PROMISE: A Network for Real-World Evidence in Rare Pediatric Cancers
Official title: PROMISE — Pediatric Rare-tumor Observational MultI-Source Evidence Network
Funded at Children's Hosp of Philadelphia · NIH-11571678 · Data updated 2026-11-12
A network will collect and harmonize pediatric cancer patient records from multiple centers to create high-quality real-world data that can support quicker, regulatory-ready insights when randomized trials aren’t feasible.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $1,498,821 |
| NIH project number | 1U01FD008977-01 |
| Project ID | NIH-11571678 on NIH RePORTER |
Who could benefit from this research
✓Could help
Pediatric, adolescent, and young adult patients with rare or ultra-rare cancers whose care is captured in participating centers’ electronic health records.
✕Less likely to help
Patients whose data are not included in participating sites or whose conditions are not among rare pediatric tumors may see limited direct benefit.
What this research studies
PROMISE aims to build a scalable, multi-center data system that gathers and standardizes pediatric cancer information from electronic health records. The project will align real-world data with rare tumor registries and regulatory expectations to support meaningful analyses. Researchers will study disease trajectories and real-world treatment effects, and may develop external control cohorts when randomized trials aren’t possible. This work focuses on improving evidence generation to guide care for children with rare cancers.
Why it matters
Potential benefit: If successful, this network could accelerate understanding and regulation of treatments for rare pediatric cancers, potentially improving outcomes for affected children and young adults.
How similar studies have gone: Prior real-world data networks have shown promise in supporting regulatory decisions, though this exact multi-center rare pediatric focus is innovative.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Aplenc, Richard — Children's Hosp of Philadelphia
- Study coordinator: Aplenc, Richard
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.