scholarly journals Estimation of Survival Probabilities for Use in Cost-effectiveness Analyses: A Comparison of a Multi-state Modeling Survival Analysis Approach with Partitioned Survival and Markov Decision-Analytic Modeling

2016 ◽  
Vol 37 (4) ◽  
pp. 427-439 ◽  
Author(s):  
Claire Williams ◽  
James D. Lewsey ◽  
Daniel F. Mackay ◽  
Andrew H. Briggs
Keyword(s):  
Cost Effectiveness ◽  
A Priori ◽  
Progression Free Survival ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Lymphocytic Leukemia ◽  
Health States ◽  
Analytic Modeling ◽  
Life Years ◽  
Markov Decision

Modeling of clinical-effectiveness in a cost-effectiveness analysis typically involves some form of partitioned survival or Markov decision-analytic modeling. The health states progression-free, progression and death and the transitions between them are frequently of interest. With partitioned survival, progression is not modeled directly as a state; instead, time in that state is derived from the difference in area between the overall survival and the progression-free survival curves. With Markov decision-analytic modeling, a priori assumptions are often made with regard to the transitions rather than using the individual patient data directly to model them. This article compares a multi-state modeling survival regression approach to these two common methods. As a case study, we use a trial comparing rituximab in combination with fludarabine and cyclophosphamide v. fludarabine and cyclophosphamide alone for the first-line treatment of chronic lymphocytic leukemia. We calculated mean Life Years and QALYs that involved extrapolation of survival outcomes in the trial. We adapted an existing multi-state modeling approach to incorporate parametric distributions for transition hazards, to allow extrapolation. The comparison showed that, due to the different assumptions used in the different approaches, a discrepancy in results was evident. The partitioned survival and Markov decision-analytic modeling deemed the treatment cost-effective with ICERs of just over £16,000 and £13,000, respectively. However, the results with the multi-state modeling were less conclusive, with an ICER of just over £29,000. This work has illustrated that it is imperative to check whether assumptions are realistic, as different model choices can influence clinical and cost-effectiveness results.

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.

Cost-effectiveness analysis of proton versus photon therapy with respect to risk of growth hormone deficiency.

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. e17553-e17553
Author(s):  
Raymond Mailhot ◽  
Jane Kim ◽  
Abby Hollander ◽  
Jeff M. Michalski ◽  
Nancy Tarbell ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Cost Effectiveness ◽  
Growth Hormone Deficiency ◽  
Proton Therapy ◽  
Cost Effective ◽  
Childhood Cancer Survivors ◽  
Cns Tumors ◽  
Hormone Deficiency ◽  
Health States ◽  
Life Years

e17553 Background: Proton therapy has been endorsed as a radiotherapy (RT) modality with the potential to avert many RT associated comorbidities. Currently, few proton centers exist, and no evidence-based data as yet adequately informs allotment of care. Childhood cancer survivors of central nervous system (CNS) malignancies have high prevalences of growth hormone deficiency (GHD) after hypothalamic exposure, notable for its cost of treatment greater than $10,000/year. We propose methodology to help guide proton referral of pediatric patients with CNS tumors through cost-effective analysis comparisons for proton and photon hypothalamic received dose. Methods: A Markov cohort model was designed to assess the expected costs and effectiveness for specific RT doses to the hypothalamus. Patients entered the model receiving proton or photon RT for CNS tumors at four and twelve years of age and were followed for 60 more years of life. Patients could experience two health states: GHD or healthy. Risk of GHD was based on data by Merchant et al. Costs were measured in USD and captured cost of GHD and cost of RT. Cost of proton course relative to photon was estimated at $160K. Effectiveness was measured in quality-adjusted life years (QALYs). The main outcome measure used for comparison was the incremental cost-effectiveness ratio (ICER). We assumed a societal willingness to pay threshold of $50,000/QALY. Results: Data were used to generate tables incorporating the differential cost of proton RT to project ICERs for different combinations of hypothalamic RT dose as displayed in the Table below. Conclusions: Despite the high cost of proton therapy, the cost of GHD alone can yield proton RT a cost-effective (even cost-minimizing) strategy when compared to photon RT. This work provides a guide for identifying a set of patients for which proton therapy is particularly cost-effective. [Table: see text]

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.

scholarly journals COST-EFFECTIVENESS OF POST-AUTOTRANSPLANT LENALIDOMIDE IN PERSONS WITH MULTIPLE MYELOMA.

2021 ◽  
Vol 13 (1) ◽  
Author(s):  
Monia Marchetti ◽  
Robert Peter Gale ◽  
Giovanni Barosi
Keyword(s):  
Cost Effectiveness ◽  
Progression Free Survival ◽  
Published Data ◽  
Fixed Duration ◽  
Health States ◽  
Life Years ◽  
The Us ◽  
Markov Decision Model ◽  
Eu And Us ◽  
The Eu

Considerable data indicate posttransplant lenalidomide prolongs progression-free survival and probably survival after an autotransplant for plasma cell myeloma (PCM).  However, optimal therapy duration is unknown, controversial and differs in the EU and US.  We compared outcomes and cost-effectiveness of 3 posttransplant lenalidomide strategies in EU and US settings: (1) none; (2) until failure; and (3) 2-year fixed duration.  We used a Markov decision model which included 6 health states and informed by published data.  The model estimated the strategy of lenalidomide given to failure achieved 1.06 quality-adjusted life years (QALYs) at costs per QALY gained of €29,232 in the EU   and $133,401 in the US settings.  Two-year fixed-duration lenalidomide averted €7,286 per QALY gained in the EU setting and saved 0.84 QALYs at $60,835 per QALY gained in the US setting.  These extremely divergent costs per QALY in the EU and US settings resulted from large differences in costs of posttransplant lenalidomide and of 2nd-line therapies driven by whether posttransplant failure was on- or off-lenalidomide.  In Monte Carlo simulation analyses which allowed us to account for variability of inputs, 2-year fixed-duration lenalidomide remained the preferred strategy for improving health-care sustainability in the EU and US settings.

scholarly journals Estimating the Relative Treatment Effect and Corresponding Cost-Effectiveness Estimates of Inotuzumab Ozogamicin Vs. Blinatumomab for Adults with Philadelphia Chromosome-Negative (Ph-) Relapsed/Refractory (R/R) B-Cell Acute Lymphoblastic Leukaemia (B-ALL) in the United Kingdom (UK)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3427-3427 ◽  
Author(s):  
Simone Critchlow ◽  
Miranda Cooper ◽  
Ilse van Oostrum ◽  
Verna L Welch ◽  
T. Alexander Russell-Smith
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Complete Response ◽  
Health State ◽  
Health States ◽  
Term Survival ◽  
Long Term Survival ◽  
Treatment Comparison ◽  
Life Years

Introduction: Inotuzumab ozogamicin (InO), is a novel anti-CD22 antibody-calicheamicin conjugate approved in R/R B-ALL due to its high hematologic remission rate (81%) based on the phase 3 INO-VATE trial comparing to investigators choice (IC). The TOWER trial demonstrated the efficacy and safety of blinatumomab (Blina) for treatment of Ph- B-ALL versus IC. The relative effectiveness of InO versus Blina was investigated by applying indirect treatment comparison (ITC) methods. A UK-based cost-effectiveness model (CEM) submitted to the Scottish Medicines Consortium (SMC) explored the impact of treatment differences with regard to mean life years (LY) gained and quality-adjusted life years (QALY). Methods: As R/R ALL is a terminal disease if left untreated, achievement of complete response/complete response with incomplete count recovery (CR/CRi) in conjunction with stem cell transplant (SCT) is essential for long-term survival. The three most important outcomes related to treatment are thus the level of response determined by CR/CRi, the rate of SCT, and overall survival (OS). Without potentially curative therapy such as SCT, there is no evidence to suggest long-term survival is possible. Therefore, to compare InO to Blina, comparisons of these outcomes were explored using patient-level data from the INO-VATE ALL trial and aggregate data from the TOWER trial. The CEM structure contained four health states categorising patients based on 'No CR/CRi & no SCT', 'CR/CRi and no SCT' and patients receiving SCT ('SCT/Post SCT') - with progression-free survival (PFS) and OS modelled within these states. States were clinically validated as relevant to treatment of the disease. Death was the fourth health state. Different methods were incorporated to allocate Blina patients to the respective health-states. For levels of response (CR/CRi) and SCT a matching-adjusted indirect comparison (MAIC) and a Bucher ITC were explored. As CR/CRi and SCT rates are not mutually exclusive, a multinomial ITC was also conducted. Once allocated into respective health states, OS and PFS were modelled. Three ITC methods were used to compare OS; a simulated treatment comparison (STC), MAIC and a standard network meta-analysis. In the absence of PFS data for Blina, PFS was assumed to have the same relative treatment effect as OS. Quality of life data within the model for the 'No CR/CRi & no SCT' and 'CR/CRi and no SCT' were informed from InO trial data, while SCT quality of life was informed from the literature with time-varying utilities. Costs were incorporated from a UK perspective using 2017 sources and were those submitted to the SMC. Results were annually discounted at 3.5%. Results: Health state proportions for Ph- InO patients were used as the basis to estimate corresponding Blina proportions and show 49.3% of patients treated with InO reach SCT. With higher odds for CR/CRi and SCT for InO, the ITC results consistently indicate Blina leads to lower proportions of patients receiving SCT (19.1-22.5%) and CR/CRi (25.2-33.3%). ITCs comparing OS outcomes for InO versus blinatumomab show negligible differences between treatments, consistently across the three methods. All combinations of the various methods were explored using the list price for both treatments. The results of the CEM ranged from 0.91-1.14 incremental QALYs for InO versus Blina, while LYs ranged from 2.03-2.59 resulting from higher rates of SCT. The incremental cost-effectiveness ratio (ICER) ranged from £3,700 to £7,010 for InO versus Blina. Extensive scenario analysis indicates that InO is a cost-effective option compared to Blina at a willingness to pay threshold of £20,000 per QALY. The SMC recommended InO as a cost-effective use of resources citing an ICER of £6,754 in the CEM when using the MAIC; InO was associated with a mean survival gain of >29 months over Blina corresponding to this ICER. Conclusions: Outcomes from the ITC indicate that InO provides patients with a greater probability of achieving CR/CRi and/or receiving a subsequent SCT versus Blina. As CR/CRi followed by SCT are essential for long-term survival and potential cure, the mean OS gain in the model cited in the SMC recommendation is intuitive as it aligns with the superior CR/CRi and SCT odds ratios associated with InO. Further research is required to determine the long-term PFS and OS following SCT in R/R B-ALL, beyond what can be reliably captured within clinical trials. Disclosures Critchlow: BresMed Health Solutions Ltd.: Consultancy. Cooper:BresMed Health Solutions Ltd.: Consultancy. van Oostrum:Ingress Health: Employment; Pfizer: Consultancy; Merck: Consultancy; Janssen: Consultancy; AstraZeneca: Consultancy. Welch:Pfizer Inc: Employment, Equity Ownership. Russell-Smith:Pfizer: Employment, Equity Ownership.

scholarly journals Cost–effectiveness of tacrolimus for the treatment of moderate-to-severe lupus nephritis in China

2019 ◽  
Vol 8 (13) ◽  
pp. 1125-1141
Author(s):  
Soyoung Kim ◽  
Adrian Yit Reen Ooi ◽  
Thomas Stephens ◽  
Hongsi Jiang
Keyword(s):  
Cost Effectiveness ◽  
Lupus Nephritis ◽  
Cost Effective ◽  
Original Form ◽  
Health States ◽  
Effectiveness Model ◽  
Life Years ◽  
Cost Effective Treatment ◽  
Tacrolimus Therapy ◽  
The Cost

Aim: Therapy for lupus nephritis (LN) requires treatment with immunosuppressive regimens, often including intravenous cyclophosphamide (IVCY), mycophenolate mofetil (MMF) or azathioprine. Additionally, tacrolimus (original form or generic) is recommended to treat LN patients in Asia, including China. However, the cost–effectiveness of tacrolimus therapy has not previously been assessed. We aimed to estimate the cost–effectiveness of tacrolimus in the treatment of moderate-to-severe LN versus standard therapies in China. Materials & methods: This cost–effectiveness model combined a decision-tree/Markov-model structure to map transitions between health states during induction and maintenance treatment phases. Induction with tacrolimus, IVCY or MMF, was followed by tacrolimus, MMF or azathioprine maintenance. Results: According to the model, during induction, complete remission rates were higher with tacrolimus versus IVCY (relative risk 1.40 vs IVCY [deterministic sensitivity analysis minimum 0.92, maximum 2.13]) and time to response was shorter. Relapse rates were lower with tacrolimus versus azathioprine or MMF during maintenance. Tacrolimus induction and maintenance was the most cost-effective regimen, incurring the lowest total costs (CN¥180,448) with the highest quality-adjusted life-years. Conclusion: The model demonstrated that tacrolimus use in both induction and maintenance therapy may be an efficacious and cost-effective treatment for LN in China.

scholarly journals Fluorouracil plasma monitoring: systematic review and economic evaluation of the My5-FU assay for guiding dose adjustment in patients receiving fluorouracil chemotherapy by continuous infusion

2015 ◽  
Vol 19 (91) ◽  
pp. 1-322 ◽  
Author(s):  
Karoline Freeman ◽  
Martin Connock ◽  
Ewen Cummins ◽  
Tara Gurung ◽  
Sian Taylor-Phillips ◽  
...  
Keyword(s):  
Liquid Chromatography ◽  
Cost Effectiveness ◽  
Social Services ◽  
High Performance ◽  
Dose Adjustment ◽  
Progression Free Survival ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Base Case ◽  
Practical Implementation

Background5-Fluorouracil (5-FU) is a chemotherapy used in colorectal, head and neck (H&N) and other cancers. Dose adjustment is based on body surface area (BSA) but wide variations occur. Pharmacokinetic (PK) dosing is suggested to bring plasma levels into the therapeutic range to promote fewer side effects and better patient outcomes. We investigated the clinical effectiveness and cost-effectiveness of the My5-FU assay for PK dose adjustment to 5-FU therapy.ObjectivesTo systematically review the evidence on the accuracy of the My5-FU assay compared with gold standard methods [high-performance liquid chromatography (HPLC) and liquid chromatography-mass spectrometry (LC-MS)]; the effectiveness of My5-FU PK dosing compared with BSA; the effectiveness of HPLC and/or LC-MS compared with BSA; the generalisability of published My5-FU and PK studies; costs of using My5-FU; to develop a cost-effectiveness model.Data sourcesWe searched MEDLINE, EMBASE, Science Citation Index and other databases between January and April 2014.MethodsTwo reviewers independently screened titles and abstracts with arbitration and consensus agreement. We undertook quality assessment. We reconstructed Kaplan–Meier plots for progression-free survival (PFS) and overall survival (OS) for comparison of BSA and PK dosing. We developed a Markov model to compare My5-FU with BSA dosing which modelled PFS, OS and adverse events, using a 2-week cycle over a 20 year time horizon with a 3.5% discount rate. Health impacts were evaluated from the patient perspective, while costs were evaluated from the NHS and Personal Social Services perspective.ResultsA total of 8341 records were identified through electronic searches and 35 and 54 studies were included in the clinical effectiveness and cost-effectiveness reviews respectively. There was a high apparent correlation between My5-FU, HPLC and LC-MS/mass spectrometer but upper and lower limits of agreement were –18% to 30%. Median OS were estimated as 19.6 [95% confidence interval (CI) 17.0 to 21.0] months for PK versus 14.6 (95% CI 14.1 to 15.3) months for BSA for 5-FU + folinic acid (FA); and 27.4 (95% CI 23.2 to 38.8) months for PK versus 20.6 (95% CI 18.4 to 22.9) months for BSA for FOLFOX6 in metastatic colorectal cancer (mCRC). PK versus BSA studies were generalisable to the relevant populations. We developed cost-effectiveness models for mCRC and H&N cancer. The base case assumed a cost per My5-FU assay of £61.03. For mCRC for 12 cycles of a oxaliplatin in combination with 5-fluorouracil and FA (FOLFOX) regimen, there was a quality-adjusted life-year (QALY) gain of 0.599 with an incremental cost-effectiveness ratio of £4148 per QALY. Probabilistic and scenario analyses gave similar results. The cost-effectiveness acceptability curve showed My5-FU to be 100% cost-effective at a threshold of £20,000 per QALY. For H&N cancer, again, given caveats about the poor evidence base, we also estimated that My5-FU is likely to be cost-effective at a threshold of £20,000 per QALY.LimitationsQuality and quantity of evidence were very weak for PK versus BSA dosing for all cancers with no randomised controlled trials (RCTs) using current regimens. For H&N cancer, two studies of regimens no longer in use were identified.ConclusionsUsing a linked evidence approach, My5-FU appears to be cost-effective at a willingness to pay of £20,000 per QALY for both mCRC and H&N cancer. Considerable uncertainties remain about evidence quality and practical implementation. RCTs are needed of PK versus BSA dosing in relevant cancers.FundingThe National Institute for Health Research Health Technology Assessment programme.

Cost-Effectiveness of Rituximab Post CVP Induction for the Treatment of Low-Grade NHL.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4486-4486
Author(s):  
John C. Hornberger ◽  
Carolina Reyes ◽  
Ashwini Shewade ◽  
David Loecke ◽  
Nancy Valente
Keyword(s):  
Cost Effectiveness ◽  
B Cell ◽  
Progression Free Survival ◽  
The United States ◽  
Sensitivity Analyses ◽  
Low Grade ◽  
Health States ◽  
Economic Implications ◽  
Expected Gain ◽  
Life Years

Abstract Background: Extended Rituximab (R) therapy after CVP (cyclophosphamide, vincristine, prednisone) induction in the first-line treatment of low-grade, CD20-positive, B-cell NHL significantly prolongs progression-free survival (PFS). A societal cost-effectiveness analysis is performed to estimate projected lifetime clinical and economic implications of this treatment. Methods: A Markov model with 3 health states (progression-free, post-progression, and death) was developed to estimate the direct medical costs and quality-adjusted life-years (QALYs) for a representative patient cohort with low-grade, CD20-positive, B-cell NHL. Kaplan-Meier estimates of PFS and overall survival (OS) up to 4 years are obtained from the ECOG/CALGB intergroup E1496 trial. After 4 years, transition rates are assumed to be the same in both arms. Costs include drug and administration costs, adverse events, treatment of relapses, and end-of-life costs. Incremental costs associated with R are based on Medicare reimbursement rates and published drug prices. Utilities are derived from the literature and a 3% discount rate is employed. Results: Projected mean OS is 1.2-yrs longer for patients assigned to extended R therapy versus observation alone (15.2 v 14.0 yrs). Extended R therapy is estimated to cost $42,822 on average, with an expected gain of 0.85 years of quality-adjusted survival. Over a lifetime, the cost per QALY gained is $57,515. Sensitivity analyses performed on all variables contributing to the model results showed that utility for follicular NHL patients (range: 0.68–1) and number of R infusions (range: 8–17) most influence the incremental cost-effectiveness ratio (ICER). Conclusion: The ICER of extended R dosing following CVP induction remains well below the threshold considered acceptable for oncology in the United States. Additional analysis of long term follow-up data will be useful for further evaluation of clinical and economic implications of this treatment for low-grade, CD20-positive, B-cell NHL patients.

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.

Cost-effectiveness of rituximab maintenance therapy for patients with follicular lymphoma: The Spanish perspective

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 8092-8092
Author(s):  
J. Gómez Codina ◽  
M. Provencio ◽  
A. Rueda ◽  
F. Capote ◽  
F. Carbonell ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Maintenance Therapy ◽  
Follicular Lymphoma ◽  
Maintenance Treatment ◽  
Progression Free Survival ◽  
Base Case ◽  
Health States ◽  
Free Survival ◽  
Rituximab Maintenance ◽  
Life Years

8092 Background: In patients with relapsed or refractory follicular lymphoma (FL) who attain a response with either cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) alone or Rituximab + CHOP, maintenance treatment with Rituximab has shown to significantly improve overall survival (OS) (85% at 3 years vs. 77%, p=0.011) and progression free survival (PFS) (51,5 vs. 14.9 months, p<0.001) as compared to observation alone (OA). We analyzed the cost-effectiveness, from a Spanish perspective, of Rituximab maintenance therapy (375mg/m2 every 3 months until progression or for 2 years) versus OA according to the population and data described for the European Organization for Research Treatment of Cancer (EORTC) 20981 study (van Oers MHJ Blood 2006). Methods: Incremental cost-effectiveness was assessed through a deterministic, three health states model (disease-free, progression and death) transition model. Base case model: PFS and OS were extrapolated from EORTC 20981 data using a Weibull distribution, Rituximab maintenance benefit was assumed to last 5 years, 10 years time horizon, 3.5% discount rate on costs and benefits, and Spanish National Health Service perspective (direct costs only). Resource use was estimated from a Spanish expert panel and EORTC 20981 study. Unit costs were obtained from local databases (May 2006 €). Health states utility values were derived from an ad hoc study. Sensitivity analyses were performed for all mentioned variables. Results: For the base case, more quality-adjusted life years (QALY), life-years (LY) and progression-free survival years per patient on maintenance therapy were obtained versus OA (incremental values of 0.85, 0.94 and 1.46, respectively). Total cost per patient was higher with Rituximab than with OA (+8,026€). Incremental cost per QALY gained was 9,358€, with a cost per LY gained of 8.493€ and a cost per PFS year gained of 5,485€. In the sensitivity analysis, values ranged between 7.263€ and 22.160€ per QALY gained. Conclusions: This study confirms that in patients with relapsed /refractory FL who attain a response with further therapy, maintenance treatment with Rituximab compared to observation alone is cost-effective. No significant financial relationships to disclose.

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