Efficacy versus effectiveness trials: informing guidelines for asthma management

David Price* (Corresponding Author), Elizabeth V. Hillyer, Thys van der Molen

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

32 Citations (Scopus)


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.

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.
Original languageEnglish
Pages (from-to)50-57
Number of pages58
JournalCurrent Opinion in Allergy and Clinical Immunology
Issue number1
Publication statusPublished - Feb 2013


  • asthma
  • effectiveness
  • efficacy
  • pragmatic
  • real life


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