OBJECTIVES: Nowadays, market access of pharmaceuticals requires an 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 procedures 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.

METHODS: To identify strategies of success and failure, published documents of the early benefit assessments with ODs in Germany were analyzed considering the results of their price negotiations. German decisions were compared to individual decisions made at European level in terms of time and content.

RESULTS: Other European countries seem to look at the German HTA model for reference. Although, some decisions at European level seem to anticipate the outcome of the German assessment in case non-orphan requirements would apply. While the German umbrella organization of SHIs (GKV-SV) most often agrees on linear pricing models, different models are adopted in France and the UK. These include capping-models or performance-based reimbursement models, exemplarily seen for some ODs. However, Germany still represents one of the most expeditious market access processes, except for Strimvelis®.

CONCLUSIONS: Seven years after the implementation of AMNOG, the pharmaceutical industry and legislators are still gaining experience from the procedures. European authorities appear to be more flexible in terms of further reimbursement opportunities in addition to the usual linear pricing model, considering the underlying evidence. GKV-SV should show more flexibility in terms of negotiation opportunities to secure successful market access strategies for future products, especially upcoming gene therapies.