By: Dionna Green, M.D., Director, Office of Pediatric Therapeutics, Lynne Yao, M.D., Director, Division of Pediatrics and Maternal Health, Center for Drug Evaluation and Research, and Barbara Buch, M.D., Associate Director for Medicine, Center for Biologics Evaluation and Research
Increasing the availability of safe and effective medicines for children is a key priority for the U.S. Food and Drug Administration. Prior to the late 1990s, very few medicines were specifically approved for use in children because of ethical concerns about including children in clinical trials. This resulted in doctors often using their professional judgement in recommending use of medicines approved for use in adults that had not been reviewed by the FDA for safety and effectiveness in children if he or she believed that the medicine was the best available treatment option for the child.
To address this critical public health issue, the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were enacted by Congress in 2002 and 2003, respectively, to incentivize and require pediatric studies under certain circumstances. The FDA has worked to develop the required infrastructure, operational improvements, regulatory science research and policies to implement these laws, resulting in increased availability of medicines and vaccines for children.
FDA Reaches Milestone for Pediatric Labeling Changes
The FDA recently reached a milestone of 1,000 medicines that include evidence-based pediatric information in the product labeling. Some of these medicines have been approved by the FDA for children and others have been updated to include additional information to help health care professionals make informed decisions when treating children. This includes medicines to treat health conditions such as certain infectious diseases, cancer and neurologic diseases that previously had no approved treatments for children. This accomplishment reflects the efforts of the FDA, federal partners, industry, researchers, patients, families, advocacy groups and numerous other stakeholders who helped inform the current approach to developing medicines for children.
Children and their caregivers have played a pivotal role in advancing pediatric medical product development. In addition to the hundreds of thousands of children who have participated in clinical trials over the last 20 years, children and their families have also provided invaluable advice and perspectives during the development process for many medicines.
As we look to the future, the FDA will continue collaborating with stakeholders to advance treatment options for children in several key areas:
1. Improving timeliness of pediatric medical product development.
Making new safe and effective treatment options available for children sooner is a high priority for future pediatric medical product development. This includes strategies such as earlier collaboration with stakeholders in the development process to plan for pediatric studies and to work with stakeholders to promote innovative approaches to facilitate pediatric medical product development.
One example of the FDA’s recent progress in expediting medicines for children is the draft guidance, “Pediatric Extrapolation,” that provides recommendations for leveraging available data (e.g., from adult trials), when appropriate, to support the development of medicines for children.
2. Engaging patients and their families in clinical research.
The FDA will continue to promote access to clinical trials for all children when appropriate and safeguard children participating in clinical trials.
The FDA remains committed to encouraging diversity in clinical trial participation, including the enrollment of children from underrepresented minority groups in clinical trials and will continue to engage with stakeholders to encourage this important goal. The agency engages patients and caregivers to ensure that their perspectives are incorporated throughout the medical product development process. These efforts will help promote clinical research to benefit children of all ages and backgrounds.
3. Collaborating with international regulators.
The FDA engages with international regulators to help assure children around the world participate in clinical trials that are well designed and meet international regulatory, ethical and safety standards. The FDA’s international collaboration efforts will continue to help improve pediatric medical product development globally by increasing the efficiency of clinical trial enrollment, expanding information and knowledge-sharing with global experts and by aiming to ensure children are not exposed to unnecessary (e.g., duplicative) trials.
The FDA is committed to taking steps in these key areas to promote clinical research for children, particularly for pediatric populations that face unique challenges such as newborns, children with rare diseases and children with cancer. The agency’s continued collaboration with patients and their families, industry, international regulators, and the pediatric clinical research community will help shape the future of pediatric medical product development.