EVENTS AND MEETING

B4B policy initiatives are aimed to advance awareness and knowledge of rare neurological diseases via cross-border collaboration and to enable better diagnosis and management of patients with these diseases.

These initiatives unite all interested parties, who are working:

• to increase the visibility, recognition and awareness of rare neurological disorders in order to facilitate early diagnosis of these conditions;
• to promote and facilitate partnership and collaboration between physicians, researchers, patient advocates, carers, policy- and decision-makers and industry;
• to encourage and support research and the translation of scientific breakthroughs into clinical practice;
• to contribute to improved standard of care for patients with rare neurological disorders which is agreed across Europe, and to ensure equity of access to diagnosis, treatment and care.

In particular events such as the European Parliament meetings are aimed to coordinate the efforts of existing groups in Europe with the shared goal of improving the treatment and care of patients with rare neurological disorders.

THE EUROPEAN PARLIAMENT MEETINGS

Rare neurological diseases of childhood pose a serious medical health issue in Europe. Although individually uncommon, collectively there are thousands of rare diseases that affect a large number of people.
The need to collaborate to focus on these disorders was highlighted during the meeting: RARE NEUROLOGICAL DISEASES OF CHILDHOOD: WE TREAT THE CHILD TO TREAT THE ADULT organized by the Brains for Brain Foundation at the European Parliament in Brussels on December 2nd 2010. The main objective of the meeting was to acknowledge the growing interest of the European Union Commission in both rare and neurological disorders. The meeting was also aimed to increase visibility of B4B, create and share information about rare neurological diseases, raise awareness of the issue, identify patients’ needs and catalyze research efforts toward the understanding and treatment of rare neurological disorders. The meeting represented an important step in identifying challenges to improve the quality of life of families and patients affected by rare neurological disorders. To this effect participants endorsed a “Declaration of Principles” launching a new initiative to facilitate research in rare neurological diseases and to improve their diagnosis and treatment.

To reinforce the necessity to work together and center attention on rare neurological disorders of infants and children the B4B Foundation has more recently organized the roundtable: “PAVING THE WAY FOR A COMPETITIVE AND DYNAMIC EU KNOWLEDGE ECONOMY: THE WAY FORWARD IN RARE DISEASES” which was held again at the EU Parliament in Brussels, on November 26, 2013. The meeting rallied numerous relevant stakeholders to discuss initiatives aiming to create a model of intersectoral cooperation that could facilitate the set-up of a European PhD Programme in the area of rare neurological diseases of children. In line with the core principles established by “Towards a Maastricht for Research”, the Brains for Brain Foundation has in fact created a network of Universities and Scientific Societies to start a doctorate programme aimed at furthering the knowledge on neurometabolic diseases amongst young physicians and scientists in order to establish an European Network of specialized experts and maintain excellence in Europe. Such initiative intends to enhance an advance awareness and knowledge about rare diseases via cross-border collaboration and to enable better diagnosis and management of patients affected by these diseases.
Holding these meetings, B4B wished to demonstrate the unity of intent of family associations, biotechnology and pharmaceutical industries and the scientific community in stimulating interest in rare neurological diseases and advance care for affected children.

The B4B EP Roundtable in particular represents a major step toward the establishment of a successful EU cross border collaboration and cooperation to raise awareness about rare diseases of childhood and keep them on the health-care agenda. Although individually rare by definition, rare diseases collectively affect millions of people worldwide. Joint forces to tackling them are essential to ensure that affected children are given the priority they deserve and that their needs are met.

With the purpose of impacting the Institutional Leaders and the Society to the problem of rare diseases and encouraging constructive action on the part of policy makers, B4B actively participated to the Workshop “SCIENCE AND INNOVATION: CLOSER THAN RARE!” held at the European Parliament in Brussesl on 17th February 2014. The workshop, hosted by MEPs Maria da Graça CARVALHO, and Mario DAVID under the patronage of the Presidency of the Portuguese Republic, First Lady of Portugal Maria Cavaco Silva, was also attended by Tonio BORG, Commissioner for Health and Consumer Policy, European Commission.

During the meeting it was generally agreed that much of European research lacks overall coordination and remains a little fragmentary.

One of the big challenges with rare disease is diagnosis.

Diagnosing a rare diseases is in fact often a long and complex process. This is mainly due to the fact that scientific knowledge is generally insufficient and scattered. The development of joint actions and collaborative efforts were encouraged since cooperation is the crucial key necessary to overcome the gaps in research in the area of rare diseases in EU.

It was highlighted the importance of pooling knowledge, resources and expertise on rare diseases, funding related research and authorising the best possible medicines for these diseases.
Participants agreed to continue their hard work so that in the near future, each and every patient affected by a rare disease can access the best possible diagnosis and care.