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To what extent is response-adaptive randomization used in clinical trials? A systematic review using Cortellis
Oleksandr Sverdlov1, Jone Renteria2, Kerstine Carter3
1Advanced Quantitative Sciences, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Response-adaptive randomization (RAR) is increasingly used in adaptive clinical trials. This systematic review details RAR trial characteristics, applications, and positive outcomes, supporting its value in optimizing trial design.
Area of Science:
- Clinical Trials Methodology
- Biostatistics
- Drug Development
Background:
- Response-adaptive randomization (RAR) shows increasing adoption in clinical trials.
- A comprehensive review of RAR trial context, characteristics, and acceptance was previously unavailable.
- This study addresses the need for systematic evaluation of RAR implementation.
Purpose of the Study:
- To systematically review clinical trials employing response-adaptive randomization.
- To characterize the use, context, and outcomes of RAR trials.
- To establish a baseline for understanding the evolving application of RAR.
Main Methods:
- Systematic review of clinical trials utilizing RAR elements.
- Data sourced from the Cortellis Regulatory Intelligence database.
- Pre-specified selection process and summary of trial characteristics.
Main Results:
- Identified 39 RAR trials (22 completed, 17 ongoing) from 170 records.
- Majority were Phase 2 studies in oncology, neurology, and infectious diseases, using small molecules and biologics.
- Seven completed trials reported positive outcomes, with two providing pivotal data for regulatory approval.
Conclusions:
- Response-adaptive randomization is increasingly utilized in complex adaptive trials across various therapeutic areas and phases.
- This systematic review offers a baseline for tracking RAR dynamics.
- Highlights RAR as a valuable methodology for optimizing future clinical trial designs.
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