just read an interesting article: Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?
The authors propose an alternative methodology based on multiple criteria such as rarity, manufacturing complexity, availability of alternatives, disease severity, research undertaken and several others, given that most orphan drugs due to the higher price ranges hardly will meet the traditional cost-effectiveness thresholds imposed by various HTA agencies. Indeed the various countries are having different systems in order to evaluate orphan drugs, for example IQWIG in Germany sees the additional medical benefit as a given for orphan drugs while other authorities have different requirements regarding the need and scope of economic evaluations.
I think this paper gives some very welcome alternatives as to what assessment criteria to be used. In my view it should ultimately be left with every EU member state, similar to what the authors proposed, to decide which is the best option for their respective healthcare system and assigning the monetary value they would like to place onto an evaluation rather than centralized purchasing procedures as once suggested. Orphan drugs, due to their limited and very specific patient population, are certainly best suited for innovative access agreements linked to real world evidence development in case of uncertainty at launch.