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Activity Number:
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307
- Clinical Trial Design-3
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Type:
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Contributed
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Date/Time:
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Tuesday, July 31, 2018 : 8:30 AM to 10:20 AM
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Sponsor:
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Biopharmaceutical Section
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Abstract #329858
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Presentation
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Title:
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Incorporating Historical Information into the Analysis of Clinical Trials- a Case Study
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Author(s):
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Guochen Song* and Yiqing Tian and John Zhong and Stacy Lindborg
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Companies:
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Biogen and Q2 Sulotions and Biogen and Biogen Idec
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Keywords:
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Bayesian borrowing;
power prior;
commensurate prior;
hierarchical model;
hazard ratio;
spinal muscular atrophy
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Abstract:
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Nusinersen was first approved by FDA to treat spinal muscular atrophy (SMA) based on the positive result of a randomized, double blinded phase III study in the infantile onset population. At the time of the phase III study, data from the phase II study and data from a natural history study were also available. Borrowing from the existing information to enhance the analysis of the phase III study will improve the efficiency of using data. The Bayesian hierarchical borrowing method was planned on a piece-wise exponential model to integrate the observational and phase II data into the pivotal trial data analysis. As an exploratory analysis, the power prior method and commensurate prior method with different specifications were also explored. This presentation will demonstrate how the different borrowing techniques narrowing the creditable interval of the hazard ratio. The operating characteristics of different methods in the Frequentist sense will be also be compared.
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Authors who are presenting talks have a * after their name.
