Workshop: Using Patient-Reported Outcomes in Diabetes Research and Practice
The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and the American Diabetes Association (ADA) are pleased to co-sponsor this workshop on “Using Patient Reported Outcomes in Diabetes Research and Practice.”
Diabetes and its treatments affect quality of life for people living with the disease and their families. Clinicians, researchers, and regulators generally agree that the assessment of Patient Reported Outcomes (PROs) (defined as "any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else." National Quality Forum, Jan 10, 2013) in people with diabetes is important and can significantly contribute to treatment decisions, regulatory approval, and guideline development. PROs complement biomedical outcomes or a clinical diagnosis by helping to understand the patient experience of living with a disease, often termed Health Related Quality of Life (HRQoL); including understanding physical, emotional, and social functioning and the demands and tradeoffs related with the treatment regimen. Important PRO domains might include assessments of perceived wellness, symptoms, functioning, emotional status, social and family functioning, satisfaction with care and disease specific issues such as burnout, fear of hypoglycemia and trade-offs between the benefit and demands of various treatment regimens.
Despite the increased call for routine use of PROs in care and research, there remains a lack of agreement about what domains should be measured and what measures to include. While some disease areas, such as cancer, are moving toward more consistent use of PROs, diabetes care and research does not currently have standardized approaches to PRO assessments, and there is little agreement about the essential PRO domains to assess. Some key issues to consider in PRO selection include psychometric issues, such as reliability (consistently measures the domain of interest); various forms of validity, such as concurrent and predictive validity (relates to or predicts another important outcome such as glycemic control or diabetes complications); and construct validity (measures what it is intended to measure). Other considerations include the method and patient burden (time to complete or other costs) of measurement, the degree of interpretability and actionable information from the measure, and whether it is valid to use for various populations including various age groups, disease states, languages, and cultures. In diabetes, as in many diseases, another important requirement is that it captures disease-specific considerations related to symptoms, self-management, regimen, or device use.
To accelerate the standardized use of validated, generalizable, and standardized PRO measures in diabetes research and practice there is a need to harmonize efforts and identify the highest priority research gaps. To facilitate this area of research, the workshop will address these key topics and questions:
- Understanding PRO/HRQoL Measurement
- What are they and why are they important to the key end users (patient/caregiver, healthcare provider/system, researcher, regulator)?
- Understanding the science of developing and validating PROs
- When are disease specific versus generic measures needed; lessons from diabetes and other chronic diseases?
- Examine advances in diabetes and in other chronic diseases
- Key Domains for measurement of PROs
- Identify key domains of measurement relating to diabetes management and treatment demands as well as broader social, physical, and emotional status
- Identify Research Needs and Gaps
- Priorities in research to address key end-user needs for assessing PROs (patient/caregiver, healthcare provider/system, researcher, device and drug regulator)
- Understanding population and disease specific research gaps: T1D vs T2D, stage of disease (duration, complications, regimen complexity), development/lifespan, specific therapeutic devices or therapies, and understudied populations/groups at elevated risk (e.g., r/e minority, low income/low resource environment)
- Identify measurement (domains or specific measures) that are ready for standard use across research (clinical, behavioral) or need to be developed to facilitate meaningful cross trial comparison
David G. Marrero, Ph.D.
Director of the UA Center for Border Health
University of Arizona Health Sciences
David Maahs, M.D., Ph.D.
Professor, Department of Pediatrics
Chief, Division of Endocrinology and Diabetes
Stanford University School of Medicine
Marisa E. Hilliard, Ph.D.
Assistant Professor, Pediatrics-Psychology
Baylor College of Medicine
American Diabetes Association
Erika Gebel Berg, Ph.D.
Alicia McAuliffe-Fogarty, Ph.D., C.Psychol.
National Institutes of Health
Christine Hunter, Ph.D. (OBSSR)
Guillermo Arreaza-Rubín, M.D. (NIDDK)
Barbara Linder, M.D., Ph.D. (NIDDK)
Andrew Bremer, M.D., Ph.D. (NIDDK)
Luke Stoeckel, Ph.D. (NIDDK)
October 20, 2017