The US Department of Health and Human Services, National Institutes of Health has a continuing requirement for a data coordinating center for the Best Pharmaceutical for Children Act (BPCA).

The Best Pharmaceuticals for Children Act (BPCA) mandates the establishment of a pediatric drug development program for studying on- and off-patent drugs in children set forth under Part B, Title IV, section 409I (a) and (b) of the Public Health Service Act (PHS Act). The original BPCA of 2002 directed the Secretary of the Department of Health and Human Services (DHHS), acting through the Director of the National Institutes of Health (NIH), and delegated to the Director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the authority and responsibility for establishment and conduct of the research program. The BPCA legislation authorizes the NIH, in consultation with the Food and Drug Administration (FDA) and with experts in pediatric research, to identify therapeutic gaps in pediatric diseases, disorders or conditions in which more complete knowledge of treatment strategies, including drugs and biologics, may be beneficial to the pediatric population. BPCA has been reauthorized several times, most recently in 2022.

The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), specifically the Obstetric and Pediatric Pharmacology and Therapeutics Branch (OTTPB), administers the research program (hence forth termed the “BPCA Clinical Program”) and works cooperatively with other NIH Institutes and Centers (ICs) with significant pediatric research portfolios to fulfill the mandate to improve pediatric therapeutics. The BPCA authorizes three main objectives for the Clinical Program:

1. Establishing priorities for pediatric therapeutics;

2. Funding clinical trials that will improve the knowledge of the dosing, safety, and effectiveness of drugs used in children; and

3. Submitting clinical trials data to the FDA for label change and then disseminating the findings for the research and clinical care communities.

Since the BPCA was initiated, NICHD has collaborated with NIH colleagues in other Institutes and Centers, the FDA, and many experts in pediatrics, public health, and pharmacology to determine priorities in pediatric therapeutics and to conduct trials in those prioritized areas that lead to drug label changes. Because of these collaborations, and from input gathered during previous years, the BPCA Clinical Program has prioritized and studied more than twenty (20) therapeutic areas, has conducted more than fifty (50) clinical studies, has submitted close to thirty (30) clinical study reports to the FDA, and has had led to more than 20 updates to pediatric drug labels in the off-patent space.

The NICHD is planning to award a contract for a Best Pharmaceuticals for Children Act (BPCA) Data Coordinating Center (DCC) responsible for providing the requisite personnel, facilities and services to support aspects of data management, analysis, and regulatory reporting for the pediatric clinical trials, select preclinical data conducted under the authority of the BPCA, and other high priority regulatory rigorous clinical trials.

The NICHD currently maintains an infrastructure of contracts to provide support for the various clinical activities in the BPCA Clinical Program. This infrastructure requires a high level of interaction with the NICHD and other responsible parties. This structure, henceforth termed “the Consortium”, works together to construct, implement, monitor, and audit pre-clinical and clinical activities to improve the knowledge and labeling of drugs, biologics, and devices used in children. The Consortium may also collaborate with other NICHD or NIH-funded networks, nonprofit organizations, other federal agencies, or pharmaceutical/biotechnology companies. Below are the responsible parties within the Consortium that support BPCA initiatives and projects:

BPCA Data Coordinating Center (DCC; this contract) oversees data quality for the trials conducted under the BPCA program, including data capture, data management, and data storage, supports data safety monitoring and auditing for BPCA clinical trial activities, and monitors regulatory rigorous efficacy and other high-risk clinical trials.
BPCA Pediatric Trials Network (PTN) serves as the network of researchers, sites, and other consultants that act as the infrastructure for the conduct and support of safe and effective BPCA pediatric clinical trials and related services.
BPCA Logistics and Support Services provide literature reviews, scientific analyses, market surveys and conduct workshops, symposia, and conferences for the development of diverse pediatric therapeutic approaches to be utilized in the prioritization and selection of pediatric therapeutics for further study by BPCA.

• A major responsibility of the BPCA DCC will be to provide state of the art data capture, data management, and data quality assurance for approximately 30 ongoing pediatric clinical trials/projects and up to 10 additional new trials or projects added yearly, each with approximately 15 clinical study sites. This also includes the integration of data from preclinical studies. The DCC’s primary responsibility will be to ensure integrity of the data collected and regulatory compliance with FDA and NICHD policies on the conduct of clinical and preclinical studies. The activities conducted by the BPCA DCC will support data collection, data tracking, data analysis, and data storage for the generation and submission of all necessary reports to the FDA and to the NICHD Data Repository (DASH). The Contractor shall understand, perform, and coordinate all of the regulatory responsibilities for NICHD, including the submission of Investigational New Drug (IND) applications and New Drug Applications (NDA), as applicable, under the BPCA authorization. The regulatory requirements of BPCA also include assisting NICHD in tracking Proposed Pediatric Study Requests, submitting meeting requests along with meeting packages, and the submission of final datasets to the NICHD Data Repository (DASH) and/or the FDA Docket.  The responsibilities of the BPCA-DCC will fall within the following categories:
• Develop a Transition Plan for the following: (1) Archiving of data and trial master files for  20 closed studies; (2) continuance of regulatory, statistical and data management support for approximately 30 ongoing BPCA clinical trials and; (3) continuance of regulatory, statistical, and data management support for up to 10 additional new trials and projects per year during the performance of this contract
• Establish and manage an electronic data collection, data analysis, and data storage system (in the form of de-identified datasets) that will support all pediatric clinical and preclinical activities under the BPCA and other high priority NICHD studies that is compliant with all federal, NIH, and NICHD policies, including FISMA.  The data from these activities will be placed into datasets, developed and maintained by the Contractor for submission to the FDA in the form of a final study report and a public use dataset, as well as for publications, presentations, and other needs
• Provide leadership and support for all regulatory requirements associated with BPCA-related Investigational New Drug (IND) Applications and New Drug Applications (NDA or supplemental NDA), including coordination and management of all communications with FDA for clinical and preclinical trials and data, as well as submissions for other high priority NICHD studies
• Develop, coordinate, and maintain a BPCA Data Safety Monitoring Plan, to include the establishment and/or maintenance of the Data Safety Monitoring Board
• Provide data quality assurance and auditing necessary for protocol implementation and data quality review for all clinical and preclinical sites, estimating approximately 120 clinical sites for each contract year and over 1000 participant enrollments per year
• Provide monitoring and assure training compliance for high-risk, regulatory rigorous clinical trials, such as randomized clinical trials or efficacy studies
• Provide statistical leadership for the analysis of clinical trials data for submission to the FDA, including interim analyses and analyses for the final study report
• Participate in the Steering Committee and Executive Committee of the BPCA Consortium
• Provide expertise and collaborate with PTN and other investigators in protocol development, particularly for pediatric clinical trials