PDUFA VIII RECOMMENDATIONS
July 28, 2026
The Prescription Drug User Fee Act (PDUFA) presents an opportunity every five years to improve regulations of oncology products as applied to children and adolescents by attaching amendments to this must-pass regulation. It currently expires in September 2027. The recommendations below focus on improving FDA’s implementation of the Research to Accelerate Cures and Equity (RACE) for Children Act (RACE Act) and the Pediatric Research Equity Act (PREA).
The RACE Act requires adult cancer drugs that are molecularly relevant in pediatric cancers be evaluated in children. Although the RACE Act has stimulated an increase in pediatric oncology studies, sponsors do not conduct these studies until an adult oncology product is approved.
PREA currently requires that pediatric studies must be conducted only when the same indication occurs in children and adults.
RECOMMENDATION #1
FDA should require sponsors, under the RACE Act, to submit plans, strategies, study designs and milestones with timelines for pediatric oncology after filing with FDA the first dosing and safety adult studies.
Currently, sponsors are required to submit individual Pediatric Study Plans (iPSPs) within 60 calendar days after an end-of-phase 2 meeting for an adult product. This time interval can unnecessarily delay the onset of study of a drug’s effect on children. If sponsors were required to start their pediatric studies prior to the approval of the adult agent, earlier planning and strategies for assessing the safety and efficacy of pediatric drugs would hasten children’s access.
RECOMMENDATION #2
FDA should require sponsors to evaluate pediatric trials for all adult agents that are biologically relevant to pediatric disease. Following agreement and a trial has begun, sponsors should be required to supply sufficient drugs for pediatric testing in academic, public and private settings best positioned to evaluate agents.
Science has changed since PREA was codified. Research now reveals similar molecular and genomic features for certain adult and pediatric diseases, requiring that the PREA “indication” idea be updated as it applies to the similarity of adult diseases in childhood. Data show that about 20% of new drugs are exempt from pediatric testing requirements because the adult condition does not occur in children, despite having use to treat diseases in children.
PREA should be updated to ensure that pediatric studies are required for adult drugs designated for rare and orphan conditions, including through removal of orphan exemptions for supplemental indications. This recommendation would expand pediatric evidence generation by ensuring that drugs with potential relevance to pediatric cancers or related health needs in children undergoing treatment for or in remission from pediatric cancer are no longer excluded from study requirements. Similar to Recommendation 1, sponsors should include plans, milestones and strategies for conducting pediatric studies of biologically relevant adult agents.
RECOMMENDATION #3
FDA Should Issue a Report to Congress Every Five Years on the Planning, and Initiation of Pediatric Studies of the RACE Act for Children Act as Well as Require Pediatric Testing for All Biologically Relevant Adult Agents.
The purpose of requiring FDA to formally Report to Congress is to track and document for public assessment the application of modern scientific insights to the care and treatment of children. Data have been reported on RACE Act studies conducted after approval of adult cancer drugs, some of which are novel oncology agents. The FDA Report to Congress should include details about the timing and progress about the conduct of all pediatric studies, including, for example:
• The timing of the planning and onset of pediatric studies in relation to the timing of FDA’s review and approval of an adult oncology product;
• The pace of pediatric patient accrual to RACE Act studies;
• The number, type and pace of pediatric safety and efficacy studies conducted beyond fulfilling RACE Act requirements;
• The timing of the clinical application of such studies in relation to RACE study onset;
• A tally and analysis of noncompliance letters for unnecessary delays in conducting required pediatric studies;
• A description of sponsors’ progress in conducting, as appropriate, all pediatric studies of adult drugs.
