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更多不必要的虚构世界 - 第4部分:镰状细胞病的crizanlizumab、voxelotor和L-谷氨酰胺的ICER证据报告

More Unnecessary Imaginary Worlds - Part 4: The ICER Evidence Report for Crizanlizumab, Voxelotor and L-Glutamine for Sickle Cell Disease.

作者信息

Langley Paul C

机构信息

Adjunct Professor, College of Pharmacy, University of Minnesota.

出版信息

Innov Pharm. 2020 Apr 30;11(2). doi: 10.24926/iip.v11i2.3123. eCollection 2020.

Abstract

A number of commentaries have been published over the past 4 years by the present author on the manifest flaws in the reference case value assessment framework of the Institute for Economic and Clinical Review. The recent release of the evidence report on sickle cell disease continues ICER's commitment to what has been described as the creation of imaginary worlds to support value assessment. The purpose of the present commentary is to continue the critiques that have been presented for earlier evidence reports. This is important because of the apparent willingness to take ICER's recommendations at face value rather than a critical review of the value assessment framework. The case presented here points to a number of weaknesses in the ICER framework: (i) the fabrication of imaginary constructs with a lifetime cost-per-incremental QALY framework; (ii) the consequent failure to meet the standards of normal science; (iii) the illogical reliance of assumptions drawn from the literature to create future scenarios; (iv) the rejection of hypothesis testing in favor of 'approximate information' and (v) a belief that in the construction of QALYS that the EQ-5D-3L utility scale has ratio properties. This last point is demonstrably false which means that the ICER value assessment framework collapses. It is impossible mathematically, a failure to meet the axioms of fundamental measurement, for an ordinal utility scale to be combined with time spent in a disease state. The result is that the pricing and access recommendations for Crizanlizumab, Voxelotor and L-glutamine in sickle cell disease (SCD) are complete nonsense and should be rejected.

摘要

在过去四年里,本文作者发表了多篇评论文章,指出经济与临床评估研究所(ICER)参考病例价值评估框架存在明显缺陷。最近发布的镰状细胞病证据报告延续了ICER对所谓创造虚构世界以支持价值评估的坚持。本评论文章的目的是继续对早期证据报告所提出的批评。这很重要,因为人们显然愿意照单全收ICER的建议,而不是对价值评估框架进行批判性审视。这里所举的案例指出了ICER框架的一些弱点:(i)用每增加一个质量调整生命年(QALY)的终身成本框架构建虚构概念;(ii)因此未能达到常规科学的标准;(iii)不合逻辑地依赖从文献中得出的假设来创建未来情景;(iv)拒绝假设检验而倾向于“近似信息”;以及(v)认为在构建QALY时,EQ - 5D - 3L效用量表具有比率属性。最后这一点显然是错误的,这意味着ICER的价值评估框架崩塌。对于一个序数效用量表而言,要与处于疾病状态的时间相结合,在数学上是不可能的,这无法满足基本测量的公理。结果是,关于镰状细胞病(SCD)中克立硐珠单抗、伏索利托和L - 谷氨酰胺的定价及准入建议完全是无稽之谈,应该被摒弃。

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