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

摘要

The current paradigms for effectiveness research and drug development face increasingly acute challenges in cost, timing, and applicability, given their reliance on classic randomized controlled trials. Opportunities to engage these challenges by the use of large simple trial (LST) designs and the integration of research at the point of care prompted the Institute of Medicine's Roundtable on Value & Science-Driven Health Care (the Roundtable) and the Forum on Drug Discovery, Development, and Translation (the Forum) to convene a workshop, Large Simple Trials and Knowledge Generation in a Learning Health System, which is summarized in this publication. Experts from a wide range of disciplines-including health information technology, research funding, clinical research methods, statistics, patients, product development, medical product regulation, and clinical outcomes research-met to marshal a better understanding of the issues, options, and approaches to accelerating the use of LSTs. This publication summarizes discussions on the potential of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development, including efficacy and effectiveness assessments, in a continuously learning health system. The work of the Roundtable is focused on moving toward a continuously learning health system, one in which every care encounter is an opportunity for learning and evidence is applied to ensure and improve best care practices. Since its inception in 2006, the Roundtable has set out to help realize this vision through the support, involvement, and activities of senior leadership from key stakeholders in the health care system.