Advancing the role of real-world evidence in comparative effectiveness research

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. Plain language summary: What is this article about?: This article discusses how researchers and healthcare decision-makers can determine the best way to compare the effectiveness of different drugs. This type of research, known as comparative effectiveness research (CER), helps to make better healthcare decisions by providing information on how new drugs perform compared with existing treatments. The article aims to offer a tool that guides analysts in choosing the right method for their CER based on the data they have. What were the results?: The study found that there are no existing papers that clearly explain which method to use for different CER questions under various conditions. To address this gap, the authors created a tool- a methods' flowchart- that will facilitate a transparent way of choosing which method should be used for a specific CER question. This tool helps researchers start with a specific question and then choose the best method to answer it, rather than forcing a one-size-fits-all approach. What do the results of the study mean?: The results mean that researchers and decision-makers now have a clear guide to help them choose the most appropriate methods for their CER questions. This new tool aims to make CER more standardized and consistent, which can lead to higher quality research and better, evidence-based decisions in healthcare. Ultimately, this can improve patient care by ensuring that new therapies meet real-world needs. Shareable abstract: This study introduces a methods flowchart to guide comparative effectiveness research (CER). It helps identify the best analytical approach, ensuring standardized, high-quality, evidence-based healthcare decisions. #CER #decision-making #comparative-effectiveness, #evidencebased