The Data Mine: How Real-World Evidence is Rewriting Pediatric Cancer Trials

WASHINGTON, D.C. – November 10, 2026 — Data has long been the currency of the modern global economy, but in the realm of pediatric oncology, it is rapidly becoming the most critical infrastructure for survival. Today, Blood Cancer United announced a strategic alliance that underscores this shift. Partnering with the Rally Foundation for Childhood Cancer Research and health technology company Tempus, the organization has launched Dare to Dream REALworld: AML. It is an initiative that treats the electronic health records of children with acute myeloid leukemia (AML) as a foundational resource to accelerate the development of new therapies.

Historically, the geopolitical and economic forces driving healthcare innovation have favored vast patient populations. Pediatric AML, however, is a rare cancer. Its small patient population makes traditional, randomized clinical trials notoriously difficult, expensive, and agonizingly slow. REALworld: AML seeks to bypass these structural bottlenecks by building a robust real-world data set from current, former, and deceased pediatric patients. It is a strategic pivot from the conventional clinical trial model, transforming fragmented patient histories into an actionable, unified registry.

A Smarter Trial Design for Rare Cancers

The most significant bottleneck in rare disease research is the traditional randomized controlled trial. Placing critically ill children into standard-of-care control groups when investigational therapies are available presents profound ethical and logistical challenges. The FDA has increasingly recognized this friction, advancing guidance on the use of real-world evidence (RWE) and external control arms (ECAs) in drug development.

An external control arm allows researchers to compare patients receiving an investigational treatment in a trial with carefully validated, historical information from similar patients who previously received standard treatment. By leveraging the data collected through REALworld: AML, trial sponsors could potentially evaluate promising treatments more efficiently without requiring a placebo or standard-of-care arm.

"For children with rare diseases like AML, time matters," said Gwen Nichols, M.D., chief medical officer of Blood Cancer United. "Because patient populations are small, clinical trials can be difficult to conduct, and research can move more slowly than families can afford to wait. REALworld: AML gives children and families an opportunity to contribute directly to research while helping us build the evidence researchers need to design more efficient studies and advance better treatment options."

Regulatory analysts note that while the FDA's Oncology Center of Excellence has encouraged innovative trial designs for small populations, success hinges on the rigor of the data. External populations must be meticulously matched to treatment groups to avoid bias, requiring deep, standardized data on lines of therapy, concomitant medications, and biomarkers. This is precisely the operational gap the new initiative aims to fill.

The Intersection of Big Tech and Non-Profit Synergy

To build a registry capable of withstanding strict regulatory scrutiny, the initiative relies heavily on private-sector technological infrastructure. Tempus is deploying its personal health concierge app, Olivia, to centralize the intake and organization of patient records.

This collaboration highlights a growing synergy between non-profit advocacy and commercial artificial intelligence platforms. Tempus utilizes advanced de-identification processes that meet and exceed HIPAA standards, systematically removing 18 specific categories of personal identifiers before the data is analyzed for research. For participating families, the app serves a dual purpose: it provides a secure hub to manage complex medical records while simultaneously funneling anonymized data into the broader research ecosystem.

"The promise of Olivia is to ensure every patient receives the full benefit of what medicine offers, while also accelerating scientific discovery to improve those benefits," said Chris Scotto DiVetta, SVP of Olivia at Tempus. "Olivia puts meaningful health and educational resources directly into the hands of families, while creating new opportunities for them to play an active role in advancing pediatric AML research."

The ethical implications of handling such sensitive information are vast. Pediatric medical ethics dictate stringent safeguards, particularly when dealing with vulnerable populations. To address this, independent institutional review boards meticulously evaluate the consent forms and data handling procedures. Tempus utilizes an expert determination method involving independent third-party experts to evaluate data de-identification, ensuring that the risk of re-identification remains statistically negligible. This level of security is paramount to maintaining public trust and encouraging broader participation from affected communities.

Data Philanthropy and the Human Element

Behind the algorithmic de-identification and regulatory frameworks lies the profound emotional engine driving this initiative: data philanthropy. For families navigating the devastation of childhood cancer, donating medical records offers a tangible way to influence the future of medicine.

For the Breyfogle family, the promise of this registry is deeply personal. Twin daughters Kenedi and Kendal were diagnosed with AML shortly after birth. Following six months of intensive chemotherapy, both entered remission. Tragically, Kendal relapsed and passed away in September 2017, while Kenedi remains in remission today.

Abby Breyfogle is now sharing Kenedi's health records and plans to share Kendal's, ensuring both experiences contribute to the collective intelligence of the AML research community. The project's inclusion of records from deceased patients is a critical, albeit sensitive, component. It requires complex posthumous consent protocols but ensures that the most aggressive and fatal forms of the disease are represented in the data.

"When you've lived through pediatric AML, you understand how urgently families need better options," said Abby Breyfogle. "Knowing that researchers can learn from both Kenedi's and Kendal's experiences means so much to our family. Our hope is that what we've been through can help the next family who hears the words 'your child has cancer.'"

Dean Crowe, Founder and CEO of Rally Foundation for Childhood Cancer Research, echoed this sentiment. "Families confronting pediatric AML urgently need research approaches designed around the realities of rare childhood cancers. We are proud to have helped co-found REALworld: AML and support an initiative that puts families at the center of research."

Integrating with a Global Ecosystem

REALworld: AML does not exist in a vacuum. It is a strategic component of Dare to Dream, Blood Cancer United's broader initiative to transform pediatric care. The cornerstone of this larger project is PedAL, the first pediatric acute leukemia master clinical trial.

Operating across more than 100 medical centers globally, PedAL has already screened over 660 children, directing them toward targeted subtrials, including those evaluating venetoclax and ziftomenib. The real-world evidence gathered through the new registry will theoretically complement the clinical data generated by PedAL, creating a comprehensive feedback loop of patient outcomes and biological markers.

Furthermore, the collaborative nature of the PedAL trial, which leverages the massive infrastructure of the Children's Oncology Group and major European cooperative groups, highlights the necessity of borderless medical research. With sites spanning from Australia to Puerto Rico, the sheer geographic diversity of the trial participants provides a rich, varied dataset. When combined with the longitudinal real-world evidence from the new registry, researchers will possess an unprecedented, multidimensional view of how different genetic profiles respond to novel therapeutics.

However, the ultimate success of this initiative will depend on its ability to integrate with the broader landscape of pediatric oncology data. Major federal and institutional efforts, such as the National Cancer Institute's Childhood Cancer Data Initiative and existing clinical registries, are already working to build interconnected data ecosystems. To maximize its impact and avoid the historical pitfalls of data fragmentation, the insights mined from REALworld: AML must eventually flow seamlessly into these larger global repositories.

By treating patient data not just as a medical record, but as a vital, shared resource, this alliance is laying the groundwork for a more agile and responsive medical infrastructure. It is a model that, if successful, will not only rewrite the rules of pediatric AML research but could serve as a blueprint for conquering rare diseases across the globe.