Identifying target populations to align with decision makers' needs.

ABSTRACT

Randomized trials estimate the average treatment effect within individuals that are eligible, invited and agree to enroll. However, decision makers often require evidence that extends beyond the trial's enrolled population to inform policy or actions for their specific target population. Each decision maker has distinct target populations, the composition of which may not often align with that of the trial population. As researchers, we should identify a decision maker for whom we aim to generate evidence early in the research process. We can then specify a target population of their interest and determine if a policy or action can be informed using results from a trial alone, or if additional complementary real-world data and analysis are required. In this commentary, we outline five key groupings of decision makers policymakers, payers, purchasers, providers, and patients. We then specify relevant target populations for decision makers interested in the effectiveness of beta-blockers following a myocardial infarction with preserved ejection fraction. Finally, we summarize the scenarios in which results from a randomized trial may or may not apply to these target populations and suggest relevant analytic approaches that can generate evidence to better align with a decision makers' needs.