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
Webinar Key Points Will Include:
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
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