scholarly journals Immunoglobulin Replacement Therapy is critical and cost-effective in increasing life expectancy and quality of life in patients suffering from Common Variable Immunodeficiency Disorders (CVID): A health-economic assessment

PLoS ONE ◽  
2021 ◽  
Vol 16 (3) ◽  
pp. e0247941
Author(s):  
Philippe van Wilder ◽  
Irina Odnoletkova ◽  
Mehdi Mouline ◽  
Esther de Vries

Background Common variable immunodeficiency disorders (CVID), the most common form of primary antibody deficiency, are rare conditions associated with considerable morbidity and mortality. The clinical benefit of immunoglobulin replacement therapy (IgGRT) is substantial: timely treatment with appropriate doses significantly reduces mortality and the incidence of CVID-complications such as major infections and bronchiectasis. Unfortunately, CVID-patients still face a median diagnostic delay of 4 years. Their disease burden, expressed in annual loss of disability-adjusted life years, is 3-fold higher than in the general population. Hurdles to treatment access and reimbursement by healthcare payers may exist because the value of IgGRT is poorly documented. This paper aims to demonstrate cost-effectiveness and cost-utility (on life expectancy and quality) of IgGRT in CVID. Methods and findings With input from a literature search, we built a health-economic model for cost-effectiveness and cost-utility assessment of IgGRT in CVID. We compared a mean literature-based dose (≥450mg/kg/4wks) to a zero-or-low dose (0 to ≤100 mg/kg/4wks) in a simulated cohort of adult patients from time of diagnosis until death; we also estimated the economic impact of diagnostic delay in this simulated cohort. Compared to no or minimal treatment, IgGRT showed an incremental benefit of 17 life-years (LYs) and 11 quality-adjusted life-years (QALYs), resulting in an incremental cost-effectiveness ratio (ICER) of €29,296/LY and €46,717/QALY. These results were robust in a sensitivity analysis. Reducing diagnostic delay by 4 years provided an incremental benefit of six LYs and four QALYs compared to simulated patients with delayed IgGRT initiation, resulting in an ICER of €30,374/LY and €47,495/QALY. Conclusions The health-economic model suggests that early initiation of IgGRT compared to no or delayed IgGRT is highly cost-effective. CVID-patients’ access to IgGRT should be facilitated, not only because of proven clinical efficacy, but also due to the now demonstrated cost-effectiveness.

2019 ◽  
Author(s):  
Elin Lindsäter ◽  
Erland Axelsson ◽  
Sigrid Salomonsson ◽  
Fredrik Santoft ◽  
Brjánn Ljótsson ◽  
...  

BACKGROUND Stress-related disorders are associated with significant suffering, functional impairment, and high societal costs. Internet-based cognitive behavioral therapy (ICBT) is a promising treatment for stress-related disorders but has so far not been subjected to health economic evaluation. OBJECTIVE The objective of this study was to evaluate the cost-effectiveness and cost-utility of ICBT for patients with stress-related disorders in the form of adjustment disorder (AD) or exhaustion disorder (ED). We hypothesized that ICBT, compared with a waitlist control (WLC) group, would generate improvements at low net costs, thereby making it cost-effective. METHODS Health economic data were obtained in tandem with a randomized controlled trial of a 12-week ICBT in which patients (N=100) were randomized to an ICBT (n=50) or a WLC (n=50) group. Health outcomes and costs were surveyed pre- and posttreatment. We calculated incremental cost-effectiveness ratios (ICERs) based on remission rates and incremental cost-utility ratios (ICURs) based on health-related quality of life. Bootstrap sampling was used to assess the uncertainty of our results. RESULTS The ICER indicated that the most likely scenario was that ICBT led to higher remission rates compared with the WLC and was associated with slightly larger reductions in costs from pre- to posttreatment. ICBT had a 60% probability of being cost-effective at a willingness to pay (WTP) of US $0 and a 96% probability of being cost-effective at a WTP of US $1000. The ICUR indicated that ICBT also led to improvements in quality of life at no net societal cost. Sensitivity analyses supported the robustness of our results. CONCLUSIONS The results suggest that ICBT is a cost-effective treatment for patients suffering from AD or ED. Compared with no treatment, ICBT for these patients yields large effects at no or minimal societal net costs. CLINICALTRIAL ClinicalTrials.gov NCT02540317; https://clinicaltrials.gov/ct2/show/NCT02540317


10.2196/14675 ◽  
2019 ◽  
Vol 21 (9) ◽  
pp. e14675
Author(s):  
Elin Lindsäter ◽  
Erland Axelsson ◽  
Sigrid Salomonsson ◽  
Fredrik Santoft ◽  
Brjánn Ljótsson ◽  
...  

Background Stress-related disorders are associated with significant suffering, functional impairment, and high societal costs. Internet-based cognitive behavioral therapy (ICBT) is a promising treatment for stress-related disorders but has so far not been subjected to health economic evaluation. Objective The objective of this study was to evaluate the cost-effectiveness and cost-utility of ICBT for patients with stress-related disorders in the form of adjustment disorder (AD) or exhaustion disorder (ED). We hypothesized that ICBT, compared with a waitlist control (WLC) group, would generate improvements at low net costs, thereby making it cost-effective. Methods Health economic data were obtained in tandem with a randomized controlled trial of a 12-week ICBT in which patients (N=100) were randomized to an ICBT (n=50) or a WLC (n=50) group. Health outcomes and costs were surveyed pre- and posttreatment. We calculated incremental cost-effectiveness ratios (ICERs) based on remission rates and incremental cost-utility ratios (ICURs) based on health-related quality of life. Bootstrap sampling was used to assess the uncertainty of our results. Results The ICER indicated that the most likely scenario was that ICBT led to higher remission rates compared with the WLC and was associated with slightly larger reductions in costs from pre- to posttreatment. ICBT had a 60% probability of being cost-effective at a willingness to pay (WTP) of US $0 and a 96% probability of being cost-effective at a WTP of US $1000. The ICUR indicated that ICBT also led to improvements in quality of life at no net societal cost. Sensitivity analyses supported the robustness of our results. Conclusions The results suggest that ICBT is a cost-effective treatment for patients suffering from AD or ED. Compared with no treatment, ICBT for these patients yields large effects at no or minimal societal net costs. Trial Registration ClinicalTrials.gov NCT02540317; https://clinicaltrials.gov/ct2/show/NCT02540317


2020 ◽  
Vol 14 (8) ◽  
Author(s):  
Anna Parackal ◽  
Jean-Eric Tarride ◽  
Feng Xie ◽  
Gord Blackhouse ◽  
Jennifer Hoogenes ◽  
...  

Introduction: Recent health technology assessments (HTAs) of robot-assisted radical prostatectomy (RARP) in Ontario and Alberta, Canada, resulted in opposite recommendations, calling into question whether benefits of RARP offset the upfront investment. Therefore, the study objectives were to conduct a cost-utility analysis from a Canadian public payer perspective to determine the cost-effectiveness of RARP. Methods: Using a 10-year time horizon, a five-state Markov model was developed to compare RARP to open radical prostatectomy (ORP). Clinical parameters were derived from Canadian observational studies and a recently published systematic review. Costs, resource utilization, and utility values from recent Canadian sources were used to populate the model. Results were presented in terms of increment costs per quality-adjusted life years (QALYs) gained. A probabilistic analysis was conducted, and uncertainty was represented using cost-effectiveness acceptability curves (CEACs). One-way sensitivity analyses were also conducted. Future costs and QALYs were discounted at 1.5%. Results: Total cost of RARP and ORP were $47 033 and $45 332, respectively. Total estimated QALYs were 7.2047 and 7.1385 for RARP and ORP, respectively. The estimated incremental cost-utility ratio (ICUR) was $25 704 in the base-case analysis. At a willingness-to-pay threshold of $50 000 and $100 000 per QALY gained, the probability of RARP being cost-effective was 0.65 and 0.85, respectively. The model was most sensitive to the time horizon. Conclusions: The results of this analysis suggest that RARP is likely to be cost-effective in this Canadian patient population. The results are consistent with Alberta’s HTA recommendation and other economic evaluations, but challenges Ontario’s reimbursement decision.


2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 8043-8043
Author(s):  
Mavis Obeng-Kusi ◽  
Daniel Arku ◽  
Neda Alrawashdh ◽  
Briana Choi ◽  
Nimer S. Alkhatib ◽  
...  

8043 Background: IXA, CAR, ELO and DARin combination with LEN+DEXhave been found superior in efficacy compared to LEN+DEX in the management of R/R MM. Applying indirect treatment comparisons from a network meta-analysis (NMA), this economic evaluation aimed to estimate the comparative cost-effectiveness and cost-utility of these four triplet regimens in terms of progression-free survival (PFS). Methods: In the absence of direct treatment comparison from a single clinical trial, NMA was used to indirectly estimate the comparative PFS benefit of each regimen. A 2-state Markov model simulating the health outcomes and costs was used to evaluate PFS life years (LY) and quality-adjusted life years (QALY) with the triplet regimens over LEN+DEX and expressed as the incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR). Probability sensitivity analyses were conducted to assess the influence of parameter uncertainty on the model. Results: The NMA revealed that DAR+LEN+DEX was superior to the other triplet therapies, which did not differ statistically amongst them. As detailed in the Table, in our cost-effectiveness analysis, all 4 triplet regimens were associated with increased PFSLY and PFSQALY gained (g) over LEN+DEX at an additional cost. DAR+LEN+DEX emerged the most cost-effective with ICER and ICUR of $667,652/PFSLYg and $813,322/PFSQALYg, respectively. The highest probability of cost-effectiveness occurred at a willingness-to-pay threshold of $1,040,000/QALYg. Conclusions: Our economic analysis shows that all the triplet regimens were more expensive than LEN +DEX only but were also more effective with respect to PFSLY and PFSQALY gained. Relative to the other regimens, the daratumumab regimen was the most cost-effective.[Table: see text]


2018 ◽  
Author(s):  
Fanny Kählke ◽  
Claudia Buntrock ◽  
Filip Smit ◽  
Matthias Berking ◽  
Dirk Lehr ◽  
...  

BACKGROUND Work-related stress is widespread among employees and associated with high costs for German society. Internet-based stress management interventions (iSMIs) are effective in reducing such stress. However, evidence for their cost-effectiveness is scant. OBJECTIVE The aim of this study was to assess the cost-effectiveness of a guided iSMI for employees. METHODS A sample of 264 employees with elevated symptoms of perceived stress (Perceived Stress Scale≥22) was assigned to either the iSMI or a waitlist control condition (WLC) with unrestricted access to treatment as usual. Participants were recruited in Germany in 2013 and followed through 2014, and data were analyzed in 2017. The iSMI consisted of 7 sessions plus 1 booster session. It was based on problem-solving therapy and emotion regulation techniques. Costs were measured from the societal perspective, including all direct and indirect medical costs. We performed a cost-effectiveness analysis and a cost-utility analysis relating costs to a symptom-free person and quality-adjusted life years (QALYs) gained, respectively. Sampling uncertainty was handled using nonparametric bootstrapping (N=5000). RESULTS When the society is not willing to pay anything to get an additional symptom-free person (eg, willingness-to-pay [WTP]=€0), there was a 70% probability that the intervention is more cost-effective than WLC. This probability rose to 85% and 93% when the society is willing to pay €1000 and €2000, respectively, for achieving an additional symptom-free person. The cost-utility analysis yielded a 76% probability that the intervention is more cost-effective than WLC at a conservative WTP threshold of €20,000 (US $25,800) per QALY gained. CONCLUSIONS Offering an iSMI to stressed employees has an acceptable likelihood of being cost-effective compared with WLC. CLINICALTRIAL German Clinical Trials Register DRKS00004749; https://www.drks.de/DRKS00004749 INTERNATIONAL REGISTERED REPOR RR2-10.1186/1471-2458-13-655


2021 ◽  
Author(s):  
Padraig Dixon ◽  
Edna Keeney ◽  
Jenny C Taylor ◽  
Sarah Wordsworth ◽  
Richard Martin

Polygenic risk is known to influence susceptibility to cancer. The use of data on polygenic risk, in conjunction with other predictors of future disease status, may offer significant potential for preventative care through risk-stratified screening programmes. An important element in the evaluation of screening programmes is their cost-effectiveness. We undertook a systematic review of papers evaluating the cost-effectiveness of screening interventions informed by polygenic risk scores compared to more conventional screening modalities. We included papers reporting cost-effectiveness outcomes in the English language published as articles or uploaded onto preprint servers with no restriction on date, type of cancer or form of polygenic risk modelled. We excluded papers evaluating screening interventions that did not report cost-effectiveness outcomes or which had a focus on monogenic risk. We evaluated studies using the Quality of Health Economic Studies checklist. Ten studies were included in the review, which investigated three cancers: prostate (n=5), colorectal (n=3) and breast (n=2). All study designs were cost-utility papers implemented as Markov models (n=6) or microsimulations (n=4). Nine of ten papers scored highly (score >75 on a 0-100) scale) when assessed using the Quality of Health Economic Studies checklist. Eight of ten studies concluded that polygenic risk informed cancer screening was likely to be more cost-effective than alternatives. However, the included studies lacked robust external data on the cost of polygenic risk stratification, did not account for how very large volumes of polygenic risk data on individuals would be collected and used, did not consider ancestry-related differences in polygenic risk, and did not fully account for downstream economic sequalae stemming from the use of polygenic risk data in these ways. These topics merit attention in future research on how polygenic risk data might contribute to cost-effective cancer screening.


2021 ◽  
Vol 9 ◽  
Author(s):  
Hui Jun Zhou ◽  
Jing Cao ◽  
Hui Shi ◽  
Nasheen Naidoo ◽  
Sherehe Semba ◽  
...  

Background: Hepatitis C virus (HCV) genotype 1 is the most prevalent HCV infection in China. Sofosbuvir-based direct antiviral agent (DAA) regimens are the current mainstays of treatment. Sofosbuvir/velpatasvir (SOF/VEL) and sofosbuvir/ledipasvir (SOF/LDV) regimens became reimbursable in China in 2020. Thus, this study aimed to identify the optimal SOF-based regimen and to inform efficient use of healthcare resources by optimizing DAA use in treating HCV genotype 1.Methods and Models: A modeling-based cost-utility analysis was conducted from the payer's perspective targeting adult Chinese patients with chronic HCV genotype 1 infection. Direct medical costs and health utilities were inputted into a Markov model to simulate lifetime experiences of chronically infected HCV patients after receiving SOF/LDV, SOF/VEL or the traditional strategy of pegylated interferon (pegIFN) + ribavirin (RBV). Discounted lifetime cost and quality adjusted life years (QALYs) were computed and compared to generate the incremental cost utility ratio (ICUR). An ICUR below the threshold of 31,500 $/QALY suggests cost-effectiveness. Deterministic and probabilistic sensitivity analyses were performed to examine the robustness of model findings.Results: Both SOF/LDV and SOF/VEL regimens were dominant to the pegIFN + RBV regimen by creating more QALYs and incurring less cost. SOF/LDV produced 0.542 more QALYs but cost $10,390 less than pegIFN + RBV. Relative to SOF/LDV, SOF/VEL had an ICUR of 168,239 $/QALY which did not meet the cost-effectiveness standard. Therefore SOF/LDV was the optimal strategy. These findings were robust to linear and random variations of model parameters. However, reducing the SOF/VEL price by 40% would make this regimen the most cost-effective option.Conclusions: SOF/LDV was found to be the most cost-effective treatment, and SOF/VEL was also economically dominant to pegIFN + RBV. These findings indicated that replacing pegIFN + RBV with DAA regimens could be a promising strategy.


2018 ◽  
Vol 3 (3) ◽  
pp. 2473011418S0028
Author(s):  
Aditya Karhade ◽  
John Kwon

Category: Other Introduction/Purpose: Orthopedic surgery lies at the forefront of the initiative to deliver value-based care. As a result, cost utility analyses have gained increased importance in the orthopedic literature. While other authors have previously reviewed the quality of US-based cost-utility analyses in orthopedic trauma, spine, sports, and arthroplasty, a similar analysis specific to foot and ankle surgery has not been performed. As such, the practicing foot and ankle surgeon does not yet have a comprehensive analysis of the available cost-utility analyses or the quality of the available evidence. The purpose of this study was to perform a systematic review of cost-utility analyses in foot and ankle surgery. Methods: A systematic review of foot and ankle studies was conducted to identify cost-utility based analyses published between 2000 and 2017. Of 687 studies screened by abstract, eight cost-utility foot and ankle studies were identified that met inclusion criteria and were scored by the Quality of Health Economic Studies instrument for overall quality. Results: Of the eight studies that met inclusion criteria, four were published in the U.S. Regarding topic, three examined end-stage arthritis, two examined diabetic foot ulcers, two examined ankle fractures and one examined ankle sprains. Cost-effective interventions identified by these studies included total ankle arthroplasty over ankle fusion or nonoperative treatment, adjunctive hyperbaric oxygen therapy for diabetic foot ulcers, suture button fixation over syndesmotic screws for unstable supination-external rotation type IV ankle fractures, and below knee cast and fracture boot immobilization over tubular bandages for acute severe ankle sprains respectively. The mean Quality of Health Economic Studies scores for these studies was 81.9 with interquartile range, 25th percentile score of 74.8 to 75th percentile score of 86. Conclusion: Despite the increasing focus on value-based care delivery, there are few foot and ankle cost-utility analyses in the current literature. Nonetheless, the quality of existing analyses is high as rated by the Quality of Health Economic Studies and certain interventions have been identified as cost-effective as highlighted above. The findings of this review can be used to help design future analyses in order to best demonstrate the cost-effectiveness of foot and ankle interventions.


Open Heart ◽  
2020 ◽  
Vol 7 (1) ◽  
pp. e001184
Author(s):  
Nasrin Tayyari Dehbarez ◽  
Camilla Palmhøj Nielsen ◽  
Bettine Wulff Risør ◽  
Claus Vinther Nielsen ◽  
Vibeke Lynggaard

ObjectivesTo enhance adherence to cardiac rehabilitation (CR), a patient education programme called ‘learning and coping’ (LC-programme) was implemented in three hospitals in Denmark. The aim of this study was to investigate the cost–utility of the LC-programme compared with the standard CR-programme.Methods825 patients with ischaemic heart disease or heart failure were randomised to the LC-programme or the standard CR-programme and were followed for 3 years.A societal cost perspective was applied and quality-adjusted life years (QALY) were based on SF-6D measurements. Multiple imputation technique was used to handle missing data on the SF-6D. The statistical analyses were based on means and bootstrapped SEs. Regression framework was employed to estimate the net benefit and to illustrate cost-effectiveness acceptability curves.ResultsNo statistically significant differences were found between the two programmes in total societal costs (4353 Euros; 95% CI –3828 to 12 533) or in QALY (–0.006; 95% CI –0.053 to 0.042). At a threshold of 40 000 Euros, the LC-programme was found to be cost-effective at 15% probability; however, for patients with heart failure, due to increased cost savings, the probability of cost-effectiveness increased to 91%.ConclusionsWhile the LC-programme did not appear to be cost-effective in CR, important heterogeneity was noted for subgroups of patients. The LC-programme was demonstrated to increase adherence to the rehabilitation programme and to be cost-effective among patients with heart failure. However, further research is needed to study the dynamic value of heterogeneity due to the small sample size in this subgroup.


2012 ◽  
Vol 30 (34_suppl) ◽  
pp. 116-116
Author(s):  
Sarmad Sadeghi ◽  
Afsaneh Barzi ◽  
Michael W. Kattan ◽  
Neal J. Meropol

116 Background: LS diagnosis (Dx) in CRC patients (probands or Pds) and their first degree relatives (FDRs) impacts the management, outcomes and quality of care. Lack of a uniform approach to screening in academic and community centers is an impediment. Recent studies advocate universal immunohistochemistry (IHC) testing for Pds; however, these analyses exclude clinical criteria e.g., Amsterdam (Ams), Bethesda (Beth), and PMs, e.g., MMRpro (Mpro), MMRpredict (Mpre), and PREMM (PRE) due to concerns for reliability. This comprehensive comparison of all LS screening strategies (STs) aims to identify a cost effective process measure that addresses this need. Methods: We performed a cost effectiveness analysis with a societal viewpoint using TreeAge software. 21 STs for Pd and general population (GP) screening in a population of 100,000 were examined assuming a 3% LS prevalence (Prev) in Pd and 0.23% in GP, 5 FDRs per LS (Dx), 50% LS Prev in FDRs, and 90% germline testing (GT) compliance in Pds and GP and 52% in FDRs. Sensitivity, life years gained (LYG), and incremental cost effectiveness ratios (ICERs) were calculated. Results: See table. Conclusions: This study suggests that Mpro is a cost-effective first step in screening for LS in Pds, and its routine use may be considered as a possible process measure for quality of care in CRC patients. Up-front IHC +/- BRAF, GT could be reserved for Pds where history is incomplete. [Table: see text]


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