Affiliation:
1. Certara Drug Development Solutions Certara USA, Inc. Princeton New Jersey USA
2. Sanofi, R&D Gentilly France
3. AstraZeneca, R&D Paris France
4. Integrated Drug Development, Certara London UK
5. Pfizer Inc. Groton Connecticut USA
6. Bristol‐Myers Squibb Princeton New Jersey USA
Abstract
Many new opportunities surround rare pediatric disease drug development, thanks to key advances in regulatory thinking and in the scientific community. As rare disease drug development brings challenges to the developers in terms of limited understanding of natural history, heterogeneity in drug response, as well as difficulty recruiting patients in pivotal trials, there has never been a greater need for quantitative integration. To understand how International Consortium for Innovation and Quality in Pharmaceutical Development (IQ) member companies approach pediatric rare disease drug development, the rare pediatric subteam of the Clinical Pharmacology Leadership Group (CPLG) sponsored Pediatrics Working Group conducted a baseline survey to assess the four main pillars of this quantitative innovation, namely, biomarkers and surrogate end points, statistical methodologies, model‐informed drug development, as well as public–private partnerships. The survey was administered by IQ and yielded 13 evaluable responders from represented companies. This article presents the key findings from this baseline identifying survey, highlighting the key blind spots, and providing insightful expert opinions to address those gaps. In summary, we call an urgent attention to the community on the opportunities to enhance integration and within‐industry learnings from this analysis on aspects related to platform studies, end‐to‐end quantitative integration, and sharing of trial‐level placebo data for better understanding of disease progression and more efficient trial designs. We collectively hope that these findings will stimulate discussion and debate around cross‐industry sharing and collaboration to better delineate principles and further enhance the efficiency of rare pediatric disease drug development.
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