Rare diseases are a major public health issue. Despite new legislation to support the authorisation of orphan medicinal products to treat rare diseases, many products do not reach patients in time, leading to continued decline in quality of life and/or loss of life. Delays in treating patients vary from country to country and are in part due to differences in pricing and reimbursement systems across Europe.
It is essential that pricing and reimbursement (P&R) processes across Europe:
- ensure that patients with rare diseases get quick and consistent access to potentially life-saving orphan medicines
- maintain incentives for academia and industry to develop new medicines
- enable European healthcare systems to be financially sustainable
“By helping to improve the consistency of pricing and reimbursement processes for orphan drugs across member states, these new principles offer a pathway towards better and more equal access to these medicines while keeping healthcare systems sustainable” said Prof Lieven Annemans, Professor of Health Economics at Ghent University, Belgium and Chair of the Working Group.
The article, titled “Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)”, has been published in the Orphanet Journal of Rare Diseases and proposes that policymakers and rare disease stakeholders in member states use the recommendations from ORPH-VAL, to explore where local P&R systems diverge from the principles, and work together to identify solutions that will improve overall access to medicines for patients with rare diseases across the EU.