Recent controversies on comparative effectiveness research investigations: Challenges, opportunities, and pitfalls

Abstract

The purpose of comparative effectiveness research (CER) is to improve health outcomes by developing and disseminating evidence-based information about which currently available interventions and practices are most effective for patients. While Randomized Controlled Trials (RCT) are the hallmark of scientific proof, when they have been used to compare interventions used in variable ways by different clinicians (Comparative Effectiveness RCTs, CER RCTs) they have at times, generated controversy. Usually the background for the CER RCT is a range of ‘standard therapy’ or ‘standard of care’. This may have been adopted on observational data alone, pilot data. At times, such prior data may derive from populations that differ from the population in which the widely variable standard approach is being applied. We believe controversies related to these CER-RCTs result from confusing ‘accepted’ therapies and ‘rigorously evaluated therapies”. We first define evidence based medicine, and consider how well neonatology conforms to that definition. We then contrast the approach of testing new therapies and those already existing and widely adopted, as in CER-RCTs. We next examine a central challenge in incorporating the control arm within CER, and aspects of the ‘titrated’ trial. We finally briefly consider some ethical issues that have arisen, and briefly discuss the wide range of neonatology practices that could be subject to CER-RCTs or alternative CER-based strategies that might inform practice in the absence of RCTs. Throughout, we emphasize the lack of awareness of the lay community, and indeed many researchers or commentators, in appreciating the wide variation of standard of care. There is a corresponding need to identify the best uses of available resources that will lead to the best outcomes for our patients. We conclude that CER is an essential methodology in modern neonatology to address many unanswered questions and test unproven therapies in newborn care.

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What Is New about Transfusions for Preterm Infants? An Update

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