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Basu, C, Ma, X, Mo, M, et al. Pharmacokinetic/pharmacodynamic data extrapolation models for improved pediatric efficacy and toxicity estimation, with application to secondary hyperparathyroidism. Pharmaceutical Statistics. 2020; 882– 896. https://doi.org/10.1002/pst.2043
In most drug development settings, the regulatory approval process is accompanied by extensive studies performed to understand the drug’s pharmacological properties, i.e. absorption, distribution, and its mechanism of action. In this paper, we attempt to utilize the rich pharmacology data to inform the borrowing of information from adults during pediatric drug development. In pediatric settings, it is especially crucial that we are parsimonious with the patients recruited for experimentation. We illustrate our approaches in the context of clinical trials of the drug cinacalcet for treating the disease secondary hyperparathyroidism (HPT) in pediatric and adult patients with chronic kidney disease (CKD), where we model both parathyroid hormone (efficacy endpoint) and corrected calcium levels (safety endpoint).