Do people have differing motivations for participating in a stated-preference study? Results from a latent-class analysis.

BACKGROUND: Researchers and policy makers have long suspected that people have differing, and potentially nefarious, motivations for participating in stated-preference studies such as discrete-choice experiments (DCE). While anecdotes and theories exist on why people participate in surveys, there is a paucity of evidence exploring variation in preferences for participating in stated-preference studies. METHODS: We used a DCE to estimate preferences for participating in preference research among an online survey panel sample. Preferences for the characteristics of a study to be conducted at a local hospital were assessed across five attributes (validity, relevance, bias, burden, time and payment) and described across three levels using a starring system. A D-efficient experimental design was used to construct three blocks of 12 choice tasks with two profiles each. Respondents were also asked about factors that motivated their choices. Mixed logistic regression was used to analyze the aggregate sample and latent class analysis identified segments of respondents. RESULTS: 629 respondents completed the experiment. In aggregate "study validity" was most important. Latent class results identified two segments based on underlying motivations: a quality-focused segment (76%) who focused most on validity, relevance, and bias and a convenience-focused segment (24%) who focused most on reimbursement and time. Quality-focused respondents spent more time completing the survey (p < 0.001) and were more likely to identify data quality (p < 0.01) and societal well-being (p < 0.01) as motivations to participate. CONCLUSIONS: This information can be used to better understand variability in motivations to participate in stated-preference surveys and the impact of motivations on response quality.

Care Management to Reduce Disparities and Control Hypertension in Primary Care: A Cost-effectiveness Analysis.

BACKGROUND: Project ReD CHiP (reducing disparities and controlling hypertension in primary care) care management was a clinic-based intervention that aimed to improve blood pressure control through improved care coordination and provide self-management support to patients from racially and socioeconomically. OBJECTIVE: To evaluate the cost-effectiveness of ReD CHiP care management versus standard care to treat hypertension in diverse communities. RESEARCH DESIGN: Microsimulation model from a health care sector perspective over 15 years. We used the published literature to inform our model including the ReD CHiP trial and the age-specific and race-specific cardiovascular disease risk equations. Deterministic and probabilistic sensitivity analyses were conducted to assess the uncertainty. SUBJECTS: Primary prevention in a racially diverse setting. MEASURES: Costs per quality-adjusted life years (QALYs) to produce an incremental cost-effectiveness ratio (ICER). RESULTS: ReD CHiP had an increase of $2114 and 0.04 QALYs. The ICER was $52,850/QALY. Predominately African American (ICER: $48,250/QALY) and elderly populations (ie, age 65+) derived value from ReD CHiP (ICER: $39,525/QALY). The value of ReD CHiP varied with changes in the reduction in systolic blood pressure (5 mm Hg reduction, ICER: $133,300/QALY; 15 mm Hg reduction, ICER: $18,767/QALY). Probabilistic sensitivity analysis indicated that ReD CHiP CM was cost-effective in over 90% of simulations, based on a willingness-to-pay of $100,000/QALY. CONCLUSIONS: ReD CHiP care management is cost-effective to prevent negative consequences of hypertension. African American and elderly patients have more favorable ICERs, recommending targeted interventions to improve health equity among vulnerable patient populations.

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

PURPOSE: Patient preference information (PPI) have an increasing role in regulatory decision-making, especially in benefit-risk assessment. PPI can also facilitate prioritization of symptoms to treat and inform meaningful selection of clinical trial endpoints. We engaged patients and caregivers to prioritize symptoms of Duchenne and Becker muscular dystrophy (DBMD) and explored preference heterogeneity. METHODS: Best-worst scaling (object case) was used to assess priorities across 11 symptoms of DBMD that impact quality of life and for which there is unmet need. Respondents selected the most and least important symptoms to treat among a subset of five. Relative importance scores were estimated for each symptom, and preference heterogeneity was identified using mixed logit and latent class analysis. RESULTS: Respondents included patients (n = 59) and caregivers (n = 96) affected by DBMD. Results indicated that respondents prioritized "weaker heart pumping" [score = 5.13; 95% CI (4.67, 5.59)] and pulmonary symptoms: "lung infections" [3.15; (2.80, 3.50)] and "weaker ability to cough" [2.65; (2.33, 2.97)] as the most important symptoms to treat and "poor attention span" as the least important symptom to treat [- 5.23; (- 5.93, - 4.54)]. Statistically significant preference heterogeneity existed (p value < 0.001). At least two classes existed with different priorities. Priorities of the majority latent class (80%) reflected the aggregate results, whereas the minority latent class (20%) did not distinguish among pulmonary and other symptoms. CONCLUSIONS: Estimates of the relative importance for symptoms of Duchenne muscular dystrophy indicated that symptoms with direct links to morbidity and mortality were prioritized above other non-skeletal muscle symptoms. Findings suggested the existence of preference heterogeneity for symptoms, which may be related to symptom experience.

Patient-centered benefit-risk assessment in duchenne muscular dystrophy.

INTRODUCTION: This study quantified caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregiver and patient differences were also explored. METHODS: A best-worst scaling survey (BWS) was administered to caregivers and patients. Across 9 profiles, respondents selected the best and worst attributes. Utility scores were estimated using mixed logistic regression. RESULTS: Respondents indicated greatest preference for therapies that maintain their current level of cough strength for 10 years or for 2 years. Preference scores for risks were low: 50% chance of diarrhea and 4 additional blood draws per year. CONCLUSION: There is a strong preference for pulmonary benefit and willingness to trade off risks and burden to achieve these benefits. In exchange for maintaining cough strength for 10 years, respondents were willing to tolerate high probabilities of diarrhea and additional blood draws. Muscle Nerve 55: 626-634, 2017.

Developing a Patient-Centered Benefit-Risk Survey: A Community-Engaged Process.

OBJECTIVES: To provide a community-engaged process to inform the design of a stated-preferences experiment. The process involved integrating patients and caregivers of people with Duchenne/Becker muscular dystrophy, advocates, clinicians, and the sponsor in conceptualizing and developing a benefit-risk survey on the basis of phase III trial results. METHODS: Our community-engagement process for the development of a stated-preference survey included a set of five guiding principles with a foundation in the principles of community-engaged research. Engagement efforts were carried out through an informal network of three committees. Members of the leadership, stakeholder, and review committees comprised patients, caregivers, clinicians, advocacy leadership, and industry representatives. RESULTS: Committee members participated in 15 hours of formal engagement including interviews and conference calls that ranged from 45 to 90 minutes, plus additional less-formal ad hoc communication. Committees comprised 20 individuals across three committees including adults with DMD (n = 6), parents of children with DMD (n = 6), clinicians (n = 3), members of research and advocacy organizations (n = 4), and an industry representative (n = 1). Community engagement informed attribute selection, survey length, word choice, and eligibility criteria. Challenges in the process included managing diverse stakeholder perspectives, time requirements, and the inherent tension between outcomes used in clinical trials versus attributes that correspond to patient- and family-relevant outcomes. CONCLUSIONS: We demonstrated how community engagement can successfully influence study design to support the design of a relevant survey instrument that is ethical, acceptable, meaningful to the community, and enhances patient-centered benefit-risk assessment for regulatory decision making.

Serving Older Adults with Complex Care Needs: A New Benefit Option for Medicare.

Medicare was originally designed to protect beneficiaries from the financial burden of acute episodes of illness. As lifespans lengthen, Medicare must adapt to serve beneficiaries with substantial long-term physical or cognitive impairment who need personal care assistance. These beneficiaries often incur high out-of-pocket costs for Medicare-covered services as well as home and community care not covered by Medicare. This latter category of care is often key to continued independence. To improve Medicare's capacity to serve such beneficiaries, and to prevent unnecessary institutionalization, this issue brief, one in a series on Medicare's future challenges, proposes a complex care benefit option that would include home and community services, and describes how it might be structured to balance the goals of improving care for beneficiaries and ensuring affordability.

Role Preferences in Medical Decision Making: Relevance and Implications for Health Preference Research.

Health preference research (HPR) is being increasingly conducted to better understand patient preferences for medical decisions. However, patients vary in their desire to play an active role in medical decisions. Until now, few studies have considered patients' preferred roles in decision making. In this opinion paper, we advocate for HPR researchers to assess and account for role preferences in their studies, to increase the relevance of their work for medical and shared decision making. We provide recommendations on how role preferences can be elicited and integrated with health preferences: (1) in formative research prior to a health preference study that aims to inform medical decisions or decision makers, (2a) in the development of health preference instruments, for instance by incorporating a role preference instrument and (2b) by clarifying the respondent's role in the decision prior to the preference elicitation task or by including role preferences as an attribute in the task itself, and (3) in statistical analysis by including random parameters or latent classes to raise awareness of heterogeneity in role preferences and how it relates to health preferences. Finally, we suggest redefining the decision process as a model that integrates the role and health preferences of the different parties that are involved. We believe that the field of HPR would benefit from learning more about the extent to which role preferences relate to health preferences, within the context of medical and shared decision making.

Economic Evaluation in Kidney Transplantation: A Scoping Review and Novel PESTLE Stakeholder Gaps Analysis.

AIM: The objective of this study was to systematically review the literature related to the economic evaluation of kidney transplantation to determine the extent of current research and identify gaps for future research. SUBJECT AND METHODS: We searched 4 medical and 2 economic electronic databases as well as hand-searching reference lists of review articles and other pertinent articles. Exclusion criteria included articles that did not include original work (ie, reviews), were not in English, and were not journal articles or economic working papers (eg, commentaries, theses, abstracts). Full-text data abstraction included qualitative and quantitative parameters with the intent to perform a gap analysis for future research. RESULTS: A total of 299 articles were included and spanned a 48-year period from 1968 to 2016, with >73% published in 2000 or after. The most common topics included immunosuppression drugs, dialysis vs kidney transplantation, organ allocation, and the potential market for donor organs. Most articles were from the United States and originated from 73 medical journals and 34 economic journals or working paper centers. There were 58 articles dealing with costing, 153 using cost-effectiveness, 69 using economic modeling, 6 performing systematic reviews with meta-analyses, and 13 exploring the qualitative financial environment of individuals and the economy. CONCLUSIONS: Research gaps were identified in every parameter used to evaluate the studies, and a new system of gap analysis for scoping reviews was also proposed.

Does price disclosure in pharmaceutical advertising result in price transparency? Evidence from a randomized experiment.

Background: Policies that mandate list price disclosure in direct-to-consumer pharmaceutical advertising (DTCPA) cite price transparency among the benefits. The expectation is that price transparency will lead to changes in consumer behavior that will ultimately lower healthcare costs. Objective: The objective of this study was to assess the impact of price transparency on perceived level of information and consumer behaviors, specifically intentions to seek treatment and intentions to comparison shop. Methods: A nine-arm randomized experiment was conducted to expose respondents to television advertisements for prescription drugs that varied by price disclosure type (no price/control, list price only, or price plus, which disclosed the list price and typical out-of-pocket cost) and indicated condition (deep vein thrombosis/pulmonary embolism [DVT/PE], diabetes, or rheumatoid arthritis [RA]). The sample was recruited from US adult members of the nationally representative Amerispeak online panel. Results: The sample included 2138 respondents. For ads featuring prescription drugs for DVT/PE, findings provide no evidence of an impact from price disclosure on perception of sufficient information. For ads for prescription drugs for diabetes, there was no evidence of an impact from list price only, but the price plus group was more likely than the control group to report the ad provided sufficient information (OR = 2.475). For ads for RA prescription drugs, both the list price only group (OR = 3.380) and price plus group (OR = 2.720) were more likely to report sufficient information than the control. Findings provide no evidence of an impact from price disclosure on consumer behaviors (i.e., intention to seek treatment or intention to comparison shop). Conclusions: Mandatory DTCPA list price disclosure may not be the most effective tool for improving price transparency and affecting consumer behavior.

Attention to price disclosures in direct-to-consumer prescription drug advertising and the impact on drug perceptions.

BACKGROUND: Mandatory disclosure of the price of prescription drugs within direct-to-consumer pharmaceutical advertisements (DTCA) has been proposed as a potential means of curbing rising drug costs in the United States. While price transparency in DTCA has widespread public support, empirical evidence regarding the effects of such drug prices disclosures remains limited. OBJECTIVES: This study assessed the degree to which a price disclosure was noticed, the individual characteristics associated with price disclosure recognition, and the impact on perceived drug affordability, effectiveness, and safety. METHODS: A randomized experiment was conducted among 2,138 members of the Amerispeak online panel. Participants were shown a television commercial for a drug treating either type II diabetes, deep vein thrombosis/pulmonary embolism (DVT/PE), or rheumatoid arthritis (RA) that disclosed the list price, disclosed the list price plus the average out-of-pocket cost, or had no price disclosure. RESULTS: Roughly forty percent of participants noticed when a price was disclosed while 20-24% noticed information about individual costs varying (the higher of these percentages occurred when the average out-of-pocket cost was provided). Attention did not vary systematically with the cost amount. Recognition of the different elements of the price disclosure were most predicted by sociodemographic variables such as race, education, and income as well as health characteristics. Price disclosure altered perceived affordability of the advertised medication in a manner consistent with the costs provided, but such consistent significant effects were not found for perceived drug effectiveness and safety. CONCLUSIONS: Repeated exposure to price disclosure in television DTCA or supplementary sources of price information may be necessary to increase attention to drug price information, especially among those who are most vulnerable to the burden of drug costs. Price transparency appears useful for adjusting affordability perceptions, but additional research needs to examine how such perceptions factor into healthcare decision-making and drug pricing.