Observational Studies in a Learning Health System - Workshop Summary


Note: Proceedings contain the opinion of the presenters, but do NOT reflect the conclusions of the Health and Medicine Division or the National Academies. Learn more about the differences between Reports and Proceedings.

Clinical research is constantly advancing, although perhaps not fast enough to meet the challenges and seize the opportunities presented. New tools are emerging. While challenges remain, these tools have the potential to accelerate the research process and to allow an approach to clinical research that applies the most appropriate methods given the requirements of the situation. This approach includes the leveraging of the information collected in the process of delivering care to drive processes for new insights and continuous improvement, which is at the heart of a learning health system. An IOM workshop, sponsored by the Patient Centered Outcomes Research Institute, was convened to identify the leading approaches to observational studies, chart the course for the use of this growing utility, and guide and grow their use in the most responsible fashion possible.

Through a series of expert presentations and discussions, workshop participants considered how observational studies can be made to be more rigorous and internally valid, how to deal with bias, the use of observational studies to generalize findings from randomized controlled trials, and how to evaluate treatment heterogeneity. Additionally, presentations at the workshop considered the role of observational studies predicting individual responses to treatment. This document summarizes the workshop.