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Activity Number: 304 - Extrapolation as a Default Strategy in Pediatric Drug Development: Implications to Clinical Trial Designs and Statistical Analysis
Type: Topic Contributed
Date/Time: Wednesday, August 5, 2020 : 10:00 AM to 11:50 AM
Sponsor: Biopharmaceutical Section
Abstract #312255
Title: Real World Evidence Utilization in Pediatric Clinical Development Reflected by US Product Labeling: Statistical Review
Author(s): Yodit Seifu* and Freda Cooner and Margaret Gamalo-Siebers and Junjing Lin and Junshan Qiu and Shiling Ruan and Ros Walley and Sophie Barthel
Companies: and Amgen Inc. and Eli Lilly & Co and Takeda and FDA/CDER and Novartis and UCB Celltech and PRA Health Sciences
Keywords: Pediatric ; Real World Data; Real World Evidence; US labels

We surveyed the FDA label website in order to get an understanding of the FDA’s past acceptance of effectiveness evidence generated from real world data (RWD) in support of drug approval in new indications. A pre-specified set of words and phrases were used in the clinical section of US labels to search for labels that have used real world evidence (RWE). The final data was extracted on 2 February 2019. An iterative review process resulted in 26 drugs as having clear use of RWE in the clinical section of the label. In 20 out of the 26 labels, the label includes some information on pediatric patients. In order to assess the utilization of RWE in pediatric patients we conducted detailed review of these 20 labels. The type of RWD used to generate information on pediatric patients and the associated statistical analysis that was used to assess effectiveness will be summarized together with the additional available pediatric data from clinical trials. With this analysis we hope to provide deeper understanding of the rational for including RWE in these labels.

Authors who are presenting talks have a * after their name.

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