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Activity Number: 496 - Estimand Framework and Its Impact on Drug Development in Oncology
Type: Topic Contributed
Date/Time: Wednesday, July 31, 2019 : 10:30 AM to 12:20 PM
Sponsor: Lifetime Data Science Section
Abstract #304521 Presentation
Title: Estimand Framework – Are We Asking the Right Questions? a Case Study in the Solid Tumor Setting
Author(s): Michelle Casey * and Evgeny Degtyarev and Maria Jose Lechuga and Paola Aimone and Feng Feng Liu and Viktoriya Stalbovskaya and Rui Tang and Emily Butler and Oliver Sailer
Companies: Pfizer and Novartis and Pfizer, Inc. and Novartis Pharma AG and AstraZeneca and Merus and Servier and GlaxoSmithKline and Boehringer Ingelheim Pharma GmbH & Co
Keywords: Estimand; Solid Tumors; Intercurrent events; Time to event data; EFSPI SIG Estimands in Oncology

The estimand framework requires a precise definition of the treatment effect (“estimand”) as different ways of accounting for “intercurrent” events post randomization may result in different scientific questions.

The initiation of alternative therapy is common in oncology clinical trials. Two possible ways to account for this intercurrent event are to censor at initiation or to consider the start of therapy as an event. The new framework clarifies that these address different questions (“does the drug delay relapse if no patient had received new therapy” vs “does the drug delay relapse or start of new therapy”). The estimand framework facilitates discussions during clinical trial design to ensure alignment between the key question of interest, the analysis, and interpretation.

This presentation is a result of a cross-industry collaboration to connect the ICH E9 addendum concepts to applications. Data from previously reported randomized phase 3 studies will be used to consider common intercurrent events in solid tumor studies, to illustrate different scientific questions and the consequences of the estimand choice for study design, data collection, analysis, and interpretation.

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

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