Most randomized controlled trials are unable to generate information about a product’s real-world effectiveness. Therefore, payers use real-world evidence (RWE) generated in observational studies to make decisions regarding formulary inclusion and coverage. While some payers generate their own RWE, most cautiously rely on RWE produced by manufacturers who have a strong financial interest in obtaining coverage for their products.
We propose a process by which an independent body would certify observational studies as generating valid and unbiased estimates of the effectiveness of the intervention under consideration. This proposed process includes (a) establishing transparent criteria for assessment, (b) implementing a process for receipt and review of observational study protocols from interested parties, (c) reviewing the submitted protocol and requesting any necessary revisions, (d) reviewing the study results, (e) assigning a certification status to the submitted evidence, and (f) communicating the certification status to all who seek to use this evidence for decision making.
Accrediting organizations such as the National Center for Quality Assurance and the Joint Commission have comparable goals of providing assurance about quality to those who look to their accreditation results. Although we recognize potential barriers, including a slowing of evidence generation and costs, we anticipate that processes can be streamlined, such as when familiar methods or familiar datasets are used. The financial backing for such activities remains uncertain, as does identification of organizations that might serve this certification function. We suggest that the rigor and transparency that will be required with such a process, and the unassailable evidence that it will produce, will be valuable to decision makers.
1Co-director, Center for Drug Safety and Effectiveness, Johns Hopkins University, and Professor of Medicine and Epidemiology, Division of General Internal Medicine, Department of Medicine, Johns Hopkins Medicine/Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland.
2Principal, Big Health Data, Falmouth, Maine. 3Master’s Student, Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland. 4Professor of Pharmaceutical Outcomes Research and Policy, School of Pharmacy, University of Washington, Seattle. 5Senior Medical Affairs Leader−Neuroscience, Global Medical Affairs, AstraZeneca, Cambridge, Massachusetts. 6Vice President, Global Services and Real-World Evidence Solutions, IMS Health, Plymouth Meeting, Pennsylvania. 7Co-director, Center for Drug Safety and Effectiveness, Johns Hopkins University, and Associate Professor of Medicine and Epidemiology, Division of General Internal Medicine, Department of Medicine, Johns Hopkins Medicine/Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland.