Php258 - development of a real world evidence study design tool

The main objective was to develop a holistic and concise tool to encourage a methodical approach to Real World Evidence (RWE) study planning and implementation. A literature review was performed to identify publications with decision and/or visual aids describing best practices for planning and/or conducting RWE studies. Articles with English abstracts were identified through Embase and MEDLINE database searches, and through supplemental internet searches of relevant professional organization websites. Following conceptual framework development, a tool was designed and pilot tested with 30 RWE professionals in 9 European countries via 6 individual and group interviews, before finalisation. 371 abstracts were identified; title and abstract screening was completed by a single assessor; 3 articles and 6 online sources were selected for detailed review. Of these, most focused on a subset of RWE study types, such as pragmatic trials or database studies, or on a subset of RWE objectives, such as evaluating clinical effectiveness. Only two sources included visual aids (one a detailed interactive online resource and the other a brief illustrative diagram). A gap was identified for a concise tool that would support decision-making on RWE study design. The newly developed tool captures a multi-step decision process, including consideration of: research objective(s), product development status, study conduct in routine practice, outcomes of interest, data availability in routine practice, need for primary data collection and randomisation. Finally, the tool guides on: appropriate study type and methodology (covering options under the categories of retrospective, prospective observational and pragmatic trial) and applicable regulatory standards. The new study design tool addresses a gap in the literature for a concise, easy-to-follow aid that captures a broad range of RWE study types and objectives. The tool can enhance RWE decision-making involving multiple stakeholders and replace ad-hoc RWE study planning processes with a robust approach.