P119: Early Insights: Impact of the RACE Act on U.S. Pediatric Oncology Regulatory Strategy
University of North Carolina/Glaxosmithkline United States
The objective is to evaluate the effect of the FDA Reauthorization Act of 2017 – Section 504 (RACE Act) on pediatric regulatory requirements by reviewing FDA-approved oncology products with molecular targets relevant to pediatrics.
Original NDAs/BLAs for oncology products with molecular targets likely to contribute to the growth/progression of pediatric cancer submitted to the FDA on or after August 18, 2020 were reviewed. All data collected were publicly available on the FDA website and analyzed using descriptive statistics.
Of the 47 marketing applications for oncology products filed on or after August 18, 2020 and approved by the FDA, only 9 (19%) were within the scope of the RACE Act (i.e., had a relevant molecular target and were original NDA/BLAs). 35 applications were supplemental applications of previously approved oncology products and were thus excluded from this review. There were 12 original applications, but 3 of those applications were for products targeting a non-relevant molecular target. Among the 9 relevant applications, 2 were for products that were neither granted waivers nor deferrals. Four full pediatric waivers and 3 partial waivers were granted. Of the 3 partial waivers, 2 were for patients <1 year of age and one was for neonate patients (0-28 days). The rationales for all full and partial waivers granted were “Necessary studies are impossible or highly impracticable.”
Deferrals were granted for 4 out of 9 applications for reasons including “The drug or biological product is ready for approval for use in adults before the pediatric studies are complete”, “Pediatric studies should be delayed until additional safety or effectiveness data have been collected”, and “There is another appropriate reason for deferral”. Post-marketing requirements for the applications that received deferrals included planned pediatric studies to determine the appropriate dose, safety, pediatric pharmacokinetics, tolerability, efficacy, and pediatric-specific pre-clinical models.
The purpose of the RACE Act was to expand treatment options for pediatric oncology patients by extending pediatric study requirements to original marketing applications for products with molecular targets relevant to pediatrics as well as previously exempt oncology products with orphan drug designations. This represents a shift in the Agency’s focus from clinical indication of the cancer drug to molecular mechanism of action. Since the implementation of the RACE Act in August of 2020, only 9 products approved by the FDA were within the scope of the legislation and had pediatric data publicly available.
While the present study was limited to the use of publicly available data, this review provides insight into the early phase of implementation of the RACE Act. Our preliminary review of these products determined that despite this legislation’s intent to expand pediatric study requirements to expedite the development of cancer therapies, many products are still being granted waivers and deferrals of pediatric studies. This observation suggests that conducting pediatric oncology trials may still prove to be difficult due to challenges in patient recruitment, formulation development, and lack of information on pediatric safety and efficacy data.
Sponsors are still required to submit pediatric study plans and provide evidence to justify any waivers or deferrals during the development of new molecular entities with relevant molecular targets. When developing the regulatory strategy for a product, sponsors should still consider timing and resources invested in their pediatric programs due to these requirements. As more oncology products are approved in the coming years, further effects of the RACE Act on the pediatric oncology drug development landscape will be ascertained.