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The Childhood Cancer Data Initiative Coordinated Pediatric, Adolescent, and Young Adult Rare Cancer Initiative

All childhood cancers are considered rare; however, about 11% are very rare cancers—less than two cases per million people are seen each year. These very rare childhood cancers may be hard to diagnose and lack effective treatments and standardized care. There is also little information or understanding of how or why these cancers occur and how they behave in a young person’s body over time.

Through collection of standardized data and community collaboration, the CCDI Coordinated Pediatric, Adolescent, and Young Adult Rare Cancer Initiative could advance our understanding of very rare childhood cancers and help improve outcomes for children, adolescents, and young adults (AYAs) with these cancers.

Which childhood cancers are very rare?

Very rare childhood cancers include many different histologies. Some examples of very rare cancers include adrenocortical, nasopharyngeal, thyroid, and any other (extra-cranial) carcinomas, as well as desmoplastic small round cell tumors, gastrointestinal or gonadal stromal tumors, neuroendocrine tumors, pancreatoblastoma, pleuropulmonary blastoma, and retinoblastoma.

What are the goals of this initiative?

The CCDI Coordinated Pediatric and AYA Rare Cancer Initiative aims to:

  • Build on knowledge and experience gained from other rare cancer programs.
  • Establish the structure and resources needed to study rare childhood cancers and develop effective treatments.

What is this initiative?

At the heart of the CCDI Coordinated Pediatric and AYA Rare Cancer Initiative is a longitudinal, observational study that will follow children and AYAs with very rare cancers over time. Data from this study could help make meaningful comparisons across multiple cancer types and identify molecular targets. These data could also inform treatment and care recommendations.

Additionally, the initiative will include a rare childhood cancer data registry and tumor boards.

Rare Childhood Cancer Registry

The primary goal of this initiative is to develop a registry containing a core data set on all rare cancer participants. It will include real-world data that can inform the design of rare childhood cancer clinical trials. For example, registry data for subsets of tumors could be used to create external control groups for trials.

Tumor Boards

The initiative will create national and international molecular tumor boards that will bring together disease experts, radiologists, pathologists, and genetic counselors to provide treatment recommendations. These tumor boards will be piloted with one or a few select cancer types initially and will collaborate with already established molecular tumor boards.

Who can participate in the longitudinal, observational study?

The study will begin enrollment in 2025. To join, a child or AYA must be diagnosed with a rare solid tumor. Enrollment will open to those with blood cancers in the future. All participants will also be enrolled in the CCDI Molecular Characterization Initiative.

A child or AYA may enroll from the Children’s Oncology Group Project:EveryChild, which is open to young people with cancer who are 25 years old or younger. If they are not being seen at a Children’s Oncology Group–affiliated hospital, they can refer themselves to the study. In this case, the child or AYA must be 39 years old or younger. Additional enrollment details will be available closer to when the study opens.

Where can you access data from the initiative?

Data generated and collected through this initiative will be available through the CCDI Data Ecosystem. More information about these data will be shared as the initiative progresses.

Who is CCDI collaborating with on this initiative?

To accomplish the goals of the CCDI Coordinated Pediatric and AYA Rare Cancer Initiative, CCDI is collaborating with organizations and initiatives in the United States and abroad. These include:

  • The Children’s Oncology Group
  • The NCI Center for Cancer Research Pediatric Oncology Branch
  • Cancer Moonshot℠

Other groups include advocacy organizations, national and international rare tumor efforts, regulatory agencies, and other consortia.

Who can you contact with questions about the initiative?

For questions about the CCDI Coordinated Pediatric and AYA Rare Cancer Initiative, contact Dr. Mary Frances Wedekind Malone at maryfrances.wedekindmalone@nih.gov.

For questions about CCDI, email NCIChildhoodCancerDataInitiative@mail.nih.gov.

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