Identifying the “Blip on the Radar Screen”: Leveraging Big Data in Defining Drug Safety and Efficacy in Pediatric Practice

Abstract

The immense amount of electronic health data (pharmacy and administrative claims, electronic health records, and clinical registries) that is being generated every day in the care of patients has the potential to be leveraged for improving clinical decisions at the point of care, uncovering or validating drug efficacy and drug safety. The potential use of big data for improving safe and effective use of medications is especially important in children because of their low drug exposure relative to adults. Electronic health data is collected primarily for clinical or billing purposes and not for research purposes. The major steps involved in data acquisition, extraction, aggregation, analysis, modeling, and interpretation are discussed. It is important to understand the limitation of big data and utilize appropriate study design and statistical methods. Possible applications are presented along with specific examples of how big data has been used in drug research to find that blip on the radar screen that may give an efficacy or safety signal that can lead to further investigation.

A Foundation for Inductive Reasoning in Harnessing the Potential of Big Data

“…Given the uncertainty associated with Big Data, which is usually observational or nonexperimental (Christensen & Davis, 2018), classification and causal reasoning models are particularly necessary for deriving understanding and meaning (Johnson, Rajeev-Kumar, & Keil, 2016). Toward this end, epidemiology, which is considered the twin discipline of statistics (Bhopal, 2016) offers concepts and frameworks that can serve as a firm basis for reasoning with Big Data in healthcare.…”

Los Big Data: conceptos relacionados y algunas aplicaciones en pediatría

“…Otra aplicación de los RES ha sido en la investigación fármaco-epidemiológica, donde se ha podido estudiar el uso de medicamentos, la eficacia y la seguridad, particularmente para población pediátrica. Esto debido a la baja exposición a los medicamentos que suelen tener los niños en comparación con los adultos 29 .…”

Developmental Pharmacotherapy: The Interface Between Ontogeny and Drug Effect

“…With the broad acceptance, utilization, and continuously improving detail and accuracy of patient data being incorporated into the electronic medical record, the value of mining "big data sets" cannot be overstated, providing a broad view of drug use, response, and safety in the real-world setting. Each of these four articles [6][7][8][9] provides insight and substance to the importance of data-informed modeling and simulation-guided clinical trial design; study execution; data-driven modification, if necessary;…”

“…Clinical trials in target pediatric patients with targeted disease(s) are fundamental to a better understanding of age‐size influenced disposition characteristics and the definition of optimal age‐appropriate drug doses in children . Four detailed, expert reviews address the current status of drug development in children and the incorporation of innovative study designs available to best capture the most from limited data sets. The data derived from clinical trials integrated with modern modeling and simulation methodologies enhances the accuracy of dose projections for a patient population as well as the individual patient.…”

“…With the broad acceptance, utilization, and continuously improving detail and accuracy of patient data being incorporated into the electronic medical record, the value of mining “big data sets” cannot be overstated, providing a broad view of drug use, response, and safety in the real‐world setting. Each of these four articles provides insight and substance to the importance of data‐informed modeling and simulation‐guided clinical trial design; study execution; data‐driven modification, if necessary; successful trial conclusion; and proper analysis with relevant, accurate interpretation. Pediatric health care providers are best served when the results from a comprehensive, multifaceted, thoughtful, methodical clinical drug development program is included in the sanctioned official product labeling for all to use in their pharmacotherapeutic decision tree.…”