Why Observational Studies Should Be Among The Tools Used In Comparative Effectiveness Research

Abstract

Doctors, patients, and other decision makers need access to the best available clinical evidence, which can come from systematic reviews, experimental trials, and observational research. Despite methodological challenges, high-quality observational studies have an important role in comparative effectiveness research because they can address issues that are otherwise difficult or impossible to study. In addition, many clinical and policy decisions do not require the very high levels of certainty provided by large, rigorous randomized trials. This paper provides insights and a framework to guide good decision making that involves the full range of high-quality comparative effectiveness research techniques, including observational research.

The GRACE Checklist for Rating the Quality of Observational Studies of Comparative Effectiveness: A Tale of Hope and Caution

“…[2][3][4][5][6][7][8][9][10][11][12][13][14][15] These papers have face validity and largely appear reasonable, but there is little, if any, evidence that any of these recommendations can actually distinguish studies of sufficient quality to merit serious evaluation for a particular clinical or payment decision. For example, some guidelines address potential conflicts of interest by calling for full disclosure, a standard journal practice that relies on individual assessment of potential conflicts.…”

Dabigatran Versus Warfarin for Atrial Fibrillation in Real-World Clinical Practice

“…In the absence of such evidence, well-designed observational studies serve an important role, and the synthesis of data from these studies can prove valuable, in evidence appraisal. 5,6 Highquality observational studies, unlike many RCTs, may have better generalizability to real-world practice as they often have less stringent inclusion criteria and patients are treated in a clinical setting under usual care. We conducted this review within the framework that observational studies are an important contribution to the totality of evidence to understand the benefits and risks of an intervention, especially when there are gaps in evidence from RCTs.…”

“…As such, high-quality observational studies can importantly contribute to the totality of evidence for the benefits and risks of an intervention, especially when there are gaps in evidence from RCTs. [5][6][7] Under this framework, we sought to review real-world observational evidence for the comparative effectiveness and safety of dabigatran and warfarin among patients with NVAF.…”

Evolving Reimbursement And Pricing Policies For Devices In Europe And The United States Should Encourage Greater Value

“…For example, the comparative effectiveness research initiative in the United States has focused attention on pragmatic randomized controlled trials, which take place in real-world practices as well as observational studies and patient registries. 28,29 Linking Evidence Of Value To Reimbursement Value-based reimbursement, an approach increasingly of interest to US private payers, may provide a viable option to better incorporate evidence into reimbursement decisions. In a survey of employer-sponsored health plans, Niteesh Choudhry and coauthors 30 estimated that 81 percent of large employers plan to offer this approach in the near future.…”