Evaluating discrete choice experiment willingness to pay [DCE-WTP] analysis and relative social willingness to pay [RS-WTP] analysis in a health technology assessment of a treatment for an ultra-rare childhood disease [CLN2].

BACKGROUND: Conventional cost-effectiveness analysis [CEA] using cost per QALY thresholds may counteract other incentives introduced to foster development of treatments for rare and ultra-rare diseases. Therefore, alternative economic evaluation methods were explored, namely Discrete Choice Experiment Willingness to Pay (DCE-WTP) and Relative Social Willingness to Pay (RS-WTP), to value interventions for an ultra-rare childhood disease, Neuronal Ceroid Lipofuscinosis type 2 (CLN2). RESEARCH DESIGN AND METHODS: Treatment for CLN2 was valued from a citizen's ('social') perspective using DCE-WTP and RS-WTP in a survey of 4,009 United Kingdom [UK] adults. Three attributes (initial quality of life, treatment effect, and life expectancy) were used in both analyses. For DCE-WTP, a cost attribute (marginal income tax increase) was also included. Optimal econometric models were identified. RESULTS: DCE-WTP indicated that UK adults are willing to pay incremental increases through taxation for improvements in CLN2 attributes. RS-WTP identified a willingness to allocate >40% of a pre-assigned healthcare budget to prevent child mortality and approximately 15% for improved health status. CONCLUSIONS: Both techniques illustrate substantive social WTP for CLN2 interventions, despite the small number of children benefitting. This highlights a gap between UK citizens' willingness to spend on rare disease interventions and current funding policies.

Measuring trade-offs in nephrology: a systematic review of discrete choice experiments and conjoint analysis studies.

Background: Discrete choice experiment (DCE), conjoint analysis or adaptive conjoint analysis methods are increasingly applied to obtain patient, clinician or community preferences in nephrology. This study systematically reviews the above-mentioned published choice studies providing an overview of the issues addressed, methods and findings. Methods: Choice studies relating to nephrology were identified using electronic databases, including Medline, Embase, PsychINFO and Econlit from 1990 to 2015. For inclusion in the review, studies had to primarily relate to kidney disease and include results from statistical (econometric) analyses of respondents' choice or preference. Studies meeting the inclusion criteria were assessed against a range of systematic review criteria, and methods and results summarized. Results: We identified 14 eligible studies from Europe, Australasia, North America and Asia, reporting preferences for treatment or screening, patient experiences, quality of life (QOL), health outcomes and priority-setting frameworks. Specific contexts included medical interventions in kidney transplantation and renal cell carcinoma, health policies for organ donation and allocation, dialysis modalities and end-of-life care, using a variety of statistical models. The characteristics of 'time' (i.e. transplant waiting time, dialysis hours, transport time) and QOL (pre- and post-transplant, or pre- and post-dialysis) consistently influenced patient and clinician preferences across the choice studies. Conclusions: DCE are increasingly used to obtain information about key preferences in kidney transplantation and dialysis. These study methods provide quantitative information about respondents' trade-offs between conflicting clinical and policy objectives, and can establish how preferences vary among stakeholder groups.

Discrete choice experiments in health economics: a review of the literature.

BACKGROUND: Discrete choice experiments (DCEs) are increasingly used in health economics to address a wide range of health policy-related concerns. OBJECTIVE: Broadly adopting the methodology of an earlier systematic review of health-related DCEs, which covered the period 2001-2008, we report whether earlier trends continued during 2009-2012. METHODS: This paper systematically reviews health-related DCEs published between 2009 and 2012, using the same database as the earlier published review (PubMed) to obtain citations, and the same range of search terms. RESULTS: A total of 179 health-related DCEs for 2009-2012 met the inclusion criteria for the review. We found a continuing trend towards conducting DCEs across a broader range of countries. However, the trend towards including fewer attributes was reversed, whilst the trend towards interview-based DCEs reversed because of increased computer administration. The trend towards using more flexible econometric models, including mixed logit and latent class, has also continued. Reporting of monetary values has fallen compared with earlier periods, but the proportion of studies estimating trade-offs between health outcomes and experience factors, or valuing outcomes in terms of utility scores, has increased, although use of odds ratios and probabilities has declined. The reassuring trend towards the use of more flexible and appropriate DCE designs and econometric methods has been reinforced by the increased use of qualitative methods to inform DCE processes and results. However, qualitative research methods are being used less often to inform attribute selection, which may make DCEs more susceptible to omitted variable bias if the decision framework is not known prior to the research project. CONCLUSIONS: The use of DCEs in healthcare continues to grow dramatically, as does the scope of applications across an expanding range of countries. There is increasing evidence that more sophisticated approaches to DCE design and analytical techniques are improving the quality of final outputs. That said, recent evidence that the use of qualitative methods to inform attribute selection has declined is of concern.

Who should be prioritized for renal transplantation?: Analysis of key stakeholder preferences using discrete choice experiments.

BACKGROUND: Policies for allocating deceased donor kidneys have recently shifted from allocation based on Human Leucocyte Antigen (HLA) tissue matching in the UK and USA. Newer allocation algorithms incorporate waiting time as a primary factor, and in the UK, young adults are also favoured. However, there is little contemporary UK research on the views of stakeholders in the transplant process to inform future allocation policy. This research project aimed to address this issue. METHODS: Discrete Choice Experiment (DCE) questionnaires were used to establish priorities for kidney transplantation among different stakeholder groups in the UK. Questionnaires were targeted at patients, carers, donors / relatives of deceased donors, and healthcare professionals. Attributes considered included: waiting time; donor-recipient HLA match; whether a recipient had dependents; diseases affecting life expectancy; and diseases affecting quality of life. RESULTS: Responses were obtained from 908 patients (including 98 ethnic minorities); 41 carers; 48 donors / relatives of deceased donors; and 113 healthcare professionals. The patient group demonstrated statistically different preferences for every attribute (i.e. significantly different from zero) so implying that changes in given attributes affected preferences, except when prioritizing those with no rather than moderate diseases affecting quality of life. The attributes valued highly related to waiting time, tissue match, prioritizing those with dependents, and prioritizing those with moderate rather than severe diseases affecting life expectancy. Some preferences differed between healthcare professionals and patients, and ethnic minority and non-ethnic minority patients. Only non-ethnic minority patients and healthcare professionals clearly prioritized those with better tissue matches. CONCLUSIONS: Our econometric results are broadly supportive of the 2006 shift in UK transplant policy which emphasized prioritizing the young and long waiters. However, our findings suggest the need for a further review in the light of observed differences in preferences amongst ethnic minorities, and also because those with dependents may be a further priority.

Balancing patient preferences and clinical needs: community versus hospital based care for patients with suspected DVT.

OBJECTIVE: To establish patients' preferences and willingness to pay (WTP) for different service models for suspected deep vein thrombosis (DVT). METHODS: We analysed patient responses to a discrete choice experiment (DCE) questionnaire which had been targeted at patients in Leicester, UK. The questionnaire elicited preferences/WTP for attributes of DVT provision including speed of diagnosis; access; continuity of care; and minimizing hospital visits. Additionally we evaluated trade-offs between clinical and service attributes. We analysed responses from 256 patients with suspected DVT (65% response rate). RESULTS: Respondents are WTP pound 4.82 per extra hour of dedicated DVT service provision; pound 17.12 per hospital visit avoided; pound 115.73 per day's reduction in diagnostic wait; and pound 179.32 for 'much' not 'some' continuity, or pound 56.88 for 'some' not 'lack' of continuity in nursing. CONCLUSIONS: Research evaluating different DVT service models usually reports on clinical efficacy in centres of excellence. Results show prompt diagnosis is valued by patients and may improve efficacy by reducing unnecessary anticoagulation. However, patients value 'process' measures such as continuity of care also. To ensure optimal provision, clinical benefit measurement ought to be augmented with information on patients' preferences.