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BACKGROUND: Clinical practice guidelines recommend indefinite anticoagulation for a first unprovoked venous thromboembolism (VTE). OBJECTIVE: To estimate the benefit-harm tradeoffs of indefinite anticoagulation in patients with a first unprovoked VTE. DESIGN: Markov modeling study. DATA SOURCES: Systematic reviews and meta-analyses for the long-term risks and case-fatality rates of recurrent VTE and major bleeding. Published literature for costs, quality of life, and other clinical events. TARGET POPULATION: Patients with a first unprovoked VTE who have completed 3 to 6 months of initial anticoagulant treatment. TIME HORIZON: Lifetime. PERSPECTIVE: Canadian health care public payer. INTERVENTION: Indefinite anticoagulation with direct oral anticoagulants. OUTCOME MEASURES: Recurrent VTE events, major bleeding events, costs in 2022 Canadian dollars (CAD), and quality-adjusted life-years (QALYs). RESULTS OF BASE-CASE ANALYSIS: When compared with discontinuing anticoagulation after initial treatment in a hypothetical cohort of 1000 patients aged 55 years, indefinite anticoagulation prevented 368 recurrent VTE events, which included 14 fatal pulmonary emboli, but induced an additional 114 major bleeding events, which included 30 intracranial hemorrhages and 11 deaths from bleeding. Indefinite anticoagulation cost CAD $16 014 more per person and did not increase QALYs (-0.075 per person). RESULTS OF SENSITIVITY ANALYSIS: Model results were most sensitive to the case-fatality rate of major bleeding and the annual risk for major bleeding during extended anticoagulation. LIMITATION: The model assumed that risks for recurrent VTE and major bleeding measured in clinical trials at 1 year remained constant during extended anticoagulation. CONCLUSION: Clinicians should use shared decision making to incorporate individual patient preferences and values when considering treatment duration for unprovoked VTE. PRIMARY FUNDING SOURCE: Canadian Institutes of Health Research.
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Tromboembolia Venosa , Humanos , Tromboembolia Venosa/tratamiento farmacológico , Tromboembolia Venosa/prevención & control , Análisis Costo-Beneficio , Calidad de Vida , Canadá , Anticoagulantes/efectos adversos , Hemorragia/inducido químicamente , RecurrenciaRESUMEN
PURPOSE: Breast cancer risk has conventionally been assessed using family history (FH) and rare high/moderate penetrance pathogenic variants (PVs), notably in BRCA1/2, and more recently PALB2, CHEK2, and ATM. In addition to these PVs, it is now possible to use increasingly predictive polygenic risk scores (PRS) as well. The comparative population-level predictive capability of these three different indicators of genetic risk for risk stratification is, however, unknown. METHODS: The Canadian heritable breast cancer risk distribution was estimated using a novel genetic mixing model (GMM). A realistically representative sample of women was synthesized based on empirically observed demographic patterns for appropriately correlated family history, inheritance of rare PVs, PRS, and residual risk from an unknown polygenotype. Risk assessment was simulated using the BOADICEA risk algorithm for 10-year absolute breast cancer incidence, and compared to heritable risks as if the overall polygene, including its measured PRS component, and PV risks were fully known. RESULTS: Generally, the PRS was most predictive for identifying women at high risk, while family history was the weakest. Only the PRS identified any women at low risk of breast cancer. CONCLUSION: PRS information would be the most important advance in enabling effective risk stratification for population-wide breast cancer screening.
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Neoplasias de la Mama , Proteína BRCA2/genética , Neoplasias de la Mama/diagnóstico , Neoplasias de la Mama/epidemiología , Neoplasias de la Mama/genética , Canadá/epidemiología , Femenino , Predisposición Genética a la Enfermedad , Humanos , Medición de Riesgo , Factores de RiesgoRESUMEN
INTRODUCTION: Acute poisoning represents a major cause of morbidity and mortality, and many of these patients are admitted to the intensive care unit (ICU). However, little is known regarding ICU costs of acute poisoning. METHODS: This was a retrospective matched database analysis of patients admitted to the ICU with acute poisoning from 2011 to 2014. It was performed in 2 ICUs within a single tertiary care hospital system. All patient information, outcomes, and costs were stored in the hospital data warehouse. Control patients were defined as randomly selected age-, sex-, severity index-, and comorbidity index-matched nonpoisoned ICU patients (1:4 matching ratio). RESULTS: A total of 8452 critically ill patients were admitted during the study period, of whom 277 had a diagnosis of acute poisoning. The mean age was 44.5 years, and the most common xenobiotics implicated were sedative hypnotics (20.2%), antidepressants (15.2%), and opioids (10.5%). Of these, 73.6% of poisonings were deemed intentional. In-hospital mortality of poisoned patients was 5.1%, compared to 11.1% for control patients (P < .01). The median ICU length of stay (LOS) for poisoned patients was 3.0 days, compared with 4.0 days for control patients (P < .01). The mean total cost for poisoned patients was CAD$18 958. Control patients had a significantly higher mean total cost of CAD$60 628 (P < .01). The xenobiotics associated with the highest costs were acetaminophen (CAD$18 585), toxic alcohols (CAD$16 771), and opioids (CAD$12 967). CONCLUSIONS: In our cohort, we confirmed the long-held belief that patients admitted to the ICU with a primary diagnosis of poisoning have a lower mortality rate, ICU LOS, and overall cost per ICU admission than nonpoisoned patients. However, poisoned patients still accrue significant daily costs, with the highest costs attributed to xenobiotics with known antidotes, such as acetaminophen, toxic alcohols, and opioids.
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Cuidados Críticos/economía , Costos de la Atención en Salud/estadística & datos numéricos , Hospitalización/economía , Unidades de Cuidados Intensivos/economía , Intoxicación/economía , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Estudios de Casos y Controles , Resultados de Cuidados Críticos , Femenino , Mortalidad Hospitalaria , Humanos , Masculino , Persona de Mediana Edad , Intoxicación/mortalidad , Estudios Retrospectivos , Índice de Severidad de la Enfermedad , Adulto JovenRESUMEN
BACKGROUND: Bronchopulmonary dysplasia (BPD) is among the most severe chronic lung diseases and predominantly affects premature infants. There is a general understanding of BPD's significant impact on the short-term outcomes however there is little evidence on long-term outcomes. Our study estimates the lifetime clinical outcomes, quality of life, and healthcare costs of BPD and associated complications. METHODS: We developed a microsimulation model to estimate lifetime clinical and economic burden of BPD among extreme preterm infants (≤28 weeks gestational age at birth) and validated it against the best available Canadian data. We further estimate the cumulative incidence of major complications associated with BPD, differentiated by BPD severity and gestational age category. RESULTS: We find, on average, patients with BPD and resulting complications will incur over CAD$700,000 in lifetime health systems costs. We also find the average life expectancy of BPD patients to be moderately less than that of the general population and significant reductions in quality-adjusted life year due to major complications. Healthcare utilization and quality of life measures vary dramatically according to BPD severity, suggesting significant therapeutic headroom for interventions that can prevent or mitigate the effects of BPD for patients. CONCLUSIONS: Our study adds a significant expansion of existing evidence by presenting the lifetime burden of BPD based on key patient characteristics. Given the extreme cost burden at the earliest stage of life and lifetime negative impact on quality of life, there is larger headroom for investment in prevention and mitigation of severe BPD than is currently available.
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Displasia Broncopulmonar , Atención a la Salud , Displasia Broncopulmonar/epidemiología , Displasia Broncopulmonar/terapia , Canadá , Atención a la Salud/estadística & datos numéricos , Edad Gestacional , Humanos , Lactante , Recién Nacido , Recien Nacido Prematuro , Calidad de Vida , Resultado del TratamientoRESUMEN
BACKGROUND.: This study estimates the maximum price at which mesenchymal stem cell (MSC) therapy is deemed cost-effective for septic shock patients and identifies parameters that are most important in making treatment decisions. METHODS: We developed a probabilistic Markov model according to the sepsis care trajectory to simulate costs and quality-adjusted life years (QALYs) of septic shock patients receiving either MSC therapy or usual care over their lifetime. We calculated the therapeutic headroom by multiplying the gains attributable to MSCs with willingness-to-pay (WTP) threshold and derived the maximum reimbursable price (MRP) from the expected net monetary benefit and savings attributable to MSCs. We performed scenario analyses to assess the impact of changes to assumptions on the study findings. A value of information analysis is performed to identify parameters with greatest impact on the uncertainty around the cost-effectiveness of MSC therapy. RESULTS: At a WTP threshold of $50,000 per QALY, the therapeutic headroom and MRP of MSC therapy were $20,941 and $16,748, respectively; these estimates increased with the larger WTP values and the greater impact of MSCs on in-hospital mortality and hospital discharge rates. The parameters with greatest information value were MSC's impact on in-hospital mortality and the baseline septic shock in-hospital mortality. CONCLUSION: At a common WTP of $50,000/QALY, MSC therapy is deemed to be economically attractive if its unit cost does not exceed $16,748. This ceiling price can be increased to $101,450 if the therapy significantly reduces both in-hospital mortality and increases hospital discharge rates.
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Análisis Costo-Beneficio , Economía Médica , Trasplante de Células Madre Mesenquimatosas/economía , Choque Séptico/terapia , Anciano , Análisis Costo-Beneficio/estadística & datos numéricos , Mortalidad Hospitalaria/tendencias , Humanos , Unidades de Cuidados Intensivos , Cadenas de Markov , Persona de Mediana Edad , Modelos Económicos , Alta del Paciente/estadística & datos numéricos , Años de Vida Ajustados por Calidad de Vida , Evaluación de la Tecnología Biomédica , Estados Unidos/epidemiologíaRESUMEN
OBJECTIVE: To compare costs and quality-adjusted life years of patients receiving endoscopic polypectomy in the clinic those receiving endoscopic sinus surgery from the perspective of Canada's health system. METHOD: We developed a Markov model to simulate costs and quality-adjusted life years among a cohort of patients with chronic rhinosinusitis with polyps who received endoscopic polypectomy in clinic or endoscopic sinus surgery, over a patient's lifetime. Our study focused on patients with chronic rhinosinusitis with polyps who have the predominant symptom of bilateral nasal obstruction. Cost data were obtained from Canadian sources. Health utility values were derived from Sino-nasal Outcome Test (SNOT-22) scores reported in the published literature. A cycle length of six months was used. Both costs and quality-adjusted life years were discounted using an annual rate of 1.5%. We conducted one-way and probabilistic sensitivity analyses to assess the robustness of the study findings. RESULTS: Endoscopic polypectomy in clinic was a cost-saving option as it had a lower cost (C$736 vs. C$6,728) and produced more quality-adjusted life years (13.85 vs 13.06) compared to endoscopic sinus surgery. Our findings were, however, highly sensitive to health utility values. Results from probabilistic sensitivity analysis showed that endoscopic polypectomy in clinic had a lower cost and was more effective compared to endoscopic sinus surgery in 99.86% of iterations. CONCLUSION: From the perspective of the publicly funded healthcare system, endoscopic polypectomy in clinic was economically attractive compared to endoscopic sinus surgery for chronic rhinosinusitis with polyps patients who have a primary symptom of bilateral nasal obstruction. The cost-effectiveness results are, however, highly dependent on the relative impact of endoscopic polypectomy in the clinic and endoscopic sinus surgery on post-surgery health utility values.
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Endoscopía/economía , Endoscopía/métodos , Obstrucción Nasal/cirugía , Pólipos Nasales/cirugía , Años de Vida Ajustados por Calidad de Vida , Rinitis/cirugía , Sinusitis/cirugía , Canadá , Análisis Costo-Beneficio , Femenino , Humanos , Masculino , Cadenas de MarkovAsunto(s)
Animales de Laboratorio , Investigación Biomédica/estadística & datos numéricos , Países Desarrollados/estadística & datos numéricos , Países en Desarrollo/estadística & datos numéricos , Publicaciones Periódicas como Asunto/ética , Edición/ética , Investigadores/estadística & datos numéricos , Animales , Autoria , Reacción de Prevención , Investigación Biomédica/normas , Ensayos Clínicos como Asunto/estadística & datos numéricos , Países Desarrollados/economía , Países en Desarrollo/economía , Guías como Asunto , Humanos , Internacionalidad , National Institutes of Health (U.S.)/economía , Publicación de Acceso Abierto/economía , Publicación de Acceso Abierto/estadística & datos numéricos , Publicación de Acceso Abierto/provisión & distribución , Publicaciones Periódicas como Asunto/normas , Edición/normas , Edición/provisión & distribución , Investigadores/educación , Investigadores/ética , Investigadores/normas , Apoyo a la Investigación como Asunto/normas , Apoyo a la Investigación como Asunto/estadística & datos numéricos , Literatura de Revisión como Asunto , Estados UnidosRESUMEN
BACKGROUND: Liver biopsy remains the gold standard for the diagnosis of liver fibrosis, but its use as a diagnostic tool is limited by its invasive nature and high cost. OBJECTIVE: The aim of this study was to systematically review the cost-effectiveness of transient elastography (TE) with and without controlled attenuation parameter (CAP) for the diagnosis of liver fibrosis or steatosis in patients with hepatitis B, hepatitis C, alcoholic liver disease and non-alcoholic fatty liver disease. METHODS: An economic literature search was performed. Eligibility criteria included systematic reviews, health technology assessments or economic evaluations of TE compared to liver biopsy and other non-invasive tests. After abstract screening, full-text reports of potentially relevant articles were assessed in duplicate. The methodological quality of the included studies was also appraised. RESULTS: The database search yielded 253 records; four cost-effectiveness and four cost-utility studies were included. The methodological quality of the included studies varies. High-quality cost-effectiveness studies not only suggested that TE is less costly but also less accurate than liver biopsy. The incremental cost-effectiveness ratio (ICER) of TE improves with a greater level of diagnostic accuracy and a higher degree of liver fibrosis. High-quality cost-utility studies indicated that TE is a cost-effective alternative to biopsy with ICER between $9000 and $14 000 per QALY for patients with hepatitis C. We did not find studies that assessed the cost-effectiveness of TE with CAP for the diagnosis of liver steatosis. CONCLUSIONS: Transient elastography is an economically attractive alternative to liver biopsy and other non-invasive diagnostic tests especially for patients with a higher degree of liver fibrosis.
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Análisis Costo-Beneficio , Diagnóstico por Imagen de Elasticidad/economía , Cirrosis Hepática/diagnóstico por imagen , Biopsia/economía , Hepatitis B/complicaciones , Hepatitis C/complicaciones , Humanos , Cirrosis Hepática/patología , Hepatopatías Alcohólicas/complicaciones , Enfermedad del Hígado Graso no Alcohólico/complicacionesRESUMEN
BACKGROUND: Diabetic retinopathy (DR) is one of the leading causes of vision loss and blindness in Canada. Eye examinations play an important role in early detection. However, DR screening by optometrists is not always universally covered by public or private health insurance plans. This study assessed whether expanding public health coverage to include diabetic eye examinations for retinopathy by optometrists is cost-effective from the perspective of the health care system. METHODS: We conducted a cost-utility analysis of extended coverage for diabetic eye examinations in Prince Edward Island to include examinations by optometrists, not currently publicly covered. We used a Markov chain to simulate disease burden based on eye examination rates and DR progression over a 30-year time horizon. Results were presented as an incremental cost per quality-adjusted life year (QALY) gained. A series of one-way and probabilistic sensitivity analyses were performed. RESULTS: Extending public health coverage to eye examinations by optometrists was associated with higher costs ($9,908,543.32) and improved QALYs (156,862.44), over 30 years, resulting in an incremental cost-effectiveness ratio of $1668.43/QALY gained. Sensitivity analysis showed that the most influential determinants of the results were the cost of optometric screening and selected utility scores. At the commonly used threshold of $50,000/QALY, the probability that the new policy was cost-effective was 99.99%. CONCLUSIONS: Extending public health coverage to eye examinations by optometrists is cost-effective based on a commonly used threshold of $50,000/QALY. Findings from this study can inform the decision to expand public-insured optometric services for patients with diabetes.
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Retinopatía Diabética/diagnóstico , Cobertura del Seguro/economía , Tamizaje Masivo/métodos , Programas Nacionales de Salud/economía , Años de Vida Ajustados por Calidad de Vida , Anciano , Canadá , Costo de Enfermedad , Análisis Costo-Beneficio , Retinopatía Diabética/economía , Progresión de la Enfermedad , Humanos , Cadenas de Markov , Tamizaje Masivo/economía , Persona de Mediana Edad , Optometristas/economía , Isla del Principe Eduardo , Probabilidad , Factores de TiempoRESUMEN
The coronavirus disease 2019 (COVID-19) pandemic has increased public awareness of the influence of epidemiological and economic decision models on public policy decisions. Alongside this is an increased scrutiny on the development, analysis, reporting and utilisation of decision models for public policy making. Therefore, it is important that model developers can clearly explain and justify to all stakeholders what is included and excluded from a model developed to support decision-making, to both improve transparency and trust in decision-making. Our aim is to provide tools for improving communication between modellers and decision-makers, leading to improved transparency in decision-making. To do so, we extend the recently described directed acyclic graphs with omitted objects displayed (DAGWOOD) approach from Haber et al. (Ann Epidemiol 68:64-71, 2022) to decision analytic models, giving the decision analytic models with omitted objects displayed (DAMWOOD) approach. DAMWOOD is a framework for the identification of objects omitted from a decision model, as well as for consideration of the effects of omissions on model outcomes. Objects omitted from a decision model are classed as either an exclusion (known and unknown confounders), misdirection (alternative model pathways) or structure (e.g. model type, methods for estimating relationships between objects). DAMWOOD requires model developers to use explicit statements and provide illustration of included and omitted objects, supporting communication with model users and stakeholders, allowing them to provide input and feedback to modellers about which objects to include or omit in a model. In developing DAMWOOD, we considered two challenges we encountered in modelling for pandemic policy response. First, the scope of the decision problem is not always made sufficiently explicit by decision-makers, requiring modellers to intuit which policy options should be considered, and/or which outcomes should be considered in their evaluation. Second, there is rarely sufficient transparency to ensure stakeholders can see what is included in models and why. This limits stakeholders' ability to advocate to decision-makers for the prioritisation of specific outcomes and challenge the model results. To illustrate the application of DAMWOOD, we apply it to a previously published COVID-19 vaccine allocation optimisation model. The DAMWOOD diagrams illustrate the ways in which it is possible to improve the communication of model assumptions. The diagrams make explicit which outcomes are omitted and provide information on the expected impact of the omissions on model results. We discuss the usefulness of DAMWOOD for framing the decision problem, communicating the model structure and results and engaging with those making and affected by the decisions the model is developed to inform.
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COVID-19 , Toma de Decisiones , Técnicas de Apoyo para la Decisión , Humanos , Formulación de Políticas , ComunicaciónRESUMEN
INTRODUCTION: Deciding whether to stop or extend anticoagulant therapy indefinitely after completing at least 3 months of initial treatment for a first unprovoked venous thromboembolism (VTE) remains a challenge for clinicians, patients and policy makers. Guidelines suggest an indefinite duration of anticoagulant therapy in these patients, yet its benefits, harms and costs have not been formally assessed. The aim of this proposed modelling study is to assess the differences in clinical benefits, harms and costs of stopping versus continuing anticoagulant therapy indefinitely for a first unprovoked VTE. METHODS AND ANALYSIS: We will develop a probabilistic Markov model, adopting a 1-month cycle length and a lifetime horizon, to estimate life-years, quality-adjusted life-years, costs and the incremental cost-effectiveness ratios for a simulated population of patients with a first unprovoked VTE who will receive indefinite duration of anticoagulant therapy versus a population who will not receive extended treatment after completing 3 months of initial anticoagulant therapy. The economic evaluation will adopt a third-party payer perspective relating to a Canadian publicly funded healthcare system. Estimates for the probability of relevant clinical events will be informed by systematic reviews and meta-analyses, while costs and utility values will be obtained from published Canadian sources. Stratified analyses based on sex, age and site of initial VTE will also be performed to identify subgroups of patients with a first unprovoked VTE in whom continuing anticoagulant therapy indefinitely might prove to be clinically beneficial and cost-effective over stopping treatment. We will also conduct sensitivity and scenario analyses to assess robustness of study findings to changes in individual or groups of key parameters. ETHICS AND DISSEMINATION: Ethical approval is not applicable for this study. The results will be disseminated through presentations at relevant conferences and in a manuscript that will be submitted to a peer-reviewed journal.
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Tromboembolia Venosa , Humanos , Tromboembolia Venosa/tratamiento farmacológico , Tromboembolia Venosa/inducido químicamente , Análisis Costo-Beneficio , Canadá , Anticoagulantes/efectos adversosRESUMEN
BACKGROUND: Although outcome studies generally demonstrate the superiority of a total shoulder arthroplasty (TSA) over a hemiarthroplasty (HA), comparative cost-effectiveness has not been well studied. From a publicly funded health-care system's perspective, this study compared the costs and quality-adjusted life-years (QALYs) in patients who underwent TSA with those in patients who underwent HA. METHODS: We conducted a cost-utility analysis using a Markov model to simulate the costs and QALYs for patients undergoing either TSA or HA over a lifetime horizon to account for costs and medically important events over the patient lifetime. Subgroup analyses by age groups (≤50 or >50 years) were performed. A series of sensitivity analyses were performed to assess robustness of study findings. The results were presented in 2019 U.S. dollars. RESULTS: TSA was dominant as it was less costly ($115,785 compared with $118,501) and more effective (10.21 compared with 8.47 QALYs) than HA over a lifetime horizon. Changes to health utility values after TSA and HA had the largest impact on the cost-effectiveness findings. At a willingness-to-pay (WTP) threshold of $50,000 per QALY gained, HA was not found to be cost-effective. The probability that TSA was cost-effective was 100%. CONCLUSIONS: Based on a WTP of $50,000 per QALY gained, from the perspective of Canada's publicly funded health-care system, TSA was found to be cost-effective in all patients, including those ≤50 years of age, compared with HA. LEVEL OF EVIDENCE: Economic and Decision Analysis Level II. See Instructions for Authors for a complete description of levels of evidence.
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Artritis Reumatoide/cirugía , Artroplastía de Reemplazo de Hombro/economía , Hemiartroplastia/economía , Osteoartritis de la Cadera/cirugía , Años de Vida Ajustados por Calidad de Vida , Anciano , Artritis Reumatoide/economía , Artroplastía de Reemplazo de Hombro/estadística & datos numéricos , Análisis Costo-Beneficio/estadística & datos numéricos , Femenino , Hemiartroplastia/estadística & datos numéricos , Humanos , Masculino , Persona de Mediana Edad , Osteoartritis de la Cadera/economía , Reoperación/economía , Reoperación/estadística & datos numéricos , Estudios Retrospectivos , Articulación del Hombro/cirugía , Resultado del TratamientoRESUMEN
INTRODUCTION: Variable costs of different radiation treatment modalities have played an important factor in selecting the most appropriate treatment for patients with intermediate-risk prostate cancer. METHODS: Analysis using a Markov model was conducted to simulate 20-year disease trajectory, quality-adjusted life years (QALYs) and health system costs of a cohort of intermediate-risk prostate cancer patients with mean age of 60 years. Clinical outcomes on toxicity and disease recurrence were measured and a probabilistic sensitivity analysis was performed, varying input parameters simultaneously according to their distributions. RESULTS: Among the six radiation treatment modalities, including conventionally fractionated intensity-modulated radiation therapy (IMRT), hypofractionated IMRT, IMRT combined with high-dose-rate (HDR) brachytherapy, HDR brachytherapy monotherapy, low-dose-rate brachytherapy monotherapy, and stereotactic body radiotherapy (SBRT), SBRT was found to be more cost-effective when compared with LDR-b and other treatment modalities, resulting in an incremental cost-utility ratio of $2985 per QALY. CONCLUSIONS: Stereotactic body radiotherapy is the most cost-effective radiation treatment modality in treatment of intermediate-risk prostate cancer, while treatment toxicity and cost data are the key drivers of the cost-utility. Further work is required with long-term follow-up for SBRT.
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Braquiterapia , Neoplasias de la Próstata , Radioterapia de Intensidad Modulada , Análisis Costo-Beneficio , Humanos , Masculino , Persona de Mediana Edad , Recurrencia Local de Neoplasia , Neoplasias de la Próstata/radioterapiaRESUMEN
Cell-based therapies hold promise to substantially curb complications from extreme preterm birth, the main cause of death in children below the age of 5 years. Exciting preclinical studies in experimental neonatal lung injury have provided the impetus for the initiation of early phase clinical trials in extreme preterm infants at risk of developing bronchopulmonary dysplasia. Clinical translation of promising therapies, however, is slow and often fails. In the adult population, results of clinical trials so far have not matched the enticing preclinical data. The neonatal field has experienced many hard-earned lessons with the implementation of oxygen therapy or postnatal steroids. Here we briefly summarize the preclinical data that have permitted the initiation of early phase clinical trials of cell-based therapies in extreme preterm infants and describe the INCuBAToR concept (Innovative Neonatal Cellular Therapy for Bronchopulmonary Dysplasia: Accelerating Translation of Research), an evidence-based approach to mitigate the risk of translating advanced therapies into this vulnerable patient population. The INCuBAToR addresses several of the shortcomings at the preclinical and the clinical stage that usually contribute to the failure of clinical translation through (a) systematic reviews of preclinical and clinical studies, (b) integrated knowledge transfer through engaging important stakeholders early on, (c) early economic evaluation to determine if a novel therapy is viable, and (d) retrospective and prospective studies to define and test ideal eligibility criteria to optimize clinical trial design. The INCuBAToR concept can be applied to any novel therapy in order to enhance the likelihood of success of clinical translation in a timely, transparent, rigorous, and evidence-based fashion.
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Displasia Broncopulmonar , Tratamiento Basado en Trasplante de Células y Tejidos , Nacimiento Prematuro , Displasia Broncopulmonar/terapia , Ensayos Clínicos como Asunto , Humanos , Recién Nacido , Recien Nacido PrematuroRESUMEN
Breast cancer is the most common cancer in women worldwide. Most current guidelines recommend using multigene profiling assays to aid the decision on the addition of chemotherapy to adjuvant hormone therapy for women who present with early-stage, hormone receptor-positive, HER2-negative disease. One of these assays is the Oncotype DX, which predicts the disease recurrence risk and adjuvant chemotherapy benefits. Given its high cost, there is an economic incentive to evaluate its surrogates, such as the Magee equations. We assessed health system costs associated with the use of the Magee scores. A probabilistic decision tree was used to calculate the difference in mean health system costs based on data obtained from a randomized trial and the published literature. Costs were calculated from a perspective of Canada's publicly funded health care system. A series of sensitivity analysis was conducted to assess the robustness of the study findings. The Magee equations were associated with a total cost savings of C$100 per patient (95% CI, -C$3068 to C$5022) compared with standard of care. The difference in costs was highly sensitive to the extent that the Magee scores could reduce the frequency of adjuvant chemotherapy and Oncotype DX requests.
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Neoplasias de la Mama , Neoplasias de la Mama/tratamiento farmacológico , Neoplasias de la Mama/genética , Costos y Análisis de Costo , Pruebas Diagnósticas de Rutina , Femenino , Humanos , RiesgoRESUMEN
Background: There is increasing evidence that premature newborns and infants with low birth weight can benefit substantially from an exclusive human milk-based diet (EHMD), consisting of human milk supplemented with a pasteurized donor human milk-derived fortifier. However, compared with the standard infant diet, EHMD also represents a significant added cost to the hospital and/or health system, thereby raising important questions about the economic feasibility of incorporating EHMD into newborn care. Design: We conducted a cost analysis and estimated the potential cost savings to a Canadian tertiary hospital based on the attributable complications averted from EHMD among low-weight neonates. A meta-analysis was performed to derive input parameters. A probabilistic analysis was conducted to determine the probability that EHMD is cost saving and 95% confidence interval (CI) around our estimates. Results: Our findings show that providing EHMD to preterm infants under 750 g at birth and at the highest risk of developing major complications is likely to be cost saving in the amount of $107,567 (95% CI: -145,229 to 360,362) per year. Extending EHMD to higher weight classes may be economically feasible depending on the pricing of the human milk-derived fortifier and the baseline risk of complications in the hospital setting. Conclusions: This comprehensive study provides critical insight for hospital-based decision makers to evaluate the potential gains and uncertainties associated with improved nutritional care for neonatal patients.
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Lactancia Materna/economía , Dieta/economía , Recién Nacido de muy Bajo Peso , Leche Humana , Canadá , Femenino , Hospitales , Humanos , Lactante , Fenómenos Fisiológicos Nutricionales del Lactante , Recién Nacido , Recien Nacido PrematuroRESUMEN
BACKGROUND: In the Canadian health care system, determining overall costs associated with a particular diagnostic subgroup of patients, in this case dizzy patients, is the first step in the process of determining where costs could be saved without compromising patient care. This study is the first Canadian study that evaluates these costs at a tertiary care hospital and will allow for the extrapolation of cost data for other similar academic health science centers, regional health initiatives, and provincial healthcare planning structures. METHODS: We conducted a retrospective cohort study of patients of any age presenting to The Ottawa Hospital (TOH), a tertiary care hospital, between January 1st, 2009 and December 31st, 2014 with a main diagnosis of dizziness or dizziness-related disease. De-identified patient information was acquired through TOH Data Warehouse and included a patient's sex, age, arrival and departure dates, Elixhauser co-morbidity score, location of presentation (emergency department or admitted inpatient) presenting complaint, final diagnosis code, any procedure codes linked to their care, and the direct and indirect hospital costs linked with any admission. We derived the mean hospital costs and 95% confidence interval for each diagnosis. We obtained the number of patients who were diagnosed with dizziness within Ontario in year 2015-16 from Canadian Institute for Health Information (CIHI). A simple frequency multiplication was performed to estimate the total cost burden for Ontario based on the cost estimate for the same year obtained from TOH. Cost data were presented in 2017 Canadian dollars. RESULTS: The average total hospital cost per patient with dizziness for the entire cohort is $450 (SD = $1334), with ED only patients costing $359 (SD = $214). The total estimated hospital cost burden of dizziness in Ontario is $31,202,000 (95% CI $29,559,000 - 32,844,000). CONCLUSIONS: The estimated annual costs of emergency department ambulatory and inpatient dizziness in Ontario was calculated to be approximately 31 million dollars per year. This is the first step in identifying potential areas for cost savings to aid local and provincial policy-makers in allocation of health care spending.
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Mareo/economía , Costos de la Atención en Salud , Centros de Atención Terciaria/economía , Adulto , Anciano , Ahorro de Costo , Mareo/diagnóstico , Mareo/terapia , Servicio de Urgencia en Hospital/economía , Femenino , Hospitalización/economía , Humanos , Masculino , Persona de Mediana Edad , Ontario , Estudios RetrospectivosRESUMEN
OBJECTIVE: We performed a return-on-investment analysis comparing the investment in surgical site infection (SSI) prevention programs in a hospital setting to the savings from averted SSI cases. DESIGN: A retrospective case costing study using aggregated patient data to determine the incidence and costs of SSI infection in surgical departments over time. We calculated return on investment to the hospital and conducted several sensitivity and scenario analyses. SETTING: Data were compiled for the Ottawa Hospital (TOH), a Canadian tertiary-care teaching institution.PatientsWe used aggregated records for all hospital patients who underwent surgical procedures between April 2010 and January 2015.InterventionWe estimated the potential cost savings of the hospital's surgical quality improvement program, namely the Surgeons National Surgical Quality Improvement Program (NSQIP) and the Comprehensive Unit-based Safety Program (CUSP). RESULTS: From 2010 to 2016, TOH invested C$826,882 (US$624,384) in surgical quality improvement programs targeting SSI incidence and accrued C$1,885,110 (US$1,423,460) in cumulative savings from averted SSI cases, generating a return of $2.28 (US$3.02) per dollar invested (95% confidence interval [CI], -0.67 to 7.37). The study findings are sensitive to the estimated cost to the hospital per SSI case and the rate reduction attributable to the prevention program. CONCLUSIONS: The NSQIP and CUSP have produced a positive return on investment at TOH; however, the result rests on several assumptions. This positive return on investment is expected to continue if the hospital can continue to reduce SSI incidence at least 0.25% annually without new investments. Findings from this study highlight the need for continuous program evaluation of the quality improvement initiatives.
Asunto(s)
Ahorro de Costo/estadística & datos numéricos , Evaluación de Programas y Proyectos de Salud/normas , Mejoramiento de la Calidad/organización & administración , Infección de la Herida Quirúrgica/prevención & control , Bases de Datos Factuales , Humanos , Ontario , Mejoramiento de la Calidad/economía , Estudios Retrospectivos , Infección de la Herida Quirúrgica/economía , Infección de la Herida Quirúrgica/epidemiología , Centros de Atención TerciariaRESUMEN
BACKGROUND: Drug-resistant epilepsy affects about one-third of children with epilepsy and is associated with high costs to the healthcare system, yet the cost effectiveness of most treatments is unclear. Use of cannabis-based products for epilepsy is increasing, and the cost effectiveness of such strategies relative to conventional pharmacologic treatments must be considered. OBJECTIVE: The objective of this systematic review was to identify economic evaluations of cannabis-based treatments for pediatric drug-resistant epilepsy. We also sought to identify and appraise decision models that have been used in economic evaluations of pharmacologic treatments (i.e., antiepileptic drugs) in this population. METHODS: Electronic searches of MEDLINE, EMBASE, and the Cochrane library, as well as a targeted grey literature search, were undertaken (11 June 2018). Model-based full economic evaluations involving cannabis-based treatments or pharmacologic treatments for drug-resistant epilepsy in children were eligible for inclusion. Two independent reviewers selected studies for inclusion, and study quality was assessed by use of the Drummond and Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklists. Study findings, as well as model characteristics, are narratively summarized. RESULTS: Nine economic evaluations involving children with drug-resistant epilepsy were identified; however, none involved cannabis-based treatments. All studies involved pharmacologic treatments compared with other pharmacologic treatments or non-pharmacologic treatments (i.e., ketogenic diet, epilepsy surgery, vagus nerve stimulation). Few studies have assessed the cost effectiveness of pharmacologic treatments in specific drug-resistant epilepsy syndromes, including Dravet and Lennox-Gastaut syndromes. Five included studies involved use of Markov models with a similar structure (i.e., health states based on seizure frequency relative to baseline). There was a wide range of methodological quality, and few studies fully addressed context-specific issues such as weight gain and treatment switching. CONCLUSION: Whether cannabis-based treatments for pediatric drug-resistant epilepsy represent good value for money has yet to be investigated. Economic evaluations of such treatments are needed and should address issues of particular importance in pediatric epilepsy, including weight gain over time, switching or discontinuation of treatments, effectiveness of interventions and comparators, and long-term effectiveness beyond the duration of available clinical studies. PROSPERO REGISTRATION: CRD42018099591.
Asunto(s)
Anticonvulsivantes/administración & dosificación , Epilepsia Refractaria/tratamiento farmacológico , Modelos Económicos , Anticonvulsivantes/economía , Niño , Análisis Costo-Beneficio , Técnicas de Apoyo para la Decisión , Epilepsia Refractaria/economía , Humanos , Marihuana Medicinal/administración & dosificación , Marihuana Medicinal/economíaRESUMEN
BACKGROUND: ICU care is costly, and there is a large variation in cost among patients. METHODS: This is an observational study conducted at two ICUs in an academic centre. We compared the demographics, clinical data, and outcomes of the highest decile of patients by total costs, to the rest of the population. RESULTS: A total of 7,849 patients were included. The high-cost group had a longer median ICU length of stay (26 versus 4 days, P < 0.001) and amounted to 49% of total costs. In-hospital mortality was lower in the high-cost group (21.1% versus 28.4%, P < 0.001). Fewer high-cost patients were discharged home (23.9% versus 45.2%, P < 0.001), and a large proportion were transferred to long-term care (35.1% versus 12.1%, P < 0.001). Patients with younger age or a diagnosis of subarachnoid hemorrhage, acute respiratory failure, or complications of procedures were more likely to be high cost. CONCLUSIONS: High-cost users utilized half of the total costs. While cost is associated with LOS, other drivers include younger age or admission for respiratory failure, subarachnoid hemorrhage, or after a procedural complication. Cost-reduction interventions should incorporate strategies to optimize critical care use among these patients.