Workshop: Using Patient-Reported Outcomes in Diabetes Research and Practice

Event Details

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.”

Background

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.

Meeting Objectives

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

Organizing Committee

Chairs

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)

Registration Deadline

October 20, 2017

Agenda

November 6, 2017

Session I: A Framework for Understanding PROs in Diabetes Research and Practice

8:30 a.m. – 8:45 a.m.

Welcome and Introductory Remarks
Judith Fradkin, M.D., National Institute of Diabetes and Digestive and Kidney Diseases
Alicia McAuliffe-Fogarty, Ph.D., CPsychol, T1D Exchange and American Diabetes Association

8:45 a.m. – 9:30 a.m.

Understanding PRO/HRQoL Measurement: Overview of the Science
Darren A. DeWalt, M.D., M.P.H., University of North Carolina at Chapel Hill
(35 minute talk/10 minute Q&A)

9:30 a.m. – 9:55 a.m.

Understanding PRO/HRQoL Measurement in Diabetes: State of the Field and Research Gaps
David G. Marrero, Ph.D., University of Arizona Health Sciences
(20 minute talk/5 minute Q&A)

9:55 a.m. – 10:15 a.m.

Break

10:15 a.m. – 12:15 p.m.

Understanding Population and Disease Specific Needs
(15 minute talks followed by panel for Q&A)

1. Understanding Population and Disease Specific Needs for Individuals with Type 2 Diabetes
   Ronald T Ackermann, M.D., M.P.H., Northwestern University Feinberg School of Medicine
2. Understanding Population and Disease Specific Needs for Individuals with Type 1 Diabetes
   David Maahs, M.D., Ph.D., Stanford University School of Medicine
3. Why must Parent/Caregiver Quality of Life be considered in PROs for Youth with Diabetes?
   Barbara J. Anderson, Ph.D., Baylor College of Medicine and Texas Children’s Hospital
4. What PROs Really Matter for Older Adults with Diabetes and their Caregivers?
   Elbert Huang, M.D., M.P.H, University of Chicago Medicine
5. Understanding PRO Domains Relevant for Populations at Elevated Risk
   Monica Peek, M.D., M.P.H, University of Chicago Medicine
6. Understanding Population and Disease Specific Needs: Panel with Audience Discussion
   Moderator: Marisa E. Hilliard, Ph.D., Baylor College of Medicine

12:15 p.m. – 1:15 p.m.

Lunch

Session II: Understanding the Needs of PRO End Users

1:15 p.m. – 1:45 p.m.

PROs that Matter to Patients and Caregivers: Panel
Jeff Hitchcock (Parent of a child with T1D)
Kelly Rawlings (Living with T1D)
Ina Mendoza (Living with T2D)

Moderator: Alicia McAuliffe-Fogarty, Ph.D., CPsychol, T1D Exchange and American Diabetes Association

1:45 p.m. – 2:05 p.m.

PROs that Matter to Healthcare Providers
Sarah D. Corathers, M.D., Cincinnati Children's Hospital
(15 minute talk/5 minute Q&A)

2:05 p.m. – 2:25 p.m.

PROs that Matter to Healthcare Systems
Sanjeev Mehta, M.D., M.P.H., Joslin Diabetes Center
(15 minute talk/5 minute Q&A)

2:25 p.m. – 2:50 p.m.

FDA Perspective: PROs in Device and Drug Approval
Elektra Papadopoulos, M.D., M.P.H., Food and Drug Administration
(20 minute talk/5 minute Q&A)

2:50 p.m. – 3:10 p.m.

Break

3:10 p.m. – 3:35 p.m.

Harmonizing PROs in Diabetes Research: Challenges and Opportunities
Søren Eik Skovlund, MSc., Bsc, Dgr., Aalborg University, Denmark
(15 minute talk/5 minute Q&A)

3:35 p.m. – 4:00 p.m.

Panel with Audience Discussion
Moderator: Christine M. Hunter Ph.D., Office of Behavioral and Social Sciences Research

Session III: Current Advances in PRO/HRQoL Measurement in Diabetes

4:00 p.m. – 4:20 p.m.

HealthMeasures: State-of-the-Science Assessment and Application of Patient Centered Outcomes
Ashley Wilder Smith, Ph.D., M.P.H., National Cancer Institute
(15 minute talk/5 minute Q&A)

4:20 p.m. – 4:40 p.m.

HRQOL / PRO in T2D Across the Lifespan
Jeffrey S. Gonzalez, Ph.D., Albert Einstein College of Medicine
(15 minute talk/5 minute Q&A)

4:40 p.m. – 5:00 p.m.

Designing Developmentally-Tailored PRO Measures of T1D HRQOL from Childhood through Adulthood
Marisa E. Hilliard, Ph.D., Baylor College of Medicine
(15 minute talk/5 minute Q&A)

5:00 p.m. – 5:20 p.m.

Use of PROs in Closed Loop Research
Korey K. Hood, Ph.D., Stanford University School of Medicine
(15 minute talk/5 minute Q&A)

5:20 p.m. – 5:30 p.m.

Wrap up and Instructions for Day 2
Christine M. Hunter Ph.D., Office of Behavioral and Social Sciences Research
Alicia McAuliffe-Fogarty, Ph.D., CPsychol, T1D Exchange and American Diabetes Association

November 7, 2017

Session IV: Next Steps: Research Gaps and Priorities

8:30 a.m. – 9:30 a.m.

Summary Remarks and Full Audience Discussion

1. Co-Chairs Summary of Key Points from Day 1 and Framing of Day 2 (10 minutes)
   David G. Marrero, Ph.D., University of Arizona Health Sciences
2. Discussant Remarks (10 minutes)
   Lawrence Fisher, Ph.D., University of California, San Francisco
3. Key Questions/Discussion with Audience (40 minutes)
   Facilitator: Lawrence Fisher, Ph.D., University of California, San Francisco

9:30 a.m. – 9:45 a.m.

Break

9:45 a.m. – 11:45 a.m.

Breakout Groups

1. What are the key things that are essential to be measured/domains
   Facilitators: David Marrero and Alicia McAuliffe-Fogarty
2. Criteria for selecting a measure/what does a measure need to do to be worthwhile for end users
   Facilitators: Marisa Hilliard and Christine Hunter

11:45 a.m. – 12:30 p.m.

Group report out, discussion and wrap-up