CHOP gets $10 million for tool to transform care for kids with rare diseases

Children’s Hospital of Philadelphia has received a $10 million dollar grant from a new federal agency to roll out a tool to gather and share data on childhood rare diseases, with the goal of transforming their care.

The tool — known as the Real-time Analysis and Discovery in Integrated And Networked Technologies, or RADIANT — is designed to gather data from the electronic health records of patients around the country in real time, so researchers and clinicians can learn from each others’ experiences with rare diseases such as pediatric brain tumors.

This kind of a tool is essential when it comes to rare diseases, which many doctors don’t see often, said Adam Resnick, research scientist in the Department of Biomedical and Health Informatics and co-executive director of the Center for Data Driven Discovery in Biomedicine at CHOP.

“If you have no clinical experience with that patient, how do you make informed decisions about their care, and what potential clinical trials are out there? So this aids with that,” said Resnick.

The effort is being funded by ARPA-H, a new federal agency modeled after DARPA, the research arm of the Pentagon, which aims to support projects that can transform health care. The grant — totaling $9,917,110 — is CHOP’s first ARPA-H award.

RADIANT will gather real-time patient data from dozens of hospitals, including CHOP, which will lead the effort.

RADIANT will be more than just a basic database of medical information. For instance, for some surgeons, the first encounter they have with a child with a brain tumor is when they see their MRI taken in the ER.

With RADIANT, surgeons will be able to use that image to search the records of patients with similar MRIs, and read about clinical trials, treatment options, and outcomes for other children with the same tumor.

RADIANT will also include genetic data, enabling researchers and clinicians to gather information from other patients with the same mutations or genetic profiles — all in real-time, without waiting for months for the information to get uploaded to a database.

“We’re trying to fundamentally change how we query in that we’re trying to query the latest, most up-to-date data as possible,” said Allison Heath, director of data technology and innovation at CHOP’s Center for Data Driven Discovery in Biomedicine.

An ultimate goal of the project is also to transform clinical trials of rare diseases in childhood.

When conducting a clinical trial of an experimental drug, researchers have to recruit a group of patients and give the drug to some but not all, so they can compare whether those who took the drug fared better than those who didn’t.

With rare diseases, it can take a long time to find enough patients to get meaningful data, and for families, the process can be heartbreaking, for they cannot control whether their loved one gets the treatment or a placebo.

Ideally, if RADIANT gathers medical information from enough patients with a particular rare disease, it may one day be possible to give all patients in a clinical trial the experimental treatment, and compare their experience to patients whose records are included in RADIANT. This means researchers wouldn’t have to gather as many patients to conduct the trial, and every trial participant would get the treatment, said Resnick.

Eliminating the placebo arm of a clinical trial would be “challenging,” he said, but worth the effort. “This is something we’re beginning to pilot as an initiative.”

According to Heath, the goal is to connect CHOP to RADIANT’s additional capabilities within the first six months of the project, then expand access to other participating hospitals.