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Abstract:
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Pediatric development program uses the concept of efficacy extrapolation at three levels: full extrapolation, partial extrapolation and no extrapolation. In a recent publication, a survey reviewed 157 products with 388 pediatric studies submitted to the FDA from 2009 through 2014. The findings revealed the change of “full extrapolation” from 14% to 34%, the change of “no extrapolation” from 18% to 37%, and a decrease of partial extrapolation. It is exciting evidence how science has evolved and increased the certainty of full extrapolation and decision-making process. In addition, modeling and simulation in extrapolation of efficacy is a powerful tool though requires further validation. Statistical methodology, such as Bayesian hierarchical models, power prior, mixture prior, and meta-analytic prior, are finding more applications. Nevertheless, methodological research is still needed to expedite drug development and to improve user friendliness of analytics in clinical research. This roundtable will focus on experiences and learnings on extrapolation methods in pediatric efficacy trials, thoughts on safety assessment, and identifying areas for enhancement and new approaches.
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