Interim analysis of continuous long-term endpoints in clinical trials with longitudinal outcomes

Statistics in Medicine
Sally Galbraith, I Marschner

Abstract

This paper discusses interim analysis for clinical trials where the primary endpoint is observed at a specific long-term follow-up time, but where repeated measures of the same outcome are also taken at earlier times. Methods are considered for improving the efficiency with which the long-term treatment difference is estimated, making use of information from shorter-term follow-up times. This approach to interim analysis has previously been studied for binary endpoints assessed at two time points during follow-up. Here we adapt and extend this methodology to include continuous endpoints assessed at an arbitrary number of follow-up times, making use of methods for analysing multivariate normal data subject to monotone missingness and unstructured mean and covariance relationships. The magnitude of efficiency gains obtained by using short-term measurements is considered, as well as how these gains depend on the number and timing of the short-term measurements. Sequential analysis of treatment differences is discussed, including the extent to which efficiency gains translate into reductions in the expected duration of a sequentially monitored trial. The methods are illustrated on a data set involving a placebo-controlled compariso...Continue Reading

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Citations

Nov 21, 2007·Journal of Biopharmaceutical Statistics·A P Grieve
Apr 26, 2011·Journal of Biopharmaceutical Statistics·Nigel Stallard
Jun 25, 2005·Statistics in Medicine·John M KittelsonScott S Emerson
Sep 16, 2006·Biometrical Journal. Biometrische Zeitschrift·Tim Friede, Meinhard Kieser
May 3, 2014·Pharmaceutical Statistics·Cornelia Ursula KunzNigel Stallard
Nov 26, 2016·Biometrical Journal. Biometrische Zeitschrift·Cornelia U KunzTim Friede
May 11, 2013·Pulmonary Circulation·Andy P GrieveMartin R Wilkins
Jan 3, 2019·Journal of Biopharmaceutical Statistics·Qing Shu LuSiu-Keung Tse
Jan 1, 2014·Therapeutic Innovation & Regulatory Science·Fei Chen, José Pinheiro
Oct 22, 2019·Statistics in Medicine·Kelly Van LanckerFilip De Ridder
Apr 5, 2021·Statistics in Medicine·Konstantinos PaterasKit C B Roes
Jul 1, 2021·Statistics in Biopharmaceutical Research·Cornelia Ursula KunzTim Friede

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