The RACE Act has successfully increased the number of pediatric clinical trials that must be conducted for cancer therapies approved in adults, according to research presented in a poster at the AACR Annual Meeting 2022.
The RACE Act took effect on August 18, 2020. The goal of the act was to accelerate the availability of drugs for pediatric cancers by requiring that all new cancer therapies approved for adults be evaluated in pediatric studies if the molecular target is relevant to pediatric cancer, and this includes therapies with an orphan drug designation.
Researchers analyzed the impact of the RACE Act by examining all new oncology drug applications or biologics license applications approved by the US Food and Drug Administration (FDA) from August 18, 2019, to August 18, 2021.
Nineteen therapies were identified during the study period — 12 approved pre-RACE and 7 approved post-RACE implementation.
In the pre-RACE period, none of the approved therapies required pediatric study. For 83.3% of the applications, this was due to orphan drug designation. The researchers noted that 91.7% of the therapies approved pre-RACE had a mechanism of action that would necessitate pediatric studies under the RACE Act.
After the RACE Act was implemented, pediatric studies were required for 42.9% of approved applications, and 100% of required pediatric studies were for orphan-designated therapies. The remaining therapies approved post-RACE had waived pediatric study requirements due to studies being “impossible or highly impracticable,” given pediatric prevalence.
“We were excited to see, even in the first year after implementation, a significant increase in required pediatric studies, especially the significant effect of closing the orphan-designation loophole,” study author Brittany Avin McKelvey, PhD, of Friends of Cancer Research in Washington, DC, said in a statement.
Dr McKelvey and colleagues also highlighted a specific example of how the RACE Act is resulting in more studies for pediatric patients. In April 2020, the FDA approved pemigatinib to treat patients with cholangiocarcinoma. The drug, which targets FGFR2 alterations, received orphan designation. Therefore, at the time, a pediatric study was not required.
Just over a year later, in May 2021, the FDA approved infigratinib, which also targets FGFR2 and also received orphan designation. However, because of the RACE Act, the drug must now be studied in pediatric patients with advanced or metastatic solid tumors with FGFR2 alterations.
“We still have many years until we will be able to see if the required pediatric studies lead to actionable data that can support labeling modifications and inclusion of information for appropriate use in pediatric patients,” Dr McKelvey said. “The first step, which RACE has helped achieve, is to encourage study of novel adult therapies in pediatric patients. The long-term measure of success will be if these studies result in patient benefit and approval for pediatric patients.”