The trials methodology research agenda—an international multi-stakeholder priority setting exercise

Abstract Background Trials methodology research priorities must be regularly reassessed to address contemporary methodological challenges. Over a decade has passed since the original priority setting exercise surveyed UK clinical trials unit directors. We conducted a two-stage international, multi-stakeholder survey to identify and rank current priorities in clinical trial methodology. Methods Stakeholders—researchers, trialists, healthcare professionals, patient and public representatives, funders, research ethics committee members, and others—were recruited through professional networks and snowball sampling. In stage 1, participants responded to four open-ended questions covering trial planning, conduct, analysis, and reporting. Responses were categorised into 34 research questions. In stage 2, participants reviewed the questions in random order, selected their top 10, and ranked them. Reverse scoring was applied, with scores summed within stakeholder groups, converted to rank-based scores, and combined so each group contributed equally regardless of size. Additional questions were independently coded by two authors against the 34 predefined questions, and new themes identified. Results In stage 1, 274 participants from 20 countries provided 2409 suggestions, categorised under 34 research questions. In stage 2, 431 respondents from 25 countries participated, of whom 341 (79%) across seven stakeholder groups completed priority ranking. The top 10 priority questions (including a joint seventh rank) were (1) designing relevant and impactful trials, (2) ensuring representative participants, (3) improving recruitment, (4) identifying and prioritising outcomes, (5) using healthcare systems data, (6) participant involvement in planning, (7=) transparency of trials, (7=) optimising trial approvals, (9) reporting and presenting findings, and (10) sharing and communicating findings. The top three questions appeared in all seven stakeholder groups’ top 10. Patient and public representatives ( n = 40) ranked ‘participant involvement in planning’ highest, while ethics committee members ( n = 9) uniquely prioritised ‘consent and safety’. ‘Innovative trials designs’ and ‘artificial intelligence in trials’ emerged as the most frequent new themes suggested. Conclusions This work provides an updated stakeholder-informed agenda for trials methodology research. The top three priorities were designing relevant and impactful trials, ensuring representative participants, and improving recruitment, each appeared in the top 10 for all stakeholder groups. Despite consensus on core priorities, differences between professional and patient-public stakeholders highlight areas for future methodology research and funding.