF trial results (3) but does not strengthen the external validity of these results or the likelihood that these estimates will match real-world cost-effectiveness. Given that this combination therapy is novel, data are not sufficient to estimate real-world effectiveness or indication drift. However, we believe that estimating the value of a treatment early in the adoption process is important because these analyses can shape therapy adoption, inform purchasing negotiations (for example, through such evidence reports as those by the California Technology Assessment Forum), and guide the direction of further research needs. In our analysis, we varied important areas of uncertainty in sensitivity analyses and found that these changes did not substantially affect the cost-effectiveness. Nevertheless, we agree that estimates derived from randomized clinical trials may not accurately estimate a therapy's real-world value. Differences in cost-effectiveness in trial and real-world settings can be secondary to differences in patient selection and other treatment practices and changes in long-term effectiveness. Although we emphasized that our findings are applicable only for cohorts that match those of the PARADIGM-HF trial, indication drift is a potential concern. We agree that analysis of the long-term effectiveness and value of novel therapies is critical. At present, the appropriate mechanism by which to continue monitoring effectiveness during adoption remains a work in progress. We hope that improved data collection and continued integration between clinical and economic data will strengthen our ability to revise economic evaluations as more data become available.