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Activity Number:
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208
- Substantial Evidence Using Bayesian Statistics in New Drug Approvals
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Type:
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Invited
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Date/Time:
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Monday, July 31, 2017 : 2:00 PM to 3:50 PM
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Sponsor:
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Section on Bayesian Statistical Science
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Abstract #322141
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Title:
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Predictive Evidence Threshold Scaling: Does the Evidence Meet a Confirmatory Standard?
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Author(s):
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Satrajit Roychoudhury* and Beat Neuenschwander and Michael Branson
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Companies:
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Pfizer Inc and Novartis Pharma AG and Novartis Pharma AG
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Keywords:
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breakthrough designation ;
extrapolation ;
evidence synthesis ;
heterogeneity ;
prediction ;
real-world evidence
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Abstract:
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Making better use of all available evidence is a trend in modern drug development. However, it is important to measure the strength of available information for correct decision making. This calls for an understanding of the evidential strength of non-confirmatory evidence relative to a confirmatory standard. Such comparisons can be done via predictive evidence threshold scaling (PETS). Under PETS, the evidence meets a confirmatory standard if the predictive probability of a positive effect reaches the predictive evidence threshold from hypothetical confirmatory data. These probabilities require plausible assumptions about between-trial heterogeneity and potential biases. Two examples are discussed. The first is breakthrough designation, illustrated by a recent FDA approval of Crizotinib for the treatment of non-small-cell-lung-cancer based on phase I and II data. The second is childhood Guillain-Barre syndrome, with sparse pediatric data enriched with adult data.
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Authors who are presenting talks have a * after their name.
