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Historical Controls in Randomized Clinical Trials: Opportunities and Challenges
Kathryn T Hall1,2, Lene Vase3, Deirdre K Tobias1,2
1Division of Preventive Medicine, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts, USA.
Clinical Pharmacology and Therapeutics
|July 1, 2020
Summary
Leveraging historical placebo data from clinical trials can accelerate drug discovery and reduce patient burden. Challenges in data heterogeneity must be addressed for broader adoption of historical controls in pharmaceutical research.
Area of Science:
- Pharmaceutical research
- Clinical trial design
- Drug discovery
Background:
- Randomized controlled trials (RCTs) with placebo are the standard for evaluating new drugs.
- Vast amounts of underutilized data exist in the placebo arms of past RCTs.
- Historical placebo data are increasingly used as synthetic controls, especially in rare diseases.
Purpose of the Study:
- To examine the challenges and opportunities of using historical placebo data as controls in RCTs.
- To propose a framework for the broader utilization of historical control data in drug development.
Main Methods:
- Review of existing literature on historical control use.
- Analysis of challenges including data heterogeneity, outcome ascertainment, and patient characteristics.
- Discussion of current methodologies and their limitations.
Main Results:
- Significant untapped potential exists in historical placebo data for guiding trial design and analysis.
- Heterogeneity in trial design, outcome measurement, and patient populations presents key challenges.
- Unmeasured pharmacogenomic effects can also impact the utility of historical controls.
Conclusions:
- Broader use of historical placebo data can benefit drug developers and patients by accelerating research.
- Addressing data heterogeneity and standardizing data collection are crucial for effective implementation.
- A strategic approach is needed to overcome current limitations and facilitate wider adoption.
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