BPCA Clinical Trials

The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at the National Institutes of Health (NIH) and other NIH Institutes involved in the BPCA are collaborating with the U.S. Food and Drug Administration (FDA) to design clinical trials for pediatric populations. The process involves creation of a Proposed Pediatric Study Request (PPSR) or Written Request (WR), the formal mechanism by which FDA notifies a drug maker that additional clinical information about a drug is needed. The WR is the basis for a Request for Proposal (RFP) or Request for Application (RFA), the formal mechanism by which NIH describes, in detail, the elements of a given research study or clinical trial that it would like to see conducted to obtain needed data.

Once NICHD has initiated a funding mechanism to the most-qualified group, the final protocol for the trial is developed, with guidance from NICHD and with input from and interaction with the appropriate NIH Institute or Center, the BPCA Coordinating Center (CC), the Principal Investigator (PI), the FDA, and the Institutional Review Boards (IRB). The protocol becomes the blueprint for the trial and the basis of the Investigational New Drug (IND) submission to FDA.

All researchers and staff involved with clinical trials follow every federal regulation and ethical guideline in conducting the study to ensure the safety of participants. Frequent evaluation by those involved and by independent entities throughout the course of the trial provides added safety. For more information about clinical trials, the safety of clinical trials, and protections for participants and their confidentiality, please visit the resources section at http://www.clinicaltrials.gov.

During the clinical study, NICHD works closely with the PIs, the CC, and the Data Monitoring Committee (DMC) to oversee the safety of the participants in the trial. When the clinical study is completed, the data are submitted to the FDA for evaluation by its review staff and expert advisory panels with the intent of modifying the label to improve pediatric therapeutics. It is the intent of NICHD to make all data from these clinical trials available to interested investigators to improve information about design, conduct, and evaluation of pediatric clinical trials.

It should be noted that not all drugs listed had WRs developed. For instance, several drugs of interest to neonatologists that are on the list (dopamine-dobutamine, furosemide-bumetanide) had development of the WR deferred because of clinical and scientific complexity in designing the clinical trials. These drugs have and will continue to be discussed by NIH scientists with input from FDA staff and outside experts to resolve those complexities before the clinical studies are designed and implemented.

The http://clinicaltrials.gov Web site lists all clinical trials currently being conducted and supported by the federal government and some pharmaceutical manufacturers, including BPCA-related trials. Please visit the Web site for more information about specific trials.