Cost-effectiveness of everolimus for the treatment of advanced neuroendocrine tumours of gastrointestinal or lung origin in Canada

Background In 2016, everolimus was approved by Health Canada for the treatment of unresectable, locally advanced or metastatic, well-differentiated, non-functional, neuroendocrine tumours (NET) of gastrointestinal (GI) or lung origin in adult patients with progressive disease. This analysis evaluated the cost-effectiveness of everolimus in this setting from a Canadian societal perspective.Methods A partitioned survival model was developed to compare the cost per life-year (LY) gained and cost per quality-adjusted life-year (QALY) gained of everolimus plus best supportive care (BSC) versus BSC alone in patients with advanced or metastatic NET of GI or lung origin. Model health states included stable disease, disease progression, and death. Efficacy inputs were based on the RADIANT-4 trial and utilities were mapped from quality-of-life data retrieved from RADIANT-4. Resource utilization inputs were derived from a Canadian physician survey, while cost inputs were obtained from official reimbursement lists from Ontario and other published sources. Costs and efficacy outcomes were discounted 5% annually over a 10-year time horizon, and sensitivity analyses were conducted to test the robustness of the base case results.Results Everolimus had an incremental gain of 0.616 QALYs (0.823 LYs) and CA$89,795 resulting in an incremental cost-effectiveness ratio of CA$145,670 per QALY gained (CA$109,166 per LY gained). The probability of cost-effectiveness was 52.1% at a willingness to pay (WTP) threshold of CA$150,000 per QALY.Conclusions Results of the probabilistic sensitivity analysis indicate that everolimus has a 52.1% probability of being cost-effective at a WTP threshold of CA$150,000 per QALY gained in Canada.

Download Full-text

An economic evaluation of nivolumab for the treatment of squamous and non-squamous NSCLC in the Swedish setting

Nordic Journal of Health Economics ◽

10.5617/njhe.5453 ◽

2020 ◽

Author(s):

Caitlin Smare ◽

Meena Venkatachalam ◽

Emma Medin ◽

Marcus Hultberg ◽

John R Penrod ◽

...

Keyword(s):

Cost Effectiveness ◽

Incremental Cost ◽

Scenario Analysis ◽

Progression Free Survival ◽

Cost Effective ◽

Quality Adjusted Life Year ◽

Sensitivity Analyses ◽

Base Case ◽

Health States ◽

Treatment Data

The cost-effectiveness of nivolumab versus docetaxel in patients with previously treated non-small cell lung cancer (NSCLC) was estimated in a cohort-based, partitioned survival model with three health states (progression-free, progressed disease, and death) and a time horizon of 15 years. The base-case model was developed using extrapolations of progression-free survival (PFS) and overall survival (OS) data from the CheckMate 017 and 057 randomized trials, and 2015 Swedish unit costs. An annual discount rate of 3% was applied. Base-case time-on-treatment was based on PFS (CheckMate 017) or time-to-treatment discontinuation (CheckMate 057), depending on whether PFS was a close proxy for time-on-treatment. Data extrapolations from CheckMate 017 and 057 were validated against external trial and registry data. Model utilities were derived from CheckMate 017 and 057 with UK weights (base-case) and Swedish weights (scenario analysis). Uncertainty was assessed using sensitivity analyses adjusted for clinical, utility, and cost data. Outcomes included incremental cost per quality-adjusted life-year (QALY) gained. The base-case model showed that nivolumab was associated with QALY gains of 0.72 (squamous) and 0.81 (non-squamous) versus docetaxel at an incremental cost of 734,573 SEK (€69,174) and 999,032 SEK (€94,078), respectively. This resulted in an incremental cost per QALY gained for nivolumab versus docetaxel of 1,013,697 SEK (€95,459) and 1,231,664 SEK (€115,985) in squamous and non-squamous NSCLC, respectively. Scenario analysis utilizing Swedish utility weights resulted in slightly lower incremental cost per QALY gained of 855,505 SEK (€80,562) (squamous) and 1,165,401 SEK (€109,745) (non-squamous). Deterministic sensitivity analysis showed that utility weights, treatment costs, discount rates, and body weight were key drivers of cost-effectiveness. Overall, the model showed that cost-effectiveness was driven by nivolumab price, but nivolumab remained cost-effective in squamous and non-squamous NSCLC in accordance with previous appraisals by the Dental and Pharmaceutical Benefits Agency (Tandvårds- och läkemedelsförmånsverket) and New Therapies Council in Sweden. Published: Online December 2019.

Download Full-text

P14.12 The cost-effectiveness of tumor-treating fields in patients with newly diagnosed glioblastoma

Neuro-Oncology ◽

10.1093/neuonc/noz126.247 ◽

2019 ◽

Vol 21 (Supplement_3) ◽

pp. iii68-iii69

Author(s):

X Armoiry ◽

P Auguste ◽

C Dussart ◽

J Guyotat ◽

M Connock

Keyword(s):

Cost Effectiveness ◽

Cost Effective ◽

Survival Model ◽

Sensitivity Analyses ◽

Base Case ◽

Tumor Treating Fields ◽

Kaplan Meier ◽

Life Years ◽

Secondary Analyses ◽

The Cost

Abstract BACKGROUND The addition of novel therapy “Tumor-Treating fields” (TTF) to standard radio-chemotherapy with Temozolomide (TMZ) has recently shown superiority over conventional TMZ regimen in patients with glioblastoma. Despite the clinical benefit of TTF, there is a strong concern regarding the cost of this new treatment. A first cost-effectiveness analysis, which was published in 2016, was based on effectiveness outcomes from an interim analysis of the pivotal trial and used a “standard” Markov model. Here, we aimed to update the cost-effectiveness evaluation using a partitioned survival model design and using the latest effectiveness data. MATERIAL AND METHODS A partitioned survival model was developed with three mutually exclusive health states: stable disease, progressive disease, and dead. Parametric models were fitted to the Kaplan-Meier data for overall and progression-free survival. These generated clinically plausible extrapolations beyond the observed data. The perspective of the French national health insurance was adopted and the time horizon was 20 years. Base case results were expressed as cost/life-years (LY) gained (LYG). Secondary analyses were undertaken, with the results presented as cost/per quality adjusted life years (QALY) gained. Last, we undertook deterministic and probabilistic sensitivity analyses. RESULTS After applying 4% annual discounting of benefits and costs, the base case model generated incremental benefit of 0.507 LY at a incremental cost of €258,695 yielding an incremental cost effectiveness ratio (ICER) of €510,273 / LYG. Secondary analyses yielded an ICER of €667,173/QALY. Sensitivity analyses and bootstrapping methods showed the model was relatively robust. The model was sensitive to TTF device costs and the parametric model fitted to the Kaplan-Meier data for overall survival. The cost-effectiveness acceptability curve showed TTF has 0% of being cost-effective under conventional thresholds. CONCLUSION Using a partitioned survival model, uprated costs and more mature survival outcomes, TTF when compared to standard radio-chemotherapy with TMZ is not likely to be cost-effective. This has major implications in terms of access of newly eligible patients

Download Full-text

The hemodialysis prescription and cost effectiveness. Renal Physicians Association Working Committee on Clinical Guidelines.

Journal of the American Society of Nephrology ◽

10.1681/asn.v441021 ◽

1993 ◽

Vol 4 (4) ◽

pp. 1021-1027

Author(s):

J C Hornberger

Keyword(s):

Cost Effectiveness ◽

Clinical Outcomes ◽

Cost Effective ◽

Quality Adjusted Life Year ◽

Base Case ◽

Uremic Toxin ◽

Intradialytic Hypotension ◽

Case Scenario ◽

Base Case Scenario ◽

The Cost

Case-mix adjusted mortality rates for patients undergoing hemodialysis for ESRD increased during the 1980s, despite the introduction of advanced dialysis technologies. Variations in dialysis practices suggest that excess mortality may be caused by inadequate uremic-toxin clearances. Cost-effectiveness analysis was used to assess whether attempts to improve uremic-toxin clearance are cost effective, assuming that these therapies are clinically effective. The medical literature was surveyed by the use of MEDLINE to assess the likelihood of clinical outcomes on the basis of the type of treatment given to the patient. Options considered in the model were delivered fractional urea clearance (Kt/V), dialysis-treatment duration, type of dialyzer membrane, dialysate, and ultrafiltration. Clinical outcomes included in the model were survival, severity of uremic symptoms, hospital days per year, and intradialytic hypotension and symptoms. Lifetime costs were calculated from data collected from a northern California dialysis center and abstracted from the literature. In the base-case scenario, it was assumed that increasing Kt/V to levels greater than 1 was effective in reducing morbidity and mortality. Under these assumptions, outpatient cost increased significantly, but the cost effectiveness of Kt/V equal to 1.5 was less than $50,000 per quality-adjusted life-year saved. These calculations indicate that, if higher levels of Kt/V prove clinically effective, they are also cost effective.

Download Full-text

Cost-Effectiveness of Particulated Juvenile Articular Cartilage Versus Matrix-Induced Autologous Chondrocyte Implantation for Patellar Chondral Lesions (176)

Orthopaedic Journal of Sports Medicine ◽

10.1177/2325967121s00295 ◽

2021 ◽

Vol 9 (10_suppl5) ◽

pp. 2325967121S0029

Author(s):

Drake LeBrun ◽

Jake Feingold ◽

Stephanie Swenson-Buza ◽

Simone Gruber ◽

Elizabeth Dennis ◽

...

Keyword(s):

Cost Effectiveness ◽

Nonoperative Management ◽

Cost Effective ◽

Sensitivity Analyses ◽

Cartilage Defects ◽

Base Case ◽

Patellar Cartilage ◽

Chondrocyte Implantation ◽

The Cost ◽

Autologous Chondrocyte

Objectives: Treatment options for articular cartilage lesions of the patella have evolved over the past several years due to the development of novel cell-based cartilage restoration techniques, including particulated juvenile allograft cartilage (PJAC) and matrix-induced autologous chondrocyte implantation (MACI). The objective of this study was to evaluate the cost -effectiveness of these modalities in the management of patellar cartilage defects. Methods: A Markov state-transition model was utilized to evaluate the cost-effectiveness of three strategies for patients with patellar chondral lesions: (1) nonoperative management, (2) PJAC, and (3) MACI. Probabilities, health utilities, and costs of surgical procedures and rehabilitation protocols were derived from institutional data and literature review. Effectiveness was assessed using quality-adjusted life-year (QALY). Cost-effectiveness was evaluated from societal and payer perspectives over a 15-year time horizon. The principal outcome measure was the incremental cost-effectiveness ratio (ICER). Sensitivity analyses were performed on pertinent model parameters to assess their effect on base case conclusions. Results: From a societal perspective, nonoperative management, PJAC, and MACI cost $4,140, $52,683, and $83,073 respectively. Nonoperative management, PJAC, and MACI were associated with 4.91, 7.07, and 7.79 QALYs gained, respectively. Therefore, PJAC and MACI were cost-effective relative to nonoperative management (ICERs $22,527/QALY and $27,456/QALY, respectively; Figure 1). Although MACI was more cost-effective than PJAC in the base case, this was strongly sensitive to the estimated probabilities of full versus intermediate benefit following PJAC and MACI (Table 1). If the probabilities of full and intermediate benefit following PJAC were assumed to be the same as those following MACI (i.e., PJAC and MACI were equally effective), then PJAC dominated MACI by being cheaper and more effective. At a $100,000/QALY willingness-to-pay threshold, MACI, PJAC, and nonoperative management were the preferred strategies in 63%, 33%, and 4% of the Monte Carlo probabilistic sensitivity analyses, respectively (Figures 2 and 3). Similar results were seen from a payer perspective. Conclusions: In the management of symptomatic patellar cartilage defects, PJAC and MACI were both cost-effective compared to nonoperative treatment in the management of symptomatic patellar cartilage defects; however, MACI was the preferred strategy in our base-case analysis. The cost-effectiveness of PJAC compared to MACI depended heavily on the probability of achieving full versus intermediate benefit after PJAC and MACI.

Download Full-text

Cost-utility Analysis of Trabecular Micro-bypass Stents (TBS) in Patients With Mild-to-moderate Open-angle Glaucoma in Italy

10.21203/rs.3.rs-267336/v1 ◽

2021 ◽

Author(s):

Antonio Maria Fea ◽

Francesco Cattel ◽

Stefano Gandolfi ◽

Giorgio Buseghin ◽

Gianluca Furneri ◽

...

Keyword(s):

Cost Effectiveness ◽

Cataract Surgery ◽

Open Angle Glaucoma ◽

Cost Effective ◽

Cost Utility ◽

Sensitivity Analyses ◽

Base Case ◽

Utility Analysis ◽

Istent Inject ◽

The Cost

Abstract BackgroundGlaucoma is a disease characterized by progressive damage of the optic nerve. Several therapeutic options are available to lower intraocular pressure (IOP). In primary open-angle glaucoma (POAG) patients with inadequate IOP control (or controlled with multiple medical therapies or for whom medical therapy is contraindicated), the implantation of micro-invasive glaucoma surgery devices (MIGS) and concomitant cataract surgery has proved to be more effective in reducing intraocular pressure (IOP), as compared to cataract surgery alone. The objective of this study is to assess the cost-effectiveness of iStent inject® device with concurrent cataract surgery vs. cataract surgery alone, in patients with mild-to-moderate POAG, adopting the Italian National Health Service (NHS) perspective.MethodsSimulation of outcomes and costs was undertaken using a Markov model with 4 health states and one-month cycles, that is used to simulate the prognosis of these patients. Efficacy data were obtained from the randomized clinical trial (RCT). A lifetime horizon was adopted in the analysis. A discount rate of 3.5% was applied to both costs and effects. The Italian National Healthcare Service (NHS) perspective was considered, therefore only healthcare direct costs (acquisition of main interventions and subsequent procedures; medications; monitoring and follow-up; adverse events). Model robustness was tested through sensitivity analyses. ResultsResults of the base-case analysis showed that the total lifetime costs were higher in the iStent inject® + concurrent cataract surgery, compared with the cataract surgery alone group (€8,368.51 vs. €7,134.71 respectively). iStent inject® + concurrent cataract surgery was cost-effective vs. cataract surgery alone, with an incremental cost-effectiveness ratio of €13,037.01 per quality-adjusted life year (QALY) gained. Both one-way deterministic and probabilistic sensitivity analyses confirmed robustness of base-case results. The acceptability curve of cost-effectiveness (CEAC) analysis showed that iStent inject® + cataract surgery would have a 98% probability of being cost-effective, compared to cataract surgery alone, when the willingness to pay (WTP) is equal to €50,000 per QALY gained.ConclusionsThe results of the cost-utility analysis confirm that iStent inject® + cataract surgery is a cost-effective option for the treatment of patients affected by mild-to-moderate POAG, compared with cataract surgery alone, when evaluated from the Italian NHS perspective. Trial registration: Not applicable

Download Full-text

Cost-effectiveness and value-based prices of the 9-valent human papillomavirus vaccine for the prevention of cervical cancer in China: an economic modelling analysis

BMJ Open ◽

10.1136/bmjopen-2019-031186 ◽

2019 ◽

Vol 9 (11) ◽

pp. e031186 ◽

Cited By ~ 3

Author(s):

Y Jiang ◽

Weiyi Ni ◽

Jing Wu

Keyword(s):

Cervical Cancer ◽

Human Papillomavirus ◽

Cost Effectiveness ◽

Hpv Vaccine ◽

Cost Effective ◽

Sensitivity Analyses ◽

Base Case ◽

Economic Modelling ◽

Current Price ◽

The Cost

ObjectivesTo evaluate the cost-effectiveness of the 9-valent human papillomavirus (HPV) vaccine for the prevention of cervical cancer in China.DesignHealth economic modelling using the Papillomavirus Rapid Interface for Modelling and Economics (PRIME) model populated with China-specific data.SettingIndividual cervical cancer prevention in China using the 9-valent HPV vaccine from the perspective of private sector purchasers in relation to receiving other HPV vaccines and not receiving vaccination for 16-year-old girls in China who had not been previously infected with HPV.ParticipantsNot applicable.InterventionsVaccination using the 9-valent, the quadrivalent and the bivalent vaccines.Primary outcome measureIncremental costs per disability-adjusted life year (DALY) prevented.ResultsIn the base case, the incremental costs per DALY prevented were, respectively, US$35 000 and US$50 455 compared with the quadrivalent and the bivalent vaccines, both of which were above the cost-effective threshold of US$25 920/DALY prevented. To be cost-effective in these comparisons, the 9-valent vaccine should be priced at $550 and $450 for the full doses, respectively. To be highly cost-effective, the price thresholds were $435 and $335. The incremental costs per DALY prevented in relation to no vaccination was US$23 012, making the 9-valent vaccine marginally cost-effective. The results were robust in most one-way sensitivity analyses including changing vaccination age to 13 and 26 years.ConclusionsAt the current price, the 9-valent HPV vaccine is not cost-effective compared with the quadrivalent and the bivalent vaccines for young girls in China who had not been previously infected with HPV. Policymakers and clinicians should keep potential vaccine recipients informed about the economic profile of the 9-valent vaccine and carefully consider expanding its use in China at the current price.

Download Full-text

Cost-effectiveness of primary prophylaxis (PP) with colony-simulating factor (CSF) when compared with secondary prophylaxis (SP) in elderly patients with diffuse aggressive lymphoma undergoing curative chemotherapy

Journal of Clinical Oncology ◽

10.1200/jco.2007.25.18_suppl.6558 ◽

2007 ◽

Vol 25 (18_suppl) ◽

pp. 6558-6558

Author(s):

K. K. Chan ◽

K. R. Imrie ◽

S. M. Alibhai

Keyword(s):

Cost Effectiveness ◽

Elderly Patients ◽

Cost Minimization ◽

Cost Effective ◽

Primary Prophylaxis ◽

Cost Utility ◽

Sensitivity Analyses ◽

Aggressive Lymphoma ◽

Base Case ◽

The Cost

6558 Background: The 2006 ASCO guideline recommends PP with CSF for elderly patients with diffuse aggressive lymphoma, partially based on previous cost-minimization analyses showing that CSF saved costs when compared with no CSF by reducing hospitalization from febrile neutropenia (FN) when the risk of FN was > 20%. However, these studies examined only one cycle of chemotherapy and did not account for costs of CSF in subsequent cycles, did not consider SP, and did not consider patients’ preferences. Methods: We conducted a cost-utility analysis to compare PP with SP in this setting using a Markov model for a time horizon of 8 cycles of chemotherapy with a government payer perspective. Costs were adjusted to 2006 $CAD. Ontario health economic data were used. The cost of hospitalization for FN was obtained from Ontario Case Costing Initiative. Data for efficacies of CSF, probabilities and utilities were obtained from published literature. Sensitivity analyses were conducted using a threshold of $100,000/QALY. Results: The base case costs for PP and SP were $22,077 and $17,641. The QALYs of PP and SP were 0.254 and 0.248. The incremental cost effectiveness ratio of PP to SP was $739,999/QALY. One-way sensitivity analyses showed that in order for PP to be cost-effective, the cost of hospitalization per episode of FN had to be > $31,138 (i.e. 2.5 times > base case), the cost of CSF per cycle had to be < $896 (base case = $1,960), the risk of FN in the 1st cycle had to be > 48% (base case = 24%), or the relative risk reduction of FN with CSF had to be > 97% (base case = 41%). Our result was robust to all other cost, probability and utility variables. First order microsimulation showed that < 17% of simulations were cost-effective. Conclusions: PP is not cost-effective when compared with SP for this population under most assumptions. PP only becomes attractive in places where the cost of hospitalization for FN is much more than that of Ontario, or the cost of CSF is under $896 per cycle. The costs of CSF and hospitalization in all cycles (instead of just one cycle) should be accounted for in any economic evaluation of CSF. Current guidelines recommending PP in this population should be revisited. No significant financial relationships to disclose.

Download Full-text

Cost-effectiveness of sequential daily teriparatide/weekly alendronate compared with alendronate monotherapy for older osteoporotic women with prior vertebral fracture in Japan

Archives of Osteoporosis ◽

10.1007/s11657-021-00891-z ◽

2021 ◽

Vol 16 (1) ◽

Author(s):

Takahiro Mori ◽

Carolyn J. Crandall ◽

Tomoko Fujii ◽

David A. Ganz

Keyword(s):

Cost Effectiveness ◽

Vertebral Fracture ◽

Cost Effective ◽

Community Dwelling ◽

Sensitivity Analyses ◽

Base Case ◽

Microsimulation Model ◽

Term Care ◽

Lifetime Horizon ◽

The Cost

Abstract Summary Using a Markov microsimulation model among hypothetical cohorts of community-dwelling older osteoporotic Japanese women with prior vertebral fracture over a lifetime horizon, we found that daily subcutaneous teriparatide for 2 years followed by weekly oral alendronate for 8 years was not cost-effective compared with alendronate monotherapy for 10 years. Purpose Teriparatide has proven efficacy in reducing osteoporotic fractures, but with substantial cost. We examined the cost-effectiveness of sequential teriparatide/alendronate (i.e., daily subcutaneous teriparatide for 2 years followed by weekly oral alendronate for 8 years) compared with alendronate monotherapy for 10 years among community-dwelling older osteoporotic women with prior clinical or morphometric vertebral fracture in Japan. Methods Using a previously validated and updated Markov microsimulation model, we obtained incremental cost-effectiveness ratios (Japanese yen [¥] (or US dollars [$]) per quality-adjusted life year [QALY]) from the perspective of a single payer responsible for both public healthcare and long-term care. We assumed a lifetime horizon with a willingness-to-pay of ¥5million (or $47,500) per QALY in the base case. We modeled the cost of biosimilar teriparatide, which has been available since November 2019 in Japan, assuming the efficacy was the same as that of the brand version. Results In the base case, sequential teriparatide/alendronate was not cost-effective compared with alendronate monotherapy. In deterministic sensitivity analyses, sequential teriparatide/alendronate would become cost-effective with 85%, 50%, and 15% price discounts to teriparatide at ages 70, 75, and 80, respectively, compared to the current biosimilar cost. Otherwise, results were especially sensitive to changes that affected efficacy of teriparatide or alendronate. In probabilistic sensitivity analyses, the probabilities of sequential teriparatide/alendronate being cost-effective were 0%, 1%, and 37% at ages 70, 75, and 80, respectively. Conclusions Among high-risk osteoporotic women in Japan, sequential teriparatide/alendronate was not cost-effective compared with alendronate monotherapy, even with the availability of biosimilar teriparatide.

Download Full-text

Detecting Germline PTEN Mutations Among At-Risk Patients With Cancer: An Age- and Sex-Specific Cost-Effectiveness Analysis

Journal of Clinical Oncology ◽

10.1200/jco.2014.60.3456 ◽

2015 ◽

Vol 33 (23) ◽

pp. 2537-2544 ◽

Cited By ~ 11

Author(s):

Joanne Ngeow ◽

Chang Liu ◽

Ke Zhou ◽

Kevin D. Frick ◽

David B. Matchar ◽

...

Keyword(s):

Cost Effectiveness ◽

Cleveland Clinic ◽

Cost Effective ◽

Cancer Surveillance ◽

Sensitivity Analyses ◽

Base Case ◽

Effective Strategy ◽

Female Patients ◽

Cost Effective Strategy ◽

The Cost

Purpose Cowden syndrome (CS) is an autosomal dominant disorder characterized by benign and malignant tumors. One-quarter of patients who are diagnosed with CS have pathogenic germline PTEN mutations, which increase the risk of the development of breast, thyroid, uterine, renal, and other cancers. PTEN testing and regular, intensive cancer surveillance allow for early detection and treatment of these cancers for mutation-positive patients and their relatives. Individual CS-related features, however, occur commonly in the general population, making it challenging for clinicians to identify CS-like patients to offer PTEN testing. Patients and Methods We calculated the cost per mutation detected and analyzed the cost-effectiveness of performing selected PTEN testing among CS-like patients using a semi-quantitative score (the PTEN Cleveland Clinic [CC] score) compared with existing diagnostic criteria. In our model, first-degree relatives of the patients with detected PTEN mutations are offered PTEN testing. All individuals with detected PTEN mutations are offered cancer surveillance. Results CC score at a threshold of 15 (CC15) costs from $3,720 to $4,573 to detect one PTEN mutation, which is the most inexpensive among the different strategies. At base-case, CC10 is the most cost-effective strategy for female patients who are younger than 40 years, and CC15 is the most cost-effective strategy for female patients who are between 40 and 60 years of age and male patients of all ages. In sensitivity analyses, CC15 is robustly the most cost-effective strategy for probands who are younger than 60 years. Conclusion Use of the CC score as a clinical risk calculator is a cost-effective prescreening method to identify CS-like patients for PTEN germline testing.

Download Full-text

Methodology and results of real-world cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone in relapsed multiple myeloma using registry data

The European Journal of Health Economics ◽

10.1007/s10198-019-01122-6 ◽

2019 ◽

Vol 21 (2) ◽

pp. 219-233 ◽

Cited By ~ 2

Author(s):

M. Campioni ◽

I. Agirrezabal ◽

R. Hajek ◽

J. Minarik ◽

L. Pour ◽

...

Keyword(s):

Multiple Myeloma ◽

Cost Effectiveness ◽

Real World ◽

Current Model ◽

Progression Free Survival ◽

Cost Effective ◽

Quality Adjusted Life Year ◽

Base Case ◽

Health States ◽

Randomised Controlled

Abstract Objective To predict the real-world (RW) cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone (KRd) versus lenalidomide and dexamethasone (Rd) in relapsed multiple myeloma (MM) patients after one to three prior therapies. Methods A partitioned survival model that included three health states (progression-free, progressed disease and death) was built. Progression-free survival (PFS), overall survival (OS) and time to discontinuation (TTD) data for the Rd arm were derived using the Registry of Monoclonal Gammopathies in the Czech Republic; the relative treatment effects of KRd versus Rd were estimated from the phase 3, randomised, ASPIRE trial, and were used to predict PFS, OS and TTD for KRd. The model was developed from the payer perspective and included drug costs, administration costs, monitoring costs, palliative care costs and adverse-event related costs collected from Czech sources. Results The base case incremental cost effectiveness ratio for KRd compared with Rd was €73,156 per quality-adjusted life year (QALY) gained. Patients on KRd incurred costs of €117,534 over their lifetime compared with €53,165 for patients on Rd. The QALYs gained were 2.63 and 1.75 for patients on KRd and Rd, respectively. Conclusions Combining the strengths of randomised controlled trials and observational databases in cost-effectiveness models can generate policy-relevant results to allow well-informed decision-making. The current model showed that KRd is likely to be cost-effective versus Rd in the RW and, therefore, the reimbursement of KRd represents an efficient allocation of resources within the healthcare system.

Download Full-text