Pediatric Drug Development

The evolution of the pediatric regulatory environment has increased the demand to develop a common scientific-based approach that is acceptable to global regulatory authorities. It is crucial to create a thoughtful plan to support efficient pediatric drug discovery and development to ensure safe and effective medicines for children. Typical challenges encountered in a pediatric program include:

  • Enrollment challenges due to small populations and multiple sponsors targeting the same population
  • Burden of research procedures
  • Limitation in use of placebo and lack of active comparators
  • Physiological differences across the entire pediatric population
  • Bridging of knowledge from adults to pediatrics

Certara has unique and extensive capabilities and experience to address these challenges and help set up your pediatric program for success. With experience working on over 100 pediatric programs, our experience in pediatric drug development is unparalleled. Our multi-disciplinary team of experts in pediatric practices provides you with end-to-end, integrated solutions, including:

  • Strategic planning of pediatric study plans (PSPs) and paediatric investigation plans (PIPs)
  • Pediatric clinical trial study design
  • Clinical pharmacology strategy and stewardship of complex pediatric programs
  • 真实世界证据
  • Juvenile toxicology
  • Innovative modeling and simulation, characterizing PK, PD, disease progression, safety and efficacy
  • Regulatory interactions specific to pediatric development including Type C or Type F (FDARA) meetings with FDA and scientific advice with EMA
  • Age-appropriate pediatric formulations
RACE for the Children Act went into effect on 2020 年 8 月 18 日

With the RACE for Children Act, the FDA is now authorized to require sponsors to submit an agreed initial Pediatric Study Plan (iPSP) with an original NDA or BLA oncology product that may target the growth or progression of a pediatric cancer. New cancer therapies with orphan-designated indications will no longer be exempt from PREA.

The iPSP must include an outline of the planned pediatric investigation(s) and any planned request for deferral or waiver with supporting documentation. According to the guidance by the FDA, a sponsor must submit the iPSP no later than 60 calendar days after the date of the end-of-phase 2 meeting.

What are the important things you need to know about the RACE Act? How can you set up your pediatric program for success?

Learn more about the RACE for the Children Act
RACE for the Children Act went into effect on August 18, 2020
Set your pediatric program up for success
Set your pediatric program up for success

Certara scientists and experts meet the needs of the regulators, enhance feasibility of the studies, and increase safety for pediatrics practices.

Certara’s quantitative methods leverage sparse data and prior information from pre-clinical studies, adult trials, literature data, and pediatric studies of related indications or drug actions. We build that knowledge into models of patient physiology, drug actions and trial characteristics, which enable us to develop and iterate clinical trial design and explore alternative dosing scenarios, in silico patient responses, drug-drug interactions, and whole trial outcomes

Our consultants also use the Simcyp™ Pediatric PBPK Simulator, the industry’s most sophisticated physiologically-based pharmacokinetic (PBPK) technology for modeling drug performance and assessing drug-drug interactions in neonates, infants, and children. The Simcyp Pediatric PBPK Simulator, routinely used by the Simcyp Consortium’s 35 leading pharmaceutical companies, contains extensive libraries on demographics, developmental physiology, and the ontogeny of drug elimination pathways. It is used by all of the industry’s leading pharma companies.

Regulatory writing, publishing and submission support for your PIP and PSP

Our regulatory science expertise in pediatric practices, programs, and development spans more than 15 therapeutic areas, including oncology, infectious diseases, and cardiovascular disorders. Core competencies include consulting, writing, preparing and managing documents, including pediatric investigational plans (PIPs) and pediatric study plans (PSPs) through global regulatory processes, and authoring scientific and commercial documents.

Oftentimes, Certara will support the PIP/PSP development plan by creating a population PK (popPK) model using adult data scaled to pediatrics. That model will integrate a range of maturation and disease factors with allometric scaling to set the best dose for the first pediatric trial cohort.

Furthermore, our GlobalSubmit™ clinical trial operations technology facilitates the eCTD submittal process, providing scalability and consistency for sponsors.

Learn more about our regulatory science solutions
Regulatory writing, publishing and submission support for your PIP and PSP
Lynne Georgopoulos
Lynne Georgopoulos,RN、MSHS、RAC 监管策略部副总裁

Lynne 拥有超过 30 年的生物制药和 CRO 经验,在产品开发的所有阶段提供战略、临床开发和监管建议。作为 Synteract 监管事务和儿科战略发展部副总裁,以及 KinderPharm 监管事务和临床开发部高级副总裁,她在过去几年里一直致力于促进创新方法来加快儿科药物的开发。她领导了许多成功的监管申报和机构互动(例如,Pre-IND、IND、NDA/BLA、EMA 科学建议,EOP2 和 Pre-NDA)。

Certara Patrick Smith
Patrick F. Smith 一体化药物开发总裁

Patrick F.Smith 博士是 Certara 一体化药物开发总裁,他领导一个全球药物开发科学家团队,在整个生命周期为客户创造价值,使患者更快获得药物。凭借逾 20 年的药物开发经验,Patrick 拥有药物开发各个阶段的工作经验,在传染病、肿瘤学和炎症,以及新颖的早期开发方案设计,乃至应用建模和模拟来解决关键开发问题方面都具有深厚的专业知识。

Patrick 此前曾联合创办了 d3 Medicine(被 Certara 于 2016 年收购),旨在帮助打造一家专门为复杂的开发计划提供战略支持的医疗公司。在创办 d3 Medicine 之前,Patrick 在 Roche 的美国临床药理学主管职位上任职超过 5 年。他在该公司的临床药理学和转化医学部门担任了多个职务,承担日益重要的职责。他一直是布法罗大学药学院兼罗斯威尔帕克癌症研究所的副教授。Patrick 已在各期刊上发表超过 125 篇同行评审的文章,其中包括曾在《新英格兰医学期刊》和《柳叶刀》上发表文章。他持有加利福尼亚大学旧金山分校药学博士学位,并在杜克大学医学中心完成了博士后临床训练。

Bio Pic TrevorJohnson
Trevor Johnson, PhD Principal Scientist

Trevor leads the development of Simcyp pediatric software designed to predict dose, drug-concentration time profiles and likely drug response from birth onwards. His current areas of research focus on pediatric oral drug absorption & biologics, ontogeny of transporters and also special population’s particularly hepatic impairment. He has over 50 research publications and 7 book contributions in the areas of pharmacokinetics, drug metabolism, pediatric drug therapy and hospital Pharmacy


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