Driving Better Patient Outcomes in Rare Neurological Diseases

While there is no cure for many of these diseases, advocates are fighting for the next best thing: timely diagnosis and treatment.

A new effort by the European Alliance for Patient Access, in collaboration with the European Federation of Neurological Associations and the European Brain Council, aims to raise awareness and further educate lawmakers on these rare neurological diseases and their challenges.

The three groups convene stakeholders from across the region this week at the European Parliament in Brussels. The meeting, titled Rare Neurological Disease in the EU: The Importance of Early Diagnosis and Real-World Evidence to Drive Better Patient Outcomes, will encourage discussion of:

• Importance of Early Detection: Treatments to prevent NMOSD and other rare disease relapses are becoming more available in Europe, but unmet medical needs still persist due to late diagnosis.Early detection and timely treatment are crucial to preventing disease attacks and ensuring the best possible disease outcome.
• Value of Real-World-Evidence: Real-world-evidence is the clinical evidence that is produced in real-world settings – outside of randomised controlled trials. These data can improve understanding of unmet needs by providing information on disease burden, patient journeys, and barriers to optimal medical management.
• Lack of Awareness: Raising awareness about these rare neurological diseases, like NMOSD, is an important first step to improving outcomes for patients and their carers, not only in Europe, but across the globe.
• Barriers to Access: Several barriers hinder patients from accessing treatment and optimal care. These include challenges such as specialist shortages, outdated treatment guidelines and inequitable access to innovative treatments.

This meeting will be a first measure for advocates and stakeholders to define next steps for the community. It will also invite members of the European Parliament to join the larger rare disease discussion, informing policies that will support patients and their families across the region.