PCN40 METHODOLOGICAL CHALLENGES OF EVALUATING THE COST-EFFECTIVENESS OF THERAPIES INCLUDED IN MULTIPLE THERAPEUTIC SCHEMES: THE CASE OF LENALIDOMIDE FOR REFRACTORY/RELAPSED MULTIPLE MYELOMA IN BRAZIL

This paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of bortezomib for the treatment of multiple myeloma patients at first relapse and beyond, in accordance with the licensed indication, based upon the evidence submission from Ortho Biotech to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The outcomes stated in the manufacturer’s definition of the decision problem were time to disease progression, response rate, survival and quality of life. The literature searches for clinical and cost-effectiveness studies were adequate and the one randomised controlled trial (RCT) included was of reasonable quality. Results from the RCT suggest that bortezomib increases survival and time to disease progression compared with high-dose dexamethasone (HDD) in multiple myeloma patients who have had a relapse after one to three treatments. Cost-effectiveness analysis based on the same trial and an observational study was reasonable and gave an estimated cost per life-year gained of £30,750, which ranged from £27,957 to £36,747 on sensitivity analysis. An attempt was made to replicate the results of the manufacturer’s model and to compare the results to the Kaplan–Meier survival curve presented in the manufacturer’s submission. In addition, a one-way sensitivity analysis and a probabilistic sensitivity analysis were undertaken, as well as additional scenario analyses. Based on these analyses the ERG suggests that the cost-effectiveness results presented in the manufacturer’s submission may underestimate the cost per life-year gained for bortezomib therapy (versus high-dose dexamethasone) when potential UK practice and scenarios are considered. The guidance issued by NICE in June 2006 as a result of the STA states that bortezomib monotherapy for the treatment of relapsed multiple myeloma is clinically effective compared with HDD but has not been shown to be cost-effective and is not recommended for the treatment of progressive multiple myeloma in patients who have received at least one previous therapy and who have undergone, or are unsuitable for, bone marrow transplantation.

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Chemotherapy-based versus chemotherapy-free stem cell mobilization (± plerixafor) in multiple myeloma patients: an Italian cost-effectiveness analysis

Bone Marrow Transplantation ◽

10.1038/s41409-021-01251-8 ◽

2021 ◽

Cited By ~ 1

Author(s):

Carlo Lazzaro ◽

Luca Castagna ◽

Francesco Lanza ◽

Daniele Laszlo ◽

Giuseppe Milone ◽

...

Keyword(s):

Multiple Myeloma ◽

Cost Effectiveness ◽

Cost Effectiveness Analysis ◽

Hematopoietic Progenitor Cell ◽

Base Case ◽

High Dose ◽

On Demand ◽

Effectiveness Analysis ◽

Cell Mobilization ◽

The Cost

AbstractGiven the availability and efficacy of the mobilizing agent plerixafor in augmenting hematopoietic progenitor cell mobilization with granulocyte colony-stimulating factor (G-CSF), there is a strong case for comparing the cost-effectiveness of mobilization with G-CSF + cyclophosphamide versus G-CSF alone. This study investigated the cost and effectiveness (i.e., successful 4 million-CD34+ collection) of G-CSF alone versus high-dose cyclophosphamide (4 g/m2) + G-CSF mobilization (± on-demand plerixafor) in patients with multiple myeloma (MM) eligible for autograft in Italy. A decision tree-supported cost-effectiveness analysis (CEA) model in MM patients was developed from the societal perspective. The CEA model compared G-CSF alone with cyclophosphamide 4 g/m2 + G-CSF (± on-demand plerixafor) and was populated with demographic, healthcare and non-healthcare resource utilization data collected from a questionnaire administered to six Italian oncohematologists. Costs were expressed in Euro (€) 2019. The CEA model showed that G-CSF alone was strongly dominant versus cyclophosphamide + G-CSF ( ± on-demand plerixafor), with incremental savings of €1198.59 and an incremental probability of a successful 4 million-CD34+ apheresis (+0.052). Sensitivity analyses confirmed the robustness of the base-case results. In conclusion, chemotherapy-free mobilization (± on-demand plerixafor) is a “good value for money” option for MM patients eligible for autograft.

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Cost–effectiveness of lenalidomide plus low-dose dexamethasone for newly diagnosed multiple myeloma patients ineligible for stem cell transplantation in China

Journal of Comparative Effectiveness Research ◽

10.2217/cer-2019-0052 ◽

2019 ◽

Vol 8 (12) ◽

pp. 979-992 ◽

Cited By ~ 1

Author(s):

Jin Lu ◽

Wendong Chen

Keyword(s):

Multiple Myeloma ◽

Stem Cell ◽

Cost Effectiveness ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Low Dose ◽

Chinese Patients ◽

Newly Diagnosed ◽

Life Years ◽

The Cost

Aim: To assess the cost–effectiveness of lenalidomide plus low dose dexamethasone (Rd) relative to bortezomib-contained therapy (BCT) for newly diagnosed multiple myeloma patients ineligible for stem cell transplantation (ndMM) in China. Materials & methods: A literature review was conducted to identify appropriate evidence for developing a cost–effectiveness model comparing Rd with BCT for lifetime health outcomes and direct medical costs in Chinese ndMM patients. Results: The estimated incremental cost–effectiveness ratio per gained quality-adjusted life years for Rd versus BCT was ¥49,793. The chance for Rd to be cost effective, under the cost–effectiveness thresholds of three-times the 2018 Chinese gross domestic goods per capita, was 90.8%. Conclusion: The cost–effectiveness of Rd relative to BCT for ndMM in Chinese patients is highly attractive.

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Economic Evaluation of Daratumumab and Pomalidomide and Dexamethasone Versus Isatuximab and Pomalidomide and Dexamethasone for Patients with Relapsed or Refractory Multiple Myeloma

Blood ◽

10.1182/blood-2020-136348 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 19-20

Author(s):

Neda Alrawashdh ◽

Abdulaali Almutairi ◽

Ali McBride ◽

Ivo Abraham

Keyword(s):

Clinical Trial ◽

Multiple Myeloma ◽

Cost Effectiveness ◽

Time Horizon ◽

Cost Utility ◽

Phase Iii ◽

Health States ◽

Time Horizons ◽

Life Years ◽

The Cost

Background. Although several new treatments are available for patients with multiple myeloma (MM), most patients eventually relapse at a median time of 8.0 months (95%CI: 6.3-8.9). Patients with relapsed and refractory MM (R/R MM) who have had several lines of previous therapy or who are refractory to lenalidomide and proteasome inhibitors require alternative options. Daratumumab and isatuximab are monoclonal antibodies that bind to the human CD38 receptor. Phase II/III clinical trials showed that isatuximab (ISA) or daratumumab (DARA) in combination with pomalidomide (POM-d) and low-dose dexamethasone (DEXA) significantly improve progression-free survival (PFS) in patients with R/R MM. No studies have assessed the comparative efficacy and cost-effectiveness of both regimens in management of R/R MM. We performed an indirect comparison of both regimens in terms of PFS and overall survival (OS) and evaluated the cost-effectiveness and cost-utility of DARA+POM-d+DEXA and ISA+POM-d+DEXA from a US payer's perspective. Methods. A partitioned survival model was developed to create three health states (pre-progression, progression, and death). The model was run three times with different time horizons (one, three and five years). To simulate health outcomes for each treatment regimen, transition probabilities between the three health states were derived from parametric exponential and lognormal distributions fitted to Kaplan-Meier (KM) curves of PFS and OS of the phase Ib clinical trial (Chari et al.; Blood 2017) for DARA+POM-d+DEXA and the phase III clinical trial (Attal et al.; Lancet 2019) for ISA+POM-d+DEXA. Wholesale acquisition costs (WAC) were obtained from RedBook for each regimen. Pre-progression costs included costs of regimens; premedication (50 mg diphenhydramine, 650 mg acetaminophen, 50 mg ranitidine); managing side effects; routine care and monitoring; and medication administration. Costs were inflated based on the medical consumer price index to the second quarter of 2020. Utilities were obtained from literature and assumed the same for both interventions. Annual discount rate of 3.5% was applied for costs and outcomes beyond the first year. The life years (LY) and quality adjusted LY (QALY) for each treatment, and the incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) were estimated in both base and probabilistic sensitivity analyses (PSA:10,000 simulations). The cost-effectiveness plane (CEP) and cost-effectiveness acceptability curves (CEAC) were plotted. Results. In the naïve patient simulation, median PFS and OS were estimated to be 9.5 months and 18 months for DARA+POM-d+DEXA, and 14.5 months and 26 months for ISA+POM-d +DEXA. As shown in the table below, ISA+POM-d+DEXA is associated with greater LY and QALY gains at one-, three- and five-year time horizons. The costs of ISA+POM-d+DEXA at one- and three- year time horizons are less than that of DARA+POM-d+DEXA, which resulted in saving (decremental) ICERs. At 5 years' time horizon, ISA+POM-d+DEXA was associated with incremental benefits (0.57 LY, 0.35 QALY) and incremental costs of $88,271 when compared with DARA+POM-d+DEXA. Per the CEAC plot, the probability that ISA+POM-d+DEXA is cost-effective was 100%, 65% and 23% at a willingness to pay threshold (WTP) of $100,000 per QALY in one-, three- and five-year time horizons. Conclusions. Clinically, ISA+POM-d+DEXA is associated with incremental survival gains of ~1 month and quality-adjusted survival gains of 0.5 month than DARA+POM-d+DEXA when patients are treated for one year. The benefits increase with treatment duration to reach ~7 months life year gains and 4 months quality-adjusted life year gains if patients treated for 5 years. Due to its lower total costs, Isatuximab based-regimen yielded saving ICERs at one and three years. However, ISA+POM-d+DEXA cost exceeded DARA+POM-d+DEXA at 5 years' time horizon to yield an ICER above the WTP. Disclosures McBride: Merck: Speakers Bureau; Pfizer: Consultancy; Sandoz: Consultancy; MorphoSys: Consultancy; Bristol-Myers Squibb: Consultancy; Coherus BioSciences: Consultancy, Speakers Bureau. Abraham:Celgene: Consultancy; Terumo: Consultancy; Rockwell Medical: Consultancy; Janssen: Consultancy; Mylan: Consultancy; Sandoz: Consultancy; Coherus BioSciences: Research Funding, Speakers Bureau; MorphoSys: Consultancy.

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Cost-Effectiveness of Lenalidomide in Multiple Myeloma Patients with 1 Prior Therapy in England and Wales,

Blood ◽

10.1182/blood.v118.21.4181.4181 ◽

2011 ◽

Vol 118 (21) ◽

pp. 4181-4181 ◽

Cited By ~ 1

Author(s):

Stephen Schey ◽

Sean Stern ◽

Sujith Dhanasiri ◽

Ruth Brown

Keyword(s):

Multiple Myeloma ◽

Cost Effectiveness ◽

Cost Effective ◽

Economic Modelling ◽

England And Wales ◽

Prior Therapy ◽

Line Therapy ◽

Life Years ◽

The Uk ◽

The Cost

Abstract Abstract 4181 Introduction: Lenalidomide plus dexamethasone (Len+Dex) is approved by EMA for patients with multiple myeloma (MM) who have received at least 1 prior therapy. In the wake of NICE's guidance on Thalidomide and Bortezomib TA 228 for 1st-line treatment of MM, Len+Dex may be the only available novel therapy option with potential to extend survival beyond traditional therapies for patients failing the NICE recommended initial treatments; thalidomide or bortezomib both in combination with melphalan and prednisone. Therefore, economic modelling was undertaken to evaluate the cost-effectiveness of Len+Dex from the perspective of the National Health Service (NHS), England and Wales. Methods: An Excel-based individual simulation model was developed to account for disease history which affects time-to-progression (TTP) and overall survival (OS) in relapse refractory MM patients. The OS for each individual within the model was calculated as the combination of that patient's TTP and post progression survival (PPS). The TTP efficacy was derived from a subgroup analysis of patients from the MM-009/010 pivotal trials who had received only 1 prior therapy. The derived equation for TTP included parameters for treatment response, Len+Dex treatment effect and certain baseline characteristics of patients with only 1 prior therapy. The PPS for patients within the model was also derived from the MM-009/010 trials; however, since 47% of patients on Dex switched to Len+Dex at progression, there was a significant Len+Dex effect in the Dex PPS. To account for this, the Dex PPS was calibrated using the longer follow up from the UK Medical Research Council (MRC) trials. Resource use for monitoring and adverse events was obtained via a survey of expert hematologists in the UK. Costs were taken from NHS sources and included a Patient Access Scheme where patients received Len free of charge beyond 26 cycles of use. Utility values were taken from published literature and applied to patients prior to progression (based on their best response rate achieved) and after progression. The robustness of the model results were assessed by sensitivity analyses whereby individual model parameters were varied across their confidence intervals (CI). Results reported include life-years (LY), quality-adjusted life-years (QALY), costs, cost per LY gained and cost per QALY gained. NICE technology appraisal methodology guidelines were followed when modelling over a lifetime horizon and applying a 3.5% discount rate for future benefits and costs. All costs presented are in British pounds. Results: Len+Dex had a substantial increase in health-related outcomes compared to Dex alone, with LYs of 5.37 vs. 2.15 and QALYs of 3.69 vs. 1.49, respectively. The associated drug costs for Len+Dex over the patients' lifetime were higher: however, survival benefits accrued over the same time period offset the cost differences between the two regimens, resulting in a cost per LY gained of 20,639 and per QALY gained of 30,153. The model results remained robust to sensitivity analysis conducted on most parameters and utility estimates. The only parameter that affected the results was the adjustment factor for Dex survival when varied across its 95% CI (see table). Discussion: These results indicated that Len+Dex at 2nd line is cost-effective for a novel drug in an orphan disease compared to Dex alone. Despite Len+Dex only being recommended by NICE for use in MM patients with 2 or more prior therapies (with the recent NICE decision to allow thalidomide and Velcade in de novo patients), treatment alternatives for 2nd line therapy with novel agents is more limited. It is important for clinicians to consider the costs and benefits of 2nd line therapy. This analysis demonstrated that Len+Dex is cost-effective for 2nd line patients and meets the commonly quoted cost per QALY threshold of 30,000. Modelled Results: Disclosures: Schey: Celgene: Consultancy. Stern:United BioSource Corporation: Employment; Celgene: Consultancy. Dhanasiri:Celgene: Employment. Brown:United BioSource Corporation: Employment; Celgene: Consultancy.

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PSY29 MODELING THE COST-EFFECTIVENESS OF BORTEZOMIB FOR THE INITIAL TREATMENT OF MULTIPLE MYELOMA IN THE UNITED STATES

Value in Health ◽

10.1016/s1098-3015(10)73031-x ◽

2010 ◽

Vol 13 (3) ◽

pp. A210

Author(s):

ST Wang ◽

H Huang ◽

H Shi ◽

MS Dun ◽

K Chen ◽

...

Keyword(s):

United States ◽

Multiple Myeloma ◽

Cost Effectiveness ◽

Initial Treatment ◽

The United States ◽

The Cost

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Cost-Effectiveness Analysis of Adding Daratumumab to Bortezomib, Melphalan, and Prednisone for Untreated Multiple Myeloma

Frontiers in Pharmacology ◽

10.3389/fphar.2021.608685 ◽

2021 ◽

Vol 12 ◽

Author(s):

Yaohua Cao ◽

Lina Zhao ◽

Tiantian Zhang ◽

Weiling Cao

Keyword(s):

Multiple Myeloma ◽

Cost Effectiveness ◽

Life Time ◽

Cost Effective ◽

Base Case ◽

Quality Adjusted Life Years ◽

Effectiveness Analysis ◽

Life Years ◽

The Cost ◽

Quality Adjusted Life

Background: To evaluate the cost-effectiveness of adding daratumumab to bortezomib, melphalan, and prednisone for transplant-ineligible newly diagnosed multiple myeloma patients.Methods: A three-state Markov model was developed from the perspective of US payers to simulate the disease development of patient’s life time for daratumumab plus bortezomib, melphalan, and prednisone (D-VMP) and bortezomib, melphalan, and prednisone (VMP) regimens. The primary outputs were total costs, expected life-years (LYs), quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs).Results: The base case results showed that adding daratumumab to VMP provided an additional 3.00 Lys or 2.03 QALYs, at a cost of $262,526 per LY or $388,364 per QALY. Sensitivity analysis indicated that the results were most sensitive to utility of progression disease of D-VMP regimens, but no matter how these parameters changed, ICERs remained higher than $150,000 per QALY.Conclusion: In the case that the upper limit of willingness to pay threshold was $150,000 per QALY from the perspective of US payers, D-VMP was not a cost-effective regimen compared to VMP.

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