Daria Julkowska | Scientific Coordinator
ERDERA - INSERM

Assistant Director, Inserm – Thematic Institute of Genetics, Genomics and Bioinformatics (IT GGB) Coordinator, European Rare Diseases Research Alliance (ERDERA) Dr Daria Julkowska is a leading architect of the European rare diseases research ecosystem. As Coordinator of ERDERA – the European Rare Diseases Research Alliance and Assistant Director at Inserm, she drives the creation of a sustainable, integrated framework that unites funding bodies, research institutions, industry, and patients across 37 countries. Over the past decade, Dr Julkowska has led major EU initiatives including E-Rare-3 and the European Joint Programme on Rare Diseases (EJP RD), mobilising more than €630 million to accelerate diagnosis, therapy development, and data integration. She has pioneered innovative public–private partnerships and policy mechanisms now adopted in Horizon Europe, and works closely with the European Commission, EMA, and EURORDIS to align research and regulatory environments. Awarded the Inserm Prize for Research Support (2024) and the EURORDIS Black Pearl Award for European Leadership(2020), she continues to champion cross-sector collaboration to translate research into tangible benefits for people living with rare diseases.

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

Rima Nabbout, MD/PhD, Hopital Necker- Enfants Malades