The Patient-Centered Outcomes Research Institute sponsored an Institute of Medicine workshop in Washington, D.C., on “Observational Studies in a Learning Health System.” The workshop initiated a comprehensive evaluation of the complementary roles of randomized controlled trials and observational studies, while looking ahead to the potential of very large data sets for observational studies in the evidence generation needs of a learning healthcare system.
Workshop discussions focused on:
- Exploring the role of observational studies (OS) in the generation of evidence to guide clinical and health policy decisions, with a focus on individual patient care, in a learning health system;
- Considering concepts of OS design and analysis, emerging statistical methods, the opportunities and challenges of OS to supplement evidence from experimental methods, identify treatment heterogeneity, and provide effectiveness estimates tailored for individual patients;
- Identifying stakeholder needs, and the barriers hindering the broader application of OS-generated evidence for decision-making; Engaging colleagues from disciplines typically underrepresented in clinical evidence discussions; and
- Suggesting strategies for accelerating progress in the appropriate use of OS for evidence generation.