Clinical trials are not one size fits all. We design and deliver pediatric studies geared to the specific needs of young patients and their caregivers, so you can bring your therapies to the children who need them at the speed of science.
From gamification strategies to protecting patients’ rights, we give pediatrics research the same creativity and care that we bring to our own families.
Senior Medical Director
Jennifer Troung, MD, MPH, earned her medical doctorate degree in pediatrics and is board certified in medical affairs. She offers 20+ years of experience in clinical pediatrics and pediatric hematology/oncology as she helps develop clinical and scientific strategies with a focus on developmental physiology, endpoints, extrapolation, and patient safety.
Vice President, Rare Disease and Pediatrics
Jonathan is a seasoned operations leader with expertise in site networks, caregiver burden, retention, and logistics in pediatric clinical trials. Drawing on nearly three decades of experience in the pharmaceutical and clinical research industries, he guides patient- and caregiver-centric clinical strategies that balance operational rigor with patient needs in rare disease and pediatric trials.
Jennifer Truong, MD - Senior Medical Director
Why take risks when Caidya can help you:
We consider endpoints, assessments, PK/PD, and formulation/device and palatability constraints for child-friendly dosage forms.
We manage consent and address caregiver constraints carefully to recruit and retain patients from limited and geographically dispersed populations.
We select countries and sites aligned with regulatory plans and local data expectations, while targeting diverse, globally-relevant populations to maximize data quality while minimizing patient and caregiver burden.
We help optimize your treatment decisions through long‑term monitoring and large‑scale data analysis, generating stronger evidence for safe pediatric treatments.
With our team, you can:
Designing pediatric cohorts begins with aligning to developmental physiology and regulatory expectations from agencies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Rather than using arbitrary age cutoffs, cohorts are typically structured across neonates, infants, children, and adolescents to reflect differences in organ maturation and disease presentation. From a study design perspective, staggered enrollment is commonly used, starting with older children to establish safety before progressing to younger groups. Operationally, this means we often select sites capable of handling age-specific procedures, such as pain management techniques that are better suited to younger patients or specialized imaging.
Adult-to-pediatric extrapolation is acceptable when the disease course and response to therapy are sufficiently similar across age groups. Regulatory guidance, including that from the International Council for Harmonisation (ICH) of Technical Requirements for Pharmaceuticals for Human Use, supports partial or full extrapolation when justified. This approach relies on pharmacokinetic and pharmacodynamic bridging, exposure-response modeling, and supportive safety data in pediatric populations. From a strategic standpoint, extrapolation can significantly reduce development burden, but it does not eliminate the need for pediatric-specific safety data or careful operational planning.
Outcome measures in pediatric studies must be age-appropriate and developmentally sensitive, unlike adult studies, which typically rely on more standardized endpoints. Endpoints must be tailored to the child’s developmental stage while remaining clinically meaningful. In younger children and infants, endpoints often rely on observer-based measures or biomarkers, whereas older children and adolescents can perform functional assessments similar to adults. Incorporating caregiver-reported outcomes is essential, as they provide insight into real-world functional benefit. Choosing endpoints that are both measurable and meaningful requires close integration of study design and patient engagement strategies.
Dosing strategies in pediatric trials typically rely on weight-based or body-surface-area–based approaches, supported by population pharmacokinetic (PK) modeling. However, formulation development is equally critical. Palatability can directly impact adherence, so children often require age-appropriate formulations such as liquids, dispersible tablets, or mini-tablets. Taste masking and tangible rewards such as sticker charts can help as well. Device considerations, particularly for therapies administered by caregivers, must also be addressed early. From an operational standpoint, delays in formulation or device readiness are a common source of program setbacks, underscoring the need for early integration of clinical pharmacology and manufacturing planning.
Enrollment challenges in pediatric trials stem from small patient populations, parental concerns, and logistical burdens. Operationally, success depends on identifying experienced pediatric sites and leveraging patient advocacy networks. From a patient and caregiver engagement perspective, mitigating these barriers requires transparent communication, flexible scheduling, and the incorporation of decentralized trial elements such as telemedicine or home visits. Reducing logistical friction is often as important as scientific design in achieving recruitment targets.
Consent and assent processes must align with regional regulatory requirements while remaining sensitive to family dynamics. In some regions, particularly under frameworks guided by the EMA, consent from both parents may be required. Children who are capable must provide assent and materials should be adapted to their level of understanding. Operationally, sites must be trained to handle complex scenarios such as divorced parents or legal guardianship, and processes must be in place for re-consenting participants as they reach the age of majority. Clear, child-friendly communications are essential to building trust.
Reducing burden is central to both recruitment and retention. From an operational perspective, hybrid trial designs that incorporate telehealth, local testing, and flexible scheduling can significantly reduce disruption to school and work. Patient and caregiver engagement strategies should prioritize convenience, including travel support and reimbursement. Minimizing visit frequency and aligning study activities with daily routines can make participation more feasible, particularly for families balancing multiple responsibilities.
Safety surveillance in pediatric trials extends beyond standard adverse event monitoring to include growth, development, and neurocognitive outcomes. For advanced therapies, long-term follow-up may extend for many years to assess durability and late-emerging risks. Regulatory authorities such as the FDA often require extended follow-up or post-marketing pediatric studies. Operationally, this necessitates early planning for long-term engagement, including the use of registries and digital tools to maintain contact with participants over time.
Global pediatric development is shaped by differing regulatory requirements, including Pediatric Study Plans in the United States and Pediatric Investigation Plans in Europe through the EMA. These frameworks differ in timing and expectations, which can complicate multi-regional trials. A successful strategy typically involves developing a harmonized global protocol with region-specific adaptations, promoting compliance while maintaining operational efficiency across geographies.
Pediatric studies most often discontinue early due to slow recruitment, unrealistic feasibility assumptions, formulation or device challenges, safety concerns, or endpoints that are not practical or meaningful. Reducing these risks requires early regulatory alignment, robust feasibility assessments, and thoughtful study design, including adaptive approaches where appropriate. Operational excellence at experienced pediatric sites, combined with strong patient and caregiver engagement, is critical to maintaining momentum and reaching study completion.