Outcome selection and measurement in pragmatic trials.

Welsing, Paco M; Oude Rengerink, Katrien; Collier, Sue; Eckert, Laurent; van Smeden, Maarten; Ciaglia, Antonio; Nachbaur, Gaelle; Trelle, Sven; Taylor, Aliki J; Egger, Matthias; Goetz, Iris; Consortium, Work Package 3 of the GetReal (2017). Outcome selection and measurement in pragmatic trials. Journal of clinical epidemiology, 90, pp. 99-107. Elsevier 10.1016/j.jclinepi.2016.12.022

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Results from pragmatic trials should reflect the comparative treatment effects encountered in patients in real-life clinical practice to guide treatment decisions. Therefore, pragmatic trials should focus on outcomes that are relevant to patients, clinical practice, and treatment choices. This sixth article in the series (see box) discusses different types of outcomes and their suitability for pragmatic trials, design choices for measuring these outcomes, and their implications and challenges. Measuring outcomes in pragmatic trials should not interfere with real-world clinical practice to ensure generalizability of trial results, and routinely collected outcomes should be prioritized. Typical outcomes include mortality, morbidity, functional status, well-being, and resource use. Surrogate endpoints are typically avoided as primary outcome. It is important to measure outcomes over a relevant time horizon and obtain valid and precise results. As pragmatic trials are often open label, a less subjective outcome can reduce bias. Methods that decrease bias or enhance precision of the results, such as standardization and blinding of outcome assessment, should be considered when a high risk of bias or high variability is expected. The selection of outcomes in pragmatic trials should be relevant for decision making and feasible in terms of executing the trial in the context of interest. Therefore, this should be discussed with all stakeholders as early as feasible to ensure the relevance of study results for decision making in clinical practice and the ability to perform the study.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Pre-clinic Human Medicine > Institute of Social and Preventive Medicine
04 Faculty of Medicine > Pre-clinic Human Medicine > CTU Bern

UniBE Contributor:

Trelle, Sven and Egger, Matthias

Subjects:

600 Technology > 610 Medicine & health
300 Social sciences, sociology & anthropology > 360 Social problems & social services

ISSN:

0895-4356

Publisher:

Elsevier

Language:

English

Submitter:

Beatrice Minder Wyssmann

Date Deposited:

04 Jul 2017 12:35

Last Modified:

22 Oct 2019 17:25

Publisher DOI:

10.1016/j.jclinepi.2016.12.022

PubMed ID:

28502810

Uncontrolled Keywords:

Generalizability Outcome measurement Pragmatic trial Real-world evidence

BORIS DOI:

10.7892/boris.101683

URI:

https://boris.unibe.ch/id/eprint/101683

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