The general aim of EUROPLAN is to contribute to improvement in access to prevention, diagnosis, treatment and care for patients with rare diseases through the production and dissemination of data and recommendations for developing strategic plans for rare diseases.
Rare diseases are diseases of low prevalence—not more than 5 per 10.000 persons in the EU. It is estimated that between 5.000 and 8.000 rare diseases are presently recognised, affecting more than 30 million people in the EU. Rare diseases are of such low prevalence that special combined efforts are needed to address them. It`s impossible to develop a public health policy specific to each rare disease thus a global rather than an individual approach should be a logical choice. Despite the progress made over the last years in the field of rare diseases, a comprehensive and evidence based approach is still missing in many EU Member States leading to an incomplete and often inadequate framework to address rare diseases. In this context, the project aims at developing recommendations on how to define a strategic plan for rare diseases. Such recommendations will include best practices to address rare diseases and information on the different steps to develop a strategic plan.
The project is built on the commitment of experienced partners who came together to assess the effectiveness of their own national programmes. On this basis, the project will define the state of the art with regard to RD in each MS participating to the project. Information on all available initiatives related to institutional framework, surveillance system, provision of care and support to patients’ groups will be collected. Indicators to assess these initiatives will be identified through a comprehensive literature review and discussion with experts. Partners will collect data on the achievements of their initiatives through ad hoc survey, interviews and analysis of available report or official regulations. On the basis of the data collected, key stakeholders will identify best practices in an open workshop considering relevance, effectiveness and sustainability of the initiatives presented.
On the basis of the best practices thus identified, recommendations will be developed by an editorial group and fifteen national consultations will be held to assess the transferability of these recommendations to different settings. The final version will be launched in an EU workshop.
The project will develop recommendations on how to establish a strategic plan for rare diseases ensuring an inclusive and broad engagement of stakeholders (ministries, regional and local authorities, health care planners, programme managers, health care professionals, researchers and patients) and a dissemination of the recommendations in all EU Member States.