The Act in Action
The implementation of the Best Pharmaceuticals for Children Act (BPCA) at the National Institutes of Health (NIH) is intended to:
- Identify drugs that need to be studied in children
- Design 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 evaluation in consideration of label modification
- Make the study data available to investigators and the public
The BPCA establishes a process for studying both on-patent and off-patent drugs for use in pediatric populations, and for improving pediatric therapeutics through collaboration on scientific investigation, clinical study design, weight of evidence, and ethical and labeling issues.
Improving Knowledge About On-Patent Drugs
These studies involve on-patent drugs that without adequate clinical data to guide their use, are being prescribed off-label for pediatric patients.
FDA requests information from the manufacturer of on-patent drugs about safety, efficacy, and appropriate dosing for pediatric populations. In regard to on-patent drugs, BPCA:
- Extends exclusivity if the necessary requested studies are conducted by the manufacturer
- Refers studies to the Foundation for the NIH (FNIH) if the manufacturer declines to do the study
- Funding for studies may come completely or in part from FNIH.
- Funding from industry or private individuals can be donated to FNIH (501c3 status).
- Currently, FNIH has approximately $3.5 million for such studies.
On-Patent Drugs Referred to the Foundation for the NIH
FNIH provides funds to NICHD for pharmacological studies of on-patent drugs, if the drug makers decline to conduct those studies. To do so, the specific NIH Institute or Center with the appropriate expertise works with NICHD to develop a request for proposal (RFP). These studies involve on-patent drugs that without adequate clinical data to guide their use, are being prescribed off-label for pediatric patients.
As of November 2005, the following on-patent drugs have been referred to FNIH for study:
- Baclofen
- Bupropion
- Dexrazoxane
- Eletriptan
- Hydroxyurea
- Morphine
- Sevelamer
- Zonisamide
FNIH is considering the cost estimates and the drug indications to determine whether or not to proceed with studies on these drugs.
BPCA Activities for Off-Patent Drugs
Under the authority of BPCA, NICHD:
- Establishes a priority list (updated at least annually) of off-patent drugs and indications that most urgently require study in pediatric populations
- Works collaboratively with FDA and relevant Institutes within NIH to determine the clinical data needed to improve pediatric therapeutics (such data are described in the Written Request, FDA's formal mechanism for notifying drug manufacturers that additional data are needed, and ascertaining if the manufacturer is interested in conducting the studies)
- Develops the clinical trial design with appropriate Institutes which is published in a request for proposal (RFP) and advertised to the public in the Federal Business Opportunities or FedBizOpps (proposals submitted in response to the RFPs are evaluated by outside experts through the standard NIH peer-review process; visit http://grants1.nih.gov/grants/peer/peer.htm for more information about the peer-review process)
- Negotiates and awards contracts
- Develops Investigational New Drug (IND) applications
- Monitors clinical trials
- Submits data to FDA for evaluation
- Makes clinical trial data publicly available
NICHD has responsibility for approximately 25 percent of BPCA funding from its annual budget. Furthermore, NICHD is responsible for organizing study design teams with FDA and other NIH Institutes for its sponsored studies. Many NIH Institutes share funding for these studies, including:
- National Cancer Institute
- National Heart, Lung, and Blood Institute
- National Institute of Dental and Craniofacial Research
- National Institute of Diabetes and Digestive and Kidney Diseases
- National Institute of Neurological Disorders and Stroke
- National Institute of Allergy and Infectious Diseases
- Eunice Kennedy Shriver National Institute of Child Health and Human Development
- National Eye Institute
- National Institute of Environmental Health Sciences
- National Institute of Arthritis and Musculoskeletal and Skin Diseases
- National Institute on Deafness and Other Communication Disorders
- National Institute of Mental Health
- National Institute on Drug Abuse
- National Institute on Alcohol Abuse and Alcoholism
- National Institute of Nursing Research
- National Human Genome Research Institute
- National Center for Research Resources
- National Center for Complementary and Alternative Medicine
- John E. Fogarty International Center
Lastly, NICHD has primary responsibility for administering, contracting, and monitoring studies to develop IND data for potential label modification and drafting label modifications for specific ages and indications for the studies conducted under BPCA.