Dr. Betsy Shenkman Appointed to Co-chair National PCORI Pediatrics Research Group

Building upon years of research in children’s health, pediatric cancer outcomes and health disparities among vulnerable populations, Betsy A. Shenkman, Ph.D., was appointed to serve as national co-chair of the Patient-Centered Outcomes Research Institute’s PCORnet Pediatrics Collaborative Research Group (CRG).

The newly formed group will help the Patient-Centered Outcomes Research Institute (PCORI) set national priorities for pediatric research using the institute’s nationwide research network, PCORnet. This vast network seeks to link large numbers of patients, health care systems, clinicians, and researchers across the country to accelerate the translation of research findings into patient-centered care. The Pediatrics CRG is tasked with leveraging the power of PCORnet and big data to identify and address some of the nation’s most pressing pediatric health problems.

According to Shenkman, children in the United States have higher rates of morbidity, behavioral health conditions, and obesity, and greater exposure to a variety of health risks when compared with children from other high-income countries. Although social and behavioral factors contribute to some of these differences, timely access to safe and effective health care is an important contributor to children’s health. However, disparities in both access to health care and health outcomes persist among ethnic and racial minorities despite state and national efforts to reduce or eliminate them.

“Improving children’s health by reducing disparities and improving the effectiveness of care through parent, child, and clinician engagement can yield lifetime benefits,” Shenkman said.

Another concern is the lack of evidence to determine which interventions are most effective for in addressing health problems that some children face, such as managing sleep disturbances in children with chronic health conditions. Addressing issues like poor sleep is critical for good health, including the prevention of obesity, yet little is known about the most effective strategies to improve sleep in children with chronic conditions.

“Strong comparative effectiveness research, that is, understanding which interventions or treatments are most effective for children, is essential to improve pediatric medical decision-making,” she said.

Children with chronic conditions are particularly vulnerable to these evidence gaps because of the impact of their disease and its treatments on their health and development.

Shenkman said she is honored to have been selected as national co-chair of PCORI’s Pediatric CRG and is eager to get to work. She and her co-chair, Christopher Forrest, M.D., Ph.D., professor of pediatrics and health care management at the University of Pennsylvania and Children’s Hospital of Philadelphia, will work closely with patients, family members, clinicians, and health system leaders to identify and prioritize the nation’s top pediatric health issues. Forrest also is principal investigator of the PCORnet pediatric clinical data research network, or PEDSnet.

The research group also will look to PCORnet and big data to help conduct research in areas that have been identified via critical clinical observations. For example, a cluster of problems among newborns that had been exposed to opiate drugs in the womb, known as neonatal abstinence syndrome, recently has been identified as a clinical priority.

Using the OneFlorida Data Trust, where Shenkman serves as the lead co-principal investigator and co-director, along with data from the other PCORnet sites such as PEDsnet, it is possible to study how to better identify these babies to intervene early and improve their health outcomes.

Although a number of smaller clinical research networks and databases around the country have made inroads into facilitating this kind of research, “No single network has sufficient numbers of investigators, clinicians, or patients to provide the quantity and quality of evidence needed to substantively improve children’s health care,” Dr. Shenkman said. “Collectively across PCORnet, however, the breadth and depth of pediatric research interests and expertise is extensive and should be harnessed to improve children’s health nationwide.”