scholarly journals Cost-Effectiveness of Pazopanib in Advanced Soft Tissue Sarcoma in the United Kingdom

Sarcoma ◽  
2014 ◽  
Vol 2014 ◽  
pp. 1-14 ◽  
Author(s):  
Jordan Amdahl ◽  
Stephanie C. Manson ◽  
Robert Isbell ◽  
Ayman Chit ◽  
Jose Diaz ◽  
...  

In the phase III PALETTE trial, pazopanib improved progression-free survival (PFS) compared with placebo in patients with advanced/metastatic soft tissue sarcomas (mSTS) who had received prior chemotherapy. We used a multistate model to estimate expected PFS, overall survival (OS), lifetime STS treatment costs, and quality-adjusted life-years (QALYs) for patients receiving pazopanib, placebo, trabectedin, ifosfamide, or gemcitabine plus docetaxel as second-line mSTS therapies. The cost-effectiveness of pazopanib was expressed as the incremental costs per QALY gained. Estimates of PFS/OS, adverse events, and utilities for pazopanib and placebo were from the PALETTE trial. Estimates of relative effectiveness of the other comparators were from an unadjusted indirect comparison versus pazopanib. Costs were from published sources. Pazopanib is estimated to increase QALYs by 0.128 and costs by£7,976 versus placebo; cost per QALY gained with pazopanib versus placebo is estimated to be£62,000. Compared with the other chemotherapies, pazopanib provides similar QALYs at a lower cost. Pazopanib may not be cost-effective versus placebo but may be cost-effective versus the most commonly used active treatments, although this conclusion is uncertain. Given the unmet need for effective treatments for mSTS, pazopanib may be an appropriate alternative to some currently used medications in the United Kingdom.

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]


2008 ◽  
Vol 24 (02) ◽  
pp. 203-211 ◽  
Author(s):  
Rodolfo A. Hernández ◽  
Jennifer M. Burr ◽  
Luke D. Vale

Objectives:The aim of this study was to assess the cost-effectiveness of screening for open-angle glaucoma (OAG) in the United Kingdom, given that OAG is an important cause of blindness worldwide.Methods:A Markov model was developed to estimate lifetime costs and benefits of a cohort of patients facing, alternatively, screening or current opportunistic case finding strategies. Strategies, varying in how screening would be organized (e.g., invitation for assessment by a glaucoma-trained optometrist [GO] or for simple test assessment by a technician) were developed, and allowed for the progression of OAG and treatment effects. Data inputs were obtained from systematic reviews. Deterministic and probabilistic sensitivity analyses were performed.Results:Screening was more likely to be cost-effective as prevalence increased, for 40 year olds compared with 60 or 75 year olds, when the re-screening interval was greater (10 years), and for the technician strategy compared with the GO strategy. For each age cohort and at prevalence levels of ≤1 percent, the likelihood that either screening strategy would be more cost-effective than current practice was small. For those 40 years of age, “technician screening” compared with current practice has an incremental cost-effectiveness ratio (ICER) that society might be willing to pay when prevalence is 6 percent to 10 percent and at over 10 percent for 60 year olds. In the United Kingdom, the age specific prevalence of OAG is much lower. Screening by GO, at any age or prevalence level, was not associated with an ICER < £30,000.Conclusions:Population screening for OAG is unlikely to be cost-effective but could be for specific subgroups at higher risk.


2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 5106-5106
Author(s):  
M. F. Botteman ◽  
S. Kaura

5106 Background: Zoledronic acid (ZOL) significantly reduces the risk of new skeletal-related events (SREs) in patients (pts) with bone metastases from RCC. This study assessed and compared the cost-effectiveness of ZOL in pts with RCC from French, German, and United Kingdom (UK) societal perspectives. Methods: This analysis was based on a retrospective analysis of RCC pts with bone metastases who were enrolled in a 9-mo trial of ZOL or placebo (PBO) plus concomitant antineoplastic therapy. A model was developed to simulate costs and quality-adjusted life-years (QALYs) experienced by study pts. The model included data and assumptions regarding SRE incidence, mortality, drug and administration costs, SRE costs, reduced quality of life (QOL) because of SREs and bone pain, and therapy duration. SRE costs were estimated using diagnosis-related group tariff information and published literature. Consistent with similar economic analyses, it was assumed that QOL decreased 20% to 80% (depending on SRE type) for 1 mo after each SRE experienced. Sensitivity analyses were performed to test the effects of alternate assumptions, with < 30,000/QALY considered cost-effective. Results: Compared with PBO-treated pts (n = 19), ZOL-treated pts (n = 27) experienced 1.07 fewer SREs/pt and gained discounted QALYs of approximately 0.1563 in France and Germany and 0.1575 in the UK. Discounted SRE-related costs were substantially lower among pts treated with ZOL vs PBO (-4,196 in France, -3,880 in Germany, and -3,355 in the UK). After including drug therapy costs, ZOL saved 1,358, 1,223, and 719 per pt in France, Germany, and the UK, respectively. In multivariate sensitivity analyses, ZOL saved costs in 67% to 77% of cases, depending on the country. ZOL resulted in a cost per QALY gained < 30,000 in approximately 93% of cases. Conclusions: Treatment with ZOL reduces SREs, improves QOL, and lowers health-related costs compared with PBO in French, German, and UK pts with bone metastases from RCC. Use of ZOL in these populations therefore provides health-related cost savings and is a cost-effective use of healthcare resources. [Table: see text]


2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 6625-6625
Author(s):  
Naomi RM Schwartz ◽  
Meghan Rose Flanagan ◽  
Joseph B Babigumira ◽  
Lotte Maria Gertruda Steuten ◽  
Joshua A. Roth

6625 Background: Neratinib after adjuvant trastuzumab significantly improves disease-free survival (DFS) in human epidermal growth factor receptor 2-postiive (HER2+) breast cancer, but the median absolute DFS gain is only 1.3 months. There has been much controversy in the clinical and lay media as to whether the substantial cost of neratinib is justified by its effects, including a prominent ASCO Post article from Dr. Vogl about a year ago. We performed a cost-utility analysis to formally assess the value of adding neratinib based on Phase III ExteNET trial results. Methods: We developed a Markov state-transition model to assess the value of neratinib in Stage I-III HER2+ breast cancer. Five-year recurrence rates were derived from ExteNet. Mortality and recurrence rates after 5 years were derived from the HERceptin Adjuvant (HERA) trial. Costs were derived from wholesale acquisition costs and peer-reviewed literature. Health state utilities were derived from ExteNET and prior publications. Outcomes included life years (LY), quality-adjusted life years (QALYs), direct medical expenditures, and cost per QALY gained. The analysis took a payer perspective over a lifetime horizon and results were discounted at 3% per year. One-way and probabilistic analyses were conducted to evaluate uncertainty. As neratinib conferred more clinical benefit in hormone receptor-positive (HR+) disease, we also assessed value in that specific subgroup. Results: Base case results are presented in Table. At typical U.S. willingness to pay thresholds of $100,000 and $150,000 per QALY gained, neratinib had 16.7% and 27.2%, probabilities of cost-effectiveness, respectively. In the HR+ subgroup, neratinib had a cost of $275,311 per QALY gained (19.9% & 31.2% probability of cost-effectiveness at $100,000 & $150,000 per QALY). Conclusions: In the first independent assessment of the value of neratinib after adjuvant trastuzumab, neratinib is not projected to be cost-effective, even among patients who derived the most clinical benefit. Future analyses should reassess the cost-effectiveness of neratinib treatment as trial data mature. Base case results. [Table: see text]


2018 ◽  
Vol 36 (6_suppl) ◽  
pp. 43-43
Author(s):  
Rahul Ramesh Khairnar ◽  
Joseph Levy ◽  
Mark Mishra

43 Background: A hydrogel rectal spacer (HRS) is an FDA-approved medical device used to increase the separation between the rectum and the prostate. A recent phase III trial demonstrated a small reduction in the incidence of RT toxicities associated with use of HRS. We conducted a cost-effectiveness analysis of HRS use in PC patients undergoing intensity modulated radiation therapy (IMRT). Methods: A multi-state Markov model was constructed to examine the cost-effectiveness of HRS in men with localized PC receiving IMRT in the US (arms: IMRT alone vs. IMRT + HRS). Subgroups included delivery site of IMRT (hospital vs. ambulatory) and baseline sexual function (SF) (general population vs. those with good SF). Based on previous studies, recurrence and survival were assumed equal for both arms. Data on SF, gastrointestinal and genitourinary toxicities incidence, as well as potential risks associated with HRS implantation were obtained from a recently published clinical trial. Health utilities and costs were derived from the literature and 2018 Physician Fee Schedule. Quality-adjusted life years (QALYs) and costs were modeled for a 5-year period from receipt of RT. Probabilistic sensitivity analysis (PSA) and value-based threshold analysis were conducted. Costs and utilities were discounted at 3% annually. Results: The per-person 5-year incremental cost for HRS administered in a hospital was $4,008 and the incremental effectiveness was 0.0273 QALYs. The incremental cost-effectiveness ratio (ICER) was $146,746 (95% credible interval from PSA $125,638 – $178,049) for PC patients undergoing HRS insertion in a hospital vs. $73,359 ($66,732 – $86,767) for patients undergoing HRS insertion in an ambulatory facility. For men with good SF, the ICER was $55,153 ($46,002 – $76,090) and $26,542 ($17,399 – $46,044) in hospital vs. ambulatory facility. Conclusions: This study is the first to evaluate the cost-effectiveness of HRS based on long-term toxicity data. Based on the current Medicare Physician Fee Schedule, HRS is cost-effective in men with good SF at a willingness to pay threshold of $100,000 and it is marginally cost-effective for the entire population depending on the facility where the HRS is inserted.


Author(s):  
Mafalda Ramos ◽  
Anastasia Ustyugova ◽  
Nikco Hau ◽  
Mark Lamotte

Aim: Cost–effectiveness (CE) analysis of empagliflozin+standard of care (SoC) compared with SoC and liraglutide+SoC, in patients with Type II diabetes and established cardiovascular disease, was conducted using evidence from cardiovascular outcomes trials. Methods: The IQVIA Core Diabetes Model was calibrated to predict same outcomes observed in EMPA-REG OUTCOME and LEADER trials. Three-year observed cardiovascular events of SoC, empagliflozin+SoC and liraglutide+SoC were derived from EMPA-REG OUTCOME trial and an indirect comparison. Time horizon was 50 years and the UK payer perspective was taken. Results: Empagliflozin+SoC dominated liraglutide+SoC with greater quality-adjusted life years and reduced costs. Base-case incremental CE ratio of 6428 GBP/QALY was observed for empagliflozin+SoC versus SoC. Conclusion: Results suggest that empagliflozin+SoC is cost effective versus SoC and liraglutide+SoC.


2015 ◽  
Vol 33 (26) ◽  
pp. 2885-2892 ◽  
Author(s):  
Isabelle Borget ◽  
Julia Bonastre ◽  
Bogdan Catargi ◽  
Désirée Déandréis ◽  
Slimane Zerdoud ◽  
...  

Purpose In the ESTIMABL phase III trial, the thyroid ablation rate was equivalent for the two thyroid-stimulating hormone (TSH) stimulation methods (thyroid hormone withdrawal [THW] and recombinant human TSH [rhTSH]) and the two iodine-131 (131I) activities (1.1 or 3.7 GBq). The objectives of this article were to present health-related quality-of-life (HRQoL) results and a cost-effectiveness evaluation performed alongside this trial. Patients and Methods HRQoL and utility were longitudinally assessed, from random assignment to the follow-up visit at 8 ± 2 months for the 752 patients with thyroid cancer, using the Short Form-36 and the EuroQoL-5D questionnaires, respectively. A cost-effectiveness analysis was performed from the societal perspective in the French context. Resource use (hospitalization for 131I administration, rhTSH, sick leaves, and transportation) was collected prospectively. We used the net monetary benefit approach and computed cost-effectiveness acceptability curves for both TSH stimulation methods and 131I activities. Sensitivity analyses of the costs of rhTSH were performed. Results At 131I administration, THW caused a clinically significant deterioration of HRQoL, whereas HRQoL remained stable with rhTSH. This deterioration was transient with no difference 3 months later. rhTSH was more effective than THW in terms of quality-adjusted life-years (QALYs; +0.013 QALY/patient) but more expensive (+€474/patient). The probability that rhTSH would be cost effective at a €50,000/QALY threshold was 47% in France. The use of 1.1 GBq of 131I instead of 3.7 GBq reduced per-patient costs by €955 (US$1,018) but with slightly decreased efficacy (−0.007 QALY/patient). Conclusion rhTSH avoids the transient THW-induced deterioration of HRQoL but is unlikely to be cost effective at its current price.


2019 ◽  
Vol 6 (Supplement_2) ◽  
pp. S749-S750
Author(s):  
Jaesh Naik ◽  
Joe Yang ◽  
David Elsea ◽  
Simone Critchlow ◽  
Laura Puzniak

Abstract Background Ventilated, hospital-acquired and ventilator-associated bacterial pneumonia (vHABP/VABP) are associated with high rates of antibiotic resistance and high morbidity and mortality in hospitalized patients. Ceftolozane/tazobactam (C/T) has shown non-inferiority to meropenem for treating HABP/VABP in a Phase III trial, ASPECT-NP. This study evaluates cost-effectiveness of C/T against meropenem in treating HABP/VABP. Methods We developed a model consisting of a short-term decision tree (reflecting the in-hospital period) followed by a long-term Markov structure (capturing lifetime costs and outcomes). Patient characteristics and clinical efficacy were informed by subjects in ASPECT-NP who received any dose of study drugs. Susceptibility was based on the Program to Assess C/T Susceptibility surveillance database. Second-line and salvage treatment were added to resemble real-world treatment patterns and used to calculate overall clinical cure and mortality rates based on results from a network meta-analysis. We analyzed two clinical scenarios: (1)”confirmed treatment’ in which C/T or meropenem is used after pathogen susceptibility is known; (2) ‘initial treatment’ of high-risk patients before susceptibility is known. Model outcomes include, percentage clinically cured, short-term mortality, direct medical costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios. Sensitivity analyses (SAs) were conducted to test the robustness of results. Results In the confirmed treatment setting, C/T had a higher cure rate (5.0 percentage points, the same below), lower short-term mortality (−5.1%), cost more ($2,728), and yielded higher lifetime QALYs (0.61) than meropenem ($4,472/QALY gained). In the initial treatment setting, C/T sustained a better clinical performance (9.5% more cure, −6.8% mortality, 1.16 more QALYs), yet cost less than meropenem (−$5,662) due to better susceptibility. The response and mortality rates from ASPECT-NP had the greatest impact on results. SAs showed that the result of C/T being cost-effective over meropenem was generally robust. Conclusion The results indicate that, compared with meropenem, C/T could be a cost-effective option for patients with vHABP/VABP in the US setting. Disclosures All authors: No reported disclosures.


Sarcoma ◽  
2018 ◽  
Vol 2018 ◽  
pp. 1-14 ◽  
Author(s):  
Santiago Zuluaga-Sanchez ◽  
Lisa M. Hess ◽  
Sorrel E. Wolowacz ◽  
Yulia D’yachkova ◽  
Emma Hawe ◽  
...  

Background. Standard first-line treatments for advanced soft tissue sarcoma (STS) have changed little for 40 years, and outcomes have been poor. Recently, the United States (US) Food and Drug Administration conditionally approved olaratumab in combination with doxorubicin (Olara + Dox) based on a randomized phase II trial that reported a significant 11.8-month improvement in median survival versus single-agent doxorubicin (Dox). The present study investigated the cost-effectiveness of Olara + Dox compared with Dox and five other standard-of-care regimens from the US payer perspective. Methods. An economic model was constructed to estimate costs and outcomes over patients’ lifetimes from start of therapy. Progression-free and overall survival were based on survival analysis of patient-level data and a meta-analysis. Adverse-event rates were based on trials. Costs were from published sources. Results. Olara + Dox resulted in an estimated additional 1.27 life-years (LYs) compared with Dox, with an increase in total expected lifetime costs of $133,653. The incremental cost-effectiveness ratio (ICER) was estimated at $105,408 per LY gained; in a fully incremental analysis, all other regimens were dominated (higher costs and lower LYs or a higher ICER). Conclusion. Olara + Dox is cost-effective for STS treatment compared with Dox and other standard-of-care regimens at willingness-to-pay thresholds of $150,000 per LY and above.


Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2366-2366
Author(s):  
Gabriel Tremblay ◽  
Anna Forsythe ◽  
Vasudha Bal ◽  
Snigdha Santra ◽  
Andrew Briggs

Abstract Background In a Phase III COMPLEMENT 2 study,ofatumumab(OFA) plusfludarabine(F) and cyclophosphamide (C) demonstrated significantly improved median progression-free survival (PFS) by 54% compared to FC treatment alone (HR=0.67, p=0.0032) in patients with relapsed chronic lymphocytic leukemia (rCLL). However, the relative value of OFA in rCLL has not been formally assessed. The objective of this study was to estimate the incremental cost per (quality-adjusted) life-year of utilizing OFA+FC vs. FC for rCLL in the US. Methods A partition survival model was developed to estimate the expected outcomes and costs of treatment of OFA+FC vs FC forrCLLover a lifetime horizon. The model includes 4 health states: PFS on treatment, PFS off-treatment, post-progression and death. Time during PFS following protocol-defined treatment duration of 6 months, was considered a treatment-free period in the model. Data on PFS, OS and frequencies of adverse events (AEs)were obtained from the Phase III clinical trial for OFA (COMPLEMENT 2). For the extrapolation of OS and PFS a piecewise approach was used, where the efficacy was based on the patient-level data (Kaplan-Meier Survivor Function) until the trial cut-off and a tail extrapolation thereafter (gamma distribution). Health state utilities and dis-utilities for AEs were obtained from previously published vignette studies. Costs incorporated in the model included drug and administration for primary and follow-up therapies, adverse event treatments, medical costs for hospitalizations and physician visits; and end of life costs. The costs were derived from databases (AnalySourceOnline, AHRQ, CMS). Results Treatment with OFA+FC led to an increase of 0.803 life years and 0.543 quality-adjusted life years (QALYs) relative to FC. The total cost of OFA+FC was higher by $6,693 per patient relative to FC. Although addition of AFA to FC lead to higher drug and adverse event costs, these were partially offset by lower follow-up costs compared to FC. The ICER per LY and per QALY gained with OFA+FC vs. FC was $8,333 and $12,322, respectively. Based on probabilistic sensitivity analyses, there wasa85% probability that OFA+FC was cost-effective compared to FC at a societal willingness-to-pay threshold of $100,000 per QALY saved. Conclusions Our analysis suggests treatment with OFA+FC compared to FC is highly cost-effective based Phase 3 within-trial analysis. These results are driven by the improved PFS and OS of OFA+FC vs. FC, as well as the treatment-free period, during which patients experienced PFS without the burden of treatment AEs or costs. Future direct comparisons of OFA+FC versus other treatment options will further clarify the cost-effectiveness of OFA+FC to inform coverage and reimbursement policy decisions. Disclosures Tremblay: Novartis Pharmaceuticals Corporation: Consultancy. Forsythe:Novartis Pharmaceuticals Corporation: Consultancy. Bal:Novartis Pharmaceuticals: Employment. Santra:Novartis Pharmaceuticals Corporation: Employment. Briggs:Novartis Pharmaceuticals Corporation: Consultancy.


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