U.S. budget impact (BI) model for selinexor, bortezomib, and dexamethasone (XVd) for the treatment of patients with previously treated multiple myeloma (MM).

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18839-e18839
Author(s):  
Mike Dolph ◽  
Gabriel Tremblay ◽  
Hoyee Leong
Keyword(s):  
Budget Impact ◽  
Case Analysis ◽  
Third Party ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Base Case Analysis ◽  
Party Payer ◽  
Previously Treated ◽  
The Absolute ◽  
To Receive

e18839 Background: Despite advances in the treatment of MM, prognosis remains poor for most patients due to its refractory nature. In a phase 3 study, a once-per-week regimen of XVd showed a novel, effective, and convenient option for patients with previously treated MM. This study was conducted to estimate the projected budget impact (BI) of XVd in patients with previously treated MM from the perspective of a third-party payer and Medicare in the US. Methods: The introduction of XVd as a treatment option for patients with previously treated MM compared to the status quo (i.e. without the introduction of XVd) was considered from a private third-party US payer and a Medicare perspective in one-year increments for the first 3 years. The model assumed market uptake of 5.9%, 7.2%, and 7.7% at years 1, 2 and 3, respectively. Total annual treatment costs (2020 US dollars [USD]) were calculated as the sum of drug costs, the costs of treating serious treatment emergent adverse events (grade ≥3), ongoing best supportive care costs, and mortality costs. Results: In the base-case analysis for a private third-party payer plan (1,000,000 members), 3 to 4 patients were projected to receive XVd out of 47 to 49 eligible patients each year. The absolute BI (Millions, USD) of including XVd from a private third-party payer plan perspective was $0.06, $0.07, $0.08 and $0.22 for years 1, 2, 3, and overall, respectively. The relative BI of including XVd was 0.33%, 0.40%, 0.43%, and 0.38% for years 1, 2, 3, and overall, respectively. This translated to a per member per month (PMPM) budget impact of $0.005, $0.006, $0.007, and $0.006 (USD), for years 1, 2, 3, and overall, respectively. In the base-case analysis from the Medicare perspective (59,499,015 members), 1,361 to 1,808 patients were projected to receive XVd out of 22,892 to 23,425 eligible patients each year. From a Medicare perspective, the absolute BI (Millions, USD) of including XVd was $29.68, $36.62, $39.42 and $105.72 for years 1, 2, 3, and overall, respectively. The relative BI of including XVd was 0.33%, 0.40%, 0.43%, and 0.38% percent for years 1, 2, 3, and overall, respectively. This translated to a PMPM budget impact of $0.041, $0.051, $0.054, and $0.049 (USD), for years 1, 2, 3, and overall, respectively. One-way sensitivity analyses showed general consistency with the base-case findings. Conclusions: Understanding the potential BI of new therapies in MM is vital for decision makers to manage spending and assess the value of treatments. The introduction of XVd presents a small and manageable BI for a third-party US payer and Medicare.

Cost-effectiveness analysis of magnetic resonance-guided focused ultrasound ablation for palliation of refractory painful bone metastases

2020 ◽  
pp. 1-7
Author(s):  
Matthew D. Bucknor ◽  
Frandics P. Chan ◽  
Jessica Y. Matuoka ◽  
Patti K. Curl ◽  
James G. Kahn
Keyword(s):  
Cost Effectiveness ◽  
Bone Metastases ◽  
Time Horizon ◽  
Focused Ultrasound ◽  
Case Analysis ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Base Case Analysis ◽  
Painful Bone Metastases

Abstract Objective The aim of this study was to determine if magnetic resonance-guided focused ultrasound (MRgFUS) is cost-effective compared with medication, for refractory pain from bone metastases in the United States. Methods We constructed a Markov state transition model using TreeAge Pro software (TreeAge Software, Inc., Williamstown, MA, USA) to model costs, outcomes, and the cost-effectiveness of a treatment strategy using MRgFUS for palliative treatment of painful bone metastases compared with a Medication Only strategy (Figure 1). Model transition state probabilities, costs (in 2018 US$), and effectiveness data (quality-adjusted life-years [QALYs]) were derived from available literature, local expert opinion, and reimbursement patterns at two U.S. tertiary academic medical centers actively performing MRgFUS. Costs and QALYs, discounted at three percent per year, were accumulated each month over a 24-month time horizon. One-way and probabilistic sensitivity analyses were performed. Results In the base-case analysis, the MRgFUS treatment strategy costs an additional $11,863 over the 2-year time horizon to accumulate additional 0.22 QALYs, equal to a $54,160/QALY ICER, thus making MRgFUS the preferred strategy. One-way sensitivity analyses demonstrate that for the base-case analysis, the crossover point at which Medication Only would instead become the preferred strategy is $23,341 per treatment. Probabilistic sensitivity analyses demonstrate that 67 percent of model iterations supported the conclusion of the base case. Conclusions Our model demonstrates that MRgFUS is cost-effective compared with Medication Only for palliation of painful bone metastases for patients with medically refractory metastatic bone pain across a range of sensitivity analyses.

scholarly journals The Value and Sustainability of Ocrelizumab in Relapsing Multiple Sclerosis: A Cost-Effectiveness and Budget Impact Analysis

2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Paolo Angelo Cortesi ◽  
Damiano Paolicelli ◽  
Marco Capobianco ◽  
Paolo Cozzolino ◽  
Lorenzo Giovanni Mantovani
Keyword(s):  
Multiple Sclerosis ◽  
Cost Effectiveness ◽  
Economic Impact ◽  
Impact Analysis ◽  
Budget Impact ◽  
Case Analysis ◽  
Base Case ◽  
Base Case Analysis ◽  
Effectiveness Analysis ◽  
The Cost

INTRODUCTION: The availability of ocrelizumab for the relapsing forms of multiple sclerosis (MS) in the Italian markets raised some questions about its economic impact and value compared to the alternative treatment options available.AIM: To assess the cost-effectiveness and budget impact of ocrelizumab compared to the most used second line disease modifying therapies (DMTs) in Italy.METHODS: The study was divided in two phases: Phase 1 – based on the development of a decision analytical Markov model to assess the cost-effectiveness of ocrelizumab compared to natalizumab and fingolimod, and Phase 2 – based on the development of a budget impact model to assess the economic impact of ocrelizumab in Italy. Both models used the National Health System perspective; a lifetime horizon was applied in the cost-effectiveness analysis and a 3-year time horizon in the budget impact. The cost-effectiveness analysis results were reported as incremental cost-effectiveness ratio (ICER) expressed as € per Quality Adjusted Life Year (QALY) gained, the budget impact analysis results were reported as difference in the overall budget (€) between a scenario with and without ocrelizumab.RESULTS: The two analyses reported ocrelizumab as a cost-effective option compared to natalizumab and fingolimod with a positive impact on the overall NHS budget. In the base-case analysis, the ICER was € 2,023 for ocrelizumab compared to fingolimod; while ocrelizumab resulted cost-saving compared to natalizumab. The sensitivity analysis confirmed the base-case analysis results. Further, the use of ocrelizumab was associated to a budget decrease of € 21 million (-2.6%) in a 3-year time horizon.CONCLUSION: The results of our cost-effectiveness and budget impact models reported ocrelizumab as an effective and efficient treatment in patients with relapsing forms of MS who failed a first line DMTs from the Italian NHS perspective.

Adjusting For Drug Wastage In Economic Evaluations Of New Therapies For Hematologic Malignancies: A Systematic Review Of The Literature and Implications

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2928-2928
Author(s):  
Matthew C. Cheung ◽  
Maureen E. Trudeau ◽  
Anca Prica ◽  
Rena Buckstein ◽  
Kelvin KW Chan
Keyword(s):  
Drug Acquisition ◽  
Case Analysis ◽  
Hematologic Malignancies ◽  
Sensitivity Analyses ◽  
Economic Evaluations ◽  
Base Case ◽  
Base Case Analysis ◽  
Drug Wastage ◽  
Acquisition Costs ◽  
The Cost

Abstract The cost of cancer care is rising to levels that appear unsustainable, in large part driven by a rapid increase in expenditures for novel therapies. In 2005, the direct costs of cancer care in the United States were $74 billion, with a prediction that these figures will double this decade as the population ages and we enter an age of biologically-tailored drug development. Historically, the evaluation of the cost-effectiveness of new drug therapies presumes that every milligram of drug is effectively utilized. With manufacturers dictating the availability of vial sizes for parenteral drugs, however, this may not be the case. A recent single-institution study in Canada estimated that an annual loss of $150 000 ($CDN) could be attributed to drug wastage of a parenteral therapy for myelodysplasia (Walker et al., Can J Hosp Pharm 2012). Whether current economic evaluations routinely model the potential for drug wastage, or the strategies to mitigate wastage, has not previously been determined. We sought to identify the frequency of drug wastage modeling in economic evaluations of modern parenteral therapies for hematologic malignancies. All US Food and Drug Administration (FDA) approved parenteral therapies from 2006-2013 (n=14) were identified for review. A systematic review of published English-language economic evaluations for these therapies was conducted and data on the conduct of the cost-analyses were extracted in duplicate. The primary outcome was the proportion of studies that explicitly modeled drug wastage in the base-case analysis, with results presented descriptively. Where possible, we calculated the impact of wastage on incremental cost-effectiveness ratios (ICERs) and drug acquisition costs. Thirty-three publications were retrieved; five of these studies were excluded (3 were non-English language and 2 were review articles). Of the remaining 27 reports, 3 were publicly-funded health technology assessments (HTAs) to inform funding decisions by the UK National Institute for Health and Care Excellence (NICE) and the remainder (n=24) were peer-reviewed publications. Sixteen economic evaluations were of rituximab-based regimens and 6 were bortezomib-based. Drug wastage was considered in the primary or base-case analysis in one third of the publications (9 of 27; 33%); wastage was considered in 2 of the 3 HTAs (67%) and 7 of the 24 peer-reviewed reports (29%). Of the 9 models that considered wastage in the base-case analysis, 2 completed sensitivity analyses in which no drug wastage occurred (i.e. vial sharing between patients). In both studies, consideration of wastage changed the calculated ICER. In one study, the ICER increased by 32% when wastage was incorporated into drug acquisition costs. In the second study, azacitidine (AZA) was compared to decitabine (DEC) for myelodysplasia, with both drugs provided as single-use vials and subject to substantial wastage. When no drug wastage was modeled, AZA was associated with higher costs than DEC (+$2 655 USD) and yielded an ICER of $15 528/QALY. When wastage for both drugs was considered, AZA was associated with lower costs (-$15 890) and AZA dominated DEC. Of the 18 publications that did not model wastage in the primary analysis, no study went on to model wastage in sensitivity analyses. In 4 of these 18 studies, the potential effect of wastage on drug acquisition costs could be calculated from data provided in the publication. The calculated drug acquisition costs if wastage occurred would potentially be +5-16% higher (mean +9%) than the costs modeled in the base-case analyses of these 4 studies. The potential costs associated with drug wastage are considered in only one third of modern cost-effectiveness models. The impact of wastage on calculated ICERs and drug acquisition costs is potentially substantial. The modeling of wastage in base-case and sensitivity analyses is recommended for future economic evaluations of new intravenous therapies for hematologic malignancies. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Cost-effectiveness of home versus hospital management of children at onset of type 1 diabetes: the DECIDE randomised controlled trial

BMJ Open ◽  
2021 ◽  
Vol 11 (5) ◽  
pp. e043523
Author(s):  
Zoe McCarroll ◽  
Julia Townson ◽  
Timothy Pickles ◽  
John W Gregory ◽  
Rebecca Playle ◽  
...  
Keyword(s):  
Type 1 Diabetes ◽  
Cost Effectiveness ◽  
Randomised Controlled Trial ◽  
Controlled Trial ◽  
Case Analysis ◽  
Base Case ◽  
Base Case Analysis ◽  
Home Management ◽  
Randomised Controlled

ObjectiveThe aim of this economic evaluation was to assess whether home management could represent a cost-effective strategy in the patient pathway of type 1 diabetes (T1D). This is based on the Delivering Early Care In Diabetes Evaluation trial (ISRCTN78114042), which compared home versus hospital management from diagnosis in childhood diabetes and found no statistically significant difference in glycaemic control at 24 months.DesignCost-effectiveness analysis alongside a randomised controlled trial.SettingEight paediatric diabetes centres in England, Wales and Northern Ireland.Participants203 clinically well children aged under 17 years, with newly diagnosed T1D and their carers.Outcome measuresThe base-case analysis adopted n National Health Service (NHS) perspective. A scenario analysis assessed costs from a broader societal perspective. The incremental cost-effectiveness ratio (ICER), expressed as cost per mmol/mol reduction in glycated haemoglobin (HbA1c), was based on the mean difference in costs between the home and hospital groups, divided by mean differences in effectiveness (HbA1c). Uncertainty was considered in terms of the probability of cost-effectiveness.ResultsAt 24 months postintervention, the base-case analysis showed a difference in costs between home and hospital, in favour of home management (mean difference −£2,217; 95% CI −£2825 to −£1,609; p<0.001). Home care dominated, with an ICER of £7434 (saved) per mmol/mol reduction of HbA1c. The results of the scenario analysis also favoured home management. The greatest driver of cost differences was hospitalisation during the initiation period.ConclusionsHome management from diagnosis of children with T1D who are medically stable represents a less costly approach for the NHS in the UK, without impacting clinical effectiveness.Trial registration numberISRCTN78114042.

scholarly journals Cost-effectiveness of Triple Therapy with Telaprevir for Chronic Hepatitis C Virus Patients in Germany

10.36469/9870 ◽  
2013 ◽  
Vol 1 (3) ◽  
pp. 239-253 ◽  
Author(s):  
Jona T. Stahmeyer ◽  
Svenja Schauer ◽  
Siegbert Rossol ◽  
Hans Heinrich Wedemeyer ◽  
Daniel Wirth ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Hepatitis C ◽  
Triple Therapy ◽  
Sensitivity Analyses ◽  
Healthcare Sector ◽  
Base Case ◽  
Life Years ◽  
Previously Treated Patients ◽  
Previously Treated ◽  
The Cost

Background: About 400,000-500,000 people are infected with hepatitis C in Germany. Long-term consequences are the development of liver cirrhosis and hepatocellular carcinoma. The introduction of first generation protease inhibitors has significantly improved the treatment of hepatitis C genotype 1 patients. The aim of the study was to assess the cost-effectiveness of triple therapy with telaprevir in Germany. Methods: We used a Markov model on disease progression and natural history to assess the cost-effectiveness of triple therapy with telaprevir compared to standard treatment with pegylated interferon and ribavirin. Model structure and inputs were discussed with clinical experts. Deterministic and probabilistic sensitivity analyses were performed to verify the robustness of results. Results: The base-case analyses shows that triple therapy results in higher costs (untreated patients: €48,446 vs. €30,691; previously treated patients: €63,228 vs. €48,603) and better outcomes (untreated patients: 16.85 qualily of life years [QALYs] vs. 15.97 QALYs; previously treated patients: 14.16 QALYs vs. 12.89 QALYs). The incremental cost-effectiveness ratio (ICER) was €20,131 per QALY and €30,567 per life year gained (LYG) for previously untreated patients. ICER in treatment experienced patients was €7,664 per QALY for relapse patients, €12,506 per QALY for partial responders and €28,429 per QALY for null responders. Results were robust in sensitivity analyses. Conclusion: Although triple therapy with telaprevir leads to additional costs, there is a high probability of being cost-effective for different thresholds. This health economic analysis makes an important contribution to current debates on cost savings and efficient resource allocation in the German healthcare sector.

Comparison of Cost-Effectiveness of Anticoagulation with Dabigatran, Rivaroxaban and Apixaban in Patients with Non-Valvular Atrial Fibrillation Across Countries

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 1164-1164 ◽  
Author(s):  
Martin Krejczy ◽  
Job Harenberg ◽  
Svetlana Marx ◽  
Konrad Obermann ◽  
Martin Wehling
Keyword(s):  
Cost Effectiveness ◽  
Markov Model ◽  
Case Analysis ◽  
Base Case ◽  
German Population ◽  
Base Case Analysis ◽  
Chads2 Score ◽  
Increased Risk ◽  
Markov Decision Model ◽  
The Cost

Abstract Abstract 1164 The three new oral anticoagulants (NOAC) dabigatran 110mg bid and 150mg bid, investigated in the RE-LY trial, rivaroxaban 20mg od of the ROCKET trial, and apixaban 5mg bid of the ARISTOTLE trial showed equivalent or superior efficacy and safety compared to warfarin in these patients. We performed a cost-effectiveness analysis for these NOACs in Germany and compared the quality of life (QALY), incremental cost effectiveness ratios (ICER), and total costs across those countries form where these data are published. The base case population was a hypothetical cohort of patients 65 years or older with AF who were at increased risk for stroke (CHADS2-score >1) and had no contraindications to anticoagulation. The time horizon was based on the life expectancy of the German population. The QALYs, health insurance costs, and ICER for the NOACs compared with warfarin were calculated for each study. The sensitivity analysis was performed for different base case prices. The Markov decision model was adopted using the TreeAge Pro 2011 program. The data of the outcomes of ischemic stroke and cerebral embolism, major and intracerebral haemorrhage, myocardial infarction, and mortality were taken from the 3 studies comparing the NOAC with INR-adjusted warfarin. Prices for clinical events and for outpatient care were taken from the institute for payment regulations in German hospitals (InEK). The base-case analysis of a 65 years old person with a >2 CHADS2 score using the data from the RE-LY study resulted in 11.41 QALYs for warfarin, 11.53 QALYs for dabigatran 110mg bid, 11.66 QALYs for dabigatran 150 mg bid. ICERs per QALY were 49640€ for dabigatran 110 mg bid and 49590€ for dabigatran 150 mg bid versus warfarin. The same base-case analysis using the data from the ROCKET AF study resulted in 10.79 QALYs for warfarin versus 11.05 QALYs for rivaroxaban. ICERs per QALY were 48980€ for rivaroxaban versus warfarin. The base-case analysis using the data from the ARISTOTLE study resulted and 11.04 QALYs for warfarin versus 11.38 QALYs for apixaban. ICERs per QALY were 49720€ for apixaban versus warfarin. According the Markov Model the daily value based daily prices were 1.25€ for dabigatran 110 mg bid, 2.50€ for dabigatran 150 mg bid, 2.60€ for rivaroxaban, and 3.10€ for apixaban in Germany. The model was highly sensitive to the daily costs for the NOACs but relatively insensitive to other model inputs. Calculating the range of NOAC prices from 0.2 to 10 Euro versus the ICERs dabigatran 110 mg bid produced the highest increase of ICERs over this range of daily costs (Tukey-Kramer test, p<0.05) Comparing these data across countries using the published data shows that the willingness to pay per QALY as well as differences in treatment costs between substantially influences the daily prices of the NOACs. The data demonstrate the necessity to analyse the cost-effectiveness separately for every study due to differences in the INR-adjusted warfarin treated control group. The better the outcome during treatment with warfarin the lower is the benefit of the NOAC. The tendency of the cost-effectiveness calculated by the Markov model is comparable across countries. Disclosures: No relevant conflicts of interest to declare.

Expanded analyses of napoli-1: Phase 3 study of MM-398 (nal-IRI), with or without 5-fluorouracil and leucovorin, versus 5-fluorouracil and leucovorin, in metastatic pancreatic cancer (mPAC) previously treated with gemcitabine-based therapy.

2015 ◽  
Vol 33 (3_suppl) ◽  
pp. 234-234 ◽  
Author(s):  
Li-Tzong Chen ◽  
Daniel D. Von Hoff ◽  
Chung-Pin Li ◽  
Andrea Wang-Gillam ◽  
Gyorgy Bodoky ◽  
...  
Keyword(s):  
Sensitivity Analyses ◽  
Open Label ◽  
Phase 3 ◽  
Open Label Study ◽  
Prior Therapy ◽  
Previously Treated ◽  
Grade 3 ◽  
Intent To Treat ◽  
To Receive ◽  
Clinical Trial Information

234 Background: MM-398 is a nanoliposomal encapsulation of irinotecan. OS in the ITT population was significantly longer with MM-398+5FU/LV over 5FU/LV alone, and the most frequent grade 3+ AEs included neutropenia, fatigue, and GI effects (diarrhea and vomiting). Expanded, pre-specified analyses of the Phase 3 study are presented. Methods: Patients (n=417) with mPAC previously treated with gemcitabine-based therapy, were randomized 1:1:1 in an open-label study to receive: (A) MM-398 (120 mg/m2 IV over 90 min) q3w; (B) 5FU (2,000 mg/m2 over 24 h) plus racemic leucovorin (LV) (200 mg/m2 over 30 min) x 4w followed by 2w rest; or (C) combination of MM-398 (80 mg/m2 IV over 90 min) prior to 5FU (2,400 mg/m2 over 46 h) and racemic LV (400 mg/m2 over 30 min) q2w. The primary endpoint was OS. The Intent To Treat (ITT) population included all randomized patients; the Per Protocol (PP) population included patients who received at least 80% of the target dose in the first 6 weeks and did not violate any inclusion/exclusion criteria. Results: Analysis of the PP populations confirmed the favorable OS, which was also reflected by the PFS, ORR and CA19-9 levels, of the combination MM-398+5FU/LV arm over the control 5FU/LV arm. The MM-398 monotherapy arm did not show a statistically significant improvement in OS compared with the control arm. Analysis of subgroups, based on pretreatment characteristics including stage at diagnosis, time since initial histological diagnosis, prior lines of therapy, time since last prior therapy, and CA19-9 levels, consistently favored OS for the MM-398+5FU/LV arm over the 5FU/LV arm. Conclusions: Expanded analysis of the PP population and sensitivity analyses support the favorability of MM-398+5FU/LV over 5FU/LV, with a manageable safety profile. Clinical trial information: NCT01494506. [Table: see text]

scholarly journals Budget impact analysis of a breast rapid diagnostic unit

2017 ◽  
Vol 24 (3) ◽  
pp. 214 ◽  
Author(s):  
M. Elmi ◽  
H. Hussain ◽  
S. Nofech-Mozes ◽  
B. Curpen ◽  
A. Leahey ◽  
...  
Keyword(s):  
Health Care ◽  
Impact Analysis ◽  
Budget Impact ◽  
Tertiary Care ◽  
Resource Planning ◽  
Budget Impact Analysis ◽  
Sensitivity Analyses ◽  
Definitive Treatment ◽  
Base Case ◽  
Diagnostic Unit

Background The Odette Cancer Centre’s recent implementation of a rapid diagnostic unit (rdu) for breast lesions has significantly decreased wait times to diagnosis. However, the economic impact of the unit remains unknown. This project defined the development and implementation costs and the operational costs of a breast rdu in a tertiary care facility.Methods From an institutional perspective, a budget impact analysis identified the direct costs associated with the breast rdu. A base-case model was also used to calculate the cost per patient to achieve a diagnosis. Sensitivity analyses computed costs based on variations in key components. Costs are adjusted to 2015 valuations using health care–specific consumer price indices and are reported in Canadian dollars.Results Initiation cost for the rdu was $366,243. The annual operational cost for support staff was $111,803. The average per-patient clinical cost for achieving a diagnosis was $770. Sensitivity analyses revealed that, if running at maximal institutional capacity, the total annual clinical cost for achieving a diagnosis could range between $136,080 and $702,675.Conclusions Establishment and maintenance of a breast rdu requires significant investment to achieve reductions in time to diagnosis. Expenditures ought to be interpreted in the context of institutional patient volumes and trade-offs in patient-centred outcomes, including lessened patient anxiety and possibly shorter times to definitive treatment. Our study can be used as a resource-planning tool for future rdus in health care systems wishing to improve diagnostic efficiency.

scholarly journals Cost-Effectiveness Analysis of Gilteritinib Versus Salvage Chemotherapy (SC) for the Treatment of Relapsed or Refractory (R/R) FLT3-Mutated (FLT3mut+) Acute Myeloid Leukemia (AML)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3859-3859 ◽  
Author(s):  
Amer M. Zeidan ◽  
Cynthia Z. Qi ◽  
Bhavik J. Pandya ◽  
Andy Garnham ◽  
Hongbo Yang ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Research Funding ◽  
Third Party ◽  
Sensitivity Analyses ◽  
Survival Models ◽  
Base Case ◽  
Health State ◽  
Lifetime Horizon ◽  
Analysis Group

Introduction: FLT3 is a frequently mutated gene in approximately one-third of AML cases and is associated with a poor prognosis. Few effective therapeutic options exist for patients with R/R FLT3mut+ AML. In 2018, gilteritinib was approved in the US as the first targeted therapy indicated for R/R FLT3mut+ AML. The efficacy of gilteritinib was established in the ADMIRAL trial, a phase 3 randomized trial, in comparison with SC (i.e., low-dose cyctarbine [LDAC], azacitidine, mitoxantrone + etoposide + cytarabine [MEC], and fludarabine + cytarabine + granulocyte colony stimulating factor + idarubicin [FLAG-IDA]). The results from the trial indicated that gilteritinib significantly improved overall survival (OS) compared to SC with 1-year survival rates of 37% vs. 17%. To inform the value of gilteritinib, this study aimed to assess the cost-effectiveness of gilteritinib for the treatment of R/R FLT3mut+ AML from a US third-party payer's perspective. Methods: A cost-effectiveness analysis (CEA) model was developed using monthly cycles and a 3% discount rate to assess the incremental cost effectiveness of gilteritinib compared to SC over a lifetime horizon. The model structure comprised a decision tree followed by two three-state partitioned survival models. The decision tree component stratified patients based on whether they received allogeneic hematopoietic stem cell transplantation (HSCT) following treatment initiation. The partitioned survival components included three health states: event-free survival (EFS), alive and post-event, and death. The selected model structure was chosen because HSCT is a key clinical event that has a significant impact on treatment outcomes for the target population. The efficacy inputs (OS and EFS) varied by HSCT status. The efficacy inputs for OS and EFS without HSCT were estimated based on the ADMIRAL trial. The efficacy inputs for OS and EFS with HSCT were assumed to be the same for both gilteritinib and SC and were based on available literature (Evers 2018). Parametric survival models or HRs were used to predict probabilities of being in different health states until year 3. Afterwards, all patients who remained alive were considered long-term survivors and their mortality risk was twice that of the general population based on literature. Treatment costs (drug, administration, and hospitalization), adverse event (AE) costs, subsequent HSCT costs, medical costs for each health state, FLT3 mutation testing, post-progression treatment, and terminal care costs were obtained from public databases and literature. For SC, the same regimen composition (i.e., LDAC, azacitidine, MEC, and FLAG-IDA) as the ADMIRAL trial was considered to estimate the treatment costs. All costs were inflated to 2018 US dollar (USD). Utilities for each health state were derived from the ADMIRAL trial and disutilities for AEs were derived from the literature. Total incremental costs, total incremental LYs, and total incremental QALYs were calculated. Incremental cost-effectiveness ratios (ICERs), defined as the incremental cost per additional gain in health effect (i.e., LY and QALY), were used to assess the economic value of gilteritinib relative to SC. Deterministic sensitivity analyses (DSA) and probabilistic sensitivity analyses (PSA) were also performed to test the robustness of the base-case results. Results: Over a lifetime horizon, the base-case model estimated that treatment with gilteritinib led to an increase of 1.55 discounted LYs and an increase of 1.29 discounted QALYs at an additional cost of $141,097 relative to SC; the corresponding incremental cost per LY gained was $90,761, and incremental cost per QALY gained was $109,741. Cost-effectiveness was most sensitive to gilteritinib cost, HSCT rate, and discount rate. In the PSA, the estimated probability that gilteritinib is cost-effective was 93.5% at an acceptable willingness-to-pay threshold of $150,000/QALY. Conclusions: Gilteritinib demonstrated greater LY and QALY improvements compared with SC. With an ICER of $109,741/QALY, gilteritinib is a cost-effective strategy from a US third-party payer's perspective, based on the $150,000/QALY threshold recommended by the US Institute for Clinical and Economic Review. Compared to SC, gilteritinib represents a new active treatment option for R/R FLT3mut+ AML patients that provides better health outcomes with a favorable cost-effectiveness profile. Disclosures Zeidan: Trovagene: Consultancy, Honoraria, Research Funding; Medimmune/AstraZeneca: Research Funding; Boehringer-Ingelheim: Consultancy, Honoraria, Research Funding; Takeda: Consultancy, Honoraria, Research Funding; Ariad: Honoraria; Novartis: Honoraria; Astellas: Honoraria; Daiichi Sankyo: Honoraria; Cardinal Health: Honoraria; Seattle Genetics: Honoraria; BeyondSpring: Honoraria; Incyte: Consultancy, Honoraria, Research Funding; Acceleron Pharma: Consultancy, Honoraria, Research Funding; Celgene Corporation: Consultancy, Honoraria, Research Funding; Abbvie: Consultancy, Honoraria, Research Funding; Otsuka: Consultancy, Honoraria, Research Funding; Agios: Honoraria; Pfizer: Consultancy, Honoraria, Research Funding; ADC Therapeutics: Research Funding; Jazz: Honoraria. Qi:Astellas: Other: I am an employee of Analysis Group, Inc., which provided paid consulting services to Astellas for the conduct of this study; Analysis Group, Inc.: Employment. Pandya:Astellas Pharmaceuticals: Employment. Garnham:Astellas: Employment. Yang:Analysis Group, Inc.: Employment; Astellas: Other: I am an employee of Analysis Group, Inc., which provided paid consulting services to Astellas for the conduct of this study. Shah:Astellas: Employment.

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