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Activity Number:
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155
- Research Reproducibility for Precision Medicine: From Controlled Experiments to Real-World Evidence
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Type:
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Topic Contributed
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Date/Time:
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Monday, July 29, 2019 : 10:30 AM to 12:20 PM
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Sponsor:
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Biopharmaceutical Section
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Abstract #305249
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Presentation
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Title:
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On Randomized Controlled Trials with Integrated Real World Evidence for Drug Development in Gene Therapy Trials
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Author(s):
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Qing Liu*
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Companies:
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Quantitative and Regulatory Medical Science, LLC
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Keywords:
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Real World Evidence;
Gene Therapy;
Rare Disease;
Bayesian Analysis;
Power;
Type 1 Error Rates
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Abstract:
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For gene therapy trials, we consider the setting where clinical development program consists of a prospective observation study real-world evidence (RWE) of patients receiving a standard of care (SOC), and a phase 3 randomized and blinded study to establish effectiveness and safety of a new drug compared with an integrated RWE of SOC. We propose an efficient inter-group (EIG) analysis approach where RWE from both the perspective observational study and the concurrent control of the parallel group design are combined via a Bayesian approach. The resulting design is 50%-65% more efficient than a traditional parallel group design. The analysis procedure controls the type 1 error rates and maintains high statistical power. This makes it feasible to conduct trials where it is difficult to enroll patients.
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Authors who are presenting talks have a * after their name.
