Medical and Regulatory Affairs

5 Things to Know About the FDA RACE for Children Act

The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a particular childhood cancer. Enacted August 18, 2017, as part of the Food and Drug Administration (FDA) Reauthorization Act (FDARA), it amends the Federal Food, Drug, and Cosmetic Act (FFDCA) to revise and extend the user-fee programs for drugs, medical devices, generic drugs, and biosimilar biological products and for other purposes.

Here are five things sponsors of pediatric oncology studies should know about the FDA RACE Act. To learn more about the legislation and the FDA’s accompanying draft guidance, download our white paper Understanding New FDA Guidance for Pediatric Oncology Studies: A Sponsor’s Guide to the FDA RACE for Children Act.

1. RACE sets new requirements for pediatric oncology studies starting this August.

RACE requires that any original new drug application or biologics license application submitted on or after August 18, 2020, for a new active ingredient must contain reports of molecularly targeted pediatric cancer investigations (unless a deferral or waiver of that requirement is granted) if the drug is:

  • Intended for the treatment of an adult cancer, and
  • Directed at a molecular target that the FDA determines to be substantially relevant to the growth or progression of a pediatric cancer.

2. The RACE Act expands upon previous legislation and policies designed to encourage pediatric drug development.

The Best Pharmaceuticals for Children Act (BPCA) of 2002 provides an incentive of additional marketing exclusivity to sponsors who voluntarily complete pediatric clinical studies outlined in a written request issued by the FDA, and the Pediatric Research Equity Act (PREA) of 2003 authorizes the agency to require drug manufacturers to complete studies in children for the same adult indications when the drugs are expected to be used in a substantial number of children.

3. The legislation comes as a response to a transformation of the paradigm of cancer drug development with molecularly targeted drugs.

Advances seen in the treatment of adult oncology indications have rarely been extended to development of pediatric cancer treatments. But extensive research has demonstrated that malignancies occurring in children and adolescents can harbor the same molecular abnormalities as those found in adult cancers, indicating that the new targeted oncology drugs may prove effective in treating pediatric patients with cancer, even if the adult cancer indication does not occur in the pediatric population.

4. RACE eliminates the orphan exemption for pediatric studies for cancer drugs directed at relevant molecular targets.

Under the PREA orphan exemption, PREA does not apply to any drug application for an indication for which orphan drug designation has been granted when that application would otherwise trigger PREA. However, the RACE requirement for pediatric investigation applies even if the adult cancer indication does not occur in the pediatric population and the drug is for an adult indication for which orphan designation has been granted.

5. The FDA maintains lists of both relevant and non-relevant molecular targets to provide further guidance to sponsors.

In collaboration with the government, academic and industry experts, and advocates, the FDA established, published, and updates two lists:

The lists are a guide for sponsors as they consider development plans for new targeted drugs and early pediatric assessments in light of the amended PREA provisions. However, sponsors of molecularly targeted oncology drugs are encouraged to seek early advice from the FDA.The passage of FDARA is a significant milestone in promoting pediatric cancer drug development that is bound to have significant implications on how sponsors plan and prepare for NDA/BLA filing of molecularly targeted oncology drugs. To learn more, download our white paper Understanding New FDA Guidance for Pediatric Oncology Studies: A Sponsor’s Guide to the FDA RACE for Children Act today.