Enhancing pediatric clinical trial feasibility
By: Robin Huff, PhD | June 21, 2016
How to mitigate the challenges of conducting clinical research with pediatric patients.
The Drug Information Association (DIA) will be holding its 2016 Annual Meeting in Philadelphia from June 26th – 30th. If your company has or will have regulatory commitments to conduct pediatric clinical trials, we hope to see you at the Wednesday afternoon session, Emergent Study Designs and Analysis Methods Addressing Issues Associated with Pediatric Clinical Trials.
Pediatric clinical trials present challenges distinct from their adult counterparts, including a less well established clinical trial infrastructure, the need to use assessments for safety and efficacy that are tailored to individual age groups, and the generally smaller number of children affected by a particular disease. There are collaborative efforts underway to address many of these challenges, such as the establishment of the Pediatric Clinical Trials Network, and work to develop pediatric clinical outcome assessments in specific therapeutic areas. A potential means of addressing the feasibility challenge inherent in having a small pediatric patient pool will be the focus of Quintiles’ presentation during the Wednesday afternoon session at DIA.
The limited pediatric patient population affected by certain diseases greatly diminishes the feasibility of conducting clinical trials of the size necessary to demonstrate statistical significance by traditional means. Feasibility difficulties are further heightened when multiple drugs approved for the same adult indication, each with a commitment to conduct a pediatric study, compete for the same patients. One potential solution to this problem is the application of Bayesian statistics to pediatric trials. During the DIA session, Quintiles will highlight work we have done to explore the application of Bayesian statistics to pediatric trials, using type-2 diabetes as a test case indication.
Bayesian statistics makes use of a prior data set – in this setting, the adult data set, to inform the design of a new clinical trial – in this case, the pediatric trial. We ran a series of simulations utilizing data sets for six drugs, two each from three different drug classes, approved for the treatment of adults with type-2 diabetes. We consistently demonstrated that the size of the pediatric trial could be reduced to nearly half of the traditional trial size when the adult data was allowed to contribute approximately 20% weight to the pediatric trial, while keeping false positives at less than 14%.
Join us at DIA to learn more about the potential for Bayesian statistics to make it feasible to generate data that will enable drugs to be labeled for pediatric use, and talk to us about other initiatives being undertaken by Quintiles’ Pediatric Center of Excellence to facilitate pediatric clinical research.