The randomized controlled trial (RCT) is the gold standard to measure the efficacy of a drug or an intervention. However, outcomes of treatment in clinical practice can differ from outcomes in RCTs with regard to both the estimated effectiveness and the estimated resource utilization.
In a randomized clinical trial (COU-AA-301), abiraterone acetate (Zytiga) was shown to be superior to prednisone in the treatment of metastatic castration-resistant prostate cancer (mCRPC). In order to analyse the external validity of the postchemotherapy clinical trial from a Swedish perspective, the outcomes and treatment patterns of abiraterone treatment in a Swedish observational study was compared to those of the pivotal clinical trial.
A comparison indicated that the Swedish patients were similar to patients included in the clinical trial in observed patient characteristics. Despite this, the Swedish patients could expect the same overall survival as patients randomized to abiraterone in the clinical trial despite shorter treatment duration, leading to a more cost-effective use of the treatment in real world compared to the clinical trial.
Scandinavian Journal of Urology, 2016; 50(4): 286-91