Context
Rare diseases affect between 500 000 and 700 000 people in Belgium. A disease is considered rare when it affects fewer than 1 in 2 000 people. Diagnosis is often difficult and slow, taking around five years on average, and most rare diseases lack curative treatments. A first Belgian Rare Diseases Plan was introduced in 2013. Since then, institutional reforms, scientific progress and European developments (such as the creation of European Reference Networks) have reshaped the landscape.
Impetus for the reform
The new plan was developed because the existing approach no longer meets current needs. Persistent challenges include long diagnostic delays, fragmented and insufficiently visible expertise, unequal access to care and treatments, insufficient data collection, lack of care coordination and complex administrative procedures. The evolving European and technological context also requires adaptation, while a more structured and coordinated organization of care is needed to reduce inequalities and improve clarity and quality.
Main purpose of the reform
The central objective of the plan is to sustainably improve the quality of life of people with rare diseases and their relatives. It aims to achieve this by strengthening the organization of care, improving access to expertise and treatments, enhancing the use of data, simplifying administrative processes, and ensuring clearer communication and coordination across the system.
Content/characteristics
The plan is structured around six domains and 25 actions:
- Identification and visibility of expertise: including mapping available expertise, improving visibility and identifying expertise gaps.
- Development of integrated care pathways: including personalized care plans tailored to patient needs, care coordination and support, and measurement of patients’ experience.
- Improvement of data and strategic information: including strengthening the central rare diseases registry and improving data collection and interoperability.
- Access to treatments and administrative simplification: including improving access to innovative therapies, supporting clinical trials and reducing administrative burdens.
- Faster diagnosis: strengthening early detection through tools, training and support for healthcare professionals.
- Information and awareness: improving access to information and increasing awareness among citizens and professionals.
A Rare Diseases Committee responsible for the coordination, monitoring and evaluation of the plan will be created. The plan was developed through a participatory (“bottom up”) approach, involving key stakeholders including patients, clinicians and institutions.
Implementation steps taken
Implementation is coordinated by the FPS Public Health and the National Institute for Health and Disability Insurance, with continued involvement of stakeholders. Key first steps include the creation of the Rare Diseases Committee, mapping expertise, developing care pathways and strengthening the national registry.
Evaluation
Implementation will be monitored and evaluated continuously, in particular through the Rare Diseases Committee, which is responsible for tracking progress, evaluating impact and supporting adjustments where necessary.