Randomized Clinical Trials That Advance Health and Health Equity

Abstract

Randomized clinical trials (RCTs) provide the highest level of evidence that shape patient care, guideline recommendations, performance benchmarks, and health care coverage decisions. JAMA Internal Medicine is issuing a call for RCTs that advance health and health equity by studying the efficacy and/or effectiveness of clinical, policy, and public health approaches. We are interested in publishing traditional individual-level, parallel group RTCs, as well as studies using other randomization approaches, such as Bayesian adaptive, cluster-randomization, stepped-wedge, crossover, and waitlist designs.Randomization accounts for known, unknown, and unobservable confounders, thus offering the most effective tool for testing causal effects with strong internal validity. However, RCTs testing efficacy can lack external validity due to tight eligibility criteria that may exclude many individuals who might benefit from the intervention, may include interventions that are not feasible in clinical practice, and interventions that are not based in communities that bear a disproportionate burden of the condition of interest. For research to equitably advance health and health care, clinical trials need to address feasible community-based interventions that include patient populations affected by the condition under study. Systematic underrepresentation of patients with disabilities, multiple comorbidities or social risks in RCTs limit clinicians' ability to decide if the results apply to the patient in front of them, which can perpetuate or even magnify health inequities. The choice of randomization design can contribute to equity goals. For example, compared with a parallelgroup design, a stepped-wedge or wait-list design may be a more attractive approach to diverse sites and participants because these designs allow more participants to access the intervention and often yield a more representative study population. In addition, Bayesian adaptive designs can improve efficiency relative to frequentist approaches. 1,2 Efforts to date to improve inclusion of underrepresented groups in RCTs 3,4 have resulted in marginal improvements. In 2022, the US Food and Drug Administration issued guidance to research sponsors recommending a recruitment plan to enroll participants from underrepresented racial and ethnic backgrounds. This optional recommendation, however, has had minimal impact. A growing body of research has highlighted that the relationship between race and ethnicity and clinical outcomes is predominantly mediated by social determinants of health.