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A Benchmark, Expand, and Calibration (BenchExCal) Trial Emulation Approach for Using Real-World Evidence to Support
Shirley V Wang1, Massimiliano Russo1,2, Robert J Glynn1
1Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Clinical Pharmacology and Therapeutics
|March 11, 2025
Summary
This study introduces a novel BenchExCal approach using real-world evidence from healthcare databases to enhance drug effectiveness evaluations and support new drug indications.
Area of Science:
- Pharmacovigilance and Drug Effectiveness Research
- Real-World Evidence (RWE) in Regulatory Science
- Comparative Effectiveness Research
Background:
- Real-world evidence (RWE) from healthcare databases is crucial for post-market drug safety and effectiveness evaluations.
- Advancements in methods and infrastructure support RWE use for effectiveness claims, including expanding drug indications.
- Current methods for using database studies to support indication expansions require enhanced confidence in validity.
Purpose of the Study:
- To propose and evaluate a structured benchmarking and calibration approach (BenchExCal) for trial emulation using RWE.
- To increase confidence in findings from database studies supporting drug indication expansions.
- To provide insights for interpreting RWE in the context of hypothetical trials for new indications.
Main Methods:
- The study proposes the Benchmark, Expand, and Calibration (BenchExCal) approach for trial emulation.
- The approach involves benchmarking an initial database study against existing Randomized Controlled Trial (RCT) evidence.
- Subsequent database studies are calibrated based on observed differences between the initial RCT and database study results.
Main Results:
- The BenchExCal approach aims to increase confidence in RWE for supporting expanded drug indications.
- Simulation studies and five planned empirical examples will evaluate the performance of the BenchExCal approach.
- The project will offer insights into using first-stage benchmarking to calibrate second-stage emulations for new indications.
Conclusions:
- The BenchExCal approach offers a structured method to enhance the reliability of RWE for regulatory decision-making.
- This methodology can improve the interpretation of database study findings for drug indication expansions.
- Further research is needed to address all clinical and data scenarios encountered in supplemental indication seeking.
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