From Small Populations to Robust Evidence: European Methodologies for Paediatric and Rare Disease Drug Development
As part of the TEDDY General Assembly and Scientific Meeting 2026, taking place from 18 to 20 September at the University of Bari Aldo Moro, Piazza Cesare Battisti, 1, 70121 Bari BA and online, TEDDY will host a dedicated panel discussion examining how innovative methodologies can contribute to addressing evidence-generation challenges in paediatric, rare disease and other small-population research.
The panel, entitled “From Small Populations to Robust Evidence: European Methodologies for Paediatric and Rare Disease Drug Development,” will take place on 19 September 2026, from 12:00 to 13:00.
The discussion will bring together representatives of three European projects: Flora Musuamba Tshinanu for ERAMET, theEcosystem for Rapid Adoption of Modelling and Simulation Methods to Address Regulatory Needs in the Development of Orphan and Paediatric Medicines, Sarah Zohar for INVENTS, theInnovative Designs, Extrapolation, Simulation Methods And Evidence-Tools For Rare Diseases Addressing Regulatory Needs and Solange Corriol-Rohou, for RealiseD, thecompRehensive mEthodological and operational Approach to cLinical trialS for rare and ultra-rarE Diseases.
The session will be moderated by Viviana Giannuzzi, Fondazione per la Ricerca Farmacologica Gianni Benzi (FGB).
The panel will use a comparative and interactive format to explore how innovative methodologies can move from development to practical use in paediatric and rare disease research. The discussion will focus on methodological maturity, regulatory and HTA acceptance, operational implementation, and the role of research networks and data infrastructures. Particular attention will also be given to the sustainability, accessibility and reuse of tools and resources developed through EU-funded projects. Bringing together perspectives from ERAMET, INVENTS and RealiseD, the session will identify opportunities for collaboration, reduce fragmentation across initiatives and highlight the remaining gaps in evidence generation and implementation.
If you are interested in attending TEDDY General Assembly and Scientific Meeting 2026, you can register through the link below:
TEDDY General Assembly – Registration Form – Fill in form
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