Government, Health & Not-for-Profit Division, Center for Research On Health and Social Care Management, SDA Bocconi School of Management, Health Economics & HTA, MEO Building, Room W210, II Floor, Via Sarfatti 10, 20136, Milan, Italy.
Italian Multiple Sclerosis (AISM) Society, Genoa, Italy.
Neurol Sci. 2023 Aug;44(8):2933-2937. doi: 10.1007/s10072-023-06825-6. Epub 2023 May 5.
Regulatory agencies have been responsive to public demand for inclusion of the patient experience in evaluating and approving therapies. Over the years, patient-reported outcome measures (PROMs) have become increasingly prevalent in clinical trial protocols; however, their influence on regulators, payers, clinicians, and patients' decision-making is not always clear. We recently conducted a cross-sectional study aimed at investigating the use of PROMs in new regulatory approvals of drugs for neurological conditions between 2017 and 2022 in Europe.
We reviewed European Public Assessment Reports (EPARs) and recorded on a predefined data extraction form whether they considered PROMs, their characteristics (e.g., primary/secondary endpoint, generic/specific instrument) and other relevant information (e.g., therapeutic area, generic/biosimilar, orphan status). Results were tabulated and summarized by means of descriptive statistics.
Of the 500 EPARs related to authorized medicines between January 2017 and December 2022, 42 (8%) concerned neurological indications. Among the EPARs of these products, 24 (57%) reported any use of PROMs, typically considered as secondary (38%) endpoints. In total, 100 PROMs were identified, of which the most common were the EQ-5D (9%), the SF-36 (6%), or its shorter adaptation SF-12, the PedsQL (4%).
Compared to other disease areas, neurology is one where the use of patient-reported outcomes evidence is inherently part of the clinical evaluation and for which core outcome sets exist. Better harmonization of the instruments recommended for use would facilitate the consideration of PROMs at all stages in the drug development process.
监管机构一直响应公众的要求，将患者体验纳入治疗评估和批准中。多年来，患者报告的结果测量（PROM）在临床试验方案中越来越普遍；然而，它们对监管机构、支付者、临床医生和患者决策的影响并不总是明确的。我们最近进行了一项横断面研究，旨在调查 2017 年至 2022 年间在欧洲，PROM 在新药监管批准中的应用情况。
我们审查了欧洲公共评估报告（EPAR），并在预先设定的数据提取表上记录了它们是否考虑了 PROM，其特征（例如，主要/次要终点、通用/特定工具）和其他相关信息（例如，治疗领域、通用/生物类似物、孤儿状态）。结果通过描述性统计进行制表和总结。
在 2017 年 1 月至 2022 年 12 月期间与授权药物相关的 500 份 EPAR 中，有 42 份（8%）涉及神经学适应症。在这些产品的 EPAR 中，24 份（57%）报告了任何 PROM 的使用，通常被认为是次要（38%）终点。共确定了 100 种 PROM，其中最常见的是 EQ-5D（9%）、SF-36（6%）或其较短的适应版 SF-12、PedsQL（4%）。
与其他疾病领域相比，神经病学是一个将患者报告的结果证据内在地纳入临床评估并存在核心结局集的领域。更好地协调推荐使用的工具将有助于在药物开发过程的所有阶段考虑 PROM。
