Patient Preferences for Treatment in Relapsed/Refractory Acute Leukemia in the United Kingdom: A Discrete Choice Experiment.

Background: Acute leukemia is a cancer of the white blood cells which progresses rapidly and aggressively. There are two types: acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). The latter has a rare subtype: acute promyelocytic leukemia (APL). For some patients, following first-line treatment, remission is not achieved ("refractory disease"), and for others the leukemia returns after achieving remission ("relapse"). For these individuals, outcomes are typically poor. It is, therefore, important to understand patients' treatment priorities in this context. Methods: Building upon formative qualitative research, an online survey containing a discrete choice experiment (DCE) was designed to explore patients' treatment preferences in the relapsed/refractory setting. The DCE attributes were mode of administration; quality of life during treatment; chance of response; duration of response; and quality of life during response. Each respondent completed twelve scenarios containing two hypothetical treatments. Participants were eligible if they lived in the United Kingdom and had a diagnosis of acute leukemia. The data were analysed using a latent class model. Results: A total of 95 patients completed the survey. The latent class analysis identified two classes. For both, chance of response was the most important attribute. For class 1, every attribute was important, whereas for class 2, the only important attributes were quality of life (during treatment and response) and chance of response. A greater proportion of respondents would fall into class 1 overall, and those with ALL or APL and those more recently diagnosed were more likely to be in class 2. Conclusion: Our results indicate that patients are strongly concerned about the chance of response, as well as quality of life (to a lesser extent), when faced with different treatment options in the relapsed/refractory setting. However, there is significant preference heterogeneity within the patient population, and other treatment characteristics also matter to many.

A Longer Life or a Quality Death? A Discrete Choice Experiment to Estimate the Relative Importance of Different Aspects of End-of-Life Care in the United Kingdom.

Background. Advocates argue that end-of-life (EOL) care is systematically disadvantaged by the quality-adjusted life-year (QALY) framework. By definition, EOL care is short duration and not primarily intended to extend survival; therefore, it may be inappropriate to value a time element. The QALY also neglects nonhealth dimensions such as dignity, control, and family relations, which may be more important at EOL. Together, these suggest the QALY may be a flawed measure of the value of EOL care. To test these arguments, we administered a stated preference survey in a UK-representative public sample. Methods. We designed a discrete choice experiment (DCE) to understand public preferences over different EOL scenarios, focusing on the relative importance of survival, conventional health dimensions (especially physical symptoms and anxiety), and nonhealth dimensions such as family relations, dignity, and sense of control. We used latent class analysis to understand preference heterogeneity. Results. A 4-class latent class multinomial logit model had the best fit and illustrated important heterogeneity. A small class of respondents strongly prioritized survival, whereas most respondents gave relatively little weight to survival and, generally speaking, prioritized nonhealth aspects. Conclusions. This DCE illustrates important heterogeneity in preferences within UK respondents. Despite some preferences for core elements of the QALY, we suggest that most respondents favored what has been called "a good death" over maximizing survival and find that respondents tended to prioritize nonhealth over conventional health aspects of quality. Together, this appears to support arguments that the QALY is a poor measure of the value of EOL care. We recommend moving away from health-related quality of life and toward a more holistic perspective on well-being in assessing EOL and other interventions. Highlights: Advocates argue that some interventions, including but not limited to end-of-life (EOL) care, are valued by patients and the public but are systematically disadvantaged by the quality-adjusted life-year (QALY) framework, leading to an unfair and inefficient allocation of health care resources.Using a discrete choice experiment, we find some support for this argument. Only a small proportion of public respondents prioritized survival in EOL scenarios, and most prioritized nonhealth aspects such as dignity and family relations.Together, these results suggest that the QALY may be a poor measure of the value of EOL care, as it neglects nonhealth aspects of quality and well-being that appear to be important to people in hypothetical EOL scenarios.

Challenges in Valuing Temporary Health States for Economic Evaluation: A Review of Empirical Applications of the Chained Time Trade-Off Method.

BACKGROUND: The time trade-off (TTO) technique is commonly used to elicit health state utilities. Nevertheless, when the health states being valued are temporary, the TTO approach may be unsuitable. A variant of TTO- chained TTO-has been suggested to be used when the health states are temporary, but little research has been done on how chained TTO should be conducted. OBJECTIVES: To systematically review the use of chained TTO in valuing temporary health states. METHODS: A systematic literature search was conducted using the following major databases: Ovid MEDLINE(R), Embase, EBM Reviews, and PsycINFO. Abstracts (full articles if necessary) were screened by two independent reviewers, with a third reviewer resolving any disagreements. RESULTS: The resulting number of articles for review was low (n = 9). All the reviewed studies used face-to-face interviews, most had small sample sizes (<100), and all studies valued a small number of health states (<7), with time horizons typically ranging from 4 weeks to 1 year. All studies discussed methodological issues of using chained TTO, and some compared the results with those generated using other preference elicitation methods. CONCLUSIONS: Chained TTO appears to be feasible, consistent, and responsive and allows the valuation of temporary health states that would improve the efficiency and accuracy of decision making in health and health care. Nevertheless, the evidence is limited due to the low number of relevant studies in the literature. Further research is needed to examine the performance and validity of chained TTO compared with conventional TTO in the valuation of temporary health states.

Whose preferences should be elicited for use in health-care decision-making? A case study using anticoagulant therapy.

The question of whose preferences to elicit in health-state valuation has been widely discussed in the literature. The importance of this debate lies in the fact that health-state utility values are used in health technology assessment (HTA); therefore, an individual's preferences can influence decision-making. If preferences differ across groups, making decisions based on one group's preferences may be suboptimal for the other. Preferences for benefits, risks, experiences and health states associated with anticoagulant therapies have been elicited by researchers due to the underutilization of warfarin and the introduction of non-vitamin K antagonist oral anticoagulants. The majority of existing studies elicit preferences from patient populations as opposed to other stakeholders such as the general public. This paper extends the preference debate by using this clinical area as a case study, with a particular focus on HTA guidelines and the recent advocacy of the use of preference information in benefit-risk assessments.