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On the feasibility of pediatric dose-finding trials in small samples with information from a preceding trial in adults.

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We consider the case of pediatric dose-finding trials with extremely limited sample size. The operating characteristics of the standard design, the Continual Reassessment Method (CRM), are only well described for… Click to show full abstract

We consider the case of pediatric dose-finding trials with extremely limited sample size. The operating characteristics of the standard design, the Continual Reassessment Method (CRM), are only well described for sample sizes of about 20 patients or more. In this simulation study, we assume the situation of a pediatric trial with only 10 patients and a preceding dose-finding trial in adults. Based on the adult data, we reduce the set of pediatric doses and formulate (partially) informative prior distributions for the pediatric trial. Our simulations show that such small pediatric dose-finding trials with robustified priors may provide sufficient operating characteristics.

Keywords: pediatric dose; trial; trial adults; feasibility pediatric; dose finding; finding trials

Journal Title: Journal of biopharmaceutical statistics
Year Published: 2021

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