Large Simple Trials and Knowledge Generation in a Learning Health System - Workshop Summary


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Despite a robust clinical research enterprise, a gap exists between the evidence needed to support care decisions and the evidence available. Streamlined approaches to clinical research provide options for progress on these challenges. Large simple trials (LSTs), for example, generally have simple randomization, broad eligibility criteria, enough participants to distinguish small to moderate effects, focus on outcomes important to patient care, and use simplified approaches to data collection. Significant opportunities, including the wide-spread adoption of electronic health records, could accelerate the potential for the use of LSTs to efficiently generate practical evidence for medical decision making and product development.

To address these opportunities, as well as challenges, the Institute of Medicine’s Roundtable on Value & Science-Driven Health Care and the Forum on Drug Discovery, Development, and Translation convened a public workshop. Workshop participants included a broad range of experts in clinical research, medical product development, patient advocacy, biostatistics, health information technology, clinical data standards, ethics, legal/regulatory issues, and health care payment and financing. The workshop highlighted the pros and cons of the design characteristics of LSTs, explored the utility of LSTs on the basis of case studies of past successes, and considered the challenges and opportunities for accelerating the use of LSTs in the context of a U.S. clinical trials enterprise. This document summarizes the workshop.