Any disease affecting fewer than 5 people in 10,000 in the EU is considered rare. Although this might appear small, it translates into approximately 246,000 people. Approximately 5,000-8,000 distinct rare diseases affect 6-8% of the EU population i.e. between 27 and 36 million people. Orphanet database contains descriptions of 6172 (in 2019) clinically unique rare diseases, whose age of onset in 69.9% is for exclusively pediatric. Indeed, minor represent 20% of the European population and their care is one of the most important priorities and challenges for Europe. Nowadays around the 50% of the medicines addressed to children and young patients have not been tested specifically for them.
In our days, drug development is undergoing a paradigm shift with an increasing emphasis on understanding pathology at the molecular and systems biology levels leading to more targeted therapies with disease modifying properties. Improved integration of the development and regulation of diagnostics, drugs and devices is a clear need in making innovation more patient-centric and personalized.
These goals should be guaranteed by maintaining the Paediatric Regulation at the core of paediatric medicines development and by implementing an effective system for giving priority to paediatric rare diseases in that contest by linking Paediatric and Orphan Regulations provisions (it would be surprising not citing orphan Regulation).
To this aim and with great pride the 95, Rare Alliance Greece, and EPTRI (European Paediatric Translational Research Infrastructure), together with Athena Research and innovation center in information communication & knowledge technologies, are very delighted to virtually welcome friends and colleagues to the 1st International Conference on Rare Diseases and Paediatric Research that will take place virtually on the 18th and 19th of November 2021.
The Conference is supported by Consorzio per Valutazioni Biologiche e Farmacologiche, EUPATI Greece (European Patients’ Academy on Therapeutic Innovation) and TEDDY (European Network of Excellence for Paediatric Research).
The main topics covered during the 1st International Conference on Rare Diseases and Paediatric Research include the following:
-The future of the Regulatory framework
-Supporting tools and funding opportunities in RD and Paediatric Research
-Regulatory tools to speed up orphan and paediatric medicines development
-Clinical Trials and non clinical research: the role of Networks and Infrastructures
-RD and Paediatric Research during the COVID-19 pandemic
The Conference will be an opportunity to enhance the professional competences for the development of expertise in the fields of RD and paediatric research, to foster innovation and knowledge-sharing.
Also, throughout the stakeholder round tables the Conference will create the opportunity to strengthen the collaboration and exchange of ideas between researchers, patients, researchers, industries, policy makers and regulators and others.
Dimitrios Athanasiou, World Duchenne Organisation (WDO), European Patient Forum (EPF), 95, Rare Alliance
Adriana Ceci, European Paediatric Translational Research Infrastructure (EPTRI), Gianni Benzi Foundation, member of CVBF
Panos Macheras, ATHENA, Athena Research Center