Best Practices for Study Informativeness – Background and development
Design, Analyze, Communicate: Working together to improve clinical study outcomes.
Background
All stakeholders in global health want the best outcomes for low-resource populations. One of the best approaches for finding, testing, and normalizing new health interventions is clinical research. BMGF has funded hundreds of clinical trials and clinical studies over the last 20 years. The investigators that BMGF have funded and their teams deserve tremendous recognition. Their research has been of huge value in advancing public health in low-resource settings.
Unfortunately, like any research funder, BMGF funds some studies that end with results that do not answer the question(s) they were designed to answer. These studies end in a variety of ways that are insufficiently persuasive to move leaders to implement a change in policy or to initiate a subsequent study in a clinical development plan. For example, studies might end without sufficient power to answer the research question; might run at a site with inadequate disease burden and fail to meet recruitment goals or run too long and lose too many participants to follow-up; or might be unclear about the optimally effective, safe, and cost-effective dose. Unfortunately, it only takes one among dozens of decisions being wrong to lead to uninformative results. In addition to traditional clinical trials for medicines, uninformative results in global health research also occur when evaluating food, vitamins, and other non-therapeutic interventions. Study weaknesses are applicable to clinical research across global health conditions and environments as well as in crisis settings.
Best Practice development
A number of global experts have, in recent years, investigated this dynamic of uninformativeness. Investigations have included surveys of low-resource trial implementers and designers, searches in the largest clinical trial registries by government administrators and educators, and assessments from disease-specific working groups.[2,3,7] In concert with Wellcome Trust and other funders, BMGF has investigated the most common design-related factors associated with studies ending successfully and informatively. These clinical study ‘features’ are presented as the ‘best practices’ of BMGF’s Design, Analyze, Communicate (DAC) program.
None of these best practices is new. Rather, they are well-regarded and accepted research methods, often referenced in international guidelines. They represent solid science, and global health researchers generally accept these practices and acknowledge the need to implement them in their research. However, some global health studies lack one or more of the best practices even though the absence of just one risks uninformative outcomes.
Please note: Implementors of studies should also always refer to relevant regulatory standards and guidelines (e.g. FDA, EMA) to assist with study/protocol design.
