Data-driven strategies for pediatric success

We design and analyze pediatric clinical trials using fit-for-purpose quantitative approaches, and we develop bridging strategies within the extrapolation framework to generate robust evidence and inform optimized dosing recommendations for pediatric populations.

Adaptive methodology tailored to your clinical questions

Our approach is driven by the clinical question to address and the context of use, rather than a specific method or tool. We adopt the most fit-for-purpose method for each case, ranging from standard PopPK/PKPD to mechanistic models such as PBPK and QSP.

From the pediatric plans to the submission ready analyses, we can be your partner. We aim to strengthen your teams in the design and analysis of pediatric trials, and offer support through technical advice and workshop training. Planning for regulatory interaction, we can support you with the strategy, the briefing documents, and advocate in person at agency meetings.

Despite being a vulnerable population with significant medical needs, children have historically been therapeutic orphans, with many drugs used off-label due to a lack of dedicated clinical data. Pediatric drug development faces a fundamental paradox: it is ethically imperative to provide safe, evidence-based treatments, yet ethically and practically challenging to conduct large-scale, traditional clinical trials in children.

To bridge the gap in pediatric care, global regulations now require or incentivize studies in children. However, because traditional phase 1–3 trials are often impractical or unethical in pediatric populations, standard development methods frequently fall short.

In response, the FDA and the EMA have shifted their focus toward model-informed drug development (MIDD). Regulatory agencies now champion modeling and simulation not just as supportive data, but as pivotal evidence to justify pediatric dosing, trial waivers, and to inform labeling.

At Pharmetheus, we specialize in Population PK & PKPD, PBPK & QSP, and Clinical Trial Simulations to translate existing adult data and prior knowledge into precise pediatric strategies.

Our MIDD approach transforms pediatric development and filing:

• Optimize pediatrics dosage selection: We predict the first-in-pediatric dosage using translational modeling approaches, and optimize dosage with high confidence by leveraging our core expertise in population PK, PKPD, and PBPK.
• Support extrapolation and inform trial design: We justify the transition of efficacy data from adults to children when disease similarity is established –and similar response to treatment is expected– and use simulations to define dosages, sparse sampling schedules, and optimal sample sizes.
• Analyze and report pediatric clinical data: We analyze and report pediatric clinical data in a high quality and timely manner using the Pharmetheus PedDes workflow and our specialized data programming capabilities.
• Prepare pediatric filing: We use our dual expertise in MIDD and regulatory science to prepare briefing books and meeting material aligned with ICH E11, E11a, and ICH M15, and participate at regulatory meetings in person.

MIDD is no longer an alternative; it is now the modern regulatory standard to ensure that pediatric patients receive the right dose, at the right time.

In addition to expert knowledge and practical experience from numerous client projects (1-10), we have developed efficient internal ways of working, script, templates and workflow (11) to conduct robust analyses and submit the analysis outcome to authorities with confidence. 

• Accelerated market access: Streamline the path to pediatric labeling by replacing lengthy Phase 3 studies with robust modeling evidence.
• Ethical consideration: Minimize the number of pediatric subjects and invasive sampling procedures.
• De-risked dosing: Identify the optimal dose for neonates through adolescents with high precision to reduce the risk of sub-therapeutic or toxic exposures.
• Ensure regulatory compliance: Navigate the mandatory initial PSP (US) and PIP (EU) requirements with confidence. With MIDD integrated in your initial submissions, you provide the quantitative justification needed to reach an Agreed initial PSP or PIP.

Selection of affiliated publications and abstracts

• Jönsson et al., Population pharmacokinetic and dose-response models of nepadutant, a selective antagonist of the NK2 receptors, in infants with colic. Br J Clin Pharmacol. 2024 Sep 26.
• Bergstrand et al., Caplacizumab Model-Based Dosing Recommendations in Pediatric Patients With Acquired Thrombotic Thrombocytopenic Purpura. J Clin Pharmacol. 2022 Nov 29;62(3):409-421.
• Röshammar et al., Pharmacokinetic modeling and simulation support for age- and weight-adjusted dosing of dabigatran etexilate in children with venous thromboembolism. J Thromb Haemost. 2021 Mar 26;19(5):1259-1270.
• Holford et al., A physiological approach to renal clearance: From premature neonates to adults. Br J Clin Pharmacol. 2024 Apr;90(4):1066-1080.
• Kloprogge et al., Artemether-lumefantrine dosing for malaria treatment in young children and pregnant women: A pharmacokinetic-pharmacodynamic meta-analysis. PLoS Medicine. 2018 Jun 12;15(6):e1002579.
• van Schaick et al., Development of a population pharmacokinetic model of prucalopride in children with functional constipation. Pharmacol Res Perspect. 2016 Jun 1;4(4):e00236.
• Sjögren et al., A Physiologically-Based Pharmacokinetic Framework for Prediction of Drug Exposure in Malnourished Children. Pharmaceutics. 2021 Feb 2;13(2):204.
• Selvaggio et al., Physiologically based pharmacokinetic modelling to support design of microarray patches delivering antiretroviral drugs to HIV positive children. PAGE 32 (2024) Abstr 10890.
• Bardol et al. Fentanyl dosage for preterm infants suggested by a pharmacokinetic, -dynamic, and -genetic model. Pediatric Research. 2025; 97: 239-245.
• Bardol et al. Pharmacokinetic and Pharmacodynamic Modeling of Clonidine and Midazolam for Sedation in Pediatric Intensive Care. Pediatric Anesthesia, 2025; 35:1053–1062.
• Ibrahim et al., A workflow for evaluating and optimizing the designs of paediatric studies. PAGE 32 (2024) Abstr 11082.

Our commitment to advancing the field of pharmacometrics is demonstrated through our active participation in scientific research and publication. This work not only contributes to the broader scientific community but also ensures our partners benefit from the most current and validated methodologies.

Whether you are in early-stage strategic planning or preparing for regulatory submission, our experts are ready to discuss what adaptive methodology can be tailored to your clinical questions. 

Ready to transform your pediatric development and filing?

Contact us to discuss your needs

• Engage us for expert advice on strategic, regulatory, or technical matters at any stage of your study or program.
• Contract with us for milestone projects to perform specific analyses, such as a complete model-based bioequivalence analysis.
• Hire an embedded scientist to work as part of your team, either full- or part-time, giving you hands-on support guided by our senior consultants.
• Empower your in-house team with customized training workshops tailored to their specific background and needs.