Type II Diabetes
Conditions
Keywords
conjoint analysis, stated-preference methods, patient centered outcome research (PCOR), community-based participatory research (CBPR), patient preference, diabetes
Brief summary
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.
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.
Interventions
Respondents receive questions asking them to choose the best and worst features of a hypothetical medication.
Respondents receive questions asking them to choose the medication they prefer between a pair of hypothetical medications.
Sponsors
Study design
Eligibility
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Patient Medication Preference as Estimated by a Choice Model | One-time survey | Preference estimate for each medicine attribute level from a conditional logit regression analysis. For each arm, there were six attributes of the medication, with 3 levels each. Within each attribute the parameter estimates for each of the levels sum to 0. If a parameter estimate for a level is above (below) 0 then the parameter is higher (lower) than average for that medication attribute. |
| Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | One-time survey | Relative attribute importance (RAI) for each attribute in each arm measures the overall importance of that attribute. It is estimated by subtracting the lowest parameter estimate from the highest parameter estimate within each attribute. The RAI was then re-scaled on a 0 to 10 scale with 0 demonstrating no importance and 10 reflecting the most important attribute in each arm. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Self-reported Difficulty in Understanding and Answering the Survey Questions | One-time survey | Questions that asked the respondents to evaluate if the Best-Worst Scaling (BWS) or Discrete Choice Experiment (DCE tasks were easy to understand and answer. |
Countries
United States
Participant flow
Recruitment details
The survey was conducted through GfK KnowledgePanel. Eligible members were invited by email to participate in the survey. All participants were required to be 18 years or older with self-reported physician-diagnosed type 2 diabetes and able to read English or Spanish. African Americans and Latinos were oversampled.
Pre-assignment details
638 respondents (34%) did not respond to the survey and 139 people among those who responded (11%) were excluded based on the screening questions.
Participants by arm
| Arm | Count |
|---|---|
| Best-Worst Scaling (BWS) (Case 2) Preference measured by best-worst scaling (case 2)
Best-Worst Scaling (Case 2): Respondents receive questions asking them to choose the best and worst features of a hypothetical medication. | 551 |
| Discrete Choice Experiment (DCE) Preference measured by discrete choice experiment
Discrete Choice Experiment: Respondents receive questions asking them to choose the medication they prefer between a pair of hypothetical medications. | 552 |
| Total | 1,103 |
Baseline characteristics
| Characteristic | Best-Worst Scaling (BWS) (Case 2) | Total | Discrete Choice Experiment (DCE) |
|---|---|---|---|
| Age, Continuous | 62.55 years | 61.93 years | 61.30 years |
| Complications No | 314 Participants | 640 Participants | 326 Participants |
| Complications Yes | 237 Participants | 463 Participants | 226 Participants |
| Education Bachelor's degree or higher | 156 Participants | 321 Participants | 165 Participants |
| Education High school | 174 Participants | 362 Participants | 188 Participants |
| Education Less than high school | 39 Participants | 82 Participants | 43 Participants |
| Education Some college | 182 Participants | 338 Participants | 156 Participants |
| Income $25,000 - $49,999 | 160 Participants | 308 Participants | 148 Participants |
| Income $50,000 - $74,999 | 111 Participants | 222 Participants | 111 Participants |
| Income $75,000 and above | 139 Participants | 300 Participants | 161 Participants |
| Income Less than $25,000 | 141 Participants | 273 Participants | 132 Participants |
| Most recent HbA1c level Don't know | 92 Participants | 183 Participants | 91 Participants |
| Most recent HbA1c level Over 7.0% but under 8.0% | 144 Participants | 297 Participants | 153 Participants |
| Most recent HbA1c level Over 8.0% but under 9.0% | 46 Participants | 105 Participants | 59 Participants |
| Most recent HbA1c level Over 9.0% | 37 Participants | 58 Participants | 21 Participants |
| Most recent HbA1c level Under 7.0% | 232 Participants | 460 Participants | 228 Participants |
| No. of HbA1c level measured in past 6 months 1 time | 253 Participants | 492 Participants | 239 Participants |
| No. of HbA1c level measured in past 6 months Don't know | 21 Participants | 32 Participants | 11 Participants |
| No. of HbA1c level measured in past 6 months More than 2 times | 246 Participants | 507 Participants | 261 Participants |
| No. of HbA1c level measured in past 6 months None | 31 Participants | 72 Participants | 41 Participants |
| No. of hypoglycemic events in the past 6 months 1 time | 80 Participants | 160 Participants | 80 Participants |
| No. of hypoglycemic events in the past 6 months 2-5 times | 144 Participants | 278 Participants | 134 Participants |
| No. of hypoglycemic events in the past 6 months More than 5 times | 49 Participants | 94 Participants | 45 Participants |
| No. of hypoglycemic events in the past 6 months None | 278 Participants | 571 Participants | 293 Participants |
| Other chronic conditions No | 90 Participants | 182 Participants | 92 Participants |
| Other chronic conditions Yes | 461 Participants | 921 Participants | 460 Participants |
| Race/Ethnicity, Customized Black, Non-Hispanic | 128 Participants | 254 Participants | 126 Participants |
| Race/Ethnicity, Customized Hispanic | 117 Participants | 236 Participants | 119 Participants |
| Race/Ethnicity, Customized Other, Non-Hispanic | 20 Participants | 38 Participants | 18 Participants |
| Race/Ethnicity, Customized White, Non-Hispanic | 286 Participants | 575 Participants | 289 Participants |
| Region of Enrollment United States | 551 Participants | 1103 Participants | 552 Participants |
| Self-reported health Excellent | 27 Participants | 60 Participants | 33 Participants |
| Self-reported health Fair | 103 Participants | 209 Participants | 106 Participants |
| Self-reported health Good | 233 Participants | 465 Participants | 232 Participants |
| Self-reported health Poor | 30 Participants | 53 Participants | 23 Participants |
| Self-reported health Very good | 158 Participants | 316 Participants | 158 Participants |
| Sex: Female, Male Female | 274 Participants | 553 Participants | 279 Participants |
| Sex: Female, Male Male | 277 Participants | 550 Participants | 273 Participants |
| Type of diabetes medicine used No prescription medicine | 62 Participants | 99 Participants | 37 Participants |
| Type of diabetes medicine used Only injections/shots | 48 Participants | 90 Participants | 42 Participants |
| Type of diabetes medicine used Only pills | 321 Participants | 666 Participants | 345 Participants |
| Type of diabetes medicine used Pills and injections/shots | 119 Participants | 246 Participants | 127 Participants |
| Type of diabetes medicine used Refused | 1 Participants | 2 Participants | 1 Participants |
| Years of diagnosis | 11.47 years STANDARD_DEVIATION 8.26 | 11.35 years STANDARD_DEVIATION 8.03 | 11.24 years STANDARD_DEVIATION 7.81 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 0 | 0 / 0 |
| other Total, other adverse events | 0 / 0 | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 | 0 / 0 |
Outcome results
Patient Medication Preference as Estimated by a Choice Model
Preference estimate for each medicine attribute level from a conditional logit regression analysis. For each arm, there were six attributes of the medication, with 3 levels each. Within each attribute the parameter estimates for each of the levels sum to 0. If a parameter estimate for a level is above (below) 0 then the parameter is higher (lower) than average for that medication attribute.
Time frame: One-time survey
Population: Respondents who completed the survey.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | HbA1c decrease by 1% | 0.983 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | HbA1c decrease by 0.5% | 0.284 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | HbA1c decrease by 0% | -1.267 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | HbA1c stable 6 days/week | 0.859 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | HbA1c stable 4 days/week | -0.030 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | HbA1c stable 2 days/week | -0.828 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | No hypoglycemia | 0.640 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | Hypoglycemia during the day | -0.145 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | Hypoglycemia during day and/or night | -0.495 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | No nausea | 1.158 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | Nausea for 30 min/day | -0.262 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | Nausea for 90 min/day | -0.896 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | 1 pill/day | 0.561 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | 2 pills/day | 0.128 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | 1 pill and 1 injection/day | -0.689 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | $10 out-of-pocket cost | 0.900 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | $30 out-of-pocket cost | -0.051 utility of |
| Best-Worst Scaling (Case 2) | Patient Medication Preference as Estimated by a Choice Model | $50 out-of-pocket cost | -0.850 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | 2 pills/day | 0.348 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | HbA1c decrease by 1% | 0.362 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | No nausea | 0.739 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | HbA1c decrease by 0.5% | 0.261 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | $50 out-of-pocket cost | -0.628 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | HbA1c decrease by 0% | -0.623 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | Nausea for 30 min/day | 0.236 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | HbA1c stable 6 days/week | 0.395 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | 1 pill and 1 injection/day | -0.805 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | HbA1c stable 4 days/week | 0.073 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | Nausea for 90 min/day | -0.975 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | HbA1c stable 2 days/week | -0.469 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | $30 out-of-pocket cost | 0.077 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | No hypoglycemia | 0.325 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | 1 pill/day | 0.456 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | Hypoglycemia during the day | 0.104 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | $10 out-of-pocket cost | 0.550 utility of |
| Discrete Choice Experiment | Patient Medication Preference as Estimated by a Choice Model | Hypoglycemia during day and/or night | -0.429 utility of |
Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model
Relative attribute importance (RAI) for each attribute in each arm measures the overall importance of that attribute. It is estimated by subtracting the lowest parameter estimate from the highest parameter estimate within each attribute. The RAI was then re-scaled on a 0 to 10 scale with 0 demonstrating no importance and 10 reflecting the most important attribute in each arm.
Time frame: One-time survey
Population: Respondents who completed the survey.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Best-Worst Scaling (Case 2) | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | HbA1c decrease | 10 units on a scale |
| Best-Worst Scaling (Case 2) | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Duration of stable HbA1c | 7.496 units on a scale |
| Best-Worst Scaling (Case 2) | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Frequency of hypoglycemia | 5.043 units on a scale |
| Best-Worst Scaling (Case 2) | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Duration of nausea | 9.125 units on a scale |
| Best-Worst Scaling (Case 2) | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Treatment burden | 5.553 units on a scale |
| Best-Worst Scaling (Case 2) | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Out-of-pocket cost | 7.777 units on a scale |
| Discrete Choice Experiment | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Treatment burden | 7.358 units on a scale |
| Discrete Choice Experiment | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | HbA1c decrease | 5.745 units on a scale |
| Discrete Choice Experiment | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Duration of nausea | 10 units on a scale |
| Discrete Choice Experiment | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Duration of stable HbA1c | 5.040 units on a scale |
| Discrete Choice Experiment | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Out-of-pocket cost | 6.873 units on a scale |
| Discrete Choice Experiment | Weight Respondents Assign to Medication Attribute (Relative Attribute Importance) Assessed by a Choice Model | Frequency of hypoglycemia | 4.395 units on a scale |
Self-reported Difficulty in Understanding and Answering the Survey Questions
Questions that asked the respondents to evaluate if the Best-Worst Scaling (BWS) or Discrete Choice Experiment (DCE tasks were easy to understand and answer.
Time frame: One-time survey
Population: Respondents who completed the survey.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Strongly disagree | 20 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Disagree | 49 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Neither agree nor disagree | 123 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Agree | 248 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Strongly agree | 105 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Refused | 6 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Strongly disagree | 18 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Disagree | 73 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Neither agree nor disagree | 133 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Agree | 225 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Strongly agree | 94 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Refused | 8 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Strongly disagree | 8 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Disagree | 9 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Neither agree nor disagree | 82 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Agree | 314 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Strongly agree | 132 Participants |
| Best-Worst Scaling (Case 2) | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Refused | 6 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Disagree | 13 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Strongly disagree | 13 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Agree | 262 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Disagree | 36 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Refused | 0 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Neither agree nor disagree | 111 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Strongly agree | 89 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Agree | 292 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Neither agree nor disagree | 83 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Strongly agree | 99 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Refused | 2 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to understand the questions | Refused | 1 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Strongly agree | 126 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Strongly disagree | 15 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Strongly disagree | 12 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Disagree | 61 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | My answers are consistent with my preferences | Agree | 318 Participants |
| Discrete Choice Experiment | Self-reported Difficulty in Understanding and Answering the Survey Questions | I found it easy to answer all the questions | Neither agree nor disagree | 123 Participants |