Monetary value of health-a practical decision-making framework combining equity considerations and WTP.

OBJECTIVE: We estimate the first monetary value of a health gain in Croatia to inform the debate about the appropriate "demand-side" cost-effectiveness thresholds in Croatia but also Central and Eastern Europe, where such debates are still uncommon. We test the empirical support for two equity considerations: age and severity operationalized as proportional shortfall (PS), and propose a pragmatic framework for combining equity considerations with the monetary value of health into a single threshold. METHODS: We used the contingent valuation method to elicit the willingness to pay per Quality-Adjusted Life Year (QALY) in Croatia, using a representative sample of the population (N = 1,500, online survey). 29 EQ-5D health states were valued using payment scales and open-ended question as payment vehicles. To test the hypotheses, we used both parametric tests and non-parametric tests. Multilinear regression was employed to test the theoretical validity of the results. RESULTS: The monetary value of a health gain in Croatia is equivalent to 1.15 of GDP per capita (equaling €17,000). Age of patients seems to be an important equity-related characteristic. The WTP per QALY in the age-neutral risk group (€11,900) was nearly equivalent to the WTP per QALY in the adult (neutral) risk group (€11,700) but lower by 16% compared to the WTP per QALY estimated in children (€14,200; p = 0.00). WTP estimates are theoretically valid and to, a small degree, scale sensitive. There is a positive association between the level of proportional shortfall and willingness to pay. To increase the usefulness of our results for the policy-makers, we combine the elicited preferences into a single decision-making framework and construct several cost-effectiveness thresholds based on willingness to pay and equity-related preferences. Based on empirical results, cost-effectiveness thresholds could range up to €20,308 for the most severe health conditions in children or could be lowered to €16,777 for less severe health conditions. DISCUSSION: In Central and Eastern Europe, in spite of a growing understanding of the importance of further developing value-based assessment frameworks there has been very little empirical research to guide, inform and promote this development. Countries in this region use mainly GDP-based thresholds without empirical evidence to support such important decisions. This may lead to thresholds that are too high, with detrimental consequence for the pricing and reimbursement systems.

VOLY: The Monetary Value of a Life-Year at the End of Patients' Lives.

OBJECTIVE: We explored the monetary value of the end-of-life (EoL) health gains, that is, the value of a life-year (VOLY) gained at the end of a patient's life in Croatia. We tested whether the nature of the illness under valuation (cancer and/or rare disease) is a factor in the valuation of EoL-VOLYs. The aim was for our results to contribute to the health and longevity valuation literature and more particularly to the debate on the appropriate cost-effectiveness threshold for EoL treatments as well as to provide input into the debate on the justifiability of a cancer and/or a rare disease premium when evaluating therapies. METHODS: A contingent valuation was conducted in an online survey using a representative sample of the Croatian population (n = 1500) to calculate the willingness to pay for gains in the remaining life expectancy at the EoL, from the social-inclusive-individual perspective, using payment scales and an open-ended payment vehicle. Our approach mimics the actual decision-making problem of deciding whether to reimburse therapies targeting EoL conditions such as metastatic cancer whose main purpose is to extend life (and not add quality to life). RESULTS: Average EoL-VOLY across all scenarios was estimated at €67,000 (median €40,000). In scenarios that offered respondents 1 full year of life extension, EoL-VOLY was estimated at €33,000 (median €22,000). Our results show that the type of illness is irrelevant for EoL-VOLY evaluations. CONCLUSIONS: The pressure to reimburse expensive therapies targeting EoL conditions will continue to increase. Delivering "value for money" in healthcare, both in countries with relatively higher and lower budget restrictions, requires the valuation of different types of health gains, which should, in turn, affect our ability to evaluate their cost effectiveness.

Access to Healthcare and Health Literacy in Croatia: Empirical Investigation.

Health literacy is related to different health-related outcomes. However, the nature of the relationship between health literacy and health outcomes is not well understood. One pathway may lead from health literacy to health outcomes by means of access to healthcare. The goal of the current study is to explore the association between health literacy and the particular measure of access to healthcare-unmet medical need-for the first time in Croatia and, to the best of our knowledge, for the first time in the EU context. We use data obtained from face-to-face interviews in a large nationally representative sample of the Croatian population (n = 1000) to estimate the level of health literacy and self-reported access to care and investigate the association between health literacy and self-perceived barriers to access. Our study showed that limited and problematic health literacy is prevalent and associated with higher rates of unmet medical need. Unmet need is largely caused by long waiting lists. It is therefore essential to design health services fitting the needs of those who have limited and/or problematic health literacy as well as enhance health education with the potential of improving the access to care and health outcomes as well as design policies that reduce waiting times.

Budget Impact Analysis of Pharmacist-Led Medication Management in Cardiovascular and Type 2 Diabetic Patients.

The paper aims to identify and measure the costs and savings associated with the delivery of Comprehensive Medication Management (CMM) services in Croatia in patients diagnosed with hypertension accompanied by at least one additional established cardiovascular disease (CVD) and/or type 2 diabetes mellitus (DMT2) who use five or more medicines daily. The budget impact analysis (BIA) employed in this study compares the total costs of CMM to the cost reductions expected from CMM. The cost reductions (or savings) are based on the reduced incidence of unwanted clinical events and healthcare service utilisation rates due to CMM. The BIA model is populated by data on medication therapy costs, labour, and training from the pilot CMM intervention introduced in Zagreb's main Health Centre, while relevant international published sources were used to estimate the utilisation, incidence, and unwanted clinical events rates. Total direct costs, including pharmacists' labour and training (EUR 2,667,098) and the increase in the cost of prescribed medication (EUR 5,182,864) amounted to EUR 7,849,962 for 3 years, rendering the cost per treated patient per year EUR 57. CMM is expected to reduce the utilisation rates of healthcare services and the incidence of unwanted clinical events, leading to a total 3-year reduction in healthcare costs of EUR 7,787,765. Given the total CMM costs of EUR 7,849,962, CMM's 3-year budget impact equals EUR 92,869, rendering per treated patient an incremental cost of CMM EUR 0.67. Hence, CMM appears to be an affordable intervention for addressing medication mismanagement and irrational drug use.

Pricing and Reimbursement of Patent-Protected Medicines: Challenges and Lessons from South-Eastern Europe.

BACKGROUND: Efficiency and transparency of pricing and reimbursement (P&R) rules and procedures as well as their implementation in South-eastern Europe (SEE) lag substantially behind Western European practice. Nevertheless, P&R systems in SEE are rarely critically assessed, warranting a detailed and wider-encompassing exploration. OBJECTIVE: Our study provides a comparative assessment of P&R processes for patent-protected medicines in ten SEE countries-EU member states: Croatia, Slovenia, Hungary, Romania and Bulgaria; and non-EU countries: Albania, Montenegro, Serbia, North Maceodina, Bosnia and Herzegovina. P&R systems are compared and evaluated through a research framework that focuses on: (1) public financing of patent-protected medicines, (2) definition of benefit packages, (3) requirements for the submission of reimbursement dossiers, (4) assessment and appraisal processes, (5) reimbursement decision making, (6) processes that occur post reimbursement, and (7) pricing. The study aims to contribute to the discussion on improving the efficiency and quality of P&R of patent-protected medicines in the region. METHODS: We conducted a non-systematic literature review of published literature, as well as policy briefs and reports on healthcare systems in the SEE region along with legal documents framing the P&R procedures in local languages. The information gathered from these various sources was then discussed and clarified through structured telephone interviews with relevant national experts from each SEE country, mainly current and former senior officials and/or executives of the funding and assessment/ appraisal bodies (total of 20 interviews conducted in late 2019). RESULTS: Capacity building through sharing knowledge and information on successful reforms across borders is an opportunity for SEE countries to further develop their P&R policies and increase (equitable) access to patent-protected medicines (especially expensive medicines), increasing affordability and containing costs. Simple yet robust and systematic decision-making frameworks that rely on international health technology assessment (HTA) procedures and are based on the pursuit of transparency seem to be the most cost-effective approach to strengthening P&R systems in SEE. CONCLUSIONS: Further reforms aiming to develop transparent and robust national decision-making frameworks (including oversight) and build institutional HTA-related and decision-making capacity are awaited in most of SEE countries, especially the non-EU members. In non-EU SEE countries, these efforts could increase access to patent-protected medicines, which is-at the moment-very limited. The EU-member SEE countries operate more developed P&R systems but could further benefit from developing their procedures, oversight and value-for-money assessment toolbox and capacity, hence further improving the transparency and efficiency of procedures that regulate access to patent-protected medicines.

Is it too expensive to fight cancer? Analysis of incremental costs and benefits of the Croatian National Plan Against Cancer.

This cost-effectiveness study analyses the expected impacts of activities proposed by the Croatian National Plan Against Cancer (NPAC) on cancer incidence and survival rates, as related to their respective costs. We evaluated the impact of the NPAC on two main outcomes, namely, reduced incidence and the improved survival of cancer patients, expressed as life years gained (LYGs), which enabled the calculation of incremental cost-effectiveness ratios (ICERs) in the form of cost per LYG. In the analysis of costs, we considered both the direct costs of NPAC activities as well as the wider indirect societal costs of cancer, thus permitting the calculation of the ICER both from the narrower national health insurer's perspective (accounting only for the direct costs) and the wider societal perspective (accounting both for the direct and indirect costs). We estimated that on average, for all patients benefiting from the implementation of the NPAC in Croatia, an additional LYG would be yielded at the additional cost of €1.021 (societal perspective). The NPAC can, for some sites, even be considered a dominant intervention due to the negative cost/LYG ratio, meaning that it generates additional LYGs while at the same time, reducing total societal costs. Taking a narrower health insurer's perspective (i.e., accounting only for the direct costs), the NPAC produces an additional LYG at an additional cost of €1.408. Both cost per LYG estimates can be considered cost-effective investment options.

Mitigating hypothetical bias in willingness to pay studies: post-estimation uncertainty and anchoring on irrelevant information.

One possible source of hypothetical bias in willingness to pay (WTP) estimates is response uncertainty, referring to subject's uncertainty about the value of the good under assessment. It has been argued that uncertainty can be measured using the post-valuation 'certainty question' that asks: 'How certain are you about your stated WTP?' and marks the degree of certainty on a quantitative or a qualitative scale. Research has shown that the self-reported certainty evaluations can help mitigate hypothetical bias and obtain increasingly accurate WTP estimates. These study reports present a simple test of reliability of post-valuation certainty assessment and then looks at the empirical evidence for clues regarding the general usefulness of certainty adjustment in mitigating hypothetical bias in WTP studies. We find that the post-estimation uncertainty scores are malleable, i.e., significantly correlated with entirely irrelevant information. We conclude that more robust evidence could justify the routine inclusion of certainty evaluation in WTP studies although in the meantime the interpretation of certainty-adjusted WTP values should be approached cautiously.

The impact of the design of payment scales on the willingness to pay for health gains.

The questionnaire format applied in a CV study represents the way in which the WTP estimates are obtained. Payment scales are often used in CV studies as the questionnaire format of choice. The study summarized here analyzes the impact of the design of two payment scales (PS) on the monetary value of QALY gains. The scales differed in terms of their end-points, mid points, and coarseness. We judged the performance of the two PS against several indicators: the average WTP per QALY estimates, post-estimation uncertainty levels, the existence of mid-point concentration, and the dependency on end-points. Our results show that PS design influences respondents' WTP values. The results also suggest that a more detailed scale with a more realistic range may help respondents to elicit values closer to their "true" WTP values, hence produce higher-quality outcomes. Further research and pretesting strategies are suggested to explore and minimize the effects of PS design on WTP estimates, which may ultimately increase the quality of WTP estimates.

Valuing QALYs in Relation to Equity Considerations Using a Discrete Choice Experiment.

BACKGROUND: To judge whether an intervention offers value for money, the incremental costs per gained quality-adjusted life-year (QALY) need to be compared with some relevant threshold, which ideally reflects the monetary value of health gains. Literature suggests that this value may depend on the equity context in which health gains are produced, but the value of a QALY in relation to equity considerations has remained largely unexplored. OBJECTIVE: The objective of this study was to estimate the social marginal willingness to pay (MWTP) for QALY gains in different equity subgroups, using a discrete choice experiment (DCE). Both severity of illness (operationalized as proportional shortfall) and fair innings (operationalized as age) were considered as grounds for differentiating the value of health gains. METHODS: We obtained a sample of 1205 respondents, representative of the adult population of the Netherlands. The data was analysed using panel mixed multinomial logit (MMNL) and latent class models. RESULTS: The panel MMNL models showed counterintuitive results, with more severe health states reducing the probability of receiving treatment. The latent class models revealed distinct preference patterns in the data. MWTP per QALY was sensitive to severity of disease among a substantial proportion of the public, but not to the age of care recipients. CONCLUSION: These findings emphasize the importance of accounting for preference heterogeneity among the public on value-laden issues such as prioritizing health care, both in research and decision making. This study emphasises the need to further explore the monetary value of a QALY in relation to equity considerations.

What Influences Patients' Decisions When Choosing a Health Care Provider? Measuring Preferences of Patients with Knee Arthrosis, Chronic Depression, or Alzheimer's Disease, Using Discrete Choice Experiments.

OBJECTIVE: To investigate what influences patients' health care decisions and what the implications are for the provision of information on the quality of health care providers to patients. DATA SOURCES/STUDY SETTING: Dutch patient samples between November 2006 and February 2007. STUDY DESIGN: Discrete choice experiments were conducted in three patient groups to explore what influences choice for health care providers. DATA COLLECTION: Data were obtained from 616 patients with knee arthrosis, 368 patients with chronic depression, and 421 representatives of patients with Alzheimer's disease. PRINCIPAL FINDINGS: The three patients groups chose health care providers on a different basis. The most valued attributes were effectiveness and safety (knee arthrosis); continuity of care and relationship with the therapist (chronic depression); and expertise (Alzheimer's disease). Preferences differed between subgroups, mainly in relation to patients' choice profiles, severity of disease, and some background characteristics. CONCLUSIONS: This study showed that there is substantial room for (quality) information about health care providers in patients' decision processes. This information should be tailor-made, targeting specific patient segments, because different actors and factors play a part in their search and selection process.

The value of a QALY: individual willingness to pay for health gains under risk.

BACKGROUND: There is an increased interest in the monetary value of a quality-adjusted life-year (QALY). Past studies commonly derived willingness to pay (WTP) for certain future QALY gains. However, obtaining valid WTP per QALY estimates proved to be difficult. OBJECTIVE: We conducted a contingent valuation study and estimated the individual WTP per QALY under risk. We demonstrate the impact of probability weighting on WTP per QALY estimates in the Netherlands. RESULTS: Our estimates of the value of a QALY are in the range of €80,000-110,000 when the weighting correction was applied, and €250,500 without correction. The validity of these estimates, applying probability weighting, appears to be good. CONCLUSIONS: Given the reasonable support for their validity and practical meaningfulness, the estimates derived while correcting for probability weighting may provide valuable input for the debate on the consumption value of health. While decision makers should not apply these estimates without further consideration, since strictly individual valuations may not carry all relevant information and values for societal decision-making, the current estimates may provide a good and informed basis for further discussion and study of this important topic.

Valuing QALY gains by applying a societal perspective.

Interpreting the outcomes of cost utility analyses requires an appropriately defined threshold for costs per quality-adjusted life year (QALY). A common view is that the threshold should represent the (consumption) value a society attaches to a QALY. So far, individual valuations of personal health gains have mainly been studied rather than potentially relevant social values. In this study, we present the first direct empirical estimates of the willingness to pay for a QALY from a societal perspective. We used the contingent valuation approach, valuing QALYs under uncertainty and correcting for probability weighting. The estimates obtained in a representative sample of the Dutch population (n = 1004) range from €52,000 to €83,000, depending on the specification of the societal perspective. The scale sensitivity was weak, however.

Inquiry into the relationship between equity weights and the value of the QALY.

BACKGROUND: A commonly held view of the decision rule in economic evaluations in health care is that the final incremental cost-effectiveness ratio needs to be judged against some threshold, which is equal for all quality-adjusted life-year (QALY) gains. This reflects the assumption that "a QALY is a QALY" no matter who receives it, or the equity notion that all QALY gains are equally valuable, regardless of the context in which they are realized. If such an assumption does not adequately reflect the distributional concerns in society, however, different thresholds could be used for different QALY gains, whose relative values can be seen as "equity weights." AIM: Our aim was to explore the relationship between equity or distributional concerns and the social value of QALYs within the health economics literature. In light of the empirical interest in equity-related concerns as well as the nature and height of the incremental cost-effectiveness ratio threshold, this study investigates the "common ground" between the two streams of literature and considers how the empirical literature estimating the incremental cost-effectiveness ratio threshold treats existing distributional considerations.

GET MORE, PAY MORE? An elaborate test of construct validity of willingness to pay per QALY estimates obtained through contingent valuation.

Estimates of WTP per QALY can be taken as an indication of the monetary value of health gains, which may carry information regarding the appropriate height of the cost-effectiveness threshold. Given the far-reaching consequences choosing a particular threshold, and thus the potential relevance of WTP per QALY estimates, it is important to address the validity of these estimates. This study addresses this issue. Our findings offer little support to the validity of WTP per QALY estimates obtained in this study. Implications for general WTP per QALY estimates and further research are discussed.

Discounting future health gains: an empirical enquiry into the influence of growing life expectancy.

We tested the influence of the growth in life expectancy over time on social time preferences for health. Growing life expectancy of future generations should raise social discount rates for health because of diminishing marginal utility of additional health gains and equity reasons reflecting the desire for a more equitable distribution of benefits over generations. This influence has, however, been largely ignored in empirical studies. We provide a first comprehensive analysis of how time preferences for health gains vary with projected growth rates, indicating the importance of subjective expectations about the growth in life expectancy in the elicitation of social time preference. Six hundred and fifty-six respondents, representative of the Dutch population, completed one of four questionnaires, differing in the projected growth in life expectancy. Results showed that individuals discount future health gains at different rates, depending on the latency period and on the projected or expected growth in life expectancy. As hypothesized, discount rates increased with higher growth rates. The association between observed discount rates and expectations regarding future life expectancy was confirmed, suggesting that discount rates for health may depend on future life expectancy. In light of our results, specifying life expectancy of future generations in time preference exercises appears appropriate.

Health effects in significant others: separating family and care-giving effects.

BACKGROUND: Changes in the health of patients may affect the health of so-called "significant others" in 2 distinct ways. First, an individual may provide informal care to the patient and be burdened by the process of care giving. We label this indirect effect of a patient's health on the health of the care giver the "care-giving effect." Second, a person may suffer from health losses because someone in his or her social environment is ill, regardless of his or her care-giving status. The health of the patient then directly affects the health of this significant other, which we label the "family effect." METHODS: We investigate the occurrence of the family and care-giving effect in a convenience sample of Dutch care givers (n = 751). The family effect was approximated by the health status of the patient (measured on EuroQol-VAS), and the care-giving effect by the number of the care-giving tasks was provided. It was assumed that care givers' health is positively associated with patients' health, that is, the family effect, and negatively associated with care-giving burden, that is, the care-giving effect. Relationships are studied using multivariate regressions. RESULTS: Our results support the existence of both types of health effects. The analysis shows that the 2 effects are separable and independently associated with the health of care givers. Not accounting for the family effect conflates the care-giving effect. CONCLUSIONS: If the goal of health care policy is to optimize health, all important effects should be captured. The scope of economic evaluations should also include health effects in significant others. This study suggests that significant others include both care givers and broader groups of affected individuals, such as family members.

Willingness to pay for a quality-adjusted life-year: the individual perspective.

OBJECTIVE: The aim of this study was to elicit the individual willingness to pay (WTP) for a quality-adjusted life-year (QALY). METHODS: In a Web-based questionnaire containing contingent valuation exercises, respondents valued health changes in five scenarios. In each scenario, the respondents first valued two health states on a visual analog scale (VAS) and expressed their WTP for avoiding a decline in health from the better health state to the worse, using a payment scale followed by a bounded open contingent valuation question. ANALYSIS: WTP per QALY was calculated for QALY gains calculated using VAS valuations, as well as the Dutch EQ-5D tariffs, the two steps in the WTP estimations and each scenario. Heterogeneity in WTP per QALY ratios was examined from the perspective of: 1) household income; and 2) the level of certainty in WTP indicated by respondents. Theoretical validity was analyzed using clustered multivariate regressions. RESULTS: A total of 1091 respondents, representative of the Dutch population, participated in the survey. Mean WTP per QALY was € 12,900 based on VAS valuations, and € 24,500 based on the Dutch EuroQoL tariffs. WTP per QALY was strongly associated with income, varying from € 5000 in the lowest to € 75,400 in the highest income group. Respondents indicating higher certainty exhibited marginally higher WTP. Regression analyses confirmed expected relations between WTP per QALY, income, and other personal characteristics. CONCLUSION: Individual WTP per QALY values elicited in this study are similar to those found in comparable studies. The use of individual valuations in social decision-making deserves attention, however.

Caring for and caring about: disentangling the caregiver effect and the family effect.

Besides patients' health and well-being, healthcare interventions may affect the well-being of significant others. Such 'spill over effects' in significant others may be distinguished in two distinct effects: (i) the caregiving effect and (ii) the family effect. The first refers to the welfare effects of providing informal care, i.e., the effects of caring for someone who is ill. The second refers to a direct influence of the health of a patient on others' well-being, i.e., the effects of caring about other people. Using a sample of Dutch informal caregivers we found that both effects exist and may be comparable in size. Our results, while explorative, indicate that economic evaluations adopting a societal perspective should include both the family and the caregiving effects measured in the relevant individuals.

The invisible hands made visible: recognizing the value of informal care in healthcare decision-making.

The healthcare sector depends heavily on the informal care provided by families and friends of those who are ill. Informal caregivers may experience significant burden as well as health and well-being effects. Resource allocation decisions, in particular from a societal perspective, should account explicitly for these effects in the social environment of patients. This is not only important to make a complete welfare economic assessment of treatments, but also to ensure the lasting involvement of informal caregivers in the care-giving process. Measurement and valuation techniques for the costs and effects of informal care have been developed and their use is becoming more common. Decision-makers in healthcare - and eventually families and patients - would be helped by more uniformity in methods.