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
A voz das pessoas com doenças raras na EuropaEURORDIS A voz internacional das pessoas com doenças raras, Rare Diseases InternacionalRare Disease International Reúne doentes, famílias e especialistas para partilhar experiências num fórum multilinguístico. RareConnect O programa Rare Barometer é uma iniciativa da EURORDIS para a realização de inquéritos para transformar a experiência das pessoas com doenças raras em números e factos que podem ser partilhados com os responsáveis pela tomada de decisões.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 Adira ao maior encontro europeu das partes interessadas no âmbito das doenças raras na Conferência Bienal Europeia sobre Doenças Raras e Produtos Órfãos. A ECRD é uma iniciativa da EURORDISEuropean Conference on Rare Diseases