Guest Blog–Conversations on Cancer – Pediatric Cancers: Navigating the Challenges Together

In conjunction with the European Medicines Agency (EMA), the FDA Oncology Center of Excellence (OCE) held a one-hour Conversations on Cancer public panel discussion on November 19, 2024, to examine an array of challenging decisions faced by members of the pediatric oncology community. The panel discussion featured current CAC2 Board Member Nicole Scobie (Zoe4Life) and past CAC2 Board Member Donna Ludwinski (Solving Kids’ Cancer).

Because of the rarity of cancers arising in childhood and adolescence and high unmet medical need, pediatric oncology drug development is inherently complex. As a result, pediatric oncology clinician investigators are faced with challenges related to clinical trial design and conduct as well as specific treatment decisions for their patients. Regulatory decision-making also typically requires careful, nuanced consideration of the legal requirements to develop and standards for demonstration of safety and efficacy in context of small patient numbers.

The panel featured speakers with a range of perspectives on the complex and challenging decisions related to clinical care, research, and regulatory decision making in pediatric oncology. The conversation was an international collaboration between FDA and EMA and highlighted the experiences of pediatric patients with cancer, their families, their doctors, and regulators.

The goal of this panel discussion was to foster a better understanding of:

• patient, parent, clinician-investigator, and regulator perspectives on the challenges associated with all aspects of pediatric cancer treatment and product development.
• how decisions made by patients and their parents impact their day-to-day lives.
• types of decisions made by clinicians and investigators and their perspectives on shared decision-making.
• how regulators approach difficult decisions related to trial design, conduct, and review of applications for approval of new medical products to treat pediatric cancers.