Supplementary material: Advancing the role of real-world evidencein comparative effectiveness research

These are peer-reviewed supplementary materials for the article 'Advancing the role of real-world evidence in comparative effectiveness research' published in the Journal of Comparative Effectiveness Research.Appendix A: Modified SPIDER framework for inclusion/exclusion criteria of publications*Appendix B: Search strategy (Embase and MEDLINE via embase.com)Appendix C: PRISMA Flow Diagram for new systematic reviews which included searches of databases, registers and other sourcesAppendix D: Mapping of methods flowchart elements to evidence sought from included guidance documentsAppendix E: Details of included guidance documents and reporting of data relevant to CER methodological decision-making (a checkmark denotes that the topic is discussed in the publication, whereas a cross indicates that the topic is not addressed)Aim: Comparative effectiveness research (CER) is essential for making informed decisions about drug access. It provides insights into the effectiveness and safety of new drugs compared with existing treatments, thereby guiding better healthcare decisions and ensuring that new therapies meet the realworld needs of patients and healthcare systems. Objective: To provide a tool that assists analysts and decision-makers in identifying the most suitable analytical approach for answering a CER question, given specific data availability contexts. Methods: A systematic literature review of the scientific literature was performed and existing regulatory and health technology assessment (HTA) guidance were evaluated to identify and compare recommendations and best practices. Based on this review a methods flowchart that synthesizes current practices and requirements was proposed. Results: The review did not find any papers that clearly identified the most appropriate analytical approach for answering CER questions under various conditions. Therefore, a methods flowchart was designed to inform analyst and decision makers choices starting from a well-defined scientific question. Conclusion: The proposed methods flowchart offers clear guidance on CER methodologies across a range of settings and research needs. It begins with a well-defined research question and considers multiple feasibility aspects related to CER. This tool aims to standardize methods, ensure rigorous and consistent research quality and promote a culture of evidencebased decision-making in healthcare.

本数据集为发表在《比较有效性研究杂志》上的文章《推进真实世界证据在比较有效性研究中的作用》的同行评审补充材料。附录A：出版物纳入/排除标准的修改版SPIDER框架；附录B：检索策略（通过embase.com的Embase和MEDLINE数据库）；附录C：针对包含数据库、登记处和其他来源的新系统评价的PRISMA流程图；附录D：方法流程图元素与从纳入的指导文件中寻求的证据的映射；附录E：纳入的指导文件细节以及与比较有效性研究（CER）方法论决策相关的数据报告（勾选表示出版物中讨论了该主题，而叉号表示未涉及）。目标：比较有效性研究（CER）对于药物可及性决策的明智决策至关重要。它揭示了新药与现有治疗方法的疗效和安全性，从而指导更佳的医疗服务决策，并确保新疗法满足患者和医疗体系的真实世界需求。目标：提供一种工具，协助分析师和决策者根据特定的数据可用性情境，确定回答CER问题的最合适的分析方法。方法：通过对科学文献的系统文献综述，并评估现有的监管和健康技术评估（HTA）指南，以识别和比较建议和最佳实践。基于此综述，提出了一个综合当前实践和要求的方法流程图。结果：综述未发现任何明确识别在各种条件下回答CER问题的最合适分析方法的论文。因此，设计了一种方法流程图，从明确定义的科学问题出发，为分析师和决策者的选择提供信息。结论：提出的方法流程图为不同设置和研究需求下的CER方法论提供了清晰的指导。它始于一个明确的研究问题，并考虑与CER相关的多个可行性方面。该工具旨在标准化方法，确保严格和一致的研究质量，并促进医疗保健中的基于证据的决策文化。