Policies to Foster Pediatric Drug Development
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
have been completed since PREA & BPCA reathorization in 2007
pediatric labeling changes
Since 1998 thanks to PREA and BPCA
have recived pediatric exclusivity under BPCA
Unique Research Challenges
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. A recent multi-stakeholder workshop highlighted the range of challenges facing pediatric researchers and explored ways to overcome obstacles, particularly in the oncology space. Learn more here.
Advancing Solutions Together
America’s biopharmaceutical companies are committed to advancing innovative treatment options for pediatric patients. Collaboration will be instrumental in harnessing innovative research approaches and addressing the scientific and operational challenges to conducting pediatric research.
Provisions within the 21st Century Cures Act, as well as the PDUFA VI agreement, will help accelerate pediatric medicine development. Learn more here.
Collaborative, pre-competitive initiatives can help address some of the identified scientific and operational challenges in pediatric oncology medicine development.
- In October 2016, Children’s Cause for Cancer Advocacy (CCCA), PhRMA, and BIO convened a workshop that brought together, for the first time, the full spectrum of key pediatric oncology stakeholders to address some of the identified scientific and operational challenges in pediatric oncology medicine development. (These included 15 biopharmaceutical companies, patient and provider advocacy groups, FDA, NIH, and some of the country’s leading pediatric oncologists). The workshop participants resolved to work together to pursue the creation of a pre-competitive public-private partnership to explore potential approaches to solving identified challenges.
- A newly announced pediatric clinical trial network, the Institute for Advanced Clinical Trials for Children (I-ACT), seeks to improve the quality and timely completion of global pediatric studies to address the pediatric evidence gap.