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In locally advanced (LA) laryngeal/hypopharyngeal squamous cell carcinoma (LHSCC), larynx preservation (LP) strategies aim at the cure of the disease while preserving a functional larynx, thus avoiding total laryngectomy and the associated impact on the quality of life. In the last decades, apart from transoral and open-neck organ preservation approaches, several non-surgical regimens have been investigated: radiotherapy alone, alternate, concurrent or sequential chemoradiation, and bioradiotherapy. Despite major progress, the identification of reliable and effective predictors for treatment response remains a clinical challenge. This review examines the current state of LP in LA-LHSCC and the need for predictive factors, highlighting the importance of the PRESERVE trial in addressing this gap. The PRESERVE trial represents a pivotal initiative aimed at finding the optimal therapy for laryngeal preservation specific to each patient through a retrospective analysis of data from previous LP trials and prospectively validating findings. The goal of the PRESERVE trial is to develop a comprehensive predictive classifier that integrates clinical, molecular, and multi-omics data, thereby enhancing the precision and efficacy of patient selection for LP protocols.
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INTRODUCTION: The treatment of patients with cardiogenic shock (CS) encompasses several health technologies including Impella pumps and venoarterial extracorporeal membrane oxygenation (VA-ECMO). However, while they are widely used in clinical practice, information on resource use and quality of life (QoL) associated with these devices is scarce. The aim of this study is, therefore, to collect and comparatively assess clinical and socioeconomic data of Impella versus VA-ECMO for the treatment of patients with severe CS, to ultimately conduct both a cost-effectiveness (CEA) and budget impact (BIA) analyses. METHODS AND ANALYSIS: This is a prospective plus retrospective, multicentre study conducted under the scientific coordination of the Center for Research on Health and Social Care Management of SDA Bocconi School of Management and clinical coordination of Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific Institute in Milan. The Impella Network stemmed for the purposes of this study and comprises 17 Italian clinical centres from Northern to Southern Regions in Italy. The Italian network qualifies as a subgroup of the international Impella Cardiac Surgery Registry. Patients with CS treated with Impella pumps (CP, 5.0 or 5.5) will be prospectively recruited, and information on clinical outcomes, resource use and QoL collected. Economic data will be retrospectively matched with data from comparable patients treated with VA-ECMO. Both CEA and BIA will be conducted adopting the societal perspective in Italy. This study will contribute to generate new socioeconomic evidence to inform future coverage decisions. ETHICS AND DISSEMINATION: As of May 2024, most of the clinical centres submitted the documentation to their ethical committee (N=13; 76%), six centres received ethical approval and two centres started to enrol patients. Study results will be published in peer-reviewed publications and disseminated through conference presentations.
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Análisis Costo-Beneficio , Oxigenación por Membrana Extracorpórea , Corazón Auxiliar , Choque Cardiogénico , Humanos , Choque Cardiogénico/terapia , Choque Cardiogénico/economía , Oxigenación por Membrana Extracorpórea/economía , Oxigenación por Membrana Extracorpórea/métodos , Corazón Auxiliar/economía , Estudios Prospectivos , Estudios Retrospectivos , Italia , Calidad de Vida , Estudios Multicéntricos como Asunto , Presupuestos , Estudios Observacionales como AsuntoRESUMEN
AIM: Rectus abdominal diastasis (RAD) can cause mainly incontinence and lower-back pain. Despite its high incidence, there is no consensus regarding surgical indication. We aimed at comparing RAD repair (minimally invasive technique with mesh implant) with no treatment (standard of care - SOC) through cost-effectiveness and budget impact analyses from both National Healthcare Service (NHS) and societal perspectives in Italy. METHODS: A model was developed including social costs and productivity losses derived by the online administration of a socio-economic questionnaire, including the EuroQol for the assessment of quality of life. Costs for the NHS were based on reimbursement tariffs. RESULTS: Over a lifetime horizon, estimated costs were 64,115 for SOC and 46,541 for RAD repair in the societal perspective; QALYs were 19.55 and 25.75 for the two groups, respectively. Considering the NHS perspective, RAD repair showed an additional cost per patient of 5,104 compared to SOC, leading to an ICUR of 824. RAD repair may be either cost-saving or cost-effective compared to SOC depending on the perspective considered. Considering a current scenario of 100% SOC, an increased diffusion of RAD repair from 2 to 10% in the next 5 years would lead to an incremental cost of 184,147,624 for the whole society (87% borne by the NHS) and to incremental 16,155 QALYs. CONCLUSION: In light of the lack of economic evaluations for minimally invasive RAD repair, the present study provides relevant clinical and economic evidence to help improving the decision-making process and allocating scarce resources between competing ends.
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Introduction: The aim of the study was to define a core outcome set (COS) to be measured following cataract surgery for the postoperative evaluation of monofocal intraocular lenses (IOLs). Compared to current COSs, the present work provides updates considering the advances in the technology due to the development of new generation monofocal IOLs, which are characterized by a safety profile comparable to standard monofocal IOLs but with an extended range of intermediate vision. Methods: Healthcare professionals (ophthalmologist surgeons) and patients were involved in the selection of outcomes to be included in the COS, starting from a list of indicators retrieved from a systematic literature search. The search considered observational studies with both a retrospective or prospective design, case studies and classic randomized controlled trials (RCTs). A mixed methodology integrating a Delphi-driven and an expert panel approach was adopted to reach an agreement among clinicians, while patients were involved in the completion of a questionnaire. Results: The final COS included 15 outcomes. Eleven outcomes, all clinical, were considered for inclusion after a joint discussion among ophthalmologists; seven outcomes were linked to visual acuity, while the remaining to contrast sensitivity, refractive errors, aberrations and adverse events. Measurement metrics, method of aggregation and measurement time point of these outcomes were specified. The most important aspects for the patients were (1) quality of life after cataract surgery, (2) the capacity to perform activities requiring good near vision (e.g., reading), (3) spectacle independence, and (4) safety of movements without fear of getting hurt or falling (intermediate vision). Discussion: In a context with limited healthcare resources, it is important to optimize their use considering also the preferences of end-users, namely patients. The proposed COS, developed involving both ophthalmologists and patients, provides an instrument for the postoperative evaluation of different technologies in the context of monofocal IOLs, which can be used not only in clinical trials but also in clinical practice to increase the body of real-world evidence.
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INTRODUCTION: Ovarian cancer (OC) is the eighth most common cancer among women, and homologous recombination deficiency (HRD) is present in approximately 50% of these patients. For this group, poly(ADP-ribose) polymerase (PARP) inhibitors are more likely to be effective. The aim of the study was to investigate the cost-effectiveness of HRD testing versus BRCA testing (which identifies mutations present only in 25% of patients) in Italy to optimize the treatment management, possibly with PARP inhibitors. METHODS: A cost-effectiveness partition survival model was developed to estimate the expected costs and outcomes (life years, LYs; quality-adjusted life years, QALYs) with lifetime horizon of HRD testing versus BRCA testing alone in women with high-grade serous or endometrioid advanced ovarian cancer. The option to perform the tests in sequence, that is, the BRCA test followed by the HRD test, in patients with BRCA-negative test was also considered, and the model accounted for the National Healthcare Service (NHS) perspective in Italy. The treatments represented the best available options according to the initial test results and according to PARP inhibitors available in Italy. A 3% discount rate was applied. Both deterministic and probabilistic sensitivity analyses were performed to test the robustness of the model results. RESULTS: HRD testing was shown to be a cost-effective strategy compared to BRCA testing (incremental cost-utility ratio 22,610/QALY) and a cost-saving strategy compared to the sequence of tests. The probabilistic sensitivity analysis showed that the HRD test is cost-effective compared to BRCA testing in 98.5% of model simulations considering a willingness-to-pay threshold of 50,000/QALY. CONCLUSION: The identification of genetic anomalies in patients with advanced OC is a costly process. Regardless, HRD upfront testing compared to BRCA testing had a cost-effective profile, allowing the efficient use of healthcare resources and better life expectancy and quality of life for patients.
Ovarian cancer has been the most lethal gynecological tumor for years. Recently, there have been notable advances due to the introduction of poly(adenosine diphosphate-ribose polymerase) (PARP) inhibitor drugs, which have significantly increased the survival rates of women affected by advanced-stage disease. At least 50% of ovarian tumors have a defect in the DNA repair mechanism, known as homologous recombination deficiency, and the mechanism of action of these drugs involves blocking the DNA repair mechanisms implemented by neoplastic cells. The identification of patients with homologous recombination deficiency through a genetic test, with consequent optimized treatment management, possibly with PARP inhibitors, resulted in better life expectancy, even when adjusted for the quality of life, than the management of patients starting from BRCA testing alone. The homologous recombination deficiency testing strategy can be considered cost-effective from the National Healthcare Service perspective in Italy. These findings provide evidence of the value of a new diagnostic option for clinicians and payers to optimize the management of women with high-grade serous or endometrioid advanced ovarian cancer.
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Neoplasias Ováricas , Inhibidores de Poli(ADP-Ribosa) Polimerasas , Humanos , Femenino , Inhibidores de Poli(ADP-Ribosa) Polimerasas/uso terapéutico , Análisis de Costo-Efectividad , Calidad de Vida , Carcinoma Epitelial de Ovario , Neoplasias Ováricas/diagnóstico , Neoplasias Ováricas/tratamiento farmacológico , Neoplasias Ováricas/genética , ItaliaRESUMEN
INTRODUCTION: Rotator cuff tear (RCT) is a painful, progressive condition resulting from damage to the rotator cuff tendons and is the leading cause of shoulder-related disability. Surgical repair of rotator cuff is an established standard of care (SOC); however, failure of the procedure can occur. In this context, the use of collagen-based bioinductive implant REGENETEN showed long-term improvements in clinical scores. The aim of the study was to assess the cost-effectiveness of REGENETEN combined with SOC (SOC + REGENETEN) compared to SOC alone from both National Healthcare Service (NHS) and societal perspectives in Italy. METHODS: A decision analytic model was developed to estimate the number of tears healed and costs for the two considered treatment strategies over 1 year. Clinical data were retrieved from the literature, and the clinical pathways for the management of patients with RCTs were retrieved from four key opinion leaders in Italy. RESULTS: Over a 1-year time horizon, healed lesions were 90.70% and 72.90% for surgical repair of RCTs with and without REGENETEN, respectively. Considering the NHS perspective, mean costs per patient were 7828 and 4650 for the two strategies, respectively, leading to an incremental cost-effectiveness ratio (ICER) of 17,857 per healed tear. From the societal perspective, the mean costs per patient were 12,659 for SOC and 11,784 for REGENETEN, thus showing savings of 4918 per healed tear when the bioinductive implant is used. The sensitivity analyses confirmed the robustness of the model results. CONCLUSION: In the context of paucity of cost-effectiveness studies, our findings provide additional evidence for clinicians and payers regarding the value of a new treatment option that supports a tailored approach for the management of patients with RCTs.
The rotator cuff refers to a group of four muscles, with tendons connected to the upper arm bone, which act together to allow lifting and rotating the shoulder. A tear of the rotator cuff can affect either a single tendon or multiple tendons. Typical first-line treatment includes conservative therapies, which aim to alleviate pain and reduce functional impairment, but are often ineffective. Persisting disease is usually managed through conventional surgical repair. Recently, REGENETEN, a collagen-based bioinductive implant derived from purified bovine Achilles tendon, positioned over the site of the damaged rotator cuff, achieved successful rotator cuff tendon repair with an increase in healed tears of 17.80% at 1 year compared to conventional surgery. Considering the National Healthcare Service perspective in Italy, the cost needed to achieve one additional healed tear using REGENETEN compared to conventional surgery is 17,857. From the societal perspective, which includes patients' productivity losses from hospital admission to return to work, the use of REGENETEN may be cost-saving compared to conventional surgery. The findings of our study provide evidence for clinicians and payers to support the value of a new treatment option for patients with rotator cuff lesions.
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Lesiones del Manguito de los Rotadores , Humanos , Lesiones del Manguito de los Rotadores/cirugía , Análisis Costo-Beneficio , Resultado del Tratamiento , Manguito de los Rotadores/cirugía , ColágenoRESUMEN
OBJECTIVES: Percutaneous coronary intervention (PCI) represents the standard treatment for ST-elevated myocardial infarction, nevertheless, mortality and heart failures are frequent. Pressure-controlled intermittent coronary sinus occlusion (PiCSO) might reduce infarct size showing better patients' outcomes. We evaluated the cost-effectiveness of PCI+PiCSO compared to PCI from the National Healthcare Service (NHS) perspective in Italy. METHODS: A Markov model was developed to estimate life years (LYs), quality-adjusted life years (QALYs) and costs. A micro-costing analysis has been performed to inform the cost of PCI+PiCSO procedure. Sensitivity analyses were performed to test the robustness of the model results. RESULTS: Considering a willingness-to-pay threshold of 50,000/QALY for the ICUR and a cost for PCI+PiCSO procedure of 14,654, the innovative strategy may be cost-effective compared to PCI alone from the Italian NHS perspective, showing an ICUR of 17,530/QALY (ICER 14,631/LY) over a lifetime horizon; the probabilistic sensitivity analysis showed that PCI+PiCSO is cost-effective in 78.8% of simulations.Considering the above mentioned willingness-to-pay threshold, PCI+PiCSO strategy would be cost-effective over a lifetime horizon considering a cost for PCI+PiCSO procedure lower than 28,160. CONCLUSION: PCI+PiCSO procedure may be considered a cost-effective technology that allows reducing cardiac events, while improving patients' life expectancy and quality of life.
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Seno Coronario , Insuficiencia Cardíaca , Intervención Coronaria Percutánea , Humanos , Intervención Coronaria Percutánea/métodos , Análisis de Costo-Efectividad , Calidad de Vida , Análisis Costo-Beneficio , Años de Vida Ajustados por Calidad de VidaRESUMEN
BACKGROUND: Asthma is a common chronic inflammatory airway affecting over 260 million people worldwide, and characterized, in the large majority of cases, by the so-called "type 2 inflammation". Fractional exhaled nitric oxide (FENO) testing is noninvasive point-of-care tool to assess type 2 inflammation and therefore improve asthma management. It has been suggested to determine eligibility for a specific biologic therapy and predict likelihood to respond. The aim of this study was to estimate the overall economic impact of an extensive use of FENO testing on the Italian population with asthma, including extra costs of testing and savings generated by more appropriate prescriptions, increased adherence and lower frequency of exacerbations. METHODS: A cost of illness analysis was firstly performed to estimate the yearly economic burden from the National Healthcare Service (NHS) perspective in Italy of the management of asthmatic patients with standard of care (SOC) according to the application of GINA (Global Initiative for Asthma) guidelines; then, we evaluated the changes in the economic burden in patient management by introducing FENO testing into clinical practice. The cost items considered were: visits/exams, exacerbations, drugs, management of adverse events caused by short-term oral corticosteroids use. Efficacy of FeNO test and SOC is based on literature evidence. Costs refer to published data or Diagnosis Related Group/outpatient tariffs. RESULTS: Considering one asthma visit every 6 months, the total yearly cost for the management of patients with asthma in Italy is 1,599,217,876 (409.07 per patient), while for FENO testing strategy this figure is 1,395,029,747 (356.84 per patient). An increased utilization rate of FENO testing from 50 to 100% of patients may lead to savings for the NHS from about 102 to 204 million compared to SOC. CONCLUSIONS: Our study showed that FeNO testing strategy may improve the management of asthmatic patients leading to significant savings for the NHS.
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Asma , Óxido Nítrico , Humanos , Pruebas Respiratorias , Espiración , Asma/diagnóstico , Asma/tratamiento farmacológico , Asma/epidemiología , InflamaciónRESUMEN
Time-driven activity-based costing (TDABC) is suggested to assess costs within the value-based healthcare approach, but there is a paucity of applications in chronic diseases such as deep vein thrombosis (DVT) and leg ulcers. In this context, we applied TDABC in a cost-effectiveness analysis comparing venous stenting to compression ± anticoagulation (standard of care-SOC) from both hospital and societal perspectives in Italy. TDABC was applied to both treatments to assess costs that were included in a cost-effectiveness model. Clinical inputs were retrieved from the literature and integrated with real-world data. The Incremental Cost Utility Ratio (ICUR) of stenting compared to SOC was EUR 10,270/QALY and EUR 8962/QALY for hospital and societal perspectives, respectively. The mean cost per patient for venous stenting of EUR 5082 was higher than the Diagnosis-Related Group (DRG) reimbursement (EUR 4742). For SOC, an ulcer healing in 3 months costs EUR 1892, of which EUR 302 (16%) is borne by the patient versus a reimbursement of EUR 1132. TDABC showed that venous stenting may be cost-effective compared with SOC but that reimbursement rates may not completely cover the real costs, which are partially sustained by the patients. A more efficient policy for covering the real costs may be beneficial for both clinical centers and patients.
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Úlcera de la Pierna , Trombosis de la Vena , Humanos , Análisis Costo-Beneficio , Úlcera de la Pierna/terapia , Factores de Tiempo , Trombosis de la Vena/terapia , Atención a la SaludRESUMEN
Impella and VA-ECMO are two possible therapeutic courses for the treatment of patients with cardiogenic shock (CS). The study aims to perform a systematic literature review and meta-analyses of a comprehensive set of clinical and socio-economic outcomes observed when using Impella or VA-ECMO with patients under CS. A systematic literature review was performed in Medline, and Web of Science databases on 21 February 2022. Nonoverlapping studies with adult patients supported for CS with Impella or VA-ECMO were searched. Study designs including RCTs, observational studies, and economic evaluations were considered. Data on patient characteristics, type of support, and outcomes were extracted. Additionally, meta-analyses were performed on the most relevant and recurring outcomes, and results shown using forest plots. A total of 102 studies were included, 57% on Impella, 43% on VA-ECMO. The most common outcomes investigated were mortality/survival, duration of support, and bleeding. Ischemic stroke was lower in patients treated with Impella compared to the VA-ECMO population, with statistically significant difference. Socio-economic outcomes including quality of life or resource use were not reported in any study. The study highlighted areas where further data collection is needed to clarify the value of complex, new technologies in the treatment of CS that will enable comparative assessments focusing both on the health impact on patient outcomes and on the financial burden for government budgets. Future studies need to fill the gap to comply with recent regulatory updates at the European and national levels.
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AIM: Major depressive disorder is considered one of the most frequent diseases in the general population, and treatment-resistant depression (TRD) represents the subset with more significant clinical and social impact. Large, robust phase III studies have shown safety and efficacy of esketamine nasal spray plus SSRI/SNRI antidepressants (ADs) compared with SSRI/SNRI plus placebo nasal spray in patients with TRD. The main aim of this study was to perform a cost-utility analysis comparing esketamine plus ADs with ADs alone in TRD patients, from the societal perspective in Italy. A secondary analysis focused on the National Healthcare Service (NHS) perspective. METHODS: A Markov multistate model has been developed to estimate quality-adjusted life years and economic outcomes of both treatment strategies over 5 years considering the initiation of esketamine in the different treatment lines, from 3 to 5 (3L-5L). The model has been populated with data from literature and real-world evidence. The analysis from the societal perspective considered direct healthcare costs and patients' productivity losses. In addition to the incremental cost-utility ratio (ICUR), the incremental net monetary benefit (INMB) has been calculated as (incremental benefit × WTP) - incremental cost and by applying a willingness-to-pay (WTP) of 50,000/QALY. Deterministic and probabilistic sensitivity analyses have been performed to assess the robustness of the model results. RESULTS: From the societal perspective, the ICUR ranged between 16,314 and 22,133 per QALY according to the different treatment lines, while it was over the threshold of 100,000/QALY for the NHS perspective. The INMB was positive and ranged from 2259 to 2744 across treatment lines in the societal perspective; the INMB begins to occur earlier when moving towards subsequent lines of treatment (3.9 years for 3L, 3.6 years for 4L and 3.5 years for 5L). The analyses showed also that the advantage in terms of INMB is maintained for a wide range of societal preferences expressed by WTP thresholds, and in particular for values above 22,200, 16,400 and 17,100 for 3L, 4L and 5L, respectively. CONCLUSION: The study showed that esketamine may be a cost-effective opportunity from the societal perspective for the management of patients with treatment-resistant depression. In the future, data collected from observational studies or registries, which can include the collection of productivity losses and also costs sustained by the patients, will be able to provide further evidence in order to improve the reliability of the model results.
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Trastorno Depresivo Mayor , Inhibidores de Captación de Serotonina y Norepinefrina , Humanos , Análisis Costo-Beneficio , Depresión , Rociadores Nasales , Reproducibilidad de los Resultados , Antidepresivos/uso terapéutico , Años de Vida Ajustados por Calidad de VidaRESUMEN
PURPOSE: Resource modeling aims to explicitly quantify the effects of adopting new health care technologies in settings with capacity-related constraints. The aim of this analysis was to use resource modeling to explore the effects of the uptake of first-line treatment with daratumumab on wait lists and wait times in patients with untreated multiple myeloma. Two formulations were compared: the standard IV formulation (DARA-IV) and a recently approved SC formulation (DARA-SC). METHODS: First, semi-structured interviews at six oncologic centers were used to retrieve data on the management of patients given a DARA-IV regimen. Second, a discrete event simulation (DES) model was built to estimate the effects on resource consumption, wait lists, and wait times in scenarios with different incident numbers of patients treated with either DARA-IV or DARA-SC. FINDINGS: In all of the simulated scenarios with more incident patients initiated on first-line treatment with DARA-IV, the actual capacity of infusion chairs was not enough to meet the demand, leading to increases in wait times and wait lists. In the highest-demand scenario, 17 more infusion chairs per center would be required to avoid such increases. Treatment with DARA-SC would allow centers to meet the demand with their actual capacity. IMPLICATIONS: DES modeling can effectively be used to formally explore the effects of different formulations on the use of limited resources, wait lists, and wait times at the facility level. Based on the findings from this analysis, DARA-SC may free up resources and prevent short- and long-term costs to infusion centers.
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Mieloma Múltiple , Humanos , Mieloma Múltiple/tratamiento farmacológico , Anticuerpos Monoclonales/uso terapéutico , Protocolos de Quimioterapia Combinada AntineoplásicaRESUMEN
Aims: To perform a cost-effectiveness analysis (CEA) comparing personalised dosimetry with standard dosimetry in the context of selective internal radiation therapy (SIRT) with TheraSphere for the management of adult patients with locally advanced hepatocellular carcinoma (HCC) from the Italian Healthcare Service perspective. Materials and methods: A partition survival model was developed to project costs and the quality-adjusted life years (QALYs) over a lifetime horizon. Clinical inputs were retrieved from a published randomised controlled trial. Health resource utilisation inputs were extracted from the questionnaires administered to clinicians in three oncology centres in Italy, respectively. Cost parameters were based on Italian official tariffs. Results: Over a lifetime horizon, the model estimated the average QALYs of 1.292 and 0.578, respectively, for patients undergoing personalised and standard dosimetry approaches. The estimated mean costs per patient were 23,487 and 19,877, respectively. The incremental cost-utility ratio (ICUR) of personalised versus standard dosimetry approaches was 5,056/QALY. Conclusions: Personalised dosimetry may be considered a cost-effective option compared to standard dosimetry for patients undergoing SIRT for HCC in Italy. These findings provide evidence for clinicians and payers on the value of personalised dosimetry as a treatment option for patients with HCC.
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OBJECTIVES: A recent debate in health economics and outcomes research community identified option value as one of the elements warranting consideration in the assessment of medical technologies. To conduct a scoping review of contributions on option value in the healthcare sector and identify relevant conceptual aspects and methods used to incorporate it in standard economic evaluations. METHODS: A systematic search was conducted up to July 2020 to identify contributions from electronic bibliographic database and gray literature. Data on the proposed definitions of option value, theoretical implications of its use in economic evaluations, and methods used to estimate it were extracted and analyzed. RESULTS: We found 57 eligible studies. Three different definitions emerged: insurance value, real option value, and option value of survival. Focusing on the latter (24 studies), we analyzed in depth 8 empirical applications across 7 therapeutic areas. The most relevant methodological challenges were on the perspective used in economic evaluations and how to robustly manage forecasting uncertainty, update cost-effectiveness thresholds, and avoid double-counting issues. For empirical studies assessing the total value of the technology, including option value, estimates ranged from +7% to +469% of its conventional value. CONCLUSIONS: This review synthesizes theoretical and empirical aspects on option value of healthcare technologies and proposes a terminology to distinguish 3 different concepts identified. Future work should focus primarily on agreeing on whether option value should be included in economic evaluations and, if so, on developing and validating reliable methods for its ex-ante estimation.
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Análisis Costo-Beneficio , Economía Médica , Evaluación de la Tecnología Biomédica , Bases de Datos Factuales , Toma de DecisionesRESUMEN
There is limited evidence on the scope and overall benefit of patient-centred drug development decisions. The present study assessed patients' preferences for the characteristics of an ideal migraine treatment through a discrete choice experiment in order to inform decision-making and drug development processes. We investigated the preferences according to five treatment attributes identified from a systematic literature review and two focus group elicitations. The heterogeneity of preferences was also investigated. Overall, the respondents considered the presence of adverse events, duration of treatment effect, reduction of symptom intensity, speed of effect and cost born by the patient as the most relevant treatment features. As expected, the patients preferred treatments with lower levels of adverse events and costs and treatments with greater speed, duration of treatment effect and effectiveness in reducing symptom intensity. There was significant preference heterogeneity only for the presence of adverse events. Compared to men, women had significantly higher preferences for quicker treatment effect and limited adverse events and reported higher preferences for costly treatments. The results of our survey help address research and development strategies in the pharmaceutical industry and public policy regarding treatments that are clinically effective and responsive to the needs expressed by patients.
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Trastornos Migrañosos , Prioridad del Paciente , Conducta de Elección , Desarrollo de Medicamentos , Femenino , Humanos , Masculino , Trastornos Migrañosos/tratamiento farmacológico , Encuestas y Cuestionarios , Revisiones Sistemáticas como AsuntoRESUMEN
A precise assessment of the efficacy of first-/second-line endocrine therapies (ET) ± target therapies (TT) in clinically-relevant subgroups of hormone receptor-positive (HR+)/HER2-negative metastatic breast cancer (MBC) has not yet been conducted. To improve our current knowledge and support clinical decision-making, we thus conducted a systematic literature search to identify all first-/second-line phase II/III randomized clinical trials (RCT) of currently approved or most promising ET ± TT. Then, we performed a meta-analysis to assess progression-free (PFS) and/or overall survival (OS) benefit in several clinically-relevant prespecified subgroups. Thirty-five RCT were included (17,595 patients). Pooled results show significant reductions in the risk of relapse or death of 26-41% and 12-27%, respectively, depending on the clinical subgroup. Combination strategies proved to be more effective than single-agent ET (PFS hazard ratio (HR) range for combinations: 0.60-0.65 vs. HR range for single agent ET: 0.59-1.37; OS HR range for combinations: 0.74-0.87 vs. HR range for single agent ET: 0.68-0.98), with CDK4/6-inhibitors(i) + ET being the most effective regimen. Single agent ET showed comparable efficacy with ET+TT combinations in non-visceral (p = 0.63) and endocrine sensitive disease (p = 0.79), while mTORi-based combinations proved to be a valid therapeutic option in endocrine-resistant tumors, as well as PI3Ki + ET in PIK3CA-mutant tumors. These results strengthen international treatment guidelines and can aid therapeutic decision-making.
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BACKGROUND: Deep vein thrombosis (DVT) is one of the major health problems worldwide with potentially serious outcomes related to mortality and morbidity. We provide a current view on how patients with DVT are managed in routine practice compared with the recommendations of published clinical guidelines. METHODS: A literature review was conducted on studies reporting diagnostic and treatment patterns for acute DVT. Four dimensions were evaluated to compare the differences between clinical practice and clinical guidelines recommendations: diagnostic pathway, prescription of pharmacologic treatment and related duration, and prescription of compression therapy. For each aspect, the agreement with the corresponding guideline has been estimated as a percentage ranging from 0% (no agreement) to 100% (full agreement). RESULTS: Sixteen studies reported clinical practices in 10 countries. Among them, Japan showed the highest agreement with guidelines, followed by the UK and Switzerland. Hong Kong showed the highest agreement with diagnosis guidelines, Spain for drug treatment, UK for treatment duration, and France for compression therapy. Conversely, Germany reported a complete disagreement with guidelines for diagnosis, followed by low agreement level by UK and Italy, and Switzerland reported a lower agreement level with prescription of compression therapy. CONCLUSIONS: The implementation of clinical guidelines for the management of patients with DVT varies among countries from strict adherence to a complete lack of adherence. In this context, registries may be a useful tool to analyze clinical practice and produce findings that may be generalizable across populations.