questions (e.g., how, when, for whom, and in what settings are treatments best used?), yet the current research paradigm, based on a hierarchical arrangement of study designs, assigns greater weight or strength to evidence produced from methods higher in the hierarchy, without necessarily considering the appropriateness of the design for the particular question under investigation. For example, the advantages of strong internal validity, a key characteristic of the randomized controlled trial (RCT)—long considered the gold standard in clinical research—are often muted by constraints in time, cost, and limited external validity or applicability of results. And, although the scientific value of well-designed clinical trials has been demonstrated, for certain research questions, this approach is not feasible, ethical, or practical and may not yield the answer needed. Similarly, issues of bias and confounding inherent to observational, simulation, and quasi-experimental approaches may limit their use and enhancement, even for situations and circumstances requiring a greater emphasis on external validity.
Especially given the growing capacity of information technology to capture, store, and use vastly larger amounts of clinically rich data and the importance of improved understanding of an intervention’s effect in real-world practice, the advantages of identifying and advancing methods and strategies that draw research closer to practice become even clearer.
Against the backdrop of the growing scope and scale of evidence needs, limits of current approaches, and potential of emerging data resources, the Institute of Medicine (IOM) Roundtable on Evidence-Based Medicine, now the Roundtable on Value & Science-Driven Health Care convened the Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches workshop. The issues motivating the meeting’s discussions are noted in Box S-1, the first of which is the need for a deeper and broader evidence base for improved clinical decision making. But also important are the needs to improve the efficiency and applicability of the process. Underscoring the timeliness of the discussion is recognition of the challenges presented by the expense, time, and limited generalizability of current approaches, as well as of the opportunities presented by innovative research approaches and broader use of electronic health records that make clinical data more accessible. The overall goal of the meeting was to explore these issues, identify potential approaches, and discuss possible strategies for their engagement.
Participants examined ways to expedite the development of clinical effectiveness information, highlighting the opportunities presented by innovative study designs and new methods of analysis and modeling; the size and expansion of potentially interoperable administrative and clinical datasets; and emerging research networks and data resources. The presentations and discussion emphasized approaches to research and learning that had the potential to supplement, complement, or supersede RCT findings and