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Activity Number:
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124
- Special Topics in Clinical Trial Design and Analysis
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Type:
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Contributed
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Date/Time:
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Monday, July 31, 2017 : 8:30 AM to 10:20 AM
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Sponsor:
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Biopharmaceutical Section
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Abstract #324554
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View Presentation
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Title:
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Phase II Trials and the Use of Registry Controls
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Author(s):
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Ruta Brazauskas* and Brent Logan and Raphael Fraser
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Companies:
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Institute for Health & Society-Medical College of Wisconsin and Institute for Health & Society-Medical College of Wisconsin and Institute for Health & Society-Medical College of Wisconsin
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Keywords:
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Phase II trials ;
Randomized clinical trials ;
Single arm trials ;
Observational database ;
Data registry
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Abstract:
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Randomized trials are recognized as the best way to evaluate treatment effect on the outcome of interest. We consider hematopoietic cell transplantation (HCT) setting where randomized phase II trials are difficult to conduct due to the rarity of patients available for study, and there is an additional option of comparing single arm phase II trial data to a registry control population. Center for International Blood and Marrow Research collects extensive data on patient risk factors and outcomes. This observational database can serve as a source of controls for a single arm phase II trial. Using a matched registry control group or using covariate adjustment does not guarantee an unbiased comparison but it may be more efficient (lower variability) and cost effective than randomized phase II trials in terms of requiring fewer patients on trial to detect the same treatment effect. We explore the trade offs in bias/efficiency between randomized phase II trials and single arm trials benchmarked against a registry control group by simulating trials of each type from real populations defined by existing completed randomized HCT clinical trials as well as available registry data.
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Authors who are presenting talks have a * after their name.
