Series: Pragmatic trials and real world evidence: Paper 6. Outcome measures in the real world

Journal of Clinical Epidemiology
Paco M WelsingWork Package 3 of the GetReal consortium

Abstract

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 ...Continue Reading

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Citations

Jun 11, 2020·Journal of General Internal Medicine·Elena ByhoffUNKNOWN Society of General Internal Medicine
Apr 24, 2020·Journal of Clinical Epidemiology·Rafael Dal-RéStefan K James

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