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

dataset

posted on 2024-10-11, 14:04 authored by Monica Daigl, Seye Abogunrin, Felipe Castro, Sarah F McGough, Rachele Hendricks Sturrup, Cornelis Boersma, Keith Abrams

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 sources
Appendix D: Mapping of methods flowchart elements to evidence sought from included guidance documents
Appendix 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.