Conversations on Cancer – Pediatric Cancers: Navigating the Challenges Together [Video]

In conjunction with the European Medicines Agency (EMA), the FDA Oncology Center of Excellence (OCE) will hold 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.

Each year, over 15,000 children, adolescents, and young adults 0-19 years of age in the U.S. and 300,000 globally are diagnosed with cancer. Although the survival rate for pediatric cancer is approximately 85%, it remains the most common cause of death by disease in children. There are approximately 500,000 survivors living in the U.S. today, many of whom suffer from late effects of treatment that adversely impact their quality of life. Childhood cancers are rare and have high survival rates but remain the first cause of death from disease in children and adolescents. In 2022, a total of 13 800 new cancer cases were estimated in children and adolescents (aged 0-19 years) and 2 144 were estimated to die from this disease. Adolescents and very small children are most affected. The most common childhood cancers are leukaemia, lymphomas and brain tumours. Incidence, mortality rates, and survival percentages vary among EU countries and regions.

For these patients and their families, treatment is often arduous, prolonged, and fraught with challenges. The need to make challenging decisions can begin at the time of diagnosis and continue throughout treatment and into survivorship, and this decision-making is often shared between families or caregivers, the patient, and their healthcare providers. In the U.S. and Western Europe, where the concept of autonomy in medical decision-making is prioritized, pediatric assent is a critical component of informed consent and shared determination.

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 will feature speakers with a range of perspectives on the complex and challenging decisions related to clinical care, research, and regulatory decision making in pediatric oncology. This Conversation on Cancer forum is an international collaboration with our colleagues at the EMA and aims to highlight the experiences of pediatric patients with cancer, their families, their doctors, and regulators.