anis Mimouni joined Critical Path Institute (C-Path) on 1 September 2025 as Associate Director for Regulatory Science. In this role, he works closely with the European Office leadership and the global C-Path teams to coordinate EU-funded projects, including, but not limited to, those in the field of rare diseases; and to support the coordination of specific global efforts led by the European Office. He leads the operational components of European projects, ensuring alignment with regulatory-science priorities and serving as a point of contact for cross-functional efforts. Prior to joining C-Path, Yanis co-led the European Rare Diseases Research Alliance (ERDERA), a ten-year Horizon Europe Partnership uniting more than 170 organisations across 37 countries and leveraging over €340 million in funding. ERDERA aims to accelerate diagnosis, clinical research readiness, and therapeutic innovation for rare diseases by strengthening research infrastructures, data services, and clinical research networks. As Scientific Deputy Coordinator, Yanis oversaw the Clinical Research Network and contributed to transversal services, regulatory support, and data-related services. He has been deeply engaged in ensuring that ERDERA activities aligned with European and international regulatory-science priorities. C-Path is actively involved in the ERDERA project, where Yanis continues to play an important role representing C-Path. In parallel to ERDERA, Yanis has also been actively involved in linked IHI and European Commission-funded projects, notably RealiseD and INVENTS, tackling innovation in clinical-trial methodologies and operations, the regulatory framework for in-silico trials, and the systematic embedding of patient perspectives into clinical research. In these projects, he continues to contribute by bridging their outputs with C-Path expertise, further reinforcing C-Path’s global leadership in regulatory science and patient-focused drug development. He holds a PharmD (Doctor of Pharmacy) and an MSc in Pharmaceutical Medicine from Université Claude Bernard Lyon 1, as well as several postgraduate diplomas in clinical trial evaluation, paediatric drug development, and orphan drugs. He also holds certifications in data science. Yanis is a passionate builder of international collaborations who thrives at the crossroads of regulatory science, data innovation, and patient engagement. Based in Paris, he enjoys connecting communities across Europe and beyond.
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.