Multidimensional Thresholding for Individual-Level Preference Elicitation.

OBJECTIVES: Multiple methods are available for collecting health preference information. However, information on the design and analysis of novel methods is limited. This article aims to provide the first introduction into the design and analysis of multidimensional thresholding (MDT). METHODS: We introduce MDT as a 2-step approach: First, participants rank the largest possible improvements in all considered attributes by their importance. Second, participants complete a series of systematically combined trade-off questions. Hit-and-Run sampling is used for obtaining preference weights. We also use a computational experiment to compare different MDT designs. RESULTS: The outlined MDT can generate preference information suitable for specifying a multiattribute utility function at the individual level. The computational experiment demonstrates the method's ability to recover preference weights at a high level of precision. While all designs in the computation experiment perform comparably well on average, the design outlined in the paper stands out with a high level of precision even if differences in relative attribute importance are large. CONCLUSION: MDT is suitable for preference elicitation, in particular if sample sizes are small. Future research should help improve the methods (e.g., remove the need for an initial ranking) to increase the potential reach of MDT.

Multistakeholder Perceptions of Additional Value Elements for United States Value Assessment of Health Interventions.

OBJECTIVES: Limitations in conventional cost-effectiveness methods have led to calls for incorporation of additional value elements in assessments of health technologies. However, gaps remain in how additional value elements may inform decision making. This study aimed to prioritize additional value elements from the perspective of healthy individuals without a specific condition or indicated for a specific treatment in the United States among a multistakeholder panel and compare the importance of perspective-specific value elements. METHODS: Additional value elements were prioritized in 2 phases: (1) we identified and categorized additional value elements in a targeted literature review, and (2) we convened a multistakeholder group-based preference elicitation study (N = 28) to evaluate the description of each value element and rank and generate normalized weights of each value element for its significance in value assessment. The importance of additional value elements was also weighted relative to patient-centric value elements. RESULTS: The rank and weight of contextual value elements among 28 stakeholders were "severity of the disease" (26.2%), "disadvantaged and vulnerable target populations highly represented" (21.8%), "broader economic impact" (17.3%), "risk protection" (13.8%), "rarity of the disease" (11.3%), and "novel mechanism of action" (9.7%). Relative weight of the additional value elements versus patient-centric value elements was 52% and 48%, respectively. CONCLUSIONS: Study findings may inform priority setting for value frameworks and emerging US government assessments. The group-based elicitation method is repeatable and useful for structured deliberative processes in value assessment and may help improve the consistency and predictability of what is important to stakeholders.

Using the Online Elicitation of Personal Utility Functions Approach to Derive a Patient-Based 5-Level Version of EQ-5D Value Set: A Study in 122 Patients With Rheumatic Diseases From Germany.

OBJECTIVES: Traditional preference elicitation methods, such as discrete choice experiments or time trade-off, usually require large sample sizes. This can limit their applicability in patient populations, where recruiting enough participants can be challenging. The objective of this study was to test a new method, called the Online elicitation of Personal Utility Functions (OPUF) approach, to derive an EQ-5D-5L value set from a relatively small sample of patients with rheumatic diseases. METHODS: OPUF is a new type of online survey that implements compositional preference elicitation techniques. Central to the method are 3 valuation steps: (1) dimension weighting, (2) level rating, and (3) anchoring. An English demo version of the OPUF survey can be accessed at https://valorem.health/eq5d5l. From the responses, a personal EQ-5D-5L utility function can be constructed for each participant, and a group-level value set can be derived by aggregating model coefficients across participants. RESULTS: A total of 122 patients with rheumatic disease from Germany completed the OPUF survey. The survey was generally well received; most participants completed the survey in less than 20 minutes and were able to derive a full EQ-5D-5L value set. The precision of mean coefficients was high, despite the small sample size. CONCLUSIONS: Our findings demonstrate that OPUF can be used to derive an EQ-5D-5L value set from a relatively small sample of patients. Although the method is still under development, we think that it has the potential to be a valuable preference elicitation tool and to complement traditional methods in several areas.

Development and Valuation of a Preference-Weighted Measure in Age-Related Macular Degeneration From the Vision Impairment in Low Luminance Questionnaire: A MACUSTAR Report.

OBJECTIVES: This study generates VILL-UI (Vision Impairment in Low Luminance - Utility Index), a preference-weighted measure (PWM) derived from the VILL-33 measure for use in patients with age-related macular degeneration (AMD) and valued to generate United Kingdom and German preference weights. METHODS: A PWM consists of a classification system to describe health and utility values for every state described by the classification. The classification was derived using existing data collected as part of the MACUSTAR study, a low-interventional study on AMD, conducted at 20 clinical sites across Europe. Items were selected using psychometric and Rasch analyses, published criteria around PWM suitability, alongside instrument developer views and concept elicitation work that informed VILL-33 development. An online discrete choice experiment (DCE) with duration of the health state was conducted with the United Kingdom and German public. Responses were modeled to generate utility values for all possible health states. RESULTS: The classification system has 5 items across the 3 domains of VILL-33: reading and accessing information, mobility and safety, and emotional well-being. The DCE samples (United Kingdom: n = 1004, Germany: n = 1008) are broadly representative and demonstrate good understanding of the tasks. The final DCE analyses produce logically consistent and significant coefficients. CONCLUSIONS: This study enables responses to VILL-33 to be directly used to inform economic evaluation in AMD. The elicitation of preferences from both United Kingdom and Germany enables greater application of VILL-UI for economic evaluation throughout Europe. VILL-UI fills a gap in AMD in which generic preference-weighted measures typically lack sensitivity.

Preferences as fairness judgments: a critical review of normative frameworks of preference elicitation and development of an alternative based on constitutional economics.

Preference elicitation is widely used within health economic evaluations to inform coverage decisions. However, coverage decisions involve questions of social justice and it is unclear what role empirical evidence about preferences can play here. This study reviews the prevalent normative frameworks for using population-based preference elicitation and the criticisms they face, and proposes an alternative based on constitutional economics. The frameworks reviewed include a supposedly value-neutral framework of preferences as predictors of choice, preference utilitarian frameworks that aim to maximize preference satisfaction, and substantive consequentialist frameworks that aim to maximize happiness, health, or capabilities. The proposed alternative implements the idea that indices of social value are tools for conflict resolution, rather than tools for maximization. Preference elicitation is used for validating values generated by multi-criteria decision analysis results within representative processes of stakeholder deliberation.

Exploring health preference heterogeneity in the UK: Using the online elicitation of personal utility functions approach to construct EQ-5D-5L value functions on societal, group and individual level.

A new method has recently been developed for valuing health states, called 'Online elicitation of Personal Utility Functions' (OPUF). In contrast to established methods, such as time trade-off or discrete choice experiments, OPUF does not require hundreds of respondents, but allows estimating utility functions for small groups and even at the individual level. In this study, we used OPUF to elicit EQ-5D-5L health state preferences from a (not representative) sample of the UK general population, and then compared utility functions on the societal-, group-, and individual level. A demo version of the survey is available at: https://eq5d5l.me. Data from 874 respondents were included in the analysis. For each respondent, we constructed a personal EQ-5D-5L value set. These personal value sets predicted respondents' choices in three hold-out discrete choice tasks with an accuracy of 78%. Overall, preferences varied greatly between individuals. However, PERMANOVA analysis showed that demographic characteristics explained only a small proportion of the variability between subgroups. While OPUF is still under development, it has important strengths: it can be used to construct value sets for patient reported outcome instruments such as EQ-5D-5L, while also allowing examination of underlying preferences in an unprecedented level of detail. In the future, OPUF could be used to complement existing methods, allowing valuation studies in smaller samples, and providing more detailed insights into the heterogeneity of preferences across subgroups.

Identifying pregnant and postpartum women's priorities for enhancing nutrition support through social needs programmes in a resource-constrained urban community in South Africa.

BACKGROUND: Malnutrition remains a pressing public health concern for mothers and children in South Africa. Despite the government's multisectoral response, unaddressed social needs prevent some mothers getting full benefit from interventions, spanning financial planning, income stability, housing, access to government services, social support, and provision of affordable, nutritious foods. Engaging with mothers and prioritising their concerns is important if we wish to overcome obstacles to women benefiting from government nutrition interventions. This study aimed to identify the programmes that women perceived as a priority in addressing the social needs of mothers of young infants and pregnant women to enhance nutrition in a resource-constrained urban township in South Africa. METHODS: A cross-sectional study employed a quantitative preference elicitation survey, administered to 210 mothers and pregnant women from five primary healthcare facilities in Soweto. The survey tool was developed with the community to identify unmet social needs and potential solutions, which were synthesised with findings from the literature. The survey described 15 programmes, grouped into three delivery levels: clinics, community, and government. Participants were required to rank programme options in two stages. First, they selected their top two programmes within each delivery level. Subsequently, they allocated stickers to indicate the strength of their preference among the top programmes across the levels. Rankings were analysed using descriptive statistics. RESULTS: The highest priority was given to five programmes. Two delivered at the community level: Women's economic empowerment groups and Job search assistance, two at the clinic level: Social needs assessment and referral, and Prescription-based food, and one at the government level: Free quality childcare. The lowest-ranked programmes were two clinic-based programmes, specifically Maternal nutrition groups and Couple antenatal education. CONCLUSION: Women expressed strong views about which programmes should be prioritised to support mothers and pregnant women in addressing their social needs and improving nutrition. Key areas included providing support with job searching and entrepreneurship, accessing childcare and the healthy foods recommended at clinics, as well as finding information on available community and government services. Leveraging multisectoral collaboration, aligned policy objectives, efficient public financing, and strengthened implementation capacity will be pivotal in delivering these programmes.

Suitability of Preference Methods Across the Medical Product Lifecycle: A Multicriteria Decision Analysis.

OBJECTIVES: This study aimed to understand the importance of criteria describing methods (eg, duration, costs, validity, and outcomes) according to decision makers for each decision point in the medical product lifecycle (MPLC) and to determine the suitability of a discrete choice experiment, swing weighting, probabilistic threshold technique, and best-worst scale cases 1 and 2 at each decision point in the MPLC. METHODS: Applying multicriteria decision analysis, an online survey was sent to MPLC decision makers (ie, industry, regulatory, and health technology assessment representatives). They ranked and weighted 19 methods criteria from an existing performance matrix about their respective decisions across the MPLC. All criteria were given a relative weight based on the ranking and rating in the survey after which an overall suitability score was calculated for each preference elicitation method per decision point. Sensitivity analyses were conducted to reflect uncertainty in the performance matrix. RESULTS: Fifty-nine industry, 29 regulatory, and 5 health technology assessment representatives completed the surveys. Overall, "estimating trade-offs between treatment characteristics" and "estimating weights for treatment characteristics" were highly important criteria throughout all MPLC decision points, whereas other criteria were most important only for specific MPLC stages. Swing weighting and probabilistic threshold technique received significantly higher suitability scores across decision points than other methods. Sensitivity analyses showed substantial impact of uncertainty in the performance matrix. CONCLUSION: Although discrete choice experiment is the most applied preference elicitation method, other methods should also be considered to address the needs of decision makers. Development of evidence-based guidance documents for designing, conducting, and analyzing such methods could enhance their use.

UK Valuation of EQ-5D-5L, a Generic Measure of Health-Related Quality of Life: A Study Protocol.

OBJECTIVES: A high-quality and widely accepted UK EQ-5D-5L value set is urgently required to enable the latest version of EQ-5D scored using recent UK public preferences to inform policy including health technology assessments submitted to the National Institute for Health and Care Excellence. This article outlines the study protocol for the generation of a new EQ-5D-5L UK value set. METHODS: Twelve hundred interviews will be undertaken using the composite time trade-off elicitation technique for 102 health states (86 from the international EQ-5D-5L valuation protocol, plus 16 with best predictive performance in an extended design used in the Native American EQ-5D-5L valuation). The sample will be UK adults (age ≥18 years) proportionately representative across England, Wales, Scotland, and Northern Ireland, representative for age, sex, ethnicity, and socioeconomic group, with inclusion of participants with/without health problems. Participants will choose to be interviewed via videoconference (by Zoom) or in-person in a central venue. Data quality will be rigorously assessed. RESULTS: The value set will be generated using tobit random effects and heteroscedastic tobit models (with censoring at -1) using all data, excluding time trade-off values highlighted by participants as ones they would reconsider and data from interviewers failing protocol compliance. Quality and acceptance will be achieved by public involvement, regular Steering Group meetings, independent assessment of data quality at 4 time points, and final endorsement of data and analyses. CONCLUSION: This study will produce a UK value set for the EQ-5D-5L for use in prospective and retrospective data sets containing EQ-5D-5L data.

Optimizing Uptake of Long-Acting Injectable Pre-exposure Prophylaxis for HIV Prevention for Men Who Have Sex with Men.

Pre-exposure prophylaxis (PrEP) is a highly effective HIV prevention tool. Long-acting injectable PrEP (LAI-PrEP) offers another opportunity to reduce HIV. However, how at-risk individuals will consider LAI-PrEP over other modes of administration is unclear. We conducted a discrete choice experiment on preferences for PrEP among a sample of N = 688 gay, bisexual, and other men who have sex with men (GBMSM). We analyzed preferences for mode of administration, side-effects, monetary cost, and time cost using a conditional logit model and predicted preference for PrEP options. LAI-PrEP was preferred, despite mode of administration being the least important PrEP attribute. Side-effects were the most important attribute influencing preferences for PrEP (44% of decision); costs were second-most-important (35% of decision). PrEP with no side-effects was the most important preference, followed by monthly out-of-pocket costs of $0. Practitioners and policymakers looking to increase PrEP uptake should keep costs low, communicate clearly about PrEP side-effects, and allow the use of patient-preferred modes of PrEP administration, including LAI-PrEP.

RESUMEN: La profilaxis prexposición (PrEP) es una herramienta de prevención del VIH muy eficaz. La PrEP inyectable de acción prolongada (LAI-PrEP) ofrece otra oportunidad para reducir el VIH. Sin embargo, no está claro cómo las personas en riesgo considerarán LAI-PrEP sobre otros modos de administración. Realizamos un experimento de elección discreta sobre las preferencias por la PrEP entre una muestra de N = 688 hombres homosexuales, bisexuales y otros hombres que tienen sexo con hombres (GBMSM). Analizamos las preferencias por el modo de administración, los efectos secundarios, el costo monetario y el costo del tiempo mediante un modelo logit condicional y la preferencia prevista por las opciones de PrEP. Se prefirió LAI-PrEP, a pesar de que el modo de administración es el atributo de PrEP menos importante. Los efectos secundarios fueron el atributo más importante que influyó en las preferencias por la PrEP (44% de la decisión); los costos fueron los segundos más importantes (35% de la decisión). La PrEP sin efectos secundarios fue la preferencia más importante, seguida de costos de bolsillo mensuales de $0. Los médicos y legisladores que buscan aumentar la aceptación de la PrEP deben mantener los costos bajos, comunicar claramente los efectos secundarios de la PrEP y permitir el uso de los modos de administración de la PrEP preferidos por los pacientes, incluido LAI-PrEP.

Preference Elicitation and Treatment Decision-Making Among Men Diagnosed With Prostate Cancer: Randomized Controlled Trial Results of Healium.

BACKGROUND: Elicitation of patients' preferences is an integral part of shared decision-making, the recommended approach for prostate cancer decision-making. Existing decision aids for this population often do not specifically focus on patients' preferences. Healium is a brief interactive web-based decision aid that aims to elicit patients' treatment preferences and is designed for a low health literate population. OBJECTIVE: This study used a randomized controlled trial to evaluate whether Healium, designed to target preference elicitation, is as efficacious as Healing Choices, a comprehensive education and decision tool, in improving outcomes for decision-making and emotional quality of life. METHODS: Patients diagnosed with localized prostate cancer who had not yet made a treatment decision were randomly assigned to the brief Healium intervention or Healing Choices, a decision aid previously developed by our group that serves as a virtual information center on prostate cancer diagnosis and treatment. Assessments were completed at baseline, 6 weeks, and 3 months post baseline, and included decisional outcomes (decisional conflict, satisfaction with decision, and preparation for decision-making), and emotional quality of life (anxiety/tension and depression), along with demographics, comorbidities, and health literacy. RESULTS: A total of 327 individuals consented to participate in the study (171 were randomized to the Healium intervention arm and 156 were randomized to Healing Choices). The majority of the sample was non-Hispanic (272/282, 96%), White (239/314, 76%), married (251/320, 78.4%), and was on average 62.4 (SD 6.9) years old. Within both arms, there was a significant decrease in decisional conflict from baseline to 6 weeks postbaseline (Healium, P≤.001; Healing Choices, P≤.001), and a significant increase in satisfaction with one's decision from 6 weeks to 3 months (Healium, P=.04; Healing Choices, P=.01). Within both arms, anxiety/tension (Healium, P=.23; Healing Choices, P=.27) and depression (Healium, P=.001; Healing Choices, P≤.001) decreased from baseline to 6 weeks, but only in the case of depression was the decrease statistically significant. CONCLUSIONS: Healium, our brief decision aid focusing on treatment preference elicitation, is as successful in reducing decisional conflict as our previously tested comprehensive decision aid, Healing Choices, and has the added benefit of brevity, making it the ideal tool for integration into the physician consultation and electronic medical record. TRIAL REGISTRATION: ClinicalTrials.gov NCT05800483; https://clinicaltrials.gov/study/NCT05800483.

Preference Variation: Where Does Health Risk Attitude Come Into the Equation?

OBJECTIVES: Decisions about health often involve risk, and different decision makers interpret and value risk information differently. Furthermore, an individual's attitude toward health-specific risks can contribute to variation in health preferences and behavior. This study aimed to determine whether and how health-risk attitude and heterogeneity of health preferences are related. METHODS: To study the association between health-risk attitude and preference heterogeneity, we selected 3 discrete choice experiment case studies in the health domain that included risk attributes and accounted for preference heterogeneity. Health-risk attitude was measured using the 13-item Health-Risk Attitude Scale (HRAS-13). We analyzed 2 types of heterogeneity via panel latent class analyses, namely, how health-risk attitude relates to (1) stochastic class allocation and (2) systematic preference heterogeneity. RESULTS: Our study did not find evidence that health-risk attitude as measured by the HRAS-13 distinguishes people between classes. Nevertheless, we did find evidence that the HRAS-13 can distinguish people's preferences for risk attributes within classes. This phenomenon was more pronounced in the patient samples than in the general population sample. Moreover, we found that numeracy and health literacy did distinguish people between classes. CONCLUSIONS: Modeling health-risk attitude as an individual characteristic underlying preference heterogeneity has the potential to improve model fit and model interpretations. Nevertheless, the results of this study highlight the need for further research into the association between health-risk attitude and preference heterogeneity beyond class membership, a different measure of health-risk attitude, and the communication of risks.

Barriers and Expectations for Patients in Post-Osteoporotic Fracture Care in France: The EFFEL Study.

OBJECTIVES: This study aimed to quantify the relative importance of barriers to better secondary prevention of osteoporotic fractures and of care expectations expressed by patients with osteoporotic fractures in France. METHODS: A qualitative exploration of potential barriers to care and expectations was undertaken through a systematic literature review and in-depth patients interviews. A list of 21 barriers and 21 expectations was identified. These were presented to 324 subjects with osteoporotic fractures, identified in a representative sample of the French population, in the form of best-worst scaling questionnaires. Patients rated the relative importance of the attributes, and arithmetic mean importance scores were calculated and ranked. A Bayesian hierarchical model was also performed to generate a relative importance score. Latent class analysis was performed to identify potential subgroups of patients with different response profiles. RESULTS: A total of 7 barriers were rated as the most important, relating to awareness of osteoporosis and coordination of care. The highest-ranked barrier, "my fracture is not related to osteoporosis," was significantly more important than all the others (mean importance score 0.45; 95% confidence interval 0.33-0.56). A similar ranking of attributes was obtained with both the arithmetic and the Bayesian approach. For expectations, no clear hierarchy of attributes was identified. Latent class analysis discriminated 3 classes of respondents with significant differences in response profiles (the educated environmentalists, the unaware, and the victims of the system). CONCLUSIONS: Better quality of care of osteoporosis and effective secondary fracture prevention will require improvements in patient education, training of healthcare professionals, and coordination of care.

Delivering patient-centered care through shared decision making in overactive bladder.

INTRODUCTION: Men and women living with overactive bladder (OAB) face many treatment decisions as they progress through the treatment pathway. Decisions to pursue specific therapies are highly preference sensitive and ideal for shared decision making (SDM). The aim of this narrative review is to provide urologists with a practical summary of methods to elicit preferences and facilitate SDM to promote patient-centered care for OAB. METHODS: We explore OAB as a preference sensitive condition through a review of treatment outcomes and present available data on prediction tools, patient preferences, and decision aids. We propose a paradigm for applying Everyday SDM to OAB care. RESULTS: Clinical outcome data points to equipoise (balanced outcomes) between options for first-, second-, and third-line OAB therapies, making OAB preference sensitive and appropriate for SDM. Methods to personalize care through individualized outcome prediction calculators and tools to elicit patient preferences are emerging. While patient information about OAB is readily available, we identified few OAB decision aids that facilitate patient preference elicitation and SDM. CONCLUSIONS: OAB is a preference sensitive condition, where treatment is largely based on the patient's preferences and values. SDM is an ideal approach to supporting patients through these treatment decisions. We propose the application of Everyday SDM, a personalized, clinically efficient methodology as a method to support patient-centered OAB care.

Two for the price of one: If moving beyond traditional single-best discrete choice experiments, should we use best-worst, best-best or ranking for preference elicitation?

This study undertook a head-to-head comparison of best-worst, best-best and ranking discrete choice experiments (DCEs) to help decide which method to use if moving beyond traditional single-best DCEs. Respondents were randomized to one of three preference elicitation methods. Rank-ordered (exploded) mixed logit models and respondent-reported data were used to compare methods and first and second choices. First choices differed from second choices and preferences differed between elicitation methods, even beyond scale and scale dynamics. First choices of best-worst had good choice consistency, scale dynamics and statistical efficiency, but this method's second choices performed worst. Ranking performed best on respondent-reported difficulty and preference; best-best's second choices on statistical efficiency. All three preference elicitation methods improve efficiency of data collection relative to using first choices only. However, differences in preferences between first and second choices challenge moving beyond single-best DCE. If nevertheless doing so, best-best and ranking are preferred over best-worst DCE.

The use of composite time trade-off and discrete choice experiment methods for the valuation of the Short Warwick-Edinburgh Mental Well-being Scale (SWEMWBS): a think-aloud study.

PURPOSE: To identify patterns and problems in completing composite time trade-off (C-TTO) and discrete choice experiment (DCE) exercises for the valuation of the Short Warwick-Edinburgh Mental Well-being Scale (SWEMWBS) to inform the optimisation of a valuation protocol. METHODS: Fourteen cognitive interviews were conducted in the UK using concurrent and retrospective think-aloud and probing techniques. Each participant completed 8 C-TTO tasks and 8 DCE tasks within a computer-assisted personal interview setting. Verbal information was transcribed verbatim. Axial coding and thematic analysis were used to organise the qualitative data and identify patterns and problems with the completion of tasks. RESULTS: While participants found the tasks generally manageable, five broad themes emerged to explain and optimise the response to the tasks. (1) Format and structure: attention to the design of practice examples, instructions, and layout were needed. (2) Items and levels: underlying relationships were discovered across different combinations of levels of SWEMWBS items. (3) Decision heuristics: participants engaged in diverse strategies to assist trade-off decisions. (4) Valuation feasibility: certain states were difficult to imagine, compare and quantify. (5) Valuation outcome: the data quality was affected by participants' discriminatory ability across states and their time trade-off decisions. CONCLUSION: The interviews contributed insights regarding the robustness of the proposed methods. The application of C-TTO and DCE valuation techniques was practical and suitable for capturing individual attitudes towards different mental well-being scenarios. A modified protocol informed by the results is being tested in a larger sample across the UK.

Transforming challenges into opportunities: conducting health preference research during the COVID-19 pandemic and beyond.

The disruptions to health research during the COVID-19 pandemic are being recognized globally, and there is a growing need for understanding the pandemic's impact on the health and health preferences of patients, caregivers, and the general public. Ongoing and planned health preference research (HPR) has been affected due to problems associated with recruitment, data collection, and data interpretation. While there are no "one size fits all" solutions, this commentary summarizes the key challenges in HPR within the context of the pandemic and offers pragmatic solutions and directions for future research. We recommend recruitment of a diverse, typically under-represented population in HPR using online, quota-based crowdsourcing platforms, and community partnerships. We foresee emerging evidence on remote, and telephone-based HPR modes of administration, with further studies on the shifts in preferences related to health and healthcare services as a result of the pandemic. We believe that the recalibration of HPR, due to what one would hope is an impermanent change, will permanently change how we conduct HPR in the future.

Comparison of online and face-to-face valuation of the EQ-5D-5L using composite time trade-off.

OBJECTIVE: The aim of this study was to compare online, unsupervised and face-to-face (F2F), supervised valuation of EQ-5D-5L health states using composite time trade-off (cTTO) tasks. METHODS: The official EuroQol experimental design and valuation protocol for the EQ-5D-5L of 86 health states were implemented in interviewer-assisted, F2F and unsupervised, online studies. Validity of preferences was assessed using prevalence of inconsistent valuations and expected patterns of TTO values. Respondent task engagement was measured using number of trade-offs and time per task. Trading patterns such as better-than-dead only was compared between modes. Value sets were generated using linear regression with a random intercept (RILR). Value set characteristics such as range of scale and dimension ranking were evaluated between modes. RESULTS: Five hundred one online and 1,134 F2F respondents completed the surveys. Mean elicited TTO values were higher online than F2F when compared by health state severity. Compared to F2F, a larger proportion of online respondents did not assign the poorest EQ-5D-5L health state (i.e., 55555) the lowest TTO value ([Online] 41.3% [F2F] 12.2%) (p < 0.001). A higher percentage of online cTTO tasks were completed in 3 trade-offs or fewer ([Online] 15.8% [F2F] 3.7%), (p < 0.001). When modeled using the RILR, the F2F range of scale was larger than online ([Online] 0.600 [F2F] 1.307) and the respective dimension rankings differed. CONCLUSIONS: Compared to F2F data, TTO tasks conducted online had more inconsistencies and decreased engagement, which contributed to compromised data quality. This study illustrates the challenges of conducting online valuation studies using the TTO approach.

Comparing clotting factors attributes across different methods of preference elicitation in haemophilia patients.

INTRODUCTION: Emerging, systematic approaches for capturing patient input, such as preference elicitation, can provide valuable information for the benefit-risk assessment of medical products for treating bleeding disorders, such as haemophilia. AIM: This study aims to identify existing and develop new methods to capture, rank and summarize preference scores for clotting factor therapies. METHODS: Haemophilia patient preference data were compiled from studies identified through literature review and publicly available US FDA patient-focused drug development meeting documents. Text mining was performed to identify major themes across studies. A standardized preference score was estimated and aggregated. RESULTS: Ten preference studies that employed qualitative (n = 3), and quantitative methods (n = 7) met the inclusion criteria. Text mining of qualitative and quantitative studies revealed similar themes as the standardized preference attribute importance. We found that seven quantitative studies employed discrete choice experiments (DCE)/conjoint analysis (CA) and examined a range of 5-12 attributes. For DCE/CA studies published prior to 2014 (n = 4), safety attributes (inhibitor and viral safety) were among the most important attributes, accounting for ~46% of the total utility measured. DCE/CA studies published after 2014 (n = 3) focused on frequency of infusion and reduction of bleeding risk, accounting for ~67% of the total utility. Interestingly, two studies that used different preference elicitation approaches (DCE and a monadic conjoint approach) both ranked infusion frequency as the most important attribute. CONCLUSIONS: Although there are few published patient preference studies for haemophilia, the results of this study can be viewed in the larger context of enhancing scientific methods of incorporating patient input in medical product development.

Ranking the Criteria Used in the Appraisal of Drugs for Reimbursement: A Stated Preferences Elicitation With Health Technology Assessment Stakeholders Across Jurisdictional Contexts.

OBJECTIVES: Our goal was to estimate the relative importance assigned to health technology assessment (HTA) criteria by stakeholders involved in the HTA process. HTA is an increasingly common framework used in the appraisal of drugs for public reimbursement. It identifies clinical, economic, social, and organizational criteria to be considered. The criteria can vary across jurisdictions and are typically appraised by multidisciplinary expert committees. Guidance on the relative weighing of criteria is often absent. METHODS: We elicited stakeholders' preferences using a single-scenario discrete choice experiment and a best-worst scaling model with conviction scores to assess the weights assigned to selected criteria by HTA stakeholders. We recruited 111 HTA stakeholders across multiple jurisdictions, including members of expert committees, clinical and economic experts, patients, and public payer representatives. Each judged twelve hypothetical cancer drug profiles for suitability for public funding and identified which characteristics were best and worst. In addition to standard discrete choice experiment and best-worst scaling models, we estimated a hybrid model to obtain a ranking of criteria by importance they played in the appraisal. RESULTS: A strong clinical benefit proved the most important criterion, followed by cost considerations, presence of adverse events, and availability of other treatments. The importance of clinical benefit was moderated by unmet need, adverse events, and number of patients. CONCLUSION: Policymakers might want to consider providing an explicit weighing scheme, or moving to a 2-stage selection process with an assessment of the quality of clinical evidence as a gatekeeping step for a full HTA review.