Cost-Effectiveness of Adding Imatinib to Chemotherapy in Adult Patients with Chromosom-Positive Acute Lymphoblastic Leukemia (Ph+ALL): A Canadian Perspective

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 2401-2401 ◽  
Author(s):  
Khalid El Ouagari ◽  
Kimbach Tran ◽  
Jennifer Stephens ◽  
Marc Botteman

Abstract BACKGROUND: Ph+ALL is a rare, high-risk, aggressive form of acute leukemia, affecting primarily adults and the elderly. Patients with high-risk forms of ALL typically have extremely poor prognosis and incur high disease-related costs. Successful use of imatinib in patients with Ph+CML has led to the administration of imatinib in recent clinical studies for patients with Ph+ALL. Given the changing treatment landscape of Ph+ALL and the recent development of additional targeted treatments for this disease, an appraisal of the economic burden associated with Ph+ALL is warranted for evaluation of appropriate treatment options and the potential value associated with novel therapies. This study explores the cost effectiveness of imatinib plus conventional chemotherapy (CC) regimens versus CC alone in adult Ph+ALL patients. METHODS: A Markov model simulated a hypothetical cohort of 1000 adult Ph+ALL patients receiving imatinib+CC or CC alone. Patients were distributed over time into three health states: alive without disease progression (DFS), alive with disease progression (DS), or dead. Probabilities of being in the states were derived from the published literature. In the absence of relevant data pertaining to Ph+ALL, assumptions about costs and utilities were derived from a cost analysis of CML. A Canadian health care payer perspective was considered, therefore only direct medical costs were included in the analysis. Patients were followed for a total of ten years in monthly intervals. All outcomes were discounted at a 5% rate per annum. RESULTS: Based on the model framework and assumptions, the total discounted survival was 1.09 years for CC and 4.13 years for imatinib+CC. Total discounted disease free survival was 0.76 year for CC and 2.69 years for imatinib+CC. Assuming utility weights of 0.854 and 0.596 for DFS and DS, respectively, the total discounted quality adjusted life years (QALY) were estimated to be 0.84 versus 3.16 for CC and imatinib+CC, respectively. Thus, the net incremental gain in discounted quality adjusted survival was 2.32 QALYs. The monthly costs of DFS and DS were estimated at $168 and $848, respectively. The net costs associated with imatinib were $101,929. The incremental cost per QALY of imatinib+CC v. CC alone was approximately $44,048 (i.e., $101,929 divided by 2.32 QALYs). CONCLUSIONS: Our results suggest that imatinib in the treatment of Ph+ALL is a cost-effective use of health-care resources, and should be considered in patients who are appropriate candidates for such therapy.

2018 ◽  
Vol 53 (2) ◽  
pp. 134-143 ◽  
Author(s):  
Quang A. Le ◽  
Joel W. Hay ◽  
Russell Becker ◽  
Yamei Wang

Background: The US Food and Drug Administration has recently approved abaloparatide (ABL) for treatment of women with postmenopausal osteoporosis (PMO) at high risk of fracture. With increasing health care spending and drug prices, it is important to quantify the value of newly available treatment options for PMO. Objective: To determine cost-effectiveness of ABL compared with teriparatide (TPTD) for treatment of women with PMO in the United States. Methods: A discrete-event simulation (DES) model was developed to assess cost-effectiveness of ABL from the US health care perspective. The model included three 18-month treatment strategies with either placebo (PBO), TPTD, or ABL, all followed by additional 5-year treatment with alendronate (ALN). High-risk patients were defined as women with PMO ⩾65 years old with a prior vertebral fracture. Baseline clinical event rates, risk reductions, and patient characteristics were based on the Abaloparatide Comparator Trial in Vertebral Endpoints (ACTIVE) trial. Results: Over a 10-year period, the DES model yielded average total discounted per-patient costs of $10 212, $46 783, and $26 837 and quality-adjusted life-years (QALYs) of 6.742, 6.781, and 6.792 for PBO/ALN, TPTD/ALN, and ABL/ALN, respectively. Compared with TPTD/ALN, ABL/ALN accrued higher QALYs at lower cost and produced an incremental cost-effectiveness ratio (ICER) of $333 266/QALY relative to PBO/ALN. In high-risk women, ABL/ALN also had more QALYs and less cost over TPTD/ALN and yielded an ICER of $188 891/QALY relative to PBO/ALN. Conclusion and Relevance: ABL is a dominant treatment strategy over TPTD. In women with PMO at high risk of fracture, ABL is an alternative cost-effective treatment.


Author(s):  
Praveen Thokala ◽  
Matt Stevenson ◽  
Varun M. Kumar ◽  
Shijie Ren ◽  
Alexandra G. Ellis ◽  
...  

Abstract Background Patients with infantile-onset spinal muscular atrophy (SMA), a rare, genetic neuromuscular disease, do not achieve key motor function milestones (e.g., sitting) and have short life expectancy in the absence of treatment. Nusinersen is a disease-modifying therapy for patients with SMA. Objective The aim of this study was to estimate the cost-effectiveness of nusinersen compared to best supportive care (BSC) in patients diagnosed with infantile-onset SMA in the US. Methods A de novo economic model was developed with the following health states: “permanent ventilation”, “not sitting”, “sitting”, “walking”, and “death”. Short-term data were sourced from the pivotal clinical trials and studies of nusinersen (ENDEAR and SHINE). Motor function milestones achieved at the end of follow-up in the clinical trials were assumed to be sustained until death. Mortality risks were based on survival modelling of relevant published Kaplan–Meier data. Costs, life years (LYs), and quality-adjusted life years (QALYs) were discounted at 3% per annum, and the analyses were performed from a US health care sector perspective. Scenario analyses and sensitivity analyses were conducted to assess the robustness of the results to key parameters. Results In our base-case analysis, nusinersen treatment achieves greater QALYs and more LYs (3.24 and 7.64, respectively) compared with BSC (0.46 QALYs and 2.40 LYs, respectively), resulting in an incremental cost per QALY gained of approximately $1,112,000 and an incremental cost per LY gained of $590,000 for nusinersen compared to BSC. The incremental cost effectiveness ratios did not fall below $990,000 per QALY gained in scenario and sensitivity analyses. Results were most sensitive to the length of survival, background health care costs, and utility in the “not sitting” and “sitting” health states. Conclusions The estimated incremental cost-effectiveness of nusinersen from a US health care sector perspective exceeded traditional cost-effectiveness thresholds. Cost-effectiveness was dependent on assumptions made regarding survival, costs, utilities, and whether the motor function milestones were sustained over lifetime. Given the relatively short-term effectiveness data available for the treatment, a registry to collect long-term data of infantile-onset SMA patients is recommended.


2002 ◽  
Vol 16 (12) ◽  
pp. 873-876 ◽  
Author(s):  
Craig R Mitton

The report of the Canadian Coordinating Office for Health Technology Assessment (CCOHTA) on the use of infliximab in the treatment of refractory Crohn’s disease stated that the medication did not meet ‘conventional standards of cost-effectiveness’. It had several methodological weaknesses, however, including the derivation of the quality-adjusted life-years (QALYs) gained and the interpretation of the incremental cost utility ratios (ICURs). The validity of economic analyses is highly dependent on the underlying assumptions that are made about the implications of health care states and treatments. The authors of the report mapped utilities from three health states, taken from an American study, onto the nine health states that were considered in their economic analysis. The QALYs that were derived might not have been sensitive to small changes in health outcomes. Moreover, the indirect costs of Crohn’s disease and its complications were ignored. Therefore, it is possible that the benefits of infliximab therapy were underestimated. The high ICURs that were quoted in the report do not necessarily mean that infliximab is not valuable, because opportunity costs were not considered. Instead of calculating the ICUR, a preferable approach would be to determine the benefit of this therapy, compared with that which could be derived from alternative uses of the same amount of health care resources. A ‘balance sheet’ approach would allow decision-makers to determine whether the additional cost of infliximab therapy would be justified by the health care gains that it produces. It is inappropriate to assign an arbitrary cut-off point to cost effectiveness, as defined by ICURs, especially when considering new and expensive treatments for severely ill patients who have few other therapeutic alternatives. Because only a small number of patients would require infliximab, the overall expenditure that would be required to make it available may be manageable.


Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 565-565
Author(s):  
Petros Pechlivanoglou ◽  
Linda Luu ◽  
Qing Li ◽  
Paul J. Gibson ◽  
Uma H. Athale ◽  
...  

Abstract Introduction: Though cure rates for childhood acute lymphoblastic leukemia (ALL) have improved significantly, outcomes for children with relapsed ALL remain poor. Novel immunotherapies have proved effective in relapsed disease. Blinatumomab, a bispecific T-cell engager targeting CD19, was recently shown to improve disease-free and overall survival among children with high-risk relapsed disease (Brown et al, 2021) prior to proceeding to stem cell transplant. Blinatumomab however also represents a significant cost burden to institutions and healthcare systems. Our objective was thus to determine the cost-effectiveness of blinatumomab vs. standard of care therapy among children with high-risk relapsed ALL. Methods: We used a childhood ALL-specific policy simulation model previously developed using real world clinical, healthcare utilization, and cost population-based data from Ontario, Canada, and restricted it to children with B-lineage ALL who relapsed with 18 months of their original diagnosis to capture a population of patients with high-risk relapse who survived their original month of re-induction chemotherapy. This model was combined with published data from the Children's Oncology Group randomized control study AALL1331 to estimate the long-term survival, healthcare costs and quality adjusted life years associated with two courses of blinatumomab vs. two courses of standard intensive chemotherapy, followed by stem cell transplant. Combining these two sources allowed for the creation of a multi-state survival model and the estimation of the impact of blinatumomab upon relapse and survival. Healthcare costs for the administration of blinatumomab or chemotherapy were sourced from administrative data, provincial formularies, and published sources. Lifetime costs and quality-adjusted life years were calculated while Monte Carlo simulation was used to propagate parameter uncertainty in the cost-effectiveness outcomes. All costs were calculated in Canadian dollars (CAD). Results: Blinatumomab was associated with on average higher life expectancy (43.4 years vs. 36.8 years) and a higher number of quality adjust life years [2.75 QALYs gained, 95 th confidence interval (95CI) -0.44 to 5.99] but higher costs (60,420 CAD, 95CI 26,794 - 94,047) compared to standard intensive chemotherapy. The incremental cost effectiveness ratio (ICER) associated with blinatumomab was 21,970 CAD/QALY gained. However, substantial uncertainty around model parameters resulted in wide confidence intervals around cost-effectiveness outcomes. Discussion: Initial evidence indicates that the use of blinatumomab in high-risk relapse of childhood ALL as compared to standard chemotherapy is associated with a cost-effectiveness ratio that is comparable to or lower than that associated with other interventions recently introduced in pediatric ALL, and represents a cost-effective intervention for this population. However, estimates are based on limited evidence; further research is necessary to better inform several model parameters and thereby decrease uncertainty in cost-effectiveness outcomes. Disclosures Gupta: Jazz Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees.


2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18846-e18846
Author(s):  
Jesse Sussell ◽  
Joshua A. Roth ◽  
Svenn Hansen ◽  
Craig S. Meyer ◽  
Anita M. Fung

e18846 Background: Standard therapy for high-risk HER2+ early breast cancer (EBC) begins with neoadjuvant dual targeted therapy with pertuzumab (P) + trastuzumab (T) + chemotherapy (CTX). After surgery, patients who achieve pathological complete response (pCR) should complete one year of dual targeted therapy, while patients with residual disease should receive ado-trastuzumab emtansine (T-DM1). Recently, a subcutaneously administered formulation of P, T, and hyaluronidase-zzxf (Phesgo) was approved for use in the U.S. This study assesses the value of this new formulation in EBC patients vs. a strategy in which patients initiate standard therapy, but discontinue P following ascertainment of pCR. Methods: We developed a six-state Markov model to assess EBC costs and quality-adjusted life years (QALYs) from a healthcare sector perspective over a lifetime time horizon. Two strategies were modeled: 1) Neoadjuvant therapy with subcutaneous P, T, and hyaluronidase-zzxf + CTX with adjuvant continuation if pCR is achieved, and T-DM1 if not (“intervention”), and 2) neoadjuvant therapy with infused P, T + CTX with adjuvant infused T if pCR is achieved, and T-DM1 if not (“comparator”). Event-free and invasive-disease free survival were derived from the PEONY and KATHERINE/APHINITY trials, respectively. Utility values, drug prices, and procedure costs were derived from KATHERINE EQ-5D data, Wholesale Acquisition Costs, and claims analyses, respectively. We assessed comparator costs using both biosimilar and branded T pricing. The primary outcome was the incremental cost-effectiveness ratio (ICER). Outcomes were discounted at 3%/year and costs are presented in 2020 U.S. dollars. Uncertainty in outcomes was assessed through Monte Carlo simulation (1,000 replicates). Results: The table shows key results. The intervention resulted in a gain of 0.092 QALYs. With biosimilar T pricing in the comparator (base case), the intervention increased costs by $7,575 (ICER = $81,793). With branded T pricing in the comparator (scenario analysis), the intervention increased costs by $982 (ICER = $10,602). In probabilistic analyses, the intervention was favored in 52% and 81% of simulations at a willingness-to-pay of $100,000/QALY with biosimilar and branded T pricing, respectively. Conclusions: This study provides additional evidence to support adjuvant continuation of P+T among patients achieving pCR. Neoadjuvant P, T, and hyaluronidase-zzxf + CTX (with adjuvant continuation of dual targeted therapy) is expected to be cost-effective ( < $100,000/QALY) vs. neoadjuvant P and T + CTX (with adjuvant T continuation) for patients with high-risk HER2+ EBC irrespective of whether the comparator uses biosimilar or branded T.[Table: see text]


2018 ◽  
Vol 25 (5) ◽  
Author(s):  
T. Sharma ◽  
C. Tajzler ◽  
A. Kapoor

BackgroundAlthough surgical resection remains the standard of care for localized kidney cancers, a significant proportion of patients experience systemic recurrence after surgery and hence might benefit from effective adjuvant therapy. So far, several treatment options have been evaluated in adjuvant clinical trials, but only a few have provided promising results. Nevertheless, with the recent development of targeted therapy and immunomodulatory therapy, a series of clinical trials are in progress to evaluate the potential of those novel agents in the adjuvant setting. In this paper, we provide a narrative review of the progress in this field, and we summarize the results from recent adjuvant trials that have been completed.MethodsA literature search was conducted. The primary search strategy at the medline, Cochrane reviews, and http://ClinicalTrials.gov/ databases included the keywords “adjuvant therapy,” “renal cell carcinoma,” and “targeted therapy or/and immunotherapy.”ConclusionsData from the s-trac study indicated that, in the “highest risk for recurrence” patient population, disease-free survival was increased with the use of adjuvant sunitinib compared with placebo. The assure trial showed no benefit for adjuvant sunitinib or sorafenib in the “intermediate- to high-risk” patient population. The ariser (adjuvant girentuximab) and protect (adjuvant pazopanib) trials indicated no survival benefit, but subgroup analyses in both trials recommended further investigation. The inconsistency in some of the current results can be attributed to a variety of factors pertaining to the lack of standardization across the trials. Nevertheless, patients in the “high risk of recurrence” category after surgery for their disease would benefit from a discussion about the potential benefits of adjuvant treatment and enrolment in ongoing adjuvant trials.


Author(s):  
Christina Liu Cui ◽  
William Yu Luo ◽  
Bard Clifford Cosman ◽  
Samuel Eisenstein ◽  
Daniel Simpson ◽  
...  

Abstract Background Watch and wait (WW) protocols have gained increasing popularity for patients diagnosed with locally advanced rectal cancer and presumed complete clinical response after neoadjuvant chemoradiation. While studies have demonstrated comparable survival and recurrence rates between WW and radical surgery, the decision to undergo surgery has significant effects on patient quality of life. We sought to conduct a cost-effectiveness analysis comparing WW with abdominoperineal resection (APR) and low anterior resection (LAR) among patients with stage II/III rectal cancer. Methods In this comparative-effectiveness study, we built Markov microsimulation models to simulate disease progression, death, costs, and quality-adjusted life-years (QALYs) for WW or APR/LAR. We assessed cost effectiveness using the incremental cost-effectiveness ratio (ICER), with ICERs under $100,000/QALY considered cost effective. Probabilities of disease progression, death, and health utilities were extracted from published, peer-reviewed literature. We assessed costs from the payer perspective. Results WW dominated both LAR and APR at a willingness to pay (WTP) threshold of $100,000. Our model was most sensitive to rates of distant recurrence and regrowth after WW. Probabilistic sensitivity analysis demonstrated that WW was the dominant strategy over both APR and LAR over 100% of iterations across a range of WTP thresholds from $0–250,000. Conclusions Our study suggests WW could reduce overall costs and increase effectiveness compared with either LAR or APR. Additional clinical research is needed to confirm the clinical efficacy and cost effectiveness of WW compared with surgery in rectal cancer.


2012 ◽  
Vol 23 (2) ◽  
pp. 59-64 ◽  
Author(s):  
Amir A Tahami Monfared ◽  
Amy K O’Sullivan ◽  
Coleman Rotstein ◽  
George Papadopoulos

INTRODUCTION: Posaconazole prophylaxis in high-risk neutropenic patients prevents invasive fungal infection (IFI). An economic model was used to assess the cost effectiveness of posaconazole from a Canadian health care system perspective.METHODS: A decision-analytic model was developed based on data from a randomized trial comparing posaconazole with standard azole (fluconazole or itraconazole) therapy. The model was extrapolated to a lifetime horizon using one-month Markov cycles; lifetime survival was specific to the underlying disease. Drug and treatment costs associated with IFI were estimated using published literature. The model was used to estimate total costs, IFIs avoided, life-years gained and the incremental cost-effectiveness ratio of posaconazole versus standard azole therapy, in 2007 Canadian dollars.RESULTS: Based on the clinical trial data, posaconazole was associated with fewer cases of IFI (0.05 versus 0.11; P=0.003), increased life-years (2.52 years versus 2.43 years) and slightly lower costs ($6,601 versus $7,045) per patient relative to standard azole therapy over a lifetime horizon. Higher acquisition costs for posaconazole were offset by IFI-associated inpatient costs for those prophylaxed with standard azoles. Probabilistic sensitivity analysis indicated a 59% probability that posaconazole was cost-saving versus standard azole therapy and a 96% probability that the incremental cost-effectiveness ratio for posaconazole was at or below the $50,000 per life-year saved threshold.DISCUSSION: In Canada, posaconazole appears to be cost-saving relative to standard azole therapy in IFI prevention among high-risk neutropenic patients.


Neurology ◽  
2019 ◽  
Vol 92 (23) ◽  
pp. e2674-e2678 ◽  
Author(s):  
Ross Mandeville ◽  
Arvin Wali ◽  
Charlie Park ◽  
Erik Groessl ◽  
Francis O. Walker ◽  
...  

ObjectiveTo evaluate the cost-effectiveness of neuromuscular ultrasound (NMUS) for the evaluation of focal neuropathies.MethodsA prior prospective, randomized, double-blind controlled trial demonstrated that NMUS, when added to electrodiagnostic testing, resulted in improved clinical outcomes after 6 months of follow-up. From this study, we abstracted quality-adjusted life-years (QALYs) from the 36-item Short Form Health Survey and entered this health-utility estimate into a mixed trial and model-based cost-effectiveness analysis from the societal perspective. Costs of intervention (NMUS) were estimated from Medicare payment rates for Current Procedural Terminology codes. Health care use was otherwise estimated to be equal, but sensitivity analyses further examined this and other key assumptions. Incremental cost-effectiveness ratio (ICER) was used as the primary outcome with a willingness-to-pay threshold of $50,000 per QALY.ResultsThe predicted mean health outcome associated with use of NMUS was 0.079 QALY, and the mean cost was $37, resulting in an ICER of $463 per QALY. Results and conclusions remained robust across all sensitivity analyses, including variations in time horizon, initial distribution of health states, costs, and effectiveness.ConclusionsFrom a societal perspective, the addition of NMUS to electrodiagnostic testing when evaluating a focal neuropathy is cost-effective. A study of longer follow-up incorporating total health care use would further quantify the value of NMUS.ClinicalTrials.gov identifier:NCT01394822.


2017 ◽  
Vol 32 (3) ◽  
pp. 267-273 ◽  
Author(s):  
Changzheng Du ◽  
Weicheng Xue ◽  
Fangyuan Dou ◽  
Yifan Peng ◽  
Yunfeng Yao ◽  
...  

Background High-risk patients with stage II colon cancer may benefit from adjuvant chemotherapy, but identifying this patient population can be difficult. We assessed the prognosis value for predicting tumor progression in patients with stage II colon cancer, of a panel of 2 biomarkers for colon cancer: tumor budding and preoperative carcinoembryonic antigen (CEA). Methods Consecutive patients (N = 134) with stage II colon cancer who underwent curative surgery from 2000 to 2007 were included. Multivariate analysis was used to evaluate the association of CEA and tumor budding grade with 5-year disease-free survival (DFS). The prognostic accuracy of CEA, tumor budding grade and the combination of both (CEA-budding panel) was determined. Results The study found that both CEA and tumor budding grade were associated with 5-year DFS. The prognostic accuracy for disease progression was higher for the CEA-budding panel (82.1%) than either CEA (70.9%) or tumor budding grade (72.4%) alone. Conclusions The findings indicate that the combination of CEA levels and tumor budding grade has greater prognostic value for identifying patients with stage II colon cancer who are at high-risk for disease progression, than either marker alone.


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