Validation of Pediatric Patient Reported Outcomes in Chronic Diseases (PEPR) Consortium (U19)

Updated December 31, 2015

PROMIS Pediatric Banks and PROMIS Parent Proxy Banks

PEPR Overview

The National Institutes of Health (NIH) is pleased to announce the release of an FOA entitled ‘Validation of Pediatric Patient Reported Outcomes in Chronic Diseases (PEPR) Consortium (U19)’ https://grants.nih.gov/grants/guide/rfa-files/RFA-AR-15-014.html. This Consortium will capitalize on recent advances in the science of Patient Reported Outcomes (PROs) to assess the health of children with a variety of chronic diseases and conditions in clinical research and care settings. Examples of chronic diseases or conditions in children include (but are not limited to): asthma, juvenile arthritis, cystic fibrosis, diabetes, obesity and overweight, chronic kidney disease, inflammatory bowel disease, sickle cell disease, malnutrition, developmental disabilities including attention deficit/hyperactivity disorder and autism spectrum disorder, cerebral palsy and mental illnesses. Many of these diseases and conditions cause patients to have pain or experience fatigue; affect their sleep; alter their ability to physically or mentally function on a daily basis; or in other ways interfere with their social relationships and overall quality-of-life. The impact, importance, and value of pediatric self-reporting (and parent-proxy reporting) in the evaluation of treatment efficacy and effectiveness support the need for a universally applicable, common metric in order to allow clinically meaningful comparisons of treatments of disease, both within and across chronic pediatric diseases and conditions. The PEPR Consortium will feature the utilization of the Patient Reported Outcomes Measurement Information System (PROMIS®) pediatric self-report and parent proxy instruments www.nihpromis.org External Web Site Policy. These instruments include those already, or soon to be, available at the Assessment Center https://www.assessmentcenter.net/documents/InstrumentLibrary.pdf External Web Site Policy.

The main, long-term goal of the Consortium is to conduct robust clinical validation studies of child patient-reported outcomes (cPROs) in clinical trials and care settings, and to examine the impact of environmental stressors (including socioeconomic aspects) on children’s symptoms and quality of life in a variety of chronic diseases or conditions. Therefore, cPROs may advance personalized, precision medicine by coupling with detailed clinical phenotyping and/or biospecimen collections in established, well-characterized pediatric cohorts. Development of new domains that extend the measurement science of cPROs to address environmental stressors/exposures, and that fit into the PROMIS domain framework following PROMIS standards, is also possible. Successful applicants will be recognized leaders in clinical pediatric research and patient care with demonstrated expertise in leading large clinical collaborative projects, as well as recruiting and retaining cohort(s) of high quality.

PEPR Structure

The figure below highlights key features of the structure of the proposed Consortium. Details are included in the FOA. A listing of Frequently Asked Questions (FAQs) and an informational webinar for potential applicants will be forthcoming.

For NIAMS contacts listed in the FOA, please submit questions to PEPR@mail.nih.gov. In addition to the NIAMS contacts listed in the FOA, the following NIH Institutes are participating in this initiative and the individuals below may be contacted for questions or more information.

Lynne Haverkos, M.D., M.P.H.
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Telephone: 301-435-6881
Email: LH179R@nih.gov

Ellen Werner, Ph.D., M.A.
National Heart, Lung, and Blood Institute (NHLBI)
Telephone: 301-435-0050
Email: Ellen.Werner@nih.gov

Marva Moxey-Mims, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Telephone: 301-594-7717
Email: mm726k@nih.gov

Derrick Tabor, Ph.D.
National Institute on Minority Health and Health Disparities (NIMHD)
Telephone: 301-594-8950
Email: Dt103j@nih.gov

Ashley Wilder Smith, Ph.D., M.P.H.
National Cancer Institute (NCI)
Telephone: 240-276-6714
Email: smithas@mail.nih.gov

Ann E. Wagner, Ph.D.
National Institute of Mental Health (NIMH)
Telephone: 301-443-5944
Email: awagner@mail.nih.gov

Chart showing how the consortium institutions will work together and feed data into the data portal.