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Europe: EFPIA proposes changes to improve patient access to rare disease treatments

2022/06/20  EFPIA

EFPIA has joined with patient-driven rare disease group EURORDIS to propose changes intended to boost access to orphan drugs.

In April, EFPIA published data showing 37% of orphan drugs are available across Europe. The figure is down on the number for 2020, 41%, and the average for all types of medicines, 46%. In some countries, fewer than 10% of orphan drugs are available.

EFPIA and EURORDIS have published six proposals intended to increase equity of access, improve health technology assessment and pricing and reimbursement processes, and establish a moonshot program for basic and translational rare disease research.

The first equity access proposal covers a conceptual framework for international differential pricing. The idea is to lower the price of rare disease medicines in less wealthy EU member states without dragging down the rate paid by their richer neighbors. EFPIA members also again committed to filing pricing and reimbursement applications within two years of receiving market authorization.

In terms of pricing and reimbursement processes, EFPIA and EURORDIS want to see more EU-level and cross-country collaboration on orphan products and use of adaptive pathways and real-world evidence (RWE) in value assessments. The collaborators see adaptive pathways and RWE as ways to overcome the uncertainty about the evidence that is available at the time of the marketing authorization.

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