Value assessment of medicinal products by the Italian Medicines Agency (AIFA) and French National Authority for Health (HAS): Similarities and discrepancies.

Frontiers in Medical Technology Pub Date : 2022-09-05 eCollection Date: 2022-01-01 DOI:10.3389/fmedt.2022.917151
Entela Xoxi, Rossella Di Bidino, Serena Leone, Andrea Aiello, Mariangela Prada
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引用次数: 1

Abstract

The evaluation of pharmaceutical innovation and therapeutic value is an increasingly complex exercise for which different approaches are adopted at the national level, despite the need for standardisation of processes and harmonisation of public health decisions. The objective of our analysis was to compare the approaches of the AIFA (Agenzia Italiana del Farmaco) and the HAS (Haute Autorité de Santé) in assessing the same medicinal products. In Italy, the 1525/2017 AIFA Deliberation introduces a transparent scheme for the evaluation of innovative status (innovative, conditional, not innovative) based on the therapeutic added value (TAV), therapeutic need, and quality of evidence. In contrast, in France, the HAS makes judgements using the effective clinical benefit (Service Médical Rendu) and improvement of effective clinical benefit (Amélioration du Service Médical Rendu, ASMR). This analysis focused on medicinal products evaluated both by the AIFA and by the HAS from July 2017 to September 2021. Similarities between AIFA and HAS evaluations were investigated in terms of the TAV, recognition of innovativeness, and the ASMR. Both total and partial agreements were considered relevant. Therefore, raw agreement, Cohen's kappa (weighted and unweighted), and Bangdiwala's B-statistic were estimated. A total of 102 medicinal products were included in this study. Out of these, 38 (37.2%) were orphan drugs, while 56 (54.9%) had a clinical indication for the treatment of cancer. The AIFA and HAS reached a higher level of agreement on the innovativeness status compared with the TAV. A moderate total agreement emerged in the recognition of innovativeness (k = 0.463, p-value ≤0.0001), and partial agreement was substantial (equal weight k = 0.547, squared k = 0.638), while a lack of agreement resulted in a comparison of the TAV according to the AIFA and the ASMR recognised by the HAS. Indeed, whereas the AIFA determined the TAV to be important, the HAS considered it to be moderate. In addition, whereas the AIFA identified a bias towards a moderate TAV, the HAS identified a bias towards a minor ASMR. A higher level of agreement was reached, both on the TAV and on innovative status, for less critical medical products (non-cancer-related, or non-orphan, or with a standard European Medicines Agency approval). These results underline the importance of implementing European procedures that are more broadly aligned in terms of value definition criteria.

Abstract Image

意大利药品管理局(AIFA)和法国国家卫生管理局(HAS)的药品价值评估:相似性和差异。
对药品创新和治疗价值的评价是一项日益复杂的工作,尽管需要对过程进行标准化和协调公共卫生决定,但在国家一级采用了不同的方法。我们分析的目的是比较AIFA (Agenzia Italiana del Farmaco)和HAS (Haute autorit de sant)评估相同药品的方法。在意大利,1525/2017 AIFA审议引入了一个基于治疗附加值(TAV)、治疗需求和证据质量的透明方案,用于评估创新状态(创新、有条件、非创新)。相比之下,在法国,HAS使用有效临床获益(Service m 医疗器械治疗计划)和有效临床获益的改善(am 医疗器械治疗计划计划,ASMR)来进行判断。该分析侧重于2017年7月至2021年9月期间由AIFA和HAS评估的药品。在TAV、对创新性的认可和ASMR方面,研究了AIFA和HAS评估的相似性。全面协议和部分协议都被认为是相关的。因此,估计了原始一致性,Cohen的kappa(加权和未加权)和Bangdiwala的b统计量。本研究共纳入102种药品。其中,38种(37.2%)为孤儿药,56种(54.9%)具有治疗癌症的临床指征。与TAV相比,AIFA和TAV在创新地位上达成了更高的共识。在对创新性的认识中出现了适度的完全一致(k = 0.463, p值≤0.0001),部分一致是实质性的(等权k = 0.547,平方k = 0.638),而缺乏一致导致根据AIFA的TAV与HAS认可的ASMR进行比较。事实上,尽管AIFA认为TAV很重要,但HAS认为它是适度的。此外,尽管AIFA发现了对中度TAV的偏见,但HAS发现了对轻度ASMR的偏见。在TAV和创新地位方面,对于不太关键的医疗产品(非癌症相关产品,或非孤儿产品,或获得欧洲药品管理局标准批准)达成了更高层次的协议。这些结果强调了实施在价值定义标准方面更广泛一致的欧洲程序的重要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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