CONTEXT: As promised, we will keep you up to date on the FDA guidance on RWE in pediatric oncology. It seems the guidance will be available soon, but following a 2-day meeting, the Pediatric Oncology Subcommittee brought into question the role of RWE in regulatory decision-making. Looks like a bumpy road ahead for RWE.
IMPACT: High
READ TIME: 2 mins
Quality Level Mean [1 – 10]: 8
1. “In pediatric oncology, the questions raised by the committee comprised current and future resources for real-world data, the FDA framework on the use of real-world data to aid in regulatory decision-making, the limitations of these data sets, and how to understand adverse events (AEs) in real-world patient populations.”
2. “On the second day of a 2-day meeting of the Pediatric Oncology Subcommittee of the FDA’s Oncologic Drug Advisory Committee (ODAC), members and guests brought into question the role of real-world data in regulatory decision-making for pediatric oncology drugs.”
3. “Outside of clinical trials, data collected from patients treated in clinics across the country provide real-world evidence that may fill in information gaps about oncologic drugs.”
4. “Although not specific to pediatric cancers, experts believe the use of the framework to draft FDA guidelines around the use of real-world evidence in pediatric oncology is warranted.”
5. ““Real-world data from children and adolescent with cancer can be obtained from multiple sources in the potential for use as well-characterized and appropriately collected in the standardized data to create sufficient evidence,” said Gregory H. Greaman, MD, associate director for Oncology Sciences at Center for Drug Evaluation and Research at the FDA, during a presentation.”