Evidence-Based Policy Making for Public Health Interventions in Cardiovascular Diseases: Formally Assessing the Feasibility of Clinical Trials.

View Abstract

Implementation of prevention policies has often been impeded or delayed due to the lack of randomized controlled trials (RCTs) with hard clinical outcomes (eg, incident disease, mortality). Despite the prominent role of RCTs in health care, it may not always be feasible to conduct RCTs of public health interventions with hard outcomes due to logistical and ethical considerations. RCTs may also lack external validity and have limited generalizability. Currently, there is insufficient guidance for policymakers charged with establishing evidence-based policy to determine whether an RCT with hard outcomes is needed before policy recommendations. In this context, the purpose of this article is to assess, in a case study, the feasibility of conducting an RCT of the oft-cited issue of sodium reduction on cardiovascular outcomes and then propose a framework for decision-making, which includes an assessment of the feasibility of conducting an RCT with hard clinical outcomes when such trials are unavailable. We designed and assessed the feasibility of potential individual- and cluster-randomized trials of sodium reduction on cardiovascular outcomes. Based on our assumptions, a trial using any of the designs considered would require tens of thousands of participants and cost hundreds of millions of dollars, which is prohibitively expensive. Our estimates may be conservative given several key challenges, such as the unknown costs of sustaining a long-term difference in sodium intake, the effect of differential cotreatment with antihypertensive medications, and long lag time to clinical outcomes. Thus, it would be extraordinarily difficult to conduct such a trial, and despite the high costs, substantial risk for a spuriously null result. A robust framework, such as the one we developed, should be used to guide policymakers when establishing evidence-based public health interventions in the absence of trials with hard clinical outcomes.

Investigators
Abbreviation
Circ Cardiovasc Qual Outcomes
Publication Date
2020-09-28
Page Numbers
CIRCOUTCOMES119006378
Pubmed ID
32981337
Medium
Print-Electronic
Full Title
Evidence-Based Policy Making for Public Health Interventions in Cardiovascular Diseases: Formally Assessing the Feasibility of Clinical Trials.
Authors
Foti K, Foraker RE, Martyn-Nemeth P, Anderson CAM, Cook NR, Lichtenstein AH, de Ferranti SD, Young DR, Hivert MF, Ross R, Deedwania P, Whitsel LP, Appel LJ