Urgently seeking efficiency and sustainability of clinical trials in global health
Introduction
The field of global health prioritises improving health and achieving health equity for all. Research in this area is focused on improving health outcomes and addressing inequities in low-income and middle-income countries (LMICs).1,2 LMICs continue to have comparatively higher rates of and burden from infectious diseases and other communicable diseases (eg, HIV, malaria, diarrhoeal diseases, and sepsis) than do high-income countries (HICs). Among many health challenges, shorter life expectancy and higher rates of maternal and child mortality in LMICs, relative to HICs, have delayed their developmental and economic potential.3–5 Thus, interventions to reduce disease and improve health in LMICs, particularly, should be a global priority.
Progress has been made in the prevention and treatment of infectious diseases and other communicable diseases, with randomised clinical trials (RCTs) having been used to generate evidence on the effectiveness of important therapeutics. RCTs are considered robust methods for evaluating the effectiveness of therapies because these studies fulfil the primary assumption of statistical testing—ie, the equality of treatment by minimising selection bias and creating groups that are comparable in prognostic factors—thereby establishing a causal effect of the treatment on the outcome.6,7 RCTs are an important methodological tool in global health research for generating high-level evidence to inform the development of context-specific and international guidelines on preferred interventions that can be delivered at scale to populations in need.5
Although the challenges in reducing the burden of communicable diseases in LMICs are considerable, there have been important demographic and epidemiological shifts occurring rapidly in LMICs. The global population is estimated to increase from approximately 7·7 billion people in 2019, to 8·5 billion by 2030, and further to 9·7 billion people in 2050.6 The most rapid growth is projected to occur in sub-Saharan Africa, with the population size expected to double by 2050.6 With the increasing life expectancy in LMICs, there will be a consequent increase in the burden of non-communicable diseases (NCDs), because these diseases are more common in adults older than 65 years.7–9 This rapid change in population size and demographics, in addition to the rising burden of NCDs, presents many challenges for meeting the Sustainable Development Goals.8–10 A change in approach is needed to rapidly answer research questions that can accompany the changes in demographics and disease burden in LMICs, where resources are limited and health research capacity is low. In the current era, efficient research designs that can answer multiple complex research questions simultaneously with a minimised sample size and trial duration will be beneficial, but there are also many logistical and methodological barriers to overcome before such an approach is a reality.