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EUCOPE-EURORDIS Press Release on Recommendations on Orphan Medicines Pricing and Reimbursement

13. March 2017 in News Newsletter Press Releases

Expert Group Publishes Recommendations on Orphan Medicines Pricing and Reimbursement

Brussels,13th March 2017. Rare diseases are a major public health issue. Despite new legislation to support the authorization 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.

To help obtain these goals, a European multi-stakeholder expert group, ORPH-VAL, consisting of patient representatives, physicians, academics, HTA practitioners, politicians and pharmaceutical industry representatives, has published a new set of common principles to improve the consistency of decision making for orphan drugs and balance the needs of innovators and payers. The principles are grouped into four domains:

  • P&R decision criteria: defining elements of value and other considerations specific to P&R decision for OMPs.
  • P&R decision processes: minimising decision uncertainty by systematically incorporating rare disease expertise and utilising adaptive assessment processes.
  • Sustainable funding systems: defining the mechanisms of funding for OMPs to ensure long-term sustainability and equity of funding.
  • European co-ordination: pursuing greater co-ordination of OMP value assessment processes at a European level.

“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 principles outlined in this demonstrate the increasing convergence of views on a topic that is of great importance to rare disease patients. It is critical that all stakeholders – including manufacturers, payers and patients– work together to ensure that the promise of scientific and medical advancements in rare diseases reaches the patients.” said Yann Le Cam, Chief Executive Officer of rare disease patient association EURORDIS-Rare Diseases Europe and member 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 on 10th March 2017 as an early electronic article (follow the link to access the publication).

It is proposed 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.


About EURORDIS-Rare Diseases Europe

EURORDIS-Rare Diseases Europe is a unique, non-profit alliance of over 700 rare disease patient organisations from more than 60 countries that work together to improve the lives of the 30 million people living with a rare disease in Europe. By connecting patients, families and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies and patient services. Follow @eurordis or see the EURORDIS Facebook page. For more information, visit



The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) is Europe’s principal trade body for small-to-medium sized innovative companies working in the field of pharmaceuticals and medical devices. EUCOPE has supported the ORPH-VAL group by providing fundings for the secretariat and medical writing of the publication.


Contact for more information:

Prof Lieven Annemans                                           Adam Hutchings                      

+32 (0) 476241144                                                  +44 (0) 7545875817