Real-World Effectiveness & Utilisation Patterns

Studies of effectiveness and utilisation employ real-world data from cohort or case-control designs, to estimate relative effectiveness, prescribing patterns, adherence, persistence, and safety outcomes. These estimates can be used to characterise country-specific treatment pathways, drug utilisation, and resource use; to help estimate direct and indirect costs of health conditions; and, to frame the potential impacts of new treatments. In situations where randomised control trials are unsuitable, real world effectiveness and utilisation patterns studies can aid in bridging the gap between clinical trial results and clinical practice and its outcomes.  


To assess real-world effectiveness and utilisation, team members at ICON may use any of the following methods, tailored to the research question at hand:

  • Administrative database analysis
  • Chart reviews
  • Prospective data collection and registries 
  • Modelling

These studies are undertaken to support the reimbursement processes, measure the impact of competitor products and inform ongoing pharmacoeconomic analyses after a product’s efficacy has been demonstrated.  

Effectiveness and utilisation studies are useful for predicting the potential impact of new therapeutics or policies on disease burden. Alternatively they can be used to monitor changes after the introduction of new therapies, in the post-marketing phase, using real-world effectiveness and safety data.