Upcoming Webinar (4pm, 18 Feb 2026): Transforming Paediatric Rare Disease Trials
Led by expert speakers including Professor J. Stuart Elborn, the webinar will explore how modelling, simulation and site‑network strategies are transforming the design and delivery of paediatric rare disease trials.
Drug developers working in paediatric rare diseases continue to face significant hurdles, from small and geographically dispersed patient populations to ethical limits on sampling and limited early clinical data. In a field where speed is crucial, traditional development approaches often struggle to support timely regulatory and investment milestones.
An upcoming 60-minute webinar at 4pm GMT, 18th February 2026 will explore how innovative modelling and simulation techniques are helping reshape paediatric trial design. Approaches such as population PK modelling, allometric scaling, adult‑to‑child extrapolation and clinical trial simulation are enabling more accurate exposure–response predictions and more efficient sampling strategies, helping reduce reliance on healthy volunteer studies and easing the burden on children and families.
The session will also examine operational barriers commonly seen in paediatric rare disease trials, including delayed site start‑up, recruitment challenges and caregiver demands, and the role of pediatric site networks in improving feasibility by connecting specialised centres and sharing rare‑disease expertise.
A distinguished panel will lead the discussion, including Professor J. Stuart Elborn (Faculty Pro-Vice-Chancellor, Centre for Experimental Medicine, Institute for Health Sciences, School of Medicine, Dentistry and Biomedical Sciences (MDBS)). Professor Elborn is recognised internationally for his leadership in respiratory medicine and his contributions to transformative cystic fibrosis research.
Additional speakers include:
- Maria‑Cruz Morillo, Global Head, Allucent Rare Disease Center of Expertise
- Dr. Jessica K. Roberts, Senior Director of Pharmacometrics, Allucent
Drawing on real‑world examples and cross‑functional expertise, the webinar will outline practical strategies to de‑risk development, bridge adult‑to‑paediatric data gaps and deliver more efficient, ethical trials.
📅 Learn more and register for the event here