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FDA RACE for Children Act: What You Should Know

Short for Research to Accelerate Cures and Equity, the RACE Act went into effect on August 18, 2020, and significantly increased the number of required studies devoted to pediatric cancer research, drugs, and therapies.

Originally enacted in August 2017, as a part of the Food and Drug Administration (FDA) Reauthorization Act (FDARA), the RACE Act is an amendment to the Federal Food, Drug, and Cosmetic Act (FFDCA) which was created to extend user-fee programs for drugs, medical devices, and biosimilars, as well as other biological products.

Key Factors of the FDA RACE for Children Act

The hope is that the RACE Act will level the playing field for children with cancer. Here is an overview of everything you need to know about the FDA RACE for Children Act and its impact on the future of cancer drug development.

  • The revised Research to Accelerate Cures and Equity (RACE) for Children Act expands treatment options for pediatric cancer patients by authorizing that any new oncology drugs, normally reserved for adults, will also have to be tested in children. As of August 18, 2020, RACE mandates that any original drug application or biologic must resemble the molecular targets that are relevant to one of the many childhood cancers being treated. The only time this wouldn’t be the case is if a waiver is granted due to the drug being exclusively intended for an adult cancer, like lung cancer, or the FDA determines that the molecular target is a factor in the growth or advancement of a pediatric cancer.
  • The RACE Act is not only an amendment to the FFDCA but an expansion of the Best Pharmaceuticals for Children Act (BPCA) of 2002 and the Pediatric Research Equity Act (PREA) of 2003. The BPCA provides additional marketing incentives to sponsors and pharmaceutical companies that voluntarily conduct pediatric clinical studies outlined by the FDA. While PREA was originally created to require drug manufacturers and pharmaceutical companies to include children in studies where the drug in question has the same adult indications and is expected to be used in a significant number of children.
  • This new legislation comes as a response to the advances made in molecularly targeted cancer drugs that, until recently, have largely been developed for adult oncology and not pediatric cancer treatments. New research suggests that malignancies that occur in children can contain the same molecular abnormalities as those found in adult cancers. This would suggest that new oncology drugs may prove effective in treating cancer in pediatric patients, regardless of the adult cancer indicators not occurring in the pediatric population.
  • The RACE Act puts an end to the orphan exemption that is outlined in The Pediatric Research Equity Act (PREA). PREA was enacted in 2003 and failed to extend pediatric research requirements for drugs for orphan diseases. Orphan diseases include tuberculosis, cystic fibrosis, and many pediatric cancers that affect fewer than 200,000 people in the United States each year.
  • The U.S. Food & Drug Administration (FDA) has compiled a list of both relevant and non-relevant molecular targets in an effort to keep sponsors informed. The FDA updates these lists frequently.

    These lists are helpful guides for sponsors, especially in the research phase of project development. It is still recommended that sponsors of targeted oncology drugs contact the FDA for detailed guidance on the impact that the RACE Act will have on bringing their drug to market.

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