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Reducing the under-5 mortality rate (U5MR) is a key public health priority and remains a persistent challenge in low- and middle-income countries. Many interventions have been developed in attempts to lower U5MR through partnerships between governments, funding partners, researchers, product developers and non-governmental organisations. The well-intentioned desire to use data to set priorities has led to many large-scale evaluations that specify U5MR as a primary outcome. The results of these evaluations are then used to inform investments, policies and advocacy efforts. While important insights can be gained from tracking U5MR at the population level, these evaluations often present serious scientific, pragmatic and ethical challenges. In this article, we outline six problems associated with evaluations targeting a U5MR outcome. We describe why U5MR is primarily informative as an outcome measure for trials of new clinical procedures or treatments designed to cure a life-threatening condition. For interventions that aim to lower U5MR by preventing disease or other potentially fatal events at the population level, or by improving implementation of existing evidence-based interventions, we recommend evaluations that specify intermediate outcomes that offer timelier information and more reliable causal inference. We present limitations to using results of a single study as the basis for policy or funding decisions about an intervention or implementing partner. Finally, we reinforce the need for rigorous methodology, national periodic U5MR measurement, verbal and social autopsies, strong mixed-methods implementation research and reliance on local implementer knowledge.
Kenny et al. (Mon,) studied this question.