Past Childhood Cancer Data Initiative Symposiums

2023 Symposium

On March 24, 2023, NCI hosted the Childhood Cancer Data Initiative (CCDI) Annual Symposium. More than 800 researchers, clinicians, survivors, and advocates from the community gathered to discuss how enhanced data connection and sharing can address current issues and possibilities in childhood cancer research. Dialogue between speakers and attendees, both virtual and in-person, reviewed CCDI’s accomplishments and identified challenges and priorities that lie ahead.

Access the event presentation and event recording.

Symposium Welcome

CCDI Scientific Director Dr. Gregory Reaman opened the symposium by welcoming all virtual and in-person attendees and encouraging their engagement and input. Speaking on behalf of NCI Director Dr. Monica Bertagnolli, he shared her belief that CCDI demonstrates the potential power of data-sharing and collaboration to transform research and care not only for childhood cancer but for all people with cancer. 

Dr. Jaime Guidry Auvil, director of NCI’s Office of Data Sharing, and Hanna Jorgenson, a childhood cancer survivor, delivered joint keynote addresses that provided both a researcher and patient perspective. Dr. Guidry Auvil explained the power of data sharing and how, when data are made available to a range of experts, isolated data points can become collective knowledge that propels childhood cancer research forward and improves outcomes for children, adolescents, and young adults (AYAs) with cancer. 

Hanna Jorgenson discussed her osteosarcoma treatment and the long-term effects it had on her appearance and life. A decade later, at her final oncology check-up, her doctor mentioned that, because she also had an adrenal tumor in childhood, she should be tested for Li-Fraumeni syndrome. Just when Hanna thought her cancer journey had ended, the test came back positive. She has used this knowledge to be proactive about her health, and her story demonstrates how widespread knowledge and data sharing among researchers, doctors, and patients can empower survivors and ensure that doctors provide the appropriate long-term care to patients.

CCDI Progress

Speakers summarized key areas of CCDI’s progress, including: 

• The Molecular Characterization Initiative, which in its first year enrolled more than 1,000 children and AYAs newly diagnosed with brain tumors, soft tissue sarcomas, or rare cancers. 
• The CCDI Data Ecosystem, which also includes platforms and tools that researchers, doctors, and patients can use to access and explore childhood cancer data. Tools include the Childhood Cancer Data Catalog, National Childhood Cancer Registry and NCCR*Explorer, and Molecular Targets Platform.  
• The CCDI Hub, where users can access the Data Ecosystem.
• The CCDI Participant Index, which, once available, will help researchers study cohorts by linking patient data from many institutions to a single ID that will be easily searchable, enhance interoperability of data, and preserve patient privacy. 
• A National Initiative for Rare Cancers in Children and AYAs, which is in the early stages of development. The proposal includes plans for a rare pediatric cancer registry, designs for observational and interventional studies, participant recruitment, national tumor boards, and collaborative rare tumor clinics for patients and families.

Key Focus Areas

The symposium focused on three areas of opportunity for CCDI: 

1. cohorts for clinical and translational research 
2. clinical data and annotation 
3. accelerating clinical trials in childhood cancer 

Cohorts for Clinical and Translational Research

Studying childhood cancer involves significant challenges, including issues with collecting patient-reported outcomes and precise treatment and care data. Because childhood cancers are rare, longitudinal observation trials must enroll smaller groups—or cohorts—of children and AYAs with different types of cancers. Well-designed cohort studies could provide strong and reliable data to inform policy and, ultimately, decisions affecting patient health.  

Speakers agreed that national and international collaboration among childhood cancer research and care institutions to create focused cohorts is crucial for gathering as much data as possible on these rare diseases. The CCDI Molecular Characterization Initiative can play a role in collecting data that will be indispensable for organizing cohorts and learning more about soft tissue sarcomas, brain tumors, neuroblastoma, and extremely rare cancers in children and AYAs. 

Another critical tool for building cohorts is the forthcoming CCDI Participant Index. With individual patient data from various institutions linked to a single ID, researchers will be able to study the natural history, outcomes, and long-term effects of childhood cancer and its treatments.

Clinical Data and Annotation

To learn from every child and AYA with cancer, data must be collected and recorded in a way that can be easily understood, searchable, and extracted by many different users; however, no standards currently exist for annotating clinical data from electronic health records (EHRs). 

Standardizing EHR data and using them to track treatments and outcomes can help create a clear picture of a person’s entire cancer experience. Extracting clinical data from EHRs can provide researchers with real-world datasets for understanding how a certain course of treatment affects outcomes. Speakers agreed that standards for accurately collecting relevant data and extracting both structured and unstructured data from EHRs will be critical for making use of this valuable tool. They also discussed the possibilities and challenges of automated EHR data extraction using pilot programs such as ExtractEHR. 

Before people with cancer can share their data with researchers or participate in clinical trials, they must provide consent. Consent can be a barrier to data sharing. It often happens at diagnosis, an already difficult time for parents of children with cancer, and language in consent forms can be hard to understand. In addition, consents are distributed across various institutions where the patient receives care and are associated with numerous clinical trials and biological studies. As such, researchers may not have easy access to all consents. 

Speakers brought to light the need for “computable consent,” where patient privacy is assured and data sharing preferences for clinical and research purposes are electronically recorded and machine-readable for easy search and access by researchers and doctors. They also discussed projects working toward more transparency and engagement in the consent process, such as the Alliance Participant Engagement Portal and the ONC LEAP Computable Consent Project.

Accelerating Clinical Trials in Childhood Cancer

Because childhood cancer is rare, clinical trials can take years to enroll a small number of patients, and participants often must meet strict criteria to be accepted for a trial. Drug approvals based on results can take many additional years, time that children with cancer don’t have.

Speakers addressed possibilities for speeding up clinical trials and drug development for children with cancer. These included well-designed externally controlled trials; trials using real-world data and real-world evidence; and decentralized, global enrollment onto trials. Panelists agreed that the current model for clinical trials must be adjusted to more efficiently investigate childhood cancer and assure timely access to safe and effective cancer drugs.

Breakout Sessions

Following the symposium, six breakout sessions were held in person on March 25, and virtually on March 28–30, 2023, to discuss topics raised at the main symposium event. See below for presentations and recordings from the virtual sessions. Each presentation also includes a summary of the corresponding in-person session. 

1. Molecular Characterization Initiative and the potential for future cohort studies
   Presentation • Recording
    
2. Patient and family perspectives on computable consent and CCDI Participant Index
   Presentation • Recording
    
3. Electronic health record data extraction: current status and continuing challenges
   Presentation • Recording
    
4. External controls for pediatric cancer clinical trials
   Presentation • Recording
    
5. Collaborations and transformative research opportunities using data available through the CCDI ecosystem
   Presentation • Recording
    
6. Observational studies and novel interventional approaches for rare pediatric cancers 
   Presentation • Recording