The contribution of real-world evidence to cost-effectiveness analysis: case study of Dabigatran etexilate in France

Objective The goal of this study was to assess the differences between an ex ante and an ex post cost-effectiveness analysis of Dabigatran etexilate vs VKAs for the prevention of thromboembolic events in non-valvular atrial fibrillation patients and to draw lessons on the design and use of real-world data for decision making. Methods The same model was used to calculate the cost-effectiveness ratio using two sets of parameters. One set included the efficacy and safety outcomes data from RE-LY, the pivotal trial comparing Dabigatran to warfarin; cost data came from an ex ante publication. Outcomes data for the second set came from real-world data studies. Cost data were a mix of real-world data and other sources. Two treatment strategies were compared: treatment initiation by either Dabigatran or VKAs, followed by either VKAs or Dabigatran. A crude comparison of results was performed; the impact of data differences was then assessed. Probabilistic sensitivity results of the two analyses were compared. Results With real-world evidence, Dabigatran at both dosages was more effective for the prevention of ischemic strokes, intra-cranial haemorrhages, with less major extra-cranial haemorrhages and a similar risk of myocardial infarction. Using clinical trial data, Dabigatran150 mg (resp. Dabigatran110 mg) as a first-line treatment vs VKAs yielded an ICER of € 8077/QALY (resp. € 13,116/QALY). Real-world evidence scenarios were cost-saving and more effective for both dosages. Conclusion The reassessment of outcomes and cost data had an impact on results, improving the efficiency of Dabigatran. We identify methodological issues which should be discussed if post-launch RWE based cost-effectiveness data become a standard in HTA decision making.