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

  • Welsing PM Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, The Netherlands. Electronic address: p.m.j.welsing@umcutrecht.nl.
  • Oude Rengerink K Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, The Netherlands.
  • Collier S GSK, Respiratory R&D, Stockley Park West, Brentford, Middlesex TW89GS, UK.
  • Eckert L Health Economics and Outcome Research, Sanofi, Avenue Pierre Brossolette, 91385, Chilly-Mazarin, France.
  • van Smeden M Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, The Netherlands.
  • Ciaglia A International Alliance of Patients' Organizations, 49-51 East Road, London, N1 6AH, UK.
  • Nachbaur G Medical Department, GSK, 100 route de Versailles, Marly-le-Roi 78163, France.
  • Trelle S Department of Clinical Research, CTU, University of Bern, Finkenhubelweg 11, CH-3012, Bern, Switzerland.
  • Taylor AJ Global Outcomes Research, Takeda Development Centre Europe Ltd, 61 Aldwych, WC2B 4AE, London, UK.
  • Egger M Institute of Social and Preventive Medicine, University of Bern, Finkenhubelweg 11, CH-3012, Bern, Switzerland.
  • Goetz I Global Patient Outcomes & Real World Evidence, Eli Lilly and Company Ltd, Erl Wood Manor, Sunninghill Road, Windlesham, Surrey, GU20 6PH, UK.
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  • 2017-05-16
Published in:
  • Journal of clinical epidemiology. - 2017
Generalizability
Outcome measurement
Pragmatic trial
Real-world evidence
Decision Making
Endpoint Determination
Evidence-Based Medicine
Humans
Outcome Assessment, Health Care
Pragmatic Clinical Trials as Topic
Randomized Controlled Trials as Topic
Research Design
English 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.
Language
  • English
Open access status
hybrid
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Persistent URL
https://sonar.ch/global/documents/269585
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