Calling for an annual FDA report on the RACE for Children Act

 

The Food and Drug Administration’s (FDA) Oncology Center of Excellence recently hosted its 2024 Pediatric Oncology Forum, an opportunity for members of the advocacy community to hear from the FDA and then to share perspectives on current issues affecting patients and their families.

As CEO of Children’s Cancer Cause, I attended the Forum and provided remarks on a topic that we’ve been closely involved with for a very long time: the passage, implementation, and monitoring of the RACE For Children Act. I wanted to share some of my remarks here so you can also follow along with this issue and what we are doing at Children’s Cancer Cause to help ensure that policies and advocacy that resulted in enactment of the RACE Act will advance and bring new therapies to kids as quickly as possible.

The acronym in this legislation stands for Research to Accelerate Cures and Equity. This legislation was passed into law in 2017 and went into effect in August 2020. Children’s Cancer Cause helped lead the charge to pass this legislation to ensure that potentially promising new oncology drugs in development for adults would be evaluated early in children with cancer. The RACE Act requires that new adult anti-cancer drugs and biologics directed at molecular targets that are substantially relevant to pediatric cancers must be evaluated for tolerability, dosing, and preliminary effectiveness in children.

The RACE for Children Act was developed as a strategy to assess whether targeted cancer therapies for adults could increase survival rates for children, especially those with resistant or refractory tumors and those with lifelong health impairments resulting from decades-old chemotherapy treatments. With rapid advances in tumor biology and over 200 types of childhood cancer, parents, pediatric oncologists, and patients desire early access to appropriate modern cancer therapies.

Children’s Cancer Cause is now urging that the FDA initiate a recurrent formal annual review of the Agency’s implementation of the RACE for Children Act to determine its impact on pediatric oncology drug development.

GAO Report January 31, 2023 Cover Page

Cover page of 2023 GAO Report

Data reported by the Government Accountability Office (GAO) in January 2023 - covering the first two years of the RACE Act (2020-2022) - showed that the approval of five cancer agents for adults were associated with requirements for pediatric studies. In the same period, FDA received 85 initial pediatric study plans.

While the GAO’s performance audit is a careful early analysis, questions about the intent of the RACE Act on improving outcomes for children remain. We believe the FDA is obligated to answer these questions annually.

Examples in an FDA annual report might include:

  • Sponsors’ time taken to start and complete postmarketing studies after an adult approval;

  • Whether sponsors request Common Commentary mechanisms to address regulatory challenges;

  • Whether the Agency issues noncompliance letters for unnecessary delays in conducting pediatric studies (per requirement under the Pediatric Research Equity Act);

  • FDA’s response when different sponsors propose to conduct pediatric studies within the same therapeutic class.

An annual FDA report on implementation of the RACE for Children Act would increase transparency for all stakeholders committed to new therapeutic options for children, including pediatric oncology investigators in academia and industry, European regulators, and patient advocates.

We concluded our remarks by assuring the FDA that Children’s Cancer Cause will continue to work with Congress to enhance the Agency’s resources to meet the increased workload necessary to fulfill the RACE law’s requirements. Additionally, we will continue to advocate for FDA’s efforts to improve outcomes for children with cancer. We appreciate the Agency’s continued work on Cluster Calls, Common Commentaries and Guidances to promote efficiency on international collaboration of global pediatric oncology clinical trials.

We are hopeful that the FDA will evaluate and implement the formal review mechanism to enhance the understanding and effectiveness of the RACE Act.

 
PolicySteve WosahlaRACE Act, drug development, new therapies