Cost-effectiveness (CE) analysis of pembrolizumab as first-line (1L) treatment for recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) in the United States (US).

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18013-e18013
Author(s):  
Karthik Ramakrishnan ◽  
Disha Jain ◽  
Jyotika Gandhi ◽  
Diana Romana Chirovsky ◽  
Rebekah Borse

e18013 Background: Pembrolizumab received FDA approval in 1L R/M HNSCC as monotherapy (P) in patients with combined positive score (CPS) ≥1, and in combination with platinum + 5-Fluorouracil (5-FU) chemotherapy (P + C) in the total population, based on KEYNOTE 048 (KN048) trial. Taking a US payer perspective, we evaluated the CE of P and P + C versus KN048 trial comparator cetuximab + platinum + 5-FU (EXTREME regimen), and other comparators commonly used in the US (i.e. cisplatin+docetaxel+cetuximab [TPEx regimen], platinum + paclitaxel/taxane [Pt + T] and platinum+5-FU [Pt + F]). Methods: A 3-state partitioned-survival model was used to project costs and outcomes over 20 years with a 3% annual discount rate. For P, P + C and EXTREME, health state occupancy was based on KN048 trial results with long-term parametric extrapolation of progression-free survival (PFS) and overall survival (OS) guided by statistical criteria. A network meta-analysis (NMA) was applied to the P and P + C interventions in KN048 to project the PFS and OS outcomes for other comparators. The time on treatment (ToT) for P, P+C and EXTREME was derived from the KN048 trial, whereas for other comparators, ToT was approximated by PFS. Costs of 1L and subsequent treatments, disease management, adverse event, and terminal care were included. Utilities were derived using the EuroQoL five-dimension data from KN048 and a US value set. Results: CE ratios, total and incremental costs and quality-adjusted life years (QALYs) from the base case analysis are summarized in the table below. P was more effective and less costly (i.e. dominant) versus EXTREME and TPEx. P + C was highly cost-effective versus EXTREME ($1,789/QALY) and TPEx regimen (dominant). CE ratios for both P and P + C versus all comparators were below a $100,000/QALY willingness-to-pay threshold. Probabilistic sensitivity analysis also showed a 100% CE probability for P and P + C at the $100,000/QALY threshold. Conclusions: P and P + C are cost-effective treatment options in the US with CE ratios below $100,000/QALY gained versus commonly used therapies in 1L R/M HNSCC.[Table: see text]

2021 ◽  
Vol 12 ◽  
Author(s):  
Jigang Chen ◽  
Xin Tong ◽  
Mingyang Han ◽  
Songfeng Zhao ◽  
Linjin Ji ◽  
...  

Background: Glioblastoma multiforme (GBM) is a fatal type of brain tumor with a high incidence among elderly people. Temozolomide (TMZ) has proven to be an effective chemotherapeutic agent with significant survival benefits. This study aimed to evaluate the economic outcomes of radiotherapy (RT) and TMZ for the treatment of newly diagnosed GBM in elderly people in the United States (US) and China.Methods: A partitioned survival model was constructed for RT plus TMZ and RT alone among patients with methylated and unmethylated tumor status. Base case calculations and one-way and probabilistic sensitivity analyses were performed. Life-years, quality-adjusted life-years (QALYs), costs (in 2021 US dollars [$] and Chinese Yuan Renminbi [¥]), and incremental cost-effectiveness ratios (ICERs) were calculated.Results: RT plus TMZ was found to be associated with significantly higher costs and QALYs in all groups. Only US patients with methylated status receiving RT plus TMZ had an ICER ($89358.51) less than the willingness-to-pay (WTP) threshold of $100000 per QALY gained when compared with receiving RT alone. When the WTP threshold ranged from $100000 to $150000 from the US perspective, the probability of RT plus TMZ being cost-effective increased from 80.5 to 99.8%. The cost of TMZ must be lower than ¥120 per 20 mg for RT plus TMZ to be cost-effective among patients with methylated tumor status in China.Conclusion: RT plus TMZ was not cost-effective in China, and a reduction in the TMZ price was justified. However, it is highly likely to be cost-effective for patients with methylated tumor status in the US.


2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18356-e18356
Author(s):  
Shaji Kumar ◽  
Istvan Majer ◽  
Sumeet Panjabi ◽  
Jean Malacan ◽  
Rohan Medhekar ◽  
...  

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.


2021 ◽  
Author(s):  
Ronald Chow ◽  
Elizabeth Horn Prsic ◽  
Hyun Joon Shin

Introduction: A recent systematic review and meta-analysis by our group reported on thirteen published cohorts investigating 110,078 patients. Patients administered statins after their COVID-19 diagnosis and hospitalization were found to have a lower risk of mortality. Given this reported superiority, a logical next question would be whether statins are cost-effective treatment options for hospitalized COVID-19 patients. In this paper, we report on a cost-effectiveness analysis of statin-containing treatment regimens for hospitalized COVID-19 patients, from a United States healthcare perspective. Methods: A Markov model was used, to compare statin use and no statin use among hospitalized COVID-19 patients. The cycle length was one week, with a time horizon of 4 weeks. A Monte Carlo microsimulation, with 20,000 samples were used. All analyses were conducted using TreeAge Pro Healthcare Version 2021 R1.1. Results: Treatment of hospitalized COVID-19 patients with statins was both cheaper and more effective than treatment without statins; statin-containing therapy dominates over non-statin therapy. Conclusion: Statin for treatment of COVID-19 should be further investigated in RCTs, especially considering its cost-effective nature. Optimistically and pending the results of future RCTs, statins may also be used broadly for treatment of hospitalized COVID-19 patients.


2017 ◽  
Vol 24 (1) ◽  
pp. 6 ◽  
Author(s):  
V. Babashov ◽  
M.A. Begen ◽  
J. Mangel ◽  
G.S. Zaric

Background We conducted a cost-effectiveness analysis of brentuximab vedotin for the treatment of relapsed and refractory Hodgkin lymphoma (hl) in the post–autologous stem-cell transplantation (asct) failure period, from the perspective of the Canadian health care payer.Methods We developed a decision-analytic model to simulate lifetime costs and benefits of brentuximab vedotin compared with best supportive care for the treatment of patients with hl after failure of asct. Administrative data from Ontario were used to set the model parameters.Results In the base case, treatment with brentuximab vedotin resulted in incremental quality-adjusted life-years (qalys) of 0.544 and an incremental cost of $89,366 per patient, corresponding to an incremental cost-effectiveness ratio (icer) of $164,248 per qaly gained. The icer was sensitive to the cost of brentuximab vedotin, the hazard ratio used to assess the efficacy of brentuximab vedotin treatment, and health state utilities.Conclusions In light of the available information, brentuximab vedotin has an icer exceeding $100,000 per qaly gained, which is a level often classified as having “weak evidence for adoption and appropriate utilization” in Canada. However, it is worth noting that provincial cancer agencies take into account not only the costs and associated icer, but also other factors such as a lack of alternative treatment options and the clinical benefits of expensive cancer drugs. Pricing arrangements should be negotiated, and risk-sharing agreements or patient access schemes should be explored. 


Circulation ◽  
2020 ◽  
Vol 141 (15) ◽  
pp. 1214-1224 ◽  
Author(s):  
Dhruv S. Kazi ◽  
Brandon K. Bellows ◽  
Suzanne J. Baron ◽  
Changyu Shen ◽  
David J. Cohen ◽  
...  

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.


2019 ◽  
Vol 22 (4) ◽  
pp. 142-144 ◽  
Author(s):  
David Aceituno ◽  
Mark Pennington ◽  
Barbara Iruretagoyena ◽  
Matthew A Prina ◽  
Paul McCrone

IntroductionCost-effectiveness analyses that use quality-adjusted life-years (QALYs) allow comparing the value for money of interventions across different health problems. Health state utility values (HSUVs) are crucial to calculate QALYs. These are weights attached to a given health state reflecting preferences in health-related quality of life (HRQoL). In schizophrenia, there is extensive evidence about the consequences of this condition on HRQoL. Besides, several interventions have claimed to be cost-effective in terms of QALYs gained. Despite this evidence, a systematic review of HSUVs has not been conducted. Therefore, we aim to synthesise the evidence about HSUVs in schizophrenia.Methods and analysisWe will conduct a systematic review of the literature about HSUVs in people with schizophrenia following the Preferred Reporting Items for Systematic review and Meta-Analysis and the International Society for Pharmacoeconomics and Outcomes Research task force recommendations. The submissions records of eight electronic peer-reviewed databases and three health technology assessment (HTA) agencies will be searched. Quantitative synthesis will be carried out in comparable studies, using random-effects meta-analysis. Heterogeneity will be explored using meta-regression if more than 10 studies per covariate are found. A narrative synthesis and methodological quality of included studies will be also reported.DiscussionThis review will provide a synthesis of the HSUVs estimated for different states experienced by people with schizophrenia. This will inform analysts when calculating QALYs, using values in a more transparent and accountable manner. Finally, it will shed light on evidence gaps and limitations about this measure in mental health.PROSPERO registration numberCRD42019123582.


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.


10.36469/9829 ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. 90-102
Author(s):  
Louise Perrault ◽  
Dilip Makhija ◽  
Idal Beer ◽  
Suzanne Laplante ◽  
Sergio Iannazzo ◽  
...  

Background: Patients developing acute kidney injury (AKI) during critical illness or major surgery are at risk for renal sequelae such as costly and invasive acute renal replacement therapy (RRT) and chronic dialysis (CD). Rates of renal injury may be reduced with use of chloride-restrictive intravenous (IV) resuscitation fluids instead of chloride-liberal fluids. Objectives: To compare the cost-effectiveness of chloride-restrictive versus chloride-liberal crystalloid fluids used during fluid resuscitation or for the maintenance of hydration among patients hospitalized in the US for critical illnesses or major surgery. Methods: Clinical outcomes and costs for a simulated patient cohort (starting age 60 years) receiving either chloride-restrictive or chloride-liberal crystalloids were estimated using a decision tree for the first 90-day period after IV fluid initiation followed by a Markov model over the remainder of the cohort lifespan. Outcomes modeled in the decision tree were AKI development, recovery from AKI, progression to acute RRT, progression to CD, and death. Health states included in the Markov model were dialysis free without prior AKI, dialysis-free following AKI, CD, and death. Estimates of clinical parameters were taken from a recent meta-analysis, other published studies, and the US Renal Data System. Direct healthcare costs (in 2015 USD) were included for IV fluids, RRT, and CD. US-normalized health-state utilities were used to calculate quality-adjusted life years (QALYs). Results: In the cohort of 100 patients, AKI was predicted to develop in the first 90 days in 36 patients receiving chloride-liberal crystalloids versus 22 receiving chloride-restrictive crystalloids. Higher costs of chloride-restrictive crystalloids were offset by savings from avoided renal adverse events. Chloride-liberal crystalloids were dominant over chloride-restrictive crystalloids, gaining 93.5 life-years and 81.4 QALYs while saving $298 576 over the cohort lifespan. One-way sensitivity analyses indicated results were most sensitive to the relative risk for AKI development and relatively insensitive to fluid cost. In probabilistic sensitivity analyses with 1000 iterations, chloride-restrictive crystalloids were dominant in 94.7% of iterations, with incremental cost-effectiveness ratios below $50 000/QALY in 99.6%. Conclusions: This analysis predicts improved patient survival and fewer renal complications with chloriderestrictive IV fluids, yielding net savings versus chloride-liberal fluids. Results require confirmation in adequately powered head-to-head randomized trials.


2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 8030-8030 ◽  
Author(s):  
Nimer Alsaid ◽  
Ali McBride ◽  
Amit Balkrishna Agarwal ◽  
Abdulaali Mutairi ◽  
Faiz Anwer ◽  
...  

8030 Background: CAR, IXA, ELO, and DAR in triplet combination with LEN+DEX have shown superior efficacy over LEN+DEX in R/R MM, but their comparative efficacy and cost effectiveness has not been estimated. Methods: Network meta-analysis [NMA] and Bücher method were used to indirectly estimate comparative progression-free survival (PFS) efficacy. A 2-state Markov model (progression-free, progressed or death) was specified. Inputs included: cost of chemotherapy, administration, adverse events (AE) management, disease monitoring; utilities for health states; and disutilities for AEs. Incremental cost effectiveness (ICER) and cost utility ratios (ICUR) were calculated for resp. PFS life years (PFS LY) and quality adjusted life years (PFS QALY) gained in base case and probabilistic sensitivity analyses (PSA). Results: NMA and Bücher indirect comparison methods yielded similar PFS hazard ratios (HR), revealing superiority of DAR+LEN+DEX over other triplets in terms of PFS (Table). Using the exponential distribution to fit PFS data, our cost effectiveness analysis indicated that all 4 triplet regimens were associated with additional PFS LY and QALY gained over LEN+DEX at additional cost. DAR+LEN+DEX was associated with the greatest PFS LY and QALY gained at the lowest relative cost, yielding superior ICER and ICUR estimates compared to other triplet regimens. Conclusions: The superior PFS efficacy of DAR+LEN+DEX is associated with positive cost effectiveness and cost utility in the setting of R/R MM. [Table: see text]


2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S387-S387
Author(s):  
A FISCHER ◽  
M Oppe ◽  
S Stypa ◽  
V Lukyanov ◽  
I Petrakis

Abstract Background Vedolizumab intravenous (IV), a gut selective humanised monoclonal antibody, is indicated for the management of moderately to severely active Ulcerative Colitis (UC) and has been shown in the only head-to-head clinical trial within UC to be superior to adalimumab (NCT02497469). Furthermore, the novel vedolizumab subcutaneous (SC) has recently been proven to be an effective treatment of UC (NCT02611830). The objective of this study is to estimate the comparative cost-effectiveness of vedolizumab IV with updated efficacy data, and vedolizumab SC for the first time. Both vedolizumab IV and SC have been compared with other publicly reimbursed biologics for the treatment of patients with moderate-to-severe UC from a Canadian public healthcare payer perspective. Methods A hybrid decision tree/Markov model was developed to simulate the clinical course of UC, translating the disease course into costs and quality-adjusted life-years (QALYs). Comparative efficacy of vedolizumab SC, vedolizumab IV, adalimumab, infliximab IV, and golimumab were sourced from a network meta-analysis. Drug and disease management costs (2019 $CAD) were sourced from Ontario public payer schedules of benefits. Utilities were sourced from the literature. Clinical and economic outcomes were projected over a lifetime and discounted at 1.5% annually. Results Within a mixed bio-naïve/experienced population, vedolizumab SC resulted in slightly more QALYs than vedolizumab IV and dominated adalimumab (Table 1); vedolizumab SC yielded an incremental cost-effectiveness ratio (ICER) of $CAD 6,727 per QALY and $CAD 52,673 per QALY relative to golimumab and to infliximab (for which the price of a biosimilar was used), respectively. Further scenario analysis within bio-naïve populations supported the robustness of the base-case results, demonstrating that vedolizumab SC or vedolizumab IV were either dominant or cost-effective across all scenarios. Conclusion Our analysis suggests that vedolizumab SC and vedolizumab IV are a cost-effective therapeutic alternative relative to other biologics for moderate-to-severe UC in Canada. Future research will expand the analysis across all biologic comparators as they are used in the real world.


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