Abstract
In the previous chapter, the challenges and problems related to composite endpoints have been outlined. A number of guidelines exist discussing composite endpoints in various contexts such as in the context of clinical trial application for drugs or medicinal products or in the context of benefit-risk assessment. It is in the nature of things that the existing guidelines are revised and new ones are developed continuously. Therefore, it is not possible to give an exhaustive overview on the perspective of guidelines related to composite endpoints. Instead, in the following some relevant guideline documents will be highlighted, and major topics related to composite endpoints addressed in these guidelines will be outlined.
This is a preview of subscription content, log in via an institution to check access.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
CHMP. (2008a). Guideline on the evaluation of medicinal products for cardiovascular disease prevention. EMEA/CHMP/EWP/311890/2007. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003290.pdf. Accessed April 9, 2018.
CHMP. (2008b). Guideline on clinical investigation of medicinal products for the treatment of acute heart failure. CPMP/EWP/2986/03. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2015/06/WC500187797.pdf. Accessed April 9, 2018.
CHMP. (2016). Guideline on multiplicity issues in clinical trials. Draft. EMA/CHMP/44762/2017. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2017/03/WC500224998.pdf. Accessed April 9, 2018.
CPMP. (2002). Points to consider on multiplicity issues in clinical trials. CPMP/EWP/908/99. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003640.pdf. Accessed April 9, 2018.
The CAPRICORN Investigators (2001). Effect of Carvedilol on outcome after myocardial infarction in patients with left-ventricular dysfunction: The CAPRICORN randomised trial. The Lancet, 357, 1385–1390.
EUnetHTA. (2015). Endpoints used for relative effectiveness assessment: Composite endpoints. Adapted version (2015) based on endpoints used for relative effectiveness assessment of pharmaceuticals: Composite endpoints - February 2013. EUnetHTA. http://www.eunethta.eu/sites/default/files/sites/5026.fedimbo.belgium.be/files/Endpoints%20used%20for%20Relative%20Effectiveness%20Assessment%20Composite%20endpoints_Amended%20JA1%20Guideline_Final%20Nov%202015_0.pdf. Accessed April 9, 2018.
FDA. (2006). Guidance for industry - Clinical studies section of labeling for human prescription drug and biological products - Content and format. FDA. http://www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm127534.pdf. Accessed April 9, 2018.
FDA. (2007). Guidance for industry - Clinical trial endpoints for the approval of cancer drugs and biologics. FDA. http://www.fda.gov/downloads/Drugs/.../Guidances/ucm071590.pdf. Accessed April 9, 2018.
FDA. (2013). Guidance for industry - Design considerations for pivotal clinical investigations for medical devices. FDA. http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM373766.pdf. Accessed April 9, 2018.
FDA. (2017). Guidance for industry - Multiple endpoints in clinical trials. Draft. FDA. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM536750.pdf. Accessed April 9, 2018.
ICH. (1998). Statistical principles for clinical trials - E9. ICH. http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf. Accessed April 9, 2018.
IQWIG. (2015). General methods, version 4.2. IQWIG. https://www.iqwig.de/en/methods/methods-paper/general-methods-previous-versions.3021.html. Accessed April 9, 2018.
Sankoh, A. J., Li, H., & D’Agostino, R. B. (2017). Composite and multicomponent end points in clinical trials. Statistics in Medicine, 36, 4437–4440.
Snapinn, S. (2017). Some remaining challenges regarding multiple endpoints in clinical trials. Statistics in Medicine, 36, 4441–4445.
Author information
Authors and Affiliations
Rights and permissions
Copyright information
© 2017 Springer International Publishing AG, part of Springer Nature
About this chapter
Cite this chapter
Rauch, G., Schüler, S., Kieser, M. (2017). Guideline View: Recommendations and Requirements. In: Planning and Analyzing Clinical Trials with Composite Endpoints. Springer Series in Pharmaceutical Statistics. Springer, Cham. https://doi.org/10.1007/978-3-319-73770-6_3
Download citation
DOI: https://doi.org/10.1007/978-3-319-73770-6_3
Published:
Publisher Name: Springer, Cham
Print ISBN: 978-3-319-73769-0
Online ISBN: 978-3-319-73770-6
eBook Packages: Mathematics and StatisticsMathematics and Statistics (R0)