Partial Extrapolating of Efficacy from Adult to Pediatrics: A Case Study in Systemic Lupus Erythematosus

• Activity Number: 374 - Statistical Approaches for Evidence Integration and Considerations to Communication in Product Labeling
• Type: Topic-Contributed
• Date/Time: Thursday, August 12, 2021 : 12:00 PM to 1:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Juan Jose Abellan*
• Companies: GSK
• Keywords: Extrapolation; Bayesian statistics; Dynamic borrowing; Paediatrics

Abstract

The development of medicines for paediatric population presents several ethical and practical concerns. When the paediatric development of a new drug starts, there is already valuable information from the development of the same drug in adults, which can be used to support the limited evidence that can be obtained in children. To this end, several statistical methods have been proposed in the literature to formally include relevant evidence from adults in the analysis and decision-making for paediatric studies. Of those, the Robust Mixture Prior approach (Schmidli et al, 2014) seems to be gaining acceptance. Here we present a case study where such approach was successfully used to partially extrapolate evidence of efficacy from adults to paediatrics to support regulatory approval of a paediatric indication expansion for an approved adult treatment for systemic lupus erythematosus.