Abstract 1023: Evaluating the impact of the RACE act: An interim analysis

Abstract Recent approvals of novel therapeutics, including targeted small-molecules and immunotherapies, have significantly impacted cancer care. However, these advancements have not easily translated to new treatment options and approvals for pediatric cancer patients. The Research to Accelerate Cures and Equity (RACE) Act was signed into law in 2017 to accelerate the availability of drugs for pediatric cancer patients by requiring all new adult oncology therapeutics also conduct pediatric studies if the molecular target is relevant to pediatric cancer, including therapeutics with an orphan drug designation. RACE requirements were implemented on August 18th, 2020, and we evaluated the impact of the RACE Act since implementation. We evaluated all new drug applications or biologics license applications submitted and approved by the FDA from August 18, 2019-August 18, 2021. Qualifying drugs were stratified by applications approved one year before RACE implementation and applications submitted and approved one year post-RACE implementation. FDA approval letters and review documents were used to obtain information regarding pediatric study requirements and orphan drug designation. Nineteen therapeutics were identified within the study period (63.2% approved pre-RACE implementation and 36.8% approved post-RACE implementation. Only 11.8% of agents approved were indicated for pediatric use at the time of initial application, and the majority (78.9%) of approved applications received an orphan drug designation. Prior to the RACE Act, pediatric studies were waived for orphan drugs, regardless of possible applicability of the agent to pediatric cancers. However, 91.7% of the therapies approved pre-RACE implementation had a relevant mechanism of action (MoA) that may have required pediatric study if the application was submitted after RACE implementation. After RACE implementation, 71.4% of the therapies received an orphan drug designation, with 60% requiring pediatric studies due to the relevancy of the MoA. Pediatric studies were waived or exempt for all therapies during the study period prior to RACE implementation. However, following implementation of RACE, 42.9% of approved drugs require pediatric studies. The remaining therapeutics approved post-implementation had waived pediatric study requirements due to studies being impossible or highly impracticable given the pediatric prevalence in the indication. The one-year anniversary of RACE implementation provides the opportunity to begin to evaluate the effectiveness of the approach and recognize opportunities to expand its reach in the future. We found an increase in required pediatric studies after implementation, with the largest effect seen in orphan-designated therapeutics that are no longer automatically exempt. However, many pediatric study requirements are still waived for therapeutics with relevant MoA, highlighting the opportunity for future policy modifications. Citation Format: Brittany A. McKelvey, Mark Stewart, Jeff Allen. Evaluating the impact of the RACE act: An interim analysis [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2022; 2022 Apr 8-13. Philadelphia (PA): AACR; Cancer Res 2022;82(12_Suppl):Abstract nr 1023.