Off-label use of the expensive orphan drug eculizumab in France 2009-2013 and the impact of literature: focus on the transplantation field

摘要

The orphan drug eculizumab (Soliris A (R)) is one of the most expensive in the world and based on expenditures is classed among the highest in France, a scenario suggestive of off-label use. Given its pharmacological properties, it is likely to be used in organ transplantation. Our purposes were to describe the consumption trends of eculizumab for off-label indications overall and in the organ transplantation field and to assess the impact of publications on the latter use.