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A regulatory preview into pediatric drug development

Updated: Apr 2

Pediatric drug development is a crucial area of pharmaceutical research that addresses the unique medical needs of children. However, it presents several challenges, including logistical difficulties in conducting clinical trials, the need for age-appropriate formulations, and the complexity of assessing outcomes in a rapidly changing population. Historically, these challenges have led to a need for pediatric-specific data and formulations, resulting in a reliance on off-label use of adult medications, which may not be safe or effective for children.

Recognizing the importance of encouraging drug development for pediatric populations, the US government has implemented various legislative measures to provide incentives for pharmaceutical companies. The Food and Drug Administration Modernization Act (FDAMA) of 1997 was significant because it offered six months of additional marketing exclusivity for medications tested on children, as the FDA requested. The Best Pharmaceuticals for Children Act (BPCA), which extended patent exclusivity and promoted voluntary pediatric studies, further reinforced this. Currently, vouchers such as the Rare Pediatric Disease Designation (under Section 529 of the Federal Food, Drug, and Cosmetic Act (FD&C Act)) allow for priority review from the FDA. An example of a medication under this designation would be the cancer medication Kymriah (tisagenlecleucel).

To complement these incentives, the Pediatric Research Equity Act (PREA) of 2003 introduced a requirement for pharmaceutical companies to test new drugs likely to be used in children in pediatric populations, with provisions for waivers and deferrals. BPCA and PREA have become permanent fixtures in the regulatory landscape, demonstrating a commitment to pediatric drug development.

To give context to why subsidies should be provided, children have vastly different physiologies than adults, leading to altered pharmacokinetic and pharmacodynamic profiles. Thus, the standard clinical trial design may not effectively cover children, warranting further resources from companies to study medications' effects in pediatric populations effectively. This has been addressed in the Pediatric Drugs Act of 2023, targeting rare diseases that affect children who lack effective therapies. Companies producing orphan medications for adults under the act could be required to perform pediatric clinical trials, as stated in the 2023 act: "Congress previously passed the RACE for Children Act, which lifted the PREA orphan drug exemption to allow studies for some orphan drugs for cancer...The Innovation in Pediatric Drugs Act would amend PREA to lift its orphan exemption, ensuring that children with rare diseases can fully benefit fully from the pediatric research requirements."

Despite these efforts, challenges remain. A McKinsey Center for Government report noted that while market exclusivity has driven significant revenue for the industry, the benefits have primarily accrued to blockbuster drugs and larger pharmaceutical companies. Smaller companies may still find the risks outweigh the potential rewards. Additionally, it has been relatively limited because adult drug development frequently drives the development of rare pediatric diseases.

Further subsidies have been introduced to address these gaps, such as the priority review vouchers granted through the 2012 Creating Hope Act for rare pediatric disease drug development. However, more targeted incentives may be needed, particularly for neglected subsegments of the pediatric population, such as neonates and adolescents, and for drug categories like biologics and generics that currently fall outside the scope of BPCA incentives.

Similar incentives are available in Europe, with extensions of supplementary protection certificates for medicines authorized by pediatric studies. The European Medicines Agency (EMA) also provides free advice and assistance for pediatric drug development questions, further supporting this crucial area of research.

In conclusion, while significant strides have been made in stimulating pediatric drug development through a combination of subsidies and regulatory requirements, ongoing challenges highlight the need for continued innovation and adaptation in incentives and policies. Addressing these challenges is essential to ensuring that children, future leaders, and scientists can access safe and effective medications tailored to their needs.



Commissioner O of the. Rare Pediatric Disease (RPD) Designation and Voucher Programs. FDA. Published October 13, 2021.

Innovation In Pediatric Drugs Act of 2023. Children’s Cancer Cause. Accessed March 7, 2024.

‌Institute of Medicine (US) Forum on Drug Discovery D. Summary. Published 2008. Accessed March 7, 2024.

‌Office of the Commissioner. Rare Pediatric Disease Priority Review Vouchers. U.S. Food and Drug Administration. Published 2019.

Spadoni C. Pediatric Drug Development: Challenges and Opportunities. Current Therapeutic Research. Published online December 2018. doi:

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