Cost-effectiveness analysis of platinum-based chemotherapy treatment options for germline BRCA-mutated locally advanced/borderline resectable pancreatic cancer.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e16246-e16246
Author(s):  
Myles Ingram ◽  
Yoanna S Pumpalova ◽  
Jiheum Park ◽  
Francesca Lim ◽  
Gulam Abbas Manji ◽  
...  

e16246 Background: Patients with germline BRCA1/2 mutations (gBRCAm) have an increased risk for pancreatic ductal adenocarcinoma (PDAC). The NCCN 2020 guidelines recommend platinum-based chemotherapy (FOLFIRINOX or gemcitabine plus cisplatin) in patients with gBRCAm diagnosed with borderline resectable or locally advanced (BR/LA) PDAC; for patients without progression on chemotherapy, maintenance therapy with a PAPR inhibitor (PARPi) can be considered. FOLFIRINOX and gemcitabine plus cisplatin (GemCis), with or without maintenance PARPi, have not been directly compared in this patient population. The purpose of our study was to compare treatment outcomes, toxicity, costs, and quality-of-life of the two recommended platinum-based regimens, with or without maintenance PARPi (olaparib or veliparib), in patients with gBRCAm and BR/LA PDAC. Methods: We developed a decision-analytic mathematical model comparing the total cost and health outcomes of FOLFIRINOX, FOLFIRINOX + olaparib (FOLFIRNOX-O), GemCis, and GemCis + veliparib (GCV) over twelve years. The inputs for the model were estimated using clinical trial data and published literature. Natural history was used as a comparator. The primary endpoint was incremental cost-effectiveness ratios (ICERs) with a willingness-to-pay (WTP) threshold of $100,000 per quality-adjusted-life-year (QALY). Secondary endpoints included overall survival (OS), progression-free survival (PFS), life-years (LYs) and total cost of care (USD). Results: Both FOLFIRINOX strategies were dominated by the GemCis strategies and thus eliminated from the efficiency frontier. GCV was the strategy that yielded the most life-years (2.05 LYs) but the ICER of $629,697.46 for this regimen far exceeded the WTP threshold. Therefore, GemCis was the cost-effective strategy, totaling incremental QALYs of 1.31 at a cost of $61,228.40 per QALY when compared to natural history. A one-way sensitivity analysis found that the GCV becomes the cost-effective strategy when the cost of maintenance PARPi is lowered to 4% of the base case. Conclusions: Our model found that GemCis is the cost-effective option for patients with gBRCAm and BR/LA PDAC. Neither of the strategies with maintenance PARPi were cost-effective. Additional clinical trial data with adequate follow-up are needed to confirm our findings.[Table: see text]

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e16793-e16793
Author(s):  
Myles Ingram ◽  
Brianna N Lauren ◽  
Yoanna S Pumpalova ◽  
Gulam Abbas Manji ◽  
Susan Elaine Bates ◽  
...  

e16793 Background: The 2019 NCCN guidelines recommend neoadjuvant FOLFIRINOX or neoadjuvant gemcitabine plus nab-paclitaxel (G-nP) for locally advanced and borderline resectable pancreatic ductal adenocarcinoma (BR/LA PDAC). Neoadjuvant FOLFIRINOX and G-nP have yet to be directly compared in a prospective, randomized trial with BR/LA PDAC patients. The purpose of our study was to incorporate treatment outcomes, toxicity profiles, costs, and quality-of-life measures to further inform clinical decision-making. Methods: We developed a decision-analytic mathematical model to compare the total cost and health outcomes of neoadjuvant FOLFIRINOX against G-nP over twelve years. Adjuvant gemcitabine (GEM) was used as a comparator. The inputs for the model were estimated using clinical trial data and published literature. We used single-institution retrospective studies to estimate our survival data in the absence of a prospective trial. The primary endpoint was incremental cost-effectiveness ratios (ICERs) with a willingness-to-pay (WTP) threshold of $100,000 per quality-adjusted-life-year (QALY). Secondary endpoints included overall (OS) and progression-free survival (PFS), total cost of care (USD), QALYs, patient resection rate, and monthly treatment-related adverse events (TRAE) costs (USD). Results: FOLFIRINOX was the cost-effective strategy, totaling incremental QALYs of 0.21 at a cost of $52,845.96 per QALY when compared to G-nP. G-nP was also on the efficiency frontier with an ICER of $46,430.73 compared to GEM. More patients received resection with FOLFIRINOX (82.15% vs. 72.40%), but had higher TRAE costs than G-nP ($12,051.26 vs. $4,666.97). A one-way sensitivity analysis found that the FOLFIRINOX ICER exceeds the WTP threshold when TRAE costs are higher and resection rates are lower. Conclusions: Our modeling analysis finds FOLFIRNOX is the cost-effective treatment compared to G-nP for BR/LA PDAC despite having a higher cost of total care due to TRAE costs. Trial data with sufficient follow-up are needed to confirm our findings. [Table: see text]


Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1324-1324 ◽  
Author(s):  
Carolina Reyes ◽  
Gregory Gazauskas ◽  
Ursula Becker ◽  
Santiago Moreno ◽  
David L. Veenstra

Abstract Background Two recently approved therapies for previously untreated chronic lymphocytic leukemia (CLL), (1) obinutuzumab (GA101) in combination with chlorambucil (G+Clb) and (2) ofatumumab in combination with Clb (O+Clb), have shown improved progression-free survival (PFS) versus Clb alone in two separate trials. However, their relative value has not been formally assessed. The objective of this study was to compare the cost-effectiveness of G+Clb versus O+Clb in previously untreated CLL patients, as well as conduct exploratory analyses versus other comparators. Methods Patient outcomes were simulated using a 3-state Markov model that included PFS, progression, and death. PFS parameters for G+Clb were fitted to the observed G+Clb trial (CLL-11 study) data, and a network meta-analysis incorporating the results of the O+Clb (COMPLEMENT 1 study) was used to estimate the relative treatment effect of G+Clb compared to O+Clb (progression HR = 0.34). Patient populations in these two trials were similar. Drug utilization, dosing and adverse events were incorporated based on trial data, and costs were based on Medicare reimbursements and drug wholesale acquisition costs. One-way and probabilistic sensitivity analyses were conducted to assess the impact of data uncertainty on the results. In an exploratory scenario analyses, we used similar methodology to estimate the cost-effectiveness of G+Clb versus rituximab plus bendamustine (R+B, progression HR = 0.41). A meta-regression on age was used to adjust for the age difference among the patient populations and indirectly taking into account different levels of comorbidities. Results Treatment with G+Clb led to an increase of 0.83 life years and 0.79 quality-adjusted life years (QALYs) relative to O+Clb. The total cost of O+Clb was higher by $3600 per patient relative to G+Clb. Higher G+Clb drug, administration, and adverse event costs were largely offset by lower progression costs compared to O+Clb. The incremental cost per QALY gained with G+Clb vs. O+Clb was $4,500. Based on probabilistic sensitivity analyses, there was a 99% probability that G+Clb was cost-effective compared to O+Clb at a societal willingness-to-pay threshold of $100,000 per QALY saved. Table.OutcomeG+ClbO+ClbDifferenceAverage life years5.744.910.83Average QALYs3.953.160.79Total drug cost$37,192$34,260$2,932Drug administration$1,977$991$985Supportive care$141$73$68Adverse events$9,542$2,660$6,882Cost of progression$46,861$54,147$(7,286)Average total cost$95,713 $92,132 $3,581 In the exploratory scenario analysis, G+Clb was $37,700 less expensive than R+B, and led to an increase of 0.67 QALY. Conclusions Our analysis suggests treatment with G+Clb compared to O+Clb is highly cost-effective based on indirect treatment comparison data. These results are driven by the improved PFS of G+Clb vs. O+Clb, as well as lower disease progression cost. Future direct comparisons of G+Clb versus other treatment options will further clarify the cost-effectiveness of G+Clb, and inform coverage and reimbursement policy decisions. Disclosures Reyes: Genentech: Employment, Equity Ownership. Off Label Use: Rituximab + Bendamustine in CLL. Gazauskas:Genentech: Consultancy. Becker:Roche: Employment. Moreno:Roche: Employment. Veenstra:Roche: Consultancy.


2004 ◽  
Vol 20 (4) ◽  
pp. 469-474 ◽  
Author(s):  
Tanja Tomson ◽  
Ásgeir R. Helgason ◽  
Hans Gilljam

Objectives: The cost-effectiveness of the Swedish quitline, a nation-wide, free of charge service, is assessed.Methods: The study was based on data of a sample of 1,131 callers enrolled from February 1, 2000 to November 30, 2001. Outcome was measured as cost per quitter and cost per year of life saved. Cost per quitter was based on a calculation of the total cost of the quitline divided by the number of individuals who reported abstinence after 12 months. The cost per life year saved (LYS) was calculated by the use of data from the literature on average life expectancy for smokers versus quitters, the total cost of the quitline, and the cost of pharmacological treatment.Results: The number of smokers who used the quitline and reported abstinence after 1 year was 354 (31 percent). The accumulated number of life years saved in the study population was 2,400. The cost per quitter was 1,052–1,360 USD, and the cost per life year saved was 311–401 USD. A sensitivity analysis showed that, for outcomes down to an abstinence rate of 20 percent, the cost per LYS rose modestly, from 311 to 482 USD. Discounting the cost per LYS showed the cost to be 135 USD for 3 percent and 283 USD for 5 percent.Conclusions: The Swedish quitline is a cost-effective public health intervention compared with other smoking cessation interventions.


Vaccines ◽  
2020 ◽  
Vol 8 (4) ◽  
pp. 707
Author(s):  
Afifah Machlaurin ◽  
Franklin Christiaan Karel Dolk ◽  
Didik Setiawan ◽  
Tjipke Sytse van der Werf ◽  
Maarten J. Postma

Bacillus Calmette–Guerin (BCG), the only available vaccine for tuberculosis (TB), has been applied for decades. The Indonesian government recently introduced a national TB disease control programme that includes several action plans, notably enhanced vaccination coverage, which can be strengthened through underpinning its favourable cost-effectiveness. We designed a Markov model to assess the cost-effectiveness of Indonesia’s current BCG vaccination programme. Incremental cost-effectiveness ratios (ICERs) were evaluated from the perspectives of both society and healthcare. The robustness of the analysis was confirmed through univariate and probabilistic sensitivity analysis (PSA). Using epidemiological data compiled for Indonesia, BCG vaccination at a price US$14 was estimated to be a cost-effective strategy in controlling TB disease. From societal and healthcare perspectives, ICERs were US$104 and US$112 per quality-adjusted life years (QALYs), respectively. The results were robust for variations of most variables in the univariate analysis. Notably, the vaccine’s effectiveness regarding disease protection, vaccination costs, and case detection rates were key drivers for cost-effectiveness. The PSA results indicated that vaccination was cost-effective even at US$175 threshold in 95% of cases, approximating the monthly GDP per capita. Our findings suggest that this strategy was highly cost-effective and merits prioritization and extension within the national TB programme. Our results may be relevant for other high endemic low- and middle-income countries.


2021 ◽  
pp. 019459982110268
Author(s):  
Joseph R. Acevedo ◽  
Ashley C. Hsu ◽  
Jeffrey C. Yu ◽  
Dale H. Rice ◽  
Daniel I. Kwon ◽  
...  

Objective To compare the cost-effectiveness of sialendoscopy with gland excision for the management of submandibular gland sialolithiasis. Study Design Cost-effectiveness analysis. Setting Outpatient surgery centers. Methods A Markov decision model compared the cost-effectiveness of sialendoscopy versus gland excision for managing submandibular gland sialolithiasis. Surgical outcome probabilities were found in the primary literature. The quality of life of patients was represented by health utilities, and costs were estimated from a third-party payer’s perspective. The effectiveness of each intervention was measured in quality-adjusted life-years (QALYs). The incremental costs and effectiveness of each intervention were compared, and a willingness-to-pay ratio of $150,000 per QALY was considered cost-effective. One-way, multivariate, and probabilistic sensitivity analyses were performed to challenge model conclusions. Results Over 10 years, sialendoscopy yielded 9.00 QALYs at an average cost of $8306, while gland excision produced 8.94 QALYs at an average cost of $6103. The ICER for sialendoscopy was $36,717 per QALY gained, making sialendoscopy cost-effective by our best estimates. The model was sensitive to the probability of success and the cost of sialendoscopy. Sialendoscopy must meet a probability-of-success threshold of 0.61 (61%) and cost ≤$11,996 to remain cost-effective. A Monte Carlo simulation revealed sialendoscopy to be cost-effective 60% of the time. Conclusion Sialendoscopy appears to be a cost-effective management strategy for sialolithiasis of the submandibular gland when certain thresholds are maintained. Further studies elucidating the clinical factors that determine successful sialendoscopy may be aided by these thresholds as well as future comparisons of novel technology.


Author(s):  
Nayyereh Ayati ◽  
Lora Fleifel ◽  
Mohammad Ali Sahraian ◽  
Shekoufeh Nikfar

Background: Cladribine tablets are the foremost oral immune-reconstitution therapy for high disease activity relapsing multiple sclerosis (HDA-RMS). We aimed to assess the cost-effectiveness of cladribine tablets compared to natalizumab in patients with HDA-RMS in Iran. Methods: A 5-year cohort-based Markov model was developed with 11 expanded disability status score (EDSS) health states, including patients with HDA-RMS as on and off-treatment. All costs were identified from the literature and expert opinion and were measured in Iranian Rial rates, changed to the 2020 USD rate and were discounted by 7.2%. Quality adjusted life years (QALY), discounted by 3.5%, and life years gained (LYG) were adopted to measure efficacy. The final results were presented as incremental cost-effectiveness ratio that was compared to a national willingness to pay (WTP) threshold of 1 to 3 gross domestic product (GDP) per capita. Deterministic and probabilistic sensitivity analyses (D/PSA) were employed to evaluate uncertainty. Results: Cladribine tablets dominated natalizumab and yielded 6,607 USD cost-saving and 0.003 additional QALYs per patient. LYG was comparable. The main cost component was drug acquisition cost in both arms. DSA indicated the sensitivity of the results to the cost discount rates and also the patients’ body weight; while they were less sensitive to the main clinical variables. PSA indicated that cladribine tablets were cost-effective in Iran, with a probability of 57.5% and 58.6% at lower and higher limits of threshold, respectively. Conclusion: Cladribine tablets yielded higher QALYs and lower costs compared to natalizumab, in patients with HDA-RMS in Iran.


Health Scope ◽  
2021 ◽  
Vol 10 (2) ◽  
Author(s):  
Maryam Khoramrooz ◽  
Asra Asgharzadeh ◽  
Saeide Alidoost ◽  
Zeynab Foroughi ◽  
Saber Azami ◽  
...  

Context: Stroke is one of the main causes of premature death and disability, imposing significant costs on the healthcare system, especially due to expensive hospital care. Home care service is one of the interventions used in the last two decades to reduce the cost of services provided for stroke patients in different countries. Objectives: The present study aimed to systematically review studies related to the economic evaluation of home care compared to hospital care for stroke patients. Data Sources: A search was conducted between January 1990 and January 2021. PubMed, Scopus, Web of Science, and Embase databases were searched systematically. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines were used to select the studies. Data Extraction: To evaluate the quality of studies included in this systematic review, Drummond’s ten-item checklist was used. Results: Five economic evaluation studies were included in this review. The included studies reported different results regarding the effect of home care on improving different indicators and the cost-effectiveness ratio of home care to hospital care. Most previous studies reported that home care is a more cost-effective option for improving many indicators, such as physical function and quality-adjusted life years (QALY), and for reducing mortality and institutionalization, compared to hospital care. Conclusions: Home care is a more cost-effective option than hospital care for stroke patients with regard to some indicators, such as the Barthel index for Activities of Daily Living, Modified Rankin Scale (mRS), quality of life, mortality, and institutionalization. However, there are some exemptions to this conclusion. Due to limitations, such as heterogeneity of interventions in the existing studies, different levels of patients’ disabilities, different perspectives toward economic evaluation, and differences in the healthcare systems of countries, further research is needed according to the context of each country based on clinical trials.


2014 ◽  
Vol 2 (1) ◽  
Author(s):  
Sixten Borg ◽  
Ingmar Näslund ◽  
Ulf Persson ◽  
Knut Ödegaard

Background:The rising trend in the prevalence of obesity has during the past decades become a major public health concern in many countries, as obesity may lead to comorbidities and death. A frequent used marker for obesity is the Body Mass Index (BMI). The cost of treatment for obesity related diseases has become a heavy burden on national health care budget in many countries. While diet and exercise are the cornerstones of weight management, pharmaco­therapy is often needed to achieve and maintain desired weight loss.  In some cases of extreme obesity, bariatric surgery may be recommended. It is expected to increase by 50% in Sweden.Objective: The overall objective was to develop a cost-effectiveness model using the best available evidence to assess the cost-effectiveness of gastric bypass (GBP) surgical treatments for obesity in adult patients, in comparison with conventional treatment (CT), in Sweden from a healthcare perspective. With the model we also seeked to identify the lower cut-off point using BMI criteria, for the surgical intervention to be cost-effective. Methods:A micro-simulation model with an underlying Markov methodology was developed, that simulates individual patients. It simulates the outcomes of the patients in terms of treatment costs, life years, and quality adjusted life years (QALY) over his/her remaining lifetime. The costs are presented in SEK in the year 2006 price level (1 SEK ≈ 0.11 EUR ≈ 0.14 USD).Results: We estimated that the incremental cost per QALY gained will not exceed SEK 33,000 per QALY in patients with BMI < 35. In patients with BMI > 35 kg/m2, gastric bypass surgery has lower costs compared to conventional treatment. Conclusion: Gastric bypass surgery is a cost-effective intervention compared to conventional treatment consisting of watchful waiting, diet and exercise.


2021 ◽  
Author(s):  
Y. Natalia Alfonso ◽  
Adnan A Hyder ◽  
Olakunle Alonge ◽  
Shumona Sharmin Salam ◽  
Kamran Baset ◽  
...  

Abstract Drowning is the leading cause of death among children 12-59 months old in rural Bangladesh. This study evaluated the cost-effectiveness of a large-scale crèche intervention in preventing child drowning. Estimates of the effectiveness of the crèches was based on prior studies and the program cost was assessed using monthly program expenditures captured prospectively throughout the study period from two different implementing agencies. The study evaluated the cost-effectiveness from both a program and societal perspective. Results showed that from the program perspective the annual operating cost of a crèche was $416.35 (95%C.I.: $222 to $576), the annual cost per child was $16 (95%C.I.: $9 to $22) and the incremental-cost-effectiveness ratio (ICER) per life saved with the crèches was $17,803 (95%C.I.: $9,051 to $27,625). From the societal perspective (including parents time valued) the ICER per life saved was -$176,62 (95%C.I.: -$347,091 to -$67,684)—meaning crèches generated net economic benefits per child enrolled. Based on the ICER per disability-adjusted-life years averted from the societal perspective (excluding parents time), $2,020, the crèche intervention was cost-effective even when the societal economic benefits were ignored. Based on the evidence, the creche intervention has great potential for reducing child drowning at a cost that is reasonable.


2018 ◽  
Vol 212 (4) ◽  
pp. 199-206 ◽  
Author(s):  
Stephanie Nobis ◽  
David Daniel Ebert ◽  
Dirk Lehr ◽  
Filip Smit ◽  
Claudia Buntrock ◽  
...  

BackgroundWeb-based interventions are effective in reducing depression. However, the evidence for the cost-effectiveness of these interventions is scarce.AimsThe aim is to assess the cost-effectiveness of a web-based intervention (GET.ON M.E.D.) for individuals with diabetes and comorbid depression compared with an active control group receiving web-based psychoeducation.MethodWe conducted a cost-effectiveness analysis with treatment response as the outcome and a cost-utility analysis with quality-adjusted life-years (QALYs) alongside a randomised controlled trial with 260 participants.ResultsAt a willingness-to-pay ceiling of €5000 for a treatment response, the intervention has a 97% probability of being regarded as cost-effective compared with the active control group. If society is willing to pay €14 000 for an additional QALY, the intervention has a 51% probability of being cost-effective.ConclusionsThis web-based intervention for individuals with diabetes and comorbid depression demonstrated a high probability of being cost-effective compared with an active control group.Declaration of interestS.N., D.D.E., D.L., M.B. and B.F. are stakeholders of the Institute for Online Health Trainings, which aims to transfer scientific knowledge related to this research into routine healthcare.


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