scholarly journals Cost-Effectiveness Analysis of Fourth- or Further-Line Ripretinib in Advanced Gastrointestinal Stromal Tumors

2021 ◽  
Vol 11 ◽  
Author(s):  
Weiting Liao ◽  
Huiqiong Xu ◽  
David Hutton ◽  
Qiuji Wu ◽  
Kexun Zhou ◽  
...  

BackgroundThe INVICTUS trial assessed the efficacy and safety of ripretinib compared with placebo in the management of advanced gastrointestinal stromal tumors.MethodWe used a Markov model with three health states: progression-free disease, progression disease and death. We parameterized the model from time-to-event data (progression-free survival, overall survival) of ripretinib and placebo arms in the INVICTUS trial and extrapolated to a patient’s lifetime horizon. Estimates of health state utilities and costs were based on clinical trial data and the published literature. The outcomes of this model were measured in quality-adjusted life-years (QALYs), costs, and incremental cost-effectiveness ratios (ICERs). Uncertainty was tested via univariate and probabilistic sensitivity analyses.ResultsThe base-case model projected improved outcomes (by 0.29 QALYs) and additional costs (by $70,251) and yielded an ICER of $244,010/QALY gained for ripretinib versus placebo. The results were most sensitive to progression rates, the price of ripretinib, and health state utilities. The ICER was most sensitive to overall survival. When overall survival in the placebo group was lower, the ICER dropped to $127,399/QALY. The ICER dropped to $150,000/QALY when the monthly cost of ripretinib decreased to $14,057. Probabilistic sensitivity analyses revealed that ripretinib was the cost-effective therapy in 41.1% of simulations at the willingness-to-pay (WTP) threshold of $150,000.ConclusionAs the fourth- or further-line therapy in advanced gastrointestinal stromal tumors, ripretinib is not cost-effective in the US. Ripretinib would achieve its cost-effectiveness with a price discount of 56% given the present effectiveness.

2012 ◽  
Vol 30 (4_suppl) ◽  
pp. 137-137
Author(s):  
Myrlene Sanon ◽  
Anju Parthan ◽  
Douglas Taylor ◽  
John Coombs ◽  
Marc Paolantonio ◽  
...  

137 Background: Recent clinical trial data have demonstrated that 3 years (yr) of adjuvant imatinib therapy for patients with surgically resected GIST leads to a significant improvement in recurrence free survival and overall survival vs. 1 yr of therapy. The objective of this study is to assess cost-effectiveness of treating with 3 yrs vs. 1 yr of adjuvant imatinib in the US from a payer’s perspective. Methods: A Markov model was developed to predict GIST recurrence, treatment (txt) costs, and quality-adjusted survival over a lifetime horizon. Patients transitioned between 3 health states: recurrence free GIST, GIST recurrence, and death. Monthly recurrence and mortality rates for 3 yr and 1 yr imatinib were derived from SSGXVIII/AIO clinical trial. The 5-yr recurrence rate observed in the trial was extrapolated for the remaining duration of the model horizon. First recurrence after active txt was treated with imatinib 400mg daily (800mg daily if recurrence during active txt). For subsequent disease progression, patients were treated with imatinib 800mg, sunitinib or best supportive care. After 5 years, txt specific mortality rate was applied for patients with recurrence. Costs and utilities were derived from published literature. Expected costs and quality-adjusted life years (QALYs) were estimated for each txt strategy. Costs and QALYs were discounted at 3% per yr. Extensive sensitivity analyses were conducted. Results: Total lifetime cost per patient was $302,100 with 3 yrs vs. $217,800 for 1 yr of imatinib therapy. Patients on 3 yrs of imatinib had higher QALYs (8.53 vs 7.18) vs. 1yr of imatinib. Incremental cost effectiveness ratio of 3 yrs of imatinib vs 1 yr of imatinib was $62,600/QALY. Model results were sensitive to rate of GIST recurrence beyond 5 yrs and monthly cost of adjuvant imatinib. At a threshold of $100,000/QALY, 3 yr imatinib therapy was cost-effective in 100% of simulations vs. 1 yr of imatinib. Conclusions: Model results suggest that treating patients with 3 yrs of imatinib is cost-effective vs. 1 yr of imatinib below the widely used $100,000/QALY threshold. Both clinical and economic results suggest treating surgically resected GIST patients with 3 yrs of imatinib would result in improved quality-adjusted and overall survival.


2021 ◽  
Vol 21 (8) ◽  
Author(s):  
Abdollah Poursamad ◽  
Zahra Goudarzi ◽  
Iman Karimzadeh ◽  
Nahid Jallaly ◽  
Khosro Keshavarz ◽  
...  

Background: Hepatitis C virus (HCV) can lead to increased mortality, disability, and liver transplantation if left untreated, and it is associated with a possible increase in disease burden in the future, all of which would surely have a significant impact on the health system. New antiviral regimens are effective in the treatment of the disease yet expensive. Objectives: The purpose of the present study was to assess the cost-effectiveness of three medication regimens, namely, ledipasvir/sofosbuvir (LDV/SOF), velpatasvir/sofosbuvir, and daclatasvir/sofosbuvir (DCV/SOF) for HCV patients with genotype 1 in Iran. Methods: A Markov model with a lifetime horizon was developed to predict the costs and outcomes of the three mentioned medication therapy strategies. The final outcome of the study was quality-adjusted life-years (QALYs), which was obtained using the previously published studies. The study was conducted from the perspective of the Health Ministry; therefore, only direct medical costs were estimated. The results were provided as the incremental cost-effectiveness ratio (ICER) per QALY. Ultimately, the one-way and probabilistic sensitivity analyses were used to measure the strength of study results. Results: The results showed that the QALYs for LDV/SOF, DCV/SOF, and VEL/SOF were 13.25, 13.94, and 14.61, and the costs were 4,807, 7,716, and 4,546$, respectively. The VEL/SOF regimen had lower costs and higher effectiveness than the LDV/SOF and DCV/SOF regimens, making it a dominant strategy. The tornado diagram results showed that the study results had the highest sensitivity to chronic hepatitis C (CHC) and compensated cirrhosis (CC) state costs. Moreover, the scatter plots showed that the VEL/SOF was the dominant therapeutic strategy in 73% of the simulations compared to LDV/SOF and 66% of the simulations compared to DCV/SOF; moreover, it was in the acceptable region in 92% of the simulations and below the threshold. Therefore, it was considered the most cost-effective strategy. Moreover, the results showed that DCV/SOF was in the acceptable region below the threshold in 69% of the simulations compared to LDV/SOF. Therefore, the DCV/SOF regimen was more cost-effective than LDV/SOF. Conclusions: According to the present study results, it is suggested that the VEL/SOF regimen be used as the first line of therapy in patients with HCV genotype 1. Moreover, DCV/SOF can be the second-line medication regimen.


2021 ◽  
Author(s):  
Xiaoyu Xi ◽  
Xin Wang ◽  
Wenwen Xie ◽  
Yu Jia ◽  
Santiago Zuluaga Sanchez ◽  
...  

Abstract Purpose: To assess the cost-effectiveness of evolocumab, a PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitor, added to background statins therapy in patients with a recent acute coronary syndrome (ACS) event (in the past 12 months) and low-density lipoprotein cholesterol (LDL-C) levels ≥100 mg/dL in China.Methods: A health economic evaluation was performed from a Chinese healthcare perspective, using a Markov model over a lifetime horizon based on baseline CV event rate from claims database data and efficacy from the FOURIER trial. The health benefit was reflected in the decrease of LDL-C level, which led to the decrease of cardiovascular events. The cost of cardiovascular events and the utility value of each health state were derived from published literature. Sensitivity analysis were conducted to evaluate the effects of uncertainty in parameters and the robustness of the model. The cost-effectiveness of evolocumab was also explored in patients with recent MI, very high-risk (VHR) ASCVD and homozygous familiar hypercholesterolemia (HoFH).Results: In recent ACS patients, evolocumab was associated with incremental quality adjusted life years (QALYs) of 1.41 and incremental costs of 120,966 yuan vs. ezetimibe, both with background statins therapy, resulting in an ICER of 85,964 yuan per QALY gained. The probability that evolocumab is cost-effective at a threshold of 217,341 yuan (3 times per capita GDP, 2020) was 100% in patients with recent ACS, recent MI, VHR ASCVD and HoFH.Conclusion: Compared with ezetimibe, evolocumab was considered to be cost-effective in patients with a recent ACS event in China.


2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18829-e18829
Author(s):  
Kishan Patel ◽  
Stacey Stein ◽  
Janki Luther ◽  
Scott F. Huntington

e18829 Background: The IMbrave150 trial found that atezolizumab and bevacizumab significantly prolonged progression-free survival (PFS) and overall survival (OS) in patients with locally advanced metastatic or unresectable hepatocellular carcinoma (HCC), compared to sorafenib. However, atezolizumab and bevacizumab are costly treatments and are administered indefinitely until disease progression. Therefore, it is unclear whether atezolizumab-bevacizumab is cost-effective in this clinical setting. Methods: We constructed a partitioned survival model to compare the costs and effectiveness of atezolizumab-bevacizumab to sorafenib in advanced HCC. PFS and OS curves for each treatment strategy were derived from the IMbrave150 trial using parametric survival modeling. The utility of each health state and the costs of treatment, adverse events, and terminal care were derived from literature and Medicare fee schedules. We calculated the incremental cost-effectiveness ratio (ICER) of atezolizumab-bevacizumab from a US healthcare perspective, using a lifetime horizon, an annual discount rate of 3%, and a willingness-to-pay threshold of $150,000/quality-adjusted life year (QALY). One-way and probabilistic sensitivity analyses were performed to evaluate the robustness of model conclusions. Results: Atezolizumab-bevacizumab was associated with an incremental cost of $102,648 and an incremental effectiveness of 0.42 QALYs compared to sorafenib, leading to an ICER of $244,213/QALY (Table). The price of atezolizumab would need to be reduced by 40% (from ̃$9,400 to ̃$5,700 per dose) or the price of bevacizumab would need to be reduced by 47% (from ̃$8,400 to ̃$4,400 per dose) for atezolizumab-bevacizumab to be cost-effective compared to sorafenib. Alternatively, the price of both atezolizumab and bevacizumab would need to be simultaneously decreased by ̃21% for the combination therapy to be cost-effective. Our model was most sensitive to the hazard ratios (HR) of OS and PFS; varying the HRs across the 95% confidence interval reported in IMbrave150 (0.42-0.79) corresponded to ICERs of $137,435/QALY and $621,365/QALY, respectively. During probabilistic sensitivity analyses, >99%, 99%, and 90% of iterations produced ICERs greater than willingness-to-pay thresholds of $50,000/QALY, $100,000/QALY, and $150,000/QALY, respectively. Conclusions: Use of atezolizumab-bevacizumab for advanced HCC is unlikely to be cost-effective under current pricing. Significant price reduction of atezolizumab and/or bevacizumab would be required to reduce the ICER to a more widely acceptable value.[Table: see text]


2013 ◽  
Vol 31 (15_suppl) ◽  
pp. e17552-e17552 ◽  
Author(s):  
Trefor Jones ◽  
W. Robert Simons

e17552 Background: The Eisai Metastatic Breast Cancer Study Assessing Physician’s Choice Versus E7389 (EMBRACE) established clinical benefits. This study evaluates its translation into comparative economic value. Methods: Because of superior survival benefits and a non-inferior safety profile, we use a cost-effectiveness analysis. Costs include medication and administration costs, cost of toxicity management, and indirect cost. The primary endpoint is the ratio of incremental means of costs and quality adjusted life years (QALY) yielding an incremental cost effectiveness ratio (ICER). Partitioned survival analyses were evaluated using a log-logistic function for overall survival, progression free survival, response duration, and toxicity time. Health state utilities or quality weights are applied to each component and aggregated. Sensitivity analyses assessed the influence of variability in a number of parameters. Results: Overall survival based on the log-logistic extrapolation was 686 days in the eribulin group compared to 550 days in the TPC group for a difference of 136 days. Mean time without progressive disease was 214 days and 160 days, respectively, for a difference of 59 days. Time spent with Grade 3 and 4 toxicity were 3.62 and 2.25 days in the eribulin group versus 2.25 and 0.98 days in the TPC group. Response times were 15.62 and 9.64 days, 5.98 days longer in the eribulin group. After weight components of overall survival with corresponding utilities and converting to years, the QALY were 1.166 in the eribulin group compared to 0.926 in the TPC group for a mean incremental improvement of 0.24 years. Mean treatment costs were $14,302.80 AUD and $1,672.02 AUD for a difference of $12,630.78 AUD. Total incremental cost is $13,794.35 AUD. The ICER with and without discounting is $48,134.29 AUD and $45,770.97 AUD, respectively. Survival time and drug cost were the most influential variables on the ICER. Conclusions: At a threshold of $50,000 AUD per QALY, eribulin is good value at $48,134 AUD per QALY. Clinical trial information: NCT00388726.


2018 ◽  
Vol 34 (S1) ◽  
pp. 139-140
Author(s):  
Borja Garcia-Lorenzo ◽  
Tasmania del Pino-Sedeño ◽  
Maria M. Trujillo-Martin ◽  
Rodrigo Alberto Rocamora Zuniga ◽  
Juan Erviti López ◽  
...  

Introduction:Stereo-electroencephalography (SEEG) has been shown to be a valuable tool for the anatomo-electroclinic definition of the epileptogenic zone (EZ) in some patients with medically refractory epilepsy considered for surgery. In Spain, many of those patients are not offered this diagnostic procedure. The objective of our health technology assessment (HTA) report was to evaluate the effectiveness, safety and cost-effectiveness of SEEG to define the EZ in patients with refractory epilepsy considered for surgery compared to no SEEG intervention (i.e. remaining with further antiepileptic drugs).Methods:We undertook a systematic review with meta-analyses on the effectiveness and safety of SEEG. A cost-effectiveness analysis was conducted using a Markov model which simulates the costs and health outcomes of individuals for a lifetime horizon from the perspective of the Spanish National Health Service (NHS). The effectiveness measure was quality-adjusted life years (QALYs). We ran extensive sensitivity analyses, including a probabilistic sensitivity analysis.Results:The EZ was found in 92 percent of patients who underwent SEEG, 72 percent were eligible for epilepsy surgery and 33 percent were free of seizures after surgery (47 percent of those who received surgery). Any complications related to insertion and monitoring of SEEG and the subsequent intervention occurred in 1.3 percent of patients. In the base case analysis, SEEG led to higher QALYs and healthcare costs with an estimated incremental cost-effectiveness ratio of EUR 10,368 (USD 12,217) per QALY. The sensitivity analyses showed that the results of the study were robust.Conclusions:SEEG is a cost-effective technology in patients with refractory epilepsy considered for surgery when compared to no SEEG intervention.


2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e19529-e19529 ◽  
Author(s):  
Josh John Carlson ◽  
Benjamin Eckert ◽  
Marita Zimmerman

e19529 Background: MRD testing is part of the response evaluation criteria for MM, and clinical guidelines recommend MRD testing with technologies that detect malignant cells at a level of at least 1/100,000. Deep MRD negativity correlates with improved health outcomes for MM pts, and MRD status may inform treatment (tx) continuation decisions during MM maintenance therapy. We evaluated the potential cost-effectiveness of NGS MRD (clonoSEQ) vs. no MRD testing in the maintenance MM setting. Methods: We developed a Markov model with 6 health states: MRD+ or MRD- on or off tx, relapsed, or dead and compared yearly NGS MRD to no MRD testing over a lifetime horizon. The model assumed tx stops after an MRD- result; otherwise, tx continued until disease progression. Progression-free survival (PFS) and overall survival (OS) data for MRD- and MRD+ and hazard ratios for on/off tx were applied based on peer-reviewed literature. MRD- pts were assumed to have the same PFS/OS rates on and off tx, which were varied in sensitivity analyses. On tx, 2%/month of MRD+ pts became MRD-. Health state utilities were based on peer-reviewed literature and included an adverse event (AE) disutility. The cost of NGS MRD was $1,800 + $1,836 for specimen collection, with maintenance tx at $21,168/month and tx for relapsed pts at $27,422/month. We used a US health system perspective and a 3% discount rate. Results: MRD testing saved $1,156,600 over patients remaining lifetime. Health outcomes were similar and slightly favored MRD testing vs no testing (0.01 quality-adjusted life-years [QALYs]) from lower AE risk over time, suggesting a potentially dominant strategy, i.e., less costly with improved health outcomes. This result was most sensitive to the probability of MRD+ to MRD- transition and the cost of maintenance tx. Conclusions: NGS MRD testing is cost-saving, with potential QALY gains due to avoidance of tx-related AEs compared with no testing for MM patients on maintenance therapy. Further studies are needed to determine the health outcomes of MRD testing during MM maintenance tx. [Table: see text]


2021 ◽  
pp. 00333-2021
Author(s):  
Alan Martin ◽  
Dhvani Shah ◽  
Kerigo Ndirangu ◽  
Glenn A Anley ◽  
Gabriel Okorogheye ◽  
...  

BackgroundThe IMPACT trial demonstrated superior outcomes following 52 weeks of once-daily single-inhaler treatment with fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) 100/62.5/25 μg compared with once-daily FF/VI (100/25 μg) or UMEC/VI (62.5/25 μg). This study evaluates the cost-effectiveness of FF/UMEC/VI compared with FF/VI or UMEC/VI for the treatment of chronic obstructive pulmonary disease (COPD) from a United Kingdom National Health Service perspective.MethodsPatient characteristics and treatment effects from IMPACT were populated into a hybrid decision tree/Markov economic model. Costs (GB£ inflated to 2018 equivalents) and health outcomes were modelled over a lifetime horizon, with a discount rate of 3.5% per annum applied to both. Sensitivity analyses were performed to test the robustness of key assumptions and input parameters.ResultsCompared with FF/VI and UMEC/VI, FF/UMEC/VI provided an additional 0.296 and 0.145 life years (LYs; discounted), and 0.275 and 0.118 quality-adjusted life years (QALYs), at an additional cost of £1129 and £760, respectively. Incremental cost-effectiveness ratios (ICERs) for FF/UMEC/VI were £4104/QALY and £3809/LY gained versus FF/VI and £6418/QALY and £5225/LY gained versus UMEC/VI. At a willingness-to-pay threshold of £20 000/QALY, the probability that FF/UMEC/VI was cost-effective was 96% versus FF/VI and 74% versus UMEC/VI. Results were similar in a subgroup reflecting patients recommended triple therapy in the 2019 National Institute for Health and Care Excellence COPD guideline.ConclusionsFF/UMEC/VI single-inhaler triple therapy improved health outcomes and was a cost-effective option compared with FF/VI or UMEC/VI for patients with symptomatic COPD and a history of exacerbations in the UK at recognised cost-effectiveness threshold levels.


2018 ◽  
Vol 36 (4_suppl) ◽  
pp. 800-800 ◽  
Author(s):  
Sebastian Stintzing ◽  
Ilse van Oostrum ◽  
Chris Pescott ◽  
Alma Katharina Steinbach-Buechert ◽  
Bart Heeg ◽  
...  

800 Background: The randomized, phase 3 FIRE-3 trial evaluated 1L FOLFIRI + cetuximab or bevacizumab in patients with RAS wt mCRC; overall survival favored FOLFIRI + cetuximab by > 8 months. The purpose of this analysis was to evaluate the cost-effectiveness of FOLFIRI + cetuximab vs that of FOLFIRI + bevacizumab as 1L treatment for patients in Germany with RAS wt mCRC (including the patient subgroup with RAS wt, left-sided [LS] primary tumors, as LS is a predictive factor). Methods: A standard oncology 3–health-state partitioned survival cost-utility model was developed to analyze the costs and health benefits of FOLFIRI + cetuximab vs those of FOLFIRI + bevacizumab from a German payer perspective based on data from FIRE-3 and the literature. Health outcomes were reported in life-years (LYs) and quality-adjusted life-years (QALYs) gained. A 3.5% discounting rate was applied to the modeled costs and outcomes. Results: Discounted costs, health gains, and incremental cost-effectiveness ratios (ICERs) for patients with RAS wt (base case) and patients with RAS wt, LS (subgroup) mCRC are summarized in the Table. Probabilistic sensitivity analyses showed that at relevant European willingness-to-pay (WTP) thresholds of €55,000 and €80,000, FOLFIRI + cetuximab had a 64.0% and 81.6% (base case) and 80.5% and 92.4% (subgroup) probability of being cost-effective vs FOLFIRI + bevacizumab, respectively. Clinical trial information: NCT00433927. Conclusions: Based on our analyses, FOLFIRI + cetuximab is cost-effective compared with FOLFIRI + bevacizumab in patients in Germany with RAS wt mCRC at official WTP thresholds applied by relevant European health technology assessment agencies. The cost-effectiveness of FOLFIRI + cetuximab is more pronounced in the subgroup of patients with RAS wt, LS tumors.[Table: see text]


2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e20703-e20703
Author(s):  
Ashley Kim ◽  
Beth Devine ◽  
Joshua A. Roth

e20703 Background: Trial results from KEYNOTE-407 have recently led to the FDA approval for pembrolizumab + carboplatin + paclitaxel/nab-paclitaxel (pembrolizumab+chemo) in previously untreated metastatic squamous NSCLC. This is the only first-line indication for squamous NSCLC regardless of tumor expression status. Our objective was to evaluate the cost-effectiveness of pembrolizumab combination therapy in this setting from the US payer perspective. Methods: Using data from KEYNOTE-407, we developed a partitioned survival decision model to estimate the lifetime costs and effectiveness of pembrolizumab+chemo vs. chemo alone in the first-line treatment of metastatic squamous NSCLC. The base case used a Weibull curve selected based on minimum AIC/BIC and best graphical fit to extrapolate in-trial survival to a lifetime horizon. First- and second-line therapy resource use and adverse event (AE) rates were derived from KEYNOTE-407. Utility data and AE management were obtained from published literature and national sources. Direct medical costs were adjusted to 2018 US dollars, and future costs and outcomes were discounted at 3% per year. We estimated life years (LY), quality-adjusted life years (QALYs), and costs over a lifetime horizon. One-way and probabilistic sensitivity analyses were also conducted. Results: In the base case, pembrolizumab+chemo resulted in 0.51 more LYs, 0.36 more QALYs, and $233,246 in healthcare costs vs. chemo alone. Costs per LY and QALY gained were $216,180 and $309,004, respectively. One-way sensitivity analyses indicated that the results were most sensitive to survival and pre-progression utility inputs. In a threshold analysis, we found that the cost of pembrolizumab+chemo would need to be reduced by 24% per course of therapy ($176,175) in order to be cost-effective at $150,000/QALY. Conclusions: Based on current available data, our analysis suggests first-line pembrolizumab-based combination therapy in metastatic squamous NSCLC is unlikely to be cost-effective relative to implied willingness to pay in cancer in the U.S. (ie < $150,000 per QALY). Future studies should reassess cost-effectiveness as trial data mature.


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