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CONCISE REVIEW

Clinical

S. Birch1 and A.I. Ismail2*


for Health Economics and Policy Analysis, Department of Clinical Epidemiology and Biostatistics, McMaster University, 1200, Main Street West, Hamilton, Ontario, L8N 3Z5, Canada; and 2School of Dentistry, University of Michigan, 1011 N. University, D2361, Ann Arbor, MI 48109-1078; *corresponding author, ismailai@umich.edu
1Centre

Patient Preferences and the Measurement of Utilities in the Evaluation of Dental Technologies

J Dent Res 81(7):446-450, 2002

ABSTRACT
Advances in life sciences that are predicted in the 21st century will present many challenges for health professionals and policy-makers. The major questions will be how to allocate resources to pay for costs of new technologies and who will best benefit from advances in new diagnostic and treatment methods. We review in this paper the concept of utility and how it can be applied and expanded to provide data to help health professionals make decisions that are preferred by patients and the public at large. Utility is a measure of peoples well-being or preferences for outcomes. The measurement of utilities of a new diagnostic technology, for example, can be carried out with the use of simple methods that do not incorporate all of the uncertainties and potential outcomes associated with providing the test, or with more complex methods that can incorporate most uncertainties. This review describes and critiques the different measurement methods of utilities. KEY WORDS: dental technology, assessment, preferences, utilities, outcomes, health services research.

DECISION-MAKING AND PATIENT PREFERENCES


innovation health care provides new ways of protecting, Technologicalchallenges forindecision-makers abouttreatment opportunities promoting, and restoring health. Expanded involve major the health care services to make available and the ways to deliver these services. Advances in bioengineering, saliva diagnostics, and gene manipulations may lead to new methods of detecting diseases in the oral and craniofacial tissues and to ways of regenerating enamel and dentin. The ability to do more in terms of health outcomes must be countered by the desire to do better for patients and the public. Clinical effectiveness provides estimates of the expected change in health outcomes associated with different interventions. However, information on the preferences of patients is needed to inform health-care providers and decision-makers about the effects of improved outcomes, as well as with other aspects of the delivery of interventions, on patient well-being. Hence, it is important to recognize that the measurement of preferences or utilities encompasses informing a patient of all aspects of an intervention that may affect his or her well-being. Utility is a measure of individual well-being. The utility associated with a particular intervention measures the expected effect of undertaking the intervention on the individuals assessment of his or her well-being. For example, root canal treatment might produce a greater expected improvement in oral health than a simple restoration. However, to be preferred by a patient, the well-being associated with the greater health improvement must more than offset the reduction in well-being associated with the additional inconvenience, suffering, and costs associated with root canal treatment. In this case, root canal treatment might be in the best interests of the patients oral health, but not in the best interest of the patients well-being. Non-utility approaches to measuring the impact of outcomes on individuals lives have been used, including the Oral Health Impact Profile (Slade and Spencer, 1994). These approaches facilitate comparison between interventions with outcomes that are not directly comparable (e.g., periodontal and orthodontic treatments) by measuring the impact of treatments on particular aspects of individuals lives (chewing function, pain experience, social acceptability, among others). However, these measures are not expressed in terms of the how the dimensions affect the individuals own assessment of his/her well-being. Utility measures go beyond these approaches by expressing the impact of an intervention on an individuals life in terms of the full consequences of the intervention from the patients perspective (Torrance and Feeny, 1989). In this paper, we review the various methods used for measuring utilities to consider the extent to which they represent valid measures of the impact of health care interventions on patient well-being.

Methods for Utility Measurement


Received June 27, 2001; Last revision April 18, 2002; Accepted May 15, 2002

Comprehensive descriptions of the methods for utility measurement have been published in the literature (Drummond et al., 1997; Matthews et al., 1999a). In addition, the statistical properties of these measurements have been the subject

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of considerable research (Gold et al., 1996). It is not the intention of this review to repeat these. However, to discuss the validity of the approaches as measures of patient well-being, it is necessary to provide a brief description of the most widely used methods to measure utilities of health outcomes:

decay among members of the public and dentists. Other examples of the SG used to measure dental utilities include Downer et al. (1997) and Birch et al. (1998).

Willingness to pay (WTP)


WTP measures an individuals strength of preference for an intervention by the maximum amount of money an individual would sacrifice for that intervention. Matthews et al. (1999b) considered patients preferences for surgical and non-surgical treatments for periodontal treatment. Individuals were asked their willingness to pay for the different treatments.

Visual analog scales (VAS)


Investigators measure, on a rating scale, the desirability of a health state associated with an intervention by asking subjects to locate the intervention on a scale with two fixed endpoints or anchors that represent two fixed health states which are given preference weights of 1 and 0, respectively. The health state under valuation is given a value according to its relative position on this 1-0 measurement scale. Edwards et al. (1999) presented patients with descriptions of a range of possible outcomes associated with alternative approaches to managing asymptomatic third molars. Each patient was asked to score each outcome on a scale with the anchors things could not be worse and I would not be bothered at all. The scores were then used as a measure of the utility of each outcome in a decision tree to compare alternative management strategies. Other uses of rating scales include Armstrong et al. (1995) and Brickley et al. (1995).

Validity of Utility Measures


Although each of the methods described above uses individuals preferences to determine the utility scores, the way these scores are developed and used differs between and among methods. The conceptual validity of a particular utility score as a measure of utility therefore depends on the assumptions underlying the methods used to derive the utility score (Gafni et al., 1993). There are several important characteristics to consider in determining the validity of utility measurement methods:

Utility and sacrifice


An individuals strength of preference for something is determined by what he is willing to trade off (or sacrifice) to have it. Utilities are measured by the maximum sacrifice that a subject is prepared to make. Measurement methods which do not involve sacrifice are inconsistent with the utility concept and hence are not valid methods for utility measurement. The visual analogue scale does not involve the notion of sacrifice in the measurement procedure. Positioning a health state at a higher point on the scale involves no trade-off by the subject. Researchers might use the VAS as a method for scoring outcome states, but those scores cannot be interpreted as measuring individuals utilities of those states. Instead they might be used as a form of individual weighting in the construction of a health status index, but the index would not be a measure of patient utility. In contrast, the other methods involve measurement of the maximum sacrifice the individual is willing to accept in return for the health state in the form of reductions in life expectancy (TTO), probability of immediate full health or survival (SG), and personal wealth (WTP), and hence satisfy this requirement for utility measurement.

Time trade-off (TTO)


The TTO measures an individuals trade-off between health improvements and life expectancy. Individuals express their strength of preference for an intervention in terms of the time they are prepared to sacrifice at the end of their expected lifetime in return for the improvement in health state associated with the intervention. The greater the amount of future life expectancy an individual is willing to forego, the stronger is his/her preference for the health state. Variants of the TTO have been developed. Fyffe et al. (1999) asked subjects the amount of free time they would be prepared to forego each day, in the form of extra time devoted to toothbrushing, in return for a given improvement in dental health. The amount of free time that the individual would forego was used to measure the individuals utility associated with that improvement in dental health. Different interventions were compared based on their Dental Free Time Trade-off (DFTO). Other variants of the TTO are provided by Cohen et al. (1990) and Tulloch et al. (1990).

Standard gamble (SG)


The standard gamble measures an individuals trade-off between health improvements and the probability of survival. Individuals express their strength of preference for an intervention by the probability of survival they would sacrifice (and by implication the risk of death they would accept) for the health state associated with the intervention under evaluation. The subject chooses between this state and the uncertain prospect of immediate full health with a probability of p and immediate death with a probability of 1-p. Probability p is varied until the subject is indifferent between the health state and the uncertain prospect. With the utility of full health and death set equal to 1 and 0, respectively, the utility of the health state is given by the indifference level of p based on the expected value function (Torrance and Feeny, 1989). In dentistry, interventions rarely involve the possibility of death; hence, to measure utilities on a scale with the lower bound of death would lead to a bunching of interventions toward the upper end of the scale. To deal with this, dental applications of the standard gamble often use upper and lower bounds more appropriate for comparison with interventions under consideration. Fyffe and Kay (1992) used SG to measure the utilities of a posterior tooth in different stages of

Utility and consequences


The determinants of well-being are not limited to health outcomes. What the patient endures to arrive at the outcome also affects his/her well-being (Mooney and Lange, 1993; Birch et al., 1998). For example, the patient is unlikely to be indifferent to the number of visits required for a treatment, the time spent in the dentists chair, or the discomfort of a procedure. If the reduction in utility associated with these factors outweighs the improvements in utility associated with the health outcome, then from the patients perspective, the treatment represents a net reduction in well-being. Utilities based only on health outcomes, as opposed to the full consequences of a treatment, can misrepresent the patients best interests and lead to patients failing to comply with evidence-based protocols based entirely on preferences for health outcomes. These broader considerations can be incorporated into measurement methods through careful description of the package of patient consequences associated with the outcome

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(Birch et al., 1998). However, some methods of measurement have been found to be less sensitive to detecting the effects of various consequences on patient well-being (Smith, 2001). For example, the units of measurement in the TTO and the SG are remaining life expectancy and probability of survival, respectively. Subjects may be unwilling to trade off a measurable amount of life expectancy or probability of full health for a small reduction in time spent in the dentists chair. But that does not mean that the reduction in chair time does not improve wellbeing. Subjects may be willing to sacrifice other things in exchange for this reduction in chair time, but the TTO and SG do not provide a mechanism by which they can express this preference (Birch et al., 1999). Fyffe et al. (1999) use daily free time in place of life expectancy to improve the sensitivity of the time trade-off measurement. However, the trade-off remains restricted to a single commodity to be sacrificed as an indicator of value. But the importance of any commodity in contributing to individual well-being may differ between individuals. For example, some individuals might have a stronger preference for free time than others. This problem applies to all methods where the sacrifice is measured in terms of a particular commodity. In contrast, the WTP method has individuals express their trade-off over a wide range of commodities expressed through the familiar monetary unit of exchange. Money per se is not a source of utility but provides individuals with command over a wide range of commodities. In this way, the WTP approach provides a method of measurement that is much more sensitive to small changes in wellbeing, as might be produced by differences in the different aspects of the process of care.

Utility and uncertainty


The outcomes of health care interventions are uncertain, and decisions about which intervention to use are taken under conditions of uncertainty. Utility measurement therefore requires that the methods reflect the uncertainty associated with decisionmaking (Gafni and Birch, 1995). There is no reason to believe that an individuals ranking of different outcomes or strengths of preference among outcomes will be the same under uncertainty as it is under conditions of certainty. Nor can we assume that the uncertainty associated with outcomes will affect all individuals preferences the same. This will depend upon each individuals attitude toward uncertainty. The time trade-off (TTO) method and its variants, such as the Dental Free Time Trade-off (DFTO), measure an individuals trade-off between health and remaining life expectancy under conditions of certaintythe amount of time to be sacrificed in return for a certain change in health status. Hence, the scores cannot be interpreted as measures of utility where uncertainty is present, as is the case in most health care choices. Uncertainty is incorporated into the Standard Gamble (SG) procedure through the unit of measurement, the probability of immediate full health. Different values of the probability of immediate full health, p, are considered until the subject is indifferent between the health state under consideration and the uncertain prospect. The subjects attitude to uncertainty is captured in the measurement procedure. The score produced by the SG reflects the impact of both the outcome being considered together with its uncertain nature on the subjects well-being. Two individuals might have the same strength of preference for a given health state, but if they have different preferences for uncertainty, the utilities for this health state will differ between them.

The SG method is derived from the theory of expected utility (Torrance and Feeny, 1989), which is based on several axioms about individual behavior. Individual behavior has been shown to violate some of these axioms on occasions (Loomes, 1995). Although more advanced theories of utility have been presented (see Karni and Schmeidler, 1991), to date these have not generated practical approaches for utility measurement (Gafni et al., 1993). In the absence of measurement methods consistent with these more advanced theories, the SG method remains a gold standard for measuring utilities based on the normative appeal of the underlying expected utility theory (Gafni and Birch, 1995). Uncertainty is not confined to outcomes but also affects the need for interventions. An individuals preference for supporting a particular health care intervention will be influenced by the probability that the individual will need the intervention sometime in the future (Birch et al., 1999). Hence, decisions about investing public funds in different technologies, or changing the services covered by insurance plans, require information about individual preferences that incorporate uncertainty in both the need for, and outcomes produced by, the interventions under consideration. This broader role of uncertainty can be accommodated in the SG method. For example, the description of an intervention can include information on the probability of the condition. The SG procedure then measures the maximum probability of immediate full health that the subject is willing to sacrifice for the intervention, given the level of risk the subject faces of getting the condition. This improves the conceptual validity of the procedure but increases the cognitive burden on the subject. Moreover, the SG procedure may be insensitive to underlying changes in risks of conditions because of the measurement unit used, the risk of immediate death (Birch et al., 1999). Uncertainty is incorporated directly into WTP measures through probabilistic descriptions of needs (e.g., WTP for interventions for conditions with a 10% of occurring) and outcomes (e.g., WTP for an intervention with a 50% chance of reducing pain) based on the best available epidemiological or clinical information (Neumann and Johannesson, 1994; Matthews et al., 1999b). In responding to choices involving differences in levels of uncertainty, the subject can consider the sacrifice to be made across a broad range of commodities through the monetary consequences involved (Birch et al., 1999).

Interpreting the Numbers to Measure Utility


The validity of utility measurement extends to how the utility scores are used (Gafni and Birch, 1995). In many applications, information on the strengths of preference provided by subjects is transformed by the researcher to produce a user-friendly construct for the decisionmaker. Quality-adjusted life-years (QALYs) (Torrance, 1986), quality-adjusted tooth-years (QATYs) (Birch, 1986; AntczakBouckoms and Weinstein, 1987), and healthy-years equivalents (HYEs) (Mehrez and Gafni, 1989) are examples of constructs aimed at providing a meaningful measure of strength of preference. QALYs adjust length of life for quality of life by multiplying each expected life-year by a preference score for different health states based on the TTO or SG scores. This assumes separability between health state and duration (Torrance and Feeny, 1989), i.e., health states have value per se independent of duration, and the effect of a particular health state on well-being is proportionate to its duration. Such an assumption lacks conceptual support and has been found, in empirical studies (Hall et al., 1992; Kuppermann et al., 1997), to be invalid. Following Bala and Zarkin (2000), suppose an individual has expressed a utility for oral surgery without anesthesia of 0.1. To calculate the QALYgain, the difference in utility between the intervention without

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anesthesia and with anesthesia is multiplied by the number of years for which the difference in health state is experienced. If the surgery lasts an hour, then the local anesthetic that restores the patient to full health would result in a QALY-gain of 0.0001 ([0.9 x 1]/[365 x 24]). Suppose society is willing to pay up to $40,000 per QALY. The maximum society would be willing to pay for anesthesia would therefore be $4. As the authors note, most people would readily place the value of such an anesthetic to be much more than that. In other words, the assumptions used to calculate the QALY do not reflect individuals preferences. The assumption is not based on information provided by the subjects but is imposed by the researcher to construct the QALY or QATY measure (Gafni et al., 1993). Under the Healthy Years Equivalent (HYE) approach, subjects own preferences are used to make the transformation (Gafni et al., 1993). A second SG procedure is used to transform the uncertain prospect of immediate full health or immediate death into equivalent time of full health for a particular life expectancy. In this way, no restriction is imposed on the way health state and duration affect the subjects well-being. Use of the SG to measure HYEs means that the assumptions of expected utility theory are still required, and that measurement remains constrained to units of risk of survival. Nevertheless, the HYE requires fewer assumptions concerning the formulation of individuals preferences than do QALY or QATY measures (Bleichrodt, 1995). The WTP approach requires no transformation, since it is based on what is already a meaningful concept to decision-makers and subjects alike. The information used is derived directly from subjects own valuations and does not depend on underlying assumptions about the form of relationship between the determinants of well-being and the level of utility.

incomes may provide low TTO, SG, or WTP scores for an intervention, even though they perceive that the intervention would have an important impact on their well-being. Adjustment algorithms have been developed to deal with the effects of differences in circumstances on utility scores (Gafni and Birch, 1991; Johannesson and Meltzer, 1998). However, there is no reason why the utility scores derived after adjustment should be consistent with the utility scores of decision-makers. The purpose of the utility measurement exercise is to inform the decision-making process about the preferences of subjects, whatever they may be. To ignore individuals preferences that differ from the preferences of the decision-makers undermines the purpose of the utility measurement exercise. Moreover, it risks affecting adversely the uptake or compliance with the intervention under consideration.

DISCUSSION
Clinical research on oral health outcomes provides important evidence for decision-making (Bader et al., 1999). The incorporation of dental research findings into the care of individual patients is central to the maximization of benefits and reducing the harm of treatments (Clarkson et al., 1999). However, if treatment decisions are to be based on the best interests of the patients, additional information is required about the patients utilities associated with the different possible choices (Slade et al., 1998). Utility measurement assesses an individuals strength of preference for an intervention that reflects the expected effect on the subjects assessment of his/her well-being. Utility is essentially a theoretical concept, and the validity of measurement methods is established in relation to the underlying theory. Concerns about validity are not restricted to measurement methods but also include how these data are transformed to generate information on preferences for decision-makers. There may be no simple answer to treatment decisions patients with the same clinical conditions may prefer different treatments. Failure to base decisions on the patients preferences among different treatments may not serve the best interests of the patient but may have more to do with the interests of providers or funding agencies. In some cases, such as treatments for infective diseases, decision-makers may believe it appropriate to over-ride patients best interests to deal with the broader societal consequences of decisions. However, achievement of these broader social goals will depend upon the acceptance of, or compliance with, the socially beneficial decision by individual patients. Hence, decision-makers must consider patient preferences to identify strategies for the delivery of services in ways that lead to the socially preferred treatment also being in the best interests of individual patients and hence promote patient compliance (Birch and Abelson, 1993). Knowledge of patient utilities is an important input for evidence-based decision-making. Such knowledge provides the evidence about the expected consequences of service utilization on patient well-being. Without such information, we do not know whether the way we choose to use dental care resources is doing more good than harm for patients. As dental research continues to uncover new ways of treating oral conditions, it is important that consideration be given to the effects of different treatments on the well-being of the populations who bear the consequences of the proposed treatments. There is considerable experience with using utility measurement methods successfully in both patient and general public settings. However, in accepting the challenge to include

Factors Affecting Individuals Utility Scores


Individuals responses to utility measurement instruments will depend on the accuracy, completeness, and clarity of the information provided. The utilities derived from responses, and the preferences they infer, even when based on situations of full information, may differ from what providers or decision-makers would like. This might be explained by differences between subjects and decision-makers in the circumstances under which interventions are considered. The unit of measurement is an important example of the role of circumstances in the determination of utility scores. Because TTO, SG, and WTP express utility in terms of other commodities, an individuals responses may be influenced by his or her current circumstances. For example, individuals with larger incomes may have higher WTP for an intervention than individuals with lower incomes, simply because they have more money available (Weinstein and Manning, 1997). Similarly, life expectancy and probability of full health are not distributed equally across the population (Hertzman et al., 1994). An individuals capacity to sacrifice the probability of full health may influence his/her willingness to sacrifice life expectancy or probability of health as measured in TTO and SG (Donaldson et al., 2002). Where attention is focused on an individuals preferences among interventions, the capacity to trade is fixed for that individual across all interventions. As a result, the utility scores for this individual represent the impact of the interventions on the individuals wellbeing, given current circumstances. Problems arise where utility scores are used to measure the effect of an intervention on total wellbeing in a patient population, or to prioritize access to the intervention on the basis of the expected impact on patient well-being. For example, individuals with very short life expectancies or very low

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utility measurement as a standard component of dental health services research, we must be careful to ensure that we measure the right thing in the right way and for the right group.

ACKNOWLEDGMENT
This review is sponsored by USPHS/NIH/NIDCR grant number 13202-03.

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