The NIH's BPCA efforts aim to:
- Identify drugs that still need to be studied in children due to lack of dosing, safety, or efficacy data
- Sponsor clinical trials for those drugs
- Identify, through a competitive peer-review process, the best sites to conduct those studies
- Carefully monitor studies in children
- Submit data from the clinical studies to the U.S. Food and Drug Administration (FDA) for consideration of label modification
- Make the study data available to researchers and the public
Before prescription drugs are approved by the FDA for clinical use, federal legislation and regulations require they be tested:
- For safety and efficacy for use in a specific population
- At a specific dosage
- For a specific time period
The FDA's Pediatric Rule of 1994 allowed the labeling of drugs for pediatric use based on:
- Extrapolation of efficacy in adults
- Additional pharmacokinetics (how a drug affects the body), pharmacodynamics (how the body affects a drug), and safety studies in pediatric patients, if the course of the disease and the response to the drug are similar in children as in adults.
Only a small number of well-designed and well-conducted studies resulted from this rule, even though it was designed to improve pediatric labeling.
Additional legislation, passed in 1997 as part of the FDA Modernization Act, provided extra incentive to pharmaceutical companies for pediatric testing by granting an additional 6-month exclusivity period for marketing. As a result, many drugs have and continue to receive pediatric labeling under this provision.
Use of drugs outside of the prescribed label indications is considered "off-label" use. Far more drugs have been approved for clinical use in adults than for pediatric populations, which leads to a high level of "off-label" use of drugs in children who need them for various medical conditions.
As a result, the majority of drugs used to treat children are used off-label, without an adequate understanding of appropriate dose, safety, or efficacy. The BPCA's role is to address these knowledge gaps.