Drug developers face unique challenges in pediatric rare disease trials, including small and geographically dispersed patient populations, strict ethical limits on sampling and sparse early clinical data. In a competitive landscape where speed is critical and every month can influence funding rounds and regulatory milestones, traditional development approaches often fall short. However, forward-thinking strategies are transforming these challenges into opportunities to accelerate approval and market entry.
This webinar explores how expertise in modeling and simulation is reshaping pediatric trial design by simulating drug pharmacokinetics across age groups, growth phases and disease states. Approaches such as population pharmacokinetic (PK) modeling, allometric scaling, adult extrapolation and clinical trial simulation enable more accurate exposure-response predictions, optimize sparse sampling strategies to meet FDA expectations and leverage early PK/pharmacodynamic (PD) data. In appropriate pediatric-specific scenarios, these tools can support direct-to-patient development strategies, reducing reliance on redundant healthy volunteer studies, shortening timelines and minimizing burden on children and families.
Beyond study design, pediatric rare disease trials frequently encounter operational challenges, including delayed site start-ups, recruitment shortfalls, caregiver burden and protocol misalignment, which can lead to screening failures and increased costs. Pediatric site networks play a critical role in addressing these barriers by connecting specialized centers, standardizing processes and sharing rare-disease expertise through key opinion leader (KOL) collaboration.
Drawing on real-world examples and cross-functional perspectives that integrate modeling, clinical operations and site networks, this webinar presents proven strategies to de-risk development programs, bridge adult-to-pediatric data gaps and enable more efficient, ethical and feasible pediatric rare disease trials. Attendees will learn how to move from early data generation to successful site-level execution without compromising patient safety or scientific rigor.
Register for this webinar to learn how pediatric rare disease trials can be strengthened through model-informed study design and site network execution.
Speakers
J. Stuart Elborn CBE, MD, FMedSci, Honorary Professor of Medicine, Queens University Belfast; Deputy Chair, Regulatory and Quality Improvement Authority
Dr. Stuart Elborn is an accomplished leader with extensive experience across the public sector, particularly in health, social care and higher education. With a career spanning over 40 years, he has held senior leadership positions at Queen’s University Belfast, Imperial College London/Royal Brompton Hospital and in health and social care.
A leading clinical academic, Stuart has spearheaded numerous clinical trials, including groundbreaking research in cystic fibrosis that has contributed to transformative therapies now benefiting most patients with the condition. He has also played a key role in developing evidence-based guidelines for the management of cystic fibrosis and bronchiectasis.
In his university leadership roles, Stuart has driven strategic planning, governance reform and transformation initiatives focused on delivering optimal teaching and research infrastructure to support innovation. These include a state-of-the-art Simulation Center and three innovation centers in Queens.
He has championed proportionate and effective regulation through the Regulatory and Quality Improvement Authority (RQIA) where he is a passionate advocate for safe, high-quality and evidence-informed care, achieved through clear strategic intent, robust stakeholder engagement and a strong sense of purpose in implementation.
Stuart earned his Medical Degree from Queen’s University Belfast. He is a Fellow of the Royal College of Physicians (London), the European Respiratory Society and the Academy of Medical Sciences.
Message Presenter
Maria-Cruz Morillo, Allucent Center of Expertise, Rare Diseases, Allucent
Maria-Cruz Morillo, MS Pharmacy, has over 28 years of experience in drug development, with a strong background in project management, development strategy and investigational medicinal product (IMP) supply across both biotech and CRO environments. She brings a strategic, execution-focused approach to clinical development and has played a key role in the successful design and delivery of clinical programs that support market authorization for rare endocrinology and autoimmune hematology indications.
Maria-Cruz currently leads the Allucent Center of Expertise, Rare Diseases, where she integrates cross-functional capabilities to deliver tailored, strategic solutions for rare disease clinical trials, with a focus on operational excellence, feasibility and successful trial execution.
Message Presenter
Jessica K. Roberts, PhD, MSCI, MBA, Senior Director, Pharmacometrics, Allucent
Dr. Roberts has over 10 years of professional experience in clinical pharmacology and pharmacometrics. In her current position as Senior Director of Pharmacometrics at Allucent, she is responsible for directing and conducting modeling and simulation analyses and providing strategic advice to clients. She also leads the Quantitative Translational Pharmacology team at Allucent. Before this role, Dr. Roberts worked as a Consultant at Metrum Research Group and Cognigen, a division of Simulations Plus. She also worked at St. Jude Children’s Research Hospital before becoming a Consultant.
Dr. Roberts currently serves as adjunct faculty at the University of Tennessee Health Sciences Center’s School of Pharmacy and as an Associate Editor for the Obstetric and Pediatric Pharmacology Specialty section of Frontiers. She holds a Bachelor’s Degree in Biochemistry from Texas A&M University, a Doctorate degree in Pharmacology and Toxicology and a Master of Science in Clinical Investigation from the University of Utah. Dr. Roberts completed a postdoctoral fellowship in Clinical Pharmacology at the University of Utah School of Medicine. To gain a better understanding of the business landscape, she received her Master of Business Administration from Washington University in St. Louis.
Message PresenterWho Should Attend?
This webinar will appeal to VPs, Directors, Managers and Heads involved in:
- Preclinical Research & Development (R&D)
- Biostatistics and Data Science
- Regulatory Affairs/Strategy
- Clinical Project Management/Trial Oversight
- Clinical Operations/Clinical Research
- Medical Affairs/Medical Writing/Pharmacovigilance
- Chief Medical Officers (CMOs) and CEOs of small and medium-sized biotech/pharmaceutical companies
What You Will Learn
In this webinar, the featured speakers will cover:
- How model-informed drug development uses early PK/PD data, extrapolation and simulation to enable earlier, regulator-ready pediatric dose selection with fewer patients
- Why pediatric rare disease trials require adapted clinical operations including, optimized sampling, flexible protocols and caregiver-centric execution, to remain feasible and efficient
- How pediatric site networks operationalize model-informed designs by accelerating start-up, improving recruitment and ensuring consistent, high-quality execution across specialized centers
Xtalks Partner
Allucent
Allucent is on a mission to help bring new therapies to light by solving the distinct challenges of small and mid-size biopharma companies. The company is purpose-built through the convergence of leading providers to address this unmet need. Today, Allucent is a global provider of comprehensive drug development solutions, including clinical trial operations, regulatory strategy, clinical pharmacology, pharmacovigilance, and biostatistical programming consulting. With more than 30 years of experience in over 70 countries, Allucent’s individualized partnership approach provides experience-driven insights and expertise to assist its clients in successfully navigating the complexities of delivering novel treatments to patients.
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