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 virtual 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

2021 Symposium

NCI hosted the CCDI Symposium on November 9, 2021. Thirty-one expert speakers representing the childhood cancer research, care, and advocacy communities shared CCDI’s recent progress, identified next steps and priorities for the initiative, and listened to patients and advocates describe their needs and issues.

Access the recording.

Symposium Welcome

Then NCI Director Dr. Norman Sharpless opened the symposium with optimistic yet grounded remarks on the current state of childhood, adolescent, and young adult cancers, expressing hope that CCDI will contribute to progress in research and treatment. He shared his view that we are in a golden age in cancer research fueled by large amounts of data generated in the clinic and the lab. Challenges and barriers remain in collecting these data from every child diagnosed with cancer. CCDI aims to address some of these challenges to lead to new discoveries and decrease the burden of cancer for individuals, their families, and communities.

Naomi Bartley, an advocate, informaticist, and childhood cancer survivor, provided the keynote talk and graciously shared her story. She discussed her treatment, its ramifications, the impact cancer had on her life as well as her daughter’s, and the importance of the work being done under CCDI. Her perspective provided an important reminder of the long-lasting effects childhood cancer can have on families.

CCDI Progress

Speakers gave an overview of CCDI’s goals and successes to date.

A key objective for CCDI is the development of a federated infrastructure to help manage and share data, including a searchable catalog of childhood cancer data, tools, and resources. Progress was evident as researchers presented their efforts in improving data sharing, collection, and analysis.

Key Focus Areas

The symposium focused on three areas crucial to the success of CCDI and how we can learn from every child, adolescent, and young adult (AYA) with cancer.

Learn from Every Child and AYA: Molecular Characterization for Every Child Diagnosed with Cancer

Understanding the molecular characteristics of childhood cancers is vital for advancing precision care. Sequencing the genomics of every child’s cancer will enhance the current knowledge base on existing variants.

• To learn from every child with cancer, NCI established the Molecular Characterization Initiative, built on the Children’s Oncology Group’s Project:EveryChild, to perform molecular characterization on children and AYAs at diagnosis and provide information back to the child’s family and clinicians to help with treatment decisions.
• OncoKB, the first FDA-recognized database of cancer variants was created.
• Texas KidsCanSeq and Pediatric MATCH offered lessons learned from genetic testing, including the importance of having a genetic counselor on hand to deliver and discuss results or to address follow-up questions, whether those results are given in person or by telemedicine.

CCDI speakers also gave insight into which types of genetic testing are proving most useful and how they are managed and conducted at different institutions.

Extract Data from Electronic Health Records (EHRs) in Real Time

EHRs offer rich data that document someone’s full medical journey and long-term outcomes. By extracting data from EHRs, researchers can look at larger populations. This also helps engage every child and better represent underserved groups.

Engage with People with Childhood Cancer and Their Families

The symposium’s closing panel took a special focus on who this initiative will affect most directly: patients and their families. Themes included:

• the importance of making molecularly informed medicine available to all patients, no matter their insurance status, location, or treatment facility
• the need for tumor typing to be offered early, with the data available to help other families
• the opportunity to handle some clinical care and trials remotely, especially for those in underserved communities
• the importance of greater data accessibility and availability, not only to break down existing silos for future research, but so that the data can inform current treatment decisions and potentially improve outcomes

At the heart of CCDI is the desire to learn from and treat every child and AYA with childhood cancer. This includes underrepresented minority and underserved populations. Not all children and AYAs have equal access to care. The Children’s Oncology Group, an NCI-supported clinical trials group, operates state-of-the-art centers, but their facilities are not available in all parts of the country or to all patients with cancer. Intentional outreach activities need to be fully inclusive (racially, ethically, and culturally).

The CCDI Engagement Committee is an important part of CCDI to help build relationships with patients, families, and communities, especially those who are underrepresented in childhood cancer research.

Symposium Conclusion

CCDI has made significant progress over the past two years in developing a community of pediatric caregivers, data scientists, researchers, advocates, and patients and their families, all working toward a shared vision. The CCDI community has the potential to transform childhood cancer treatments and outcomes, and to act as a model for data sharing and research in adult cancers. In the coming years, CCDI aims to strengthen and grow its community while working towards its goals.

2019 Symposium

Scientific stakeholders and leaders from academia, government, industry, and advocacy organizations gathered in Washington, DC, July 29–31, 2019, for the first CCDI symposium—a scientific meeting that gained a common understanding of the current issues and opportunities in childhood cancer research that could be addressed through better data collection and utilization.

Poster Abstracts

NCI invited researchers to submit abstracts for poster presentations at the symposium. 

Areas of interest for poster submission included research, methodologies, and tools that focus on any of the following categories: 

1. scientific and clinical research data needs in childhood and AYA cancers 
2. establishing meaningful datasets for clinical care and associated research progress
3. building infrastructure to enable federation among disparate databases
4. extracting knowledge from data 
5. developing adequate and equitable policies for shared data resources

Symposium Materials 

Review the symposium agenda and access accepted poster abstracts. Note that abstracts have not been edited. Day 1, day 2, and day 3 recordings are also available.