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Activity Number: 511 - Statistical Considerations for Rare Disease Clinical Development: Opportunities and Challenges
Type: Topic Contributed
Date/Time: Wednesday, August 1, 2018 : 10:30 AM to 12:00 PM
Sponsor: Biopharmaceutical Section
Abstract #330352
Title: On Randomized Controlled Trials with Integrated Real World Evidence for Drug Development in Rare Diseases
Author(s): Qing Liu*
Companies: Amicus Therapeutics, Inc
Keywords: Real World Evidence; Two-Stage Adaptive Design; Robust Test; Rare Disease; Integrated Analysis of Efficacy; Sample Size Calculation

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.

Authors who are presenting talks have a * after their name.

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