Challenges in Pediatric Clinical Trials and How to Overcome Them

Why Pediatric Drug Development Lagged Behind

Pediatric drug development is hampered by many factors that have historically made it a neglected area within the pharmaceutical industry. These challenges are diverse in nature- spanning ethical, economic, operational, and scientific domains, and together they create significant barriers for sponsors aiming to design, conduct, and complete a pediatric clinical trial.

From an economic perspective, the limited commercial return associated with small pediatric markets has traditionally discouraged investment. Scientifically, the need for tailored formulations, age-appropriate dosing, and subgroup-specific endpoints increases the complexity of study design. And, ethically enrolling children in clinical research demands heightened scrutiny, specialized consent procedures, and strong oversight to ensure participant protection. Furthermore, recruitment remains difficult on the operational level due to smaller eligible populations and the logistical challenges of involving multiple care environments, from hospitals to specialized pediatric centers.

Regulation and Incentives

The introduction of international and regional frameworks, especially the EU Pediatric Regulation, marked a pivotal shift in how pediatric research is conducted. These policies established clearer ethical and procedural standards while offering tangible incentives to sponsors who invested in developing medicines for children.

Aligned with the International Council for Harmonisation (ICH) guidelines, these reforms harmonized regulatory expectations across the EU, USA, and Japan, introducing the Pediatric Investigation Plan (PIP) as a cornerstone of child-focused drug development. The PIP ensures that sponsors address pediatric needs proactively during the development process.

Growth and Gap

Over the past decade, the number of pediatric clinical trials has increased substantially, underscoring the success of these regulatory measures. Nevertheless, the gap between pediatric and adult randomized controlled trials remains wide across most therapeutic areas.

Economic, ethical, technological, geographical, and cultural factors continue to influence pediatric drug development and remain obstacles to efficient trial execution. These realities highlight the ongoing need for coordinated strategies that promote both scientific rigor and operational feasibility.

Keys to Successful Pediatric Trial Preparedness

To meet the growing demand for pediatric studies and ensure alignment with approved PIPs, sponsors and CROs must prioritize trial preparedness, a comprehensive approach that integrates ethical, scientific, and operational excellence.

Key success factors include:

1. Robust study design tailored to developmental stages and physiological differences across age groups.

2. Operational excellence, with trained and experienced teams managing complex logistics.

3. Ethical vigilance, ensuring that participant welfare remains the top priority.

4. Cultural and geographic sensitivity, promoting recruitment and engagement across diverse regions.

5. Effective communication with patients, caregivers, and healthcare professionals to foster trust and compliance.

Lessons from the DEEP Project

The Deferiprone Evaluation in Pediatrics (DEEP) project exemplifies the practical challenges and innovative solutions that characterize multinational pediatric clinical research. This initiative demonstrated the importance of early planning, cross-border collaboration, and adaptive trial management in overcoming logistical and regulatory hurdles, eventually advancing safe and effective treatment options for children.

Challenges in Pediatric Clinical Trials and How to Overcome Them

Pediatric patients have long been treated as ‘small adults’ in clinical research, a notion that shaped both drug development and clinical practice. Prior to the introduction of international regulations and incentives, encouraging child-specific drug development, pediatric populations routinely received off-label treatments that lacked both efficacy and safety data for their specific age groups. These drugs were often tested only in adults, leaving children exposed to uncertain benefits and unassessed risks. Not only this, but the pediatric population has also been on the verge of many other factors that challenged the suitability of drugs and treatment meant for them.

This blog throws light on the regulatory landscapes, gaps in research, and what can be expected to streamline the research culture concerned with these ‘small adults’.

Defining the Pediatric Population

Understanding and accurately defining the pediatric population is essential for sound study design. International guidelines subdivide this population into four distinct groups, each with unique clinical and developmental characteristics:

1. Pre-term and term neonates (0–27 days)

2. Infants (1–23 months)

3. Children (2–11 years)

4. Adolescents (12–18 years)

Each subgroup requires specific pharmacological considerations, ethical safeguards, and methodological adaptations.

How RCS Supports Pediatric Research

Pediatric clinical research has evolved from a neglected niche into a vital and dynamic field within global drug development. Yet, the road ahead requires continued collaboration between regulators, sponsors, CROs, and healthcare institutions.

RCS recognizes that pediatric clinical development is more than a regulatory obligation, it is a scientific, ethical, and operational commitment to a vulnerable population that deserves evidence-based care.

Our multidisciplinary teams bring together expertise in pediatric protocol design, regulatory strategy, patient engagement, and site management to support sponsors through every phase of pediatric drug development. From navigating complex regulatory submissions to managing global recruitment networks and tailoring communications for patients and families, RCS provides an end-to-end bespoke partnership because every child needs access to safe, effective, and appropriately studied medicines.