Measuring the Preferences of Patients With Type II Diabetes Using Best-worst Scaling and Discrete Choice Experiment

Advancing Stated-preference Methods for Measuring the Preferences of Patients With Type II Diabetes

In 2012, the FDA Center for Devices and Radiological Health (CDRH) issued guidance to clarify the principal benefit-risk factors FDA considers during the reviews for premarket approval applications and de novo classification requests. In addition to a detailed description of benefits and risks, CDRH listed "patient tolerance for risk and perspective on benefit" as a factor that CDRH may consider in regulatory reviews. It underlined the need for developing methods to measure patient preference and incorporate it into regulatory decision-making. The purpose of this study is to advance methods for patient and community engagement in patient-centered outcome research (PCOR) and has three objectives.

First, demonstrate good practices for patient and community involvement in PCOR projects by applying principles of community-based participatory research (CBPR).

Second, address methodological gaps pertaining to the use of stated-preference methods in studying preferences in PCOR. These include identifying the best methods for designing a preference study and strategies for analyzing variation in preferences. The investigators also seek to assess the relevance of stated-preference methods to patients and stakeholders using both qualitative and quantitative methods.

Third, demonstrate good practices for applying stated-preference methods by studying the preferences of patients with type II diabetes. While type II diabetes provides an important case study, this research will advance approaches and methods that will be broadly generalizable to other diseases, and to diverse patient and stakeholder groups.

Clinical Significance:

This project will illustrate and advance methods for assessing the values of patients and stakeholders. It will demonstrate how CBPR methods apply to PCOR studies and the value of stated-preference methods in measuring the preferences of patients and stakeholders and directing health care.

Study Overview

• Status: Unknown
• Conditions: Type II Diabetes
• Intervention / Treatment: Other: Best-Worst Scaling (Case 2); Other: Discrete Choice Experiment

Detailed Description

This study will apply the principles of CBPR to involve patients and stakeholders associated with a local community board and a national diabetes advisory board in key decisions in the project. During year 1, the investigators will utilize mixed methods to develop, pretest, and pilot the survey instruments to assess the preferences of patients with type II diabetes. In year 2, the investigators plan further engagement to finalize the survey instruments, and will implement a nationally representative, racially/ethnically diverse sample of patients with type II diabetes. Based on further consultation, the investigators will conduct statistical analysis, including stratified analyses and segmentation of patients with similar preferences. In year 3, mixed methods will be applied to assess beliefs of patients and other consumers about the relevance of this work and its generalizability to other PCOR topics. Finally, lay language reports will be developed to highlight patient and stakeholder engagement and the application of stated-preference methods to the study of the preferences of patients with type II diabetes.

• Study Type: Observational
• Enrollment (Anticipated): 1000

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins Bloomberg School of Public Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Adults with type II diabetes.

Description

Inclusion Criteria:

• Participate in the GfK's KnowledgePanel
• Self-reported Type II diabetes diagnosis

Exclusion Criteria:

• Does not have Type II diabetes diagnosis
• Unable to communicate in English or Spanish

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Best-Worst Scaling (Case 2); Preference elicitation survey using a best-worst scaling method.	Other: Best-Worst Scaling (Case 2); Respondents receive questions asking them to choose the best and worst features of a hypothetical medication.
Discrete Choice Experiment; Preference elicitation survey using a discrete choice experiment method.	Other: Discrete Choice Experiment; Respondents receive questions asking them to choose the medication they prefer between a pair of hypothetical medications.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The importance of lowering hemoglobin A1C level from patient perspective	The investigators will measure patients' view on the importance of lowering hemoglobin A1C level using surveys with two different stated-preference methods. One study arm will use the best-worst scaling (case 2) technique and the other arm will use discrete choice experiment method. Results from the two arms will be compared.	This outcome will be evaluated during a one-time survey that will be conducted one year after the start of the study.
The importance of having stable blood glucose levels from patient perspective	The investigators will measure patients' view on the importance of having stable blood glucose levels using surveys with two different stated-preference methods. One study arm will use the best-worst scaling (case 2) technique and the other arm will use discrete choice experiment method. Results from the two arms will be compared.	This outcome will be evaluated during a one-time survey that will be conducted one year after the start of the study.
The importance of avoiding hypoglycemia from patient perspective	The investigators will measure patients' view on the importance of avoiding hypoglycemia using surveys with two different stated-preference methods. One study arm will use the best-worst scaling (case 2) technique and the other arm will use discrete choice experiment method. Results from the two arms will be compared.	This outcome will be evaluated during a one-time survey that will be conducted one year after the start of the study.
The importance of avoiding nausea from patient perspective	The investigators will measure patients' view on the importance of avoiding nausea using surveys with two different stated-preference methods. One study arm will use the best-worst scaling (case 2) technique and the other arm will use discrete choice experiment method. Results from the two arms will be compared.	This outcome will be evaluated during a one-time survey that will be conducted one year after the start of the study.
The importance of minimizing treatment burden from patient perspective	The investigators will measure patients' view on the importance of minimizing treatment burden using surveys with two different stated-preference methods. One study arm will use the best-worst scaling (case 2) technique and the other arm will use discrete choice experiment method. Results from the two arms will be compared.	This outcome will be evaluated during a one-time survey that will be conducted one year after the start of the study.
The importance of medication cost from patient perspective	The investigators will measure patients' view on the importance of medication cost using surveys with two different stated-preference methods. One study arm will use the best-worst scaling (case 2) technique and the other arm will use discrete choice experiment method. Results from the two arms will be compared.	This outcome will be evaluated during a one-time survey that will be conducted one year after the start of the study.

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Self-reported difficulty in understanding and answering the survey questions	The survey will ask the respondents to evaluate if the questions are easy to understand and answer using Likert scale.	This outcome will be evaluated in a survey that will be conducted one year after the start of the study.

Collaborators and Investigators

• Sponsor: Johns Hopkins Bloomberg School of Public Health
• Collaborators: Patient-Centered Outcomes Research Institute
• Investigators: Principal Investigator: John Bridges, PhD, Johns Hopkins University

Study record dates

Study Major Dates

• Study Start: July 1, 2013
• Primary Completion (Anticipated): June 1, 2016
• Study Completion (Anticipated): June 1, 2016

Study Registration Dates

• First Submitted: November 18, 2015
• First Submitted That Met QC Criteria: December 18, 2015
• First Posted (Estimate): December 22, 2015

Study Record Updates

• Last Update Posted (Estimate): December 22, 2015
• Last Update Submitted That Met QC Criteria: December 18, 2015
• Last Verified: October 1, 2015

More Information

Terms related to this study

• Keywords: diabetes; patient preference; conjoint analysis; stated-preference methods; patient centered outcome research (PCOR); community-based participatory research (CBPR)
• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 2
• Other Study ID Numbers: PCORI 90056532 PF