BACKGROUND AND OBJECTIVE: Non-muscle-invasive bladder cancer (NMIBC) poses a significant clinical challenge, particularly when failing bacillus Calmette-Guérin (BCG) therapy, necessitating alternative treatments. Despite radical cystectomy being the recommended treatment, many patients are unfit or unwilling to undergo this invasive procedure, highlighting the need for effective bladder-sparing therapies. This review aims to summarize and report the evidence on the efficacy and to estimate the costs of bladder-preserving strategies used in NMIBC recurrence after failure of intravesical BCG therapy. METHODS: We systematically searched online databases for prospective studies investigating intravesical therapy, systemic therapy, or combination of both in patients treated previously with BCG. Owing to significant heterogeneity across the studies, a meta-analysis was inappropriate. A sensitivity analysis was performed in an exploratory manner. We used a decision-analytic Markov model to compare novel U.S. Food and Drug Administration-approved treatments with a 2-yr time horizon. KEY FINDINGS AND LIMITATIONS: A total of 57 studies published between 1998 and 2024, with 68 unique study arms and consisting of 2589 patients, were identified. The 3-mo overall response rate (ORR) across all studies, complete response rate (CRR) in concomitant carcinoma in situ (CIS) or CIS only disease, and recurrence-free rate (RFR) in papillary disease were estimated to be 52.4% (95% confidence interval [CI]: 45.4-59.2), 52.8% (95% CI: 42.9-62.6), and 26.4% (95% CI: 13.3-45.6), respectively. The 12-mo ORR, CRR, and RFR were estimated to be 78% (95% CI: 52.9-91.8), 27.8% (95% CI: 21.3-35.4), and 25.4% (95% CI: 18.2-34.2), respectively. The progression rate was estimated to be 13% (95% CI: 9-18.2). The mean proportion of patients treated with radical cystectomy was estimated to be 24.7 (range 0-85.7). The reported toxicity grades were overall mild, with a median of 3.4% (range 0-33.3%) participants experiencing a dose limiting toxicity. Compared with using radical cystectomy to treat patients failing BCG therapy, at a willingness-to-pay threshold of 100 000 USD, nadofaragene firadenovec was cost effective, with an incremental cost-effectiveness ratio (ICER) of 10 014 USD per quality-adjusted life year (QALY), while nogapendekin alfa inbakicept was less cost effective than nadofaragene firadenovec (ICER of 44 602 USD per QALY). Pembrolizumab, which dominated, was both less costly and more effective than the other strategies. CONCLUSIONS AND CLINICAL IMPLICATIONS: Salvage bladder-sparing therapies show a response rate of around 50% at 3 mo in patients with NMIBC failing BCG. However, long-term data are heterogeneous. Nevertheless, recently developed agents show promising tumor control activity. In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life. The findings of our review highlight the need for novel, more effective therapeutic strategies. PATIENT SUMMARY: In this study, we reviewed the evidence on the efficacy of bladder-preserving strategies used in patients with bladder cancer recurrence after failing bacillus Calmette-Guérin (BCG) therapy. We found that these strategies show a response rate of around 50% at 3 mo. However, long-term data are heterogeneous. Nevertheless, recently developed agents show promising tumor control activity. In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life.
- MeSH
- adjuvancia imunologická * terapeutické užití ekonomika MeSH
- analýza nákladů a výnosů * MeSH
- aplikace intravezikální MeSH
- BCG vakcína * terapeutické užití ekonomika MeSH
- invazivní růst nádoru MeSH
- lidé MeSH
- lokální recidiva nádoru MeSH
- nádory močového měchýře neinvadující svalovinu MeSH
- nádory močového měchýře * farmakoterapie patologie terapie ekonomika MeSH
- neúspěšná terapie MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- přehledy MeSH
- systematický přehled MeSH
INTRODUCTION: In a previously published randomised, placebo-controlled trial, 800 mg/day of pharmaceutical-grade chondroitin sulfate (CS) was shown to be superior to placebo in reducing pain and improving function over 6 months in patients with symptomatic knee osteoarthritis (OA). The aim of the current post hoc analyses was to evaluate the cost-effectiveness of CS compared with placebo in a European perspective using individual patient data from this clinical trial. METHODS: Patients with knee OA randomised to CS or placebo were followed up at 1, 3 and 6 months. The algo-functional Lequesne index was used to derive the EuroQol Five-Dimension Five-Level (EQ-5D-5L) score based on a validated formula. The EQ-5D-5L scores at each time point were used to calculate the changes in quality-adjusted life years (QALYs) with the area under the curve method. Costs were assessed using the average price of CS in the countries where the original study took place and where CS is currently marketed. The costs of CS in three countries were then used (i.e. the Czech Republic, Italy and Switzerland). The incremental cost-effectiveness ratio (ICER) threshold for CS to be considered cost-effective was set at 91,870 EUR per QALY (equivalent to the usually recommended threshold of US $100,000). The study used an intention-to-treat population, i.e. patients who received one dose of the study drug, and imputed missing values using the basal observation carried forward method. RESULTS: No significant differences in baseline characteristics were observed between the CS group (N = 199) and the placebo group (N = 205). The mean cost of CS for 6 months of treatment was 194.74 EUR. After 6 months of treatment, CS showed a mean ICER of 33,462 (95% CI 5130-61,794) EUR per QALY gained, indicating cost-effectiveness compared with placebo. The acceptability curve for cost-effectiveness shows that the CS treatment is likely to be cost-effective compared with placebo, with a 93% probability when the ceiling ratio is set at 91,870 EUR per QALY gained. CONCLUSIONS: These results highlight the role of CS as a cost-effective therapeutic option in the management of OA. However, further studies taking into account the use of other healthcare resources are warranted for a more complete understanding.
- MeSH
- analýza nákladové efektivity MeSH
- analýza nákladů a výnosů * MeSH
- artróza kolenních kloubů * farmakoterapie ekonomika MeSH
- chondroitinsulfáty * terapeutické užití ekonomika MeSH
- kvalitativně upravené roky života * MeSH
- lidé středního věku MeSH
- lidé MeSH
- senioři MeSH
- Check Tag
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- randomizované kontrolované studie MeSH
- Geografické názvy
- Česká republika MeSH
- Itálie MeSH
- Švýcarsko MeSH
OBJECTIVES: Decision-analytic models assessing the value of emerging Alzheimer's disease (AD) treatments are challenged by limited evidence on short-term trial outcomes and uncertainty in extrapolating long-term patient-relevant outcomes. To improve understanding and foster transparency and credibility in modeling methods, we cross-compared AD decision models in a hypothetical context of disease-modifying treatment for mild cognitive impairment (MCI) due to AD. METHODS: A benchmark scenario (US setting) was used with target population MCI due to AD and a set of synthetically generated hypothetical trial efficacy estimates. Treatment costs were excluded. Model predictions (10-year horizon) were assessed and discussed during a 2-day workshop. RESULTS: Nine modeling groups provided model predictions. Implementation of treatment effectiveness varied across models based on trial efficacy outcome selection (clinical dementia rating - sum of boxes, clinical dementia rating - global, mini-mental state examination, functional activities questionnaire) and analysis method (observed severity transitions, change from baseline, progression hazard ratio, or calibration to these). Predicted mean time in MCI ranged from 2.6 to 5.2 years for control strategy and from 0.1 to 1.0 years for difference between intervention and control strategies. Predicted quality-adjusted life-year gains ranged from 0.0 to 0.6 and incremental costs (excluding treatment costs) from -US$66 897 to US$11 896. CONCLUSIONS: Trial data can be implemented in different ways across health-economic models leading to large variation in model predictions. We recommend (1) addressing the choice of outcome measure and treatment effectiveness assumptions in sensitivity analysis, (2) a standardized reporting table for model predictions, and (3) exploring the use of registries for future AD treatments measuring long-term disease progression to reduce uncertainty of extrapolating short-term trial results by health-economic models.
- MeSH
- Alzheimerova nemoc * ekonomika farmakoterapie MeSH
- analýza nákladů a výnosů * MeSH
- ekonomické modely MeSH
- kognitivní dysfunkce * ekonomika MeSH
- kvalitativně upravené roky života MeSH
- lidé MeSH
- metody pro podporu rozhodování MeSH
- progrese nemoci MeSH
- výsledek terapie MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- srovnávací studie MeSH
OBJECTIVES: Smoking is an important risk factor leading to many diseases, which brings substantial healthcare costs as well as indirect costs due to decreased productivity. This article aims to quantify the social costs of smoking in the Czech Republic in 2019. METHODS: The prevalence-based, cost-of-illness approach is used, which assesses the costs as the sum of direct (healthcare) costs and indirect costs (productivity losses due to mortality and morbidity). The costs of healthcare utilization and pharmacotherapy in direct costs, and the costs of absenteeism, presenteeism, and premature mortality in indirect costs, are included. RESULTS: Total costs of smoking in the Czech Republic in 2019 are estimated as 2110.6 million EUR (0.94% of GDP). Direct costs amounted to 537.0 million EUR (2.9% of health expenditures in 2019) and indirect costs were 1573.6 million EUR, mainly driven by the costs of premature mortality (1062.5 million EUR). CONCLUSIONS: Despite the declining trend in the prevalence of smoking in the Czech Republic, the associated costs are considerable. Investments into strategies to reduce smoking continue to be needed.
- MeSH
- absentérství MeSH
- dospělí MeSH
- kouření * ekonomika epidemiologie MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- náklady na zdravotní péči * statistika a číselné údaje MeSH
- osobní újma zaviněná nemocí * MeSH
- předčasná smrt MeSH
- prevalence MeSH
- senioři MeSH
- výdaje na zdravotnictví statistika a číselné údaje MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- mužské pohlaví MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- Geografické názvy
- Česká republika MeSH
Current European/US guidelines recommend that molecular testing in advanced non-small cell lung cancer (aNSCLC) be performed using next-generation sequencing (NGS). However, the global uptake of NGS is limited, largely owing to reimbursement constraints. We compared real-world costs of NGS and single-gene testing (SGT) in nonsquamous aNSCLC. This observational study was conducted across 10 pathology centers in 10 different countries worldwide. Biomarker data collected via structured questionnaires (1 January-31 December 2021) were used to feed micro-costing analyses for three scenarios ['Starting Point' (SP; 2021-2022), 'Current Practice' (CP; 2023-2024), and 'Future Horizons' (FH; 2025-2028)] in both a real-world model, comprising all biomarkers tested by each center, and a standardized model, comprising the same sets of biomarkers across centers. Testing costs (including retesting) encompassed personnel costs, consumables, equipment, and overheads. Overall, 4,491 patients with aNSCLC were evaluated. Mean per-patient costs decreased for NGS relative to SGT over time, with real-world model costs 18% lower for NGS than for SGT in the SP scenario, and 26% lower for NGS than for SGT in the CP scenario. Mean per-biomarker costs also decreased over time for NGS relative to SGT. In the standardized model, the tipping point for the minimum number of biomarkers required for NGS to result in cost savings (per patient) was 10 and 12 in the SP and CP scenarios, respectively. Retesting had a negligible impact on cost analyses, and results were robust to variation in cost parameters. This study provides robust real-world global evidence for cost savings with NGS-based panels over SGT to evaluate predictive biomarkers in nonsquamous aNSCLC when the number of biomarkers to be tested exceeds 10. Widespread adoption of NGS may enable more efficient use of limited healthcare resources.
- MeSH
- analýza nákladů a výnosů MeSH
- genetické testování ekonomika metody MeSH
- lidé středního věku MeSH
- lidé MeSH
- nádorové biomarkery * genetika MeSH
- nádory plic * genetika patologie diagnóza MeSH
- náklady na zdravotní péči MeSH
- nemalobuněčný karcinom plic * genetika diagnóza patologie MeSH
- senioři MeSH
- vysoce účinné nukleotidové sekvenování * ekonomika MeSH
- Check Tag
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- pozorovací studie MeSH
- srovnávací studie MeSH
Pressure ulcers (PUs) impose a significant economic burden on healthcare systems, affecting patient quality of life and leading to substantial treatment costs. This study presents a cost-of-illness analysis of PU treatment in hospitalized patients in the Czech Republic, based on real-world clinical data. The analysis was conducted using a comprehensive methodology at a Czech university hospital, involving 304 hospitalizations. The study included all hospitalized patients with PUs. Data were collected employing a bottom-up, person-based approach, which refers to the collection and analysis of cost data at the individual patient level. This method captures detailed resource utilization for each patient. The methodology accounted for both systemic and local costs, including materials, medications, caregiver time, and procedures. The study involved 304 hospitalizations, with a mean length of stay of 13 days. The total cost of PU treatment, excluding pharmacotherapy, had a median of €678, while including pharmacotherapy, the median cost rose to €929. Younger patients incurred higher treatment costs. Significant cost variations were observed among different departments. We developed and applied a novel cost model to quantify the expenses associated with PUs, which accurately highlighted the financial burden in the hospital care setting. We present a rigorous methodology for PU cost-of-illness analysis, providing a valuable tool for future research and clinical practice. This comprehensive approach supports the development of targeted interventions to reduce the incidence and severity of PUs, ultimately improving patient care and reducing healthcare costs.
- MeSH
- dekubity * ekonomika terapie MeSH
- délka pobytu ekonomika statistika a číselné údaje MeSH
- dospělí MeSH
- hospitalizace ekonomika MeSH
- lidé středního věku MeSH
- lidé MeSH
- náklady na zdravotní péči statistika a číselné údaje MeSH
- nemocnice univerzitní * ekonomika MeSH
- osobní újma zaviněná nemocí * MeSH
- senioři nad 80 let MeSH
- senioři MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- senioři nad 80 let MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- Geografické názvy
- Česká republika MeSH
Cíl: Poskytnout přehled recentní literatury zabývající se nákladovou efektivitou screeningu duševního zdraví žen v období těhotenství. Výsledky: V databázích bylo vyhledáno 1 988 publikací, z nichž čtyři byly zařazeny do systematického review. Modely péče se screeningem jsou nákladově efektivní v porovnání se standardní péčí bez screeningu. Vícestupňové screeningové modely jsou efektivnější než jednostupňové. Na efektivitu má vliv množství falešně pozitivních případů, které ve vícestupňových modelech klesá. Závěr: Screening duševního zdraví v těhotenství se zdá být nákladově efektivní. Doporučujeme jeho využití v rámci komplexní zdravotní péče o těhotné ženy u nás.
Objective: To provide an overview of recent literature on the cost-effectiveness of mental health screening for women during pregnancy. Results: 1,988 publications were retrieved from databases, of which four were included in the systematic review. Models of care with screening are cost-effective compared with standard care without screening. Multi-level screening models are more cost-effective than single-level models. Effectiveness is affected by the number of false-positive cases, which decreases in multi-level models. Conclusion: Mental health screening in pregnancy appears to be cost-effective. We recommend its use in comprehensive health care for pregnant women in our country.
- MeSH
- analýza nákladové efektivity MeSH
- duševní zdraví MeSH
- klinická studie jako téma MeSH
- lidé MeSH
- plošný screening MeSH
- prenatální péče * ekonomika MeSH
- těhotenství MeSH
- Check Tag
- lidé MeSH
- těhotenství MeSH
- ženské pohlaví MeSH
- Publikační typ
- přehledy MeSH
- Klíčová slova
- ustekinumab CT-P43,
- MeSH
- biosimilární léčivé přípravky * farmakologie klasifikace terapeutické užití MeSH
- cílená molekulární terapie metody MeSH
- Crohnova nemoc farmakoterapie MeSH
- humanizované monoklonální protilátky farmakologie klasifikace terapeutické užití MeSH
- klinická studie jako téma MeSH
- lidé MeSH
- náklady na zdravotní péči MeSH
- ustekinumab * aplikace a dávkování farmakologie terapeutické užití MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- přehledy MeSH
Idiopatické střevní záněty představují významný zdravotní, sociální a ekonomický problém. Jejich moderní terapie je efektivní a bezpečná, současně přispívá ke snížení invalidizace, a tím i k redukci nepřímých nákladů. V České republice je dostupnost cílené léčby IBD omezena přibližně na polovinu reálné potřeby. Ke zlepšení této neuspokojivé situace je nutno detailně analyzovat přímé i nepřímé náklady, navýšit finanční prostředky na centrovou léčbu a zlepšit komunikaci mezi klíčovými partnery zdravotního i sociálního systému.
Inflammatory bowel disease poses a significant health, social and economic problem. A modern advanced therapy of IBD is effective and safe, and, simultaneously, reduces patients‘ disability together with indirect costs. The access to advanced therapy in Czech Republic is, however, a half of what we need in the real clinical practice. To improve this, we have to analyze both direct and indirect costs, increase budgets for advanced therapy and improve communication between the key stakeholders within the health and social system.
- MeSH
- cílená molekulární terapie ekonomika metody MeSH
- Crohnova nemoc ekonomika farmakoterapie MeSH
- idiopatické střevní záněty * diagnóza ekonomika farmakoterapie MeSH
- lidé MeSH
- náklady na zdravotní péči * statistika a číselné údaje MeSH
- ulcerózní kolitida ekonomika farmakoterapie MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- přehledy MeSH
- Geografické názvy
- Česká republika MeSH
The epidemiological transition has been characterized by demographic, societal and health changes in societies. Presuming that acute diseases, mostly of communicable etiology, are more important in terms of early-life mortality, whereas chronic diseases are responsible for a greater burden of disease throughout the life course, we attempt to develop an index to measure the stage of the epidemiological transition. Using Global Burden of Diseases, Risk Factors and Injuries (GBD) data available at https://vizhub.healthdata.org/gbd-compare/ on 04/04/2024 to calculate the Epidemiologic Transition Estimate (ETE) index as a ratio of YLD/YLL for the time period of 1990-2019. The values of the index ranged from 0.131 to 1.067 and 0.180 to 2.108 for males and females, respectively, across the 195 included countries. The transition process seems to be faster to females compared to males. The index shows consistently increasing values for five SDI-based country groups, with clear differences among the groups. Although more research and validation studies are needed despite all the uncertainties, the index seems to be robust to assess the progress in epidemiological transition. These findings can help to predict future social care and health system needs to address causes leading to YLD or YLL.
