Cost-Effectiveness Analysis of Ofatumumab As a Treatment for Relapsed Chronic Lymphocytic Leukemia in the United States

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2366-2366
Author(s):  
Gabriel Tremblay ◽  
Anna Forsythe ◽  
Vasudha Bal ◽  
Snigdha Santra ◽  
Andrew Briggs
Keyword(s):  
Adverse Event ◽  
Cost Effectiveness ◽  
Chronic Lymphocytic Leukemia ◽  
Cost Effective ◽  
The United States ◽  
Lymphocytic Leukemia ◽  
Phase Iii ◽  
Life Years ◽  
Quality Adjusted Life

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.

scholarly journals Cost-effectiveness and budget impact of venetoclax in combination with rituximab in relapsed/refractory chronic lymphocytic leukemia in Switzerland

2021 ◽  
Author(s):  
Michaela Barbier ◽  
Nicholas Durno ◽  
Craig Bennison ◽  
Mathias Örtli ◽  
Christian Knapp ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Chronic Lymphocytic Leukemia ◽  
Survival Data ◽  
Impact Analysis ◽  
Budget Impact ◽  
Treatment Strategies ◽  
Progression Free Survival ◽  
Cost Effective ◽  
Lymphocytic Leukemia ◽  
Life Years

Abstract Introduction Venetoclax in combination with rituximab (VEN + R) demonstrated prolonged overall survival (OS) and progression-free survival (PFS) for patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) in comparison to standard chemoimmunotherapy [bendamustine + rituximab (BR)]. We conducted a cost-effectiveness and budget impact analysis comparing VEN + R versus six comparators from the Swiss healthcare payer perspective. Methods A three-state partitioned survival model, developed in accordance with NICE and ISPOR decision modelling guidelines, was adapted to Switzerland. Model inputs were informed by the MURANO trial (survival data, patient characteristics), publicly available Swiss sources (drug prices, inpatient and outpatient costs), Swiss National Institute of Cancer Epidemiology and Registration data (incidence and prevalence values), and Swiss medical expert feedback. We used published (dis-)utility values and adverse event probabilities. Results Over a lifetime, VEN + R resulted in an expected gain of 2.60 quality-adjusted life years (QALYs) per patient and incremental costs of Swiss Francs (CHF) 147,851 compared to BR, leading to an incremental cost-effectiveness ratio of CHF 56,881/QALY gained. Other treatment strategies (for example ibrutinib versus VEN + R) resulted in higher costs and lower QALYs. Results were not different for subgroups of patients with/without deletion of chromosome 17p/tumour protein 53 mutation. In scenario analysis, changes in post-progression treatment costs demonstrated a high impact on results. We estimated an expected value of perfect information of CHF 3,318/patient. A moderate VEN + R uptake was estimated to save CHF 12.3 million during 5 years. Conclusions Using a threshold of CHF 100,000 per QALY, VEN + R was projected to be cost-effective vs BR.

scholarly journals Cost-Effectiveness of Tafamidis Therapy for Transthyretin Amyloid Cardiomyopathy

Circulation ◽  
2020 ◽  
Vol 141 (15) ◽  
pp. 1214-1224 ◽  
Author(s):  
Dhruv S. Kazi ◽  
Brandon K. Bellows ◽  
Suzanne J. Baron ◽  
Changyu Shen ◽  
David J. Cohen ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Cost Effective ◽  
The United States ◽  
Quality Adjusted Life Year ◽  
Life Years ◽  
The Us ◽  
Amyloid Cardiomyopathy ◽  
Quality Adjusted Life

Background: In patients with transthyretin amyloid cardiomyopathy, tafamidis reduces all-cause mortality and cardiovascular hospitalizations and slows decline in quality of life compared with placebo. In May 2019, tafamidis received expedited approval from the US Food and Drug Administration as a breakthrough drug for a rare disease. However, at $225 000 per year, it is the most expensive cardiovascular drug ever launched in the United States, and its long-term cost-effectiveness and budget impact are uncertain. We therefore aimed to estimate the cost-effectiveness of tafamidis and its potential effect on US health care spending. Methods: We developed a Markov model of patients with wild-type or variant transthyretin amyloid cardiomyopathy and heart failure (mean age, 74.5 years) using inputs from the ATTR-ACT trial (Transthyretin Amyloidosis Cardiomyopathy Clinical Trial), published literature, US Food and Drug Administration review documents, healthcare claims, and national survey data. We compared no disease–specific treatment (“usual care”) with tafamidis therapy. The model reproduced 30-month survival, quality of life, and cardiovascular hospitalization rates observed in ATTR-ACT; future projections used a parametric survival model in the control arm, with constant hazards reduction in the tafamidis arm. We discounted future costs and quality-adjusted life-years by 3% annually and examined key parameter uncertainty using deterministic and probabilistic sensitivity analyses. The main outcomes were lifetime incremental cost-effectiveness ratio and annual budget impact, assessed from the US healthcare sector perspective. This study was independent of the ATTR-ACT trial sponsor. Results: Compared with usual care, tafamidis was projected to add 1.29 (95% uncertainty interval, 0.47–1.75) quality-adjusted life-years at an incremental cost of $1 135 000 (872 000–1 377 000), resulting in an incremental cost-effectiveness ratio of $880 000 (697 000–1 564 000) per quality-adjusted life-year gained. Assuming a threshold of $100 000 per quality-adjusted life-year gained and current drug price, tafamidis was cost-effective in 0% of 10 000 probabilistic simulations. A 92.6% price reduction from $225 000 to $16 563 would be necessary to make tafamidis cost-effective at $100 000/quality-adjusted life-year. Results were sensitive to assumptions related to long-term effectiveness of tafamidis. Treating all eligible patients with transthyretin amyloid cardiomyopathy in the United States with tafamidis (n=120 000) was estimated to increase annual healthcare spending by $32.3 billion. Conclusions: Treatment with tafamidis is projected to produce substantial clinical benefit but would greatly exceed conventional cost-effectiveness thresholds at the current US list price. On the basis of recent US experience with high-cost cardiovascular medications, access to and uptake of this effective therapy may be limited unless there is a large reduction in drug costs.

scholarly journals Cost-Effectiveness Analysis of Obinutuzumab Versus Ofatumumab for Previously Untreated Chronic Lymphocytic Leukemia (CLL)

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1324-1324 ◽  
Author(s):  
Carolina Reyes ◽  
Gregory Gazauskas ◽  
Ursula Becker ◽  
Santiago Moreno ◽  
David L. Veenstra
Keyword(s):  
Cost Effectiveness ◽  
Chronic Lymphocytic Leukemia ◽  
Cost Effective ◽  
Lymphocytic Leukemia ◽  
Sensitivity Analyses ◽  
Age Difference ◽  
Total Cost ◽  
Life Years ◽  
The Cost ◽  
The Impact

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.

Cost-effectiveness of digital cognitive behavioral therapy (Sleepio) for insomnia: a Markov simulation model in the United States

SLEEP ◽  
2020 ◽  
Author(s):  
Michael Darden ◽  
Colin A Espie ◽  
Jenna R Carl ◽  
Alasdair L Henry ◽  
Jennifer C Kanady ◽  
...  
Keyword(s):  
United States ◽  
Cost Effectiveness ◽  
Cognitive Behavioral Therapy ◽  
Behavioral Therapy ◽  
Cost Effective ◽  
The United States ◽  
Cognitive Behavioral ◽  
Life Years ◽  
Insomnia Treatment ◽  
Group Cbt

Abstract Study Objectives To examine the cost-effectiveness and potential net monetary benefit (NMB) of a fully automated digital cognitive behavioral therapy (CBT) intervention for insomnia compared with no insomnia treatment in the United States (US). Similar relative comparisons were made for pharmacotherapy and clinician-delivered CBT (individual and group). Methods We simulated a Markov model of 100,000 individuals using parameters calibrated from the literature including direct (treatment) and indirect costs (e.g. insomnia-related healthcare expenditure and lost workplace productivity). Health utility estimates were converted into quality-adjusted life years (QALYs) and one QALY was worth $50,000. Simulated individuals were randomized equally to one of five arms (digital CBT, pharmacotherapy, individual CBT, group CBT, or no insomnia treatment). Sensitivity was assessed by bootstrapping the calibrated parameters. Cost estimates were expressed in 2019 US dollars. Results Digital CBT was cost beneficial when compared with no insomnia treatment and had a positive NMB of $681.06 (per individual over 6 months). Bootstrap sensitivity analysis demonstrated that the NMB was positive in 94.7% of simulations. Relative to other insomnia treatments, digital CBT was the most cost-effective treatment because it generated the smallest incremental cost-effectiveness ratio (−$3,124.73). Conclusions Digital CBT was the most cost-effective insomnia treatment followed by group CBT, pharmacotherapy, and individual CBT. It is financially prudent and beneficial from a societal perspective to utilize automated digital CBT to treat insomnia at a population scale.

Lenvatinib versus sorafenib for unresectable hepatocellular carcinoma: a cost–effectiveness analysis

2020 ◽  
Vol 9 (8) ◽  
pp. 553-562
Author(s):  
Hongfu Cai ◽  
Longfeng Zhang ◽  
Na Li ◽  
Bin Zheng ◽  
Maobai Liu
Keyword(s):  
Hepatocellular Carcinoma ◽  
Cost Effectiveness ◽  
Cost Effective ◽  
Quality Adjusted Life Years ◽  
Cost Effectiveness Ratio ◽  
Phase 3 ◽  
Sorafenib Treatment ◽  
Life Years ◽  
The Cost ◽  
Quality Adjusted Life

Aim: To investigate the cost–effectiveness of lenvatinib and sorafenib in the treatment of patients with nonresected hepatocellular carcinoma in China. Materials & methods: Markov model was used to simulate the direct medical cost and quality-adjusted life years (QALY) of patients with hepatocellular carcinoma. Clinical data were derived from the Phase 3 randomized clinical trial in a Chinese population. Results: Sorafenib treatment resulted in 1.794 QALYs at a cost of $43,780.73. Lenvatinib treatment resulted in 2.916 QALYs for patients weighing <60 and ≥60 kg at a cost of $57,049.43 and $75,900.36, The incremental cost–effectiveness ratio to the sorafenib treatment group was $11,825.94/QALY and $28,627.12/QALY, respectively. Conclusion: According to WHO’s triple GDP per capita, the use of lenvatinib by providing drugs is a cost-effective strategy.

scholarly journals Incremental cost-effectiveness of adult spinal deformity surgery: observed quality-adjusted life years with surgery compared with predicted quality-adjusted life years without surgery

2014 ◽  
Vol 36 (5) ◽  
pp. E3 ◽  
Author(s):  
Ian McCarthy ◽  
Michael O'Brien ◽  
Christopher Ames ◽  
Chessie Robinson ◽  
Thomas Errico ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Surgical Treatment ◽  
Incremental Cost ◽  
Adult Spinal Deformity ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Nonsurgical Patients ◽  
The Cost ◽  
Quality Adjusted Life

Object Incremental cost-effectiveness analysis is critical to the efficient allocation of health care resources; however, the incremental cost-effectiveness ratio (ICER) of surgical versus nonsurgical treatment for adult spinal deformity (ASD) has eluded the literature, due in part to inherent empirical difficulties when comparing surgical and nonsurgical patients. Using observed preoperative health-related quality of life (HRQOL) for patients who later underwent surgery, this study builds a statistical model to predict hypothetical quality-adjusted life years (QALYs) without surgical treatment. The analysis compares predicted QALYs to observed postoperative QALYs and forms the resulting ICER. Methods This was a single-center (Baylor Scoliosis Center) retrospective analysis of consecutive patients undergoing primary surgery for ASD. Total costs (expressed in 2010 dollars) incurred by the hospital for each episode of surgical care were collected from administrative data and QALYs were calculated from the 6-dimensional Short-Form Health Survey, each discounted at 3.5% per year. Regression analysis was used to predict hypothetical QALYs without surgery based on preoperative longitudinal data for 124 crossover surgical patients with similar diagnoses, baseline HRQOL, age, and sex compared with the surgical cohort. Results were projected through 10-year follow-up, and the cost-effectiveness acceptability curve (CEAC) was estimated using nonparametric bootstrap methods. Results Three-year follow-up was available for 120 (66%) of 181 eligible patients, who were predominantly female (89%) with average age of 50. With discounting, total costs averaged $125,407, including readmissions, with average QALYs of 1.93 at 3-year follow-up. Average QALYs without surgery were predicted to be 1.6 after 3 years. At 3- and 5-year follow-up, the ICER was $375,000 and $198,000, respectively. Projecting through 10-year follow-up, the ICER was $80,000. The 10-year CEAC revealed a 40% probability that the ICER was $80,000 or less, a 90% probability that the ICER was $90,000 or less, and a 100% probability that the ICER was less than $100,000. Conclusions Based on the WHO's suggested upper threshold for cost-effectiveness (3 times per capita GDP, or $140,000 in 2010 dollars), the analysis reveals that surgical treatment for ASD is cost-effective after a 10-year period based on predicted deterioration in HRQOL without surgery. The ICER well exceeds the WHO threshold at earlier follow-up intervals, highlighting the importance of the durability of surgical treatment in assessing the value of surgical intervention. Due to the study's methodology, the results are dependent on the predicted deterioration in HRQOL without surgery. As such, the results may not extend to patients whose HRQOL would remain steady without surgery. Future research should therefore pursue a direct comparison of QALYs for surgical and nonsurgical patients to better understand the cost-effectiveness of surgery for the average ASD patient.

A Phase Ib/II Study of Combined Obinutuzumab (Gazyva) and High-Dose Methylprednisolone (HDMP) As Treatment for Patients with Chronic Lymphocytic Leukemia (CLL)

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2051-2051 ◽  
Author(s):  
Januario E. Castro ◽  
Michael Y. Choi ◽  
Carlos I. Amaya-Chanaga ◽  
Natalie Nguyen ◽  
Colin MacCarthy ◽  
...  
Keyword(s):  
Chronic Lymphocytic Leukemia ◽  
Research Funding ◽  
Lymphocytic Leukemia ◽  
Phase Iii ◽  
High Dose ◽  
Free Survival ◽  
Phase Ib ◽  
High Dose Methylprednisolone ◽  
Grade 3

Abstract High-dose methylprednisolone (HDMP) and rituximab (R) is an effective non-myelosuppressive treatment regimen for patients (pts) with chronic lymphocytic leukemia (CLL). Also, this combination has shown activity even in pts who have adverse leukemia-cytogenetics, such as del17p. Phase III studies have demonstrated that CLL pts treated with chlorambucil and obinutuzumab-Gazyva (G), another anti-CD20 mAb, had a superior outcome than comparable pts treated with R-chlorambucil. We hypothesized that G-HDMP is well-tolerated and effective in the treatment of pts with CLL. Accordingly, we initiated an open-label phase Ib/II clinical study. A total of 40 pts were enrolled in two cohorts of 20 pts each (previously untreated (PU) and relapsed/refractory (RR) CLL) and treated with HDMP 1 g/m2on Day 1-3 of cycles 1-4 (28 days/cycle) and G administered based on FDA dosing recommendations for 6 cycles. The pts had a median age of 67 years + 9.1 in the RR cohort and 63 years + 8.3 in the PU cohort. The median baseline absolute lymphocyte count was 30.7 + 7.3 x1,000/mm3 for pts in the RR cohort and 47.6 + 19.7 x1,000/mm3for pts in the PU cohort. Pts showed the following cytogenetic abnormalities: del(17p) in 30% RR vs. 0% PU, del(13q) in 60% RR vs. 70% PU, del(11q) in 20% RR vs. 35% PU, and trisomy 12 in 15% RR vs. 20% PU. Most AEs were grade 1-2 (RR=87%; PU=93%) without development of dose-limiting toxicities. Only two pts needed therapy discontinuation. One pt due to pulmonary embolism and the second pt due to asymptomatic gastrointestinal bleeding that required blood transfusion and resolved spontaneously. Grade 1-2 G-infusion-related reactions (IRR) were observed in 40% and 80% of pts in the RR and PU cohorts, respectively. Grade 3-4 IRR were observed in 10% of pts in the PU cohort only. We observed cytopenias (neutropenia grade 3-4: RR=55%, PU=40%; thrombocytopenia grade 3-4: RR=35%, PU=20%; and anemia grade 3-4: RR=0%, PU=0%). There were no cases of febrile neutropenia. Two pts (10%) in the RR cohort and one pt (5%) in the PU cohort developed infection grade 1-2 that was treated with oral antibiotics but did not require study treatment discontinuation. The most frequent non-hematological adverse events (AEs) were transaminitis, hyperglycemia, and electrolyte alterations (grade 1-2). There were no treatment related deaths in either cohort. The response assessment was performed in all 40 pts by iwCLL criteria. The ORR was 100% in the PU cohort and 95% in the RR cohort. 70% of the pts in the PU cohort and 85% of the pts in the RR cohort achieved a PR. CR was observed in 30% and 10% of the pts in the PU and RR cohorts, respectively. One pt (5%) in the RR cohort and four pts (20%) in the PU cohort achieved MRDneg status (<0.01% CLL in the bone marrow by multiparameter flow cytometry). Only one pt in the RR cohort achieved SD. At a median follow-up of 12.2 months, the RR cohort had a median Progression Free Survival (PFS) of 13.6 months and median Treatment Free Survival (TFS) of 14.7 months; the median Overall Survival (OS) has not been reached. In the PU cohort, the median PFS, TFS and OS have not been reached. One pt from the RR cohort and one pt from the PU cohort died during the follow-up period due to disease progression. G-HDMP was well tolerated and all 40 pts showed hematological and clinical responses during the study treatment without development of unexpected AEs. In both cohorts, most of IRR were grade 1-2 and severe IRR (grade 3-4) were much less compared with previously published data (G-chlorambucil / CLL-11 study). Compared to pts in the CLL-11 study, cytopenias appeared to be more frequent, however, the rate of infection and need for IV antibiotics or hospitalizations was lower. Of note, the eligibility criteria allowed pts with severe cytopenias and transfusion requirement to participate in our study. Response in PU pts were higher in terms of ORR, CR and CR-MRDnegativecompared with the data from the CLL-11 study and suggests a possible synergistic activity between G and HDMP. Overall, G-HDMP was well tolerated in the PU and RR CLL pts with a lower rate of IRR making this regimen more manageable in the outpatient setting. Responses were higher than previously reported in PU pts. Responses in RR pts appear to be comparable to our previous studies using R-HDMP. Our data supports G-HDMP as an alternative combination regimen for the treatment of CLL pts. Disclosures Kipps: Celgene: Consultancy, Honoraria, Research Funding; Pharmacyclics, LLC, an AbbVie Company: Consultancy, Honoraria; Gilead: Consultancy, Honoraria, Speakers Bureau; AbbVie: Consultancy, Honoraria, Research Funding; Roche: Consultancy, Honoraria.

Is neratinib following trastuzumab in early-stage HER2-positive breast cancer cost-effective?

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
Keyword(s):  
Breast Cancer ◽  
Cost Effectiveness ◽  
Clinical Benefit ◽  
Cost Effective ◽  
Phase Iii ◽  
Base Case ◽  
Recurrence Rates ◽  
Adjuvant Trastuzumab ◽  
Her2 Breast Cancer ◽  
Life Years

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]

Evaluating the cost-effectiveness of hydrogel spacer in radiotherapy (RT) of prostate cancer (PC).

2018 ◽  
Vol 36 (6_suppl) ◽  
pp. 43-43
Author(s):  
Rahul Ramesh Khairnar ◽  
Joseph Levy ◽  
Mark Mishra
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Intensity Modulated Radiation Therapy ◽  
Cost Effective ◽  
Credible Interval ◽  
Phase Iii ◽  
Incremental Effectiveness ◽  
Life Years ◽  
The Cost ◽  
Fee Schedule

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.

Cost-effectiveness of once weekly carfilzomib 70 mg/m2 plus dexamethasone in patients with relapsed and refractory multiple myeloma in the United States.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18356-e18356
Author(s):  
Shaji Kumar ◽  
Istvan Majer ◽  
Sumeet Panjabi ◽  
Jean Malacan ◽  
Rohan Medhekar ◽  
...  
Keyword(s):  
Health Care ◽  
Multiple Myeloma ◽  
Cost Effectiveness ◽  
Progression Free Survival ◽  
Cost Effective ◽  
The United States ◽  
Sensitivity Analyses ◽  
Refractory Multiple Myeloma ◽  
Life Years ◽  
Lifetime Costs

e18356 Background: Carfilzomib plus dexamethasone (Kd) dosed once weekly at 70 mg/m2 (QW Kd70) was recently approved in the US for treating patients with relapsed and refractory multiple myeloma (RRMM). To assess the cost-effectiveness (CE) of QW Kd70 vs twice weekly Kd dosed at 27 mg/m2 (BIW Kd27), data from the phase 3 ARROW trial, which directly compared these regimens in patients with 2-3 prior lines of therapy were used. Methods: A partitioned survival model was developed for the CE analysis. Time to treatment discontinuation, progression-free survival, and overall survival (OS) were estimated from the ARROW trial. Long-term OS was extrapolated using Surveillance Epidemiology and End Results registry data after matching characteristics of patients in the registry and ARROW trial. Direct costs were estimated from a US health care payer perspective. Utilities collected in the ARROW trial using the five-level version of the EuroQol questionnaire (EQ-5D-5L) were applied to estimate the quality-adjusted life years (QALYs). Uncertainty was explored using sensitivity analyses. Two subgroups of patients refractory to lenalidomide or bortezomib were assessed. Main outcomes were mean life-years (LYs), QALYs, lifetime costs, and incremental cost-effectiveness ratios (ICERs). Results: For QW Kd70 and BIW Kd27, the model predicted mean LYs of 4.17 and 3.07 years, QALYs of 2.98 and 2.03 years, and mean total lifetime costs of $444,563 and $373,364, respectively. The incremental LYs gain, QALY gain, and incremental costs of QW Kd70 vs BIW Kd27 were estimated to be 1.10 years, 0.95 year, and $71,199, respectively, resulting in an ICER of $64,595 per LY gained and $75,204 per QALY gained. For patients refractory to lenalidomide and bortezomib, similar results were found with ICERs of $79,988 and $76,793, respectively. Conclusions: In line with ARROW trial results, this CE analysis showed that QW Kd70 is expected to provide considerable additional benefit in terms of LYs and QALYs gained compared with BIW Kd27. In the RRMM setting, QW Kd70 is cost-effective with ICERs below accepted willingness to pay thresholds in US and represents an efficient utilization of the health care budget.

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