Review articlePatient and general public preferences for health states: A call to reconsider current guidelines
Introduction
Economic evaluations serve the purpose of supporting the process of allocating the health care budget in such a way that it optimally reflects a set of objectives agreed to by society or a relevant decision maker. In general, an economic evaluation identifies whether a new treatment increases welfare by considering whether its (incremental) benefits exceed its incremental costs. In the context of a fixed budget, the approach is often used to assert whether a new treatment gains more health per invested euro than the displaced activities due to the re-allocation of resources (Claxton et al., 2011).
In economic evaluations, effects of health care interventions are commonly measured and valued in terms of Quality Adjusted Life Years (QALYs). The QALY combines quality of life (morbidity) and survival (mortality) in a single metric. Preferences indicate the desirability of health states compared to being in full health and form the quality adjustment of the QALY. These preferences are typically elicited by asking respondents to imagine being in some impaired health state for a fixed period of time. Generally, respondents are then asked to choose between this option and living shorter but in perfect health (Time Trade-Off) or between this option and a procedure restoring perfect health but with a risk of immediate death (Standard Gamble). Using such preferences, and anchoring the value of the state ‘dead’ at 0 and that of perfect health at 1, allows QALY scores for health states to be computed. These are subsequently used to determine health gains from treatment in an economic evaluation; the differences between the QALY score prior to treatment and that of the QALY score post treatment (or that between an intervention and a control group) determines the size of the gain (or loss) in quality and/or length of life. Preferences elicited from the general public (referred to here as ‘general public preferences’) are often considered to be most appropriate for use in health care decision making. However, there has always been considerable doubt that public preferences “tell the whole story” (Nord et al., 2005) and the issue remained a topic of debate throughout the past years (Dolan and Kahneman, 2008, Dolan, 2010, Dolan and Metcalfe, 2012, McTaggart-Cowan et al., 2011, Nord et al., 2009, Stiggelbout and De Vogel-Voogt, 2008).
International guidelines identify different sources of preference as appropriate for health care decision-making. In for instance the Netherlands and the United Kingdom, regulatory bodies prescribe preferences for health states used in the context of economic evaluations to be obtained from the general public. The Dutch guidelines justify this choice by stating that general public preferences are in line with the societal perspective, the insurance principle and that adaptation of patients to their condition might result in unfavorable cost-utility ratio's (Zorginstituut Nederland, 2015). In Sweden, however, patient preferences are preferred (The Dental and Pharmaceutical Benefits Agency (TLV), 2003). This choice can be of great importance: it has been shown that, on average, patients indicate a smaller impact of health impairments than expected by the general public (Krahn et al., 2003, Noel et al., 2015, Peeters and Stiggelbout, 2010, Zethraeus and Johannesson, 1999). This difference may be larger for worse health states (Burström et al., 2006), although there are studies reporting opposite effects (Lloyd et al., 2008, Pyne et al., 2009, Stolk and Busschbach, 2003), as well as studies that only report a difference when preferences are measured with a time trade-off task, rather than with a discrete choice task (Krabbe et al., 2011). In some instances using patient preferences may result in more favorable cost-utility ratios for interventions treating those patients than using public preferences (Oldridge et al., 2008), but this need not to be the case (Schackman et al., 2002). The effect of using patient or public preferences also depends on whether an intervention is purely quality of life improving or rather life prolonging, as we will highlight in this paper. With patient preferences we here mean preferences obtained in those who are actually in the health state under value and thus experience this health state.
The differences between general public and patient preferences, and the international differences in guidelines in this context, stress the need for an open and thorough discussion of the issue of source of valuation of health states to be used in economic evaluations. This paper aims to contribute to this discussion against the background of decision-making regarding the reimbursement of new health care interventions. We will start from the current practice in which the QALY, interpreted from either a welfarist or an extra-welfarist perspective (Brouwer et al., 2008), is the commonly used maximand (potentially somehow corrected for distributive concerns). In this paper, we focus on the question whose preferences should form the basis of this maximand. This discussion is to be seen against this decision making background, since the size of the identified benefit in economic evaluations may depend on normative choices regarding whose preferences for health states matter. We aim to further the discussion regarding health state valuations in the context of economic evaluations by introducing a way forward which includes the use of patient preferences alongside general public preferences. To that end, and given the dominance of the general public as source of health state valuation, we will revisit some main arguments for using general public preferences put forward in the debate. We will conclude that the current justification for the use of general public preferences for health states is, at best, incomplete.
Section snippets
Background
We first clarify some important concepts that are central to the discussion.
Revisiting the arguments
Without claiming to be exhaustive, we discuss some of the main arguments put forward in favor of using public preferences below. We will argue that none of these arguments, alone or jointly, provides a convincing argument for solely using either general public preferences or patient preferences.
Way forward
Although the properties of both the public and the patient preference perspectives have been extensively discussed in the literature, there does not seem to be any conclusive or theoretically sound justification for disregarding either perspective, as has been argued before (Drummond et al., 2009, Menzel et al., 2002, Menzel, 2014). One suggestion is that “members of the general public could be informed about the views of those who have experienced the health states that are the subject of
Conclusion
Preferences for health states are commonly derived from the general public, rather than from actual patients. We argued that both viewpoints are reasonable and compelling, and that, therefore, the challenge is to investigate possibilities to intelligently combine the different sources of information.
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