“How tired do you feel?” a doctor asks a child with a chronic disease. Or, “How well are you managing stress?”
The answers to questions like these are even more important, from many patients’ and families’ perspectives, than the particular numerical result of their lab test results.
But the answers are less useful to doctors than they could be. Doctors do not have validated tools to use such patient-reported outcomes to track progress managing a condition over time in the same way they can compare results of blood tests over time. In clinical research, they are unable to compare the answers across patients to ultimately show an experimental drug meaningfully improves fatigue or other patient-reported measures.
“Our vision is that patient-reported outcomes become like lab tests,” said Christopher Forrest, MD, PhD, a pediatrician and researcher at The Children’s Hospital of Philadelphia and professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania. “Soon doctors will use patient-reported outcomes to monitor patients’ clinical care in the same way they use lab tests or X-rays.”
Dr. Forrest and colleagues at CHOP and partner institutions received a new grant from the National Institutes of Health to advance the science of patient-reported outcome measures to one day achieve that vision.
The grant establishes CHOP as the administrative leader and one of four centers awarded as part of the Validation of Pediatric Patient-Reported Outcomes in Chronic Diseases (PEPR) Consortium. The PEPR Consortium will work to improve pediatric health and well-being by capturing the voice and experience of children and their families living with a variety of chronic diseases and conditions.
“There are a lot of outcomes that we can’t get except by child report,” said Katherine Bevans, PhD, who is co-leading the PEPR Consortium research at CHOP with Dr. Forrest. “There’s a long line of research, going back 20 years, that kids are in fact accurate and reliable reporters of their own health.”
Dr. Bevans noted that considering patient-reported outcomes is essential to ensure that clinicians and clinical researchers are achieving their goals of delivering patient- and family-centered care.
The group will test several tools for collecting children’s self-reported outcomes that were previously developed under the Pediatric PROMIS initiative. Under PROMIS, NIH-funded researchers at multiple sites, including a group led by Dr. Forrest at CHOP, developed survey tools for collecting and scoring a variety of patient-reported outcome measures in both pediatric and adult populations.
Development of new tools under PROMIS has ended, and the PEPR consortium will now continue to collect evidence to validate the tools’ effectiveness at measuring clinically meaningful outcomes specifically in populations of children with chronic diseases. They intend to connect children’s scores on PROMIS surveys to clinically meaningful outcomes so doctors and clinical researchers can know when a fluctuation in a survey score represents a meaningful improvement or worsening.
In addition, PEPR Consortium centers will include a focus on improving understanding of environmental influences on pediatric diseases, in keeping with the goals of the National Children’s Study (NCS). PEPR is one of the NIH programs receiving funds diverted from the now-cancelled NCS and tasked with answering some of the study’s important questions about children’s environment and health.
The group led by Dr. Bevans and Dr. Forrest at CHOP will integrate PROMIS survey tools into several existing studies, including a long-term study of chronic kidney disease led at CHOP by nephrology division chief Susan Furth, MD, PhD; a clinical trial for pediatric Crohn’s disease, led at CHOP by Andrew Grossman, MD; and, a clinical study of survivors of cancer treatment at St. Jude Children’s Research Hospital.
“The opportunity with the PEPR grant is to get a much more in-depth view from the patient’s perspective on how concerns that go along with chronic kidney disease including pain, mobility issues, and social functioning, affect their lives,” said Dr. Furth. “Ultimately, when we can target our interventions and include the perspectives of kids and their families, we will focus on the key areas that really make their lives better.”
Dr. Grossman noted, “The PEPR grant will afford the opportunity for us to develop and refine the tools that will allow the patient and parent voice to be inculcated into our assessment of disease activity. In turn, we will better be able to determine the effectiveness of our interventions going forward.”
As the administrative leader of the PEPR Consortium, CHOP also will support resources and technical expertise for projects undertaken by PEPR investigators at the other three centers receiving NIH PEPR awards. These centers will benefit from Dr. Forrest’s work as principal investigator of PEDSnet, a major clinical data research network that includes data from electronic health records and patient-reported data. The infrastructure developed under PEDSnet will offer a data management platform and standardized data formats and sharing for PEPR research data.
At CHOP, Dr. Forrest and Dr. Bevans are further working to build both PEPR and PEDSnet into a new center of emphasis focused on research that informs doctors’ routine treatment decisions. They aim to build a community of clinicians and researchers interested in effectiveness research and patient-reported outcomes. They encourage CHOP researchers and clinicians to contact them to discuss collaborations.