The Cost-Effectiveness of Bortezomib for the Initial Treatment of Multiple Myeloma in the United States.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 1379-1379
Author(s):  
Si-Tien Wang ◽  
Hui Huang ◽  
Hongliang Shi ◽  
Mei Sheng Duh ◽  
Kristina Chen ◽  
...  

Abstract Abstract 1379 Poster Board I-401 Background: Data from the phase III VISTA trial demonstrated superiority in terms of clinical effectiveness of bortezomib (Velcadë) plus melphalan and prednisone (VMP) relative to melphalan and prednisone alone (MP) in the initial treatment of patients with multiple myeloma (MM). The aim of this study was to utilize data from the VISTA study and published literature to compare lifetime health outcomes and the cost-effectiveness of these regimens as induction therapy for MM patients ineligible for autologous stem cell transplantation (ASCT). An indirect comparison of VMP versus thalidomide plus MP (MPT) was also conducted using published results from the IFM 99-06 clinical trial for MPT (Facon et al, Lancet 2007). The goal of this study and the derived model was to assess the relative costs and outcomes from these two trials, recognizing the limitations imposed by using data derived from independent studies. Methods: A Markov model from the US payer's perspective was developed. Simulations were performed for hypothetical cohorts of newly diagnosed MM patients with an average age of 70 years at treatment initiation and who were not eligible for ASCT. The model includes seven health states representing periods of treatment response (stable disease/minimal response, partial response, or complete response), treatment-free interval, progressive disease, second-line treatment and death. Monthly transition probabilities were estimated from patient-level VISTA trial data for VMP and MP (with a data cut-off of June 15, 2007), and from the published phase lll IFM 99-06 trial for MPT. Costs included per-protocol drug and medical costs, treatment-related adverse events, second-line treatment, and resource utilization during treatment-free interval and progressive disease. Unit costs of medications and resources were obtained from published literature. All costs were adjusted to 2009 US dollars. State-specific utility estimates were derived from patient-level EQ-5D data from the VISTA trial using US-specific weights. Health outcomes were expressed in life-years (LYs) and quality-adjusted life-years (QALYs). Both cost and health outcomes were discounted at 3%. Incremental cost-effectiveness ratios (ICERs) were calculated for VMP versus MP, and VMP versus MPT, over a lifetime horizon (approximated by 20 years). One-way sensitivity analyses were conducted by running the model with upper and lower values of key parameters to assess the general robustness of model findings and identify key drivers. Results: Model base case results for the incremental cost-effectiveness of VMP relative to MP and MPT are shown in the Table. Comparison of the model's overall survival (OS) projections with the observed differences indicates a conservative approximation of the treatment differences for VMP. The estimated OS was 4.187 years with VMP versus 2.864 years with MP and versus 4.140 years with MPT over a lifetime horizon. Lifetime direct medical costs range from $57,864 for MP to $129,902 for MPT. The cost per LY and QALY gained with VMP compared with MP is $40,051 and $56,109, respectively. VMP is dominant (cost saving and better outcomes) compared with MPT, costing 17.7% less and providing slightly more QALYs on average. One-way sensitivity analyses suggest general robustness of model findings and the key drivers include VMP/MP hazard ratio from second-line treatment to death, and the MPT/MP hazard ratio for treatment discontinuation. Conclusions: In newly diagnosed MM patients ineligible for ASCT, VMP is projected to improve long-term health outcomes, offering a substantial survival benefit compared with MP. The incremental cost-effectiveness of VMP versus MP is within the generally accepted cost-effectiveness range of $50,000 to $100,000 per QALY, suggesting that VMP is cost-effective compared with MP in the United States. Within this cost-effectiveness model, compared with MPT, VMP is dominant, yielding lower costs and better health outcomes. Disclosures: Wang: Milllennium: Research Funding. Huang: Milllennium: Employment, Equity Ownership. Shi: Millennium Pharmaceuticals, Inc.: Employment. Duh: Milllennium: Consultancy, Research Funding. Chen: Milllennium: Research Funding. Chang: Milllennium: Research Funding. Korves: Milllennium: Research Funding. Dhawan: Johnson and Johnson Research Pharmaceuticals: Employment. Cakana: Johnson & Johnson: Employment, Equity Ownership. van de Velde: Johnson & Johnson: Employment, Equity Ownership. Esseltine: Milllennium: Employment, Equity Ownership. Garrison: Milllennium: Consultancy.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2563-2563 ◽  
Author(s):  
Si-Tien Wang ◽  
Hui Huang ◽  
Abbie Ba-Mancini ◽  
Hongliang Shi ◽  
Kristina Chen ◽  
...  

Abstract Abstract 2563 Background: Results from the phase 3 VISTA trial demonstrated that bortezomib (Velcade®) plus melphalan and prednisone (VMP) has superior efficacy to MP alone in patients with newly diagnosed multiple myeloma (MM) ineligible for ASCT. In a similar patient population, the three-arm phase 3 MM-015 trial compared lenalidomide (Revlimid®) plus MP with or without continuous lenalidomide maintenance treatment after cycle 9 (MPR-R vs MPR) with MP alone. There was no difference in progression-free (PFS) or overall survival (OS) between patients who received MPR or MP during the 9-cycle induction period; MPR-R was associated with superior PFS vs MP during the R maintenance period. To assess the value of R in combination with MP followed by R maintenance therapy we indirectly estimated the incremental cost-effectiveness of VMP vs MPR-R as therapy for MM patients ineligible for ASCT. Methods: An Excel-based Markov model from the US payer's perspective was developed. Simulations were performed for hypothetical cohorts of newly diagnosed MM patients ineligible for ASCT with an average age of 70 years at treatment initiation. The model includes seven health states representing periods of treatment response (stable disease/minimal response, partial response, and complete response), treatment-free interval/maintenance treatment, progressive disease, second-line treatment, and death. Monthly transition probabilities were estimated from patient-level data for VISTA for VMP and MP (data cut-off June 15 2007; San Miguel et al NEJM 2008) and published results for MM-015 for MPR-R (data cut-off April 15 2009; Palumbo et al ASH 2009). As previously reported (Wang et al ASH 2009), costs included per-protocol drug and medical costs, treatment-related adverse event costs, second-line treatment costs, and resource utilization during the treatment-free interval and progressive disease; all costs were adjusted to 2010 US dollars. State-specific utility estimates were derived from patient-level EQ-5D data from the VISTA trial using US-specific weights. Health outcomes (as indicators of the effectiveness of therapy) were expressed in life years (LYs) and quality-adjusted LYs (QALYs). Costs and health outcomes were discounted at 3%. Incremental cost-effectiveness ratios (ICERs) were calculated for VMP vs MPR-R over a lifetime horizon (20 years). In the base case, the MPR-R vs MP hazard ratio (HR) for PFS was set to 0.499 and that for OS was set to 1, due to a lack of survival benefit with MPR-R vs MP observed in MM-015. One-way sensitivity analyses were conducted by running the model with upper and lower values of key parameters to assess the general robustness of model findings and identify key drivers. Results: Model base case results for the incremental cost-effectiveness of VMP relative to MPR-R are shown in the Table. Estimated OS was 4.187 years with VMP vs 3.409 years with MPR-R over a lifetime horizon. Lifetime direct medical costs were $119,102 with VMP vs $241,247 with MPR-R; the lifetime cost of R maintenance was $107,047. Thus, VMP appears associated with reduced costs and better outcomes vs MPR-R; VMP costs approximately 50% less than MPR-R and seems to provide slightly more QALYs (0.567) on average. One-way sensitivity analyses supported the general robustness of model findings and identified the MPR-R vs MP HR for OS as a key driver; only when this HR was set to ≤0.25 did MPR-R become cost-effective vs VMP at $100,000 per QALY. Conclusions: In newly diagnosed MM patients ineligible for ASCT, VMP appears to be associated with lower costs and better health outcomes vs MPR-R. From a cost-effectiveness perspective, R maintenance therapy therefore seems to have little benefit in this patient population. The current comparison was based on published results for MPR-R from MM-015 after a median follow-up of 9.4 months (vs 16.3 months for VMP vs MP in VISTA), at which time no survival benefit was observed for MPR-R vs MP. If longer follow-up in MM-015 indicates a survival benefit for MPR-R vs MP, a re-estimation of the incremental cost-effectiveness of VMP vs MPR-R using the present Markov model would be warranted; however, updated data (Palumbo et al EHA 2010) show no significant difference in OS between the MPR-R and MP arms at a median follow-up of 21 months. Disclosure: Wang: Millennium Pharmaceuticals: Consultancy, Research Funding. Huang:Millennium Pharmaceuticals, Inc: Employment. Ba-Mancini:Millennium Pharmaceuticals, Inc.: Employment. Shi:Millennium Pharmaceuticals, Inc: Employment. Chen:Millennium Pharmaceuticals: Consultancy, Research Funding. Korves:Millennium Pharmaceuticals, Inc: Research Funding. Dhawan:Johnson & Johnson: Employment, Equity Ownership. Cakana:Johnson & Johnson: Employment, Equity Ownership. van de Velde:Johnson & Johnson: Employment, Equity Ownership. Esseltine:Millennium Pharmaceuticals, Inc.: Employment; Johnson & Johnson: Equity Ownership. Duh:Millennium Pharmaceuticals, Inc: Consultancy, Research Funding.


Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5604-5604 ◽  
Author(s):  
Gerry Oster ◽  
Ariel Berger ◽  
Rebecca Bornheimer ◽  
Gary Binder ◽  
Yasir Nagarwala

Abstract Introduction The efficacy and safety of the novel agents, lenalidomide and bortezomib, in previously untreated MM has been demonstrated in several randomized controlled trials (Online-OnlyTs). In this study, we examine the cost-effectiveness of lenalidomide-melphalan–prednisone induction followed by lenalidomide maintenance (MPR-R), and bortezomib-melphalan-prednisone (VMP), respectively, versus melphalan-prednisone (MP), in patients with previously untreated MM. Methods We developed a partitioned-survival model to estimate expected clinical outcomes and costs in newly diagnosed MM patients receiving MPR-R, VMP, or MP as first-line therapy. The model had 3 mutually exclusive health states: (1) “progression-free, alive”; (2) “post-progression, alive”; and (3) “death.”  Progression-free survival (PFS) for MP was estimated by aggregating data across five Online-OnlyTs (Palumbo 2012, San Miguel 2008, Facon 2007, Hulin 2009, Palumbo 2006). Estimates of PFS for MPR-R and VMP were based on an adjusted indirect treatment comparison with MP, using data from MM-015 for MPR-R (Palumbo 2012) and VISTA for VMP (San Miguel 2008). Since many MP patients in both MM-015 and VISTA “crossed over” to lenalidomide and bortezomib following disease progression in these trials, we estimated post-progression survival (PPS) for MPR-R and VMP based on a review of novel agents in MM (Messori 2011), which reported mean PPS of 30.9 months. Costs of MPR-R and VMP were estimated based on actual use of study drug in Online-OnlyTs; costs of adverse events as well as other disease-related costs were estimated based on published data. Health-state utilities also were estimated using published data.  All costs were expressed in 2012 US$.  Cost-effectiveness of MPR-R and VMP versus MP was examined in terms of cost per life-year (LY) gained, cost per quality-adjusted life-year (QALY) gained, and cost per progression-free life-year gained.  Future costs and benefits were discounted at 3% annually. Results Mean estimated PFS was 3.4 years for MPR-R, 2.6 years for VMP, and 1.7 years for MP; corresponding estimates for OS were 6.0 years, 5.2 years, and 4.3 years, respectively (Table 1). Mean total expected lifetime costs (discounted) are reported in the Table. The incremental cost per life-year (LY) gained versus MP was $75,392 for MPR-R and $86,213 for VMP; corresponding estimates of the incremental cost per QALY gained were $91,794 and $106,211, respectively (Figure 1).  The incremental cost per progression-free LY (PFLY) gained versus MP was $70,666 for MPR-R and $80,565 for VMP. Conclusions In patients with previously untreated MM, cost-effectiveness ratios for MPR-R and VMP are well within the range reported for other well-accepted novel therapies in oncology. $/LY: Incremental cost per life-year gained; $/QALY: Incremental cost per quality-adjusted life-year gained; $/PFLY: Incremental cost per progression-free life-year gained Support Funded by Celgene Corporation Disclosures: Oster: Celgene: Research Funding. Off Label Use: Lenalidomide (immunomodulatory agent), bortezomib (proteosome inhibitor), melphalan (alkylator), and prednisone (steroid), are all treatments for multiple myeloma. Berger:Celgene: Research Funding. Bornheimer:Celgene: Research Funding. Binder:Celgene: Employment, Equity Ownership. Nagarwala:Celgene: Employment, Equity Ownership.


Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3122-3122
Author(s):  
Salmon Manier ◽  
Meletios A. Dimopoulos ◽  
Cyrille Hulin ◽  
Xavier Leleu ◽  
Michel Delforge ◽  
...  

BACKGROUND: Rd continuous is now a standard treatment option in patients (pts) with transplant-ineligible NDMM based on the results of the large phase 3 registrational FIRST trial showing significant PFS and OS benefits of lenalidomide (LEN) + low-dose dexamethasone until disease progression (Rd continuous) vs melphalan + prednisone + thalidomide (MPT). However, pts who relapse on frontline LEN represent a growing population for whom there is limited clinical evidence on subsequent treatment. Factors associated with relapse within 12 mos in Rd- and MPT-treated pts (LDH ≥ 200 U/L, ISS stage III, high-risk cytogenetics, ECOG PS ≥ 2, and baseline platelet count ≤ 150 × 109/L) indicate that the individual risk of progression may involve a combination of disease biology, genetics, and pt-specific factors (Facon, EHA 2019). Physicians may consider these early relapse factors when making treatment decisions. To characterize pts relapsing while receiving frontline LEN, this analysis evaluated Rd-treated pts from the FIRST trial who progressed while receiving LEN based on their time of relapse, type of relapse (CRAB [symptomatic] vs non-CRAB [nonsymptomatic]), and second-line treatment. METHODS: Pts randomized to Rd continuous (n = 535) or Rd for 18 cycles (n = 541) arms and who progressed while receiving LEN or within 60 days of treatment end (per IMWG criteria) were pooled and grouped according to their time of relapse after randomization (< 12 mos = early< 12; 12-18 mos = early12-18, and > 18 mos = late relapse). The data cutoff was January 2016. RESULTS: Of the 389 pts who relapsed, 203 had early< 12 relapse, 69 had early12-18 relapse, and 117 had late relapse. Pts who relapsed within the first 12 mos or between 12 and 18 mos had higher rates of elevated LDH, ISS Stage III disease, and ECOG PS 2 compared with those who relapsed beyond 18 mos (Table). Early< 12 and early12-18 relapses were also associated with poor PFS and OS outcomes. Median PFS in pts with early< 12, early12-18, and late relapse was 6.5, 15.9, and 26.4 mos, and median OS was 26.8, 41.6, and 78.0 mos, respectively (Figure). Pts with late relapse had more dose reductions prior to PD vs those with early relapse and were more likely to experience nonsymptomatic progression. Across the 3 groups, in pts experiencing a nonsymptomatic progression who started second-line treatment, 88% started second-line treatment prior to experiencing CRAB symptoms. The median time from PD to second-line treatment was shorter in those with CRAB relapse (1.4-2.5 mos) vs non-CRAB relapse (2.5-5.9 mos). Second-line treatment was reported in 170 (83.7%), 62 (89.9%), and 99 (84.6%) pts with early< 12, early12-18, and late relapse, respectively. The majority of these pts received bortezomib-based regimens (65.6% overall), most commonly bortezomib + dexamethasone, bortezomib + melphalan + prednisone, or bortezomib + alkylator (cyclophosphamide or bendamustine). Second PFS was similar regardless of time of relapse. CONCLUSIONS: This analysis focused on an emerging and clinically relevant population of transplant-ineligible pts relapsing on frontline LEN. Pts with late relapse on LEN more commonly had nonsymptomatic progression vs pts relapsing early. This raises the question on the possible impact of the immune stimulatory effects of LEN in pts able to remain on therapy and achieve a longer OS and warrants further investigation of the biological impact of IMiD agent-based therapies on long-term disease control. The increase in dose reductions in the late- vs early-relapse groups is also an important consideration for continuous treatment. Consistent with the prior analysis of the FIRST study exploring Rd and MPT-treated pts with early< 12 relapse (Facon, EHA), pts relapsing on LEN within 12 mos and between 12-18 mos both appeared to have functionally high-risk disease and represent a population with an unmet need that should be considered for inclusion in clinical trials investigating new therapeutic strategies. Due to the timing of the FIRST trial, limited novel-novel agent combination treatments were available at relapse, and the majority of pts received bortezomib-based second-line regimens. These results highlight the need for effective therapies for pts with early relapse. Further characterization of first- and second-line outcomes according to type of relapse are ongoing and will be reported at the meeting. Disclosures Dimopoulos: Sanofi Oncology: Research Funding. Hulin:celgene: Consultancy, Honoraria; Janssen, AbbVie, Celgene, Amgen: Honoraria. Leleu:Sanofi: Honoraria; Takeda: Honoraria; Oncopeptide: Honoraria; Karyopharm: Honoraria; Amgen: Honoraria; Carsgen: Honoraria; Incyte: Honoraria; Novartis: Honoraria; Janssen: Honoraria; Celgene: Honoraria; BMS: Honoraria; Merck: Honoraria. Delforge:Amgen, Celgene, Janssen , Takeda: Honoraria. Weisel:Adaptive Biotech: Consultancy, Honoraria; Celgene Corporation: Consultancy, Honoraria, Research Funding; Takeda: Consultancy, Honoraria; Juno: Consultancy; Janssen: Consultancy, Honoraria, Research Funding; Bristol-Myers Squibb: Consultancy, Honoraria; Amgen: Consultancy, Honoraria, Research Funding; GSK: Honoraria; Sanofi: Consultancy, Honoraria, Research Funding. Srinivasan:Celgene: Employment, Equity Ownership. Costa:Celgene: Employment, Equity Ownership. Robinson:Celgene: Employment, Equity Ownership. Facon:Celgene: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Takeda: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen: Membership on an entity's Board of Directors or advisory committees; Sanofi: Membership on an entity's Board of Directors or advisory committees.


Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3555-3555 ◽  
Author(s):  
Carin A. Uyl-de Groot ◽  
Rachel Ramsden ◽  
Janneke Boersma ◽  
Sonja Zweegman ◽  
Sujith Dhanasiri

Abstract Background: Standard of care for patients with newly diagnosed multiple myeloma (NDMM) who are ≤ 70 years of age and who are fit is induction therapy followed by high-dose chemotherapy and autologous stem cell transplantation (ASCT). However, this approach is not curative, and residual disease leads to disease relapse. Sustaining response and postponing relapse following ASCT is an important clinical goal, as early progression is associated with an increased risk of death. Lenalidomide (LEN) maintenance therapy has emerged as an important standard of care post-ASCT. Several clinical studies have shown that patients who received LEN maintenance therapy after ASCT had significantly longer progression-free survival (PFS) and overall survival (OS) in comparison with those who did not receive maintenance therapy. Other studies have indicated that active LEN maintenance treatment does not impair quality of life (Tay J, et al. Blood. 2017;130:abstract 2150). A recent EU5 cost-impact analysis suggested that LEN maintenance is potentially cost saving on direct medical costs by 24% over a 5-year period (Jackson G, et al. Blood. 2017;130:abstract 3405). To date, no study has specifically assessed the cost-effectiveness of LEN as maintenance treatment. Aims: To assess the cost-effectiveness of LEN treatment versus no maintenance treatment in transplant-eligible NDMM patients from a Dutch healthcare service perspective. Methods: A partitioned survival model structure was selected to provide a good fit to the supporting efficacy and safety data. The model was structured around 3 primary health states relevant to an NDMM patient's treatment trajectory: pre-progression state (encompassing on- and off-treatment periods), post-progression state (encompassing periods just prior to second-line treatment, on second-line treatment, and post-second-line treatment), and death. Efficacy and safety data were taken from a pooled meta-analysis of the CALGB 100104, GIMEMA RV-MM-PI-209, and IFM 2005-02 studies. Parametric models were used to estimate long-term survival. Utility data were applied from a real-world setting captured in the Connect® MM Disease Registry, which was used to calculate the progression-free (LEN), progression-free (no treatment), progressive disease (treatment-free), and progressive disease (second-line treatment) utilities. A 21 out of 28-day cycles dosing regimen for LEN was applied as recommended in the Dutch HOVON clinical guidelines. Costs (2016) and subsequent therapy data were derived from published literature and sources appropriate for the Dutch market. All drug costs are presented at list price. Healthcare resource utilization was informed from a EU5 (France, Germany, Italy, Spain, and the UK) real-world study (Ashcroft J, et al. Int J Hematol Oncol. 2018;Epub ahead of print). The total costs, life years gained (LYG) and quality-adjusted life years (QALYs) were estimated over a lifetime horizon. Multiple scenario and sensitivity analyses were conducted to test the robustness of the model results to key assumptions and data inputs. Results: The cost-effectiveness model predicted a QALY gain of 2.26 and a LYG of 2.79 for LEN in the base case analysis (Table). First-line drug costs of LEN contributed to an increase of EUR 147,707 in total costs versus no maintenance treatment. However, this was partially offset by savings of EUR 77,462 in subsequent treatment costs. LEN was shown to represent a cost-effective use of resources when compared with the Dutch willingness-to-pay (WTP) threshold for NDMM of EUR 50,000/QALY. Scenario analyses showed LEN remained cost-effective in settings representative of Dutch clinical practice. For instance, use of only the Dutch recommended dose (10 mg with dose reductions if needed) of LEN for NDMM gave an incremental cost-effectiveness ratio (ICER) of EUR 30,709. Scenario ICERs evaluating the key assumptions, aligning the subsequent therapy data as per in-trial use (EUR 49,059) and up to 83.9% of patients receiving 28 out of 28-day dosing, also remained below the WTP threshold. Conclusions: Introducing LEN as a maintenance therapy post-ASCT delays progression, improves survival, and reduces subsequent treatment-line costs. The use of LEN post-ASCT is cost-effective in comparison with no maintenance therapy in the Netherlands. Disclosures Uyl-de Groot: Merck: Research Funding; Janssen- Cilag: Research Funding; Gilead: Research Funding; Genzyme: Research Funding; Celgene Corp.: Research Funding; Boehringer Ingelheim: Research Funding; Bayer: Research Funding; AstraZeneca: Research Funding; Astellas: Research Funding; Amgen: Research Funding; Roche: Research Funding; Sanofi: Research Funding. Ramsden:Celgene Corp.: Consultancy; BresMed: Employment. Boersma:Celgene BV: Employment, Equity Ownership. Zweegman:Takeda: Membership on an entity's Board of Directors or advisory committees, Research Funding; Takeda: Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene Corp.: Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Membership on an entity's Board of Directors or advisory committees, Research Funding. Dhanasiri:Celgene International: Employment, Equity Ownership.


2021 ◽  
Vol 12 ◽  
Author(s):  
Hongfu Cai ◽  
Baohua Xu ◽  
Na Li ◽  
Bin Zheng ◽  
Zhiwei Zheng ◽  
...  

Background: This study aimed to analyze the cost effectiveness of camrelizumab in the second-line treatment of advanced or metastatic esophageal squamous cell carcinoma in China.Methods: On the basis of the ESCORT clinical trial, a partitioned survival model was constructed to simulate the patient’s lifetime quality-adjusted life years (QALYs), lifetime costs, and incremental cost-effectiveness ratio (ICER). One-way sensitivity and probability sensitivity analyses were performed to test the stability of the model.Results: Treatment of esophageal squamous cell carcinoma with camrelizumab added 0.36 QALYs and resulted in an incremental cost of $1,439.64 compared with chemotherapy, which had an ICER of $3,999 per QALY gained. The ICER was far lower than the threshold of willingness to pay for one time the GDP per capita in China. Sensitivity analysis revealed that the ICERs were most sensitive to the cost of drugs, but the parameters did not have a major effect on the results of the model.Conclusion: Camrelizumab is likely to be a cost-effective option compared with chemotherapy for patients with advanced or metastatic esophageal squamous cell carcinoma. This informs patient selection and clinical path development.


Author(s):  
Javier Aguilar-Serra ◽  
Vicente Gimeno-Ballester ◽  
Alfonso Pastor-Clerigues ◽  
Javier Milara ◽  
Ezequiel Marti-Bonmati ◽  
...  

Aim: To assess the cost–effectiveness of first-line treatment with dacomitinib compared with gefitinib in patients newly diagnosed with advanced NSCLC EGFR-positive in the context of Spain. Materials & methods: A partitioned survival model was developed including costs, utilities and disutilities to estimate quality-adjusted life-year (QALY) and incremental cost–effectiveness ratio when treating with dacomitinib versus gefitinib. Results: Dacomitinib presented higher QALYs (0.51) compared with gefitinib (0.45). Dacomitinib costs were €33,061 in comparison with €26,692 for gefitinib arm. An incremental cost–effectiveness ratio of €111,048 was obtained for dacomitinib. Conclusion: Dacomitinib was more effective in terms of QALYs gained than gefitinib. However, to obtain a cost–effectiveness alternative, a discount greater than 25% in dacomitinib acquisition cost is required.


2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e21104-e21104
Author(s):  
Nimer S. Alkhatib ◽  
Briana Choi ◽  
Hala Halawah ◽  
Matthias Calamia ◽  
Dexter Gulick ◽  
...  

e21104 Background: Crizotinib, alectinib, ceritinib, and brigatinib are approved as second line treatment for ALK+ NSCLC. Crizotinib was the first ALK inhibitor for first line therapy approved by Food and Drug Administration (2011) then ceritinib (2014), alectinib (2015), and brigatinib (2017) were approved as second line drugs. Following more data, these agents were approved as the first line therapy (2017 for ceritinib and alectinib; 2020 for brigatinib). These remain as a treatment option in patients who fail the first line therapy. Cost-effectiveness/utility analyses were conducted to assess clinical efficacy with varying costs of the agents. Methods: A three state Markov model were assumed (progression free, progression and death). Progression free survival (PFS) curves were digitized and fitted with exponential function. US payer perspective, a lifetime horizon, and discount rate of 3% were applied. Drug costs were Redbook wholesale acquisition cost. Other costs included were monitoring, adverse events and disease progression from published data (US$ 2020). Adverse events reported >5% in patients were included. Measured outcomes were PFS life years (PFSLY) and quality adjusted life years (PFSQALY). Crizotinib was the reference drug. Incremental cost-effectiveness and utility ratios (ICER/ICUR) of PFSLY and PFSQALY gained (PFSLYG, PFSQALYG) and lost were estimated. Base case (BCA) and probabilistic sensitivity analyses (PSA) were conducted. Results: Crizotinib was the reference drug for the following outcomes. For alectinib, with the decremental cost of -$14,653 (-$14,712), the incremental PFSLY of 0.16 (0.16) and PFSQALY of 0.05 (0.05) resulted in an ICER / PFSLYG of -$89,337 (-$88,604) and an ICUR / PFSQALYG of -$269,835 (-$266,510). For brigatinib, with the decremental cost of -$14,975 (-$14,954), the incremental PFSLY of 0.01 (0.01) and PFSQALY of ̃0.01 (0.02) yielded an ICER / PFSLYG of -$1,982,962 (-$1,431,631) and an ICUR / PFSQALYG of -$2,140,534 (-$570,538). For ceritinib, with the incremental cost of $7,590 ($7,514), there were decremental PFSLY of -0.01 (-0.01) and PFSQALY of -0.03 (-0.03). Conclusions: As second line treatment, crizotinib, ceritinib, and brigatinib had comparable PFSLYs and PFSQALYs while alectinib had the most PFSLY and PFSQALY and the lowest cost. Therefore, alectinib is the most cost-effective treatment for treating ALK+ NSCLC as the second line therapy.[Table: see text]


2020 ◽  
Vol 36 (5) ◽  
pp. 492-499
Author(s):  
Akashdeep Singh Chauhan ◽  
Shankar Prinja ◽  
Sushmita Ghoshal ◽  
Roshan Verma

BackgroundThe newer cancer treatment technologies hold the potential of providing improved health outcomes at an additional cost. So it becomes obligatory to assess the costs and benefits of a new technology, before defining its clinical value. We assessed the cost-effectiveness of intensity-modulated radiotherapy (IMRT) as compared to 2-dimensional radiotherapy (2-DRT) and 3-dimensional radiotherapy (3D-CRT) for treating head and neck cancers (HNC) in India. The cost-effectiveness of 3-DCRT as compared to 2-DRT was also estimated.MethodsA probabilistic Markov model was designed. Using a disaggregated societal perspective, lifetime study horizon and 3 percent discount rate, future costs and health outcomes were compared for a cohort of 1000 patients treated with any of the three radiation techniques. Data on health system cost, out of pocket expenditure, and quality of life was assessed through primary data collected from a large tertiary care public sector hospital in India. Data on xerostomia rates following each of the radiation techniques was extracted from the existing randomized controlled trials.ResultsIMRT incurs an incremental cost of $7,072 (2,932–13,258) and $5,164 (463–10,954) per quality-adjusted life year (QALY) gained compared to 2-DRT and 3D-CRT, respectively. Further, 3D-CRT as compared to 2-DRT requires an incremental cost of $8,946 (1,996–19,313) per QALY gained.ConclusionBoth IMRT and 3D-CRT are not cost-effective at 1 times GDP per capita for treating HNC in India. The costs and benefits of using IMRT for other potential indications (e.g. prostate, lung) require to be assessed before considering its introduction in India.


Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2656-2656
Author(s):  
Steven R. Arikian ◽  
Dejan Milentijevic ◽  
Gary Binder ◽  
Mara Silvia Monzini ◽  
X Henry Hu ◽  
...  

Abstract Introduction: As clinical evidence has mounted in support of novel agents and longer treatment (Tx) durations for patients (pts) with newly diagnosed multiple myeloma (NDMM), questions have arisen regarding the economic impact of extending time to progression (TTP) in these pts, and the cost consequences once pts relapse and move to a second line of Tx. Previous analysis showed that relapsed myeloma pts incurred higher monthly costs once they advanced to later lines of Tx (Gaultney, 2013). There is limited information on the cost patterns of MM pts before and after their first relapse. A claims analysis was performed to evaluate the patterns of total direct costs of care, from Tx initiation until progression, for NDMM patients and for newly relapsed patients treated with novel agents, utilizing time to next therapy (TTNT) as a proxy measure for progression. Methods: A retrospective study was conducted using a large US medical and pharmacy claims database, covering > 25 million lives annually. NDMM patients were identified with at least 2 outpatient claims or 1 inpatient medical claim associated with a diagnosis of MM (ICD-9-CM] code 203.0X), with the first such claim used to define the index date. Inclusion criteria required a minimum of 12 months' pre-index enrollment and 6 months' post-index continuous enrollment between 2006 and 2012. Pts with claims for stem cell transplantation (SCT) were excluded, to avoid confounding results from various factors based on timing, costs, and site of care of SCT. The analysis focused on NDMM and relapsed MM pts receiving lenalidomide (LEN)- or bortezomib (BORT)- based Tx, where complete claim history was available from Tx onset to initiation of subsequent Tx. Using methods similar to those described by Gaultney, patients' average monthly costs were determined, including medical (inpatient, ambulatory, and emergency room) and pharmacy (index and other drugs) costs, and total cost patterns over quarterly time periods were calculated. Average Charlson comorbidity scores were determined to compare baseline measures between pt groups. Results: 897 NDMM pts and 280 relapsed MM pts were identified with complete data through initiation of subsequent Tx. Monthly total direct costs for NDMM pts were $15,400 in the first 3 months (mos) of Tx, and declined each quarter, reaching approximately $5,000/mo at 18+ mos. At relapse, monthly costs increased to over $12,000 for the first 3 mos and followed a quarterly pattern of reduction similar to that seen for NDMM pts (Fig 1). Quarterly cost reduction patterns were consistent for patients treated with LEN or BORT for both NDMM and relapsed pts. Pts' total monthly NDMM costs over the full TTNT period averaged $8,942 with LEN vs. $11,139 for BORT (due to 54% higher monthly medical costs for BORT), while monthly drug costs were nearly identical (Table 1). The baseline Charlson comorbidity index was similar between Tx groups in both lines of Tx. Figure 1: Direct monthly costs (medical and pharmacy) for LEN- and BORT-based treatments Figure 1:. Direct monthly costs (medical and pharmacy) for LEN- and BORT-based treatments Table 1: Direct monthly costs for NDMM pts Table 1 Table 1. Conclusions: For a population of NDMM pts receiving either LEN- or BORT-based Tx without SCT, followed until TTNT, total direct monthly costs per pt declined steadily over time, decreasing by 68% from the initial quarter to the period post 18 mos. Costs spiked when pts began 2nd-line therapy, then followed a similar pattern of decline over time. This pattern may suggest that further extending the TTP for NDMM pts may also yield economic benefits for each month extended before relapse. Patterns of cost decline were similar between the LEN and BORT groups, for NDMM and for relapsed patients, although mean monthly total costs were lower for NDMM pts receiving LEN-based Tx due to lower medical costs and similar drug costs. Disclosures Arikian: Genesis Research: Consultancy. Off Label Use: Lenalidomide in newly diagnosed multiple myeloma patients . Milentijevic:Celgene Corporation: Consultancy. Binder:Celgene Corporation: Employment, Equity Ownership. Monzini:Celgene Corporation: Employment, Equity Ownership. Hu:Celgene Corporation: Employment. Nagarwala:Celgene Corporation: Employment. Hussein:Celgene Corporation: Employment. Corvino:Genesis Research LLC: Consultancy. Surinach:Genesis Research LLC: Consultancy. Usmani:Celgene Corporation: Consultancy, Honoraria, Research Funding; Millennium: Consultancy, Honoraria; Onyx: Consultancy, Honoraria, Research Funding; Sanofi: Consultancy; Array BioPharma: Research Funding; Janssen: Research Funding; Pharmacyclics: Research Funding.


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