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Activity Number: 164 - FDA Adaptive Designs and Master Protocols Guidance for Clinical Trials – Reflection and Outlook
Type: Topic Contributed
Date/Time: Monday, July 29, 2019 : 10:30 AM to 12:20 PM
Sponsor: Biopharmaceutical Section
Abstract #305272 Presentation
Title: Points to Consider in the Design of Adaptive Platform Clinical Trials in Non-Alcoholic Steatohepatitis
Author(s): Peter Mesenbrink*
Companies: Novartis Pharmaceuticals
Keywords: Adaptive Design; Interim Analysis; Type I error; Bayesian Decision Rules; Enrichment

The need for platform designs is becoming important outside of oncology in disease areas like non-alcoholic steatohepatitis (NASH)where the competition for patients is high and combination therapy will be the preferred treatment strategy in the future due to multifactorial nature of the disease. Currently there are no approved therapies for NASH and there are no validated biomarkers of disease diagnosis or disease progression. In order to find the best combination therapy to treat NASH, an adaptive platform study design would increase efficiency and allow faster decisions to be made on effective and ineffective treatments through decision rules implemented at pre-defined interim analyses throughout the cohorts of the design. We will discuss how and when interim analyses should be performed to enrich the study design and to make decisions on the effectiveness of a specific treatment combination. We will also discuss the advantages and disadvantages of a shared control group across such a design and how treatment arms that appear in multiple cohorts should be handled and how to implement a Bayesian framework to control the Type 1 error rate.

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

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