title: Expanding the Evidence Base: Comparing Randomized Controlled Trials and Observational Studies of Statins.
abstract: It is widely accepted that randomized controlled trials (RCTs) are the gold standard for demonstrating the efficacy of a given therapy (results under ideal conditions). Observational studies, on the other hand, can complement this by demonstrating effectiveness (results under real-world conditions). To examine the role that observational studies can play in complementing data from RCTs, we reviewed published studies for statins, a class of drugs that have been widely used to reduce the risk of cardiovascular (CV) events by lowering low-density lipoprotein cholesterol levels. RCTs have consistently demonstrated the benefits of statin treatment in terms of CV risk reduction and have demonstrated that more intensive statin therapy has incremental benefits over less intensive treatment. Observational studies of statin use in 'real-world' populations have served to augment the evidence base generated from statin RCTs in preselected populations of patients who are often at high CV risk and have led to similar safety and efficacy findings. They have also raised questions about factors affecting medication adherence, under-treatment, switching between statins, and failure to reach low-density lipoprotein cholesterol target levels, questions for which the answers could lead to improved patient care.
authors.affiliation: 1Department of Cardiology, Division of Medicine B, Oslo University Hospital, Oslo, Norway; 2Pfizer Ltd, Walton Oaks, Surrey, United Kingdom; and 3Department of Vascular Medicine, Durrer Center for Cardiogenetic Research, Academic Medical Center, Amsterdam, The Netherlands.
authors.forename: Dan
authors.initials: D
authors.lastname: Atar
authors.forename: Seleen
authors.initials: S
authors.lastname: Ong
authors.forename: Peter J
authors.initials: PJ
authors.lastname: Lansberg
mesh_terms: D002318:Cardiovascular Diseases / Q000517:prevention & control*; D008078:Cholesterol, LDL / Q000097:blood; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000008:administration & dosage*; D055118:Medication Adherence; D064887:Observational Studies as Topic / Q000379:methods*; D017410:Practice Guidelines as Topic; D016032:Randomized Controlled Trials as Topic / Q000379:methods*
chemical_list: D008078:Cholesterol, LDL; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors