OBJECTIVES: To assess the market access and pricing and reimbursement (P&R) processes for orphan drugs (OD) in the five European countries (EU5) of France, Germany, Italy, Spain and UK to determine decision relevant components.

METHODS: National laws and regulations were assessed via targeted literature research, including official health technology/reimbursement agency websites. Additionally, expert interviews were conducted.

RESULTS: In general, there is no extra specific pathway for ODs in EU5 with respect to P&R. The main P&R pathways remain applicable, but with some exceptions in different countries. There are possibilities for early access programs. For instance, in France and UK, ODs may enter early access programs if there are no therapeutics alternatives available, whilst under certain circumstances, the Italian NHS grants access to a drug even before its marketing authorization (“early access”=L648/96; Italy's Fondo AIFA 5%). In Italy, ODs Market Access Holders are also allowed to submit a P&R dossier for AIFA immediately after the CHMP approval (instead of waiting until Commission Decision is publicized officially) and access an accelerated evaluation process. Germany shows a different approach, where the main P&R process is simplified and an added benefit for ODs (yearly revenue below €50 million) is given by law. This “automatic” added benefit might appear an early free ride for reimbursement, but strong evidence is still needed. Missing evidence leads to time-limited benefit. In Spain, no special rules apply for ODs (same process as for non-ODs), however mandatory price deduction applied for ODs is lower than the one existing for non-ODs.

CONCLUSIONS: Even though there are similarities between the EU5 countries for the market access for ODs, processes vary between countries. An improved pathway through the European reimbursement market can be developed by knowing the specialities and the pitfalls, which can lead to improved price negotiations.