Measuring the Wellbeing of Cancer Patients with Generic and Disease-Specific Instruments.

Different wellbeing measures have been used among cancer patients. This study aimed to first investigate the sensitivity of health state utility (HSU), capability, and subjective wellbeing (SWB) instruments in cancer. A cancer-specific instrument (QLQ-C30) was included and transferred onto the cancer-specific HSU scores. Furthermore, it examined the relative importance of key life domains explaining overall life satisfaction. Data were drawn from the Multi-instrument Comparison survey. Linear regression was used to explore the extent to which the QLQ-C30 sub-scales explain HSU and SWB. Kernel-based Regularized Least Squares (KRLS), a machine learning method, was used to explore the life domain importance of cancer patients. As expected, the QLQ-C30 sub-scales explained the vast majority of the variance in its derived cancer-specific HSU (R2 = 0.96), followed by generic HSU instruments (R2 of 0.65-0.73) and SWB and capability instruments (R2 of 0.33-0.48). The cancer-specific measure was more closely correlated with generic HSU than SWB measures, owing to the construction of these instruments. In addition to health, life achievements, relationships, the standard of living, and future security all play an important role in explaining the overall life satisfaction of cancer patients.

Valuation of Treatments for Rare Diseases: A Systematic Literature Review of Societal Preference Studies.

INTRODUCTION: We sought to synthesize published empirical studies that elicited and characterized societal valuations of orphan drugs and the attributes that may drive different valuations for orphan drugs versus other treatments. METHODS: We conducted a systematic literature review (SLR) in MEDLINE and EMBASE databases up to November 2, 2020. Search terms covered societal preferences and attributes of orphan drugs (e.g., disease prevalence, severity, burden, unmet needs, and benefits). RESULTS: We identified 38 eligible publications: 33 societal preference studies and 5 reviews discussing societal valuations and attributes of orphan drugs. Most publications suggested that a majority of respondents favored allocating funds to more prevalent diseases. However, trade-off studies and discrete-choice experiments found that survey participants chose to allocate resources to orphan drugs even when the cost per unit of health benefit was greater than for therapies for more prevalent diseases. Overall, 19 of 27 studies assessing severity in treatment valuation revealed that respondents prioritized patients with severe diseases over those with milder ones for equal health benefits. Members of the general public tended to prefer treatments for diseases with no alternative or when existing alternatives had limited efficacy over diseases with clear therapeutic alternatives. There was evidence that individuals preferred sharing resources, so no patient was left without treatment. CONCLUSIONS: Our SLR indicates the general public typically attaches greater value to orphan drugs than to other treatments for common diseases. This is not because of rarity per se, but primarily because of disease severity and lack of therapeutic alternatives typically associated with rare diseases.

Orphan drugs are drugs serving a substantial public health need by treating life-threatening or chronically debilitating medical conditions affecting a small number of people with very high unmet needs. We reviewed 38 published studies looking at drug characteristics that may cause people to value orphan drugs differently versus treatments for common conditions. Most people surveyed in these publications favored health care funds going to more prevalent diseases. However, some people preferred funding orphan drugs even when the cost versus health benefit was higher compared with treatments for more common diseases. The majority of studies that investigated the impact of disease severity on the valuation of treatments found that people prioritized patients with severe disease over those with milder disease, for the same extent of health benefit. People also preferred funding treatments for diseases that have no alternative treatments, or treatments with limited benefits, over treatments for diseases with many treatments or more effective treatments. We also found evidence of a societal preference for shared resources, meaning that no patient would be left without treatment, including those who receive limited benefits from health care resources, even if this does not lead to the maximization of health benefits across society. In conclusion, our literature review indicated that the general public attaches greater value to orphan drugs versus treatments for more common diseases, not because of rarity per se, but largely because the rare diseases treated by orphan drugs are often severe and have no or few treatment options.

Correction to: Are Quality-Adjusted Life Years a Good Proxy Measure of Individual Capabilities?

Open Access This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0International License.

Health technology assessment (HTA) and economic evaluation: efficiency or fairness first.

The economic evaluation which supports Health Technology Assessment (HTA) should inform policy makers of the value to society conferred by a given allocation of resources. However, neither the theory nor practise of economic evaluation satisfactorily reflect social values. Both are primarily concerned with efficiency, commonly conceptualised as the maximisation of utility or quality adjusted life years (QALYs). The focus is upon the service and the benefits obtained from it. This has resulted in an evaluation methodology which discriminates against groups and treatments which the population would like to prioritise. This includes high cost treatments for patients with rare diseases. In contrast with prevailing methods, there is increasing evidence that the public would prefer a fairness-focused framework in which the service was removed from centre stage and replaced by the patient. However methods for achieving fairness are ad hoc and under-developed. The article initially reviews the theory of economic evaluation and argues that its focus upon individual utility and efficiency as defined by the theory omits potentially important social values. Some empirical evidence relating to population values is presented and four studies by the first author are reviewed. These indicate that when people adopt the social perspective of a citizen they have a preference for sharing the health budget in a way which does not exclude patients who require services that are not cost effective, such as orphan medicinal products (OMP's) and treatments for patients with ultra-rare diseases (URD's).

Social preferences for prioritising the treatment of disabled and chronically ill patients: beyond the order effect.

Previous evidence suggests that members of the public value life saving services differently when they are for patients with a pre-existing permanent disability and when they are for patients who become disabled at the onset of treatment - for example, as a result of treatment that is not entirely effective. However, the valuation of services in these two cases has also been found to differ with the order in which they are presented in a population survey. This casts doubt upon the validity of the results and leaves unresolved the nature of the public's true preferences. The study reported here had three main objectives: (i) to determine the considered, underlying preferences of a sample of the Australian public with respect to the treatment of the permanently disabled and chronically ill, (ii) to gain insight into the reasons for respondent's distributive preferences and (iii) to eliminate or significantly reduce the order effect. Eight semi-structured, small-group discussions were held with 66 members of the public in Victoria, Australia. Order effects were effectively eliminated. The study found substantial support among participants for the equal treatment of the permanently disabled and chronically ill regardless of when the problem commenced.

Variation in the apparent importance of health-related problems with the instrument used to measure patient welfare.

AIMS: This paper investigates the distributional implications for eight population groups of using six different instruments to measure wellbeing and to prioritise access to health services. Specifically, it examines the importance of different physical and psycho-social problems for the scores obtained using each instrument and whether scores differ because of differences in the concept measured by the instrument or because of the instrument's construction. METHODS: Patients with seven chronic conditions and a sample of the 'healthy' public were administered six instruments: two utility instruments; two self-rating scales; a subjective wellbeing instrument and the ICECAP measure of capability. Scores were regressed upon the subscales of the SF-36 and the AQoL-8D. Each instrument's 'problem mix' was measured by the numerical importance of the subscales for the instrument's score and compared with the problem mix of patients constructed from all of the instruments. RESULTS: The apparent importance of different problems varied significantly with the instrument used to assess welfare but not with the chronic conditions. The correspondence between an instrument's problem mix and the patients' problem mix was highly variable. CONCLUSION: Different instruments give prominence to different physical and psycho-social problems and consequently favour different groups of patients. Budgetary decisions which appear to be based on efficiency criteria such as the cost per quality-adjusted life year (QALY) conceal distributive effects attributable to the instrument used in the analysis. The effects are additional to the ethical questions considered in making an equity-efficiency trade-off as they arise from the measurement of efficiency.

Sharing and the Provision of "Cost-Ineffective" Life-Extending Services to Less Severely Ill Patients.

BACKGROUND: Cost-utility analysis prioritizes services using cost, life-years, and the health state utility of the life-years. Nevertheless, a significant body of evidence suggests that the public would prefer more variables to be considered in decision making and at least some sharing of the budget with services for severe conditions that are not cost-effective because of their high cost. OBJECTIVES: To examine whether this preference for sharing persists for less severe conditions when both cost effectiveness and illness severity would indicate that resources should be allocated to other services. METHODS: Survey respondents were asked to divide a budget between two patients facing life-threatening illnesses. The severity of the illnesses differed and the price of treatment was varied. RESULTS: Sharing occurred in all scenarios including scenarios in which the illness was less severe and services were not cost-effective. Results are consistent with behavior commonly observed in other contexts. CONCLUSIONS: Results suggest that sharing per se is important and that the public would support some funding of cost-ineffective services for less severe health problems.

Uncertainty and the Undervaluation of Services for Severe Health States in Cost-Utility Analyses.

OBJECTIVES: To test the hypothesis that the "severity effect"-the preference for more than utility-maximizing expenditure on severe health states-may be the result of, or exacerbated by, the uncertainty associated with the chance of contracting the illness that causes the severe health state. METHODS: Survey respondents were asked to imagine that they will contract one of two illnesses and asked to allocate a budget between two insurance policies, each of which provides services for the treatment of one of the illnesses. A person's final health state varied with the amount of insurance purchased for the illness that occurred and therefore with the level of treatment. The relative cost of the two policies was altered and the selected levels of insurance compared with the levels that would be provided by a health authority that sought to maximize total utility or quality-adjusted life-years from its own budget. RESULTS: Respondents selected more than utility-maximizing insurance for protection against severe health states. A number of psychological factors that affect measurement under uncertainty do not affect utility as currently measured. This difference may explain the present results and also explain the "severity paradox" that personal preferences as presently measured imply less expenditure on severe health states than do "social preferences" for the treatment of strangers. CONCLUSIONS: Uncertainty alters preferences. Incorporating these preferences in decision making would result in greater spending on severe health states.

Does a patient's health potential affect the social valuation of health services?

BACKGROUND: Patients with a permanent impairment may be unable to reach full health. Consequently health services which cure illnesses which are unrelated to the impairment may increase health less than services for patients with no impairment. While it has been argued that this should not lead to discrimination against impaired patients there is little evidence to determine whether this equity-efficiency trade-off is consistent with social values. OBJECTIVES: To measure the effect of permanent impairment upon the social valuation of services for unrelated illnesses. METHODS: Social valuations of services for illnesses associated with mobility, depression or pain were assessed and compared for patients with and without a permanent impairment using the Relative Social Willingness to Pay (RS-WTP) instrument. The maximum valuation of services for impaired patients was also compared with the maximum utility which could be gained when utility was measured using three multi attribute utility instruments. RESULTS: Curing the illness of impaired patients was valued 8-11 percent less than the cure of patients with no impairment. Discrimination decreased as the severity of the illness increased. Valuation of health states using the utility instruments implied significantly greater discrimination than the social valuations using the RS-WTP instrument. CONCLUSIONS: Health services are valued less highly when a patient's health potential is impaired. However discrimination is significantly less than would occur if the value of the services were limited to the value of the health state causing the impairment. The argument for disregarding a patient's limited health potential when resources are allocated therefore receives some support from social valuations but the case for completely equal treatment depends upon additional ethical arguments.

Does the use of the proportional shortfall help align the prioritisation of health services with public preferences?

It has been proposed that equity may be included in the economic evaluation of health services using the 'proportional shortfall' (PS)-the proportion of a person's QALY expectation that they would lose because of an illness. The present paper reports the results of a population survey designed to test whether PS helped to explain people's preferences for health services and whether it did this better than the absolute shortfall or the equity related variables that PS seeks to replace. Survey respondents were asked to allocate 100 votes between 13 scenarios and a standard scenario. Variation in the allocation of votes was explained by health gain and different combinations of the equity variables. Differences in votes for the comparisons were significantly related to differences in PS but the relationship was weaker than between votes and the age related variables. Cases were identified where PS suggested a priority ordering of services which was strongly rejected by respondents. It is concluded that the use of PS is unlikely to improve the alignment of priorities with public preferences.

Social preferences for prioritizing the treatment of severely ill patients: The relevance of severity, expected benefit, past health and lifetime health.

The study examined the preferences of a sample of the Australian public and health professionals regarding the relative importance of four different criteria for prioritizing between patients: the severity of the condition, the size of the benefit from the intervention, past health losses and expected lifetime health. A discussion-group methodology was adopted to elicit social preferences. This allowed participants time to consider all of the alternatives fully, to seek clarification of the task, and to engage in open debate about the issues raised. Participants traded-off cost-effectiveness for priority to the more severely ill. They placed less importance on past health and the lifetime allocation of health in deciding priority for treatment, and more importance on improving the condition of those who will be left more severely ill or disabled in the absence of treatment. The results pose a challenge to studies reporting support for the "fair innings argument". They also support the Norwegian government's decision not to pursue a life-time health loss criterion as recommended by the Norheim Commission. The study question is important given current debate both in the health economics literature and at the policy level in several jurisdictions.

Are Quality-Adjusted Life Years a Good Proxy Measure of Individual Capabilities?

BACKGROUND: There is a debate in the health economics literature concerning the most appropriate way of applying Amartya Sen's capability approach in economic evaluation studies. Some suggest that quality-adjusted life years (QALYs) alone are adequate while others argue that this approach is too narrow and that direct measures of capability wellbeing provide a more extensive application of Sen's paradigm. OBJECTIVE: This paper empirically explores whether QALYs provide a good proxy for individual capabilities. METHODS: Data is taken from a multinational cross-sectional survey of individuals with seven health conditions (asthma, arthritis, cancer, depression, diabetes, hearing loss, heart disease) and a healthy population. Each individual completed the ICECAP-A measure of capability wellbeing for adults and six health utility instruments that are used to generate QALYs, including EQ-5D and SF-6D. Primary analysis examines how well health utility instruments can explain variation in the ICECAP-A using ordinary least squares regression. RESULTS: The findings show that all seven health conditions have a negative association on overall capability as measured by the ICECAP-A index. Inclusion of health utility instruments into separate regressions improves the predictive power of capability but on average, explains less than half of the variation in capability wellbeing. Individuals with arthritis appear to be less inhibited in terms of capability losses when accounting for health utility, yet those who have depression record significant reductions in capability relative to the healthy population even after accounting for the most commonly used health utility instruments. CONCLUSION: The study therefore casts doubt on the ability of QALYs to act as a reliable proxy measure of individuals' capability.

Assessing the validity of the ICECAP-A capability measure for adults with depression.

BACKGROUND: Effectiveness and cost-effectiveness are increasingly important considerations in determining which mental health services are funded. Questions have been raised concerning the validity of generic health status instruments used in economic evaluation for assessing mental health problems such as depression; measuring capability wellbeing offers a possible alternative. The aim of this study is to assess the validity of the ICECAP-A capability instrument for individuals with depression. METHODS: Hypotheses were developed using concept mapping. Validity tests and multivariable regression analysis were applied to data from a cross-sectional dataset to assess the performance of ICECAP-A in individuals who reported having a primary condition of depression. The ICECAP-A was collected alongside instruments used to measure: 1. depression using the depression scale of the Depression, Anxiety and Stress Scale (DASS-D of DASS-21); 2. mental health using the Kessler Psychological Distress Scale (K10); 3. generic health status using a common measure collected for use in economic evaluations, the five level version of EQ-5D (EQ-5D-5L). RESULTS: Hypothesised associations between the ICECAP-A (items and index scores) and depression constructs were fully supported in statistical tests. In the multivariable analysis, instruments designed specifically to measure depression and mental health explained a greater proportion of the variation in ICECAP-A than the EQ-5D-5L. CONCLUSION: The ICECAP-A instrument appears to be suitable for assessing outcome in adults with depression for resource allocation purposes. Further research is required on its responsiveness and use in economic evaluation. Using a capability perspective when assessing cost-effectiveness could potentially re-orientate resource provision across physical and mental health care services.

Communal Sharing and the Provision of Low-Volume High-Cost Health Services: Results of a Survey.

INTRODUCTION: This paper suggests and tests a reason why the public might support the funding of services for rare diseases (SRDs) when the services are effective but not cost effective, i.e. when more health could be produced by allocating funds to other services. It is postulated that the fairness of funding a service is influenced by a comparison of the average patient benefit with the average cost to those who share the cost. METHODS: Survey respondents were asked to allocate a budget between cost-effective services that had a small effect upon a large number of relatively well patients and SRDs that benefited a small number of severely ill patients but were not cost effective because of their high cost. RESULTS: Part of the budget was always allocated to the SRDs. The budget share rose with the number sharing the cost. DISCUSSION: Sharing per se appears to characterise preferences. This has been obscured in studies that focus upon cost per patient rather than cost per person sharing the cost.

Mapping Between the Sydney Asthma Quality of Life Questionnaire (AQLQ-S) and Five Multi-Attribute Utility Instruments (MAUIs).

PURPOSE: Economic evaluation of health services commonly requires information regarding health-state utilities. Sometimes this information is not available but non-utility measures of quality of life may have been collected from which the required utilities can be estimated. This paper examines the possibility of mapping a non-utility-based outcome, the Sydney Asthma Quality of Life Questionnaire (AQLQ-S), onto five multi-attribute utility instruments: Assessment of Quality of Life 8 Dimensions (AQoL-8D), EuroQoL 5 Dimensions 5-Level (EQ-5D-5L), Health Utilities Index Mark 3 (HUI3), 15 Dimensions (15D), and the Short-Form 6 Dimensions (SF-6D). METHODS: Data for 856 individuals with asthma were obtained from a large Multi-Instrument Comparison (MIC) survey. Four statistical techniques were employed to estimate utilities from the AQLQ-S. The predictive accuracy of 180 regression models was assessed using six criteria: mean absolute error (MAE), root mean squared error (RMSE), correlation, distribution of predicted utilities, distribution of residuals, and proportion of predictions with absolute errors <0.0.5. Validation of initial 'primary' models was carried out on a random sample of the MIC data. RESULTS: Best results were obtained with non-linear models that included a quadratic term for the AQLQ-S score along with demographic variables. The four statistical techniques predicted models that performed differently when assessed by the six criteria; however, the best results, for both the estimation and validation samples, were obtained using a generalised linear model (GLM estimator). CONCLUSIONS: It is possible to predict valid utilities from the AQLQ-S using regression methods. We recommend GLM models for this exercise.

Age Weights for Health Services Derived from the Relative Social Willingness-to-Pay Instrument.

The effect of a patient's age on the social valuation of health services remains controversial, with empirical results varying in magnitude and implying a different age-value profile. This article employs a new methodology to re-examine these questions. Data were obtained from 2 independent Web-based surveys that administered the Relative Social Willingness to Pay instrument. In the first survey, the age of the patient receiving a life-saving service was varied. Patients were left with either poor mental or physical health. In the second survey, patient age was varied for a service that fully cured the patient's poor mental or physical health. In total, therefore, 4 sets of age weights were obtained: weights for life-extending services with poor physical or mental health outcomes and weights for quality-of-life improvement for patients in poor mental or physical health. Results were consistent. Increasing age was associated in each case with a monotonic decrease in the social valuation of the services. The decrease in value was quantitatively small until age 60 years. By age 80 years, the social value of services had declined by about 50%. The decline commenced at an earlier age in the context of physical health, although the magnitude of the decrement by age 80 years was unrelated to the type of service. With 1 exception, there was little difference in the valuation of services by the age of the survey respondent. Respondents aged >60 years placed a lower, not higher, value on quality-of-life improvement for elderly individuals than other respondents. There was no difference in the valuation of life-extending services.

How important is severity for the evaluation of health services: new evidence using the relative social willingness to pay instrument.

The 'severity hypothesis' is that a health service which increases a patient's utility by a fixed amount will be valued more highly when the initial health state is more severe. Supporting studies have employed a limited range of analytical techniques and the objective of the present paper is to test the hypothesis using a new methodology, the Relative Social Willingness to Pay. Three subsidiary hypotheses are: (1) that the importance of the 'severity effect' varies with the type of medical problem; (2) that the relationship between value and utility varies with the severity of the initial health state; and (3) that there is a threshold beyond which severity effects are insignificant. For each of seven different health problems respondents to a web-based survey were asked to allocate a budget to five services which would, cumulatively, move a person from near death to full health. The time trade-off utilities of health states before and after the service were estimated. The social valuation of the service measured by the budget allocation was regressed upon the corresponding increase in utility and severity as measured by the pre-service health state utility. Results confirm the severity hypothesis and support the subsidiary hypotheses. However, the effects identified are quantitatively significant only for the most severe health states. This implies a relatively limited redistribution of resources from those with less severe to those with more severe health problems.

Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement.

BACKGROUND: In most jurisdictions, policies have been adopted to encourage the development of treatments for rare or orphan diseases. While successful as assessed against their primary objective, these policies have prompted concerns among payers about the economic burden that might be caused by an annual cost per patient in some cases exceeding 100,000 Euro. At the same time, many drugs for rare disorders do not meet conventional standards for cost-effectiveness or 'value for money'. Owing to the fixed (volume-independent) cost of research and development, this issue is becoming increasingly serious with decreasing prevalence of a given disorder. METHODS: In order to critically appraise the problems posed by the systematic valuation of interventions for ultra-rare disorders (URDs), an international group of clinical and health economic experts was convened in conjunction with the Annual European ISPOR Congress in Berlin, Germany, in November 2012. Following this meeting and during subsequent deliberations, the group achieved a consensus on the specific challenges and potential ways forward. RESULTS: The group concluded that the complexities of research and development for new treatments for URDs may require conditional approval and reimbursement policies, such as managed entry schemes and coverage with evidence development agreements, but should not use as justification surrogate end point improvement only. As a prerequisite for value assessment, the demonstration of a minimum significant clinical benefit should be expected within a reasonable time frame. As to the health economic evaluation of interventions for URDs, the currently prevailing logic of cost-effectiveness (using benchmarks for the maximum allowable incremental cost per quality-adjusted life year gained) was considered deficient as it does not capture well-established social preferences regarding health care resource allocation. CONCLUSION: Modified approaches or alternative paradigms to establish the 'value for money' conferred by interventions for URDs should be developed with high priority.

Shared medical appointments for Aboriginal and Torres Strait Islander men.

BACKGROUND: Aboriginal and Torres Strait Islander health is generally the worst of any population group in Australia. Inaccessibility to health services is one possible cause of this. Shared medical appointments (SMAs) appear to be a culturally competent and appropriate way of improving access to, and the quality of, primary healthcare services for Aboriginal and Torres Strait Islander peoples. OBJECTIVE: The objective of this article is to assess the acceptability and appropriateness of SMAs as an adjunct process in primary care for Aboriginal and Torres Strait Islander men. METHODS: As part of a broader study on SMAs, three SMA sessions were delivered at an Aboriginal men's health centre in northern New South Wales. One-day training sessions in SMA facilitation were also provided to two groups of 12-14 Aboriginal health workers (AHWs). Mixed methods were used to assess patient and provider satisfaction, subjective outcomes, and operational procedures in the SMA groups, as well as interest in the SMA process by AHWs. RESULTS: Satisfaction with SMAs among Aboriginal men was unanimously positive, with the numbers in the group increasing over time. Patients most enjoyed the 'yarn up' nature of SMAs with peer support, which reduced the 'scary' and culturally 'unnatural' nature of one-on-one consultations with a general practitioner (GP). AHWs who were trained to a level to conduct SMAs saw this as an effective way of improving cultural competence in, and accessibility of, their various Aboriginal health services. DISCUSSION: The results, though not generalisable, suggest that SMAs may offer a culturally safe and appropriate tool to enhance Aboriginal and Torres Strait Islander peoples' access to primary care.