With 3 million people affected in France, rare diseases are a major public health issue.
25% of concerned people must wait, on average, four years before getting a reliable diagnosis.
For 95% of the 7,000 rare diseases identified, there is no curative treatments.
This new position paper proposes to:
- Review the conditions of rare diseases management…
- … and of orphan drugs market access in France
- Analyze the organization of the different stakeholders
- Illustrate the current situation through the study of Narcolepsy