On May 11, 2021, the subcommittee will discuss the development and successful implementation of the Pediatric Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) as a tool for eliciting the patient’s voice in oncology clinical trials to more accurately determine tolerability and toxicity of drugs under investigation. The subcommittee will also address the challenges of capturing this type of data across the age spectrum of the pediatric population and possible generalizability of the data. It will consider approaches to address concerns about excluding the patient voice of young children deemed incapable of self-reporting. The subcommittee will also focus on approaches to investigators and commercial sponsors to use the Pediatric PRO-CTCAE in toxicity assessment moving forward.

On May 12, 2021, the subcommittee will discuss real-world evidence (RWE) for regulatory use in pediatrics, real-world data (RWD) resources, and RWD and RWE to advance pediatric safety assessments of oncology drug products in children within the context of the FDA framework for RWE. Potential data sources and publicly available platforms, including those made possible through the development and implementation of the National Cancer Institute’s Childhood Cancer Data Initiative, will be discussed. The potential for use of data sources to construct external controls to evaluate effectiveness of investigational products will be considered given the frequent dependence on single arm studies due to extremely small study populations, now exaggerated by molecularly defined subtypes of the rare cancer types that occur in children.