Working together for new solutions to cancer in children

By: Margaret A. Hamburg, M.D. 

One of the greatest pleasures I have as FDA commissioner is the opportunity to meet with so many who are making a real difference in the world of health care, including researchers, doctors, drug industry leaders, foundations, and advocates for patients and families, including most recently the Alexandria Summit for Oncology. 

The group’s two-day meeting in New York City addressed a range of topics related to the challenges and advances in treatments for cancer and featured many of the leading scientists and researchers in the field, including my colleague Dr. Richard Pazdur, the director of FDA’s Office of Hematology and Oncology Products. 

Last night I had the chance to speak to the group about developments in treating pediatric cancer. I was joined by Nancy Goodman, a passionate and effective advocate, who is the founder of a patient advocacy group called Kids and Cancer. Nancy is helping to influence discussion on this topic, and her inspiring work gives enduring meaning to the life of her son Jacob, who died of cancer in 2009. 

Pediatric medicine is an area that has long faced historic obstacles, the result of the tension between our eagerness to respond as quickly as possible to treating and developing treatments for diseases in children, and our desire to protect children from potentially dangerous side effects, particularly in the early stages of research when effectiveness is not fully known. 

Today, we are in the midst of some exciting advances in the development of improved and better treatments for cancer that we expect can and will translate into new treatments for children with cancer. Scientific progress isn’t the only reason for these advances.  Regulators, researchers and industry are also thinking and acting in new ways that are allowing us to accelerate our investigation and approval process.  

Over the past 20 years we have evolved from a view that we must protect children from research, to a view that we must protect children through research, in order to assure their access to new and effective medications. Research studies are the only way to truly determine the safety and efficacy of medication in children and to avoid possible harm when children are given drugs approved only for adults. 

Recent laws have created new mechanisms to prioritize drug research and development for children, resulting in a dramatic increase in pediatric drug trials. We are already seeing progress, with more drug companies hiring pediatric experts, and some large pharmaceutical companies developing pediatric centers of excellence. 

FDA is working to promote earlier consideration and approval of treatments for children. But increased emphasis is also needed on discovery and development, which must come from the industry and the academic research community. The potential offered by our science has never been greater. We want to turn this potential into expanded treatments, new drug availability, and hopefully new cures. That’s why the enthusiasm and creativity this Summit generates is so promising. 

Margaret A. Hamburg, M.D., is the Commissioner of the Food and Drug Administration