Identifying target populations to align with decision-makers' needs.

Abstract

Randomized trials estimate the average treatment effect within individuals who 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 5 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 after 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-maker's needs. This article is part of a Special Collection on Pharmacoepidemiology.