Paediatric oncology is advancing through a combination of scientific innovation and regulatory evolution. A comparative analysis by Reggiardo et al. (2025) outlines how the US and EU have responded to unmet paediatric needs by modernizing drug development policies and incentives (Reggiardo et al., 2025).

Key Insights from the Study

  • More Approvals, Faster Pace in the US: From 2007 to 2024, the US approved 53 paediatric oncology drugs versus 35 in the EU. The US also had a significantly steeper upward trend post-2017, attributed to the enactment of the RACE for Children Act, which eliminated automatic waivers for rare diseases and introduced a mechanism-of-action (MoA) based requirement for paediatric development (Reggiardo et al., 2025; FDA, 2017).
  • Rising Non-Adult-Driven Therapies: In the EU, approvals for non-adult-driven indications increased significantly after 2018—from 34.1% to 60%—and a similar trend was seen in the US. These therapies target childhood-specific cancers rather than adapting adult products (Reggiardo et al., 2025).
  • Innovative Drugs Lead the Way: Targeted therapies and immunotherapies now dominate new approvals. In the EU, 71% of new drugs between 2018–2024 fall into these innovative categories. In the US, 75% of approvals were either targeted or immune-based agents, including five tissue-agnostic therapies that mark a major milestone in precision oncology (Reggiardo et al., 2025).
  • Greater Attention to Solid Tumours: The number of PIPs addressing solid tumours now exceeds those for haematological malignancies. This marks a reversal from historical neglect, although gaps remain in specific areas like neuroblastoma and bone tumours (Reggiardo et al., 2025).

Author Recommendations

The article recommends the following regulatory and policy actions:

  1. Formalise the MoA Approach in EU Law: Although EMA’s PDCO has applied an MoA approach in practice, this is not yet codified in EU law. Authors recommend embedding this as a legal requirement (Reggiardo et al., 2025).
  2. Introduce First-in-Child Incentives: The study supports proposals such as those from SIOPE to incentivise paediatric-specific development through first-in-child marketing authorisation benefits (Reggiardo et al., 2025).
  3. Avoid Repealing the Paediatric Regulation: The proposed repeal of the EU Paediatric Regulation, without parallel investment or replacement frameworks, is seen as risky. The current system has demonstrated measurable success and should be strengthened—not abandoned (Reggiardo et al., 2025).
  4. Encourage Regulatory Harmonisation and Cooperation: The authors stress the need for global alignment, particularly between EMA and FDA, to prevent unnecessary delays and disparities in access (Reggiardo et al., 2025).
  5. Support Digital and Precision Innovation: Emphasis is placed on integrating real-world data, genomics, and digital technologies into regulatory science, echoing trends that GMDP Academy covers in Module 8 (Reggiardo et al., 2025).

Why It Matters for GMDP Professionals

This paper reinforces the importance of early paediatric regulatory planning, cross-functional collaboration, and a firm understanding of evolving policy levers. Modules 4 (Clinical Trials), 5 (Regulatory Affairs), and 8 (Digital Technologies) at GMDP Academy offer direct training on these emerging requirements and tools.

Moving Forward: A Call for Strategic Action

The authors conclude that the field stands at a critical juncture. The US has shown that regulatory innovation like the RACE Act can accelerate approvals and shift market dynamics. Europe, with its extensive infrastructure and scientific base, risks falling behind if it dismantles rather than modernises its regulatory architecture.

For pharmaceutical professionals, this is not just a policy debate—it is a strategic imperative. The future of paediatric oncology depends on smart regulation, coordinated investment, and the leadership of well-trained professionals in development roles.

Get Involved

At GMDP Academy, we are committed to fostering the next generation of global leaders in pharmaceutical medicine. If your organization is looking to advance paediatric oncology expertise or align with the latest regulatory science developments:

🔹 Explore our Certification in Medicines Development (CMD) Program

🔹 Join specialized modules in Regulatory Affairs, Digital Technology, and Clinical Trials

🔹 Partner with us to upskill your team in line with emerging global standards

Visit gmdpacademy.org or contact admissions@gmdpacademy.org to learn more.

References

FDA. (2017). Research to Accelerate Cures and Equity for Children Act (RACE for Children Act). https://www.congress.gov/bill/115th-congress/house-bill/2430

Reggiardo, G., Natale, A., Santoro, N., Giannuzzi, V., Pansieri, C., Felisi, M., Bonifazi, D., & Ceci, A. (2025). Scientific and regulatory progress in advancing paediatric oncology drug development in the EU and in the US. Frontiers in Medicine, 12, 1642279. https://doi.org/10.3389/fmed.2025.1642279

Disclaimers

  • The material in these reviews is from various public open-access sources, meant for educational and informational purposes only
  • Any personal opinions expressed are those of only the author(s) and are not intended to represent the position of any organization(s)
  • No official support by any organization(s) has been provided or should be inferred