Estimand Framework in Oncology Drug Development – Impact and Opportunities

• 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
• Author(s): Evgeny Degtyarev* and Kaspar Rufibach and Jonathan Siegel and Viktoriya Stalbovskaya and Steven Sun
• Companies: Novartis and F. Hoffmann-La Roche and Bayer HealthCare Pharmaceuticals Inc. and Merus and J&J
• Keywords: Estimand ; ICH E9 Addendum; Time to event data; EFSPI SIG Estimands in Oncology

Abstract

A draft addendum of the ICH E9 guideline on Statistical Principles for Clinical Trials released in August 2017 introduced an estimand framework. The new framework aims at aligning trial objectives and analyses by requiring a precise definition of the treatment effect (estimand), in particular explicitly accounting for intercurrent events (i.e. events which occur after baseline but before observing the endpoint, e.g. start of another therapy). Motivational examples will be presented introducing the addendum and highlighting its impact on drug development and potential opportunities. The choice of estimands will drive the trial design and sample size, data collection, trial conduct, and analysis. The framework will support discussions among sponsors, regulators, payers, physicians, and patients about relevant treatment effects, interpretation of study results, and added value of drugs. Statisticians will play a key role in these discussions with different stakeholders about the choice of estimands. This presentation is a result of a cross-industry working group with the goal to ensure common understanding and consistent definitions for key time-to-event estimands in Oncology.