DataSheet1_Real-world data: a comprehensive literature review on the barriers, challenges, and opportunities associated with their inclusion in the health technology assessment process.ZIP

Objective: This review aimed to assess the current use and acceptance of real-world data (RWD) and real-world evidence (RWE) in health technology assessment (HTA) process. It additionally aimed to discern stakeholders’ viewpoints concerning RWD and RWE in HTA and illuminate the obstacles, difficulties, prospects, and consequences associated with the incorporation of RWD and RWE into the realm of HTA.
Methods: A comprehensive PRISMA-based systematic review was performed in July 2022 in PubMed/Medline, Scopus, IDEAS-RePEc, International HTA database, and Centre for Reviews and Dissemination with ad hoc supplementary search in Google Scholar and international organization websites. The review included pre-determined inclusion criteria while the selection of eligible studies, the data extraction process and quality assessment were carried out using standardized and transparent methods.
Results: Twenty-nine (n = 29) studies were included in the review out of 2,115 studies identified by the search strategy. In various global contexts, disparities in RWD utilization were evident, with randomized controlled trials (RCTs) serving as the primary evidence source. RWD and RWE played pivotal roles, surpassing relative effectiveness assessments (REAs) and significantly influencing decision-making and cost-effectiveness analyses. Identified challenges impeding RWD integration into HTA encompassed limited local data access, complexities in non-randomized trial design, data quality, privacy, and fragmentation. Addressing these is imperative for optimal RWD utilization. Incorporating RWD/RWE in HTA yields multifaceted advantages, enhancing understanding of treatment efficacy, resource utilization, and cost analysis, particularly via patient registries. RWE complements assessments of advanced therapy medicinal products (ATMPs) and rare diseases. Local data utilization strengthens HTA, bridging gaps when RCT data is lacking. RWD aids medical device decision-making, cancer drug reassessment, and indirect treatment comparisons. Challenges include data availability, stakeholder acceptance, expertise, and privacy. However, standardization, training, collaboration, and guidance can surmount these barriers, fostering enhanced RWD utilization in HTA.
Conclusion: This study highlights the intricate global landscape of RWD and RWE acceptance in HTA. Recognizing regional nuances, addressing methodological challenges, and promoting collaboration are pivotal, among others, for leveraging RWD and RWE effectively in healthcare decision-making.

研究目的：本系统综述旨在评估真实世界数据（real-world data, RWD）与真实世界证据（real-world evidence, RWE）在卫生技术评估（health technology assessment, HTA）流程中的当前应用与接受度；此外，本研究旨在厘清利益相关方对HTA中RWD与RWE的观点，并阐明将RWD与RWE纳入HTA领域所面临的障碍、困难、发展前景及潜在影响。
研究方法：本研究于2022年7月开展基于系统综述与荟萃分析优先报告条目（Preferred Reporting Items for Systematic Reviews and Meta-Analyses, PRISMA）的全面系统综述，检索数据库包括PubMed/Medline、Scopus、IDEAS-RePEc、国际卫生技术评估数据库（International HTA Database）及综述与传播中心（Centre for Reviews and Dissemination），并在Google Scholar及国际组织官网开展特设补充检索。本研究预设纳入标准，且采用标准化、透明化方法完成合格研究筛选、数据提取及质量评估流程。
研究结果：本研究共检索到2115篇文献，最终纳入29项（n=29）研究。在全球不同地区场景中，RWD的应用存在显著差异，随机对照试验（randomized controlled trials, RCTs）仍是主要的证据来源。RWD与RWE发挥了关键作用，其影响力超越相对有效性评估（relative effectiveness assessments, REAs），并对决策制定与成本效益分析产生显著影响。阻碍RWD纳入HTA的已识别挑战包括：本地数据获取受限、非随机试验设计复杂度高、数据质量参差不齐、隐私保护问题及数据碎片化。优化RWD的应用必须解决上述问题。将RWD/RWE纳入HTA可带来多维度优势，尤其是通过患者登记库，能够加深对治疗有效性、资源利用及成本分析的理解。RWE可补充先进治疗药品（advanced therapy medicinal products, ATMPs）与罕见病的评估工作。本地数据的应用可强化HTA能力，弥补RCT数据缺失的空白。RWD可辅助医疗器械决策、癌症药物重新评估及间接治疗比较。现存挑战还包括数据可及性、利益相关方接受度、专业人才储备及隐私保护问题。但通过标准化建设、培训、协作与政策指导，可克服上述障碍，推动HTA中RWD的更广泛应用。
研究结论：本研究阐明了HTA中RWD与RWE接受度的复杂全球格局。在医疗决策中有效利用RWD与RWE，需重视区域差异、应对方法学挑战并加强协作，此三者为关键举措之一。