ORPH-VAL WORKING GROUP
Context and issue
Rare diseases have been identified as a major public health issue in Europe following the orphan drug legislation introduced in 2000, leading in particular to the development of new orphan medicinal products. Many of these new products however do not reach patients in time, sometimes leading to continued morbidity and/or loss of life
These delays in treating patients, which vary greatly from country to country, are in part due to inconsistencies in pricing and reimbursement systems across Europe.
It is essential that pricing and reimbursement processes in Europe ensure that patients with rare diseases get quick and consistent access to potentially life-saving orphan medicines, incentives for academia and industry to development new medicines are maintained, and the healthcare system continues to be financially sustainable.
ORPH-VAL paper
Recommendations for value assessment and funding process in rare diseases
9 Principles covering recommendations on P&R decision criteria, P&R decision processes, sustainable funding systems and European coordination
Access Full paperORPH-VAL Principles in practice
Comparison of alignment of five European P&R systems
Explore the pricing and reimbursement systems for OMPs in selected European countries. An overview of where they align with or diverge from the nine principles suggested in the original OrphVal report
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