CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题：Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications.
Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications.
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使用 ICER 矩阵根据治疗获益和确定性对孤儿疗法进行分级是可行的。然而，该评估涉及基于异质性证据的主观判断。ICER 矩阵等工具可能有助于决策者在跨适应症比较疗法时评估治疗获益及其确定性。
临床证据的评价考虑健康获益（疗效和安全性）以及获益估计的确定程度。在罕见病适应症中，开展临床试验的实际和伦理挑战，尤其是在儿科患者中，往往限制了可获得的证据，使结构化评价面临挑战。在承认证据匮乏的同时，监管机构和报销机构会比较某一给定适应症替代治疗的疗效和安全性，通常是在其他治疗对相似或不同病症的获益背景下进行。本研究探讨使用临床与经济评价研究所（ICER）比较临床有效性证据评级矩阵，对一组儿科适应症罕见病疗法样本的临床获益程度（净健康获益，NHB）和效应估计的确定程度进行结构化评估的可行性。
通过OrphaNet和EMA数据库，识别出2017年1月至2020年3月期间获得欧洲药品管理局（EMA）孤儿药产品认定、获批用于16种儿科适应症的11种全身性疗法，并选用ICER证据评级矩阵进行评估：burosumab、cannabidiol、cerliponase alfa、chenodeoxycholic acid（CDCA）、dinutuximab beta、glibenclamide、metreleptin、nusinersen、tisagenlecleucel、velmanase alfa和vestronidase alfa。检索了EMA欧洲公共评估报告、PubMed、EMBASE、Cochrane Library、Clinical Key以及2016年1月至2021年4月的会议报告，以获取疗效和安全性证据。在所识别的疗法中，有两种被评定为具有“显著”净健康获益（NHB）：dinutuximab beta（神经母细胞瘤维持治疗）和nusinersen（I型SMA），一种被评定为“可比”NHB（CDCA）。其余疗法的NHB等级介于“可比”与“显著”之间。没有疗法被评定为负NHB。估计的确定性范围从“高”（dinutuximab beta用于神经母细胞瘤维持治疗）到“低”（CDCA、metreleptin和vestronidase alfa）。其他疗法的确定性等级介于“低”与“高”之间。ICER证据评级矩阵的总体评级“A”（最高）授予了两种疗法，“B+”授予了6种疗法，“C+”授予了5种疗法，“I”（最低）授予了3种疗法。不同评级作者之间的评分存在差异，所有适应症的平均一致率分别为：NHB 71.9%、确定性56.3%、总体评级68.8%。
The evaluation of clinical evidence takes account of health benefit (efficacy and safety) and the degree of certainty in the estimate of benefit. In orphan indications practical and ethical challenges in conducting clinical trials, particularly in paediatric patients, often limit the available evidence, rendering structured evaluation challenging. While acknowledging the paucity of evidence, regulators and reimbursement authorities compare the efficacy and safety of alternative treatments for a given indication, often in the context of the benefits of other treatments for similar or different conditions. This study explores the feasibility of using the Institute for Clinical and Economic Review (ICER) Evidence Rating Matrix for Comparative Clinical Effectiveness in structured assessment of both the magnitude of clinical benefit (net health benefit, NHB) and the certainty of the effect estimate in a sample of orphan therapies for paediatric indications.
Eleven systemic therapies with European Medicines Agency (EMA) orphan medicinal product designation, licensed for 16 paediatric indications between January 2017 and March 2020 were identified using OrphaNet and EMA databases and were selected for evaluation with the ICER Evidence Rating Matrix: burosumab; cannabidiol; cerliponase alfa; chenodeoxycholic acid (CDCA); dinutuximab beta; glibenclamide; metreleptin; nusinersen; tisagenlecleucel; velmanase alfa; and vestronidase alfa. EMA European Public Assessment Reports, PubMed, EMBASE, the Cochrane Library, Clinical Key, and conference presentations from January 2016 to April 2021 were searched for evidence on efficacy and safety. Two of the identified therapies were graded as "substantial" NHB: dinutuximab beta (neuroblastoma maintenance) and nusinersen (Type I SMA), and one as "comparable" NHB (CDCA). The NHB grade of the remaining therapies fell between "comparable" and "substantial". No therapies were graded as having negative NHB. The certainty of the estimate ranged from "high" (dinutuximab beta in neuroblastoma maintenance) to "low" (CDCA, metreleptin and vestronidase alfa). The certainty of the other therapies was graded between "low" and "high". The ICER Evidence Rating Matrix overall rating "A" (the highest) was given to two therapies, "B+" to 6 therapies, "C+" to five therapies, and "I" (the lowest) to three therapies. The scores varied between rating authors with mean agreement over all indications of 71.9% for NHB, 56.3% for certainty and 68.8% for the overall rating.
Using the ICER Matrix to grade orphan therapies according to their treatment benefit and certainty is feasible. However, the assessment involves subjective judgements based on heterogenous evidence. Tools such as the ICER Matrix might aid decision makers to evaluate treatment benefit and its certainty when comparing therapies across indications.
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