Some reflections on cost-effectiveness analysis.
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Biomedical subjects
Publications and source records attributed to M Johannesson.
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In this paper, we develop a simple model of the benefits and costs of being on a waiting list. The model shows that complex factors are in operation, implying that a shorter waiting time need not necessarily be preferred to a longer waiting time. We also present an empirical study, where a sample of Swedes are offered the possibility of purchasing private insurance, thus reducing waiting time for surgery beyond the three-month guarantee offered by the public sector health care system. Respondents could choose between two insurance contracts. A 'spike' model, where the probability of a zero WTP is strictly positive, was developed and estimated to obtain demand functions for private insurance.
This study examined the sensitivity towards increases in user charges for different types of drugs and among different socio-economic groups. It was based on responses by 2008 consumers of prescription drugs to a self-administered postal questionnaire sent to a random sample of 8000 inhabitants in Uppsala County in Sweden. The questionnaire included a question about whether the respondents would use fewer prescription drugs if the user charges increased by a specific amount. The increase in user charges was varied between 9 and 150% in five different subsamples. Logistic regression analysis was used to estimate the probability that a respondent would reduce consumption of prescription drugs as a function of the size of the user charges increase, socio-economic characteristics and the type of drug used. Results showed that the price sensitivity decreased with increasing age, income, education and self-rated health status. Price sensitivity was highest for antitussives and lowest for climacteric drugs. If the user charges doubled, 40% of antitussives users would reduce their consumption whereas only 11% of climacteric drugs users would reduce their consumption. It is concluded that sensitivity to increases in user charges varied greatly between different types of drugs and between socio-economic groups. The young, those with poor health status, low education and low income are most likely to decrease consumption of prescription drugs when user charges increase.
Traditionally, economic evaluations in terms of cost-effectiveness analysis are based, explicitly or implicitly, on the assumption of constant returns to scale. This assumption has been criticized in the literature and the role of cost-effectiveness as a tool for decision making has been questioned. In this paper we analyze the case of increasing returns to scale due to fixed capital costs. Cost-effectiveness analysis is regarded as a tool for estimating a cost function. To this cost function two types of decision rules can be applied, the budget approach and the constant price approach. It is shown that in the presence of fixed capital costs the application of these two decision rules to a specific patient group will give different results. The budget approach may lead to suboptimizations, while using the price as a decision rule will give optimal solutions. With fixed capital costs and when an investment can be used for treating several patient groups, these groups are no longer independent. Therefore the cost-effectiveness analysis has to be performed simultaneously for all patient groups that are potential users of the investment.
The purpose of the study was to investigate psychosocial issues concerning puberty and motherhood among CF adult females, to see how they had obtained and conceived information on these matters and how they would like information to be given. Fourteen adult CF females were interviewed. The majority of the women felt socially accepted and did not remember being ashamed over their delayed puberty. Thirteen of the women had been or were living in stable sexual relationships. However, the study revealed problems with destructive behaviour during puberty due to thoughts about premature death, secret worries over delayed puberty, poorly received information about puberty and fertility, avoidance of close relationships with the opposite sex during adolescence and concerns about being a mother with a chronic illness. Information about puberty and fertility should be given individually and in small discussion groups with teenage girls combined with thorough medical and psychological guidance concerning motherhood.
BACKGROUND: In asthma, a substantial impact of disease is on quality of life rather than survival. To date, the quality of life effects of asthma have not been quantitated. OBJECTIVE: The purpose of this study was to quantitate the quality of life effects of asthma via measurement of the health utility of asthmatic patients, and the willingness to pay for an asthma cure. A second goal was to analyze how these measures are related to the different dimensions of quality of life as measured by general and disease specific quality of life instruments. METHODS: Health utilities were measured on a scale between 0 and 1 using the rating scale, time trade off, and standard gamble methods. Willingness to pay was elicited using both the dichotomous choice and the bidding game approach. Quality of life was assessed using both a generic instrument (the SF-36) and a disease-specific instrument (the Asthma TyPE). RESULTS: Sixty-nine patients with asthma were surveyed. The mean health utility was 0.68 with the rating scale method, 0.89 with the time trade off, and 0.91 with the standard gamble. On average, patients were willing to pay between $200 to $350 dollars more per month for an asthma cure. Nearly all correlations between dimensions of quality of life, health state utilities, and willingness to pay were in the expected direction. CONCLUSION: The two quality of life instruments performed about equally well in terms of being correlated with and being able to explain the responses to the health state utility questions and the willingness to pay questions. Correlations between the dimensions of the Asthma TyPE and the SF-36 were also fairly high. Further work should focus on validating and refining the different methods of quantitating quality of life for asthma patients.
How to obtain confidence intervals for cost-effectiveness ratios is complicated by the statistical problems of obtaining a confidence interval for a ratio of random variables. Different approaches have been suggested in the literature, but no consensus has been reached. We propose an alternative simple solution to this problem. By multiplying the effectiveness units by the price per effectiveness unit, both costs and benefits can be expressed in monetary terms and standard statistical techniques can be used to estimate a confidence interval for net benefits. This approach avoids the ratio estimation problem and explicitly recognizes that the price per effectiveness unit has to be known to provide cost-effectiveness analysis with a useful decision rule.
BACKGROUND: The public's perception of autologous blood donation and transfusion as a worthwhile alternative to allogeneic blood transfusion increased dramatically with discovery of the human immunodeficiency virus. However, new concerns are being raised about the health outcomes and cost-effectiveness of the procedure. As more restrictive guidelines for autologous blood donation evolve, opposition from patients concerned about exposure to allogeneic blood may arise. Physicians' ability to reassure patients and garner their support for more restrictive policies requires an understanding of patients' concerns. The motivations, perceptions, and preferences of patients currently participating in autologous blood donation programs were investigated in this study. STUDY DESIGN AND METHODS: Results from two questionnaire studies of 647 autologous blood donors are presented. The questionnaires assessed demographics, risk perceptions, preferences, willingness to pay, and reactions to different interventions designed to decrease patient preference for autologous blood donation. RESULTS: Patients expressed a strong preference for the availability of autologous blood and indicated that they would be willing to pay substantial amounts of money even if the procedure were not covered by insurance. Despite education about the low risks of complications from allogeneic transfusions, an aversion to allogeneic transfusion and a willingness to pay for autologous blood donation persisted. Patients were not reassured by information on better infectious disease testing or physician recommendation against autologous blood donation. CONCLUSION: Patients currently participating in autologous blood donor programs strongly prefer continued access to this procedure, primarily because they remain concerned about the complications of allogeneic transfusions. They may not be significantly reassured despite increasingly rigorous and costly improvements in donor and component screening.
Ten women with cystic fibrosis (CF) were evaluated with regard to hormonal profiles during a natural and a clomiphene citrate (CC) stimulated cycle. Five of the women were found to be anovulatory during a natural cycle. All women except one did respond with ovulation to CC stimulation indicating adequate ovarian response. Neither did they show increased follicle-stimulating hormone (FSH) concentrations on day 10 after CC treatment confirming normal ovarian reserve. Clinically the anovulatory women differed from the ovulating in two aspects: more profound essential fatty acid deficiency (EFAD) and higher peak/basal insulin response during an oral glucose tolerance test. The anovulatory women had significantly lower luteal oestradiol and progesterone but higher total testosterone concentrations when compared to healthy controls and the ovulatory CF women. The pathological insulin response and high testosterone concentrations resemble those seen in women with polycystic ovarian (PCO) syndrome. However, the CF patients in our study had normal ovaries, as deduced from ultrasound examination and normal luteinizing hormone (LH)/FSH ratio. It is suggested that EFAD as well as hypersecretion of insulin may be of importance for the observed ovarian dysfunction. Further studies are needed to evaluate the relation between ovulatory mechanisms and EFAD in CF women as well as studies to compare anovulatory CF women with women with PCO syndrome.
OBJECTIVES: For contingent valuation to provide valid values for policy making, it is important that respondents be well informed about the goods they are asked to value. Few studies, however, have tested the impact of providing this information This study assessed the impact of risk information on patients' willingness to pay for autologous blood donation and derived the willingness to pay in a sample of informed patients. METHODS: Patients were randomized either to receive information about the risks of complications from allogeneic (volunteer) blood transfusions or to base their willingness to pay responses on their own prior knowledge. Four hundred twelve autologous blood donors were recruited from three study sites. Self-administered questionnaires collected information on willingness to pay, risk perceptions, and socioeconomic information. RESULTS: As predicted by our theoretical model, providing risk information reduced the variance in the willingness to pay for autologous blood donation. A tendency for information to reduce the willingness to pay was also found, suggesting that uninformed patients, on average, overestimate the risks of allogeneic blood transfusions. The median willingness to pay in the informed sample was approximately $750 to $1,100, depending on the estimation method, compared with $800 to $1,900 in the uninformed group. Willingness to pay was significantly related to perceived transfusion risk, personal income, and dread of transfusions. CONCLUSIONS: Our results are consistent with an economic model where individuals update their prior risk perceptions with new information. The willingness to pay in the informed sample was far higher than the costs of autologous blood donation, suggesting that total benefits outweigh the costs of the procedure.
Standard price indices may not account for the price decrease that results when patients switch to pharmaceutical products for which their demand-reservation price exceeds the price of the new product. In a similar way, standard price indices may not account for the price increase that results when patients switch to pharmaceuticals for which the reservation price is below the price of the new drug. This study was designed to assess whether standard pharmaceutical price indices under- or overestimate the real price development resulting from new drug introductions. We illustrate that both situations may occur. In our first example, the switch from a branded to a generic drug, we showed that standard price indices recorded a price increase of about 16%, although the price actually decreased by about 5%. In our second example, the introduction of new formulation of a currently marketed drug, we showed that standard price indices recorded a price increase of about 6%, when the actual price increased by more than 65%.
This paper investigates the effects of changes in drug therapy on drug expenditure in Sweden between 1990 and 1995. Analyses have been carried out for both the aggregate drug expenditure and for drug expenditure according to the main groups of the Anatomical Therapeutic Chemical (ATC) classification system. Changes in expenditure can be divided into 3 components: the price of drugs, the quantity of drugs consumed and a residual. The size of the residual is a measure of the effect of changes in drug treatment patterns on drug expenditure. The aggregate real drug expenditure increased by 50% between 1990 and 1995. The relative retail price index of drugs decreased by 9% and the quantity index measured in terms of defined daily doses increased by 27%. The remaining residual increased by 30%. This implies that, if the residual had been unchanged during the study period, the aggregate expenditure would have increased by only 15%. The results also show that the size of the residual varies substantially across different ATC groups. The increase in the residual was largest for drugs that affect the nervous system (ATC group N); the residual increased by 86%. From the observed changes in residual values, it can be concluded that the underlying factor responsible for the increase in drug expenditure in Sweden is changes in drug therapy from less expensive to more expensive drugs.
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Multidrug resistance (MDR1b) and cystic fibrosis transmembrane conductance regulator (CFTR) proteins are members of the "ATP-binding cassette" superfamily of transporters. They are associated with chloride channel activities and ATP secretion and have complementary patterns of expression in several organs. In the rat uterus, CFTR expression is replaced by MDR1b expression during pregnancy. We have studied whether expression of MDR1b and CFTR also vary in the lung during pregnancy. No variations in MDR1b or CFTR mRNA levels during pregnancy were detected. However, there was an unusual degree of variation in MDR1b mRNA expression in lung parenchyma between animals in both the control group and the pregnant group. If present among humans, polymorphic expression of MDR1 in lung parenchyma may explain part of the differences in lung symptomatology observed in the CF patients carrying the same mutation.
BACKGROUND: The Scandinavian Simvastatin Survival Study (4S) showed that lowering cholesterol levels with simvastatin reduces mortality and morbidity in patients with angina pectoris or previous acute myocardial infarction. Before the widespread use of cholesterol-lowering drugs in such patients is recommended, its cost effectiveness should be demonstrated. We estimated the cost effectiveness of simvastatin treatment to lower cholesterol levels in relation to the age, sex, and cholesterol level before treatment of patients with coronary heart disease. METHODS: We estimated the cost per year of life gained with simvastatin therapy. To model the increased life expectancy, hazard functions from 4S were used. The costs studied included those of the intervention and the direct and indirect costs associated with morbidity from coronary causes. We prepared separate estimates for men and women at various ages (from 35 to 70 years) and total cholesterol levels before treatment (213 to 309 mg per deciliter). RESULTS: In the analysis limited to direct costs, the cost of each year of life gained ranged from $3,800 for 70-year-old men with 309 mg of cholesterol per deciliter to $27,400 for 35-year-old women with 213 mg of cholesterol per deciliter. When we included indirect costs, the results ranged from a savings in the youngest patients to a cost of $13,300 per year of life gained in 70-year-old women with 213 mg of cholesterol per deciliter. CONCLUSIONS: In patients with coronary heart disease, simvastatin therapy is cost effective among both men and women at the ages and cholesterol levels studied.
Cystic fibrosis transmembrane conductance regulator (CFTR) mRNA expression has been found in the medial preoptic area using in situ hybridization, addressing the possibility of CFTR regulation of sexual maturation and reproductive behaviour. CFTR mRNA has also been found in the cortical deep pyramidal layer V implying possible involvement of CFTR in 'motor' function and output control over bodily movements and secretion. CFTR production in the brain regions observed in this study implicate involvement of CFTR in cerebral control over motor/visceral and endocrine systems.
This paper compares the relative performance of quality adjusted life years (QALYs) based on quality weights elicited by rating scale (RS), time trade-off (TTO) and standard gamble (SG). The standard against which relative performance is assessed is individual preference elicited by direct ranking. The correlation between predicted and direct ranking is significantly higher for TTO-QALYs than for RS-QALYs and SG-QALYs. This holds both based on mean Spearman rank correlation coefficients calculated per individual and based on two social choice rules: the method of majority voting and the Borda rule. Undiscounted TTO-QALYs are more consistent with direct ranking than discounted TTO-QALYs.
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