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

Pérdidas laborales ocasionadas por muertes prematuras en España : un análisis para el periodo 2005-2009

Fundamentos: El impacto económico que causan las enfermedades va mucho más allá del gasto sanitario, transmitiéndose a la sociedad a través de diferentes dimensiones. El principal objetivo de este estudio fue estimar la evolución de las pérdidas de productividad laboral causadas por muertes prematuras en España durante el periodo 2005-2009. Métodos: Se emplearon varias fuentes estadísticas (Registro de defunciones según la causa de muerte, Encuesta de Población Activa y Encuesta de Estructura Salarial) para desarrollar un modelo de simulación basado en el enfoque de capital humano que permitió estimar las pérdidas laborales ocasionadas por muertes prematuras para el periodo analizado. Adicionalmente, se llevaron a cabo dos escenarios alternativos en los que se analizó la influencia de la evolución epidemiológica en los resultados obtenidos. Resultados: El modelo de simulación mostró una caída de las pérdidas laborales asociadas a muertes prematuras que fue desde 8.935 millones de euros en 2005 a 8.073 millones de euros en 2009. El análisis de las causas reducidas de muerte señaló que nueve causas de enfermedad (accidentes de tráfico, tumor maligno de tráquea, suicidios, infarto agudo de miocardio, SIDA, cirrosis, tumor de mama, enfermedades cerebrovasculares y cáncer de colon) supusieron el 35,6% del total de Años Potenciales de Vida Laboral Perdidos y 36,6% del total de pérdida estimada. Las pérdidas estimadas representaron el 0,98%, 0,85% y 0,77% del Producto Interior Bruto de 2005, 2007 y 2009, respectivamente. Conclusiones: Las pérdidas laborales ocasionadas por muertes prematuras en España disminuyeron sustancialmente en el periodo analizado. Esta reducción se debió fundamentalmente a la favorable evolución epidemiológica de la mortalidad prematura.Background: The economic impact caused by diseases goes far beyond health care costs and, therefore it is transferred to the society through different dimensions. The aim of this study was to estimate the productivity losses due to premature deaths caused by diseases occurred in Spain during the period 2005-2009. Methods: We used data from several sources (Death Registry, Labour Force Survey and Wage Structure Survey) to develop a simulation model based on the human-capital approach that allowed us to estimate the labour productivity losses caused by premature deaths in the period analysed. Additionally, we also carried out two alternative scenarios in which we analysed how epidemiologic data influenced our results. Results: Our model showed the estimated loss of productivity due to premature death fell from 8,935 billion euros in 2005 to 8,073 billion euros in 2009. Nine diseases (traffic accidents, malignant tumour of the trachea, suicides, acute myocardial infarction, AIDS, cirrhosis, breast tumour, cerebrovascular disease and colon cancer) accounted for 35.6% of the total Years of Potential Productive Life Lost and 36.5% of the estimated productivity losses. The estimated losses represented 0.98%, 0.85% and 0.77% of Gross Domestic Product in 2005, 2007 and 2009, respectively. Conclusions: The labour productivity losses caused by premature deaths decreased substantially in the period analysed. This reduction was mainly due to the epidemiological evolution of premature mortality

Cost-Effectiveness of an Exercise Programme That Provided Group or Individual Training to Reduce the Fall Risk in Healthy Community-Dwelling People Aged 65–80: A Secondary Data Analysis

: Research has demonstrated that some exercise programs are effective for reducing fall rates in community-dwelling older people; however, the literature is limited in providing clear recommendations of individual or group training as a result of economic evaluation. The objective of this study was to assess the cost-effectiveness of the Otago Exercise Program (OEP) for reducing the fall risk in healthy, non-institutionalized older people. An economic evaluation of a multicenter, blinded, randomized, non-inferiority clinical trial was performed on 498 patients aged over 65 in primary care. Participants were randomly allocated to the treatment or control arms, and group or individual training. The program was delivered in primary healthcare settings and comprised five initial sessions, ongoing encouragement and support to exercise at home, and a reinforcement session after six months. Our hypothesis was that the patients who received the intervention would achieve better health outcomes and therefore need lower healthcare resources during the follow-up, thus, lower healthcare costs. The primary outcome was the incremental cost-effectiveness ratio, which used the timed up and go test results as an effective measure for preventing falls. The secondary outcomes included differently validated tools that assessed the fall risk. The cost per patient was USD 51.28 lower for the group than the individual sessions in the control group, and the fall risk was 10% lower when exercises had a group delivery. The OEP program delivered in a group manner was superior to the individual method. We observed slight differences in the incremental cost estimations when using different tools to assess the risk of fall, but all of them indicated the dominance of the intervention group. The OEP group sessions were more cost-effective than the individual sessions, and the fall risk was 10% lower

Cost-Effectiveness of an Exercise Programme that Provided Group or Individual Training to Reduce the Fall Risk in Healthy Community-Dwelling People Aged 65–80: A Secondary Data Analysis

Research has demonstrated that some exercise programs are effective for reducing fall rates in community-dwelling older people; however, the literature is limited in providing clear recommendations of individual or group training as a result of economic evaluation. The objective of this study was to assess the cost-effectiveness of the Otago Exercise Program (OEP) for reducing the fall risk in healthy, non-institutionalized older people. An economic evaluation of a multicenter, blinded, randomized, non-inferiority clinical trial was performed on 498 patients aged over 65 in primary care. Participants were randomly allocated to the treatment or control arms, and group or individual training. The program was delivered in primary healthcare settings and comprised five initial sessions, ongoing encouragement and support to exercise at home, and a reinforcement session after six months. Our hypothesis was that the patients who received the intervention would achieve better health outcomes and therefore need lower healthcare resources during the follow-up, thus, lower healthcare costs. The primary outcome was the incremental cost-effectiveness ratio, which used the timed up and go test results as an effective measure for preventing falls. The secondary outcomes included differently validated tools that assessed the fall risk. The cost per patient was USD 51.28 lower for the group than the individual sessions in the control group, and the fall risk was 10% lower when exercises had a group delivery. The OEP program delivered in a group manner was superior to the individual method. We observed slight differences in the incremental cost estimations when using different tools to assess the risk of fall, but all of them indicated the dominance of the intervention group. The OEP group sessions were more cost-effective than the individual sessions, and the fall risk was 10% lower

Economic burden and health‑related quality of life in tenosynovial giant‑cell tumour patients in Europe: an observational disease registry

Background: Tenosynovial Giant-Cell Tumour (TGCT) is a benign clonal neoplastic proliferation arising from the syn‑ ovium, causing a variety of symptoms and often requiring repetitive surgery. This study aims to defne the economic burden—from a societal perspective—associated with TGCT patients and their health-related quality of life (HRQOL) in six European countries. Methods: This article analyses data from a multinational, multicentre, prospective observational registry, the TGCT Observational Platform Project (TOPP), involving hospitals and tertiary sarcoma centres from six European countries (Austria, France, Germany, Italy, the Netherlands, and Spain). It includes information on TGCT patients’ health-related quality of life and healthcare and non-healthcare resources used at baseline (the 12-month period prior to the patients entering the registry) and after 12 months of follow-up. Results: 146 TGCT patients enrolled for the study, of which 137 fulflled the inclusion criteria. Their mean age was 44.5 years, and 62% were female. The annual average total costs associated with TGCT were €4866 at baseline and €5160 at the 12-month follow-up visit. The annual average healthcare costs associated with TGCT were €4620 at baseline, of which 67% and 18% corresponded to surgery and medical visits, respectively. At the 12-month follow up, the mean healthcare costs amounted to €5094, with surgery representing 70% of total costs. Loss of productivity represented, on average, 5% of the total cost at baseline and 1.3% at follow-up. The most-afected HRQOL dimensions, measured with the EQ-5D-5L instrument, were pain or discomfort, mobility, and the performance of usual activities, both at baseline and at the follow-up visit. Regarding HRQOL, patients declared a mean index score of 0.75 at baseline and 0.76 at the 12-month follow-up. Conclusion: The results suggest that TGCT places a heavy burden on its suferers, which increases after one year of follow-up, mainly due to the healthcare resources required—in particular, surgical procedures. As a result, this condi‑ tion has a high economic impact on healthcare budgets, while the HRQOL of TGCT patients substantially deteriorates over time

Health-related Quality of Life in Type 1 Diabetes Mellitus Pediatric Patients and Their Caregivers in Spain: An Observational Cross-Sectional Study

Objectives: This study assessed the health-related quality of life (HRQOL) of pediatric patients with type 1 diabetes mellitus (T1DM) and their caregivers.Methods: CHRYSTAL was an observational cross-sectional study conducted in Spain in 2014 on 275 patients under 18 years old diagnosed with T1DM. Patient/caregiver pairs were stratified by patients' HbA1c level (?7.5% versus <7.5%) and by presence or absence of T1DM complications and/or comorbidities. EQ-5D and PedsQL questionnaires were administered to patients and caregivers.Results: On the EQ-5D, according to caregivers' perception, 17.7% of children experienced moderate pain or discomfort, 9.7% suffered problems performing usual activities, and 13.2% demonstrated moderate anxiety or depression. Mean EQ-5D index score was 0.95 and mean visual analog scale (VAS) score was 86.1. By HbA1c level (?7.5% versus <7.5%), mean index scores were 0.94 and 0.95, and mean VAS scores were 82.8 and 89.2, respectively. Mean index scores were 0.91 for children with complications and/or comorbidities and 0.96 for children without. Mean VAS scores were 83.7 and 87.2, respectively. HRQOL per the PedsQL tool ranged from 68.1 (ages 2-4) to 73.1 (ages 13-18). EQ-5D index and VAS scores were significantly correlated (rho = 0.29-0.43) with several age groups of the PedsQL. EQ-5D scales showed significant moderate correlation between EQ-5D-Y and EQ-5D-3L proxy VAS score (rho = 0.45; p < .001).Conclusions: Patients with few complications and controlled HbA1c reported a relatively high HRQOL. The results suggest that parent-proxy EQ-5D ratings are valid for use as part of an overall health outcomes assessment in clinical studies of T1DM in pediatric patients

Social economic costs of type 1 diabetes mellitus in pediatric patients in Spain: CHRYSTAL observational study

AIMS: To estimate the social-economic costs of Type 1 Diabetes Mellitus (T1DM) in patients aged 0-17 years in Spain from a social perspective. METHODS: We conducted a cross-sectional observational study in 2014 of 275 T1DM pediatric outpatients distributed across 12 public health centers in Spain. Data on demographic and clinical characteristics, healthcare utilization and informal care were collected from medical records and questionnaires completed by clinicians and patients' caregivers. RESULTS: A valid sample of 249 individuals was analyzed. The average annual cost for a T1DM patient was €27,274. Direct healthcare costs were €4070 and direct non-healthcare cost were €23,204. Informal (familial) care represented 83% of total cost, followed by medical material (8%), outpatient and primary care visits (3.1%) and insulin (2.1%). Direct healthcare cost per patient statistically differed by glycated haemoglobin (HbA1c) level [mean cost €4704 in HbA1c ?7.5% (?58mmol/mol) group vs. €3616 in HbA1c<7.5% (<58mmol/mol) group)]; and by the presence or absence of complications and comorbidities (mean cost €5713 in group with complications or comorbidities vs. €3636 in group without complications or comorbidities). CONCLUSIONS: T1DM amongst pediatric patients incurs in considerable societal costs. Informal care represents the largest cost category

Impacto socioeconómico de las enfermedades crónicas y problemas de salud

La incorporación de la salud como pilar de las sociedades del bienestar parece ser condición suficiente para tener en cuenta el impacto que tienen las enfermedades y problemas de salud desde un punto de vista amplio. La organización de los recursos disponibles para la atención de la salud es clave para garantizar la sostenibilidad del Sistema Nacional de Salud. Para ello parece recomendable que no sólo se tenga en cuenta cuál es el valor monetario de los recursos que se precisan para que el sistema funcione, sino también otros impactos económicos y sociales relacionados con el mantenimiento o la pérdida de salud y autonomía personal. Así pues, en esta tesis doctoral se evaluó el impacto económico que originan las enfermedades y problemas de salud estimando el coste sanitario de una intervención farmacológica, las pérdidas laborales ocasionadas por enfermedades y lesiones a nivel nacional, la valoración monetaria del tiempo cuidado informal y la pérdida de bienestar que sufre las personas cuidadoras de aquellas cuya autonomía se ha visto limitada como consecuencia de una enfermedad crónica. Los resultados obtenidos proporcionan información hasta la fecha no revelada en la literatura muy útil para la toma de decisiones en el ámbito de la micro, meso y macro-gestión de los recursos sanitarios y sociosanitarios. En el futuro, cabe esperar el desarrollo de herramientas y estudios que generen información de utilidad para el diseño y aplicación de políticas públicas sostenibles que, asignando los recursos disponibles de una manera eficiente y equitativa, reduzcan el impacto social de las enfermedades y lesiones y mejoren la salud y el bienestar de la sociedad

Evaluación de las pérdidas laborales provocadas enfermedades y lesiones en España en el año 2009

The main aim of this study is to estimate the productivity losses caused by diseases and injuries occurred in Spain in 2009. Using several data sources from the National Statistics Institute and The National Institute of Social Security, a simulation model based on the human-capital approach is performed to estimate the labour losses due to premature deaths and disability (permanent and temporary).The total productivity losses are estimated to amount to 42,430 million of euros, of which 8,073 million of euros are due to the losses caused by premature deaths (19% of the total loss), 18,050 million of euros to permanent disability (42% of the total loss) and 16,306 million of euros to temporary disability (38.4% of the total loss). These figures represented 3.93% of the 2009 Gross Domestic Product of Spain, in other words, 60.3% of the total Public Health Expenditure in 2009. By type of disease or injury, diseases of musculoskeletal system and connective tissue accounted for 22.9% of the total loss, followed by external causes with 13.5%, metal disorders with 10.5% and tumors with 10.4%.El objetivo del trabajo es estimar las potenciales pérdidas laborales ocasionadas por enfermedades y lesiones durante el año 2009. Combinando varias fuentes de datos del Instituto Nacional de Estadística y el Instituto Nacional de la Seguridad Social, se desarrolla un modelo de simulación basado en el enfoque del capital humano para la estimación de pérdidas de producción asociadas a muertes prematuras y a situaciones de incapacidad laboral (permanente y temporal).Las pérdidas potenciales estimadas ascienden a valores de 42.430 millones de euros, del cual 8.073 millones de euros corresponden a pérdidas derivadas de la mortalidad prematura (un 19,0% sobre el total), 18.050 millones de euros corresponden a pérdidas asociadas a situaciones de Incapacidad Permanente (42,5% sobre el total) y 16.306 millones de euros a pérdidas asociadas a situaciones de Incapacidad Temporal (38,4% sobre el total). Ello supone una cifra equivalente al 3,93% del Producto Interior Bruto de España o, alternativamente, al 60,3% del gasto sanitario público total del año 2009. Por tipo de enfermedad o lesión, las enfermedades del sistema osteomuscular y del tejido conjuntivo representaron el 22,9% de las pérdidas, seguidos de las causas externas, con un 13,5%, los trastornos mentales, 10,5%, y los tumores, con un 10,4%. 

Social/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain

Abstract Background The aim of this study was to determine the economic burden and health-related quality of life (HRQOL) of patients with Spinal Muscular Atrophy (SMA) and their caregivers in Spain. Methods This was a cross-sectional and retrospective study of patients diagnosed with SMA in Spain. We adopted a bottom up, prevalence approach design to study patients with SMA. The patient’s caregivers completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and non-healthcare services. Costs were estimated from a societal perspective (including healthcare costs and non-healthcare costs), and health-related quality of life (HRQOL) was assessed using the EQ-5D questionnaire. The main caregivers also answered a questionnaire on their characteristics and on their HRQOL. Results A total of 81 caregivers of patients with different subtypes of SMA completed the questionnaire. Based on the reference unitary prices for 2014, the average annual costs per patient were € 33,721. Direct healthcare costs were € 10,882 (representing around 32.3% of the total cost) and the direct non-healthcare costs were € 22,839 (67.7% of the total cost). The mean EQ-5D social tariff score for patients was 0.16, and the mean score of the EQ-5D visual analogue scale was 54. The mean EQ-5D social tariff score for caregivers was 0.49 and their mean score on the EQ-5D visual analogue scale was 69. Conclusion The results highlight the burden that SMA has in terms of costs and decreased HRQOL, not only for patients but also for their caregivers. In particular, the substantial social/economic burden is mostly attributable to the high direct non-healthcare costs