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Augmenting randomized clinical trial data with historical control data: Precision medicine applications.

Boris Freidlin¹, Edward L Korn¹

¹Biometric Research Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, MD, USA.

Journal of the National Cancer Institute

|September 26, 2022

Summary

Augmenting small clinical trials with historical data is challenging in precision medicine. Strategies for using historical controls are unlikely to be effective for small, targeted patient groups.

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Area of Science:

Clinical Trials
Precision Medicine
Biostatistics

Background:

Precision medicine yields smaller patient subpopulations, complicating randomized clinical trials (RCTs).
Augmenting small RCTs with historical control data is a proposed solution for timely trials.
Existing strategies aim to mitigate bias when incorporating external control data.

Purpose of the Study:

To evaluate the utility of proposed strategies for using historical control data in precision medicine.
To demonstrate the limitations of these strategies with simple examples and case studies.

Main Methods:

Analysis of strategies for augmenting RCT control arms with historical data.
Illustrative examples and review of completed studies.

Main Results:

The evaluated strategies are unlikely to be effective for precision medicine applications.
Challenges remain in leveraging historical data for small, targeted patient populations.

Conclusions:

Current methods for incorporating historical controls are not well-suited for the unique challenges of precision medicine.
Further research is needed to develop robust methods for small-sample clinical trials in targeted therapies.