Protecting Pediatric Patients: Advancing Drug Safety Through Pharmacovigilance

Written by: Jen Truong, Senior Medical Director and Zhiqiang Yan Associate Manager, Pharmacovigilance Operation, September 2025

Children Are Not “Small Adults”

In drug development and clinical practice, children are often misunderstood as merely requiring a scaled‑down version of adult doses. However, children’s bodies are not simply miniature models of adults. Their organs and metabolic functions are still developing, which makes the way they absorb, distribute, metabolize, and excrete drugs significantly different. For instance, newborns have immature liver and kidney functions, leading to lower drug clearance and a higher risk of drug accumulation and adverse effects^[1].

Therefore, even commonly used medicines may produce different efficacy and safety outcomes in pediatric patients. Children should never be treated as “small adults.” Instead, independent clinical evidence and tailored safety monitoring systems must be established^[2].

Current Challenges in Pediatric Drug Safety

Despite growing attention from global regulators, several challenges remain in pediatric drug use:

• Widespread off‑label use: According to WHO, more than 50% of pediatric medicines are prescribed off‑label^[1].
• Lack of child‑friendly formulations: Young patients often struggle with traditional tablets, while liquid formulations, orally disintegrating tablets, and chewables remain underdeveloped^[2].
• Low participation in clinical trials: Ethical concerns and recruitment difficulties limit pediatric trial enrollment, leaving gaps in safety and efficacy data^[3].
• Difficulty assessing long‑term risks: Some drug‑related effects may only appear years later, adding complexity to pediatric pharmacovigilance.

These challenges highlight the indispensable role of pharmacovigilance in safeguarding children’s health.

The Unique Value of Pharmacovigilance in Pediatrics

Pharmacovigilance focuses on identifying, assessing, and preventing drug‑related risks. For children, it plays a particularly crucial role:

• Bridging data gaps: Real‑world data (RWD) collected through pharmacovigilance can partially compensate for the limited data from pediatric trials^[2].
• Identifying pediatric‑specific adverse reactions: Children may experience adverse effects not seen in adults, Or have a paradoxical reactions, resulting in the opposite of the intended effect and robust monitoring systems help detect these differences.
• Optimizing treatment decisions: Long‑term monitoring and large‑scale data analysis provide clinicians with stronger evidence for safe prescribing^[3].

Key Measures to Build a Pediatric Drug Safety Framework

1. Proactive safety data collection: Pediatric‑specific risk assessments should be integrated into clinical trial design from the earliest stages, ensuring unique safety signals are captured promptly^[2].
2. Lifecycle monitoring: Pediatric drug safety must extend beyond trials, covering the entire lifecycle—from development to market use—to ensure continuous risk management^[1].
3. International regulatory alignment: Regulatory authorities worldwide have strengthened pediatric requirements:
   1. In Europe, the Paediatric Regulation (Regulation (EC) No 1901/2006) has been in force since 2007 to promote research and access to medicines for children.
   2. In the United States, two key laws apply: the Best Pharmaceuticals for Children Act (BPCA), which incentivizes pediatric studies, and the Pediatric Research Equity Act (PREA), which mandates pediatric data submission unless waived.
   3. In China, the National Medical Products Administration (NMPA) has issued official guidance such as the Guidance on Pharmaceutical Development of Medicines for Pediatric Use (Trial) to strengthen pediatric drug development and monitoring^[2-7].
   4. CROs with global expertise are uniquely positioned to help sponsors meet these diverse regulatory requirements, ensuring standardization and compliance across regions.
4. Empowering families and frontline clinicians: Because children often cannot clearly articulate discomfort, caregivers and healthcare providers serve as the first line of defense in detecting adverse reactions. Training and digital reporting tools enhance efficiency and ensure timely signal reporting^[1].

The Role of CROs in Advancing Pediatric Pharmacovigilance

For pharmaceutical companies, building a comprehensive pharmacovigilance system in‑house requires significant resources. This is where CROs can add value:

• Specialized expertise: Teams with pediatric clinical backgrounds can accurately identify and interpret complex safety data.
• Advanced technology platforms: AI and big data enable real‑time signal detection, accelerating adverse event analysis.
• Global regulatory compliance: Ensuring data and processes meet EMA, FDA, NMPA, and other international standards reduces compliance risks.
• Patient‑centered approach: Beyond scientific rigor, CROs focus on minimizing the burden on children and families, making safety monitoring more supportive and less intrusive.

Future Outlook: Toward Smarter, Collaborative Safety Systems

With the rise of digital health and real‑world evidence, pediatric pharmacovigilance is evolving rapidly:

• Digital monitoring tools: Wearables and remote monitoring can capture real‑time physiological responses after drug use^[3].
• AI‑powered signal detection: Artificial intelligence accelerates the analysis of vast datasets, improving both the speed and accuracy of adverse event identification.
• Cross‑sector collaboration: In the future, pharmaceutical companies, CROs, regulators, and academia will form closer networks, collectively strengthening pediatric drug safety.

Conclusion

Drug safety is not an optional add‑on—it is the foundation of responsible drug development and clinical practice. Children, as the most vulnerable population, deserve a dedicated, systematic pharmacovigilance framework. Through science, collaboration, and global regulatory alignment, we can build a stronger safety net for young patients.

Protecting the smallest patients means safeguarding the greatest future.

Sources

1. World Health Organization (WHO). Promoting safety of medicines for children. Geneva: WHO; 2007. https://www.who.int/publications/i/item/9789241563437
2. European Medicines Agency (EMA). Paediatric Regulation (Regulation (EC) No 1901/2006 on medicinal products for paediatric use). https://www.ema.europa.eu/en/human-regulatory-overview/paediatric-medicines-overview/paediatric-regulation
3. S. Food and Drug Administration (FDA). Pediatric Product Development – including Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA). https://www.fda.gov/drugs/development-resources/pediatric-product-development
4. National Medical Products Administration (NMPA), Center for Drug Evaluation. Guidance on Pharmaceutical Development of Medicines for Pediatric Use (Trial), Notice No. 67, 2020. https://www.cde.org.cn/main/news/viewInfoCommon/95102a5facaf8fd4430d0916a24eab53
5. National Medical Products Administration (NMPA), Center for Drug Evaluation. Working Procedures for Adding Pediatric Use Information to the Labeling of Marketed Drugs (Trial) and Its Implementation Rules, Notice No. 68, 2023. https://www.cde.org.cn/main/news/viewInfoCommon/511ef16b9b968571d1ab25c2b9b46610
6. National Medical Products Administration (NMPA), Center for Drug Evaluation. Technical Guidance for Safety Information Evaluation and Reporting in Pediatric Drug Clinical Trials (Trial), Notice No. 21, 2025. https://www.cde.org.cn/main/news/viewInfoCommon/d3762909c55ef21c7fc533d8ba826d40
7. National Medical Products Administration (NMPA), Center for Drug Evaluation. Working Norms for Expedited Review of Market Approval Applications for Innovative Drugs (Trial). https://www.cde.org.cn/main/news/viewInfoCommon/ace377c025ad4f2bbf94790673b2646e
8. Caidya official website – Pediatric Clinical Research. https://www.caidya.com/what-we-do/therapeutic-areas/pediatrics/
9. Caidya official website – Pharmacovigilance Services. https://www.caidya.com/what-we-do/services/pharmacovigilance/