Shaping the Future of Pediatric Formulation Development

The U.S. Food and Drug Administration (FDA) in collaboration with the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), the IQ Pediatric Consortium, and European Paediatric Formulation Initiative (EuPFi) will host a two-day in-person workshop: Shaping the Future of Pediatric Formulation Development.

Recent progress and advancements in drug delivery technologies and formulation sciences have created new opportunities for developing better pediatric medicines. However, multiple challenges and hurdles need to be overcome before these technologies can become fully available to children.

By bringing together the pediatric community including pharmaceutical scientists, regulators and clinical counterparts from diverse functions, this workshop is intended to shed light on the barriers to and opportunities for patient-focused pediatric formulations. Advancing pediatric formulations will require sharing and building on experiences from patients/caregivers, industry, academia, and regulators. It is hoped that the participants will better understand the principles, gaps, technical, and process challenges encountered during formulation and dosage product development as well as considerations for regulatory assessments and ultimately policy development.

Funding Statement

This workshop is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award to the Center of Excellence in Regulatory Science and Innovation, U01FD005946 totaling $10,000 funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. government.

Info: Patient-centric product development in the context of pediatric formulation means using a user-focused approach that understands the physiological, developmental and psychological needs of the pediatric population that spans from neonates to adolescence. This translates into tailoring dosage forms to address factors such as dosing flexibility, taste-masking and swallowing abilities across this age group and ascertain that caregivers can deliver these medications where needed to ensure adherence to dosing regimen to impact health outcomes. This concept also applies to easing the burden of clinical studies and arriving at appropriate dosing regimens with minimal hardships on this vulnerable population. This session will present perspectives on some of these aspects to promote understanding and generate discussion on some of these key topics.

Info: This session will focus on key aspects of developing mini tablets as a dosage form for pediatrics. This session will provide a forum to discuss acceptability of mini tablets across pediatric age groups, inconsistencies in nomenclature, manufacturing and packaging challenges and limitations, and strategies for identifying appropriate food vehicles.

Info: This session features presentations on the assessment of excipients for pediatric formulations, including tools such as the IID and ELSA, delivered by FDA experts. Industry perspectives are provided through talks on challenges and considerations in selecting excipients for pediatric products, including the use of the PERA tool. Additional insights focus on excipient needs for special populations—such as neonates. The program concludes with a breakout session on the PERA tool and a forward looking panel discussion on future needs, research priorities, and regulatory challenges in pediatric excipient development.

Info: Since our last pediatric workshop in 2019, we have seen transformational innovations in drug delivery systems, the evolution of complex biologics, as well as new techniques for gene editing and gene therapy. Furthermore, recent advances in AI/ML methodologies, including agentic AI, are having a broad impact on drug discovery and development. In this session, we will discuss these recent trends and how they will shape the future of developing better drugs and delivery systems for children.

Info: This session will look towards establishing potential collaboration opportunities through shared understanding of the work done by various organizations such as industry, academia, philanthropic organizations or governments. The distinguished panel members from each of these organizations will share their perspectives through panel discussion. Workshop participants are encouraged to share their questions even ahead of time to enable productive, focused discussion on key topics as they relate to pediatric formulation development.

Discussion Points: innovation, acceleration, coordination, international harmonization, incentives

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