Tenosynovial Giant Cell Tumor Observational Platform Project (TOPP) Registry: A 2-Year Analysis of Patient-Reported Outcomes and Treatment Strategies.

BACKGROUND: The Tenosynovial giant cell tumor Observational Platform Project (TOPP) registry is an international prospective study that -previously described the impact of diffuse-type tenosynovial giant cell tumour (D-TGCT) on patient-reported outcomes (PROs) from a baseline snapshot. This analysis describes the impact of D-TGCT at 2-year follow-up based on treatment strategies. MATERIAL AND METHODS: TOPP was conducted at 12 sites (EU: 10; US: 2). Captured PRO measurements assessed at baseline, 1-year, and 2-year follow-ups were Brief Pain Inventory (BPI), Pain Interference, BPI Pain Severity, Worst Pain, EQ-5D-5L, Worst Stiffness, and -Patient-Reported Outcomes Measurement Information System. Treatment interventions were no current/planned treatment (Off-Treatment) and systemic treatment/surgery (On-Treatment). RESULTS: A total of 176 patients (mean age: 43.5 years) were included in the full analysis set. For patients without active treatment strategy -(Off-Treatment) at baseline (n = 79), BPI Pain Interference (1.00 vs. 2.86) and BPI Pain Severity scores (1.50 vs. 3.00) were numerically favorable in patients remaining Off-Treatment compared with those who switched to an active treatment strategy at year 1. From 1-year to 2-year -follow-ups, patients who remained Off-Treatment had better BPI Pain Interference (0.57 vs. 2.57) and Worst Pain (2.0 vs. 4.5) scores compared with patients who switched to an alternative treatment strategy. In addition, EQ-5D VAS scores (80.0 vs. 65.0) were higher in patients who remained -Off-Treatment between 1-year and 2-year follow-ups compared with patients who changed treatment strategy. For patients receiving systemic treatment at baseline, numerically favorable scores were seen in patients remaining on systemic therapy at 1-year follow-up: BPI Pain Interference (2.79 vs. 5.93), BPI Pain Severity (3.63 vs. 6.38), Worst Pain (4.5 vs. 7.5), and Worst Stiffness (4.0 vs. 7.5). From 1-year to 2-year follow-up, EQ-5D VAS scores (77.5 vs. 65.0) were higher in patients who changed from systemic treatment to a different treatment strategy. CONCLUSION: These findings highlight the impact D-TGCT has on patient quality of life, and how treatment strategies may be influenced by these outcome measures. (ClinicalTrials.gov number: NCT02948088).

A prospective real-world study of the diffuse-type tenosynovial giant cell tumor patient journey: A 2-year observational analysis.

BACKGROUND AND OBJECTIVES: Diffuse-tenosynovial giant cell tumor (D-TGCT) is a rare, locally aggressive, typically benign neoplasm affecting mainly large joints, representing a wide clinical spectrum. We provide a picture of the treatment journey of D-TGCT patients as a 2-year observational follow-up. METHODS: The TGCT Observational Platform Project registry was a multinational, multicenter, prospective observational study at tertiary sarcoma centers spanning seven European countries and two US sites. Histologically confirmed D-TGCT patients were categorized as either those who remained on initial treatment strategy (determined at baseline visit) or those who changed treatment strategy with specific changes documented (e.g., systemic treatment to surgery) at the 1-year and/or 2-year follow-up visits. RESULTS: A total of 176 patients were assessed, mean diagnosis age was 38.4 (SD ± 14.6) years; most patients had a knee tumor (120/176, 68.2%). For the 2-year observation period, most patients (75.5%) remained on the baseline treatment strategy throughout, 54/79 patients (68.4%) remained no treatment, 30/45 patients (66.7%) remained systemic treatment, 39/39 patients (100%) remained surgery. Those who changed treatment strategy utilized multimodal treatment options. CONCLUSIONS: This is the first prospectively collected analysis to describe D-TGCT patient treatments over an extended follow-up and demonstrates the need for multidisciplinary teams to determine an optimal treatment strategy.

Can the Consideration of Societal Costs Change the Recommendation of Economic Evaluations in the Field of Rare Diseases? An Empirical Analysis.

OBJECTIVES: To analyze whether the adoption of a societal perspective would alter the results and conclusions of economic evaluations for rare disease-related healthcare technologies. METHODS: A search strategy involving all the active substances considered as orphan drugs by the European Medicines Agency plus a list of 76 rare diseases combined with economic-related terms was conducted on Medline and the Cost-Effectiveness Registry from the beginning of 2000 until November 2018. We included studies that considered quality-adjusted life years as an outcome, were published in a scientific journal, were written in English, included informal care costs or productivity losses, and separated the results according to the applied perspective. RESULTS: We found 14 articles that fulfilled the inclusion criteria. Productivity losses were considered in 12 studies, the human capital approach being the method most frequently used. Exclusively, informal care was considered in 2 articles, being valued through the opportunity cost method. The 14 articles selected resulted in 26 economic evaluation estimations, from which incremental cost-utility ratio values changed from cost-effective to dominant in 3 estimates, but the consideration of societal costs only modified the authors' conclusion in 1 study. CONCLUSIONS: The presence of societal costs in the economic evaluation of rare diseases did not affect the conclusions of the studies except in a single specific case. In those studies where the societal perspective was considered, we did not find significant changes in the economic evaluation results due to the higher costs of treatments and the low quality-adjusted life-years gained.

Socioeconomic costs and health-related quality of life in juvenile idiopathic arthritis: a cost-of-illness study in the United Kingdom.

BACKGROUND: Juvenile idiopathic arthritis (JIA) refers to a number of rare chronic inflammatory diseases. Although JIA imposes a significant societal burden, limited data are available on the cost of JIA. The study's objective is to quantify the socioeconomic burden of JIA patients in the United Kingdom (UK), along with their health-related quality of life (HRQoL). METHODS: A bottom-up, cross-sectional, cost-of-illness analysis of 23 patients was carried out. To collect data on demographic characteristics, health resource utilization, informal care, productivity losses and HRQoL, questionnaires were administered to and completed by patients or their caregivers. The EuroQol five dimensions (EQ-5D) instrument was used to measure HRQoL. RESULTS: This study found that the average annual cost for a JIA patient was €31,546, with direct health care costs equalling €14,509 (46.0 % of total costs), direct non-health care costs amounting to €8,323 (26.4 %) and productivity losses being €8,715 (27.6 %). This was calculated using unit costs for 2012. The largest expenditures on average were accounted for by early retirement (27.0 %), followed by informal care (24.1 %), medications (21.1 %), outpatient and primary health care visits (13.2 %) and diagnostic tests (7.9 %). Important differences existed between JIA patients in need of caregiver assistance and those with no need (€39,469 vs. €25,452 respectively). Among adult JIA patients, mean EQ-5D index scores and visual analogue scale (VAS) scores were found to be 0.26 and 49.00 respectively; the same scores among caregivers were 0.66 and 67.14 respectively. CONCLUSION: JIA poses a significant cost burden on the UK society. Over half of the total average costs (54 %) are related to non-health care and productivity losses. HRQoL of JIA patients is considerably worse than the UK general population.

Social and economic costs and health-related quality of life in non-institutionalised patients with cystic fibrosis in the United Kingdom.

BACKGROUND: This study aimed to determine the societal economic burden and health-related quality of life (HRQOL) of cystic fibrosis (CF) patients in the UK. METHODS: A bottom-up cost-of-illness, cross-sectional, retrospective analysis of 74 patients was conducted aiming to estimate the economic impact of CF. Data on demographic characteristics, health resource utilisation, informal care, productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) instrument. RESULTS: Using unit costs for 2012 we found that the average annual cost for a CF patient was €48,603, with direct health care costs amounting to €20,854 (42.9 % of total costs), direct non-health care costs being €21,528 (44.3 %) and indirect costs attributable to productivity losses being €6,222 (12.8 %). On average, the largest expenditures by far were accounted for by informal care (44.1 %), followed by medications (14.5 %), acute hospitalisations (13.9 %), early retirement (9.1 %) and outpatient and primary health care visits (7.9 %). Sharp differences existed depending on whether CF patients were in need of caregiver help (€76,271 versus €26,335). In adult CF patients, mean EQ-5D index scores were 0.64 (0.93 in the general population) and mean EQ-5D visual analogue scale scores were 62.23 (86.84 in the general population); among caregivers, these scores were 0.836 and 80.85, respectively. DISCUSSION: Our analysis highlights the importance of the economic and quality of life consequences of CF from a societal perspective. The results highlight that beyond conventional costs such as acute hospitalisations, medication and outpatient and primary care visits, indirect costs related to informal care and early retirement, have significant societal implications. Similarly, our analysis showed that the average EQ-5D index score of adult CF patients was significantly lower than in the general population, an indication that a methodological bias may exist in using the latter in economic analyses. CONCLUSION: CF poses a significant cost burden on UK society, with non-health care and indirect costs representing 57 % of total average costs, and HRQOL being considerably lower than in the general population.

Economic Burden And Health-Related Quality Of Life Of Patients With Cystic Fibrosis In Bulgaria.

OBJECTIVE: The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with cystic fibrosis (CF) in Bulgaria. MATERIALS AND METHODS: We conducted a cross-sectional study of 33 patients with CF and 17 caregivers from Bulgaria. Data on socio-demographic characteristics, health resource utilisation, informal care, labor productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was evaluated with the EuroQol 5-domain (EQ-5D-3L) questionnaire. RESULTS: Median annual costs of CF in Bulgaria were € 24 152 per patient in 2012 as a reference year. Median annual costs for children were found to be significantly higher than those for adults - € 31 945 vs. € 15 714 (p = 0.012). This outcome came from statistically significant differences in costs for main informal carer (p < 0.001) and costs for other informal carers (p = 0.022). As a single cost item, drugs had the biggest monetary impact. Median annual drug costs were € 13 059. Bulgarian CF patients showed low HRQOL results - 50 median VAS score and 0.592 median health utilities. A quarter of patients even rated their health state as worse than death. CONCLUSION: CF patients from Eastern Europe remain a vulnerable population with risk factors for worse health outcomes. Our study provided a state-of-the art analysis that facilitates the elaboration, adoption and application of targeted public health policies to tackle CF-related problems at national and European level.

Costs, outcomes and challenges for diabetes care in Spain.

BACKGROUND: Diabetes is becoming of increasing concern in Spain due to rising incidence and prevalence, although little information is known with regards to costs and outcomes. The information on cost of diabetes in Spain is fragmented and outdated. Our objective is to update diabetes costs, and to identify outcomes and quality of care of diabetes in Spain. METHODS: We performed systematic searches from secondary sources, including scientific literature and government data and reports. RESULTS: Diabetes Type II prevalence is estimated at 7.8%, and an additional 6% of the population is estimated to be undiagnosed. Four Spanish diabetes cost studies were analyzed to create a projection of direct costs in the NHS and productivity losses, estimating €5.1 billion for direct costs along with €1.5 billion for diabetes-related complications (2009) and labour productivity losses represented €2.8 billion. Glycemic control (glycolysated hemoglobin) is considered acceptable in 59% of adult Type II cases, in addition to 85% with HDL cholesterol ≥40mg/dl and 65% with blood pressure <140/90 mmHg, pointing to good intermediate outcomes. However, annual figures indicate that over half of the Type II diabetics are obese (BMI >30), 15% have diabetic retinopathy, 16% with microalbuminuria, and 15% with cardiovascular disease. CONCLUSIONS: The direct health care costs (8% of the total National Health System expenditure) and the loss of labour productivity are high. The importance of a multi-sectoral approach in prevention and improvements in management of diabetes are discussed, along with policy considerations to help modify the disease course.

Social and economic costs and health-related quality of life in stroke survivors in the Canary Islands, Spain.

BACKGROUND: Cost-of-illness analysis is the main method of providing an overall vision of the economic impact of a disease. Such studies have been used to set priorities for healthcare policies and inform resource allocation. The aim of this study was to determine the economic burden and health-related quality of life (HRQOL) in the first, second and third years after surviving a stroke in the Canary Islands, Spain. METHODS: Cross-sectional, retrospective study of 448 patients with stroke based on ICD 9 discharge codes, who received outpatient care at five hospitals. The study was approved by the Research Ethics Committee of Nuestra Señora de la Candelaria University Hospital. Data on demographic characteristics, health resource utilization, informal care, labor productivity losses and HRQOL were collected from the hospital admissions databases and questionnaires completed by stroke patients or their caregivers. Labor productivity losses were calculated from physical units and converted into monetary units with a human capital-based method. HRQOL was measured with the EuroQol EQ-5D questionnaire. Healthcare costs, productivity losses and informal care costs were analyzed with log-normal, probit and ordered probit multivariate models. RESULTS: The average cost for each stroke survivor was €17 618 in the first, €14 453 in the second and €12 924 in the third year after the stroke; the reference year for unit prices was 2004. The largest expenditures in the first year were informal care and hospitalizations; in the second and third years the main costs were for informal care, productivity losses and medication. Mean EQ-5D index scores for stroke survivors were 0.50 for the first, 0.47 for the second and 0.46 for the third year, and mean EQ-5D visual analog scale scores were 56, 52 and 55, respectively. CONCLUSIONS: The main strengths of this study lie in our bottom-up-approach to costing, and in the evaluation of stroke survivors from a broad perspective (societal costs) in the first, second and third years after surviving the stroke. This type of analysis is rare in the Spanish context. We conclude that stroke incurs considerable societal costs among survivors to three years and there is substantial deterioration in HRQOL.

Assessing the Effect of Including Social Costs in Economic Evaluations of Diabetes-Related Interventions: A Systematic Review.

BACKGROUND: The economic burden of diabetes from a societal perspective is well documented in the cost-of-illness literature. However, the effect of considering social costs in the results and conclusions of economic evaluations of diabetes-related interventions remains unknown. OBJECTIVE: To investigate whether the inclusion of social costs (productivity losses and/or informal care) might change the results and conclusions of economic evaluations of diabetes-related interventions. METHODS: A systematic review was designed and launched on Medline and the Cost-Effectiveness Analysis Registry from the University of Tufts, from the year 2000 until 2018. Included studies had to fulfil the following criteria: i) being an original study published in a scientific journal, ii) being an economic evaluation of an intervention on diabetes, iii) including social costs, iv) being written in English, v) using quality-adjusted life years as outcome, and vi) separating the results according to the perspective applied. RESULTS: From the 691 records identified, 47 studies (6.8%) were selected. Productivity losses were included in 45 of the selected articles (73% used the human capital approach) whereas informal care costs in only 13 (when stated, the opportunity cost method was used in seven studies and the replacement cost in one). The 47 studies resulted in 110 economic evaluation estimations. The inclusion of social costs changed the conclusions in 8 estimations (17%), 6 of them switching from not cost-effective from the healthcare perspective to cost-effective or dominant from the societal perspective. Considering social costs altered the results from cost-effective to dominant in 9 estimations (19%). CONCLUSION: When social costs are considered, the results and conclusions of economic evaluations performed in diabetes-related interventions can alter. Wide methodological variations have been observed, which limit the comparability of studies and advocate for the inclusion of a wider perspective via the consideration of social costs in economic evaluations and methodological guidelines relating to their estimation and valuation.

The diffuse-type tenosynovial giant cell tumor (dt-TGCT) patient journey: a prospective multicenter study.

BACKGROUND: Tenosynovial giant cell tumor (TGCT) is a rare, locally aggressive neoplasm arising from the synovium of joints, bursae, and tendon sheaths affecting small and large joints. It represents a wide spectrum ranging from minimally symptomatic to massively debilitating. Most findings to date are mainly from small, retrospective case series, and thus the morbidity and actual impact of this rare disease remain to be elucidated. This study prospectively explores the management of TGCT in tertiary sarcoma centers. METHODS: The TGCT Observational Platform Project registry was a multinational, multicenter, prospective observational study involving 12 tertiary sarcoma centers in 7 European countries, and 2 US sites. This study enrolled for 2 years all consecutive ≥ 18 years old patients, with histologically diagnosed primary or recurrent cases of diffuse-type TGCT. Patient demographic and clinical characteristics were collected at baseline and every 6 months for 24 months. Quality of life questionnaires (PROMIS-PF and EQ-5D) were also administered at the same time-points. Here we report baseline patient characteristics. RESULTS: 166 patients were enrolled between November 2016 and March 2019. Baseline characteristics were: mean age 44 years (mean age at disease onset: 39 years), 139/166 (83.7%) had prior treatment, 71/166 patients (42.8%) had ≥ 1 recurrence after treatment of their primary tumor, 76/136 (55.9%) visited a medical specialist ≥ 5 times, 66/116 (56.9%) missed work in the 24 months prior to baseline, and 17/166 (11.6%) changed employment status or retired prematurely due to disease burden. Prior treatment consisted of surgery (i.e., arthroscopic, open synovectomy) (128/166; 77.1%) and systemic treatments (52/166; 31.3%) with imatinib (19/52; 36.5%) or pexidartinib (27/52; 51.9%). Treatment strategies at baseline visits consisted mainly of watchful waiting (81/166; 48.8%), surgery (41/166; 24.7%), or targeted systemic therapy (37/166; 22.3%). Patients indicated for treatment reported more impairment compared to patients indicated for watchful waiting: worst stiffness NRS 5.16/3.44, worst pain NRS 6.13/5.03, PROMIS-PF 39.48/43.85, and EQ-5D VAS 66.54/71.85. CONCLUSION: This study confirms that diffuse-type TGCT can highly impact quality of life. A prospective observational registry in rare disease is feasible and can be a tool to collect curated-population reflective data in orphan diseases. Name of registry: Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project (TOPP). TRIAL REGISTRATION NUMBER: NCT02948088. Date of registration: 10 October 2016. URL of Trial registry record: https://clinicaltrials.gov/ct2/show/NCT02948088?term=NCT02948088&draw=2 .

Health related quality of life of Canary Island citizens.

BACKGROUND: The aim of the study was to describe the health-related quality of life of Canarian population using information from the Canary Island Health Survey and three observational studies developed in the Canary Islands. METHODS: A descriptive analysis was carried out on a sample of 5.549 Canarian citizens using information from 2004 Canary Island Health Survey and three observational studies on Alzheimer's disease, Stroke and HIV. EQ-5 D was the generic tool used for revealing quality of life of people surveyed. Besides the rate of people reporting moderate or severe decrease in quality of life, TTO-index scores and visual analogue scale were used for assessing health related quality of life of people that suffer a specific diseases and general population. RESULTS: Self-perceived health status of citizens that suffer chronic diseases of high prevalence, identifies by the Canary Island Health Survey and other diseases such Alzheimer's disease, Stroke and HIV, independently examined in observational studies, are worse than self-perceived health of general population. Depression/anxiety and pain/discomfort were identified as the dimensions of the EQ-5 D with highest prevalence of problems. Alzheimer's disease and stroke were the illnesses with greater loss of quality of life. CONCLUSIONS: Health related quality of life should be integrated into a set of information along with expectancy of life, incidence and prevalence of chronic diseases for developing health policy and planning health care activities The combination of information on health related quality of life from population health surveys with data from observational studies enlarges the sources of relevant information for setting health priorities and assessing the impact of health policies.

Cost of illness and economic evaluation in rare diseases.

Rare diseases are a major cause of morbidity and mortality in high income countries and have major repercussions on individuals and health care systems. This chapter examines the health economy of rare diseases from two different perspectives: firstly, the study of the economic impact of rare diseases (Cost of Illness studies); and, secondly, cost-effectiveness evaluation, which evaluates both the costs and results of the health care technologies applied in rare diseases. From the point of view of economics, health resource allocation is based on the principle of scarcity, as there are not - and never will be- sufficient resources for all worthy objectives. Hence, policy makers should balance costs and health outcomes. Rare diseases may well represent a significant societal burden that should rightly receive appropriate prioritisation of health care resources. As new and seemingly expensive health care technologies are developed for rare diseases, it will become increasingly important to evaluate potential and real impact of these new technologies in both dimensions: social costs and health outcomes.

The Economic Impact and Health-Related Quality of Life of Spinal Muscular Atrophy. An Analysis across Europe.

Background: this study aimed to estimate the economic impact and health-related quality of life (HRQOL) of patients with spinal muscular atrophy (SMA) in three European countries. It was used a cross-sectional study carried out in France, Germany, and the United Kingdom. Data were collected from July 2015 to November 2015. Healthcare costs (hospitalizations, emergencies, medical tests, drugs used, visits to general practitioners (GPs) and specialists, medical material and healthcare transport), and non-healthcare costs (social services and informal care) were identified and valued. EuroQol instruments, the Zarit interview, and the Barthel Index were also used to reflect the burden and the social impact of the disease beyond the cost of healthcare. Results: we included 86 children with SMA, 26.7% of them had Type I, and 73.3% Type II or III. The annual average cost associated with SMA reaches €54,295 in the UK, €32,042 in France and €51,983 in Germany. The direct non-healthcare costs ranged between 79-86% of the total cost and the informal care costs were the main component of these costs. Additionally, people suffering from this disease have a very low health-related quality of life, and there are large differences between countries. Conclusions: SMA has a high socioeconomic impact in terms of healthcare and social costs. It was also observed that the HRQOL of affected children was extremely reduced. The figures shown in this study may help to design more efficient and equitable policies, with special emphasis on the support provided to the families or on non-healthcare aid.

The Burden of Spinal Muscular Atrophy on Informal Caregivers.

Spinal muscular atrophy (SMA) is one of the most common severe hereditary diseases of infancy and early childhood. The progression of this illness causes a high degree of disability; hence, a significant burden is experienced by individuals with this disease and their families. We analyzed the time taken to care for patients suffering from SMA in European countries and the burden on their informal caregivers. We designed a cross-sectional study recording data from France, Germany, Spain and the United Kingdom. The primary caregivers completed a self-administered questionnaire that included questions about the time of care, The Zarit Burden Interview, type of SMA and socio-demographic characteristics. Multivariate analyses were used to study the associations between the type of SMA, time of care and burden supported by informal caregivers. The caregivers provided 10.0 h (SD = 6.7) per day of care (the principal caregivers provided 6.9 h, SD = 4.6). The informal caregivers of patients with type I SMA had a 36.3 point higher likelihood (p < 0.05) of providing more than 10 h of care per day in comparison with caregivers of patients with type III SMA. The severity of the disease was associated with more time of care and a higher burden on the caregivers.

Considering the societal perspective in economic evaluations: a systematic review in the case of depression.

BACKGROUND: Depressive disorders are associated with a high burden of disease. However, due to the burden posed by the disease on not only the sufferers, but also on their relatives, there is an ongoing debate about which costs to include and, hence, which perspective should be applied. Therefore, the aim of this paper was to examine whether the change between healthcare payer and societal perspective leads to different conclusions of cost-utility analyses in the case of depression. METHODS: A systematic literature search was conducted to identify economic evaluations of interventions in depression, launched on Medline and the Cost-Effectiveness Registry of the Tufts University using a ten-year time horizon (2008-2018). In a two-stepped screening process, cost-utility studies were selected by means of specified inclusion and exclusion criteria. Subsequently, relevant findings was extracted and, if not fully stated, calculated by the authors of this work. RESULTS: Overall, 53 articles with 92 complete economic evaluations, reporting costs from healthcare payer/provider and societal perspective, were identified. More precisely, 22 estimations (24%) changed their results regarding the cost-effectiveness quadrant when the societal perspective was included. Furthermore, 5% of the ICURs resulted in cost-effectiveness regarding the chosen threshold (2% of them became dominant) when societal costs were included. However, another four estimations (4%) showed the opposite result: these interventions were no longer cost-effective after the inclusion of societal costs. CONCLUSIONS: Summarising the disparities in results and applied methods, the results show that societal costs might alter the conclusions in cost-utility analyses. Hence, the relevance of the perspectives chosen should be taken into account when carrying out an economic evaluation. This systematic review demonstrates that the results of economic evaluations can be affected by different methods available for estimating non-healthcare costs.

Cost-effectiveness of donepezil in the treatment of mild or moderate Alzheimer's disease.

Available treatments for Alzheimer's disease (AD) need to be evaluated in order to determine whether the clinical benefits justify their additional costs. This study evaluated the cost-effectiveness of donepezil treatment compared with no-drug treatment of mild and moderate AD from the perspective of society and the health care system in Spain. A Markov model was designed to simulate the natural history of a cohort of patients with mild and moderate AD. Monthly transition probabilities were estimated from the international literature and donepezil clinical trials. Direct medical and non-medical costs and utilities were derived from Spanish studies. Local data on tolerance and medication withdrawal rates were incorporated into the model. Incremental cost-effectiveness ratios for a range of realistic treatment options were calculated. A probabilistic sensitivity analysis was carried out using a Monte Carlo approach with 10,000 iterations. In the baseline scenario (24 months, patients initially with mild AD) incremental cost-effectiveness for direct medical costs was 20,353 euro/QALY. When all costs were taken into account, donepezil treatment was the dominant strategy. Incremental cost-effectiveness ratios vary according to the selected perspective. For the baseline scenario, donepezil treatment is cost-effective with a probability of 95% for a threshold efficiency of 25,000 euro/QALY.

Social economic costs and health-related quality of life in patients with amyotrophic lateral sclerosis in Spain.

Amyotrophic lateral sclerosis (ALS) is a process that leads to the functional disability of the individual in a relatively short period of time, with a very important limitation of autonomy and affecting the quality of life. We wished to determine the economic burden (direct and indirect costs), as well as health-related quality of life (HRQoL) in patients with ALS in Spain. A cross-sectional study was carried out on 63 patients with ALS during 2004. A retrospective assessment of the use of resources was obtained through questionnaires completed by the patients and/or the patients' caregivers. The approach used was a cost-of-illness study based on a societal perspective. We assessed the HRQoL with the EQ-5D. Mean annual cost per patient with ALS was euro36,194. The most important categories of costs were informal care, early retirement, medications, and orthopaedic devices. The mean EQ-5D index score was 0.18 and the mean EQ-5D VAS score was 29. Considerations of the costs related to caregiving to cope with the patients' disabilities, as well as the high indirect costs resulting from early retirement in patients with ALS, should become a priority for health authorities in Spain. The patients' HRQoL was very low and substantially influenced by the degree of severity of ALS.

The economic costs and health-related quality of life of people with HIV/AIDS in the Canary Islands, Spain.

BACKGROUND: The objective was to determine the economic burden, as well as the impact on HRQOL for people with HIV/AIDS in Spain in 2003. METHODS: A cross-sectional study of 572 people with HIV were recruited from outpatient clinics in the Canary Islands, Spain. Demographic, health resources utilization, indirect costs and quality of life data were collected through medical records and questionnaires filled out by people with HIV. HRQOL was measured with two generic questionnaires: SF-36 and EQ-5D. RESULTS: In 2003 annual costs of caring for patients with asymptomatic HIV, symptomatic HIV and AIDS were euro10,351, euro14,489 and euro15,750, respectively. The HRQOL with the EQ-5D was 0.78. SF-36 summary results for physical and mental health were 48.30 and 38.80, respectively. CONCLUSION: HIV/AIDS represent a high economic impact from society point of view. the structure of health care costs have changed due to these new drugs, increasing the weight of pharmaceutical treatment over total costs and decreasing the importance of inpatient care costs. In spite of the therapeutic improvements, labour losses/indirect costs still represent a high cost. Costs and HRQOL were strongly associated with severity. Although the latest drug developments have not yet been able to find the definitive cure, they have allowed an improvement in expectancy of life and in the HRQOL of the patients.

What attributes should be included in a discrete choice experiment related to health technologies? A systematic literature review.

Discrete choice experiments (DCEs) are a way to assess priority-setting in health care provision. This approach allows for the evaluation of individuals' preferences as a means of adding criteria to traditional quality-adjusted life year analysis. The aim of this systematic literature review was to identify attributes for designing a DCE in order to then develop and validate a framework that supports decision-making on health technologies. Our systematic literature review replicated the methods and search terms used by de Bekker-Grob et al. 2012 and Clark et al. 2014. The Medline database was searched for articles dated between 2008 and 2015. The search was limited to studies in English that reflected general preferences and were choice-based, published as full-text articles and related to health technologies. This study included 72 papers, 52% of which focused on DCEs on drug treatments. The average number of attributes used in all included DCE studies was 5.74 (SD 1.98). The most frequently used attributes in these DCEs were improvements in health (78%), side effects (57%) and cost of treatment (53%). Other, less frequently used attributes included waiting time for treatment or duration of treatment (25%), severity of disease (7%) and value for money (4%). The attributes identified might inform future DCE surveys designed to study societal preferences regarding health technologies in order to better inform decisions in health technology assessment.

Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy.

BACKGROUND: Decision makers have huge problems when attempting to attribute social value to the improvements achieved by new drugs, especially when considering the use of orphan drugs for rare diseases. We present the results of a pilot study aimed to investigate patient preferences regarding public funding for drugs used to treat rare diseases. METHODS: An online questionnaire was used as a discrete choice experiment (DCE) survey to explore the preferences of patients with cystic fibrosis and haemophilia in Italy. The questionnaire focused on relevant issues that were defined in a review of the literature. A conditional logistic model showed preferences for specific attributes. RESULTS: A total of 54 questionnaires (20% response rate) were completed. The issues that received the greatest attention were improvement in health, treatment cost and value for money. However, disease severity and the availability of other treatments were important social values that could not be ignored. CONCLUSIONS: The findings presented here provide evidence as to what patients with cystic fibrosis or haemophilia think are the most important considerations on which to base decisions in health technology scenarios, and regarding the priorities for funding.