Orphan drugs’ market access challenges in Europe from a German perspective

Nowadays, market access of pharmaceuticals requires an health technology assessment (HTA) in most European countries. Particularly orphan drugs (ODs) are assigned a special role in most countries. The German AMNOG process belongs to the most straightforward and transparent HTAs in Europe. These procedure with subsequent price negotiations involving drugs with orphan drug designation (ODD) were evaluated and compared with results at European level to figure out success and failure strategies in Europe, especially in Germany. Challenges for the European market access of ODs were analyzed, mainly based on learnings from AMNOG procedures supported by results from the rest of Europe, especially HAS and NICE, also reflecting the inclusion of International Reference Pricing (IRP) in the price-determination mechanisms in the different countries.