Research at Kela, The Social Insurance Institution of Finland (Kela), Helsinki, Finland.
School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Kuopio, Finland.
BMJ Open. 2024 Oct 21;14(10):e083549. doi: 10.1136/bmjopen-2023-083549.
During the last decade, extensions of therapeutic indications have been one of the most common methods to extend the lifecycle of a medical product in the post-authorisation phase and to increase the use and sales of medicines. The aim of this study was to gain understanding of the lifecycle of cancer medicines and especially the role and level of evidence extensions in comparison to first indications.
We identified all new outpatient cancer medicines approved by the European Medicines Agency between 2010 and 2020 and the extensions to their indications. We compared general study design characteristics from the European public assessment reports using critical appraisal tools and clinical added value assessments.
We identified altogether 55 new outpatient cancer medicines, 31 of which had one or more extension(s) of indication and 24 had no extension of indication. In total, there were 57 extensions. The most common extension of indication was a change in the treatment line (35%). Compared with first indications, the overall quality of studies supporting extensions was better in terms of study designs. The proportion of medicines providing CAV was higher in extensions compared with first indication of medicines with and without extensions.
Based on different assessments and perspectives, we found that extensions of indications are a very common and important part of extending the lifecycle of outpatient cancer medicines in Europe. Our findings also suggest that the clinical value of cancer medicines increases with extensions.
在过去十年中，扩大治疗适应症一直是延长医疗产品上市后生命周期和增加药品使用和销售的最常见方法之一。本研究旨在了解癌症药物的生命周期，特别是适应症扩展的作用和证据水平，与首次适应症相比。
我们确定了 2010 年至 2020 年间欧洲药品管理局批准的所有新的门诊癌症药物及其适应症的扩展。我们使用关键评估工具和临床附加值评估，比较了欧洲公共评估报告中的一般研究设计特征。
我们总共确定了 55 种新的门诊癌症药物，其中 31 种有一个或多个适应症扩展，24 种没有适应症扩展。共有 57 个扩展。最常见的适应症扩展是治疗线的变化（35%）。与首次适应症相比，支持扩展的研究在研究设计方面的整体质量更好。与无扩展的药品相比，提供 CAV 的药品比例在扩展中更高。
基于不同的评估和观点，我们发现适应症的扩展是延长欧洲门诊癌症药物生命周期的非常常见和重要的部分。我们的研究结果还表明，癌症药物的临床价值随着适应症的扩展而增加。
