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Activity Number:
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511
- Statistical Considerations for Rare Disease Clinical Development: Opportunities and Challenges
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Type:
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Topic Contributed
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Date/Time:
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Wednesday, August 1, 2018 : 10:30 AM to 12:00 PM
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Sponsor:
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Biopharmaceutical Section
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Abstract #330352
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Title:
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On Randomized Controlled Trials with Integrated Real World Evidence for Drug Development in Rare Diseases
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Author(s):
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Qing Liu*
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Companies:
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Amicus Therapeutics, Inc
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Keywords:
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Real World Evidence;
Two-Stage Adaptive Design;
Robust Test;
Rare Disease;
Integrated Analysis of Efficacy;
Sample Size Calculation
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Abstract:
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Consider clinical development program consisting 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. It was recently stated by the FDA that ``the clinical trial system we have right now is broken'', and a RWE approach for establishing safety and effectiveness for drug development is advocated. We propose the use of a blinded randomized control trial (RCT) with an integrated RWE that incorporates both intra-subject and inter-group comparisons to establish the effectiveness and safety of a new drug for patients with rare diseases. We develop an integrated analysis of efficacy to combines both inter-group and intra-patient comparisons of a new treatment to RWE SOC. The required sample size for the proposed design is nearly half of that of the parallel group design; and thus, making an adequate and well-controlled phase 3 trial for rare disease drug development feasible.
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Authors who are presenting talks have a * after their name.
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