The innovative biopharmaceutical industry is firmly committed to conducting pediatric research, and is making great strides against pediatric illnesses. Recent research has provided important new dosing, safety and efficacy information in pediatric populations that is changing the treatment landscape across a range of serious and life-threatening diseases, including:
Asthma: Pediatric patients age 6 to 11 now have a therapy option that addresses severe eosinophilic asthma, a complex and challenging condition to treat, which was previously only approved for use in children age 12 and older. This breakthrough is a significant step forward, as asthma is the third-ranking cause of hospitalization among children younger than 15.
Type 2 Diabetes: The first non-insulin drug approved to treat type 2 diabetes in pediatric patients is now available. The medicine enhances the incretin system, a natural body system that helps to regulate glucose, and improves blood sugar control.
Peanut Allergy: A new oral immunotherapy designed to reduce the incidence and severity of allergic reactions due to accidental peanut exposure is now available for children with peanut allergy. The therapy delivers a controlled daily dose of peanut protein that is gradually increased over months to build tolerance in the immune system’s overreaction to peanuts.
HIV/AIDS: New combination therapies are now available for pediatric patients, reducing the complexity of treatment for this devastating disease by offering options that can be taken once a day in a fixed-dose pill.
A robust drug development pipeline offers tremendous promise for the future. Currently there are over 2,100 industry-sponsored pediatric clinical trials underway, involving 1.2 million pediatric patients across a variety of therapeutic areas, including diseases where there is significant unmet need, such infectious diseases, neurologic conditions, genetic disorders, and several forms of cancer.
Prior to the passage of the Food and Drug Administration Modernization Act (FDAMA) in 1997, there was growing concern that many FDA-approved drugs had not been clinically tested in children, resulting in inadequate or unavailable information on dosing, safety, efficacy and side effects. Because medicines that may work one way in adults may not work the same way in children, it is important that medicines intended for children are studied in children.
The need for pediatric-specific information in drug labeling prompted action, leading to the passage of two laws that address the study of drugs in pediatric populations: the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). PREA and BPCA work together to foster pediatric drug development, providing previously unavailable information on dosing, safety, efficacy, and side effects.
Made permanent with large bipartisan support in 2012, PREA and BPCA have been key drivers of pediatric research, generating important safety and efficacy information on the use of medicines in children. This balanced approach has driven research on innovative medicines in pediatric patients, resulted in more treatment options for children, and has greatly advanced children’s medical care. Learn more here.
completed since the first temporary reauthorization of BPCA and PREA in 2007
reflecting pediatric information made since 2007
pediatric clinical trials underway worldwide
Developing medicines for pediatric diseases poses unique scientific and operational challenges. Biopharmaceutical companies are committed to working with all stakeholders to combat the issues that prevent further research and results.
America’s biopharmaceutical companies are committed to advancing innovative treatment options for pediatric patients. Collaboration is instrumental to harness innovative research approaches and address the scientific and operational challenges to conduct pediatric research.
Collaborative, pre-competitive initiatives can help address some of the identified scientific and operational challenges in pediatric oncology medicine development.