Cost-Effectiveness Methods and Newborn Screening Assessment.

Nowadays, health funding decisions must be supported by sound arguments in terms of both effectiveness and economic criteria. After more than half a century of newborn screening for rare diseases, the appropriate economic evaluation framework for these interventions is still challenging. The validity of standard methods for economic evaluation heavily relies on the availability of robust evidence, but collection of such evidence is precluded by the rareness of the conditions that may benefit from screening. Furthermore, there are a series of conceptual and methodological limitations that warrant further careful consideration when assessing the cost-effectiveness of newborn screening programs. In this chapter we provide a general overview of current economic evaluation methods and the challenges for their application to newborn screening programs.

Social/economic costs and health-related quality of life in patients with juvenile idiopathic arthritis in Europe.

OBJECTIVE: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with juvenile idiopathic arthritis (JIA) in Europe. METHODS: We conducted a cross-sectional study of patients with JIA from Germany, Italy, Spain, France, the United Kingdom, Bulgaria, and Sweden. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D-5L) questionnaire. RESULTS: A total of 162 patients (67 Germany, 34 Sweden, 33 Italy, 23 United Kingdom, 4 France, and 1 Bulgaria) completed the questionnaire. Excluding Bulgarian results, due to small sample size, country-specific annual health care costs ranged from €18,913 to €36,396 (reference year: 2012). Estimated direct healthcare costs ranged from €11,068 to €22,138; direct non-healthcare costs ranged from €7837 to €14,155 and labor productivity losses ranged from €0 to €8715. Costs are also shown to differ between children and adults. The mean EQ-5D index score for JIA patients was estimated at between 0.44 and 0.88, and the mean EQ-5D visual analogue scale score was estimated at between 62 and 79. CONCLUSIONS: JIA patients incur considerable societal costs and experience substantial deterioration in HRQOL in some countries. Compared with previous studies, our results show a remarkable increase in annual healthcare costs for JIA patients. Reasons for the increase are the inclusion of non-professional caregiver costs, a wider use of biologics, and longer hospital stays.

Review of economic studies and budget impact analysis of ocriplasmin as a treatment of vitreomacular traction. / Revisión de estudios económicos y análisis de impacto presupuestario de la ocriplasmina como tratamiento de la tracción vitreomacular.

OBJECTIVE: To review the evidence on the cost-effectiveness of ocriplasmin as a treatment for vitreomacular traction (VMT), and to estimate the impact on the Spanish National Health System (NHS). MATERIAL AND METHODS: 1) Systematic review. The following databases were searched in January 2015: MEDLINE, PREMEDLINE, EMBASE, CRD, the Cochrane Library, and key websites. Selection criteria were: full economic evaluations that compared ocriplasmin with usual care ('watch and wait' and/or vitrectomy) in patients with VMT. The outcomes to extract were costs of the alternatives and the incremental cost-effectiveness ratio. Studies of budget impact analysis were also included. The methodological quality was assessed, and a narrative synthesis of the included studies was carried out. 2) Estimation of budget impact. The impact on the budget as a result of the introduction of ocriplasmin in the NHS was estimated, including data from different sources. RESULTS: Six studies were identified, none of them performed in Spain. The two best studies concluded that ocriplasmin is cost-effective in their respective countries (Canada and United Kingdom), but only in patients with certain conditions (without epiretinal membrane, for example). The results of the budget impact analysis are different between countries. The analysis for Spain showed that the introduction of ocriplasmin would mean a saving over 1 million Euros for the NHS in 5 years. CONCLUSIONS: The cost-effectiveness of ocriplasmin has not been demonstrated in Spain. However, good studies performed in other countries found that ocriplasmin is cost-effective in selected patients. Given the current prices in Spain, ocriplasmin could involve a saving for the Spanish NHS.

Costs resulting from premature mortality due to cardiovascular causes: A 20-year follow-up of the DRECE study. / Mortalidad prematura cardiovascular y sus costes: 20 años de seguimiento del estudio DRECE.

OBJECTIVES: Cardiovascular diseases are still the leading cause of death in Spain. The DRECE study (Diet and Cardiovascular Disease Risk in Spain), based on a representative cohort of the Spanish general population, analyzed nutritional habits and lifestyle and their association with morbidity and mortality patterns. We estimated the impact, in terms of loss of productivity, of premature mortality attributed to cardiovascular diseases. METHODS: The loss of productivity attributed to premature mortality was calculated from 1991, based on the potential years of life lost and the potential years of working life lost. RESULTS: During the 20-year follow-up of a cohort of 4779 patients, 225 of these patients died (men, 152). Sixteen percent of the deaths were attributed to cardiovascular disease. The costs due to lost productivity by premature mortality exceeded 29 million euros. Of these, 4 million euros (14% of the total cost) were due to cardiovascular causes. CONCLUSIONS: Premature cardiovascular mortality in the DRECE cohort represented a significant social cost due to lost productivity.

Costs of QuantiFERON-TB Gold versus tuberculin skin test in Spanish healthcare workers.

We compared the cost of the tuberculin skin test (TST) with the QuantiFERON-TB Gold (QFT-G) test when screening for latent tuberculosis in 134 healthcare workers in Spain. The QFT-G test cost euro42.5 per healthcare worker, while the TST cost euro39.5. The tests varied in cost structure; most (70%) TST costs were due to time demands on the participants, whereas the QFT-G was more expensive in terms of consumables (50% of the total cost). Accordingly, the results depend on the hourly wages of the participants and the time they must dedicate to the tests. In the Spanish healthcare system, the societal costs of QFT-G are comparable to those of the TST, although their cost structures are quite distinct.

A systematic review of specialised palliative care for terminal patients: which model is better?

There is evidence of improved effectiveness of specialised palliative care for terminally ill patients in comparison to conventional care. However, there is uncertainty about which model is better. The objective of this systematic review was to identify studies that compare specialised palliative care models between them assessing their effectiveness or cost-effectiveness. We searched studies published between 2003 and 2006 in several electronic databases and updated the search in MEDLINE up to 2008. Papers published before 2003 were identified by means of previous systematic reviews and manual search. Studies with broad designs comparing two or more specialised palliative care programmes in adults with terminal illness were selected. Six systematic reviews, three studies on effectiveness and one cost study were included. All systematic reviews drew the conclusion that specialised palliative care is more effective than conventional care. The methodological limitations of the original studies and the heterogeneity of programmes did not allow to draw conclusions about whether a specific model of specialised palliative care is more or less effective or cost-effective than other.

Impact on health-related quality of life and perceived burden of informal caregivers of individuals with Alzheimer's disease.

This study assessed the impact on health-related quality of life (HRQL) and the perceived burden of informal caregivers of individuals with Alzheimer's disease (AD) on the Canary Islands (Spain). We utilized a multicenter, cross-sectional design, based on questionnaire responses of 237 informal caregivers of AD patients. Patients were classified according to the degree of severity utilizing the Clinical Dementia Rating Scale. Sociodemographic, HRQL (EQ-5D) and functional dependency data were gathered together with the degree of caregiver burden. Caregivers had a higher frequency of problems than did the general population for every EQ-5D dimension. Caregivers' HRQL was inversely associated with the subject dependency level and caregiver age. HRQL was higher for more educated caregivers. Variables with a negative and/or significant effect on caregivers' HRQL were high feelings of burden, more committed time to care, and older age. The caregiver burden quantified by the Zarit scale showed 83.3% of caregivers with a high level of burden.

[Socio-economic costs of mental health in the Canary Islands, Spain, in 2002]. / Costes socioeconómicos de las enfermedades mentales en las Islas Canarias en 2002.

OBJECTIVE: To evaluate the economic impact in terms of direct and indirect costs of the mental health in Canary Islands (Spain) in 2002. DESIGN: The cost-of-illness method was used. Direct and indirect costs were estimated using prevalence cost, i.e., the costs produced in 2002. The human capital theory approach has been used. SETTING: Canary Islands, Spain, including primary health care and inpatient care. PARTICIPANTS: Mental health patients. MAIN MEASUREMENTS: Direct health costs (inpatients, ambulatory care, primary health care, and drugs). Indirect costs (premature death, short-term illness, and permanent disability). RESULTS: The total costs of mental health were 189.59 million euros. The direct health costs were 81.67 million euros, constituting 43% of the total costs and 5.2% of the total public health care budget in this region. The indirect costs of mental health were 107.92 million euros, representing 57% of the total costs. CONCLUSIONS: Although this study adopts a conservative approach, the high socio-economic cost of the mental health helps us to define better the dimension of the problem to establish priorities besides opening a way towards cost-effectiveness studies that allow a more transparent debate on this topic.

Preoperative assessment.

Although anaesthetic and surgical procedures should be individualised for every patient, in practice many preoperative protocols and routines are used generally. In this article, we aim to emphasise: why preoperative assessment is important; how it should be done, and by whom; what can be expected; and the importance of test selection based on patients' needs and on scientific evidence of effectiveness. We outline the roles of preoperative medical assessment in otherwise healthy patients. Clinical history, preoperative questionnaires, physical examination, routine tests, individual risk-assessment, and fasting policies are investigated by review of published work. Cost of routine preoperative assessment, the anaesthetist's legal responsibility, and patients'views in the preoperative process are also considered. A thorough clinical preoperative assessment of the patient is more important than routine preoperative tests, which should be requested only when justified by clinical indications. Moreover, this practice eliminates unnecessary cost without compromising the safety and quality of care. Education and training of medical doctors should be more scientifically guided, emphasising the relevance of effectiveness, and cost-effectiveness in clinical decision-making and complemented by audit.

[Socioeconomic costs of cardiovascular disease and cancer in the Canary Islands (Spain) in 1998]. / Los costes socioeconómicos de las enfermedades cardiovasculares y del cáncer en las Islas Canarias en 1998.

OBJECTIVE: To evaluate the economic impact in terms of direct and indirect costs of the cardiovascular disease and cancer in the Canary Islands in 1998. METHODS: The cost-of-illness method was used. Direct and indirect costs were estimated using prevalence costs, i.e., the costs produced in 1998. Direct costs were divided into hospitalization costs, outpatient costs, primary health care costs, and drug costs while indirect costs were obtained through transformation of physical units into monetary units using the approach of human capital theory and the friction cost method. RESULTS: The total costs of cardiovascular disease and cancer were 246.11 and 193.72 million euros respectively. The direct costs of the two diseases were 134.44 and 58.04 million euros respectively, representing 55% and 30% of total costs and 16% of total health care expenditure in this region. The indirect costs of these two diseases were 111.68 and 135.68 million euros respectively, representing 45% and 70% of total costs. Use of the friction cost method revealed that indirect costs decreased by 88% for cardiovascular disease and those for cancer decreased by 77%. CONCLUSIONS: Although this study adopts a conservative approach by omitting costs associated with pain and suffering, permanent disability, and those of at-home care provided by the family, the annual socioeconomic cost of cardiovascular disease and cancer in the Canary Islands was high, amounting to 440 million euros.

[Cost analysis and potential savings related to the use of preoperative tests in the hospitals of the Canary Islands [Spain]]. / Análisis de costes y ahorros potenciales relacionados con la utilización de pruebas preoperatorias en los hospitales de Canarias.

OBJECTIVE: To estimate the potential cost savings that might take place in the Canary Islands Health Service (CHS) through the appropriate scientific use of preoperative tests, using information obtained from five public university hospitals. METHODS: A questionnaire was completed by 55 anesthesiologists, representing 60% of the total in the CHS. The questionnaire paid special attention to the most frequently used preoperative tests: chest radiograph, electrocardiogram, laboratory tests, and spirometry. The mean unit costs for the various preoperative tests were obtained from the two hospitals participating in the study. To calculate the potential cost savings derived from the appropriate use of preoperative tests, several scenarios were considered. These were characterized by different hypotheses or degrees of fulfillment of a protocol based on scientific knowledge and considered as the gold standard. RESULTS: In the ideal scenario in which the recommended scientific protocol was fulfilled in 100% of the 16 179 patients with an American Society of Anesthesiologists (ASA) grade of I-II, the economic impact would be notable, since it would free sensitive resources that could be used for other health programs. These figures could amount to approximately 1.02 million euros, without considering the cost of preoperative hospital stay. This figure could increase by up to approximately 2.13 million euros if one day of preoperative hospital stay were included and by up to 3.24 million euros if two days of preoperative hospital stay were included. CONCLUSIONS: The recent literature review and the results of the questionnaire applied in the CHS indicate that preoperative tests are of greatest benefit to patients and to society if their use is guided by scientific knowledge. In addition, resources can be freed by better selection and utilization of preoperative tests.

[The social and economic cost of diabetes mellitus]. / Los costes socioeconómicos de la diabetes mellitus.

OBJECTIVE: To evaluate the economic impact in terms of the direct costs (health care costs) and the indirect costs (effects on production caused by mortality and morbidity) of diabetes mellitus (DM) in the Canary Islands (Spain) during 1998. DESIGN: The approach used was to study the cost of the disease through looking only at people in the productive sphere (human capital). Direct and indirect costs were calculated by using costs of prevalence, i.e. the costs caused during 1998. SETTING: Direct costs were broken down into hospital care, primary care, out-patient consultations, drugs and further tests; indirect costs, into premature mortality and time off work. Measurements and main results. The total cost of DM amounted to 6468.76 million pesetas (38.88 millio ), or the equivalent of 126 168 pesetas (758.28 ) a year for each known diabetic patient. Total direct cost of DM was 4011.51 million pesetas (24.11 million ), which amounts to 2.13% of health expenditure in the Canaries; or the equivalent of 78 240 pesetas (470.23 ) a year for each known diabetic patient. Direct cost of health care was 62% of the total cost. Total indirect cost was 2457.25 million pesetas (14.77 million ), 38% of the total cost. CONCLUSIONS: Despite the conservatism of the approach, in that the study omitted costs linked to pain and suffering, permanent disability or home care by family members, the high social and economic cost of DM patients calls for cost-effectiveness studies that would permit a more transparent debate on this question.