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.

Social and environmental determinants of health among children with long-term movement impairment.

The healthcare research community increasingly recognizes the need to address social (SDOH) and environmental determinants of health (EDOH) to optimize health and healthcare. This is particularly relevant to disability and functioning and to those with child onset conditions that impair mobility and impact functioning and participation. Using the World Health Organization (WHO)'s International Classification of Functioning, Disability, and Health (ICF) as a comprehensive framework, this paper aims to discuss our understanding of the relationships between social and EDOH and outcomes among people with impaired mobility that impacts functioning. This paper offers suggestions for future developments and guidance to use SDOH and EDOH in research and clinical practice.

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.

Best-Worst Scaling and the Prioritization of Objects in Health: A Systematic Review.

BACKGROUND AND OBJECTIVE: Best-worst scaling is a theory-driven method that can be used to prioritize objects in health. We sought to characterize all studies of best-worst scaling to prioritize objects in health, to assess trends of using best-worst scaling in prioritization over time, and to assess the relationship between a legacy measure of quality (PREFS) and a novel assessment of subjective quality and policy relevance. METHODS: A systematic review identified studies published through to the end of 2021 that applied best-worst scaling to study priorities in health (PROSPERO CRD42020209745), updating a prior review published in 2016. The PubMed, EBSCOhost, Embase, Scopus, APA PsychInfo, Web of Science, and Google Scholar databases were used and were supplemented by a hand search. Data describing the application, development, design, administration/analysis, quality, and policy relevance were summarized and we tested for trends by comparing articles before and after 1 January, 2017. Multivariate statistics were then used to assess the relationships between PREFS, subjective quality, policy relevance, and other possible indicators. RESULTS: From a total of 2826 unique papers identified, 165 best-worst scaling studies were included in this review. Applications of best-worst scaling to study priorities in health have continued to grow (p < 0.01) and are now used in all regions of the world, most often to study the priorities of patients/consumers (67%). Several key trends can be observed over time: increased use of pretesting (p < 0.05); increased use of online administration (p < 0.01), and decreased use of paper self-administered surveys (p = 0.02); increased use of heterogeneity analysis (p = 0.02); an increase in having a clearly stated purpose (p < 0.01); and a decrease in comparing respondents to non-respondents (p = 0.01). The average sample size has more than doubled, from 228 to 472 respondents, but formal sample size justifications remain low (5.3%) and unchanged over time (p = 0.68). While the average PREFS score remained unchanged at 3.1/5, both subjective quality and policy relevance trended up, but changes were not statistically significant (p = 0.06 and p = 0.13). Most of the variation in subjective quality was driven by PREFS (R2 = 0.42), but it was also positively assosciated with policy relevance, heterogeneity analysis, and using a balanced incomplete block design, and was negatively associated with not using developmental methods and an increasing sample size. CONCLUSIONS: Using best-worst scaling to prioritize objects is now commonly used around the world to assess the priorities of patients and other stakeholders in health. Best practices are clearly emerging for best-worst scaling. Although legacy measures (PREFS) to measure study quality are reasonable, there may need to be new tools to assess both study quality and policy relevance.

Proximal Femoral Focal Deficiency/Congenital Femoral Deficiency: Evaluation and Management.

Proximal focal femoral deficiency is a congenital transverse deficiency in which the femur is globally smaller with a typical proximal deformity at the hip that may include distal involvement of knees, leg, and feet. Congenital femoral deficiency (CFD) describes a broader spectrum of longitudinal deficiency inclusive of proximal focal femoral deficiency. CFD may also include lateral distal femoral hypoplasia, knee cruciate ligament deficiency, rotational instability, patellar dislocation, fibular hemimelia, ray absence, and contralateral limb involvement. Treatment intends to maximize function by limb equalization and deformity correction ranging from nonsurgical management using prosthetics to amputation and may include lengthening, shortening, and complex limb reconstruction. Management decisions depend on overall severity and the patient and family's preferences and priorities. Owing to its complexity, CFD is best treated by clinicians with considerable deformity treatment experience who can help guide decision making and embark on a treatment course that will maximize the functional outcome.

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.

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.

Do Patient Preferences Align With Value Frameworks? A Discrete-Choice Experiment of Patients With Breast Cancer.

Purpose. Assess patient preferences for aspects of breast cancer treatments to evaluate and inform the usual assumptions in scoring rubrics for value frameworks. Methods. A discrete-choice experiment (DCE) was designed and implemented to collect quantitative evidence on preferences from 100 adult female patients with a self-reported physician diagnosis of stage 3 or stage 4 breast cancer. Respondents were asked to evaluate some of the treatment aspects currently considered in value frameworks. Respondents' choices were analyzed using logit-based regression models that produced preference weights for each treatment aspect considered. Aggregate- and individual-level preferences were used to assess the relative importance of treatment aspects and their variability across respondents. Results. As expected, better clinical outcomes were associated with higher preference weights. While life extensions with treatment were considered to be most important, respondents assigned great value to out-of-pocket cost of treatment, treatment route of administration, and the availability of reliable tests to help gauge treatment efficacy. Two respondent classes were identified in the sample. Differences in class-specific preferences were primarily associated with route of administration, out-of-pocket treatment cost, and the availability of a test to gauge treatment efficacy. Only patient cancer stage was found to be correlated with class assignment (P = 0.035). Given the distribution of individual-level preference estimates, preference for survival benefits are unlikely to be adequately described with two sets of preference weights. Conclusions. Although value frameworks are an important step in the systematic evaluation of medications in the context of a complex treatment landscape, the frameworks are still largely driven by expert judgment. Our results illustrate issues with this approach as patient preferences can be heterogeneous and different from the scoring weights currently provided by the frameworks.

Reporting Formative Qualitative Research to Support the Development of Quantitative Preference Study Protocols and Corresponding Survey Instruments: Guidelines for Authors and Reviewers.

BACKGROUND: Formative qualitative research is foundational to the methodological development process of quantitative health preference research (HPR). Despite its ability to improve the validity of the quantitative evidence, formative qualitative research is underreported. OBJECTIVE: To improve the frequency and quality of reporting, we developed guidelines for reporting this type of research. The guidelines focus on formative qualitative research used to develop robust and acceptable quantitative study protocols and corresponding survey instruments in HPR. METHODS: In December 2018, a steering committee was formed as a means to accumulate the expertise of the HPR community on the reporting guidelines (21 members, seven countries, multiple settings and disciplines). Using existing guidelines and examples, the committee constructed, revised, and refined the guidelines. The guidelines underwent beta testing by three researchers, and further revisions to the guidelines were made based on their feedback as well as on comments from members of the International Academy of Health Preference Research (IAHPR) and the editorial board of The Patient: Patient-Centered Outcomes Research. RESULTS: The guidelines have five components: introductory material (4 domains), methods (12), results/findings (2), discussion (2), and other (2). They are concordant with existing guidelines, published examples, beta-testing results, and expert comments. CONCLUSIONS: Publishing formative qualitative research is a necessary step toward strengthening the foundation of any quantitative study, enhancing the relevance of its preference evidence. The guidelines should aid researchers, reviewers, and regulatory agencies and promote transparency within HPR more broadly.

Collecting patient preference information using a Clinical Data Research Network: demonstrating feasibility with idiopathic pulmonary fibrosis.

Purpose: Rare diseases present challenges for accessing patient populations to conduct surveys. Clinical Data Research Networks (CDRNs) offer an opportunity to overcome those challenges by providing infrastructure for accessing patients and sharing data. This study aims to demonstrate the feasibility of collecting patient preference information for a rare disease in a CDRN, using idiopathic pulmonary fibrosis as proof of concept. Patients and methods: Utilizing a cohort of idiopathic pulmonary fibrosis (IPF) patients across a CDRN, a discrete choice experiment was administered via electronic and paper methods to collect patient preference information about benefits and risks of two therapeutic options. Survey data were augmented with data from electronic health records and patient-reported outcome surveys. Results: Thirty-three patients completed the preference experiment. The amount of choice attributable to a benefit of slowing of decline in lung function was 36%. Improving efficacy in terms of lung function was 2.16 times as important as improving efficacy in terms of shortness of breath. In terms of side effects, decreasing risk of gastrointestinal problems was 2.6 times as important as decreasing risk of sun sensitivity and 2.4 times as important as decreasing risk of liver injury. In terms of benefit-risk trade-offs, improving efficacy in terms of lung function was 1.6 times as important as decreasing risk of gastrointestinal problems. Conclusion: This study used IPF as a proof of concept to demonstrate the feasibility of collecting patient preference information in a CDRN. The network was advantageous to the study of patient preferences. Future research should continue to explore pathways for the collection and use of patient preference information across networks. The power of consolidated collection efforts may lead to the ability to use preference data to inform decision-making at the regional, specialty, or individual encounter level.

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.

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.

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.

Using Best-Worst Scaling to Investigate Preferences in Health Care.

INTRODUCTION: Best-worst scaling (BWS) is becoming increasingly popular to elicit preferences in health care. However, little is known about current practice and trends in the use of BWS in health care. This study aimed to identify, review and critically appraise BWS in health care, and to identify trends over time in key aspects of BWS. METHODS: A systematic review was conducted, using Medline (via Pubmed) and EMBASE to identify all English-language BWS studies published up until April 2016. Using a predefined extraction form, two reviewers independently selected articles and critically appraised the study quality, using the Purpose, Respondents, Explanation, Findings, Significance (PREFS) checklist. Trends over time periods (≤2010, 2011, 2012, 2013, 2014 and 2015) were assessed further. RESULTS: A total of 62 BWS studies were identified, of which 26 were BWS object case studies, 29 were BWS profile case studies and seven were BWS multi-profile case studies. About two thirds of the studies were performed in the last 2 years. Decreasing sample sizes and decreasing numbers of factors in BWS object case studies, as well as use of less complicated analytical methods, were observed in recent studies. The quality of the BWS studies was generally acceptable according to the PREFS checklist, except that most studies did not indicate whether the responders were similar to the non-responders. CONCLUSION: Use of BWS object case and BWS profile case has drastically increased in health care, especially in the last 2 years. In contrast with previous discrete-choice experiment reviews, there is increasing use of less sophisticated analytical methods.

A Scoping Review of Healthcare Costs for Patients with Cystic Fibrosis.

BACKGROUND: Cystic fibrosis (CF), one of the most common life-shortening genetic diseases, has no cure, but people living with it have seen improvements in their health and survival. The rising life expectancy and increased availability of treatment options has likely increased the lifetime costs of people living with CF. In addition, a recent drug approval for a therapy that targets the cause of the disease is one of the most expensive drugs worldwide. In light of these circumstances, it is important to have an updated understanding of the costs of CF therapy and management. This study aims to determine the extent of available literature that quantifies CF costs. METHODS: We used a scoping review framework to identify the sources and types of evidence available to determine the costs of CF therapy and management compared to the general population or a comparable population of people with other complex chronic conditions. We searched 14 databases for peer-reviewed studies and grey literature published in English since 1998. The search was conducted in August 2013 and updated in October 2014. RESULTS: We identified 28 studies that estimated overall, general CF costs. Of these, three studies compare CF costs to healthcare costs of a general population and only one of those provides a direct comparison of CF costs to the general population in order to calculate the incremental cost associated with CF. We estimate there are 98 systematic reviews that quantify the costs of comparable conditions and potentially provide a comparison group for people with CF. CONCLUSIONS: There is evidence available that attempts to quantify overall, general healthcare costs of people with CF, although much of it is outdated. However, there is much less evidence available that provides a comparison of these costs with either the general population or people with comparable conditions.

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.

Caregiver preferences for emerging duchenne muscular dystrophy treatments: a comparison of best-worst scaling and conjoint analysis.

BACKGROUND: Through Patient-Focused Drug Development, the US Food and Drug Administration (FDA) documents the perspective of patients and caregivers and are currently conducting 20 public meetings on a limited number of disease areas. Parent Project Muscular Dystrophy (PPMD), an advocacy organization for Duchenne muscular dystrophy (DMD), has demonstrated a community-engaged program of preference research that would complement the FDA's approach. OBJECTIVE: Our objective was to compare two stated-preference methods, best-worst scaling (BWS) and conjoint analysis, within a study measuring caregivers' DMD-treatment preferences. METHODS: Within one survey, two preference-elicitation methods were applied to 18 potential treatments incorporating six attributes and three levels. For each treatment profile, caregivers identified the best and worst feature and intention to use the treatment. We conducted three analyses to compare the elicitation methods using parameter estimates, conditional attribute importance and policy simulations focused on the 18 treatment profiles. For each, concordance between the results was compared using Spearman's rho. RESULTS: BWS and conjoint analysis produced similar parameter estimates (p < 0.01); conditional attribute importance (p < 0.01); and policy simulations (p < 0.01). Greatest concordance was observed for the benefit and risk parameters, with differences observed for nausea and knowledge about the drug-where a lack of monotonicity was observed when using conjoint analysis. CONCLUSIONS: The observed concordance between approaches demonstrates the reliability of the stated-preference methods. Given the simplicity of combining BWS and conjoint analysis on single profiles, a combination approach is easily adopted. Minor irregularities for the conjoint-analysis results could not be explained by additional analyses and needs to be the focus of future research.