CHOP-led clinical research network leverages big data with a family-friendly approach

PEDSnet, a national clinical research network co-led by a pediatric researcher at The Children's Hospital of Philadelphia, was approved for an $8.6 million, three-year funding award by the Patient-Centered Outcomes Research Institute (PCORI). PCORI announced the award on July 21.

PEDSnet combines "big data," the use of large quantities of electronic health records, with family engagement—involving parents as partners in research design to address concerns that are important to families, especially those living with a chronically ill child.

"Our fundamental goal is to discover which treatments and diagnostic practices are most effective for children," said Christopher Forrest, M.D., Ph.D., a CHOP pediatrician and principal investigator of PEDSnet. "We want to help parents decide which treatment option is best for their child, and when parents are engaged in research, we can prioritize outcomes by what is important to both clinicians and parents."

PEDSnet brings together eight collaborating children's hospitals, including Cincinnati Children's Hospital Medical Center, where Forrest's co-principal investigator, Peter Margolis, M.D., Ph.D., works. The network aims to pool data and expertise from multiple centers to more quickly and efficiently translate discoveries into better outcomes for children. One hospital may not treat enough children with a rare condition to yield statistically significant findings, but a network of multiple sites sharing data strengthens those results.

The current funding award is a second phase in the development of PCORnet, PCORI's overall national patient-centered clinical research resource consisting of 34 individual research networks. In the first phase, in 2014, PCORI provided PEDSnet $7 million to support the development of its infrastructure. The second phase of funding will be used to launch dozens of new research projects and to develop a long-term, financially sustainable network.

For Amy Kratchman, a family consultant at CHOP who serves on the PEDSnet Steering Board, it was important for parents like her to be involved in the clinical research from the start. "The first phase was very data-heavy and the technical information could be overwhelming, but the researchers made it a point to keep us fully informed about the material and to provide explanations beforehand," she said.

More to the point, she added, the scientists were able to step outside the "research bubble" and learn how parents' concerns may challenge the researchers' initial assumptions about what was important. Referring to a pilot project that preceded PEDSnet, Kratchman recalled that a focus group of parents of children with autism spectrum disorder rated the importance of a meaningful relationship higher than a medication that controlled their child's aggressive behavior.

"Families may have a different set of priorities than the researchers," she added, "and identifying which outcomes are important to parents and children helps to shape how the research is conducted."

Provided by Children's Hospital of Philadelphia