Choices in oncology: factors that influence patients' treatment preference

Medical treatment of cancer often entails a trade-off between outcomes of two different attributes: quality of life (QOL) and length of life (LL). This process of weighing advantages and disadvantages seems to be influenced by different factors. The main purposes of this study were (a) to investigate the relative importance of different factors on the trade-off and (b) to explore the relationship between these importance ratings and personal characteristics. We asked 199 patients with cancer to indicate to what degree they consider a number of factors to be of importance if they had to choose between two treatment modallties that differ in their expected outcomes concerning QOL and LL. The seven factors were their age at the time of decision, having a partner, having children, inability to work due to side-effects of the treatment, the nature of the side-effects, disease-related life expectancy and baseline QOL. The results indicate that six of the seven factors were of considerable to great importance when a treatment choice had to be made. The negative effects of treatment on the ability to work did not seem to be a very important consideration. Patient age and education were positively associated with importance ratings.Supported by the Dutch Cancer Society (Project IKW 90-13).     

Social tariffs and democratic choice—Do population‐based health state values reflect the will of the people?

In economic evaluations of health technologies, health outcomes are commonly measured in terms of quality‐adjusted life years (QALYs). QALYs are the product of time and health‐related quality of life. Health‐related quality of life, in turn, is determined by a social tariff, which is supposed to reflect the public's preference over health states. This study argues that, because of the tariff's role in the societal decision‐making process, it should not be understood as merely an operational (statistical) definition of health, but as a major instrument of democratic participation. I outline what implications this might have for both the method used to aggregate individual preferences, and the set of individuals whose preferences should count. Alternative tariff specifications and decision rules are explored, and future research directions are proposed.     

Towards Cost-Value Analysis in Health Care?

By describing societal value judgements in health care in numerical terms one may in theory increase the precision of guidelines for priority setting and allow decision makers to judge more accurately the degree to which different health care programs provide societal value for money. However, valuing health programs in terms of QALYs disregards salient societal concerns for fairness in resource allocation. A different kind of numerical valuation of medical interventions, that incorporates concerns for fairness, is described. The usefulness to decision makers of such numerical information remains to be tested. This revised version was published online in August 2006 with corrections to the Cover Date.     

药物经济学评价中的健康结果测量

目的 :为选择适当的药物经济学评价结果指标提供建议。方法 :比较3类不同健康结果指标的区别及其测量方法 ,并介绍各国《指南》有关结果测量的标准。结果与结论 :应根据不同的卫生保健体系、研究角度和疾病选择相应的结果测量方法。     

Calibration of Quality-Adjusted Life Years for Oncology Clinical Trials

ContextQuality-adjusted life year (QALY) estimation is a well-known but little used technique to compare survival adjusted for complications. Lack of calibration and interpretation guidance hinders implementation of QALY analyses.ObjectivesWe conducted simulation studies to assess the impact of differences in survival, toxicity rates, and utility values on QALY results.MethodsSurvival comparisons used both log-rank and Wilcoxon testing. We examined power considerations for a North Central Cancer Treatment Group Phase III lung cancer clinical trial (89-20-52).ResultsSample sizes of 100 events per treatment have low power to generate a statistically significant difference in QALYs unless the toxicity rate is 44% higher in one arm. For sample sizes of 200 per arm and equal survival times, toxicity needs to be at least 38% more in one arm for the result to be statistically significant, using a utility of 0.3 for days with toxicity. Sample sizes of 300 (500)/arm provide 80% power if there is a 31% (25%) toxicity difference. If the overall survival hazard ratio between the two treatment arms is 1.25, then samples of at least 150 patients and 13% increased toxicity are necessary to have 80% power to detect QALY differences. In study 89-20-52, there was only 56% power to determine the statistical significance of the observed QALY differences, clarifying the enigmatic conclusion of no statistically significant difference in QALY despite an observed 14.5% increase in toxicity between treatments.ConclusionThis calibration allows researchers to interpret the clinical significance of QALY analyses and facilitates QALY inclusion in clinical trials through improved study design.     

The Willingness to Pay for a Quality Adjusted Life Year: A Review of the Empirical Literature

There has been a rapid increase in the use of cost‐effectiveness analysis, with quality adjusted life years (QALYs) as an outcome measure, in evaluating both medical technologies and public health interventions. Alongside, there is a growing literature on the monetary value of a QALY based on estimates of the willingness to pay (WTP). This paper conducts a review of the literature on the WTP for a QALY. In total, 24 studies containing 383 unique estimates of the WTP for a QALY are identified. Trimmed mean and median estimates amount to 74,159 and 24,226 Euros (2010 price level), respectively. In regression analyses, the results indicate that the WTP for a QALY is significantly higher if the QALY gain comes from life extension rather than quality of life improvements. The results also show that the WTP for a QALY is dependent on the size of the QALY gain valued. Copyright © 2014 John Wiley & Sons, Ltd.     

Projected Cost-Effectiveness of Primary Angioplasty for Acute Myocardial Infarction

Objectives. This study sought to evaluate the cost-effectiveness of primary angioplasty for acute myocardial infarction under varying assumptions about effectiveness, existing facilities and staffing and volume of services.

Background. Primary angioplasty for acute myocardial infarction has reduced mortality in some studies, but its actual effectiveness may vary, and most U.S. hospitals do not have cardiac catheterization laboratories. Projections of cost-effectiveness in various settings are needed for decisions about adoption.

Methods. We created a decision analytic model to compare three policies: primary angioplasty, intravenous thrombolysis and no intervention. Probabilities of health outcomes were taken from randomized trials (base case efficacy assumptions) and community-based studies (effectiveness assumptions). The base case analysis assumed that a hospital with an existing laboratory with night/weekend staffing coverage admitted 200 patients with a myocardial infarction annually. In alternative scenarios, a new laboratory was built, and its capacity for elective procedures was either 1) needed or 2) redundant with existing laboratories.

Results. Under base case efficacy assumptions, primary angioplasty resulted in cost savings compared with thrombolysis and had a cost of $12,000/quality-adjusted life-year (QALY) saved compared with no intervention. In sensitivity analyses, when there was an existing cardiac catheterization laboratory at a hospital with ≥200 patients with a myocardial infarction annually, primary angioplasty had a cost of <$30,000/QALY saved under a wide range of assumptions. However, the cost/QALY saved increased sharply under effectiveness assumptions when the hospital had <150 patients with a myocardial infarction annually or when a redundant laboratory was built.

Conclusions. At hospitals with an existing cardiac catheterization laboratory, primary angioplasty for acute myocardial infarction would be cost-effective relative to other medical interventions under a wide range of assumptions. The procedure’s relative cost-ineffectiveness at low volumes or redundant laboratories supports regionalization of cardiac services in urban areas. However, approaches to overcoming competitive barriers and close monitoring of outcomes and costs will be needed.     

The impact of NHS expenditure on health outcomes in England: Alternative approaches to identification in all‐cause and disease specific models of mortality

Several recent studies have estimated the responsiveness of mortality to English National Health Service spending. Although broadly similar, the studies differ in how they identify the outcome equation. One approach uses conventional socio‐economic variables as instruments for endogenous health care expenditure, whereas the other exploits exogenous elements in the resource allocation formula for local budgets. The former approach has usually been applied to specific disease areas (e.g., for cancer and circulatory disease), whereas the other has only been applied to all‐cause mortality. In this letter, we compare the two approaches by using them to estimate the direct all‐cause elasticity as well as disease‐specific elasticities. We also calculate the implied all‐cause elasticity associated with the disease‐specific results. We find that the “funding rule” approach to identification can be successfully replicated and applied to disease area models. This is important because disease area models reduce the danger of aggregation bias present in all‐cause analysis, and they offer the opportunity to link estimated mortality effects to more complete measures of health outcome that reflect what is currently known about the survival and morbidity disease burden in different programmes.