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更多不必要的虚构世界——第1部分:临床与经济评论研究所关于类风湿关节炎中 Janus 激酶(JAK)抑制剂的证据报告

More Unnecessary Imaginary Worlds - Part 1: The Institute for Clinical and Economic Review's Evidence Report on Janus Kinase (JAK) Inhibitors in Rheumatoid Arthritis.

作者信息

Langley Paul C

机构信息

College of Pharmacy University of Minnesota.

出版信息

Innov Pharm. 2020 Jan 24;11(1). doi: 10.24926/iip.v11i1.2402. eCollection 2020.

Abstract

Previous commentaries in the Formulary Evaluation section of INNOVATIONS in Pharmacy have pointed to the lack of credibility in modeled claims for cost-effectiveness and associated recommendations for pricing by the Institute for Clinical and Economic Review (ICER). The principal objection to ICER reports has been that their modeled claims fail the standards of normal science: they are best seen as pseudoscience. The purpose of this latest commentary is to consider the recently released ICER evidence report for Janus Kinase (JAK) Inhibitors. As ICER continues, in the case of JAK Inhibitors, to apply its modeled cost utility framework with consequent recommendations for pricing adjustments, these recommendations also lack credibility. In contrast with previous ICER evidence reports, the present report adopts only a 12-month timeframe, one due, in large part, to ICER being unable to justify assumptions to drive its construction of imaginary worlds beyond 12 months. This commentary emphasizesagain, why the ICER methodology fails to meet the standards of normal science. Claims made by ICER for the competing JAK Inhibitor therapies lack credibility, are impossible to evaluate, let alone replicate across treatment settings. Even so, it is important to examine a number of key elements in the ICER invention of the 12-month JAK Inhibitor imaginary world. While this does not imply any degree of acceptance of the ICER methodology, one element that merits particular attention is thefailure of the ICER modeling to meet logically defensible measurement standards in its application of generic health related quality of life (HRQoL) ordinal metrics to create its QALY claims. The failure to meet the required standards of fundamental measurement means that the cost-per-QALY claims are invalid. This raises the issue of the application of Rasch Measurement Theory (RMT) in instrument development and the potential role of patient centric outcome (PCO) instruments that represent the patient voice in value claims. The case made here is that the ICER approach should be abandoned as an unnecessary distraction. If we are to meet standards for the discovery of new facts in therapy response then our focus must be on proposing credible, evaluable and replicable claims within disease states. Instruments, such as the Rheumatoid Arthritis Quality of Life (RAQoL)questionnaire that build on the common construct that QoL is the extent to which human needs are fulfilled should be the basis for value claims. HRQoL Instruments that are clinically focused and reflect the value calculus of providers and not patients in measuring response by symptoms and activity limitations are irrelevant. This puts to one side the belief that incremental cost-per-QALY models, the construction of imaginary worlds are, in any sense, a 'gold standard'; a meme embraced by the health technology assessment profession. Claims for incremental cost per QALY outcomes and recommendations for pricing and access driven by willingness to pay thresholds are irrelevant to formulary decisions.

摘要

《药学创新》“处方集评估”板块之前的评论指出,临床与经济评论学会(ICER)在成本效益的模型化声明及相关定价建议方面缺乏可信度。对ICER报告的主要反对意见是,其模型化声明未能达到正常科学的标准:它们最好被视为伪科学。这篇最新评论的目的是审视ICER最近发布的关于Janus激酶(JAK)抑制剂的证据报告。在JAK抑制剂的案例中,ICER继续应用其模型化成本效用框架并随之提出定价调整建议,这些建议同样缺乏可信度。与ICER之前的证据报告不同,本报告仅采用了12个月的时间范围,这在很大程度上是因为ICER无法为其构建超过12个月的虚构世界的假设提供正当理由。这篇评论再次强调了为什么ICER的方法未能达到正常科学的标准。ICER对相互竞争的JAK抑制剂疗法所做的声明缺乏可信度,无法进行评估,更不用说在不同治疗环境中进行复制了。即便如此,审视ICER在12个月的JAK抑制剂虚构世界中的一些关键要素还是很重要的。虽然这并不意味着对ICER方法有任何程度的认可,但一个值得特别关注的要素是,ICER在应用通用的健康相关生活质量(HRQoL)序贯指标以创建其质量调整生命年(QALY)声明时,其模型未能达到逻辑上合理的测量标准。未能达到基本测量的要求标准意味着每QALY成本声明是无效的。这就引出了拉施测量理论(RMT)在工具开发中的应用问题,以及以患者为中心的结果(PCO)工具在价值声明中代表患者声音的潜在作用。这里提出的观点是,应该摒弃ICER的方法,因为它是一种不必要的干扰。如果我们要达到在治疗反应中发现新事实的标准,那么我们的重点必须是在疾病状态下提出可信、可评估和可复制的声明。诸如类风湿性关节炎生活质量(RAQoL)问卷之类的工具,基于生活质量是人类需求满足程度的共同概念构建,应该成为价值声明的基础。那些以临床为重点、在通过症状和活动受限来衡量反应时反映提供者而非患者价值计算的HRQoL工具是不相关的。这就摒弃了一种观点,即增量每QALY模型、虚构世界的构建在任何意义上都是“黄金标准”,这是卫生技术评估行业所信奉的一个观点。每QALY结果的增量成本声明以及由支付意愿阈值驱动的定价和准入建议与处方集决策无关。

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