采访嘉宾：

        Dr. Catherijne A. J. Knibbe is hospital pharmacist-clinical pharmacologist in the St. Antonius Hospital in Nieuwegein, Professor of Individualized Drug Treatment at the Division of Systems Biomedicine and Pharmacology of the Leiden Academic Center for Drug Research (LACDR) at Leiden University, and holds a guest appointment at the NICU of the Erasmus MC, Sophia Children Hospital, Rotterdam. She leads a research line on optimization of pharmacotherapy in special patient populations like neonates, children and morbidly obese patients, with use of pharmacometric approaches including population PKPD modelling or PBPK modelling. Dr. Knibbe became a Fellow (FCP) of the American College of Clinical Pharmacology (ACCP) in 2019 and is a member of the Central Committee of Research Involving Human Subjects (the 'CCMO') in the Hague

采访者：

        Xueting Yao（么雪婷）, Ph. D, Assistant Research Scientist in the Drug Clinical Trial Center, Peking University Third Hospital, Peking University. Dr. Yao received Ph.D from Peking Union Medical College Hospital, Chinese Academy of Medical Sciences. She worked at Peking University Third Hospital from August 2019. Her research interests include phase I clinical trial of innovative drug for metabolic disease, MIDD, and physiologically-based pharmacokinetic modelling for children.  

Dr. Xueting Yao on behalf of the organization committee of ISQP:

Xueting Yao:

 Prof. Catherijne A. J. Knibbe is an outstanding clinical pharmacologist in the Division of Pharmacology at Leiden University working on drug research in children. She combined clinical responsibilities as a hospital pharmacist-clinical pharmacologist in Intensive Care and started a research group developing rational dosing schemes for pediatric indications using population PK-PD modeling within a multi-center platform, including Division of Pharmacology, LACDR, Leiden, Erasmus MC/Sophia Children's Hospital Rotterdam and UMCU/Wilhelmina Children's Hospital. Prof. Knibbe will have a conference report with topic of “Challenges and Opportunity for Drug Research in Children: How to Apply Modeling and Simulation in the Clinic” in ISQP conference. Before the formal report, on behalf of the organizing committee, I had an email interview with Prof. Knibbe. Let’s take a look at her perspective on children drug research.

Xueting Yao: Dear Prof. Knibbe, it is a great honor to invite you to share with us. Drug development and rational dosing schemes for children has been widely concerned. U.S FDA, EMA, and China NMPA, have issued a series of guidelines to promote pediatric drug development. How do you think the current situation of pediatric drug development? What do you think are the most difficult challenges?

Prof. Knibbe: Currently, more and more studies are being performed in children of different ages which is very good news! This applies to studies on new drugs that are being developed by the industry and (academic) studies on existing drugs. For studies on novel drugs funded by companies, the challenge is I think to define what kind and how much data are required to define the PK, effects and safety of the drug in children whereby population PK PD modelling techniques should be optimally applied to minimize the burden for the children participating in the trial. I still see trials submitted to IRBs in which children are exposed to full PK curves, which is often not justified given the data of the drug that are already out there, or that can be predicted using Physiologically-based PK modelling (PBPK) techniques. Optimizing these trials using the best data analysis techniques and convincing the industry and regulatory that based on these (minimal) data the PKPD can be solidly estimated in children is therefore the greatest challenge.   

Xueting Yao: The model informed drug development, specifically physiological based pharmacokinetics and population pharmacokinetic and pharmacodynamic model, is an invaluable and proven tool that is mushrooming in the pediatric studies. But how to apply modeling and simulation in pediatric drug development and in the clinic? And in the clinic, how to benefit the individual pediatric patients using these modeling technologies?

Prof. Knibbe: For studies on existing drugs that are performed in the clinic, population PKPD modelling is in my opinion the preferred tool for two reasons: 1. The burden for the individual child participating in the study is minimized because these techniques allow for the analysis of sparse and unbalanced data as opposed to standard two-stage techniques that require full PK curves and 2. Studies can be performed within the context of clinical practice in which the child is treated with the drug of interest because the technique can handle varying or missing sampling times. As such, costs are reduced and dedicated trials are not always required as these studies can be combined with clinical care. However, when these type of scarce data from children are analyzed , particularly when participating children vary in age and weight, a prerequisite is that advanced expertise with this type of modelling is available to prevent the development of models that do not well capture the variability or lack predictive power that is required to design adapted drug dosing, so this is always a point of concern. Similarly, for PBPK modelling, advanced expertise is required and therefore, for use in the clinic I would say that there is only limited room for this tool. Only in case predictions are needed in age groups or drugs for which other scaling methods have proven to result in incorrect predictions, like young infants and neonates, PBPK should be used. We are actually working on a paper in which we aim to define for what drugs and what age groups PBPK is required as compared to allometric scaling or linear weight-based scaling as tools to predict clearance.

Xueting Yao: With the deepening in pediatric scientific research, artificial intelligence, and machine learning technological progress, how do you think the opportunities and prospect for the drug research or precision medicine in children?

Prof. Knibbe: All of these methods will contribute together to better analysis techniques to analyze the sparse data that are available in children. For now, I think that while the techniques are out there, we need people with expertise to use these techniques and apply them to good data whereby for studies on existing drugs, I advocate to always check whether historical data can be used with or without prospective data that are to be gathered. It is often stated that there is a scarcity on data in children, but for many drugs I think there are many data, we just need to analyze them with optimal data analysis techniques.

Xueting Yao: Do you have any future expectations for drug research in children or pediatric drug development?

Prof. Knibbe: I hope that efforts across the world on characterizing the PKPD of drugs in children varying from preterm neonate to adolescents will advance optimal efficacy and safety of drugs in children.

Xueting Yao:

大会报告信息：

大会报告主题：儿童药物研究的挑战与机遇：如何在临床中应用建模和模拟

                    （Challenges and Opportunity for Drug Research in Children: How to Apply Modeling and Simulation in the Clinic）

报告时间：12月6日上午，分会主题8：儿童新药开发：中美指南解读及目前挑战与未来机

赵立波  教授

        医学博士、主任药师、教授、博士生导师，先后就职于北京大学人民医院、北京儿童医院和北京大学第三医院，主要从事临床药理学/临床药学科研教学工作，在个体化用药及临床试验管理等方面有一定的经验。主持国家自然基金课题2项、重大新药创制专项子课题1项，北京市自然基金2项、首发基金1项。以第一作者或通讯作者发表SCI收录论文30余篇。兼任中国药理学会临床药理专委会/定量药理专委会委员，中国药学会药学服务专委会委员，北京药学会理事及TDM专委会副主任委员等。

赵  维  教授

山东大学，教授、博士生导师；山东大学临床药学系主任、教研室主任。山东大学临床药理研究所所长；国家药品监督管理局创新药物临床研究与评价重点实验室常务副主任；山东省千佛山医院I期临床试验研究中心主任；山东省儿童药物临床评价与研发工程技术研究中心主任；山东省临床药学与临床药理重点实验室主任；山东省儿童抗感染治疗健康医疗大数据科技创新联盟主任；山东省泰山学者青年专家；山东大学杰出中青年学者。担任国家药品监督管理局新药审评专家；欧盟药监局数据外推委员会、建模与仿真委员会常务委员、审评专家；法国药监局儿科药物评审委员会副主任；中欧临床试验优化设计联盟主席。发表论文120余篇。总影响因子超过450分。主持5项国家级、2项省部级和4项国际合作项目。主持70余项药代动力学、有效性和安全性研究；获得国家药品监督管理局药物批件、欧盟药监局药物研究计划PIP批件10余项。

ISQP重要时间：
        2021年9月30日                优惠注册截止日期
        2021年12月5日-6日         大会正式议程（含现场注册）
        2021年12月7日                东亚论坛（线上会议）
        2021年12月25日-12月4日   会前和会后培训班
大会网站： https://isqp2021.sciconf.cn