With the passage of the Pediatric Research Equity Act (PREA) of 2003, drug manufacturers were required to submit an initial pediatric study plan (iPSP) prior to commencement of Phase 3 studies (or new drug application [NDA]/biologics license application [BLA] submission in the absence of a Phase 3 study) for studies involving a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration. The iPSP is expected to contain an assessment of safety and effectiveness of the investigational drug for the proposed indication in all relevant pediatric subpopulations. In certain situations, a deferral or waiver for conducting pediatric studies can be obtained.
Prior to the RACE for Children Act, the pediatric study plans for oncology drugs were generally proposals to request waivers for pediatric assessments because the adult cancer indications for which a drug was developed did not occur, or occurred only rarely, in pediatric patients, making pediatric studies impossible or highly impracticable. The RACE for Children Act amended PREA to require pediatric investigation of certain targeted cancer drugs based on molecular mechanisms of action rather than the clinical indication for original BLAs/NDAs submitted on or after August 18, 2020, unless a deferral or waiver is granted.
An iPSP for a molecularly targeted oncology drug should address the drug’s molecular target and its relevance to one or more cancers that occur in the pediatric population. An iPSP is expected to include the following elements:
- Description of the cancer(s) in the pediatric population for which the drug warrants early evaluation
- Overview of the drug product
- Overview of planned extrapolation of effectiveness to the pediatric population
- Planned request for drug-specific waivers and partial waivers with justification
- Planned request for deferrals of pediatric studies
- Tabular summary of proposed non-clinical and clinical studies
- Age-appropriate formulation including details of existing/planned excipients
- Non-clinical proof-of-concept studies (planned and completed)
- Data to support clinical studies in pediatric patients
- Planned pediatric clinical study or studies
- Timeline of the pediatric development plan
- Agreements for pediatric studies with other regulatory agencies
Our new white paper Understanding New FDA Guidance for Pediatric Oncology Studies: A Sponsor’s Guide to the FDA RACE for Children Act focuses on other considerations for developing an iPSP for a molecularly targeted cancer drug, including planned pediatric clinical studies, considerations for rare cancers, and early advice on pediatric development meetings, as well as conditions for obtaining planned waivers or deferrals. Click here to download.