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Activity Number: 228 - Innovative Statistical Designs with Real Life Case Studies for New Paradigms in Oncology Drug Development
Type: Topic Contributed
Date/Time: Monday, July 30, 2018 : 2:00 PM to 3:50 PM
Sponsor: Biopharmaceutical Section
Abstract #328933 Presentation
Title: A Generalized Design for a Confirmatory Basket Trial
Author(s): Robert Beckman and Xiaoyun (Nicole) Li*
Companies: Georgetown University and Merck
Keywords: basket trials; histology independent indications; rare diseases; cancer; biomarkers; confirmatory development

Increasingly, tumors are defined on a molecular basis rather than only on histology, and targeted agents which address these molecular subtypes are being approved. This profusion of molecular subtypes creates "rare" diseases as subsets of common cancers, leading to difficulties in enrolling sufficiently large cohorts for confirmatory trials. However, if the molecular subtype is shared across various histologies, these may be pooled into a basket trial. To date, basket trials have been primarily for exploratory early development. In this presentation, we consider qualitative designs for confirmatory basket trials. These confirmatory basket designs will provide patients in niche indications with enhanced access to novel therapies, facilitate development and full approval for niche indications, allow accelerated approval for indications within a basket based on a surrogate endpoint, reduce development cost by combining trials into one, and enhance the ability of regulatory authorities to evaluate risk and benefit in niche indications.

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

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