Pediatric Clinical Trials

Among the mandates of BPCA is to design and sponsor clinical trials on therapeutics in pediatric populations. Doing so requires collaboration among NICHD, other NIH institutes, non-government organizations, and the U.S. Food and Drug Administration (FDA). Specific goals include the following:

  • Identify therapeutic gaps in pediatric diseases, disorders or conditions in which expanded knowledge of treatment strategies, including drugs and biologics, may be required
  • 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

View a listing of current BPCA clinical trials (PDF 268 KB).

These clinical trials are conducted under regulatory rigor so that the data can be submitted to the FDA for label change consideration. Requirements include the following:

  • Carefully monitoring all BPCA-sponsored studies in children for compliance with all federal regulations
  • Submitting quality data from the clinical studies to the FDA for evaluation and additional analyses in consideration of label modification
  • Making de-identified study data available to investigators and the public

The ultimate outcome of the clinical trials and research efforts of the BPCA program is a change in a medication label to improve the knowledge and safe use of that drug in the pediatric population. 

In addition to a label change, there are other avenues for sharing BPCA-supported resources with researchers, healthcare providers, and the public, including publications, training opportunities, lectures, collaborations, and working group recommendations and presentations.​

The process usually begins with Written Request (WR)—​the formal mechanism through which the FDA notifies pharmaceutical companies that additional clinical information is needed for an off-patent drug for a selected indication and/or in specific populations. FDA first issues a WR to the drug sponsor. If the sponsor turns down the WR, it is then issued to NIH/NICHD to perform the study.

In some cases, NIH can start the process for an off-patent drug by sending a Proposed Pediatric Study Request (PPSR) to FDA. A PPSR is similar to a WR in that it describes the information needed to change labeling for a certain drug.

After meeting with the FDA to review a study’s design, the Pediatric Trials Network (PTN) finalizes the clinical trial design and begins the process to select the most appropriate investigators and sites to conduct the trial.

​The approved protocol serves as the blueprint for the clinical trial and, later, as the basis of the Investigational New Drug (IND) application to FDA.

The BPCA Data Coordinating Center (DCC) collects study data using electronic case report forms. It also monitors trial sites, analyzes and submits data to the FDA, and supports the BPCA Data Monitoring Committee which provides safety oversight for all BPCA studies.

Once the clinical trial is completed, the DCC submits trial data to the FDA in the form of a Clinical Study Report (CSR). If appropriate, the data are then used to change drug labels.

The de-identified data from the completed BPCA trials are available on the NICHD Data and Specimen Hub (DASH) a centralized resource for researchers to store and access data from NICHD-funded research studies to use for secondary research.

Not all WRs or PPSRs received from FDA result in clinical studies. Some reasons a study may not occur include:

  • Various ethical issues, including parental permission and the child's assent
  • Lack of necessary technology to monitor patients and test very small amounts of blood
  • Lack of incentives for pharmaceutical companies to study drugs in neonates, infants, and children
  • Unforeseeable nature of some clinical responses in immature individuals
  • Possibility of catastrophic unanticipated reactions
  • Threat of effect on growth or health long after the drug's administration
  • Difficulty in predicting dose-response or concentration-response relationships by extrapolating data obtained from adults
  • Ethical considerations for conducting clinical research in children
  • Lack of suitable infrastructure for conducting pediatric pharmacology research

In addition, not all drugs or therapeutic areas listed in the Priority List under BPCA currently have WRs or are under active study. For example, a drug may have additional research needs—such as determining appropriate outcome measures, new formulations, or other concerns—before a clinical trial begins.  

In September 2010, NICHD awarded a contract to establish a Pediatric Trials Network (PTN)  ​at Duke Clinical Research Institute (DCRI).

​PTN's main objectives are to:

  • Provide an environment and appropriate infrastructure for conducting safe and effective pediatric clinical trials for BPCA
  • Perform ancillary activities in support of these trials

PTN conducts pediatric clinical drug trials in a variety of therapeutic areas, including cardiovascular diseases, cancer, infectious diseases, gastroenterology, respiratory diseases, neonatology, and medical devices.

​​​In addition, lists all clinical trials sponsored or funded by the federal government, including BPCA-related trials, in addition to trials managed by pharmaceutical companies, academic medical centers, voluntary groups, and other organizations.

For more information and resources about clinical studies in children, visit NIH's Clinical Research Trials and You.

You're Invited

NICHD and DCRI are interested in candidates who have clinical trial concepts and who would like to collaborate with PTN. Pediatric physicians can submit ideas using a concept sheet (DOC 104 KB).  

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