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
La voz de los pacientes de enfermedades raras en EuropaEURORDIS La voz internacional de las personas que tienen enfermedades rarasRare Disease International Reuniendo a pacientes, familiares y expertos para compartir experiencias en un foro moderado en distintos idiomas. RareConnect El Programa Rare Barometer es una iniciativa de EURORDIS que realiza encuestas para transformar las experiencias de los pacientes con enfermedades raras en cifras que puedan compartirse con los responsables políticos.Rare Barometer Una campaña internacional de sensibilización que se celebra todos los años el último día de febrero, El Día de las Enfermedades Raras es una iniciativa de EURORDISRare Disease Day Únete al mayor grupo de partes interesadas en toda Europa, en la Conferencia Europea sobre Enfermedades Raras y Medicamentos Huérfanos que se celebra cada dos años. ECRD es una iniciativa de EURORDISEuropean Conference on Rare Diseases