Observational research, randomised trials, and two views of medical science

Abstract

Two views exist of medical science: one emphasises discovery and explanation, the other emphasises evaluation of interventions. This essay analyses in what respects these views differ, and how they lead to opposite research hierarchies, with randomisation on top for evaluation and at bottom for discovery and explanation. The two views also differ strongly in their thinking about the role of prior specification of a research hypothesis. Hence, the essay explores the controversies surrounding subgroup analyses and multiplicity of analyses in observational research. This exploration leads to a rethinking of the universally accepted hierarchy of strength of study designs, which has the randomised trial on top: this hierarchy may be confounded by the prior odds of the research hypothesis. Finally, the strong opinions that are sometimes displayed in pitting the two types of medical science against each other may be explained by a difference in "loss function": the difference in penalty for being wrong. A longer, more detailed version of this paper is found in supplementary Text S1. © 2008 Jan P. Vandenbroucke.