Ellen M. Janssen

Review of the evidence: prevalence of medical conditions in the United States population with serious mental illness

OBJECTIVE Persons with serious mental illness (SMI) have high rates of premature mortality from preventable medical conditions, but this group is underrepresented in epidemiologic surveys and we lack national estimates of the prevalence of conditions such as obesity and diabetes in this group. We performed a comprehensive review to synthesize estimates of the prevalence of 15 medical conditions among the population with SMI. METHOD We reviewed studies published in the peer-reviewed literature from January 2000 to August 2012. Studies were included if they assessed prevalence in a sample of 100 or more United States (US) adults with schizophrenia or bipolar disorder. RESULTS A total of 57 studies were included in the review. For most medical conditions, the prevalence estimates varied considerably. For example, estimates of obesity prevalence ranged from 26% to 55%. This variation appeared to be due to differences in measurement (e.g., self-report versus clinical measures) and underlying differences in study populations. Few studies assessed prevalence in representative, community samples of persons with SMI. CONCLUSIONS In many studies, the prevalence of medical conditions among the population with SMI was higher than among the overall US population. Screening for and monitoring of these conditions should be common practice in clinical settings serving persons with SMI.

Conducting a Discrete-Choice Experiment Study Following Recommendations for Good Research Practices: An Application for Eliciting Patient Preferences for Diabetes Treatments

OBJECTIVES To consolidate and illustrate good research practices in health care to the application and reporting of a study measuring patient preferences for type 2 diabetes mellitus medications, given recent methodological advances in stated-preference methods. METHODS The International Society for Pharmacoeconomics and Outcomes Research good research practices and other recommendations were used to conduct a discrete-choice experiment. Members of a US online panel with type 2 diabetes mellitus completed a Web-enabled, self-administered survey that elicited choices between treatment pairs with six attributes at three possible levels each. A D-efficient experimental design blocked 48 choice tasks into three 16-task surveys. Preference estimates were obtained using mixed logit estimation and were used to calculate choice probabilities. RESULTS A total of 552 participants (51% males) completed the survey. Avoiding 90 minutes of nausea was valued the highest (mean -10.00; 95% confidence interval [CI] -10.53 to -9.47). Participants wanted to avoid low blood glucose during the day and/or night (mean -3.87; 95% CI -4.32 to -3.42) or one pill and one injection per day (mean -7.04; 95% CI -7.63 to -6.45). Participants preferred stable blood glucose 6 d/wk (mean 4.63; 95% CI 4.15 to 5.12) and a 1% decrease in glycated hemoglobin (mean 5.74; 95% CI 5.22 to 6.25). If cost increased by

Improving the quality of discrete-choice experiments in health: How can we assess validity and reliability?

1, the probability that a treatment profile would be chosen decreased by 1%. CONCLUSIONS These results are consistent with the idea that people have strong preferences for immediate consequences of medication. Despite efforts to produce recommendations, ambiguity surrounding good practices remains and various judgments need to be made when conducting stated-preference studies. To ensure transparency, these judgments should be described and justified.

Education and patient preferences for treating type 2 diabetes: a stratified discrete-choice experiment

ABSTRACT Introduction: The recent endorsement of discrete-choice experiments (DCEs) and other stated-preference methods by regulatory and health technology assessment (HTA) agencies has placed a greater focus on demonstrating the validity and reliability of preference results. Areas covered: We present a practical overview of tests of validity and reliability that have been applied in the health DCE literature and explore other study qualities of DCEs. From the published literature, we identify a variety of methods to assess the validity and reliability of DCEs. We conceptualize these methods to create a conceptual model with four domains: measurement validity, measurement reliability, choice validity, and choice reliability. Each domain consists of three categories that can be assessed using one to four procedures (for a total of 24 tests). We present how these tests have been applied in the literature and direct readers to applications of these tests in the health DCE literature. Based on a stakeholder engagement exercise, we consider the importance of study characteristics beyond traditional concepts of validity and reliability. Expert commentary: We discuss study design considerations to assess the validity and reliability of a DCE, consider limitations to the current application of tests, and discuss future work to consider the quality of DCEs in healthcare.

Research as an event: a novel approach to promote patient-focused drug development

Purpose Diabetes is a chronic condition that is more prevalent among people with lower educational attainment. This study assessed the treatment preferences of patients with type 2 diabetes by educational attainment. Methods Patients with type 2 diabetes were recruited from a national online panel in the US. Treatment preferences were assessed using a discrete-choice experiment. Participants completed 16 choice tasks in which they compared pairs of treatment profiles composed of six attributes: A1c decrease, stable blood glucose, low blood glucose, nausea, treatment burden, and out-of-pocket cost. Choice models and willingness-to-pay (WTP) estimates were estimated using a conditional logit model and were stratified by educational status. Results A total of 231 participants with a high school diploma or less education, 156 participants with some college education, and 165 participants with a college degree or more completed the survey. Participants with a college degree or more education were willing to pay more for A1c decreases (

Identifying and prioritizing concerns associated with prosthetic devices for use in a benefit-risk assessment: a mixed-methods approach

58.84, standard error [SE]: 10.6) than participants who had completed some college (

Live, Learn, Pass It on

28.47, SE: 5.53) or high school or less (

Caregiver Treatment Preferences for Children with a New versus Existing Attention-Deficit/Hyperactivity Disorder Diagnosis

17.56, SE: 3.55) (p≤0.01). People with a college education were willing to pay more than people with high school or less to avoid nausea, low blood glucose events during the day/night, or two pills per day. Conclusion WTP for aspects of diabetes medication differed for people with a college education or more and a high school education or less. Advanced statistical methods might overcome limitations of stratification and advance understanding of preference heterogeneity for use in patient-centered benefit–risk assessments and personalized care approaches.

Assessing Rationality in Discrete Choice Experiments in Health: An Investigation into the Use of Dominance Tests

Patient groups are increasingly engaging in research to understand patients’ preferences and incorporate their perspectives into drug development and regulation. Several models of patient engagement have emerged, but there is little guidance on how to partner with patient groups to engage the disease community. Our group has been using an approach to engage patient groups that we call research as an event. Research as an event is a method for researchers to use a community-centered event to engage patients in their own environment at modest incremental cost. It is a pragmatic solution to address the challenges of engaging patients in research to minimize patients’ frustration, decrease the time burden, and limit the overall cost. The community, the event, and the research are the three components that constitute the research as an event framework. The community represents a disease-specific community. The event is a meeting of common interest for patients and other stakeholders, such as a patient advocacy conference. The research describes activities in engaging the community for the purpose of research. Research as an event follows a six-step approach. A case study is used to demonstrate the six steps followed by recommendations for future implementation.

Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy

ABSTRACT Objective: We identified and prioritized concerns reported by stakeholders associated with novel upper-limb prostheses. Methods: An evidence review and key-informant engagement, identified 62 concerns with upper-limb prostheses with implantable components. We selected 16 concerns for inclusion in a best-worst scaling (BWS) prioritization survey. Focus groups and BWS were used to engage stakeholders at a public meeting on prostheses. In 16 BWS choice tasks, attendees selected the most and least influential concern when choosing an upper-limb prosthesis. Aggregate data were analyzed using choice frequencies and conditional logit analysis. Latent class analysis examined heterogeneity in priorities. Estimates were adjusted to importance ratios which indicate how influential each concern is in the decision making process. Results: Forty-seven (47) stakeholders from diverse backgrounds completed the BWS survey (response rate 51%). On aggregate, the most influential concern was reliability of the device (importance ratio: 13%), and least influential was the concern of an outdated device (importance ratio: 1%). Latent class analysis identified two classes with approximately 50% of participants each. The first class was most influenced by effectiveness of the device. The second class was most influenced by minimizing risks. Conclusion: In this pilot, we identified heterogeneity in how participants prioritize concerns for upper-limb prostheses.