Certara Drug Development Solutions, Certara USA, Inc., Princeton, New Jersey, USA.
Sanofi, R&D, Gentilly, France.
Clin Pharmacol Ther. 2024 Dec;116(6):1433-1441. doi: 10.1002/cpt.3422. Epub 2024 Aug 15.
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.
由于监管思维和科学界的重大进步，许多新的机会围绕着儿科罕见病药物的开发。由于对自然史的有限了解、药物反应的异质性以及在关键试验中招募患者的困难，儿科罕见病药物的开发给开发者带来了挑战，因此，定量整合比以往任何时候都更加重要。为了了解国际创新与制药质量联盟 (IQ) 成员公司如何开展儿科罕见病药物的开发，临床药理学领导小组 (CPLG) 的儿科罕见病小组赞助了儿科工作组，开展了一项基线调查，以评估这种定量创新的四个主要支柱，即生物标志物和替代终点、统计方法、模型指导药物开发以及公私合作伙伴关系。该调查由 IQ 进行，来自代表公司的 13 名应答者可进行评估。本文介绍了这一基线识别调查的主要发现，强调了关键的盲点，并提供了有见地的专家意见来解决这些差距。总之，我们呼吁社区关注通过分析与平台研究、端到端定量整合以及分享试验级安慰剂数据相关的方面来加强整合和行业内学习的机会，以更好地了解疾病进展并设计更有效的试验。我们共同希望这些发现将激发围绕跨行业共享和合作的讨论和辩论，以更好地阐明原则并进一步提高儿科罕见病药物开发的效率。
