Galeone Carlotta, Bruzzi Paolo, Jommi Claudio
Bicocca Applied Statistics Center (B-ASC), Università degli Studi di Milano-Bicocca, Milano, Lombardia, Italy
Biostatistics & Outcome Research, Statinfo, Renate, Lombardia, Italy.
BMJ Open. 2021 Jan 13;11(1):e041259. doi: 10.1136/bmjopen-2020-041259.
In 2017, the Italian Medicines Agency (Agenzia Italiana del Farmaco, AIFA) introduced a standardised process to appraise innovativeness of medicines. Innovative medicines are provided speeder market access and dedicated funds. Innovativeness criteria are: unmet therapeutic need, added therapeutic value and quality of the evidence (Grading of Recommendations Assessment, Development and Evaluation method). We investigated the role played by these three criteria on the final decision aimed to understand how the new Italian innovativeness appraisal framework was implemented.
A desk research gathered AIFA's appraisal reports on innovativeness and data analyses were conducted. No patients were directly involved in this study.
We scrutinised all 77 appraisal reports available on AIFA's website (2017-2020).
The impact of the three domains on final decision was investigated through a series of univariate analyses.
Among 77 appraisal reports on innovativeness available, 49 (64%) and 28 (36%) were for oncology and non-oncology medicines, respectively. The appraisals were equally distributed among 'fully innovative' (36%), 'conditionally innovative' (30%) and 'not innovative' (34%). Added therapeutic value was the most important driver on innovativeness decision, followed by quality of the evidence. Drugs for rare diseases and with paediatric/mixed indications were appraised 'innovative' by a larger proportion, but no statistical significance was found.
Despite some limitations, including the moderate number of appraisals, this paper provides an insight into the determinants of innovativeness appraisals for medicines in Italy and the accuracy of the appraisal process. This has important implications in terms of transparency and accountability in the prioritisation process applied to innovative medicines.
2017年，意大利药品管理局（Agenzia Italiana del Farmaco，AIFA）引入了一种标准化程序来评估药品的创新性。创新药物可获得更快的市场准入和专项资金。创新性标准包括：未满足的治疗需求、增加的治疗价值和证据质量（推荐分级评估、发展与评价方法）。我们调查了这三个标准在最终决策中所起的作用，旨在了解意大利新的创新性评估框架是如何实施的。
进行案头研究，收集AIFA关于创新性的评估报告并进行数据分析。本研究未直接涉及患者。
我们仔细审查了AIFA网站上提供的所有77份评估报告（2017 - 2020年）。
通过一系列单变量分析研究这三个领域对最终决策的影响。
在77份可用的创新性评估报告中，49份（64%）是关于肿瘤药物的，28份（36%）是关于非肿瘤药物的。评估在“完全创新”（36%）、“有条件创新”（30%）和“不创新”（34%）之间均匀分布。增加的治疗价值是创新性决策中最重要的驱动因素，其次是证据质量。罕见病药物以及有儿科/混合适应症的药物被评为“创新”的比例更高，但未发现统计学意义。
尽管存在一些局限性，包括评估数量适中，但本文深入了解了意大利药品创新性评估的决定因素以及评估过程的准确性。这在应用于创新药物的优先排序过程中的透明度和问责制方面具有重要意义。
