By Bhargavi Pandit, global head regulatory affairs, Galderma
For decades clinicians often extrapolated adult doses for pediatric patients. A 2008–2012 review found that roughly 50% to 75% of drugs used in pediatrics lacked adequate study to support appropriate labeling until regulatory changes in 2020 reshaped practice.
The U.S. RACE for Children Act, fully effective Aug. 18, 2020, removed automatic pediatric waivers for oncology when the adult indication was the only basis. The FDA published a list of molecular targets with pediatric activity and the potential to show efficacy across pediatric tumors. The EMA and its Paediatric Committee applied similar tightening, requiring pediatric study plans with milestones before adult marketing applications in many cases.
Between 2020 and 2023 regulators granted fewer waivers and deferrals and increased scrutiny on pediatric investigation plans (PIPs) and pediatric study plans (PSPs). Sponsors faced mandatory early engagement, accelerated submissions, and unplanned studies. Larger companies could allocate resources or partner with academic groups to meet requirements; smaller biotechs often confronted compressed timelines, higher costs, and operational strain, leading some to reprioritize, seek additional funding, delay programs, or discontinue development.
The RACE/iPSP requirement applied to original applications submitted on or after Aug. 18, 2020. Drugs including larotrectinib, capivasertib and crizotinib had to develop initial pediatric plans and align with the FDA before filing for adult marketing authorization after that date.
Over time industry perspective shifted. Sponsors began to see pediatric mandates not merely as regulatory obligations but as strategic opportunities. Early pediatric development can yield pediatric exclusivity, strengthen competitive positioning, accelerate overall development and increase long-term asset value. Investing earlier in translational biology and including adolescents in trials from the outset can produce stronger adult and pediatric evidence and prevent pediatric plans from becoming bottlenecks to accelerated adult filings.
Early pediatric commitments also help differentiate assets in crowded mechanisms such as PARP, KRAS and PIK3. A clear, robust pediatric plan can enhance credibility with investigators, regulators, investors and partners. Partnerships with networks and institutions such as the Children’s Oncology Group, ITCC, St. Jude and Pediatric MATCH enable faster trial starts, access to rare patient populations and collaborative translational research. Robust sponsored studies can be incorporated into initial PSP or PIP submissions.
Regulatory agencies have encouraged early interactions to help sponsors and pediatric review divisions align on novel development paths. Building capabilities in pediatric PK/PD modeling, simulations for small cohorts and trial design for rare populations strengthens sponsor credibility with regulators and supports more efficient development.
For smaller companies, pediatric readiness can be a deal-making asset. Assets with mechanisms relevant in pediatrics and an agreed initial PSP or PIP can be more attractive to potential acquirers, de-risking the asset and increasing shareholder value.
The pediatric mandate has changed oncology drug development from a compliance-driven exercise to a driver of scientific rigor and long-term strategy. Mechanism-based pediatric relevance has raised expectations across the industry, requiring clearer scientific narratives and integrated development decisions regardless of company size.
Embedding pediatric strategy into oncology programs elevates the value placed on pediatric patients and shifts development toward deliberate stewardship rather than reactionary compliance. Companies that integrate pediatric planning early will be better positioned to navigate regulatory complexity and to shape the future of oncology therapeutics across age groups.
About the author: Bhargavi Pandit is a regulatory affairs leader specializing in oncology and rare disease drug development. She is global head of regulatory affairs for immunology and rare disease at Galderma. Previously she led global regulatory strategy for first-in-class oncology therapies at AstraZeneca and has held roles at Karyopharm Therapeutics, Boehringer Ingelheim and GSK. She holds an M.S. in Regulatory Affairs from Northeastern University and a Bachelor of Pharmacy from Mumbai University.
Leave a Reply