We are very honored to welcome you to the 1st International Conference on Rare Diseases and Paediatric Research that will take place virtually from the 18th to the 19th of November.
The Conference is organised by the ‘’95’’ Rare Alliance Greece together with Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF), Athena Research and innovation center in information communication and knowledge technologies.
The Conference is supported by the European Joint Programme on Rare Diseases (EJPRD), European Paediatric Translational Research Infrastructure (EPTRI), EURORDIS Rare Diseases Europe, European Patients’ Forum (EPF), EUPATI Greece (European Patients’ Academy on Therapeutic Innovation), Greek Patients’ Association and TEDDY (European Network of Excellence for Paediatric Research).
In a fast-changing regulatory environment, the Conference is mainly aimed at opening the discussion with all relevant stakeholders on the challenging topic of research in paediatric and rare diseases and answer the way that all the participants can contribute to high quality paediatric and RD research, aimed at the development of accessible, available and affordable therapies for paediatric and rare diseases.
Indeed, minors represent 20% of the European population and their care is one of the most important priorities and challenges for Europe. Nowadays around 50% of the medicines addressed to children and young patients have not been tested specifically for them. On the other hand 50% of the rare disease patients are children and only 5% of them have an approved treatment.
With a mandate to cover this high unmet need, currently, 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, extensive off-label use in the pediatric population, drugs and devices is a clear need in making innovation more patient-centric and personalised.
The need for appropriate quality health care engenders inequalities and difficulties in access to treatment and care and often results in heavy social and financial burdens on all pediatric patients.
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 the unmet need like neonates, pediatric cancers and rare diseases in that context, by further promoting and possibly linking paediatric and orphan regulations provisions.
The Conference will be an opportunity to be updated of all current developments, be an active participant and contribute to this European discussion. Your participation will enhance your professional competences, support the development of expertise in the fields of EU Regulation, RD and paediatric research, foster innovation and promote knowledge-sharing.
High level professionals’ representatives from the European Commission, European Parliament, European Medicines Agency, national government representatives, patient organizations and international networks will be among the speakers.
Also, throughout the stakeholder discussion sessions, the Conference will create the opportunity to strengthen the collaboration and exchange of ideas between 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