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Efficacy versus effectiveness trials: informing guidelines for asthma management

Price, David; Hillyer, Elizabeth V.; van der Molen, Thys

Current Opinion in Allergy & Clinical Immunology: February 2013 - Volume 13 - Issue 1 - p 50–57
doi: 10.1097/ACI.0b013e32835ad059
MECHANISMS OF ALLERGY AND ADULT ASTHMA: Edited by Stephen T. Holgate and J. Andrew Grant

Purpose of review: Randomized controlled trials, known as efficacy trials and long considered the gold standard for evidence-based asthma guidelines, are designed to test whether interventions have a benefit for selective patient populations under ideal conditions. The goal of pragmatic trials and observational studies instead is to understand real-life efficacy, known as effectiveness. This review summarizes the strengths and limitations of efficacy and effectiveness trials, results of recent effectiveness trials in asthma and initiatives promoting effectiveness research.

Recent findings: Recent pragmatic trials and observational studies have examined outcomes of interventions for diverse real-life patient populations, including smokers and patients with variable adherence, inhaler technique and baseline asthma control. Study results challenge practice guidelines regarding relative effectiveness of leukotriene receptor antagonists and inhaled corticosteroids (ICS); supplement guidelines with regard to effectiveness of interventions in smokers; and begin to address gaps in guidelines regarding choice of ICS and inhaler device. Initiatives are ongoing to refine methods of observational research and to harmonize asthma outcomes for better integration of results from all types of trials.

Summary: Results of pragmatic trials and observational studies are an important component of the evidence needed to inform guideline recommendations and decision-making by healthcare providers, patients and policymakers.

aAcademic Primary Care, Division of Applied Health Sciences, University of Aberdeen, Aberdeen

bResearch in Real Life Ltd, Cambridge, UK

cDepartment of General Practice, Groningen Research Institute for Asthma and COPD, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

Correspondence to David Price, Academic Primary Care, Division of Applied Health Sciences, University of Aberdeen, Polwarth Building, Foresterhill, Aberdeen AB25 2ZD, UK. Tel: +44 1224 554588; fax: +44 1224 550683; e-mail: david@rirl.org

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