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.

Update of the Dutch Manual for Costing in Economic Evaluations.

OBJECTIVES: In 2000, the first "Dutch Manual for Costing: METHODS and Reference Prices for Economic Evaluations in Healthcare" was published, followed by an updated version in 2004. The purpose of the Manual is to facilitate the implementation and assessment of costing studies in economic evaluations. New developments necessitated the publication of a thoroughly updated version of the Manual in 2010. The present study aims to describe the main changes of the 2010 Manual compared with earlier editions of the Manual. METHODS: New and updated topics of the Manual were identified. The recommendations of the Manual were compared with the health economic guidelines of other countries, eliciting strengths and limitations of alternative methods. RESULTS: New topics in the Manual concern medical costs in life-years gained, the database of the Diagnosis Treatment Combination (DBC) casemix System, reference prices for the mental healthcare sector and the costs borne by informal care-givers. Updated topics relate to the friction cost method, discounting future effects and options for transferring cost results from international studies to the Dutch situation. CONCLUSIONS: The Action Plan is quite similar to many health economic guidelines in healthcare. However, the recommendations on particular aspects may differ between national guidelines in some respects. Although the Manual may serve as an example to countries intending to develop a manual of this kind, it should always be kept in mind that preferred methods predominantly depend on a country's specific context.

Burden of illness of Pompe disease in patients only receiving supportive care.

BACKGROUND: Pompe disease is an orphan disease for which enzyme replacement therapy (ERT) recently became available. This study aims to estimate all relevant aspects of burden of illness--societal costs, use of home care and informal care, productivity losses, and losses in health-related quality of life (HRQoL)--for adult Pompe patients only receiving supportive care. METHODS: We collected data on all relevant aspects of burden of illness via a questionnaire. We applied a societal perspective in calculating costs. The EQ-5D was used to estimate HRQoL. RESULTS: Eighty adult patients (87% of the total Dutch adult Pompe population) completed a questionnaire. Disease severity ranged from mild to severe. Total annual costs were estimated at 22,475 (range 0-169,539) per adult Pompe patient. Patients on average received 8 h of home care and 19 h of informal care per week. Eighty-five percent of the patients received informal care from one or more caregivers; 40% had stopped working due to their disease; another 20% had reduced their working hours. HRQoL for Pompe patients who only received supportive care was estimated at 0.72, 17% lower than the Dutch population at large. CONCLUSIONS: Adult Pompe disease is associated with a considerable burden of illness at both the societal and patient levels. The disease leads to substantial costs and dependency on medical devices, home care, and informal care, and has a high impact on the patient's social network. In addition, patients are limited in their ability to work and have significantly reduced HRQoL.

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.

Relationships among self-management, patient perceptions of care, and health economic outcomes for decision-making and clinical practice in type 2 diabetes.

OBJECTIVES: Type 2 diabetes (T2D) treatment involves complex interactions between biological, psychological, and behavioral factors of care, requiring multifaceted efforts in clinical practice and disease management to reduce health and economic burdens. We aimed to quantify correlations among these factors and characterize their level of inclusion in economic analyses that are part of informed medical decision-making. METHODS: A comprehensive, stepwise systematic literature review was performed on published articles dated 1993 to 2008 using medical subject heading and keyword searches in electronic reference libraries. Data were collected using standardized techniques and were analyzed descriptively. RESULTS: A total of 97 articles fulfilling all inclusion criteria were reviewed, including 16 on economic models (17% of articles). Most studies were retrospective (41 of 97; 42%) and from managed care perspectives (66%). Oral antidiabetic drugs were a central focus, appearing in 83% of studies. Patient behaviors, particularly medication adherence and persistence in real-world settings, are well researched (n=65) and may influence diabetes outcomes, cardiovascular risk, mortality rates, and treatment-specific resource use (e.g., hospitalizations) and costs (

Lifestyle intervention: from cost savings to value for money.

Prevention of unhealthy lifestyles has sometimes been promoted as simultaneously reducing costs and improving public health but this will unlikely prove to be true. Additional medical costs in life years gained due to treatment of unrelated diseases may offset possible savings in related diseases, but are often ignored both in health promotion policies and in economic evaluations of life-prolonging interventions. Many national guidelines explicitly recommend excluding these costs from economic evaluations or leave inclusion up to the discretion of the analyst. This may result in too favorable estimations of cost-effectiveness, feeding the unjustified optimism among policymakers regarding lifestyle interventions as a cost-saving option. However, prevention may still be a cost-effective way to improve public health, even when it does not result in cost savings, but this should be judged taking all future costs into account and be based on the true value for money provided by lifestyle interventions.

Cost-effectiveness of collaborative care for the treatment of major depressive disorder in primary care. A systematic review.

BACKGROUND: The effectiveness of collaborative care for patients with major depressive disorder in primary care has been established. Assessing its cost-effectiveness is important for deciding on implementation. This review therefore evaluates the cost-effectiveness of collaborative care for major depressive disorder in primary care. METHODS: A systematic search on economic evaluations of collaborative care was conducted in Pubmed and PsychInfo. Quality of the studies was measured with the Cochrane checklist and the CHEC-list for economic evaluations. Cost-effectiveness and costs per depression-free days were reported. RESULTS: 8 studies were found, involving 4868 patients. The quality of the cost effectiveness studies, according to the CHEC-list, could be improved. Generally, the studies did not include all relevant costs and did not perform sensitivity analysis. Only 4 out of 8 studies reported cost per QALY, 6 out of 8 reported costs per depression-free days. The highest costs per QALY reported were $49,500, the highest costs per depression-free day were $24. CONCLUSIONS: Although studies did not fulfil all criteria of the CHEC-list, collaborative care is a promising intervention and it may be cost-effective. However, to conclude on the cost-effectiveness, depression research should follow economic guidelines to improve the quality of the economic evaluations.

Uncertainty in decision-making: value of additional information in the cost-effectiveness of lifestyle intervention in overweight and obese people.

OBJECTIVES: To quantify the uncertainty in the cost-effectiveness of lifestyle intervention versus standard care in overweight and obese people in Switzerland and to determine if further research is necessary based on current information. There has been an increasing interest in using value of information analysis in medical decision-making to quantify the uncertainty in decision-making, and to identify the need for further research. METHODS: Value of information analysis was applied on a probabilistic cost-effectiveness model to evaluate the uncertainty by calculating the patient expected value of perfect information (EVPI), population EVPI, and partial EVPI. The costs were expressed in Swiss Francs (CHF), price year 2006. RESULTS: Overall, the EVPI was higher in overweight than in obese people. The maximum population EVPI was CHF 6.8 million in overweight people and CHF 3.2 million in moderate obese people, representing the upper limit on costs associated with decision uncertainty. The partial EVPI estimated a higher uncertainty in the model parameters such as utilities, body mass index, cardiovascular risk factors, and systolic blood pressure in overweight and moderate obese subjects. CONCLUSIONS: The EVPI analysis indicates that there is some uncertainty regarding the choice between lifestyle intervention and standard care. The parameter EVPI suggests that if further research is commissioned, this should focus on the effectiveness of lifestyle intervention on cardiovascular risk factors and utilities.

Welfarism vs. extra-welfarism.

'Extra-welfarism' has received some attention in health economics, yet there is little consensus on what distinguishes it from more conventional 'welfarist economics'. In this paper, we seek to identify the characteristics of each in order to make a systematic comparison of the ways in which they evaluate alternative social states. The focus, though this is not intended to be exclusive, is on health. Specifically, we highlight four areas in which the two schools differ: (i) the outcomes considered relevant in an evaluation; (ii) the sources of valuation of the relevant outcomes; (iii) the basis of weighting of relevant outcomes and (iv) interpersonal comparisons. We conclude that these differences are substantive.

Costs and effects of secondary prevention with perindopril in stable coronary heart disease in Poland: an analysis of the EUROPA study including 1251 Polish patients.

OBJECTIVES: To estimate the long-term impact of treatment with perindopril on costs and health effects in patients with stable coronary artery disease in Poland. METHODS: The cost-effectiveness analysis was based on data from a randomized double-blind, placebo-controlled trial. A decision-tree analysis was employed, including Monte Carlo and bootstrapping techniques. This study was a sub-study of the EUROPA (European Trial on Reduction of Cardiac Events with Perindopril in Stable Coronary Artery Disease) trial (n = 12,218; mean follow-up 4.2 years). Resource use was based on data from Polish EUROPA study patients (n = 1251), while effectiveness was based on the whole EUROPA study. The health gain of perindopril in life-years was based on overall EUROPA study results, and the adapted Polish life expectancy of patients not dying during the trial. Costs were calculated in new Polish zloty (PLN), year 2003 values; euro1 = PLN4.053. Only direct healthcare costs related to cardiovascular events and medication use were studied. RESULTS: When observed mortality was combined with life expectancy beyond the end of the study, perindopril use showed a gain in life expectancy of 0.182 life-years (SD +/- 0.129) at a cost of PLN1983 (SD +/- 103) with discounting of 5% per annum on costs and no discounting on effects. This resulted in an incremental cost-effectiveness ratio (ICER) of PLN10 896 per life-year gained. The probability that the ICER for perindopril was below the threshold of PLN60 000 was 88%. The overall results were insensitive to discount rates for costs and life-years. CONCLUSIONS: Perindopril leads to a reduction in the risk of coronary events among patients with stable heart disease. When the expected improvement in life expectancy is combined with associated medical costs, there is a high probability that perindopril is cost effective, given the threshold of PLN60 000 per life-year gained.

Unrelated medical costs in life-years gained: should they be included in economic evaluations of healthcare interventions?

Which costs and benefits to consider in economic evaluations of healthcare interventions remains an area of much controversy. Unrelated medical costs in life-years gained is an important cost category that is normally ignored in economic evaluations, irrespective of the perspective chosen for the analysis. National guidelines for pharmacoeconomic research largely endorse this practice, either by explicitly requiring researchers to exclude these costs from the analysis or by leaving inclusion or exclusion up to the discretion of the analyst. However, the inclusion of unrelated medical costs in life-years gained appears to be gaining support in the literature.This article provides an overview of the discussions to date. The inclusion of unrelated medical costs in life-years gained seems warranted, in terms of both optimality and internal and external consistency. We use an example of a smoking-cessation intervention to highlight the consequences of different practices of accounting for costs and effects in economic evaluations. Only inclusion of all costs and effects of unrelated medical care in life-years gained can be considered both internally and externally consistent. Including or excluding unrelated future medical costs may have important distributional consequences, especially for interventions that substantially increase length of life. Regarding practical objections against inclusion of future costs, it is important to note that it is becoming increasingly possible to accurately estimate unrelated medical costs in life-years gained. We therefore conclude that the inclusion of unrelated medical costs should become the new standard.

The "health benefit basket" in the Netherlands.

This contribution describes the entitlements in Dutch health care and explores how these entitlements are determined and to whom they apply. The focus is on services of curative care. No comprehensive positive or negative list of individual services is included in formal laws. Instead, the legislation states only what general types of medical services are covered and generally the "usual care" criterion determines to which interventions patients are entitled. This criterion is not very restrictive and yields local variations in service provision, which are moderated by practice guidelines. It is conceivable, however, that the recent introduction of the DBC financing system will change the reimbursement and therefore benefit-setting policy.

Standardisation of costs: the Dutch Manual for Costing in economic evaluations.

The lack of a uniform costing methodology is often considered a weakness of economic evaluations that hinders the interpretation and comparison of studies. Standardisation is therefore an important topic within the methodology of economic evaluations and in national guidelines that formulate the formal requirements for studies to be considered when deciding on the reimbursement of new medical therapies. Recently, the Dutch Manual for Costing: Methods and Standard Costs for Economic Evaluations in Health Care (further referred to as "the manual") has been published, in addition to the Dutch guidelines for pharmacoeconomic research. The objectives of this article are to describe the main content of the manual and to discuss some key issues of the manual in relation to the standardisation of costs. The manual introduces a six-step procedure for costing. These steps concern: the scope of the study;the choice of cost categories;the identification of units;the measurement of resource use;the monetary valuation of units; andthe calculation of unit costs. Each step consists of a number of choices and these together define the approach taken. In addition to a description of the costing process, five key issues regarding the standardisation of costs are distinguished. These are the use of basic principles, methods for measurement and valuation, standard costs (average prices of healthcare services), standard values (values that can be used within unit cost calculations), and the reporting of outcomes. The use of the basic principles, standard values and minimal requirements for reporting outcomes, as defined in the manual, are obligatory in studies that support submissions to acquire reimbursement for new pharmaceuticals. Whether to use standard costs, and the choice of a particular method to measure or value costs, is left mainly to the investigator, depending on the specific study setting. In conclusion, several instruments are available to increase standardisation in costing methodology among studies. These instruments have to be used in such a way that a balance is found between standardisation and the specific setting in which a study is performed. The way in which the Dutch manual tries to reach this balance can serve as an illustration for other countries.

Unit costs of inpatient hospital days.

BACKGROUND: Costs of inpatient days in hospitals are frequently the main drivers of total treatment costs, and their unit cost can markedly affect the outcomes of an economic evaluation. In many countries, the availability of unit cost data is limited and unit costs are often based on data from hospitals participating in clinical trials. OBJECTIVE: To provide data about unit costs of inpatient hospital days in The Netherlands from a healthcare provider's perspective and to give an insight into the extent to which cost categories and total costs differ between hospitals. DESIGN: Unit costs were collected from 22 wards and 11 intensive care units (ICUs) of general and university hospitals involved in clinical trials with 'piggybacked' economic evaluations. Direct costs, such as costs of nursing and medical materials, were calculated by dividing the annual cost per category of the nursing department by the annual number of inpatient days. Indirect costs, such as overheads and accommodation, were allocated to the nursing departments by applying direct allocation. All costs were expressed in 1998 euros (EUR). RESULTS: The mean costs per inpatient day were EUR230 (range: EUR154-EUR311) in general hospitals and EUR323 (range: EUR209-EUR400) in university hospitals. The mean costs per inpatient day in an ICU were EUR1125 (EUR919-EUR1560). Between 38-48% of the total costs were made up of nursing costs. All cost categories showed wide variations between hospitals. CONCLUSIONS: The results of this study were used to develop standard costs for inpatient days in The Netherlands and may contribute to the comparability and generalisability of economic evaluations.

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.

Cost-effectiveness of breast cancer control strategies in Central America: the cases of Costa Rica and Mexico.

This paper reports the most cost-effective policy options to support and improve breast cancer control in Costa Rica and Mexico. Total costs and effects of breast cancer interventions were estimated using the health care perspective and WHO-CHOICE methodology. Effects were measured in disability-adjusted life years (DALYs) averted. Costs were assessed in 2009 United States Dollars (US$). To the extent available, analyses were based on locally obtained data. In Costa Rica, the current strategy of treating breast cancer in stages I to IV at a 80% coverage level seems to be the most cost-effective with an incremental cost-effectiveness ratio (ICER) of US$4,739 per DALY averted. At a coverage level of 95%, biennial clinical breast examination (CBE) screening could improve Costa Rica's population health twofold, and can still be considered very cost-effective (ICER US$5,964/DALY). For Mexico, our results indicate that at 95% coverage a mass-media awareness raising program (MAR) could be the most cost-effective (ICER US$5,021/DALY). If more resources are available in Mexico, biennial mammography screening for women 50-70 yrs (ICER US$12,718/DALY), adding trastuzumab (ICER US$13,994/DALY) or screening women 40-70 yrs biennially plus trastuzumab (ICER US$17,115/DALY) are less cost-effective options. We recommend both Costa Rica and Mexico to engage in MAR, CBE or mammography screening programs, depending on their budget. The results of this study should be interpreted with caution however, as the evidence on the intervention effectiveness is uncertain. Also, these programs require several organizational, budgetary and human resources, and the accessibility of breast cancer diagnostic, referral, treatment and palliative care facilities should be improved simultaneously. A gradual implementation of early detection programs should give the respective Ministries of Health the time to negotiate the required budget, train the required human resources and understand possible socioeconomic barriers.

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.