Pediatric Clinical Trials: Tackling Clinical and Therapeutic Development Challenges Head-On

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

Having recognized this, Pediatric and Rare diseases legislation adopted in US, EU and in many other countries have addressed the need for drug development in these neglected populations and have had a substantial impact on the overall development of new medicines. However, while progress has been made many conditions are still without approved treatments and healthcare professionals relying on off-label options or inadequate studies.

By conducting well-designed and ethically conducted pediatric clinical trials, the safety and effectiveness of medications for the youngest patients can be improved and their overall healthcare outcomes enhanced.

Join this webinar to learn about shaping the future of therapeutic clinical development in pediatric clinical trials.

Speakers

Richard Vesely, Vice President, Regulatory Strategy, Allucent

With over 12 years of experience at the European Medicines Agency as Pediatric Coordinator and Head of Office for Rheumatology, Immunology, Gastroenterology and Respiratory Diseases, Richard Vesely is an expert in regulatory procedures in the European Union. He led the team in evaluating marketing authorizations, scientific advice and pediatric and orphan designation procedures. With 30 years of clinical and research experience as a pediatrician, pediatric immunologist and rheumatologist, Richard has expertise in the development of paediatric investigation plans and initial pediatric study plans.

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Vanessa Beddo, PhD, Vice President, Biostatistical Consulting, Allucent

Vanessa Beddo 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.

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Maria-Cruz Morillo, Global Therapeutic Operations Lead, Rare Diseases, Allucent

Maria-Cruz Morillo, MS Pharmacy, has a strong background in drug development with 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 their cross functional expertise to provide strategic solutions that foster successful outcomes in rare disease clinical trials and ensure excellence in delivery.

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Jessica K. Roberts, Director, Pharmacometrics, Allucent

Jessica Roberts 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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