Jansen Marieke S, Dekkers Olaf M, le Cessie Saskia, Hooft Lotty, Gardarsdottir Helga, de Boer Anthonius, Groenwold Rolf H H
Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands.
Department of Endocrinology and Metabolic Disorders, Leiden University Medical Center, Leiden, The Netherlands.
Pharmacoepidemiol Drug Saf. 2025 Jan;34(1):e70074. doi: 10.1002/pds.70074.
Real-world evidence (RWE) is increasingly considered in regulatory and health technology assessment (HTA) decision-making, though perspectives on its relevance may vary. Expanding on a recent review regarding regulatory decisions, this study aimed to identify factors influencing the need for RWE in HTA decision-making, confirm and enrich factors with stakeholder views, and evaluate similarities and differences between regulatory and HTA needs.
Previous scoping review methodology was used to identify factors influencing the need for RWE in HTA decision-making. Semi-structured interviews with stakeholders were conducted to confirm and enrich literature-derived factors for both regulatory and HTA contexts. Insights from the reviews and interviews were combined to explore similarities and differences in RWE needs across these domains.
The HTA review, featuring 118 articles, revealed two major themes and six subthemes, encompassing 45 factors. The need for RWE depended on (1) questions addressable with RWE, and (2) contextual factors. Stakeholder interviews confirmed literature-derived factors. While contextual factors aligned between regulatory and HTA decision-making, question-related factors partly differed. Unlike the benefit-risk assessment in regulatory decision-making, RWE serves as direct input for the HTA, and involves specific details and a broader scope. Regulators require RWE for orphan status submissions, alternative approval pathways and to evaluate the impact of risk minimization measures, whereas HTA uses RWE to guide comparator selection, evaluate treatment implementation, quality of care and general healthcare impacts.
Contextual factors that influence the need for RWE are similar between regulatory and HTA decision-making, with variations seen in questions addressable with RWE.
在监管和卫生技术评估（HTA）决策中，真实世界证据（RWE）越来越受到重视，尽管对其相关性的看法可能存在差异。本研究在近期一项关于监管决策的综述基础上进行拓展，旨在确定影响HTA决策中RWE需求的因素，通过利益相关者的观点确认并丰富这些因素，并评估监管需求与HTA需求之间的异同。
采用先前的范围综述方法来确定影响HTA决策中RWE需求的因素。对利益相关者进行半结构化访谈，以确认并丰富从文献中得出的监管和HTA背景下的因素。将综述和访谈的见解结合起来，探讨这些领域中RWE需求的异同。
HTA综述纳入了118篇文章，揭示了两个主要主题和六个子主题，涵盖45个因素。对RWE的需求取决于（1）可用RWE解决的问题，以及（2）背景因素。利益相关者访谈确认了从文献中得出的因素。虽然背景因素在监管决策和HTA决策之间是一致的，但与问题相关的因素部分存在差异。与监管决策中的效益-风险评估不同，RWE是HTA的直接输入，涉及具体细节和更广泛的范围。监管机构要求在提交孤儿药认定申请、替代批准途径以及评估风险最小化措施的影响时提供RWE，而HTA则使用RWE来指导对照选择、评估治疗实施情况、护理质量和总体医疗影响。
监管决策和HTA决策中影响RWE需求的背景因素相似，但可用RWE解决的问题存在差异。
