Transposition and implementation of EU rare disease policy in Eastern Europe

ABSTRACT

Introduction: А series of European Union (EU) political decisions have made rare diseases one of the cornerstones of the common European health policy. Adopted in 2009, Council Recommendation on an action in the field of rare diseases aimed to serve as a policy-making guideline. However, the implementation report, which followed it, neither performed detailed cross-country comparison, nor assessed the impact of the policies.

Areas covered: A 10-indicator set was elaborated to structure the review and to describe rare disease activities in 14 Eastern European countries.

Expert commentary: Taking into account all indicators, EU member states outperform candidate and potential candidate countries in terms of rare disease policy planning and implementation. Hungary is the top performer, followed by Bulgaria and Czech Republic. Non-EU countries form the bottom tier, with Serbia being the best ranked among them. While EU adhesion is a major facilitator for planning and adopting rare disease policies, local stakeholders are the triggering factor for their successful implementation. European reference networks are likely to be the future of rare disease activities in the EU. They need to synchronize and closely collaborate with all important EU projects in the field of rare diseases if they are to achieve their objectives.

KEYWORDS:

• Rare diseases
• national plans
• orphan drugs
• European reference networks
• registries
• EU

Declaration of interest

AV Pejcic is awarded with the Scholarship of the Ministry of Education, Science and Technological Development of the Republic of Serbia for PhD students. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This work was partially supported by the Ministry of Education, Science and Technological Development of the Republic of Serbia under Grant number 175014. Publication of results was not contingent to Ministry’s censorship or approval.