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1.
Background: In light of constrained budgets and the need to fund efficient treatment options, this study set out to assess the cost-effectiveness of sorafenib as a first-line treatment of hepatocellular carcinoma (HCC) compared to best supportive care (BSC) from the military hospital perspective in Egypt.

Methods: A decision analytic Markov model simulated disease progression with clinical parameters and utility values derived from published data. Data on direct medical costs were collected from the local healthcare system or payer. Costs and effects were discounted at 3.5% annually and reported in USD using purchasing power parity adjustments. Deterministic and probabilistic sensitivity analyses were conducted.

Results: Mortality occurred less frequently in the sorafenib group (sorafenib group: 99.96%, BSC group: 99.99%). The total quality-adjusted life years (QALYs) of the sorafenib cohort were estimated to be 46.24 compared with 42.27 for the BSC cohort, which resulted in an incremental gain of 3.96 QALYs. The total costs for the sorafenib and BSC cohorts were USD 4,229,940 and USD 3,092,886, respectively (incremental cost = $1,137,054), resulting in an incremental cost-effectiveness ratio (ICER) of USD 286,776 per QALY gained for the sorafenib cohort. One-way sensitivity analyses that addressed the uncertainty of the BSC estimates indicated that the progression-free survival for BSC and utility value of progression had the greatest effects on the results.

Conclusion: This study concluded that sorafenib does offer increased survival and quality-of-life at an increased cost but at an ICER that exceeds the nationally accepted cost-effectiveness threshold. The findings support healthcare decision-making of the efficient allocation of healthcare system resources to improve the health of the Egyptian population. Whether sorafenib is cost-effective in specific sub-groups with additional risk factors needs to be addressed in future studies.  相似文献   


2.
Background and objective: Dapagliflozin is the first SGLT2 inhibitor available in China, where the disease burden of diabetes and its complications is very heavy. Because a new diabetes treatment strategy for diabetes should consider its cost-effectiveness, compared with an existing treatment, this study aimed to examine the cost-effectiveness between dapagliflozin and metformin treatment in China.

Methods: The Cardiff Diabetes Model (CDM) was used to estimate cost effectiveness and macro- and micro-vascular outcomes of dapagliflozin vs metformin. The CDM effectiveness inputs were derived from indirect comparative efficacy data from meta-analysis of 71 studies comparing monotherapy and add-on therapy of dapagliflozin vs metformin: dapagliflozin or metformin monotherapy, add-on therapy with other oral hypoglycemic agents, and add-on therapy with insulin. Direct medication costs and medical costs on treating diabetes were calculated based on published and local sources. A discount rate of 3% was applied to both costs and health effects. Univariate and probabilistic sensitivity analyses (PSA) were performed to assess uncertainties.

Results: The total healthcare costs accumulated over the lifetime on dapagliflozin treatment arm was 8,626 Chinese yuan higher than the metformin treatment arm for an individual patient, and the quality adjusted life years (QALYs) gained with dapagliflozin treatment was 0.8 more than metformin treatment. Therefore, an incremental cost-effectiveness ratio was 10,729 yuan per QALY gained for dapagliflozin treatment arm vs metformin treatment arm. The cost-effectiveness results were robust to various sensitivity analyses.

Conclusion: Dapagliflozin treatment was more cost-effective compared with metformin treatment for Chinese type 2 diabetes patients. However, the findings of favorable cost-effectiveness results for dapagliflozin are largely driven by the effects of favorable weight profile on clinical, utility, and costs in the Cardiff model.  相似文献   


3.
Aims: To conduct a lifetime cost-effectiveness analysis (CEA) of rasburicase compared with standard of care (SOC) for tumor lysis syndrome (TLS) in children with hematologic malignancies from the Chinese healthcare system perspective.

Materials and methods: The CEA was performed using a decision tree model with a lifetime horizon. The model explores the cost-effectiveness of rasburicase vs SOC for both preventing TLS and treating established TLS among pediatric patients with acute myeloid leukemia (AML), acute lymphoid leukemia (ALL), and non-Hodgkin’s lymphoma (NHL). Both the prophylaxis-use model and treatment-use model incorporate long-term health states of the diseases: survival without TLS and death. The efficacy data of rasburicase and SOC were obtained from published literature. Drug costs, healthcare resource utilization (HRU), and adverse event (AE) management costs were obtained via a published study with clinical experts. Costs in US dollar and quality-adjusted life year (QALY) are reported, and incremental cost-effectiveness ratios (ICERs) were also calculated. Uncertainties due to parameter fluctuations in the model were assessed through one-way sensitivity analysis and probabilistic sensitivity analysis (PSA).

Results: During TLS prevention, compared with SOC, the ICER of rasburicase treatment in China are $17,580.04/QALY, $5,783.45/QALY, and $5,391.00/QALY for pediatric patients with AML, ALL, and NHL, respectively. For the established TLS treatment, compared with SOC, the ICERs of rasburicase treatment are $2,031.18/QALY, $1,142.93/QALY, and $990.37/QALY for pediatric patients with AML, ALL, and NHL, respectively.

Limitations: The clinical data for SOC are based on the published study in China, and the rasburicase prevention or treatment failure rate was either calculated based on the risk ratio or directly from the clinical study among non-Chinese pediatric patients. Another study limitation was the lack of utility data for pediatric patients with TLS and without TLS. Thus, the utility scores of pediatric cancer survivors were used as an alternative.

Conclusion: Rasburicase is estimated to be a cost-effective alternative to SOC in the prevention and treatment of TLS among Chinese pediatric patients with AML, ALL, and NHL.  相似文献   


4.
Aim: To evaluate the cost-effectiveness of secukinumab, a fully human anti-interleukin-17A monoclonal antibody, compared to other clinically used biologics (adalimumab, infliximab, and ustekinumab) in Japan for the treatment of moderate-to-severe psoriasis from the healthcare system (total costs) and patient co-payment (using different frequencies of drug purchase) perspectives.

Methods: A decision-tree (first year)/Markov model (subsequent years), with an annual cycle, was developed. The model adopted a 5-year time horizon. Efficacy inputs were obtained from a mixed-treatment comparison analysis, and other model inputs were collected from published literature and local Japanese sources. Model outcomes included quality-adjusted life years (QALYs) and an incremental cost-effectiveness ratio (ICER) in terms of cost per QALY gained. The annual discounting rate of 2% was applied to both costs and outcomes.

Results: Results for the healthcare system perspective showed that secukinumab had the highest number of quality-adjusted life years (QALYs) (4.07) vs other biologics, dominated ustekinumab and infliximab, and the ICER of secukinumab compared to adalimumab was ¥8,418,222/QALY gained. In the patient co-payment perspective with the monthly purchase of drugs, ustekinumab had the lowest co-payment cost, followed by infliximab, adalimumab, and secukinumab. In the patient co-payment perspective with a once every 3 months purchase of secukinumab and adalimumab, the co-payment costs of secukinumab, adalimumab, and ustekinumab became comparable, and infliximab had the highest co-payment cost.

Limitations: Only short-term efficacy data was modeled, as there was a lack of data on long-term outcomes. Treatment sequencing was restricted to first-line biologic treatment. Drop-out rates for comparators were assumed to be equivalent to secukinumab in the absence of available data.

Conclusions: Secukinumab is a cost-efficient treatment for moderate-to-severe psoriasis, providing greater health outcomes to patients at lower total costs compared to infliximab and ustekinumab, as well as comparable patient co-payment relative to other biologic treatments.  相似文献   


5.
Aims: Multiple sclerosis (MS) is a chronic, autoimmune, and inflammatory disease. If the first-line medicines are not effective enough, specialists will prescribe second-line medicines, such as natalizumab and fingolimod. This study aimed to compare the cost-effectiveness and cost-utility of fingolimod with those of natalizumab in patients with relapsing-remitting multiple sclerosis (RRMS) in Iran, Fars province in 2016.

Materials and methods: This study was a cost-effectiveness and cost-utility study in which a Markov model was used. The study used the census method to evaluate 81 patients with MS in Iran, Fars province who were being treated with fingolimod and natalizumab. In this study, costs were collected from the societal perspective, and the outcomes were the mean of relapse avoided rate and QALY. The cost data collection form, Kurtzke Expanded Disability Status Scale, and EQ-5D-3L questionnaire were used to collect the required data.

Results: The results showed that, compared to natalizumab, patients who used fingolimod had decreased costs (58,087 vs 201,707), increased QALYs (8.09 vs 7.37), and a better relapse avoided rate (6.27 vs 5.83) per patient over the lifetime. The results of the sensitivity analysis showed that the results of the study were robust. Also, the results of the scatter plots showed that fingolimod was more cost-effective based on the QALY and relapse avoided rate in 62% and 56%, respectively, of the simulations for the thresholds below $15,657 for the studied patients.

Conclusions: According to the results of this study, the cost-effectiveness and cost-utility of fingolimod were higher than those of natalizumab. Therefore, it is recommended that treatment with fingolimod be the first priority of second-line treatment for MS patients, and policy-makers and health managers are encouraged to make efforts in order to increase insurance coverage and reduce the out-of-pocket payments of these patients.  相似文献   


6.
Aims: To carry out a cost-utility analysis comparing the cost-effectiveness of levodopa carbidopa intestinal gel (LCIG) with standard of care (SOC) in patients with advanced Parkinson’s Disease (aPD) unsuitable for apomorphine or deep brain stimulation (DBS). LCIG is the only treatment option in this small, but clinically important, population.

Methods: A Markov model with 25 disease states based on disease stage and off-time status plus death. Patients enter the model with aPD spending >50% of their waking day in the off-state. Patients progress through the model in 6-monthly cycles for 20 years to approximate lifetime treatment and capture long-term costs and effects of therapy. Inputs are based on LCIG clinical trials for clinical outcomes and health state utilities, the literature for health state transitions and use UK-based input data wherever possible (drug costs, disease/adverse event management costs, discontinuation rates, mortality rates).

Limitations: Data collection can be challenging in this small, elderly population with advanced disease, therefore some model inputs were estimated, rather than collected directly. It was assumed that a reduction in off-time was the only benefit after the first year of treatment with LCIG; this is a conservative approach, since there may be additional clinical benefits.

Results: There is a considerable incremental gain in quality adjusted life years (QALYs) for patients treated with LCIG of 1.26 QALY with an associated incremental cost-effectiveness ratio (ICER) of £52,110. If the impact on caregivers is included, the ICER reduces to £47,266.

Conclusions: In cases where there is an orphan population, with no alternative treatment options, HTA assessments have a broader decision-making framework and the ICER is interpreted in this context. In the setting of a very small population, with considerable unmet need, LCIG represents value for money, as reflected by funding approval across the UK.  相似文献   


7.
Background: Polymyxin B and colistin are nephrotoxic drugs used in the treatment of carbapenem-resistant Enterobacteriaceae. The aim of this study is to evaluate the burden of costs due to polymyxin associated AKI and propose a simulated break-even price for new therapies.

Methods: The pharmacoeconomic model is based on two large cross-sectional studies of polymyxin nephrotoxicity. Total direct costs in patients with and without renal failure were compared. The direct cost of each hemodialysis section (USD82.94) and daily hospital charges (USD934.85) were based on the values used in a major public hospital in the city where the clinical study was performed. The break-even price of new drugs was simulated considering eventual new drugs as effective as polymyxins, but less nephrotoxic in different percentages. Outcomes of patients after hospital discharge were not evaluated.

Results: Total direct cost of the group of patients who survived without AKI was significantly lower than total direct cost of the groups either with AKI or the group who died without AKI. There was a tendency of even higher costs in those who died with AKI and dialysis. Direct cost of hemodialysis was not as important as the longer hospitalization after sepsis. Considering daily cost of polymyxin is USD60, drugs with 50% less AKI could be considered cost-beneficial if the daily cost is lower than USD160.

Conclusions: AKI in patients with carbapenem-resistant Enterobacteriaceae treated with polymyxin increases both length of stay in hospital and total costs.  相似文献   


8.
Aims: This study aimed to evaluate the budget impact of niraparib and olaparib in patients with platinum-sensitive, recurrent ovarian cancer from a US third party payer perspective.

Materials and methods: A budget impact model was constructed to assess the additional per member per month (PMPM) costs associated with the introduction of niraparib and olaparib, two poly ADP-ribose polymerase ribose polymerase (PARP) inhibitors recently approved to be used in platinum-sensitive, recurrent ovarian cancer patients with and without a gBRCA mutation. The model assessed both pharmacy costs and medical costs. Pharmacy costs included adjusted drug costs, coinsurance, and dispensing fees. Medical costs included costs associated with disease monitoring and management of adverse events from the treatment. Epidemiological data from the literature were used to estimate the target population size. The analysis used 1-year time frame, and patients were assumed on treatment until disease progression or death. All costs were computed in 2017 USD. One-way sensitivity analyses were conducted to evaluate the model robustness.

Results: In a hypothetical plan of 1,000,000 members, 206 patients were estimated to be potential candidates for niraparib or olaparib maintenance treatment after applying all epidemiological parameters. At listed 30-day supply WAC prices of $14,750 for niraparib and $13,482 for olaparib, budget impacts of these two drugs were $0.169 PMPM and $0.156 PMPM, respectively, most of which were contributed by pharmacy costs. Sensitivity analyses suggested that assumptions around market share, platinum-sensitive rate after first treatment, and WAC prices affected results the most.

Limitations: In this model, it was assumed that adopting niraparib and olaparib would not affect utilization of existing medications. Also, the estimated clinical parameters from clinical trials could differ from real-world data.  相似文献   


9.
Aims: Percutaneous closure of a patent foramen ovale (PFO) is known to lower the risk of recurrent stroke in patients with a cryptogenic stroke. However, the economic implications of transcatheter PFO closure are less well known. From a UK payer perspective, a detailed economic appraisal of PFO closure was performed for prevention of recurrent ischemic stroke in patients with a PFO who had experienced a cryptogenic stroke.

Materials and methods: A Markov cohort model was constructed using a 5-year time-horizon with a patient mean age of 45.2 years, reflecting the characteristics reported in the REDUCE trial. Transition probabilities, clinical inputs, costs, and utility values were ascertained from published and national costing sources. Total costs, incremental costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios were calculated, utilizing a discount rate of 3.5%. A range of univariate and probabilistic sensitivity analyses were also performed.

Results: When applying a willingness-to-pay (WTP) threshold of £20,000/QALY in accordance with NICE guidelines, PFO closure compared with antiplatelet therapy alone showed a beneficial cost/QALY of £18,584, attained at 4 years. Applying discount rates of 0% and 6% had a negligible effect on the base-case model findings. PFO closure demonstrated a 76.9% probability of being cost-effective at a WTP threshold of £20,000/QALY at a 5-year time-horizon.

Limitations: This model focused specifically on UK stroke patients and typically enrolled young (mean age <65 years old) patients. Hence, caution should be taken when comparing data vs non-UK populations, and it remains unclear how older patients might have affected cost-effectiveness findings, as the risk of paradoxical embolism can persist as patients age.

Conclusion: Percutaneous closure of a PFO is cost-effective compared with antiplatelet therapy alone, underlining the economic benefits potentially afforded by this treatment in selected patients.  相似文献   


10.
Background: Non-vitamin K antagonist oral anticoagulants (NOACs) have been included in international guidelines as important alternatives to vitamin K antagonists (VKAs) for the treatment of venous thromboembolism (VTE) and stroke prevention in non-valvular atrial fibrillation (NVAF). Meanwhile, in the Netherlands, NOACs are widely used next to VKAs. The objective of this study is to estimate the cost-effectiveness of treatment with rivaroxaban compared to VKAs in NVAF and VTE patients in the Netherlands, using data from international prospective observational phase IV studies.

Methods: Two models were developed to represent NVAF and VTE patients, populated with patients from the XANTUS (NCT01606995) and XALIA (NCT01619007) international prospective observational studies. The 1-year cost-effectiveness of rivaroxaban use, compared to VKAs, was explored in a population consisting of NVAF and VTE patients (base case) as well as for four scenarios with sub-populations: NVAF patients only, VTE patients only, NVAF patients with unstable international normalized ratio (INR), and NVAF patients using an INR self-measuring device.

Results: In the base case, rivaroxaban saved €72,350 and gained 21 quality-adjusted life-years (QALYs) in a simulation of 2,000 patients over the use of VKAs. Ergo, rivaroxaban was dominant over VKAs. The probabilistic sensitivity analysis showed a probability of 85% for rivaroxaban being dominant and 100% at a willingness-to-pay threshold of €20,000/QALY. Rivaroxaban appeared to be dominant in all scenarios as well, except for the NVAF-patients-only scenario where the incremental cost-effectiveness ratio (ICER) was €157/QALY.

Conclusions: In patients with NVAF or VTE, rivaroxaban treatment is likely to be cost-effective and a potentially cost-saving alternative to VKA in the Netherlands.  相似文献   


11.
Aims: Broad molecular profiling of patients with advanced non-small cell lung cancer (NSCLC) is strongly advised to optimize genomic matching with available targeted treatment options or investigational agents. Unlike conventional molecular diagnostic testing, or smaller hotspot panels, comprehensive genomic profiling (CGP) identifies genomic alterations across hundreds of clinically relevant cancer genes from a single tissue specimen. The present study sought to estimate the budget impact of increased use of CGP using a 324-gene panel (FoundationOne) vs non-CGP (represented by a mix of conventional molecular diagnostic testing and smaller NGS hotspot panels) and the number needed to test with CGP to gain 1 life year.

Materials and methods: A decision analytic model was developed to assess the budget impact of increased CGP in advanced NSCLC from a US private payer perspective. Model inputs were based on published literature (epidemiology and treatment outcomes), real-world data (testing and rates, medical service costs), list prices for CGP and anti-cancer drugs, and assumptions for clinical trial participation.

Results: Among 2 million covered lives, 532 had advanced NSCLC; 266 underwent molecular diagnostic testing. An increase in CGP among those tested, from 2% to 10%, was associated with $0.02 per member per month budget impact, of which $0.013 was attributable to costs of prolonged drug treatment and survival and $0.005 to testing cost. Approximately 12 patients would need to be tested with CGP to add 1 life year.

Limitations: The model incorporated certain assumptions to account for inputs with a limited evidence profile and simplify the possible post-CGP treatments.

Conclusions: An increase in CGP utilization from 2% to 10% among patients with advanced NSCLC undergoing molecular diagnostic testing was associated with a modest budget impact, most of which was attributable to increased use of more effective treatments and prolonged survival.  相似文献   


12.
Aims: There is an increasing interest in understanding patients’ preferences in the area of healthcare decision-making to better match treatment with patients’ preferences and improve treatment uptake and adherence. The aim of this study was to elicit the preferences of patients with a depressive disorder regarding treatment modalities.

Materials and methods: In a discrete-choice experiment, patients chose repetitively between two hypothetical depression treatments that varied in four treatment attributes: waiting time until the start of treatment, treatment intensity, level of digitalization, and group size. A Bayesian-efficient design was used to develop 12 choice sets, and patients’ preferences and preference variation was estimated using a random parameters logit model.

Results: A total of 165 patients with depression completed the survey. Patients preferred short (over long) waiting times, face-to-face (over digital) treatment, individual (over group) treatment, and one session per week over two sessions per week or one session per 2 weeks. Patients disfavoured digital treatment and treatment in a large group. Waiting time and treatment intensity were substantially less important attributes to patients than face-to-face (vs digital) and group size. Significant variation in preferences was observed for each attribute, and sub-group analyses revealed that these differences were in part related to education.

Limitations: The convenience sample over-represented the female and younger population, limiting generalizability. Limited information on background characteristics limited the possibilities to explore preference heterogeneity.

Conclusion: This study demonstrated how different treatment components for depression affect patients’ preferences for those treatments. There is significant variation in treatment preferences, even after accounting for education. Incorporating individual patients’ preferences into treatment decisions could potentially lead to improved adherence of treatments for depressive disorders.  相似文献   


13.
Aims: To estimate the impact of anti-vascular endothelial growth factor (VEGF) agents on visual impairment and blindness avoided in patients with diabetic macular edema (DME) and on associated patient and caregiver productivity loss in Japan.

Methods: This study compared the impact of current care (estimated at 53.8% utilization of anti-VEGF agents using current data) with that of hypothetical care (characterized by a higher utilization of anti-VEGF agents [80.0%], as estimated by an expert panel) of DME patients. A population-based Markov model (two-eye approach) simulated visual acuity (Early Treatment Diabetic Retinopathy Study [ETDRS] letters) transitions over 5 years with DME treatments (intravitreal aflibercept, ranibizumab, and triamcinolone acetonide, and grid/focal laser) in patients with DME. Patient and caregiver productivity loss was determined using the human capital method.

Results: In total, 570,000 DME patients were included in the model over 5 years. Increased utilization of anti-VEGF agents resulted in 6,659 fewer cases of severe visual impairment (SVI; 26–35 ETDRS letters) or blindness (0–25 ETDRS letters) compared with the current care approach (26,023 vs 32,682 cases; 20.38% reduction) over this period. Increased utilization of anti-VEGF agents also contributed to productivity loss savings of ¥12.58 billion (US $115.64 million) (i.e., 17.01%) at the end of year 5. The total overall saving over 5 years was ¥45.83 billion (US $421.27 million) (13.45%).

Limitations: Few Japanese data were available, and assumptions were made for some inputs. Vision changes dependent on the function of both eyes were not studied. Only intravitreal (not sub-Tenon’s) injections of triamcinolone were considered in this model. Direct costs were not considered.

Conclusions: Increased utilization of anti-VEGF agents can reduce SVI and legal blindness in patients with DME in Japan. This would also be associated with substantial savings in patient and caregiver productivity loss.  相似文献   


14.
15.
Aims: Non-vitamin K antagonist oral anticoagulants (NOACs) and vitamin K antagonists (VKAs) are used to prevent stroke in patients with atrial fibrillation (AF). This paper aimed to evaluate the clinical efficacy and safety of NOACs when compared to VKAs by calculating the number needed to treat (NNT) at 2 years using incidence rates and hazard ratios (HRs) derived from a meta-analysis of studies conducted in real-world settings.

Materials and methods: HRs were sourced from a published systematic literature review and a meta-analysis of real-world evidence on the use of NOACs vs VKAs. Rivaroxaban, dabigatran, and apixaban vs VKAs were investigated. The efficacy outcomes included: a composite of ischaemic stroke and systemic embolism (IS/SE), ischaemic stroke (IS), and all-cause mortality. The safety analysis assessed major bleeding and intracranial haemorrhage (ICH).

Results: Superiority of NOACs vs VKAs was observed in 10/15 comparisons. Treating patients with rivaroxaban and dabigatran was associated with a reduced risk of IS and all-cause mortality compared to VKAs, with one death prevented every 22 and 32 patients, respectively, and one IS prevented every 206 and 166 patients, respectively. Rivaroxaban was significantly associated with a reduced risk of IS/SE compared to VKA (NNT: 107). No significant differences were observed between apixaban and VKAs. Dabigatran and apixaban were associated with a reduced risk of major bleeding compared to VKA (NNT: 59 and 38, respectively). No significant difference was observed between rivaroxaban and VKAs regarding major bleeding. Rivaroxaban, dabigatran, and apixaban were significantly associated with a reduced risk of ICH (NNT: 205, 115, and 108, respectively).

Limitations: Heterogeneity in definitions of major bleeding across studies.

Conclusions: The NNT calculation, when approached and interpreted properly, is a practical measure of the effectiveness of a treatment. The calculation based on HRs showed that NOACs are safe and effective alternatives to VKAs in real life.  相似文献   


16.
Aims: This study analyzed discrepancies in the quantity of medical services supplied by physicians under different payment systems for patients with different health statuses and illnesses by means of a field experiment.

Methods: Based on the laboratory experiment of Heike Hennig-Schmidt, we designed a field experiment to examine fee-for-service (FFS), capitation (CAP), and diagnosis-related group (DRG) payment systems. Medical students were replaced with 220 physicians as experimental subjects, which more closely reflected the clinical choices made by physicians in the real world. Under the three payment mechanisms, the quantity of medical services provided by physicians when they treated patients with different health statuses and illnesses were collected. Finally, relevant statistics were computed and analyzed.

Results: It was found that payment systems (sig. = 0.000) and patient health status (sig. = 0.000) had a stronger effect on physicians’ choices regarding quantity of medical services than illness types (sig. = 0.793). Additionally, under the FFS and CAP payment systems, physicians overserved patients in good and intermediate health while underserving patients in bad health. Under the DRG payment system, physicians overserved patients in good health and underserved patients in intermediate and bad health. Correspondingly, under FFS and CAP, the proportional loss of benefits was the highest for patients in bad health and the lowest for patients in good and intermediate health; while under DRGs, patients in good and intermediate health had the largest and smallest loss of benefits, respectively.

Limitations: In order to increase external effects of experiment results, we used the field experiment to replace laboratory experiment. However, the external effects still existed because of the blurring and abstraction of the parameters.

Conclusions: Medical treatment cost and price affected the quantity of medical services provided by physicians. Therefore, we proposed that a mix of payment systems could address the common interests of physicians and patients, as well as influence incentives from payment systems.  相似文献   


17.
Objective: To investigate preferences for fertility treatment from the Australian general population with the aims of calculating the willingness to pay in tax contribution for attributes (characteristics) that make up treatment and for an “ideal” fertility treatment program. We also assessed whether willingness-to-pay varies by the relationship status or sexual orientation of the patient.

Methods: A stated preference discrete choice experiment was administered to a panel of 801 individuals representative of the Australian general population. Seven attributes of fertility treatment under three broad categories were included: outcome, process, and cost. Attributes were identified through published literature, focus group discussions, expert knowledge, and a pilot study. A Bayesian fractional experimental design was used, and data analysis was performed using a generalized multinomial logit model. Further analyses included interaction terms and latent class modeling.

Results: Six of the seven attributes influenced the choice of a treatment program. Under process attributes, individuals preferred: continuity of care of clinic staff, where patients are seen by the same doctor but different nurses at each visit; “alternative” treatments being offered to all patients; and onsite clinic counseling and peer-support groups. Personalization and tailoring of the treatment journey were not important. Among outcome attributes, the improved success rate of having a baby per cycle and significant side-effects were considered important. Cost of treatment also influenced the choice of treatment program. Individual preferences for fertility treatment were not associated with patients’ relationship status or sexual orientation. Latent class modeling revealed sub-groups with distinct fertility treatment preferences.

Conclusion: This study provides important insights into the attributes that influence the preferences of fertility treatment in Australia. It also estimates socially-inclusive willingness-to-pay values in tax contributions for an “ideal” package of treatment. The results can inform economic evaluations of fertility treatment programs.  相似文献   


18.
Background: Validation of overall survival (OS) extrapolations of immune-checkpoint inhibitors (ICIs) during the National Institute for Health and Care Excellence (NICE) Single Technology Assessment (STA) process is limited due to data still maturing at the time of submission. Inaccurate extrapolation may lead to inappropriate decision-making. The availability of more mature trial data facilitates a retrospective analysis of the plausibility and validity of initial extrapolations. This study compares these extrapolations to subsequently available longer-term data.

Methods: A systematic search of completed NICE appraisals of ICIs from March 2000 to December 2017 was performed. A targeted search was also undertaken to procure published OS data from the pivotal clinical trials for each identified STA made available post-submission to NICE. Initial Kaplan-Meier curves and associated extrapolations from NICE documentation were extracted to compare the accuracy of OS projections versus the most mature data.

Results: The review identified 11 STAs, of which 10 provided OS data upon submission to NICE. The extrapolations undertaken considered parametric or piecewise survival models. Additional data cut-offs provided a mean of 18 months of OS beyond the end of the original data. Initial extrapolations typically under-estimated OS from the most mature data cut-off by 0.4–2.7%, depending on the choice of assessment method and use of the manufacturer- or ERG-preferred extrapolation.

Conclusion: Long-term extrapolation of OS is required for NICE STAs based on initial immature OS data. The results of this study demonstrate that the initial OS extrapolations employed by manufacturers and ERGs generally predicted OS reasonably well when compared to more mature data (when available), although on average they appeared to underestimate OS. This review and validation shows that, while the choice of OS extrapolation is uncertain, the methods adopted are generally aligned with later-published follow-up data and appear appropriate for informing HTA decisions.  相似文献   


19.
Aims: To model direct medical costs associated with reductions in cardiovascular disease (CVD) events in T2DM patients reported in the CANVAS and EMPA-REG trials, which assessed the cardiovascular safety of canagliflozin and empagliflozin, respectively.

Materials and methods: Costs were modeled from a US managed care organization (MCO) perspective for the CVD outcomes included in both trials: three-point major adverse cardiovascular event (MACE) and its components (cardiovascular-related death, nonfatal myocardial infarction, nonfatal stroke), as well as heart failure requiring hospitalization. The rate of CVD events averted (difference between study drug and placebo) was projected to the portion of an MCO T2DM population matching the respective trial’s inclusion criteria. A targeted literature search for paid amounts directly associated with each CVD event provided the unit costs, which were applied to the projected number of events averted, to calculate costs avoided per member per year (PMPY). One-way sensitivity analyses were performed on events averted, unit costs, and percentages of trial-applicable patients.

Results: Based on three-point MACE events averted, costs avoided PMPY of $6.17 (range: $1.27–$10.94) for CANVAS and $2.75 ($0.19–$4.83) for EMPA-REG were estimated. Costs avoided for individual components of MACE ranged from $0.77 to $3.84 PMPY for CANVAS and from -$0.97 (additional costs) to $1.54 for EMPA-REG. PMPY costs avoided for heart failure were $2.72 for CANVAS and $1.32 for EMPA-REG.

Limitations and conclusions: Models assumed independent, non-recurrent outcomes and were restricted to medical costs directly associated with the trial-reported events. The reductions in CVD events in T2DM patients reported for both CANVAS and EMPA-REG project to a positive cost avoidance for these events in an MCO population. The analysis did not include an assessment of the impact on total cost, as the costs associated with adverse events, drug utilization or other clinical outcomes were not examined.  相似文献   


20.
Background: Chemotherapy-induced nausea and vomiting (CINV) are among the most common and debilitating side-effects patients experience during chemotherapy, and are associated with considerable acute care use and healthcare cost. It is estimated that 70–80% of CINV could be prevented through appropriate use of CINV prophylaxis; however, suboptimal CINV compliance and control remains an issue in clinical practice. Netupitant/palonosetron (NEPA) is a fixed combination of serotonin-3 (5-HT3) and neurokinin-1 (NK1) receptor antagonists (RAs), respectively, indicated for the prevention of acute and delayed nausea and vomiting associated with highly emetogenic chemotherapy (HEC) and moderately emetogenic chemotherapy (MEC). Phase 3 clinical trials showed a significantly higher complete response rate in both acute and delayed CINV in chemotherapy-naïve patients receiving NEPA compared to patients receiving palonosetron.

Objective: The objective of this study was to estimate the budgetary impact of adding NEPA to a US payer or practice formulary for CINV prophylaxis.

Methods: A model was developed to estimate the impact of adding NEPA to the formulary of a hypothetical US payer with 1.15 million members, including 150,000 (13%) Medicare beneficiaries. The model compared the annual total costs of CINV-related events and CINV prophylaxis in two scenarios: base year (no NEPA) and comparator year (10% and 5% NEPA usage in HEC and MEC patients, respectively). A univariate sensitivity analysis was conducted to explore the effect of variability in model parameters on the budget impact.

Results: A total of 2,021 patients were eligible to receive CINV prophylaxis. With NEPA, CINV prophylaxis costs increased by 0.7% ($3,493,630 vs $3,518,760) while medical costs associated with CINV events decreased by 3.9% ($15,118,639 vs $14,532,442), resulting in a net cost saving of $561,067 (3.0%) for the health plan ($18,612,269 vs $18,051,202), or Background: Chemotherapy-induced nausea and vomiting (CINV) are among the most common and debilitating side-effects patients experience during chemotherapy, and are associated with considerable acute care use and healthcare cost. It is estimated that 70–80% of CINV could be prevented through appropriate use of CINV prophylaxis; however, suboptimal CINV compliance and control remains an issue in clinical practice. Netupitant/palonosetron (NEPA) is a fixed combination of serotonin-3 (5-HT3) and neurokinin-1 (NK1) receptor antagonists (RAs), respectively, indicated for the prevention of acute and delayed nausea and vomiting associated with highly emetogenic chemotherapy (HEC) and moderately emetogenic chemotherapy (MEC). Phase 3 clinical trials showed a significantly higher complete response rate in both acute and delayed CINV in chemotherapy-naïve patients receiving NEPA compared to patients receiving palonosetron.

Objective: The objective of this study was to estimate the budgetary impact of adding NEPA to a US payer or practice formulary for CINV prophylaxis.

Methods: A model was developed to estimate the impact of adding NEPA to the formulary of a hypothetical US payer with 1.15 million members, including 150,000 (13%) Medicare beneficiaries. The model compared the annual total costs of CINV-related events and CINV prophylaxis in two scenarios: base year (no NEPA) and comparator year (10% and 5% NEPA usage in HEC and MEC patients, respectively). A univariate sensitivity analysis was conducted to explore the effect of variability in model parameters on the budget impact.

Results: A total of 2,021 patients were eligible to receive CINV prophylaxis. With NEPA, CINV prophylaxis costs increased by 0.7% ($3,493,630 vs $3,518,760) while medical costs associated with CINV events decreased by 3.9% ($15,118,639 vs $14,532,442), resulting in a net cost saving of $561,067 (3.0%) for the health plan ($18,612,269 vs $18,051,202), or $0.04 per member per month. This was equivalent to saving $5,011 per patient moved to NEPA. Among all 5-HT3 RA?+?NK1 RA regimens, NEPA was associated with the lowest CINV-related costs, leading to the lowest total cost of care.

Conclusions: Adding NEPA to a payer or practice formulary results in a net decrease in the total budget due to a substantial reduction in CINV event-related resource utilization and medical costs, and an increase in pharmacy costs <1%, saving over $5,000 per patient.  相似文献   


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