Rima Nabbout | MD/PhD
Hopital Necker- Enfants Malades

Rima Nabbout is Professor of Paediatric Neurology at Université de Paris cité and Director of the French reference centre for rare Epilepsies at Necker Enfants Malades, APHP, Paris, France.
She is elected president of the French society for pediatric Neurology.
She is coordinator of RHU Innov4-ePiK, director of the epilepsy chair (GEEN-DS) at Institut Imagine, INSERM U1163, where she leads the epilepsy program. Her research focuses on childhood rare and genetic epilepsies aiming to advance holistic care through syndromes’ delineation, deep phenotype-genotype correlations, biomarkers and underlying mechanisms identification, therapies’ development and psycho-social and economic evaluation of these diseases.
She is member of ERN EPICARE steering committee, coordinator of the TF on transition and member of 2 task forces on nosology and Big-data of the ILAE. She is member of the ExCo of ERDERA, leading the expertise workstream and of IHI EU project, Realised. She is president of the scientific committee of the BNDMR (French registry for rare diseases) and member of the scientific council of INSERM.
She is principal Investigator in multiple clinical trials on rare epilepsies with an interest in the development of patients’ centered end-points and innovative methodologies for personalized therapies. She has authored over 300 peer-reviewed papers and received Horizons 2020, FP7, national grants and philanthropy funds.

Appearances:

Pre-Congress Workshops - Monday 27th October @ 10:00

Innovative Methodological Approaches Accelerating Rare Diseases Drug Development: Bridging Data, Innovative Design, and Decision-Making

Developing effective therapies for rare and ultra-rare diseases (U)RD is hindered by inherent challenges such as limited patient numbers, disease heterogeneity, and complexities in defining clinically meaningful endpoints. Accelerating progress requires novel methodological approaches across the entire R&D spectrum. European initiatives like ERDERA, INVENTS, and RealiseD are tackling these challenges head-on through collaborative, multi-stakeholder efforts. This session will showcase how these projects are developing and implementing cutting-edge methodologies to optimise clinical trial design, leverage diverse data sources including Real World Data (RWD), streamline evidence generation, and ensure alignment with regulatory and HTA requirements, all centered on patient needs.

We will hear about ERDERA's work on innovative data integration, regulatory-grade natural history cohorts, disease progression modelling, and clinical trial simulation. INVENTS will present its generalizable framework for evidence assessment in small populations, including in silico trials and innovative designs. RealiseD will highlight its comprehensive approach to optimising (U)RD clinical trials, focusing on innovative designs, patient-centered outcome measures (PCOMs), regulatory/HTA co-creation, and the development of a practical playbook.

Moderator: Daria Julkowska, Scientific Coordinator, ERDERA - INSERM