1. Design of Trials with Composite Endpoints with the R Package CompAREdesign
- Author
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Cortés Martínez, Jordi, Bofill Roig, Marta, Gómez Melis, Guadalupe, Universitat Politècnica de Catalunya. Departament d'Estadística i Investigació Operativa, and Universitat Politècnica de Catalunya. GRBIO - Grup de Recerca en Bioestadística i Bioinformàtica
- Subjects
Biometria ,FOS: Computer and information sciences ,Biometry ,Statistics ,Study design ,Estadística ,Pharmaceutical technology ,Statistics - Applications ,ARE ,Clinical trial ,Methodology (stat.ME) ,Matemàtiques i estadística::Estadística matemàtica [Àrees temàtiques de la UPC] ,Applications (stat.AP) ,Tecnologia farmacèutica ,Statistics - Methodology ,Composite endpoints - Abstract
Composite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional hazard assumption usually does not hold when using a composite endpoint, even when the premise remains true for their components. Consequently, the conventional formulae for sample size calculation do not longer apply. We present the R package CompAREdesign by means of which the key elements of trial designs, such as the sample size and effect sizes, can be computed based on the information on the composite endpoint components. CompAREdesign provides the functions to assess the sensitivity and robustness of design calculations to variations in initial values and assumptions. Furthermore, we describe other features of the package, such as functions for the design of trials with binary composite endpoints, and functions to simulate trials with composite endpoints under a wide range of scenarios. This work was supported by the Ministerio de Economía y Competitividad (Spain) under Grant PID2019- 104830RB-I00 and the Departament d’Empresa i Coneixement de la Generalitat de Catalunya (Spain) under Grant 2017 SGR 622 (GRBIO). Marta Bofill Roig is a member of the EU Patient Centric Clinical Trial Platforms (EU-PEARL). This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation program and EFPIA and Children’s Tumor Foundation, Global Alliance for TB Drug Development nonprofit organization, Springworks Therapeutics Inc. This publication reflects the authors’ views. Neither IMI nor the European Union, EFPIA, nor any Associated Partners are responsible for any use that may be made of the information contained herein.
- Published
- 2022
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