Drug Development in Pediatrics
Children are not simply “small adults,” yet many therapies used in pediatrics were historically developed and tested primarily in adults. This session on Drug Development in Pediatrics explores how science, regulation, and ethics are evolving to produce medicines specifically studied in children, with formulations, dosing, and safety profiles suited to their needs. Participants will consider the full pipeline—from preclinical research and age-appropriate trial design to long-term safety monitoring and post-marketing surveillance.
Clinicians, researchers, and regulators who attend Pediatrics Conference are often seeking clarity on how to balance innovation with protection. In this session, you will review regulatory frameworks that encourage pediatric investigation, including incentives and obligations for companies to generate pediatric data. Case examples will highlight successful pediatric trials in areas such as oncology, rare diseases, infectious diseases, and chronic inflammatory conditions, emphasising lessons learned about endpoints, recruitment, assent, and consent.
A key theme is building robust pediatric drug development pathways that reflect the diversity of childhood—different ages, developmental stages, and co-morbidities. Participants will discuss formulation challenges (such as palatability, dosing flexibility, and excipient safety), pharmacokinetic and pharmacodynamic differences, and strategies for model-informed dosing. The session will also cover pragmatic trials, real-world evidence, and use of registries to complement traditional randomised designs, particularly for rare diseases where large trials are not feasible.
Ethical issues—including risk–benefit assessment, involvement of children and families in trial design, and communication of uncertain long-term effects—will be woven throughout. Attendees will also reflect on global equity: how to ensure that children in low- and middle-income countries benefit from advances and are not only viewed as trial populations without local access. By the end, participants will have a clearer understanding of how to evaluate pediatric drug evidence, contribute to research, and advocate for safer, more effective medicines for children.
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Submit Your Abstract Here →Core Themes in Drug Development in Pediatrics
Regulatory and ethical foundations
- Reviewing frameworks that promote pediatric studies while safeguarding child participants.
- Considering how assent, parental consent, and ongoing communication support ethical trial participation.
Age-appropriate formulations and dosing
- Designing medicines that are palatable, flexible in dosing, and safe in terms of excipients and administration routes.
- Using developmental pharmacology and modelling to determine doses that balance efficacy and safety across age groups.
Trial design and endpoints
- Selecting endpoints and outcome measures that matter to children and families as well as regulators.
- Incorporating adaptive designs, real-world data, and novel trial models to answer questions efficiently.
Post-approval monitoring and access
- Tracking long-term safety, off-label use, and real-world effectiveness after a drug reaches the market.
- Advocating for equitable access so that children in different regions benefit from newly developed treatments.
Practice Insights and Collaboration Opportunities
Engaging clinical teams in research
Encouraging pediatric services to participate in trials and observational studies with appropriate support.
Partnering with families and youth
Including children, adolescents, and caregivers in shaping trial design, outcomes, and information materials.
Interpreting pediatric evidence at the bedside
Translating trial results into practical prescribing decisions, monitoring plans, and shared decision-making.
Addressing off-label and unlicensed use
Documenting and reviewing off-label prescribing while advocating for formal studies when evidence is weak.
Building international networks
Joining collaborative research groups to tackle rare conditions and share data across borders.
Promoting transparency and communication
Ensuring families receive clear information about benefits, risks, and uncertainties throughout the drug lifecycle.
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