BPCA INNOVATIVE TRIAL DESIGNS AND ASSAY DEVELOPMENTS IN PEDIATRIC THERAPEUTICS

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For the last 25 years the scientific community, including academia, the National Institutes of Health (NIH), the Food and Drug Administration (FDA), and the pharmaceutical industry have worked to improve the knowledge of medications used in children. Congress has passed legislations to provide incentives for drug development plans in pediatrics. The responsibility for the implementation and oversight for improving drug development has been delegated primarily to the FDA (for on-patent drugs) and to the NIH (for off-patent drugs). Even though the legislations have improved the number of clinical studies conducted in children, gaps remain in areas such as developmental pharmacology, ontogeny of drug metabolism and effects, validation of outcome measures and endpoints in pediatric research and novel trial designs that improve research knowledge and at the same time decrease patient burden. The latter topic is the focus of this task order. For that reason, the NICHD encourage the Best Pharmaceuticals for Children Act (BPCA) Clinical Program, specifically the Pediatric Trials Network (PTN), to begin to incorporate and adopt a new paradigm of promoting clinical research in personalized pediatric therapeutics that will provide a greater understanding of the interrelationship among disease processes, and therapies across the developmental spectrum. New technologies (like artificial intelligence); new approaches to health care including electronic health records, and a 100-year pandemic that impacted all aspects of life and every type of system—including the health care system and how research is done. This is even more important in pediatrics. This recent shift requires novel therapeutic approaches in pediatric drug development research and hence the need for this Innovative Trial Design and Assay developments project to be re-issued under the BPCA PTN. This Task Order is for the PTN to provide innovative trial designs important for the advancement of pediatric therapeutics that can lead to advances in scientific knowledge and improvements in pediatric drug labeling in this current PTN cycle. In addition, methods and assays developed as part of these trials will also be performed under this task order and used as future resources for other investigators in the field of pediatric therapeutics. Examples of trial designs include but are not limited to the following: • Platform Trials • Randomized Pragmatic Trials • Therapeutic related Registries • Electronic health system pilot programs to promote research The data collated from the clinical trials (including trial designs, outcome measures, biomarkers, and endpoints) can ultimately be incorporated and utilized for many therapeutic areas in pediatric medicine based on the scientific needs determined by the BPCA prioritization process as well as the priorities of the NICHD. Therefore, it is evident that although all individual trials (studies) can provide useful information, the integration of this acquired knowledge from the different studies in this task order, will provide a more comprehensive data analysis that will lead to a better scientific assessment to determine the best treatment approaches in adolescent and pediatric populations. More specifically, while each of these trials are important, receipt of the final report that includes the integrated data from all clinical trials performed under this task, is necessary to retain the comprehensive nature of the research conducted. The integration of the results of these innovative trial designs and the lab resources developed as part of the trials, will be presented in a final report, and will provide data to advance the science and knowledge gaps in the dosing, effectiveness and safety of therapeutics used in the pediatric populations. The integration of the data will also inform and provide a scientific platform for the development of a personalized therapeutics hub within the NICHD and across other related NIH Networks.