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Activity Number:
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304
- Extrapolation as a Default Strategy in Pediatric Drug Development: Implications to Clinical Trial Designs and Statistical Analysis
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Type:
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Topic Contributed
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Date/Time:
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Wednesday, August 5, 2020 : 10:00 AM to 11:50 AM
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Sponsor:
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Biopharmaceutical Section
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Abstract #312866
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Title:
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A Quantitative Method to Evaluate Exposure-Response Similarity Between Pediatrics and Adults to Support Efficacy Extrapolation
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Author(s):
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Junshan Qiu and Margaret Gamalo-Siebers and Freda Cooner and Junjing Lin and Yodit Seifu and Yaning Wang*
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Companies:
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FDA/CDER and Eli Lilly & Co and Amgen Inc. and Takeda and and FDA/CDER/DPM
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Keywords:
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Exposure-Response;
Similarity;
Pediatrics;
Non-parametric
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Abstract:
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US FDA has allowed extrapolation of efficacy from adults and an abbreviated pediatric development program where there is an expectation of a similar exposure-response (E-R) relationship in addition to a similar disease progression and treatment response in children, when compared to adults. Historically, the assessment of E-R similarity was based on visual inspection of two E-R curves. This talk will discuss a more quantitative method to compare exposure-response relationships between pediatric and adult populations to inform decision making in pediatric drug development. This method clarified that non-inferiority instead of similarity is required to extrapolate adult efficacy to pediatric patients. It also provided reliable objective criteria for non-inferiority assessment and decision making for efficacy extrapolation in pediatric population.
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Authors who are presenting talks have a * after their name.
