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Activity Number: 540 - SPEED: Clinical Trial Design, Longitudinal Analysis, and Other Topics in Biopharmaceutical Statistics
Type: Contributed
Date/Time: Wednesday, August 1, 2018 : 11:35 AM to 12:20 PM
Sponsor: Biopharmaceutical Section
Abstract #332897
Title: Statistical Considerations of Single Pivotal Vs Two Replicated Confirmatory Studies
Author(s): Zijiang Yang*
Companies: Janssen R&D
Keywords: clinical trials; pivotal study; statistically persuasive; simulation

Adequate and well controlled studies are the basis for a successful NDA. The FDA usually requires two independent confirmatory studies, each with a significant p-value (< 0.05) as one of the requirements for registration. Though not very common, the concept of "Statistically Persuasive" has been introduced to provide an alternative path to approval with only one pivotal trial that requires the "same amount of evidence" as two replicated studies. Achieving the higher bar of p< 0.00125 is considered as statistically equivalent to two independent studies with a threshold of p< 0.05. This presentation explores the similarities and differences between these two options. We will also discuss the feasibility of conducting two confirmatory studies simultaneously and analyzing the aggregated study data for statistically persuasive results through simulation.

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

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