BPCA PHARMACOEPIDEMIOLOGY (REAL WORLD DATA) STUDIES

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Since the early 1990s, the scientific community, including academia, NIH, 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 in pediatrics 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, the validation of outcome measures and endpoints in pediatric research, and most recently utilizing databases and electronic health records (real world health data) to leverage clinical research. After more than 30 years of legislation, and in the face of burgeoning technological advances in clinical care and research, it has become clear that to be successful in continuing to close knowledge gaps in the area of pediatric drug development to appropriately treat pediatric patients for a variety of diseases, an integrated and comprehensive approach to pediatric drug development research is required. Furthermore, NICHD and others in the scientific community are concluding that the harmonization of clinical research data (i.e., PK, pharmacodynamic, pharmacogenomic, efficiency, and safety data) with the use of existing clinically relevant databases, registries and other means of collating real world patient health data are vital to the success of effectively treating pediatric patients in a personalized way.