National Academy of Medicine

Real-World Evidence to Guide the Approval and Use of New Treatments

By Steven Galson and Gregory Simon
October 18, 2016 | Discussion Paper

Research regarding new treatments (drugs, biological products, and high-risk devices) often begins with a broad assessment of disease epidemiology, disease burden, and shortcomings of existing treatments. That research may draw from diverse data sources, including real-world data generated by health system operations.

The clinical research phase of treatment development typically follows a well-established pathway from initial evaluation of safety to preliminary evaluation of therapeutic efficacy to pivotal trials intended to support regulatory approval for marketing. Those pivotal trials focus on key questions of efficacy (typically in comparison to placebo or some analogous control condition) and safety (especially serious or previously unrecognized adverse effects). This focus is consistent with the responsibility of the U.S. Food and Drug Administration (FDA) for assuring the safety and efficacy of drugs, biological products, and medical devices at the time of approval.

Regulatory approval is sometimes followed by systematic postmarketing evaluation to address a wider range of practical or real-world questions. This more pragmatic research again draws from more varied sources of data drawn from diverse clinical settings.



Suggested Citation

Galson, S. and G. Simon. 2016. Real-World Evidence to Guide the Approval and Use of New Treatments. NAM Perspectives. Discussion Paper, National Academy of Medicine, Washington, DC. doi: 10.31478/201610b


Disclaimer: The views expressed in this paper are those of the authors and not necessarily of the authors’ organizations, the National Academy of Medicine (NAM), or the National Academies of Sciences, Engineering, and Medicine (the National Academies). The paper is intended to help inform and stimulate discussion. It is not a report of the NAM or the National Academies. Copyright by the National Academy of Sciences. All rights reserved.