There remains a crucial need to include children in clinical development as historically they have been underrepresented in clinical trials. This is due to the complexities of including children, their vulnerabilities and the lack of motivation for drug developers to pursue marketing authorization for pediatric populations. These challenges faced in developing pediatric medicine are not dissimilar to those encountered in orphan drug development and often overlap, with rare pediatric diseases being particularly disadvantaged.
With limited experience among stakeholders in regulating and conducting pediatric clinical trials, several factors contribute to the lagging availability of scientifically tested and approved treatments for these vulnerable populations, including:
- small population sizes and the need for age-appropriate formulations
- ethical conservatism and regulatory restrictions
- specific ADME considerations and limitations in blood sampling
- differences in body size, development and compliance during puberty
- particularities of neonates and infants contributing to heterogeneity
- the involvement of families in treatment and in pediatric clinical trials
View this webinar and explore strategies to address the unique challenges of developing improved medicines for children with both rare and non-rare diseases, including:
- Innovative clinical trial solutions, including modern statistical methodology approaches, modelling, simulation and extrapolation of efficacy and safety from other populations
- Optimization of blood sample requirements to minimize frequency of invasive draws, their volume and promoting analytical micro methods
- Working with patient advocacy groups, the patient’s families and the application of patient-centric trial principles to improve recruitment, the patient consent process and compliance
- Cross-functional stakeholder collaboration including regulators on the scientific and regulatory progress of pediatric and rare disease drug development, discussing individual applications with regulators, participation at specialized conferences and regulatory and legislation consultations
About the Presenters
Richard Veselý, MD – Vice President, Regulatory Strategy
With over 12 years of experience at the European Medicines Agency as Paediatric Coordinator and Head of Office for Rheumatology, Immunology, Gastroenterology, and Respiratory Diseases, Dr. Veselý is an expert in regulatory procedures in the European Union. He led the team in evaluating marketing authorizations, scientific advice, and paediatric and orphan designation procedures. With 30 years of clinical and research experience as a paediatrician, paediatric immunologist, and rheumatologist, Dr. Veselý has expertise in the development of paediatric investigation plans and initial paediatric study plans.
Vanessa Beddo, PhD – Vice President, Biostatistical Consulting
Vanessa Beddo, PhD regularly provides innovative and complex study design solutions in support of efficiently navigated regulatory paths, building on her 15+ years of experience in clinical trial design and analysis. As VP of Biostatistical Consulting at Allucent, she is also responsible for regulatory body interactions on an ongoing basis throughout program lifecycles on behalf of her clients. Prior to this role, Dr. Beddo served in various management positions involving oversight of statistical/programming staff and leadership with respect to departmental initiatives, processes, and training. Her expertise also includes clinical development of pharmaceuticals and biologics, having served as the lead statistician on Phase I-IV clinical trials and drug submissions leading to successful drug approvals.
Maria-Cruz Morillo, MS — Global Therapeutic Operations Lead, Rare Diseases
Maria-Cruz Morillo, MS Pharmacy, has deep knowledge in drug development after 27 years of experience in Project Management and Strategy and IMP Supply in both biotech and CROs. Her strategic mindset and demonstrated ability to design and implement effective study protocols has contributed significantly to the achievement of market authorizations for numerous rare endocrinology and hematology autoimmune programs. Maria-Cruz is currently working as the head of Allucent’s team of Rare Disease experts, integrating our cross functional expertise to provide strategic solutions that foster successful outcomes in rare disease clinical trials and ensure excellence in delivery.
Jessica K. Roberts, PhD – Director, Pharmacometrics
Jessica has worked in modeling and simulation for over 10 years with an emphasis on pediatric patients. With experience from the NICU and PICU at Primary Children’s Hospital and oncology protocols at St. Jude Children’s Research Hospital before becoming a consultant, Jessica has had the opportunity to contribute to studies in neonates, infants, and pediatric patients for the treatment of infectious diseases, pain management, oncology, and respiratory illnesses, to name a few. Her experience has focused on optimal sampling in pediatric patients for clinical trials as well as selection of the best dose for treatment in various pediatric age groups. During her career, Jessica has published over 35 research articles, invited editorials, invited reviews, and invited book chapters, and given many oral and poster presentations that have focused on drug development in neonate, infant, and pediatric patients.
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