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CCDI Activities Enhance NCI’s Childhood and AYA Cancer Research

, by Norman E. Sharpless, M.D.

Child Patient with Bear

The goal of NCI's Childhood Cancer Data Initiative is to expand the breadth and quality of the data on pediatric and AYA cancers and make the data more accessible and easier to use for researchers.

Credit: iStock

In July 2019, researchers, advocates, and many others came together to brainstorm ways to develop more efficient and more effective means of collecting, analyzing, and sharing the data needed to speed progress against cancers that occur in children and adolescents and young adults (AYAs).  

This effort served as one springboard for NCI’s planning of the Childhood Cancer Data Initiative (CCDI), a $50 million federal investment made in 2020, with an additional $50 million proposed each year for the next 9 years. 

Because cancers in children and AYAs are rare—representing approximately 4% of cancers diagnosed annually in the United States—there are many research challenges.

For example, tissue samples from this patient population are limited in availability. Additionally, the clinical and biologic data generated from these samples typically reside in different databases across the country and are not broadly available. This has made it extremely difficult for researchers to aggregate and use these samples and data to better understand childhood and AYA cancers—and to develop new ways to prevent, diagnose, and treat them. 

The ultimate aim of the CCDI, of course, is to make more rapid progress in improving treatments for childhood and AYA cancers. We believe we can achieve that goal by expanding the breadth and quality of the data accessible to researchers and making those data much easier to access and use.

At the core of this initiative are our efforts to optimize this research infrastructure by creating a data “ecosystem.” This ecosystem will seamlessly connect the repositories and registries where these data are stored, allowing scientists to learn from the information gathered from each child and AYA with cancer without having to seek it from multiple institutions or researchers. 

Childhood Cancer Data Initiative Working Group Report

NCI has been busy over the past year moving the CCDI forward. In November 2019, the institute convened an ad hoc working group of the Board of Scientific Advisors (BSA) to provide general guidance regarding future priorities for the initiative. 

In addition to members of the BSA, the group included several National Cancer Advisory Board (NCAB) members and other individuals with expertise in pediatrics, bioinformatics and clinical informatics, technology, and drug development, among other areas. The working group presented its report, which includes 24 specific recommendations, at the joint BSA/NCAB meeting on June 15, and it was unanimously accepted.

I am grateful to the CCDI working group for their diligence over the past several months in developing and drafting this important report. NCI leadership will carefully review the report and begin to make decisions about how to implement the working group’s recommendations. 

An update on those plans will be provided at the NCAB meeting scheduled for early September.

Ongoing CCDI Activities

Since the initiative’s launch last July, NCI has undertaken a range of research activities to lay the foundation for developing and supporting the CCDI. These efforts align with the BSA working group’s recommendations and are designed to enhance existing pediatric cancer and survivorship research activities, as well as support new ones.

CCDI activities currently underway include: 

  • Conducting a comprehensive review of existing childhood and AYA cancer data, data repositories, and analytic tools that can be connected under the CCDI
  • Developing the National Childhood Cancer Registry (NCCR), as part of the CCDI data ecosystem, to enhance access to patient-linked childhood and AYA cancer and survivorship data
  • Supporting ongoing research to develop a preclinical data commons—a platform for submitting, sharing, and analyzing data from studies involving cancer models (e.g., cell lines or animal research) that can help to prioritize which agents to pursue in human clinical trials for childhood and AYA cancer
  • Building the technical infrastructure of the data ecosystem, which will connect various types of cancer and clinical care data and tools, including the NCCR and preclinical data commons
  • Expanding comprehensive data collection to include more institutions engaged in childhood and AYA cancer and survivorship research
  • Continuing to enhance data sharing and publication policies to promote open access to data and journal articles without paywalls

The CCDI extends NCI's ongoing support of childhood and AYA cancer research, as well as complements efforts authorized by two pieces of federal legislation: the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act and the Research to Accelerate Cures and Equity (RACE) for Children Act

The STAR Act encourages NCI to enhance biospecimen collection, biobanking, and related resources for childhood and AYA cancer patients and survivors, with an emphasis on those cancer types for which current treatments are least effective. CCDI data resources will support and enhance access to and use of data generated from a broad range of childhood and AYA cancer research collected through STAR Act-supported projects.

The RACE for Children Act directed NCI and the Food and Drug Administration to solicit feedback from physicians, researchers, patients, and other stakeholders to develop a list of molecular targets relevant to the growth and progression of a childhood cancer. CCDI activities will respond to mandates in the RACE for Children Act through research and data resources that validate these molecular targets, and further contribute to the development of safe and effective therapies for childhood and AYA cancers.  

The CCDI also builds on prior NCI programs, such as TARGET and Pediatric MATCH, which employ molecular and targeted clinical approaches to generate critical data sets that catalog the molecular changes and treatment responses in children with several types of cancer. These programs have increased our understanding of how these cancers develop, thereby improving the accuracy of diagnosis and classification, and identifying new candidate molecular targets for better treatments. I’m confident that CCDI will further these successes.

We believe the CCDI will have a great impact on our ability to prevent, diagnose, and treat children, adolescents, and young adults with cancer. At the same time, we believe what we learn from CCDI will serve as a model to improve the use and sharing of data for adult cancers as well as other diseases. 

I look forward to updating the community on CCDI activities via Cancer Currents and social media. You can also stay current on our efforts by going to the CCDI page on and by signing up for email updates