Beyond Causality: Additional Benefits of Randomized Controlled Trials for Improving Health Care Delivery

Abstract

Policy Points Policymakers at federal and state agencies, health systems, payers, and providers need rigorous evidence for strategies to improve health care delivery and population health. This is all the more urgent now, during the COVID-19 pandemic and its aftermath, especially among low-income communities and communities of color. Randomized controlled trials (RCTs) are known for their ability to produce credible causal impact estimates, which is why they are used to evaluate the safety and efficacy of drugs and, increasingly, to evaluate health care delivery and policy. But RCTs provide other benefits, allowing policymakers and researchers to: 1) design studies to answer the question they want to answer, 2) test theory and mechanisms to help enrich understanding beyond the results of a single study, 3) examine potentially subtle, indirect effects of a program or policy, and 4) collaborate closely to generate policy-relevant findings. Illustrating each of these points with examples of recent RCTs in health care, we demonstrate how policymakers can utilize RCTs to solve pressing challenges.

Figure 1

Randomized Controlled Trials Can Make Credibly Estimating Causal Impact Easier: A Stylized Illustration of Health Insurance's Impact on Health [Colour figure can be viewed at wileyonlinelibrary.com]