The 2020 Pediatric Mandate That Shook Up Oncology Research
By Bhargavi Pandit, global head regulatory affairs, Galderma
For decades, clinicians have used extrapolations from adult doses and prescribed them to pediatrics. According to a 2008‑2012 review, approximately 50% to 75% of drugs used in pediatric medicine had not been studied adequately to provide appropriate labeling information — until 2020.
The FDA, EMA, and other health authorities declared that pediatric plans are no longer a late-stage formality but rather a prerequisite to adult development in many therapeutic areas, especially in oncology and targeted therapy.
When Pediatrics Became The Priority
The true inflection point began on August 18, 2020, when the U.S. RACE for Children Act under the FDA Reauthorization Act of 2017 came into full effect, and companies could no longer receive automatic pediatric waivers from the FDA for oncology solely based on an adult indication. With greater scrutiny on precision drugs and immunotherapies, the FDA published a list of relevant molecular targets that demonstrated activity in pediatrics and had the potential to generate evidence of efficacy in other pediatric tumors beyond the adult indication.
Across the Atlantic, EMA and its Paediatric Committee (PDCO) followed up with similar tightening measures. Between 2020 and 2023, which included more rigorous pediatric investigation plan (PIP) evaluations, fewer waivers and deferrals were granted, and there was increased scrutiny on sponsors to agree to pediatric investigations for novel therapies with potential. The EMA soon required sponsors to agree to a pediatric study plan (PSP) with study-related milestones in order to file a marketing authorization application for the adult indication.
Development programs had long anticipated routine pediatric waivers, especially in oncology, but were then confronted with mandatory early engagement, unplanned studies, and accelerated PIP/initial PSP submissions. Having in-house expertise and resources to design pediatric studies became challenging, especially for smaller biotech companies feeling the pressure of compressed timelines, increased costs, and operational challenges colliding with limited resources.
When the regulators’ thinking shifted, sponsor companies reacted with a mix of urgency and frustration. Many expressed concerns about committing to pediatric studies based on the relevance of mechanism of action data in adults, while others accelerated and embraced pediatric development by engaging early with relevant stakeholders. An important caveat to note is that the RACE/iPSP requirement applied to only original applications submitted on or after Aug. 18, 2020.
Drugs such as larotrectinib, capivasertib, crizotinib — all investigating benefits in adult cancer indications — had to quickly turn around and develop an initial pediatric plan and align with the FDA prior to filing for the adult marketing authorization after Aug. 18, 2020. Larger pharmaceutical companies could leverage their operational infrastructure and expand R&D resources to cover costs for pediatric development. In some cases, drugs like capivasertib used academic collaborations to lead their initial pediatric development. Meanwhile, for smaller biotech, this structural shift meant that they suddenly faced a new regulatory cost and risk that was previously avoidable. For firms with limited funding or limited pipeline breadth, this forced reprioritization and requesting additional funding from investors, delay, or even termination of some programs — or to defer pediatric studies indefinitely.
Pharma Soon Saw The Upsides Of A Pediatric Focus
Once the oncology industry settled into this new normal of including pediatric development as part of their overall strategy, sponsors started recognizing that these pediatric mandates are not simply a compliance check but an inflection point. Assigning resources for investigating pediatric-related tumors could provide drug developers with pediatric exclusivity, strengthen their competitive positioning, accelerate development, and expand the long-term value of their oncology assets. Companies understood the importance of investing earlier in translational biology to gain stronger adult and pediatric value stories and ultimately achieve better outcomes by recruiting adolescent patients from the beginning.
Sponsors also realized that early pediatric commitments could help differentiate assets in particularly saturated mechanisms, such as PARP, KRAS, and PIK3. For instance, if two companies are developing drugs in the same class and indication, the company with a well-thought-out, robust pediatric plan up front may hold more credibility with investigators, regulators, investors, and potential partners and be considered as having the more complete and responsible global strategy. With proactive investment in pediatric development, wherein the drug performs exceptionally well in early phases, pediatric investigation plans no longer become the rate-limiting step to accelerated filing for adult indications.
A few other upsides of incorporating pediatric development early in the regulatory and development strategy include building relationships and engaging with esteemed institutions such as Children’s Oncology Group (COG), Innovative Therapies for Children and Adolescents with Cancer (ITCC), St. Jude, and Pediatric MATCH networks. This helps sponsors access rapid trial startup models, tap into rare patient populations, and collaborate on translational research. In fact, robust investigatory sponsored studies could even be proposed as part of the initial PSP or PIP submissions. These partnerships may also lead to future adult collaborations or novel biomarker insights that ultimately help companies strengthen their pipeline. Companies can build a strong ecosystem around their asset, boosting visibility and accelerating trial completion to gain the advantage of speed to market in a rapidly evolving oncology landscape.
Agencies like the FDA also support early interactions with sponsors to understand assets in their pipeline and how the pediatric review division at the agency can support novel drug development in unmet need conditions. Companies that invest in building infrastructure to support pediatric PK/PD modeling and simulations for small cohorts and design trials for rare populations can strengthen confidence and credibility with the FDA and EMA.
For smaller companies that may view pediatric drug development as a hurdle, they can instead leverage their pediatric development readiness as a deal-making asset. When larger companies eventually look to acquire biotechs, one of the biggest attractions is a mechanism of action relevant in pediatrics with an agreed initial PSP (iPSP) with FDA or PIP with the EMA. In such situations, the acquiring companies have confidence in accelerating adult drug development because of its pediatric readiness that de-risks the asset and ultimately creates value for the shareholders and partners.
What The Pediatric Mandate Means For Future Oncology Drug Development
The pediatric mandate shake-up has undeniably reshaped the contours of oncology drug development. What began as a source of industry anxiety has emerged as one of the most transformative catalysts for scientific rigor, regulatory alignment, and long-term strategic thinking. Companies that once viewed pediatric obligations as a compliance burden are now leveraging them as engines of innovation — strengthening their translational foundations, accelerating global development, and building pipelines that reflect a more equitable commitment to all patients.
In many ways, pediatric oncology has become the new proving ground of regulatory excellence. Sponsors that step into this space with intention, transparency, and early planning are not just fulfilling a mandate; they are redefining what responsible, future-ready drug development looks like. The shift toward mechanism-based pediatric relevance has elevated expectations across the industry, forcing every company, regardless of size, to articulate clearer scientific narratives and make bolder, more integrated development decisions.
Ultimately, the true impact of these regulatory changes extends far beyond compliance timelines or PIP negotiations. It represents a fundamental shift in how we value pediatric patients in the global drug development ecosystem. By embedding pediatric strategy into the heart of oncology programs, the industry can move from a reactive posture to one of deliberate stewardship. And that shift is not only good for business; it is a moral imperative, too. As the FDA, EMA, and other agencies look to harmonize regulations, pediatric drug development will be at the epicenter of this change.
The companies that embrace this new reality will not only navigate regulatory complexity more effectively but will shape the future of oncology therapeutics irrespective of age and disease. When pediatric development stops being an afterthought and becomes a guiding principle, we do more than meet mandates. As science-driven organizations, we should deliver on the promise of innovation for the patients who need it most.
About The Author:
Bhargavi Pandit is a regulatory affairs leader with expertise in oncology and rare disease drug development. She currently serves as the global head of regulatory affairs for immunology and rare disease at Galderma, where she drives global strategies for breakthrough therapies. Previously, at AstraZeneca, she led global regulatory strategy for first-in-class oncology therapies, achieving FDA Fast Track, Project ORBIS, and Priority Review designations, and securing pivotal approvals. Her career spans leadership roles at Karyopharm Therapeutics, Boehringer Ingelheim, and GSK, where she shaped global submissions, pediatric oncology programs, and cross-functional development strategies. Recognized for her impact in the field, Bhargavi was honored with the Marquis Who’s Who award in 2024. She holds an M.S. in Regulatory Affairs from Northeastern University and a Bachelor of Pharmacy from Mumbai University and is passionate about translating complex regulatory science into actionable strategies that bring transformative therapies to patients worldwide.