Orphan Drugs: EURORDIS & EFPIA Push for Broader Access


In Europe, patient access to Orphan Medicinal Products (OMP) for the treatment of rare diseases remains inequitable. With the common goal of ensuring broader and faster access to OMPs across Europe, EURORDIS and EFPIA have joined forces to propose a series of solutions.

According to a 2020 study, fragmentation of reimbursement policies across Europe translates into unequal access with the number of reimbursed OMPs ranging from over 100 in Germany, France, Italy, and the Netherlands to zero in Armenia.

Rare Diseases Europe (EURORDIS), the patient-driven alliance of patient organisations representing 1000 rare disease patient organisations in 74 countries and the European Federation of Pharmaceutical Industries and Associations (EFPIA), the organisation that represents the biopharmaceutical industry operating in Europe, have issued a joint statement on their shared goal to ensure broader and faster access to OMPs to all European patients.

Specifically, the statement presents the output of a structured dialogue between the two organisations to identify a series of solutions, among which are Equity-Based Tiered Pricing, EU-level and cross-country collaboration for OMPs, and the adaptation of country-level HTA and P&R frameworks to the specificities of rare diseases

The European Union introduced the Orphan Medicinal Product (OMP) Regulation in 2000 to address patient access to OMPs yet current debate has called for revision of that regulation, arguing that root causes to impaired patient access to OMPs are complex and multi-faceted.

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