Training Sessions
Opening Session
- Welcome & overview of the TEDDY Network Donato Bonifazi
- Introduction from the KIDS groups All the groups
- Welcome & introduction to the ERDERA training Rima Nabbout
- The role of advocacy and patient organizations Dimitrios Athanasiou
Rare and Genetic Diseases Specificities
- Overview of rare and genetic diseases in pediatric patients Persephone Augoustides-Savvopoulou
- Challenges in diagnosis, research and care Rima Nabbout
- Case studies and interactive discussion Bernadette Sheehan Gilroy
Patients’ and Children’s Rights
- Legal and ethical considerations in pediatric research Silvia Torretta
- The role of young patients in matters related to their health Suja Somanadhan
- Group discussion and real-world examples Francesco La Penna
Translational Research Challenges
- Innovative therapies and the translational research journey with EPTRI Donato Bonifazi
- RD unmet needs and healthcare impact with prioritization in health and research decision-making Dimitrios Athanasiou
- Interactive session with case-based scenarios Rima Nabbout
Shared activities from the TEDDY KIDS Network
- Establish a solid network: a perspective from a charity Eleonora Passeri
- Designing a paediatric multinational trial with kids Ornela Cullufe
- Participating in the TEDDY Network Board Fiona Rako
Patient Engagement in Clinical Trials
- Active Patient Involvement in Clinical Trials: PROMs, PREMs, and PISs Emanuela Crea
- Consent/assent, clinical study protocol, and lay summaries Silvia Torretta
- Q&A and participant reflections All
Orphan Medicinal Products & Pharmacovigilance
- Availability and access to orphan drugs in the EU Dimitrios Athanasiou & Rima Nabbout
- Pharmacovigilance and off-label use in pediatric settings Sophia Trantza
Closing Session
- Key takeaways from the training Francesco La Penna
- Participant feedback and evaluation All the groups
- Closing remarks Donato Bonifazi