What is included in a rare disease national plan/ strategy?

The main objective of a national rare disease plan or strategy is to recognise rare diseases as a national public health priority and define specific national care pathways that will ultimately reduce the burden of these diseases on patients, their families and their carers. Read more.

A plan/ strategy includes relevant national measures to improve care at every stage of the patient’s journey, from diagnosis to access to treatments and therapies, through long-term follow-up, social care and services. A national rare disease plan/strategy often includes measures to foster innovative research in the field of rare diseases. Each measure needs to be associated with a defined budget.

A national plan/ strategy requires a solid governance composed of a multi-stakeholder governing committee (including patient representatives) that monitors the implementation of the national measures against agreed timelines and public health indicators.

Rare Diseases: a domain of very high European added-value

Due to the rarity, heterogeneity and complexity of each of the over 6,000 rare diseases, no one country alone can tackle the issues surrounding rare diseases. This is why European action to bring scarce knowledge and expertise together across borders has a strong added value.

National rare disease policies are intrinsically linked with European rare disease policies. EU regulations and policies that have been developed to foster cross-country collaboration need to be integrated into national policies.

On 8 June 2009, the Ministers of Health of the 28 EU Member States adopted the EU Council Recommendation on “an action in the field of rare diseases” which recommended EU Member States “to elaborate and adopt a plan or strategy […] aimed at guiding and structuring relevant actions in the field of rare diseases within the framework of their health and social system”.

The strategic areas below constitute the backbone of a national plan or strategy for rare diseases in European countries:

  1. Governance
  2. Definition, codification, inventorying of rare diseases and patient registries
  3. Research (including development of orphan drugs, innovative treatments)
  4. Centres of Expertise and European Reference Networks (ERNs)
  5. Social services and programmes adapted to rare diseases
  6. Empowerment of patient organisations
  7. Sustainability of the national plan or strategy

Please click here to access the list of main European recommendations and policies as well as EURORDIS documents necessary to design and build a good national plan or strategy for rare diseases. It is an invaluable one-stop shop of key references to be used as advocacy tools for concrete improvements in favour of your National Rare Disease Community.

Page created: 13/02/2019
Page last updated: 26/04/2019
The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseasesRare Disease International Bringing together patients, families and experts to share experiences in a moderated multi-language forum. RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases