The Italian Assessment System for Innovative Medicines: An 8-Year Retrospective Analysis of Outcomes, Criteria, Timelines and Funding Dynamics

IF 3.3 4区医学 Q1 ECONOMICS

Applied Health Economics and Health Policy Pub Date : 2026-03-04 DOI:10.1007/s40258-026-01032-8

Lorenzo Martellone, Camilla Servidio, Marcello Vaccaro, Katiuscja Malandrini, Adriana Coluccia, Alessandra Oliva, Claudio Maria Mastroianni, Giacomo Polito

{"title":"The Italian Assessment System for Innovative Medicines: An 8-Year Retrospective Analysis of Outcomes, Criteria, Timelines and Funding Dynamics","authors":"Lorenzo Martellone, Camilla Servidio, Marcello Vaccaro, Katiuscja Malandrini, Adriana Coluccia, Alessandra Oliva, Claudio Maria Mastroianni, Giacomo Polito","doi":"10.1007/s40258-026-01032-8","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><p>The Italian assessment system for innovative medicines represents a distinctive European regulatory approach. Integrated with the Italian Medicines Agency (AIFA), since 2017 it has provided a formal evaluation based on three predefined criteria, Therapeutic Need (TN), Added Therapeutic Value (ATV) and Quality of Evidence (QoE), granting access to dedicated funds and simplified procedures.</p><h3>Objective</h3><p>This study investigated the evolution of the Italian system from 2017 to the 2025 reform, focusing on outcomes, decision criteria, timelines, and financial implications.</p><h3>Methods</h3><p>Publicly available AIFA data on 266 assessments were analysed and stratified by outcome, therapeutic area, orphan status and Advanced Therapy Medicinal Product (ATMP) designation. Logistic regression models were used to assess the internal consistency of appraisal criteria and to explore whether regulatory attributes modified their association with innovativeness outcomes. Time to recognition (TIR) and Decision-to-Implementation Interval (DII) were calculated for “Full Innovative” medicines. Annual spending was compared to fund availability to assess the financial impact of innovative medicines.</p><h3>Results</h3><p>Among the 266 evaluations, approximately 29% were classified as “Full Innovative”, 29% “Conditionally Innovative” and 42% “Not Innovative”. Added Therapeutic Value and QoE showed the strongest associations with positive designations, while orphan and ATMP status showed no independent association. Access timelines were comparable across therapeutic areas, with slightly longer delays for ATMPs. Spending exceeded fund limits several times but fell below budget after fund unification in 2022.</p><h3>Conclusions</h3><p>The Italian system has been applied consistently over time, with appraisal outcomes largely reflecting the formal decision criteria rather than regulatory status. The 2025 reform may represent a turning point, potentially changing designation patterns and their determinants. Ongoing monitoring is needed to ensure access does not compromise evidence standards or sustainability, offering lessons that may be relevant to other health systems considering similar mechanisms.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 3","pages":"541 - 551"},"PeriodicalIF":3.3000,"publicationDate":"2026-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-026-01032-8.pdf","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Applied Health Economics and Health Policy","FirstCategoryId":"3","ListUrlMain":"https://link.springer.com/article/10.1007/s40258-026-01032-8","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ECONOMICS","Score":null,"Total":0}

引用次数: 0

Abstract

Background

The Italian assessment system for innovative medicines represents a distinctive European regulatory approach. Integrated with the Italian Medicines Agency (AIFA), since 2017 it has provided a formal evaluation based on three predefined criteria, Therapeutic Need (TN), Added Therapeutic Value (ATV) and Quality of Evidence (QoE), granting access to dedicated funds and simplified procedures.

Objective

This study investigated the evolution of the Italian system from 2017 to the 2025 reform, focusing on outcomes, decision criteria, timelines, and financial implications.

Methods

Publicly available AIFA data on 266 assessments were analysed and stratified by outcome, therapeutic area, orphan status and Advanced Therapy Medicinal Product (ATMP) designation. Logistic regression models were used to assess the internal consistency of appraisal criteria and to explore whether regulatory attributes modified their association with innovativeness outcomes. Time to recognition (TIR) and Decision-to-Implementation Interval (DII) were calculated for “Full Innovative” medicines. Annual spending was compared to fund availability to assess the financial impact of innovative medicines.

Results

Among the 266 evaluations, approximately 29% were classified as “Full Innovative”, 29% “Conditionally Innovative” and 42% “Not Innovative”. Added Therapeutic Value and QoE showed the strongest associations with positive designations, while orphan and ATMP status showed no independent association. Access timelines were comparable across therapeutic areas, with slightly longer delays for ATMPs. Spending exceeded fund limits several times but fell below budget after fund unification in 2022.

Conclusions

The Italian system has been applied consistently over time, with appraisal outcomes largely reflecting the formal decision criteria rather than regulatory status. The 2025 reform may represent a turning point, potentially changing designation patterns and their determinants. Ongoing monitoring is needed to ensure access does not compromise evidence standards or sustainability, offering lessons that may be relevant to other health systems considering similar mechanisms.

查看原文本刊更多论文

意大利创新药物评估系统：对结果、标准、时间表和资金动态的8年回顾性分析。

背景：意大利创新药物评估体系代表了一种独特的欧洲监管方法。自2017年以来，它与意大利药品管理局（AIFA）进行了整合，根据三个预定义标准(治疗需求（TN）、附加治疗价值（ATV）和证据质量（QoE）提供了正式评估，并提供了专用资金和简化程序。目的：本研究调查了意大利从2017年到2025年改革的演变，重点关注结果、决策标准、时间表和财政影响。方法：对266项公开的AIFA评估数据进行分析，并按结果、治疗领域、孤儿状态和先进治疗药物（ATMP）指定进行分层。采用Logistic回归模型评估评价标准的内部一致性，并探讨监管属性是否会改变其与创新结果的关联。计算“完全创新”药物的识别时间（TIR）和决策到实施间隔（DII）。将年度支出与可用资金进行比较，以评估创新药物的财务影响。结果：266项评价中，“完全创新”占29%，“有条件创新”占29%，“不创新”占42%。添加治疗价值和QoE与阳性标记的相关性最强，而孤儿和ATMP状态没有独立的相关性。各治疗领域的获得时间具有可比性，atmp的延迟时间略长。但在2022年基金统一后，支出数多次超过了基金限额，但仍低于预算。结论：随着时间的推移，意大利系统一直被应用，评估结果在很大程度上反映了正式的决策标准，而不是监管状态。2025年的改革可能是一个转折点，可能会改变指定模式及其决定因素。需要进行持续监测，以确保获取不会损害证据标准或可持续性，提供可能与考虑类似机制的其他卫生系统相关的经验教训。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Applied Health Economics and Health Policy Economics, Econometrics and Finance-Economics and Econometrics

CiteScore

6.10

自引率

2.80%

发文量

期刊介绍： Applied Health Economics and Health Policy provides timely publication of cutting-edge research and expert opinion from this increasingly important field, making it a vital resource for payers, providers and researchers alike. The journal includes high quality economic research and reviews of all aspects of healthcare from various perspectives and countries, designed to communicate the latest applied information in health economics and health policy. While emphasis is placed on information with practical applications, a strong basis of underlying scientific rigor is maintained.