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.
- PREA gives the FDA the authority to require pediatric studies requires pediatric studies for certain new medicines approved for use in adults where the indication for use in children would be comparable to that for adults, and produce formulations appropriate for children (e.g., liquid or chewable tablets).
- BPCA complements PREA requirements by providing sponsors with an incentive (6 months additional marketing exclusivity) to undertake voluntary pediatric studies at the request of FDA, many of which may not be required under PREA. BPCA has proven to be an effective incentive for stimulating pediatric drug development, especially in cases where PREA requirements do not apply (for example, for orphan diseases, including rare cancers).
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.
"Before BPCA and PREA became law, more than 80% of the drugs approved for adult use were being used in children, even though the safety and effectiveness had not been established in children. Today that number has been reduced to about 50%.” – Dr. Lynne Yao, FDA