2023 - 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.