On 11-12 December 2017, WHO convened about 30 experts in public health, clinical trials, epidemiology and regulatory to attend the 1 st WHO R&D Blueprint consultation on therapeutic evaluation in Public Health Emergencies (PHEs). The meeting was hosted by the University Medical Center Utrecht (UMCU) in Zeist, The Netherlands. This first consultation aimed to engage participants in a series of efforts to improve the design, implementation, and conduct of therapeutic and prophylactic studies (both clinical trials and observational studies) during Public Health Emergencies, through an inclusive and evidence-based process. The Blueprint priority pathogens and the current landscape of experimental molecules were used to frame the conversation and illustrate the group’s thinking and rationale based on lessons learned from past and current outbreaks. Participants agreed to initiate a work plan with 4 working groups: (i) a comprehensive methodological document on therapeutic study designs; (ii) a decision tree to guide the design of a therapeutic trial and promote discussion around key methodological choices; (iii) statistical and mathematical methodology for therapeutic and prophylactic studies; (iv) generic annotated protocols for various study designs. With respect to the above working groups, participants agreed to develop tools and material during year 2018. A next F2F consultation is planned in Q2 2018 and will provide the opportunity to collectively review activities of the groups.
