RESUMEN
Health technology assessment (HTA) of medical devices (MDs) increasingly rely on real-world evidence (RWE). The aim of this study was to evaluate the type and the quality of the evidence used to assess the (cost-)effectiveness of high risk MDs (Class III) by HTA agencies in Europe (four European HTA agencies and EUnetHTA), with particular focus on RWE. Data were extracted from HTA reports on the type of evidence demonstrating (cost-)effectiveness, and the quality of observational studies of comparative effectiveness using the Good Research for Comparative Effectiveness principles. 25 HTA reports were included that incorporated 28 observational studies of comparative effectiveness. Half of the studies (46%) took important confounding and/or effect modifying variables into account in the design and/or analyses. The most common way of including confounders and/or effect modifiers was through multivariable regression analysis. Other methods, such as propensity score matching, were rarely employed. Furthermore, meaningful analyses to test key assumptions were largely omitted. Resulting recommendations from HTA agencies on MDs is therefore (partially) based on evidence which is riddled with uncertainty. Considering the increasing importance of RWE it is important that the quality of observational studies of comparative effectiveness are systematically assessed when used in decision-making.
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Evaluación de la Tecnología Biomédica , Análisis Costo-Beneficio , Europa (Continente) , Humanos , Evaluación de la Tecnología Biomédica/métodosRESUMEN
OBJECTIVES: Developing and validating a discrete event simulation model that is able to model patients with heart failure managed with usual care or an early warning system (with or without a diagnostic algorithm) and to account for the impact of individual patient characteristics in their health outcomes. METHODS: The model was developed using patient-level data from the Trans-European Network - Home-Care Management System study. It was coded using RStudio Version 1.3.1093 (version 3.6.2.) and validated along the lines of the Assessment of the Validation Status of Health-Economic decision models tool. The model includes 20 patient and disease characteristics and generates 8 different outcomes. Model outcomes were generated for the base-case analysis and used in the model validation. RESULTS: Patients managed with the early warning system, compared with usual care, experienced an average increase of 2.99 outpatient visits and a decrease of 0.02 hospitalizations per year, with a gain of 0.81 life years (0.45 quality-adjusted life years) and increased average total costs of 11 249. Adding a diagnostic algorithm to the early warning system resulted in a 0.92 life year gain (0.57 quality-adjusted life years) and increased average costs of 9680. These patients experienced a decrease of 0.02 outpatient visits and 0.65 hospitalizations per year, while they avoided being hospitalized 0.93 times. The model showed robustness and validity of generated outcomes when comparing them with other models addressing the same problem and with external data. CONCLUSIONS: This study developed and validated a unique patient-level simulation model that can be used for simulating a wide range of outcomes for different patient subgroups and treatment scenarios. It provides useful information for guiding research and for developing new treatment options by showing the hypothetical impact of these interventions on a large number of important heart failure outcomes.
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Simulación por Computador/normas , Insuficiencia Cardíaca/complicaciones , Simulación de Paciente , Simulación por Computador/tendencias , Insuficiencia Cardíaca/fisiopatología , HumanosRESUMEN
BACKGROUND: Empirical identification of the direct impact of hospitalisation in the change in utility could provide an interpretation for some of the unexplained variance in quality of life responses in clinical practice and clinical trials and provide assistance to researchers in assessing the impact of a hospitalisation in the context of economic evaluations. This study had the goal of determining the impact of nonfatal hospitalisations on the quality of life of a cohort of patients previously diagnosed with heart failure by using their quality of life measurements before and after hospitalisation. METHODS: The impact of hospitalisation on health-related quality of life was estimated by calculating the difference in utility measured using the EQ-5D-3L in patients that were hospitalised and had records of utility before and after hospitalisation. The variation in differences between the utilities pre and post hospitalisation was explained through two multiple linear regression models using (1) the individual patient characteristics and (2) the hospitalisation characteristics as explanatory variables. RESULTS: The mean difference between health-related quality of life measurement pre and post hospitalisation was found to be 0.020 [95% CI: - 0.020, 0.059] when measured with the EQ-5D index, while there was a mean decrease of - 0.012 [95% CI: - 0.043, 0.020] in the utility measured with the visual analogue scale. Differences in utility variation according to the primary cause for hospitalisation were found. Regression models showed a statistically significant impact of body mass index and serum creatinine in the index utility differences and of serum creatinine for utilities measured with the visual analogue scale. CONCLUSIONS: Knowing the impact of hospitalisation on health-related quality of life is particularly relevant for informing cost-effectiveness studies designed to assess health technologies aimed at reducing hospital admissions. Through using patient-level data it was possible to estimate the variation in utilities before and after the average hospitalisation and for hospitalisations due to the most common causes for hospital admission. These estimates for (dis) utility could be used in the calculations of effectiveness on economic evaluations, especially when discrete event simulations are the employed modelling technique.
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Insuficiencia Cardíaca/psicología , Hospitalización , Calidad de Vida , Anciano , Anciano de 80 o más Años , Estudios de Cohortes , Análisis Costo-Beneficio/métodos , Femenino , Insuficiencia Cardíaca/economía , Humanos , Masculino , Persona de Mediana Edad , Encuestas y CuestionariosRESUMEN
OBJECTIVES: To assess cost effectiveness of abatacept versus adalimumab, each administered with methotrexate, in treating patients with rheumatoid arthritis (RA) stratified according to baseline anticitrullinated protein antibody (ACPA) levels (marker of poor prognosis in RA). METHODS: A payer-perspective cost-effectiveness model simulated disease progression in patients with RA who had previously failed conventional disease-modifying antirheumatic drugs and were starting biologic therapy. Patients commenced treatment with abatacept or adalimumab plus methotrexate and were evaluated after 6 months. Therapy continuation was based on the European League Against Rheumatism treatment response; disease progression was based on the Health Assessment Questionnaire Disability Index score. These score changes were used to estimate health state utilities and direct medical costs. Quality-adjusted life-years (QALYs) and incremental cost per QALY gained were calculated by baseline ACPA groups (Q1, 28-234 AU/ml; Q2, 235-609 AU/ml; Q3, 613-1045 AU/ml; and Q4, 1060-4894 AU/ml). Scenario analysis and one-way and probabilistic sensitivity analyses were used to evaluate robustness of model assumptions. RESULTS: Abatacept resulted in QALY gain versus adalimumab in ACPA Q1, Q3, and Q4; between-treatment difference (difference: Q1, -0.115 Q2, -0.009 Q3, 0.045; and Q4, 0.279). Total lifetime discounted cost was higher for abatacept versus adalimumab in most quartiles (Q2, £77,612 vs. £77,546; Q3, £74,441 vs. £73,263; and Q4, £78,428 vs. £76,696) because of longer time on treatment. Incremental cost per QALY for abatacept (vs. adalimumab) was the lowest in the high ACPA titer group (Q4, £6200/QALY), followed by the next lowest titer group (Q3, £26,272/QALY). CONCLUSIONS: Abatacept is a cost effective alternative to adalimumab in patients with RA with high ACPA levels.
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Abatacept/economía , Abatacept/uso terapéutico , Adalimumab/economía , Adalimumab/uso terapéutico , Antirreumáticos/economía , Antirreumáticos/uso terapéutico , Artritis Reumatoide/tratamiento farmacológico , Análisis Costo-Beneficio , Metotrexato/economía , Metotrexato/uso terapéutico , Péptidos Cíclicos/inmunología , Años de Vida Ajustados por Calidad de Vida , Adulto , Anciano , Anciano de 80 o más Años , Evaluación de la Discapacidad , Progresión de la Enfermedad , Quimioterapia Combinada , Femenino , Humanos , Masculino , Persona de Mediana Edad , PronósticoRESUMEN
BACKGROUND: In an ageing population, it is inevitable to improve the management of care for community-dwelling elderly with incontinence. A previous study showed that implementation of the Optimum Continence Service Specification (OCSS) for urinary incontinence in community-dwelling elderly with four or more chronic diseases results in a reduction of urinary incontinence, an improved quality of life, and lower healthcare and lower societal costs. The aim of this study was to explore future consequences of the OCSS strategy of various healthcare policy scenarios in an ageing population. METHODS: We adapted a previously developed decision analytical model in which the OCSS new care strategy was operationalised as the appointment of a continence nurse specialist located within the general practice in The Netherlands. We used a societal perspective including healthcare costs (healthcare providers, treatment costs, insured containment products, insured home care), and societal costs (informal caregiving, containment products paid out-of-pocket, travelling expenses, home care paid out-of-pocket). All outcomes were computed over a three-year time period using two different base years (2014 and 2030). Settings for future policy scenarios were based on desk-research and expert opinion. RESULTS: Our results show that implementation of the OSCC new care strategy for urinary incontinence would yield large health gains in community dwelling elderly (2030: 2592-2618 QALYs gained) and large cost-savings in The Netherlands (2030: health care perspective: 32.4 Million - 72.5 Million; societal perspective: 182.0 Million - 250.6 Million). Savings can be generated in different categories which depends on healthcare policy. The uncertainty analyses and extreme case scenarios showed the robustness of the results. CONCLUSIONS: Implementation of the OCSS new care strategy for urinary incontinence results in an improvement in the quality of life of community-dwelling elderly, a reduction of the costs for payers and affected elderly, and a reduction in time invested by carers. Various realistic policy scenarios even forecast larger health gains and cost-savings in the future. More importantly, the longer the implementation is postponed the larger the savings foregone. The future organisation of healthcare affects the category in which the greatest savings will be generated.
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Costos de la Atención en Salud , Enfermeras Especialistas/economía , Incontinencia Urinaria/terapia , Anciano , Ahorro de Costo , Costo de Enfermedad , Análisis Costo-Beneficio , Técnicas de Apoyo para la Decisión , Política de Salud , Humanos , Vida Independiente , Países Bajos , Calidad de Vida , Incontinencia Urinaria/economía , Incontinencia Urinaria/prevención & controlRESUMEN
Objectives: The aims were to compare the performance of cardiovascular risk calculators, Framingham Risk Score (FRS) and QRISK2, in RA and matched non-RA patients and to evaluate whether their performance could be enhanced by the addition of CRP. Methods: We conducted a retrospective analysis, using a clinical practice data set linked to Hospital Episode Statistics (HES) data from the UK. Patients presenting with at least one RA diagnosis code and no prior cardiovascular events were matched to non-RA patients using disease risk scores. The overall performance of the FRS and QRISK2 was compared between cohorts, and assessed with and without CRP in the RA cohort using C-Index, Akaike Information Criterion (AIC) and the net reclassification index (NRI). Results: Four thousand seven hundred and eighty RA patients met the inclusion criteria and were followed for a mean of 3.8 years. The C-Index for the FRS in the non-RA and RA cohort was 0.783 and 0.754 (P < 0.001) and that of the QRISK2 was 0.770 and 0.744 (P < 0.001), respectively. Log[CRP] was positively associated with cardiovascular events, but improvements in the FRS and QRISK2 C-Indices as a result of inclusion of CRP were small, from 0.764 to 0.767 (P = 0.026) for FRS and from 0.764 to 0.765 (P = 0.250) for QRISK2. The NRI was 3.2% (95% CI: -2.8, 5.7%) for FRS and -2.0% (95% CI: -5.8, 4.5%) for QRISK2. Conclusion: The C-Index for the FRS and QRISK2 was significantly better in the non-RA compared with RA patients. The addition of CRP in both equations was not associated with a significant improvement in reclassification based on NRI.
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Algoritmos , Artritis Reumatoide/complicaciones , Proteína C-Reactiva/fisiología , Enfermedades Cardiovasculares/etiología , Adolescente , Adulto , Factores de Edad , Anciano , Femenino , Humanos , Masculino , Persona de Mediana Edad , Estudios Retrospectivos , Medición de Riesgo/métodos , Factores de Riesgo , Índice de Severidad de la Enfermedad , Factores de Tiempo , Adulto JovenRESUMEN
BACKGROUND: The validation of health economic (HE) model outcomes against empirical data is of key importance. Although statistical testing seems applicable, guidelines for the validation of HE models lack guidance on statistical validation, and actual validation efforts often present subjective judgment of graphs and point estimates. OBJECTIVES: To discuss the applicability of existing validation techniques and to present a new method for quantifying the degrees of validity statistically, which is useful for decision makers. METHODS: A new Bayesian method is proposed to determine how well HE model outcomes compare with empirical data. Validity is based on a pre-established accuracy interval in which the model outcomes should fall. The method uses the outcomes of a probabilistic sensitivity analysis and results in a posterior distribution around the probability that HE model outcomes can be regarded as valid. RESULTS: We use a published diabetes model (Modelling Integrated Care for Diabetes based on Observational data) to validate the outcome "number of patients who are on dialysis or with end-stage renal disease." Results indicate that a high probability of a valid outcome is associated with relatively wide accuracy intervals. In particular, 25% deviation from the observed outcome implied approximately 60% expected validity. CONCLUSIONS: Current practice in HE model validation can be improved by using an alternative method based on assessing whether the model outcomes fit to empirical data at a predefined level of accuracy. This method has the advantage of assessing both model bias and parameter uncertainty and resulting in a quantitative measure of the degree of validity that penalizes models predicting the mean of an outcome correctly but with overly wide credible intervals.
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Interpretación Estadística de Datos , Toma de Decisiones , Complicaciones de la Diabetes/terapia , Guías como Asunto , Modelos Económicos , Teorema de Bayes , Complicaciones de la Diabetes/economía , Humanos , Fallo Renal Crónico/economía , Fallo Renal Crónico/terapia , Probabilidad , Diálisis Renal/economía , Diálisis Renal/estadística & datos numéricos , Estudios de Validación como AsuntoRESUMEN
OBJECTIVE: RA is associated with a 50-60% increase in risk of cardiovascular (CV) death. This study aimed to compare management of CV risk factors in RA and matched non-RA patients. METHODS: A retrospective cohort study was conducted using UK clinical practice data. Patients presenting with an incident RA diagnosis were matched 1:4 to non-RA patients based on a propensity score for RA, entry year, CV risk category and treatment received at index date (date of RA diagnosis). Patients tested and treated for CV risk factors as well as those attaining CV risk factor management goals were evaluated in both groups. RESULTS: Between 1987 and 2010, 24 859 RA patients were identified and matched to 87 304 non-RA patients. At index date, groups had similar baseline characteristics. Annual blood pressure, lipids and diabetes-related testing were similar in both groups, although CRP and ESR were higher in RA patients at diagnosis and decreased over time. RA patients prescribed antihypertensives increased from 38.2% at diagnosis to 45.7% at 5 years, from 14.0 to 20.6% for lipid-lowering treatments and from 5.1 to 6.4% for antidiabetics. Similar treatment percentages were observed in non-RA patients, although slightly lower for antihypertensives. Modest (2%) but significantly lower attainment of lipid and diabetes goals at 1 year was observed in RA patients. CONCLUSION: There were no differences between groups in the frequency of testing and treatment of CV risk factors. Higher CV risk in RA patients seems unlikely to be driven by differences in traditional CV risk factor management.
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Artritis Reumatoide/complicaciones , Enfermedades Cardiovasculares/etiología , Adolescente , Adulto , Anciano , Antihipertensivos/uso terapéutico , Artritis Reumatoide/epidemiología , Enfermedades Cardiovasculares/epidemiología , Enfermedades Cardiovasculares/prevención & control , Manejo de la Enfermedad , Utilización de Medicamentos/estadística & datos numéricos , Femenino , Humanos , Hipoglucemiantes/uso terapéutico , Hipolipemiantes/uso terapéutico , Masculino , Persona de Mediana Edad , Estudios Retrospectivos , Factores de Riesgo , Reino Unido/epidemiología , Adulto JovenRESUMEN
BACKGROUND: The conditional reimbursement policy for expensive medicines in The Netherlands requires data collection on actual use and cost-effectiveness after the initial decision to reimburse a drug. This introduces new sources of uncertainty (less important in a randomized controlled trial than in daily practice), which may affect priorities for further research. OBJECTIVES: This article focuses on determining the impact of including these uncertainties at the time a decision is made, and whether more complex models are always needed to address prioritization of additional research. METHODS: We constructed a typical decision model for chronic progressive diseases with four health states and parameters related to transition and exacerbation probabilities, costs, and utilities. Different scenarios are built on the basis of three additional uncertainties: persistence, compliance, and broadening of indication. Persistence refers to treatment duration. Compliance describes the fraction of treatment benefit obtained because of not taking the medication as prescribed. Broadening of indication reflects a shift in the severity distribution at treatment start. These uncertainties were parameterized in the model and included in the value-of-information analysis. RESULTS: The most important parameters were transition probabilities. Broadening of indication had little impact on the overall uncertainty. Compliance and persistence were important when establishing priorities for further research. Major differences with respect to the reference scenario were due to the parameterization of compliance in the decision model. CONCLUSIONS: The usual practice of modeling only randomized controlled trial data at the time the decision on conditional reimbursement is made can lead to wrong decisions. Additional uncertainties arising from outcomes studies should be anticipated at an early stage and included in the model because this can have a strong impact on the prioritization of further research.
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Técnicas de Apoyo para la Decisión , Progresión de la Enfermedad , Incertidumbre , Análisis Costo-Beneficio/economía , Humanos , Cumplimiento de la Medicación , Países BajosRESUMEN
BACKGROUND: Subcutaneous allergen immunotherapy (SCIT) and sublingual allergen immunotherapy (SLIT) are safe and effective treatments of allergic rhinitis, but high levels of compliance and persistence are crucial to achieving the desired clinical effects. OBJECTIVE: Our objective was to assess levels and predictors of compliance and persistence among grass pollen, tree pollen, and house dust mite immunotherapy users in real life and to estimate the costs of premature discontinuation. METHODS: We performed a retrospective analysis of a community pharmacy database from The Netherlands containing data from 6486 patients starting immunotherapy for 1 or more of the allergens of interest between 1994 and 2009. Two thousand seven hundred ninety-six patients received SCIT, and 3690 received SLIT. Time to treatment discontinuation was analyzed and included Cox proportional hazard models with time-dependent covariates, where appropriate. RESULTS: Overall, only 18% of users reached the minimally required duration of treatment of 3 years (SCIT, 23%; SLIT, 7%). Median durations for SCIT and SLIT users were 1.7 and 0.6 years, respectively (P < .001). Other independent predictors of premature discontinuation were prescriber, with patients of general practitioners demonstrating longer persistence than those of allergologists and other medical specialists; single-allergen immunotherapy, lower socioeconomic status; and younger age. Of the persistent patients, 56% were never late in picking up their medication from the pharmacy. Direct medication costs per nonpersistent patient discontinuing in the third year of treatment were 3800, an amount that was largely misspent. CONCLUSION: Real-life persistence is better in SCIT users than in SLIT users, although it is low overall. There is an urgent need for further identification of potential barriers and measures that will enhance persistence and compliance.
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Desensibilización Inmunológica , Cooperación del Paciente , Rinitis Alérgica Perenne/terapia , Administración Sublingual , Adulto , Alérgenos/administración & dosificación , Alérgenos/inmunología , Animales , Desensibilización Inmunológica/economía , Desensibilización Inmunológica/métodos , Desensibilización Inmunológica/psicología , Femenino , Humanos , Inyecciones Subcutáneas , Masculino , Persona de Mediana Edad , Países Bajos , Cooperación del Paciente/psicología , Cooperación del Paciente/estadística & datos numéricos , Poaceae/inmunología , Polen/inmunología , Pyroglyphidae , Estudios Retrospectivos , Rinitis Alérgica , Rinitis Alérgica Perenne/etiología , Árboles/inmunologíaRESUMEN
OBJECTIVES: This study primarily aimed to develop a validated Dutch translation of the 28 items of the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) II. A secondary aim was to provide a worked example of a scientifically valid translation process. METHODS: A four-step process was applied: (1) forward translation, (2) backward translation, (3) quantitative validation (two back-translated English versions vs. original English version), and (4) qualitative validation (one Dutch version vs. original English version), resulting in the final Dutch CHEERS II checklist. RESULTS: During quantitative validation, the average scores indicated high language comparability (1.88 (SD 0.70); 1.70 (SD 0.73)) and interpretation similarity (1.77 (SD 0.81); 1.54 (SD 0.74)). Four items required formal revision. In the qualitative validation step, feedback primarily focused on specific terms 'outcomes,' 'benefits and harms,' '(year of) conversion,' 'any,' and 'characterizing.' CONCLUSION: Despite English being the common language of science, translating research instruments remains relevant to enhance clarity, accessibility, and inclusivity. The Dutch translation can be used by students, regulators, researchers, or others to report and evaluate reporting of economic evaluations. Our detailed description of the applied methodology can facilitate future translations of research instruments.
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Lista de Verificación , Informe de Investigación , Humanos , Análisis Costo-BeneficioRESUMEN
The aim of this study was first, to introduce a comprehensive, de-novo health economic (HE) model incorporating the full range of activities involved in toileting and containment care (T&CC) for people with incontinence, capturing all the potential benefits and costs of existing and future Digital Health Technologies (DHT) aimed at improving continence care, for both residential care and home care. Second, to use this novel model to evaluate the cost-effectiveness of the DHT TENA SmartCare Identifi in the implementation of person-centred continence care (PCCC), compared with conventional continence care for Canadian nursing home residents. The de-novo HE model was designed to evaluate technologies across different care settings from the perspective of several stakeholders. Health states were based on six care need profiles with increasing need for toileting assistance, three care stages with varying degrees of toileting success, and five levels of skin health. The main outcomes were incremental costs and quality-adjusted life years. The effectiveness of the TENA SmartCare Identifi was based primarily on trial data combined with literature and expert opinion where necessary. Costs were reported in CAD 2020. After 2 years, 21% of residents in the DHT group received mainly toileting as their continence care strategy compared with 12% in the conventional care group. Conversely, with the DHT 15% of residents rely mainly on absorbent products for incontinence care, compared with 40% with conventional care. On average, residents lived for 2.34 years, during which the DHT resulted in a small gain in quality-adjusted life years of 0.015 and overall cost-savings of $1,467 per resident compared with conventional care. Most cost-savings were achieved through reduced costs for absorbent products. Since most, if not all, stakeholders gain from use of the DHT-assisted PCCC, widespread use in Canadian residential care facilities should be considered, and similar assessments for other countries encouraged.
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Salud Digital , Servicios de Atención de Salud a Domicilio , Humanos , Canadá , Análisis Costo-Beneficio , Casas de Salud , Modelos EconómicosRESUMEN
The aim of this study was to perform a 1-yr trial-based cost-effectiveness analysis (CEA) of tiotropium versus salmeterol followed by a 5-yr model-based CEA. The within-trial CEA, including 7,250 patients with moderate to very severe chronic obstructive pulmonary disease (COPD), was performed alongside the 1-yr international randomised controlled Prevention of Exacerbations with Tiotropium (POET)-COPD trial comparing tiotropium with salmeterol regarding the effect on exacerbations. Main end-points of the trial-based analysis were costs, number of exacerbations and exacerbation days. The model-based analysis was conducted to extrapolate results to 5 yrs and to calculate quality-adjusted life years (QALYs). 1-yr costs per patient from the German statutory health insurance (SHI) perspective and the societal perspective were 126 (95% uncertainty interval (UI) 55-195) and 170 (95% UI 77-260) higher for tiotropium, respectively. The annual number of exacerbations was 0.064 (95% UI 0.010-0.118) lower for tiotropium, leading to a reduction in exacerbation-related costs of 87 (95% UI 19-157). The incremental cost-effectiveness ratio was 1,961 per exacerbation avoided from the SHI perspective and 2,647 from the societal perspective. In the model-based analyses, the 5-yr costs per QALY were 3,488 from the SHI perspective and 8,141 from the societal perspective. Tiotropium reduced exacerbations and exacerbation-related costs, but increased total costs. Tiotropium can be considered cost-effective as the resulting cost-effectiveness ratios were below commonly accepted willingness-to-pay thresholds.
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Albuterol/análogos & derivados , Broncodilatadores/economía , Enfermedad Pulmonar Obstructiva Crónica/economía , Derivados de Escopolamina/economía , Anciano , Albuterol/economía , Teorema de Bayes , Broncodilatadores/administración & dosificación , Análisis Costo-Beneficio , Método Doble Ciego , Femenino , Costos de la Atención en Salud , Humanos , Masculino , Persona de Mediana Edad , Modelos Económicos , Probabilidad , Enfermedad Pulmonar Obstructiva Crónica/tratamiento farmacológico , Años de Vida Ajustados por Calidad de Vida , Xinafoato de Salmeterol , Derivados de Escopolamina/uso terapéutico , Bromuro de Tiotropio , Resultado del TratamientoRESUMEN
BACKGROUND: There is an 80% prevalence of two or more psychiatric symptoms in psychogeriatric patients. Multiple psychiatric symptoms (MPS) have many negative effects on quality of life of the patient as well as on caregiver burden and competence. Irrespective of the effectiveness of an intervention programme, it is important to take into account its economic aspects. METHODS: The economic evaluation was performed alongside a single open RCT and conducted between 2001 and 2006. The patients who met the selection criteria were asked to participate in the RCT. After the patient or his caregiver signed a written informed consent form, he was then randomly assigned to either IRR or UC.The costs and effects of IRR were compared to those of UC. We assessed the cost-utility of IRR as well as the cost-effectiveness of both conditions. Primary outcome variable: severity of MPS (NPI) of patients; secondary outcome variables: general caregiver burden (CB) and caregiver competence (CCL), quality of life (EQ5D) of the patient, and total medical costs per patient (TiC-P). Cost-utility was evaluated on the basis of differences in total medical costs). Cost-effectiveness was evaluated by comparing differences of total medical costs and effects on NPI, CB and CCL (Incremental Cost-Effectiveness Ratio: ICER). CEAC-analyses were performed for QALY and NPI-severity. All significant testing was fixed at p<0.05 (two-tailed). The data were analyzed according to the intention-to-treat (ITT)-principle. A complete cases approach (CC) was used. RESULTS: IRR turned out to be non-significantly, 10.5% more expensive than UC ( 36 per day). The number of QALYs was 0.01 higher (non-significant) in IRR, resulting in 276,290 per QALY. According to the ICER-method, IRR was significantly more cost-effective on NPI-sum-severity of the patient (up to 34%), CB and CCL (up to 50%), with ICERs varying from 130 to 540 per additional point of improvement. CONCLUSIONS: No significant differences were found on QALYs. In IRR patients improved significantly more on severity of MPS, and caregivers on general burden and competence, with incremental costs varying from 130 to 540 per additional point of improvement. The surplus costs of IRR are considered acceptable, taking into account the high societal costs of suffering from MPS of psychogeriatric patients and the high burden of caregivers. The large discrepancy in economic evaluation between QALYs (based on EQ5D) and ICERs (based on clinically relevant outcomes) demands further research on the validity of EQ5D in psychogeriatric cost-utility studies. (Trial registration nr.: ISRCTN 38916563; December 2004).
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Prestación Integrada de Atención de Salud/métodos , Casas de Salud/organización & administración , Psicoterapia/métodos , Anciano , Anciano de 80 o más Años , Trastornos del Conocimiento/economía , Trastornos del Conocimiento/terapia , Análisis Costo-Beneficio , Prestación Integrada de Atención de Salud/economía , Femenino , Evaluación Geriátrica , Humanos , Masculino , Pruebas Neuropsicológicas , Casas de Salud/economía , Psicoterapia/economía , Años de Vida Ajustados por Calidad de VidaRESUMEN
BACKGROUND: Heart failure is a major health concern associated with significant morbidity, mortality, and reduced quality of life in patients. Home telemonitoring (HTM) facilitates frequent or continuous assessment of disease signs and symptoms, and it has shown to improve compliance by involving patients in their own care and prevent emergency admissions by facilitating early detection of clinically significant changes. Diagnostic algorithms (DAs) are predictive mathematical relationships that make use of a wide range of collected data for calculating the likelihood of a particular event and use this output for prioritizing patients with regard to their treatment. OBJECTIVE: This study aims to assess the cost-effectiveness of HTM and a DA in the management of heart failure in the Netherlands. Three interventions were analyzed: usual care, HTM, and HTM plus a DA. METHODS: A previously published discrete event simulation model was used. The base-case analysis was performed according to the Dutch guidelines for economic evaluation. Sensitivity, scenario, and value of information analyses were performed. Particular attention was given to the cost-effectiveness of the DA at various levels of diagnostic accuracy of event prediction and to different patient subgroups. RESULTS: HTM plus the DA extendedly dominates HTM alone, and it has a deterministic incremental cost-effectiveness ratio compared with usual care of 27,712 (currency conversion rate in purchasing power parity at the time of study: 1=US $1.29; further conversions are not applicable in cost-effectiveness terms) per quality-adjusted life year. The model showed robustness in the sensitivity and scenario analyses. HTM plus the DA had a 96.0% probability of being cost-effective at the appropriate 80,000 per quality-adjusted life year threshold. An optimal point for the threshold value for the alarm of the DA in terms of its cost-effectiveness was estimated. New York Heart Association class IV patients were the subgroup with the worst cost-effectiveness results versus usual care, while HTM plus the DA was found to be the most cost-effective for patients aged <65 years and for patients in New York Heart Association class I. CONCLUSIONS: Although the increased costs of adopting HTM plus the DA in the management of heart failure may seemingly be an additional strain on scarce health care resources, the results of this study demonstrate that, by increasing patient life expectancy by 1.28 years and reducing their hospitalization rate by 23% when compared with usual care, the use of this technology may be seen as an investment, as HTM plus the DA in its current form extendedly dominates HTM alone and is cost-effective compared with usual care at normally accepted thresholds in the Netherlands.
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OBJECTIVES: To assess the cost-effectiveness of various combinations of urate lowering therapy (ULT) and anti-inflammatory treatment in the management of newly diagnosed gout patients, from the Dutch societal perspective. METHODS: A probabilistic patient-level simulation estimating costs and quality-adjusted life years (QALYs) comparing gout and hyperuricemia treatment strategies was performed. ULT options febuxostat, allopurinol and no ULT were considered. Flare treatments naproxen, colchicine, prednisone, and anakinra were considered. A Markov Model was constructed to simulate gout disease. Health states were no flare, and severe pain, mild pain, moderate pain, or no pain in the presence of a flare. Model input was derived from patient level clinical trial data, meta-analyses or from previously published health-economic evaluations. The results of probabilistic sensitivity analyses were presented using incremental cost-effectiveness ratios (ICERs), and summarized using cost-effectiveness acceptability curves (CEACs). Scenario analyses were performed. RESULTS: The ICER for allopurinol versus no ULT was 1,381, when combined with naproxen. Febuxostat yielded the highest utility, but also the highest costs (4,385 vs. 4,063 for allopurinol), resulting in an ICER of 25,173 when compared to allopurinol. No ULT was not cost-effective, yielding the lowest utility. For the gout flare medications, comparable effects on utility were achieved. Combined with febuxostat, naproxen was the cheapest option (4,404), and anakinra the most expensive (4,651). The ICER of anakinra compared to naproxen was 818,504. Colchicine and prednisone were dominated by naproxen. CONCLUSION: Allopurinol and febuxostat were both cost-effective compared to No ULT. Febuxostat was cost-effective in comparison with allopurinol at higher willingness-to-pay thresholds. For treating gout flares, colchicine, naproxen and prednisone offered comparable health economic implications, although naproxen was the favoured option.
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Quimioterapia Combinada , Supresores de la Gota , Gota , Modelos Económicos , Ácido Úrico/sangre , Antiinflamatorios/economía , Antiinflamatorios/uso terapéutico , Análisis Costo-Beneficio , Costos y Análisis de Costo , Gota/sangre , Gota/tratamiento farmacológico , Gota/economía , Supresores de la Gota/economía , Supresores de la Gota/uso terapéutico , HumanosRESUMEN
BACKGROUND: Real-world disease models spanning multiple treatment lines can provide insight into the (cost) effectiveness of treatment sequences in clinical practice. OBJECTIVE: Our objective was to explore whether a disease model based solely on real-world data (RWD) could be used to estimate the effectiveness of treatments for patients with castration-resistant prostate cancer (CRPC) that could then be suitably used in a cost-effectiveness analysis. METHODS: We developed a patient-level simulation model using patient-level data from the Dutch CAPRI registry as input parameters. Time to event (TTE) and overall survival (OS) were estimated with multivariate regression models, and type of event (i.e., next treatment or death) was estimated with multivariate logistic regression models. To test internal validity, TTE and OS from the simulation model were compared with the observed outcomes in the registry. RESULTS: Although patient characteristics and survival outcomes of the simulated data were comparable to those in the observed data (median OS 20.6 vs. 19.8 months, respectively), the disease model was less accurate in estimating differences between treatments (median OS simulated vs. observed population: 18.6 vs. 17.9 [abiraterone acetate plus prednisone], 24.0 vs. 25.0 [enzalutamide], 20.2 vs. 18.7 [docetaxel], and 20.0 vs. 23.8 months [radium-223]). CONCLUSIONS: Overall, the disease model accurately approximated the observed data in the total CRPC population. However, the disease model was unable to predict differences in survival between treatments due to unobserved differences. Therefore, the model is not suitable for cost-effectiveness analysis of CRPC treatment. Using a combination of RWD and data from randomised controlled trials to estimate treatment effectiveness may improve the model.
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OBJECTIVES: To develop a stochastic population model of disease progression in chronic obstructive pulmonary disease (COPD) that includes the effects of COPD exacerbations on health-related quality of life, costs, disease progression, and mortality and can be used to assess the effects of a wide range of interventions. METHODS: The model is a multistate Markov model with time varying transition rates specified by age, sex, smoking status, COPD disease severity, and/or exacerbation type. The model simulates annual changes in COPD prevalence due to COPD incidence, exacerbations, disease progression (annual decline in the forced expiratory volume in 1 second as percentage of the predicted value), and mortality. The main outcome variables are quality-adjusted life years, total exacerbations, and COPD-related health care costs. Exacerbation-related input parameters were based on quantitative meta-analysis. All important model parameters are entered into the model as probability distributions. To illustrate the potential use of the model, costs and effects were calculated for 3-year implementation of three different COPD interventions, one pharmacologic, one on smoking cessation, and one on pulmonary rehabilitation using a time horizon of 10 years for reporting outcomes. RESULTS: Compared with minimal treatment the cost/quality-adjusted life year was 8,300 for the pharmacologic intervention, 10,800 for the smoking cessation therapy, 8,700 for the combination of the pharmacologic intervention and the smoking cessation therapy, and 17,200 for the pulmonary rehabilitation program. The probability of the interventions to be cost-effective at a ceiling ratio of 20,000 varied from 58% to 100%. CONCLUSIONS: The COPD model provides policy makers with information about the long-term costs and effects of interventions over the entire chain of care, from primary prevention to care for very severe COPD and includes uncertainty around the outcomes.
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Cadenas de Markov , Modelos Teóricos , Enfermedad Pulmonar Obstructiva Crónica/terapia , Calidad de Vida , Cese del Hábito de Fumar/métodos , Factores de Edad , Análisis Costo-Beneficio , Progresión de la Enfermedad , Costos de la Atención en Salud , Humanos , Dinámica Poblacional , Enfermedad Pulmonar Obstructiva Crónica/economía , Enfermedad Pulmonar Obstructiva Crónica/fisiopatología , Años de Vida Ajustados por Calidad de Vida , Índice de Severidad de la Enfermedad , Factores Sexuales , Fumar/efectos adversos , Procesos EstocásticosRESUMEN
INTRODUCTION: Hospitals are increasingly forced to consider the economics of technology use. We estimated the incremental cost-consequences of remifentanil-based analgo-sedation (RS) vs. conventional analgesia and sedation (CS) in patients requiring mechanical ventilation (MV) in the intensive care unit (ICU), using a modelling approach. METHODS: A Markov model was developed to describe patient flow in the ICU. The hourly probabilities to move from one state to another were derived from UltiSAFE, a Dutch clinical study involving ICU patients with an expected MV-time of two to three days requiring analgesia and sedation. Study medication was either: CS (morphine or fentanyl combined with propofol, midazolam or lorazepam) or: RS (remifentanil, combined with propofol when required). Study drug costs were derived from the trial, whereas all other ICU costs were estimated separately in a Dutch micro-costing study. All costs were measured from the hospital perspective (price level of 2006). Patients were followed in the model for 28 days. We also studied the sub-population where weaning had started within 72 hours. RESULTS: The average total 28-day costs were 15,626 with RS versus 17,100 with CS, meaning a difference in costs of 1474 (95% CI -2163, 5110). The average length-of-stay (LOS) in the ICU was 7.6 days in the RS group versus 8.5 days in the CS group (difference 1.0, 95% CI -0.7, 2.6), while the average MV time was 5.0 days for RS versus 6.0 days for CS. Similar differences were found in the subgroup analysis. CONCLUSIONS: Compared to CS, RS significantly decreases the overall costs in the ICU. TRIAL REGISTRATION: Clinicaltrials.gov NCT00158873.
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Analgesia/economía , Sedación Consciente/economía , Piperidinas/economía , Respiración Artificial/economía , Analgesia/tendencias , Sedación Consciente/tendencias , Análisis Costo-Beneficio/economía , Análisis Costo-Beneficio/tendencias , Estudios Cruzados , Estudios de Seguimiento , Humanos , Unidades de Cuidados Intensivos/economía , Unidades de Cuidados Intensivos/tendencias , Tiempo de Internación/economía , Tiempo de Internación/tendencias , Países Bajos/epidemiología , Piperidinas/uso terapéutico , Remifentanilo , Respiración Artificial/tendenciasRESUMEN
OBJECTIVES: To compare the cost-effectiveness of first-line gefitinib, erlotinib, afatinib, and osimertinib in patients with non-small cell lung cancer (NSCLC) harbouring epidermal growth factor receptor (EGFR) mutations. METHODS: A systematic review and network meta-analysis (NMA) were conducted to compare the relative efficacy of gefitinib, erlotinib, afatinib, and osimertinib in EGFR-mutated NSCLC. To assess the cost-effectiveness of these treatments, a Markov model was developed from Dutch societal perspective. The model was based on the clinical studies included in the NMA. Incremental costs per life-year (LY) and per quality-adjusted life-year (QALY) gained were estimated. Deterministic and probabilistic sensitivity analyses (PSA) were conducted. RESULTS: Total discounted per patient costs for gefitinib, erlotinib, afatinib, and osimertinib were 65,889, 64,035, 69,418, and 131,997, and mean QALYs were 1.36, 1.39, 1.52, and 2.01 per patient, respectively. Erlotinib dominated gefitinib. Afatinib versus erlotinib yielded incremental costs of 27,058/LY and 41,504/QALY gained. Osimertinib resulted in 91,726/LY and 128,343/QALY gained compared to afatinib. PSA showed that gefitinib, erlotinib, afatinib, and osimertinib had 13%, 19%, 43%, and 26% probability to be cost-effective at a threshold of 80,000/QALY. A price reduction of osimertinib of 30% is required for osimertinib to be cost-effective at a threshold of 80,000/QALY. CONCLUSIONS: Osimertinib has a better effectiveness compared to all other TKIs. However, at a Dutch threshold of 80,000/QALY, osimertinib appears not to be cost-effective.