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 people1. Orphanet database contains descriptions of 6172 (in 2019) clinically unique rare diseases, whose age of onset in 69.9% is for exclusively pediatric2. 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.
To this aim and with great pride the Athena Research and innovation center in information communication & knowledge technologies and Consorzio per Valutazioni Biologiche e Farmacologiche, together with 95, Rare Alliance Greece and EURORDIS (The Voice of Rare Disease Patients in Europe), 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 from the 31st May and the 1st of June 2021.
The Conference is supported by EPTRI (European Paediatric Translational Research Infrastructure), 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
- Innovative technologies and Health Data
- Clinical Trials and 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), Alliance 95
Adriana Ceci, Gianni Benzi Foundation
Panos Macheras, ATHENA, Athena Research Center