Office of Biostatistics & Epidemiology, Center for Biologics Evaluation and Research, US Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, US Food and Drug Administration (FDA), Silver Spring, Maryland, USA.
CPT Pharmacometrics Syst Pharmacol. 2021 Apr;10(4):286-290. doi: 10.1002/psp4.12597. Epub 2021 Mar 5.
As part of the US Food and Drug Administration (FDA)'s Prescription Drug User Fee Act (PDUFA) VI commitments, the Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) are conducting a model-informed drug development (MIDD) pilot program. Sponsor(s) who apply and are selected will be granted meetings that aim to facilitate the application of MIDD approaches throughout the product development lifecycle and the regulatory process. Due to their complex mechanisms of action and limited clinical experience, cell and gene therapies have the potential to benefit from the application of MIDD methods, which may facilitate their safety and efficacy evaluations. Leveraging data that are generated from all stages of drug development into appropriate modeling and simulation techniques that inform decisions remains challenging. Additional discussions regarding the application of quantitative modeling approaches to drug development decisions, such as through the MIDD pilot program, may be crucial for both the sponsor(s) and regulatory review teams. Here, we share some perspectives on the opportunities and challenges for utilizing MIDD approaches for product review, which we hope will encourage investigators to publish their experiences and application of MIDD in gene therapy product development.
作为美国食品和药物管理局 (FDA) 处方药用户收费法案 (PDUFA) VI 承诺的一部分，生物制品评估和研究中心 (CBER) 和药物评估和研究中心 (CDER) 正在开展模型指导药物开发 (MIDD) 试点计划。申请并被选中的申办方将被授予会议，旨在促进在整个产品开发生命周期和监管过程中应用 MIDD 方法。由于其复杂的作用机制和有限的临床经验，细胞和基因疗法有可能受益于 MIDD 方法的应用，这可能有助于它们的安全性和疗效评估。利用药物开发各个阶段生成的数据，将其转化为告知决策的适当建模和模拟技术仍然具有挑战性。通过 MIDD 试点计划等方式，就定量建模方法在药物开发决策中的应用进行额外的讨论，对于申办方和监管审查团队都可能至关重要。在这里，我们分享了一些关于利用 MIDD 方法进行产品审查的机会和挑战的观点，我们希望这将鼓励研究人员发表他们在基因治疗产品开发中应用 MIDD 的经验。
