2025 年 11 月 26 日
Pediatric Drug Development: Why It’s Critical to Submission Strategy and How Modeling, Regulatory, and Clinical Insights Shape Success
Hear perspectives from industry, regulatory science, and academia that illustrate how an integrated approach can de-risk pediatric programs
Senior Director, Clinical Pharmacology Consulting, Co-Lead of Certara’s Rare and Neglected Innovation Engine
Dr. Hay joined Certara in 2022 with 25+ years of clinical pharmacology experience having started his career as Senior Clinical Scientist at the Centre for Human Drug Research (CHDR), Leiden. More recently he worked as Senior Pharmacokinetics Assessor and Deputy Unit Manager at the Medicine and Healthcare Products Regulatory Agency (MHRA), UK where he also had a leading role with the Access Consortium (Regulatory agencies of Australia, Canada, Singapore, Switzerland and UK).
Justin has also been a member of the EMA’s former Modelling and Simulation Working Party (MSWP). He has a special interest in biologics, CNS research, pain management and pediatric pharmacology. Justin has a PhD from the University of Adelaide, Australia.
Senior Director, Regulatory Strategy
Dr. Eva Gil Berglund is a pharmacist by training and has a PhD in Clinical Pharmacology, both from Uppsala University, Sweden. She has been a Clinical Pharmacology reviewer at the Swedish Medical Products Agency for over 20 years and a Senior Expert for 12 years, working with all types of molecules in marketing applications, clinical trials and scientific advice procedures in the EMA Network of National agencies. Eva has been working in all therapeutic areas and has extensive knowledge in antivirals, antibiotics, CNS active drugs, oncology, rheumatology, inhalation products etc.
Eva actively involved in drafting of several EU Clinical Pharmacology guidance documents and Rapporteur of several (drug-drug and drug-food interactions, PBPK, pediatrics, pharmacogenetics, etc.), been extensively involved in inter-regional harmonization activities and in the work of EMA working parties Pharmacokinetics Working Party and Paediatrics Working Party. Eva joined Certara in 2019 and provides her Clinical Pharmacology experience and Regulatory strategy knowledge in GAP analyses, regulatory stress tests and moc meetings, regulatory interactions, filing and clin pharm response support, pediatric submissions (PIP, PSP, new indications). Eva is part of the Clinical Pharmacology Regulatory Strategy team and also part of the Pediatric Centre of Excellence. Her inspiration is scientific development and its practical application, optimizing drug development, pushing regulatory science forward, and improving patient access to efficacious and safe drugs.
Director, Clinical Pharmacology and Translational Medicine
Paola Coppola is currently Director Clinical Pharmacology at Certara. She has 15+ years of clinical pharmacology and regulatory experience having worked as Senior Pharmacokinetics Assessor at the Medicines and Healthcare products Regulatory Agency (MHRA), UK and in a number of roles in Industry such as Head of Clinical Pharmacokinetics and Marketed Products Clinical Pharmacology Lead at AstraZeneca, UK, and Pharmacokinetics Scientist in Angelini, Italy. She has been an observer of the EMA PK Working Party and Modelling & Simulation Working Party.
Paola has extensive experience in early phase clinical trials and strong interest in the evaluation of PK in special populations such as pregnancy and pediatrics. Having worked on a MHRA project on the evaluation of PK in pregnancy in collaboration with The Bill & Melinda Gates Foundation, she has authored a number of publications and presentations on that matter. Paola is a member of the Clinical Pharmacology Regulatory Strategy team and of the Pediatric Center of Excellence at Certara. She obtained her MSc in Biological Sciences from the University Federico II of Naples, Italy and a Post graduate Master from the Business School ISTUD, Italy.
This blog was originally published in April 2023, and has been updated for accuracy.
常见问题解答
What is a Paediatric Investigation Plan (PIP)?
A Paediatric Investigation Plan (PIP) is a comprehensive development program that outlines how a pharmaceutical company intends to study and ensure the safety and efficacy of a medicine for use in children. The PIP details the measures and timelines for conducting pediatric studies, aiming to generate the necessary data to support the authorization of medicines for pediatric populations. In the European Union, the PIP is a regulatory requirement intended to ensure that children have timely access to appropriately tested medicines and to encourage the development of medicines specifically for pediatric use.
Does the United States FDA require sponsors to submit the equivalent of a PIP?
Yes, the FDA requires sponsors to submit a pediatric study plan (PSP). A PSP is a structured document that outlines the strategy and specific steps a sponsor intends to take to evaluate the safety, efficacy, and appropriate use of a drug or biologic in children. It typically includes proposed study designs, age-appropriate formulations, timelines, and considerations for ethical conduct in pediatric populations. The plan is developed to ensure that medicines are properly tested in children, addressing their unique medical needs and regulatory requirements.
How can model-informed drug development approaches be used to increase the efficiency of pediatric drug development?
Model-informed drug development (MIDD) significantly enhances the efficiency of pediatric drug development by utilizing existing data and advanced modeling techniques to minimize the need for large, dedicated clinical trials. By extrapolating efficacy and safety data from a reference population, MIDD predicts drug responses in a target population, often facilitating regulatory approval without separate Phase 3 studies. These approaches optimize dose selection using population and physiologically-based pharmacokinetic models, support efficient trial design through informed sample sizes and schedules and incorporate biomarkers or natural history data as surrogate endpoints to reduce placebo use and accelerate development. Regulatory agencies like the FDA routinely accept MIDD analyses to support dosing decisions, trial waivers, and expanded labeling, especially with early sponsor engagement through programs such as the FDA MIDD Pilot Program. Overall, MIDD offers a scientifically robust and ethical pathway to accelerate pediatric medicine development.
What do paediatric investigation plan (PIPs) waivers and modifications refer to?
Drug developers can request PIP modifications, waivers, and deferrals from the PDCO. A waiver exempts the sponsor from conducting pediatric clinical studies on an investigational therapy. A deferral allows the sponsor to delay conducting pediatric studies until after adult trials are completed. If a sponsor encounters difficulties or needs to change the PIP after it has been agreed upon with regulators, they can request modifications to it.
携手 Certara 推进儿科药物开发
Certara 通过创新建模与模拟技术及监管策略，引领儿科药物开发变革。我们独有的儿科 MIDD 方法，将尖端生物模拟与监管科学结合，助力设计符合全球监管标准、确保儿童用药安全有效的儿科研究方案。
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