Using real world data for pharmacoeconomic assessments - a case study

Abstract

Pembrolizumab monotherapy for non-small cell lung cancer (NSCLC) was deemed not cost-effective due to limited data, high uncertainty regarding long term results and a considerable budget impact. However, due to confidential price agreements it was approved in 2017, as first-line treatment for metastatic NSCLC that expresses PD-L1 ≥ 50 %. Given potential discrepancies between clinical efficacy and real-world effectiveness due to differing treatment guidelines and inclusion criteria, it’s uncertain whether the treatment is truly cost-effective, especially since projections are based on immature data. Our objectives are to (1) re-evaluate the economic feasibility of pembrolizumab in treating lung cancer within the Norwegian healthcare framework with real world data; (2) to identify the possible variables contributing to the gap between its efficacy in controlled studies and its effectiveness in a clinical setting and (3) to provide a framework for assessing the cost-effectiveness of treatments with immature data.