Pharmaceutical Technology - March 2021

Pharmaceutical Technology - Regulatory Sourcebook - March 2021

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42 Pharmaceutical Technology REGULATORY SOURCEBOOK MARCH 2021 P h a r mTe c h . c o m Quality Collaboration E xtrapolation to the pediatric population using avail- able data obtained from a reference population is a well-recognized approach to achieve more efficient pediatric drug development and avoid unnecessary pediatric clinical trials. While regulations regarding the use of extrapolation date back to 1994 (1), there are still signif- icant challenges in the implementation of pediatric extrap- olation (2–4). To ensure the "voice of industry" was heard as the regulatory landscape evolved, the Clinical Pharma- cology Leader Group (CPLG) of the IQ Consortium (IQ) established the Pediatric Working Group (PWG) in 2011, and it has evolved to be an integral part of the implementation and dissemination of the use of extrapolation as a framework to optimize the development of therapeutics for pediatric use (Figure 1). The PWG currently consists of 38 members from 24 dif- ferent organizations, and includes representatives from IQ corporate members, regulatory agencies, and non-IQ mem- ber companies to strengthen its diversity in expertise and perspective on pediatric drug development. In collaboration with the pharmaceutical industry, regulatory agencies, ac- ademia, and non-profit organizations, the PWG has orga- nized/co-organized several workshops and webinars to inform relevant stakeholders and interested parties (5–7). The PWG has also published on related topics to describe the challenges and successful applications of pediatric ex- trapolation (8, 9). This paper highlights two case studies describing pediatric medicines development programs using a pediatric extrapolation framework, endorsed by the Eu- ropean Medicines Agency (EMA) and/or FDA. These case studies demonstrate the application of model-informed quantitative approaches to integrate prior knowledge of the disease and available data within and/or across indications to support selection of dose and regimen for pediatric Phase II/III studies. This extrapolation framework has also been successful in waiving the need for a placebo control arm in pediatric Phase III studies, and in supporting pediatric formulation qualification and labeling claims. Sebastian Haertter, S.Y. Amy Cheung, Angela James, Ashley Strougo, Christina Bucci-Rechtweg, Jing Liu, Meina Tao Tang, Paulien Ravenstijn, Raafat Bishai, Satyendra Suryawanshi, and Solange Corriol-Rohou; all authors are members of the IQ Consortium. PAULISTA - STOCK.ADOBE.COM Extrapolating Data from Adult Clinical Trials to Advance Pediatric Drug Development Extrapolating data from adult clinical trials to a pediatric population is a proven method to avoid unnecessary pediatric clinical trials. Representatives of the Pediatric Working Group of the IQ Consortium share case studies that demonstrate how adult clinical trial data can be used to support the dose selection and regimen—or waive the need for a placebo control arm—for pediatric studies.

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