Greater Quality-adjusted Life Years Research Articles

Cost-effectiveness analysis of mosunetuzumab for treatment of relapsed or refractory follicular lymphoma after two or more lines of systemic therapy in the United States

Aims Mosunetuzumab has received accelerated approval by the US Food and Drug Administration for adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy. We evaluated the cost-effectiveness of mosunetuzumab for the treatment of R/R FL from a US private payer perspective. Materials and methods A partitioned survival model simulated lifetime costs and outcomes of mosunetuzumab against seven comparators: axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), tazemetostat (taz, EZH2 wild-type only), rituximab plus lenalidomide (R-Len) or bendamustine (R-Benda), obinutuzumab plus bendamustine (O-Benda), and a retrospective real-world cohort (RW) based on current patterns of care derived from US electronic health records (Flatiron Health). Efficacy data for mosunetuzumab were from the pivotal Phase II GO29781 trial (NCT02500407). Relative treatment efficacy was estimated from indirect treatment comparisons (ITCs). Costs included were related to treatment, adverse events, routine care, and terminal care. Except for drug costs (March 2023), all costs were inflated to 2022 US dollars. Costs and quality-adjusted life-years (QALYs) were used to calculate incremental cost-effectiveness ratios (ICERs). Net monetary benefit (NMB) was calculated using a willingness-to-pay (WTP) threshold of $150,000/QALY. Results Mosunetuzumab dominated taz, tisa-cel, and axi-cel with greater QALYs and lower costs. Mosunetuzumab was projected to be cost-effective against R-Benda, O-Benda, and RW with ICERs of $78,607, $42,731, and $21,434, respectively. Mosunetuzumab incurred lower costs but lower QALYs vs. R-Len. NMBs showed that mosunetuzumab was cost-effective against comparators except R-Len. Limitations Without head-to-head comparative data, the model had to rely on ITCs, some of which were affected by residual bias. Model inputs were obtained from multiple sources. Extensive sensitivity analyses assessed the importance of these uncertainties. Conclusion Mosunetuzumab is estimated to be cost-effective compared with approved regimens except R-Len for the treatment of adults with R/R FL.

Cost‐effectiveness of maribavir versus conventional antiviral therapies for post‐transplant refractory cytomegalovirus infection with or without genotypic resistance: A US perspective

AbstractThis study evaluated the cost‐effectiveness of maribavir versus investigator‐assigned therapy (IAT; valganciclovir/ganciclovir, foscarnet, or cidofovir) for post‐transplant refractory cytomegalovirus (CMV) infection with or without resistance. A two‐stage Markov model was designed using data from the SOLSTICE trial (NCT02931539), real‐world multinational observational studies, and published literature. Stage 1 (0–78 weeks) comprised clinically significant CMV (csCMV), non‐clinically significant CMV (n‐csCMV), and dead states; stage 2 (78 weeks–lifetime) comprised alive and dead states. Total costs (2022 USD) and quality‐adjusted life years (QALYs) were estimated for the maribavir and IAT cohorts. An incremental cost‐effectiveness ratio was calculated to determine cost‐effectiveness against a willingness‐to‐pay threshold of $100 000/QALY. Compared with IAT, maribavir had lower costs ($139 751 vs $147 949) and greater QALYs (6.04 vs 5.83), making it cost‐saving and more cost‐effective. Maribavir had higher acquisition costs compared with IAT ($80 531 vs $65 285), but lower costs associated with administration/monitoring ($16 493 vs $27 563), adverse events (AEs) ($11 055 vs $16 114), hospitalization ($27 157 vs $33 905), and graft loss ($4516 vs $5081), thus making treatment with maribavir cost‐saving. Maribavir‐treated patients spent more time without CMV compared with IAT‐treated patients (0.85 years vs 0.68 years), leading to lower retreatment costs for maribavir (cost savings: −$42 970.80). Compared with IAT, maribavir was more cost‐effective for transplant recipients with refractory CMV, owing to better clinical efficacy and avoidance of high costs associated with administration, monitoring, AEs, and hospitalizations. These results can inform healthcare decision‐makers on the most effective use of their resources for post‐transplant refractory CMV treatment.

Cost-effectiveness analysis of dupilumab versus omalizumab, mepolizumab, and benralizumab added to the standard of care in adults with severe asthma in Colombia

ABSTRACT Background Cost-effectiveness studies evaluate health technologies and help choose treatments. The current study compared dupilumab to omalizumab, mepolizumab, and benralizumab in Colombian adults with severe uncontrolled type 2 asthma. Methods Over a 5-year period, a Markov model was utilized to assess the costs of biological treatments and management of exacerbations, comparing various doses of exacerbations, comparing various doses of dupilumab, omalizumab, mepolizumab, and benralizumab as add-on treatments. It included a 5% annual discount rate per local HTA, and set willingness-to-pay at three times GDP per capita per quality-adjusted life year (QALY) in Colombia. Results Dupilumab (200 mg) exhibited greater QALYs and reduced overall costs compared to mepolizumab (100 mg), benralizumab (30 mg), and omalizumab (450 mg and 600 mg), with the incremental cost-effectiveness ratio (ICER) per QALYgained being -$5.429, -$6.269, -$196.567 and -$991.007, respectively. Dupilumab had greater QALYs and costs versus omalizumab 300 mg (ICERof $200.653 per QALY, above the willingness-to-pay threshold of 3 × GDP per capita). Sensitivity analyses were consistent with base case results. Conclusions Dupilumab 200 mg was strongly dominant versus omalizumab 450 mg and 600 mg, mepolizumab 100 mg, and benralizumab 30 mg; however, cost-effectiveness was not demonstrated versus omalizumab 300 mg. These results could assist healthcare professionals in choosing an appropriate biologic for treating severe type 2 asthma.

Ibrutinib Plus RCHOP versus RCHOP Only in Young Patients with Activated B-Cell-like Diffuse Large B-Cell Lymphoma (ABC-DLBCL): A Cost-Effectiveness Analysis.

The standard treatment for Diffuse Large B-Cell Lymphoma (DLBCL) is rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (RCHOP). However, many patients require subsequent treatment after relapsed disease. The ABC subtype of DLBCL (ABC-DLBCL) has a worse prognosis, and the PHOENIX trial explored adding ibrutinib to RCHOP for this patient population. The trial showed favorable outcomes for younger patients, and our study aimed to inform clinical decision-making via a cost-effectiveness model to compare RCHOP with and without ibrutinib (I-RCHOP). A Markov decision analysis model was designed to compare the treatments for patients younger than 60 years with ABC-DLBCL. The model considered treatment pathways, adverse events, relapses, and death, incorporating data on salvage treatments and novel therapies. The results indicated that I-RCHOP was more cost-effective, with greater quality-adjusted life years (QALY, 15.48 years vs. 14.25 years) and an incremental cost-effectiveness ratio (ICER) of CAD 34,111.45/QALY compared to RCHOP only. Sensitivity analyses confirmed the model's robustness. Considering the high market price for ibrutinib, I-RCHOP may be more costly. However, it is suggested as the preferred cost-effective strategy for younger patients due to its benefits in adverse events, overall survival, and quality of life. The decision analytic model provided relevant and robust results to inform clinical decision-making.

Cost-effectiveness of FOLFOX6+Bevacizumab Versus FOLFOX6+Cetuximab in Stage IV Colorectal Cancer Patients in Shiraz, Iran.

Colorectal cancer is one of the most common cancers in the world, with about one million cases diagnosed annually. Various treatment methods can be used to treat colorectal cancer, including chemotherapy with different drug regimens. Considering the need to opt for more effective and less expensive drugs in the treatment of this disease, the present study aimed to compare the cost-effectiveness of FOLFOX6+Bevacizumab with FOLFOX6+Cetuximab in patients with stage IV colorectal cancer referred to medical centers in Shiraz, Iran, in 2021. Using a decision tree, the cost-effectiveness and cost-utility of the 2 drug regimens were compared in all studied patients through the census method. Having a societal perspective, this study considered direct medical costs, direct non-medical costs, and indirect costs. The effectiveness indicators included the rate of major response to the drug combination used and the Quality-adjusted Life Year (QALY). The data were analyzed using Treeage 2011 and Excel 2016 software. In order to ensure the robustness of the results, one-way and probabilistic sensitivity analyses were performed as well. The results showed that the expected costs, the effectiveness (major response rate), and the QALYs of the FOLFOX6+Bevacizumab drug regimen were $16746.13(USD), .49, and .19, respectively, and those of the FOLFOX6+Cetuximab regimen were, respectively, $15191.05 (USD), .68, and .22. Therefore, FOLFOX6+Cetuximab compared to FOLFOX6+Bevacizumab was less costly and more effective and had a greater QALY, thus being considered as the dominant option. Also, the results of the sensitivity analyses showed that there was a bit of uncertainty. Considering that the FOLFOX6+Cetuximab regimen was more cost-effective, it is suggested to be prioritized in preparing clinical guidelines for Iranian colorectal cancer patients. In addition, increasing the basic and supplementary insurance coverage for this drug combination as well as the use of remote technology to guide patients by oncologists can be solutions to reduce direct and indirect costs of the patients.

Cost-Utility Analysis of a Medication Adherence-Enhancing Educational Intervention for Glaucoma

To evaluate the cost utility of a glaucoma medication-enhancing intervention compared to standard of care over a lifetime from the United States Department of Veterans Affairs (VA) payer perspective. Model-based cost-utility analysis of a glaucoma medication-enhancing intervention from a randomized clinical trial. Veterans with glaucoma, or suspected glaucoma who were prescribed topical glaucoma medications, had their visual field assessed within the last 9 months, and endorsed poor glaucoma medication adherence. Veterans were randomized either to a behavioral intervention to promote adherence or to a standard of care (control) session about general eye health. A decision analytic model was developed to simulate lifelong costs and quality-adjusted life years (QALYs) for an intervention tested in a randomized clinical trial at a single VA eye clinic. Costs included direct medical costs that the VA payer would incur, as informed initially by the clinical trial and then by published estimates. Health-state quality of life was based on published utility values. Scenario analyses included addition of booster interventions, a 3% decline in chance of staying medication adherent annually, and the combination of the two. Analyses were also conducted in the following subgroups: those with companion versus not, and those with once-daily versus more than once-daily dosing frequency. Incremental cost-effectiveness ratio (ICER). Compared to standard of care, the intervention dominated resulting in lower costs ($23 339.28 versus $23 504.02) and higher QALYs (11.62 versus 11.58). Among the 4 subgroups, the intervention dominated for 3 of them. In the fourth subgroup, those with more than once-daily dosing, the ICER was $2625/QALY. Compared to standard of care, an intervention with booster interventions led to an ICER of $3278/QALY. Assuming both a 3% annual loss in chance of continuing to be adherent and addition of booster interventions, the ICER increased to $71 371/QALY. From a VA payer perspective over a lifetime, the glaucoma medication-enhancing behavioral intervention dominated standard of care in terms of generating cost savings and greater QALYs. Proprietary or commercial disclosure may be found after the references.

Economic Evaluation: A Randomized Pragmatic Trial of a Primary Care-based Cognitive Behavioral Intervention for Adults Receiving Long-term Opioids for Chronic Pain.

Chronic pain is prevalent and costly; cost-effective nonpharmacological approaches that reduce pain and improve patient functioning are needed. Report the incremental cost-effectiveness ratio (ICER), compared with usual care, of cognitive behavioral therapy aimed at improving functioning and pain among patients with chronic pain on long-term opioid treatment. Economic evaluation conducted alongside a pragmatic cluster randomized trial. Adults with chronic pain on long-term opioid treatment (N=814). A cognitive behavioral therapy intervention teaching pain self-management skills in 12 weekly, 90-minute groups delivered by an interdisciplinary team (behaviorists, nurses) with additional support from physical therapists, and pharmacists. Cost per quality adjusted life year (QALY) gained, and cost per additional responder (≥30% improvement on standard scale assessment of Pain, Enjoyment, General Activity, and Sleep). Costs were estimated as-delivered, and replication. Per patient intervention replication costs were $2145 ($2574 as-delivered). Those costs were completely offset by lower medical care costs; inclusive of the intervention, total medical care over follow-up was $1841 lower for intervention patients. Intervention group patients also had greater QALY and responder gains than did controls. Supplemental analyses using pain-related medical care costs revealed ICERs of $35,000, and $53,000 per QALY (for replication, and as-delivered intervention costs, respectively); the ICER when excluding patients with outlier follow-up costs was $106,000. Limited to 1-year follow-up; identification of pain-related utilization potentially incomplete. The intervention was the optimal choice at commonly accepted levels of willingness-to-pay for QALY gains; this finding was robust to sensitivity analyses.

IDENTIFYING THE MOST EFFECTIVE TREATMENT ALGORITHM FOR THERAPIES TO TREAT MODERATE TO SEVERE ULCERATIVE COLITIS

Abstract BACKGROUND There has been an ongoing expansion in available biologic and small molecule therapies to treat moderate to severe ulcerative colitis (UC). The sequence in which these therapies are used has been a topic of high interest among clinicians. In the absence of precision medicine for UC, it would be useful to define the sequence of therapies that may predict the best outcomes. Limited data exists regarding the most effective treatment algorithms for achieving optimal patient outcomes. As such, this study endeavors to identify treatment algorithms when considering the UC therapies of vedolizumab, infliximab, ustekinumab, and tofacitinib. METHODS A Markov model was used to identify the UC treatment algorithm that would potentially yield the greatest quality adjusted life years (QALYs) over 1 year. The base case was a 35-year old male with moderate to severe UC who had not previously received biologic or immunomodulator therapy. Four medical therapies were considered: (1) infliximab + azathioprine, (2) tofacitinib, (3) ustekinumab, and (4) vedolizumab. Colectomy was assumed to be the least desirable treatment option. These four therapies were combined in eight separate potential treatment algorithms (Figure 1). Tofacitinib was only included after anti-TNF therapy per its USA prescribing indication. With each therapy, simulated individuals could enter remission or response, develop a significant adverse event or infection, or lose response to the therapy. Transition probabilities for response, remission, and adverse event rates were derived from relevant clinical trials. Lymphoproliferative risks were derived from age-specific SEER (Surveillance, Epidemiology, and End Results) data and treatment-specific risks from CESAME. Utility estimates were derived from previously published studies. The time horizon was 1 year. Primary analyses consisted of first order Monte Carlo simulation of 100 trials of cohorts consisting of 100,000 individuals. RESULTS Algorithms incorporating vedolizumab as first-line therapy resulted in the greatest quality of life and proportion of patients in remission at 1 year in comparison to strategies where combination therapy with infliximab + azathioprine or ustekinumab were included as first line therapy (Table 1). Vedolizumab followed by infliximab + azathioprine and then ustekinumab resulted in the greatest quality of life advantage. The benefit of this treatment sequence over other sequences ranged from 0.008 to 0.011 QALYs at 1 year. CONCLUSION In this model, using vedolizumab prior to infliximab + azathioprine, ustekinumab and tofacitinib is predicted to yield greater quality of life and a greater percentage of patients with clinical remission at the end of 1 year. Further analyses assessing model inputs, probabilistic analyses, and strategy cost-effectiveness are required.

What's in a Name? A Cost-Effectiveness Analysis of the Noninvasive Follicular Thyroid Neoplasm with Papillary-Like Nuclear Features' Nomenclature Revision.

Background: The noninvasive subtype of encapsulated follicular variant of papillary thyroid carcinoma (eFVPTC) has been reclassified as noninvasive follicular thyroid neoplasm with papillary-like nuclear features (NIFTP) in 2016 to reflect the indolent behavior and favorable prognosis of this type of tumor. This terminology change has also de-escalated its management approach from cancer treatment to a more conservative treatment strategy befitting a benign thyroid neoplasm. Objective: To characterize the reduced health care costs and improved quality of life (QOL) from management of NIFTP as a nonmalignant tumor compared with the previous management as eFVPTC. Methods: A cost-effectiveness analysis was performed by creating Markov models to simulate two management strategies for NIFTP: (i) de-escalated management of the tumor as NIFTP involving lobectomy with reduced follow-up, (ii) management of the tumor as eFVPTC involving completion thyroidectomy/radioactive iodine ablation for some patients, and follow-up recommended for carcinoma. The model was simulated for 5 and 20 years following diagnosis of NIFTP. Aggregate costs and quality-life years were measured. One-way sensitivity analysis was performed for all variables. Results: Over a five-year simulation period, de-escalated management of NIFTP had a total cost of $12,380.99 per patient while the more aggressive management of the tumor as eFVPTC had a total cost of $16,264.03 per patient (saving $3883.05 over five years). Management of NIFTP provided 5.00 quality-adjusted life years, whereas management as eFVPTC provided 4.97 quality-adjusted life years. Sensitivity analyses showed that management of NIFTP always resulted in lower costs and greater quality-adjusted life years (QALYs) over the sensitivity ranges for individual variables. De-escalated management for NIFTP is expected to produce ∼$6-42 million in cost savings over a five-year period for these patients, and incremental 54-370 QALYs of increased utility in the United States. Conclusion: The degree of cost savings and improved patient utility of de-escalated NIFTP management compared with traditional management was estimated to be $3883.05 and 0.03 QALYs per patient. We demonstrate that these findings persisted in sensitivity analysis to account for variability in recurrence rate, surveillance approaches, and other model inputs. These findings allow for greater understanding of the economic and QOL impact of the NIFTP reclassification.

Which injected antiosteoporotic medication is worth paying for? A cost-effectiveness analysis of teriparatide, zoledronate, ibandronate, and denosumab for postmenopausal osteoporotic women in China.

The purpose of this study was to evaluate the cost-effectiveness of four injected antiosteoporotic medications including teriparatide, zoledronate, ibandronate, and denosumab for postmenopausal osteoporotic women in China. A Markov microsimulation model was used to compare the cost-effectiveness of the four drugs above in Chinese postmenopausal osteoporotic women with no fracture history of hip, vertebral, or wrist at various ages (65, 70, 75, and 80) of therapy initiation from the health care payer perspective. Denosumab was dominant (ie, lower costs and greater quality-adjusted life-years [QALYs]) compared with other strategies at all ages studied. The incremental cost-effectiveness ratios (ICERs) of zoledronate or ibandronate versus no treatment were $4,482.88/ QALYs or $11,378/QALYs, respectively, at age 65 years, and the results at other ages were similar. In contrast, the incremental cost-effectiveness ratio of teriparatide strategy compared with no treatment exceeded the pre-determined threshold of a willingness-to-pay of $31,512/QALY regardless of the adoption of the patient assistance program at all ages studied, and a threshold analysis showed that teriparatide without patient assistance program became cost-effective when the annual drug cost is decreased to $1,644.87 (current cost: $8,764.65). The cost-effectiveness decision did not change in most of the one-way sensitivity analyses. A scenario analysis considering no offset effect of denosumab showed that zoledronate had the potential to become the optimal option relative to denosumab. In probabilistic sensitivity analyses, the probabilities of denosumab being cost-effective compared with other strategies were 100% at a willingness-to-pay of $31,512/QALY. Among postmenopausal osteoporotic women in China, denosumab therapy is cost-effective at all ages examined from the health care payer perspective, compared with teriparatide, zoledronate, or ibandronate. This study will help clinicians and policymakers make better decisions about the relative economic value of osteoporosis treatments in China.

PD55-02 MOLECULAR SUBTYPING TO STRATIFY THE TREATMENT OF MUSCLE-INVASIVE BLADDER CANCER: A COST EFFECTIVENESS ANALYSIS

You have accessJournal of UrologyBladder Cancer: Invasive IV (PD55)1 Sep 2021PD55-02 MOLECULAR SUBTYPING TO STRATIFY THE TREATMENT OF MUSCLE-INVASIVE BLADDER CANCER: A COST EFFECTIVENESS ANALYSIS Diana Magee, Douglas Cheung, Beate Sander, and Girish Kulkarni Diana MageeDiana Magee More articles by this author , Douglas CheungDouglas Cheung More articles by this author , Beate SanderBeate Sander More articles by this author , and Girish KulkarniGirish Kulkarni More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000002089.02AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: The gold standard treatment for muscle invasive bladder cancer (MIBC) is neoadjuvant chemotherapy (NAC) followed by radical cystectomy. However, response to NAC is unpredictable. Molecular subtypes allow for an improved ability to select a tailored treatment course. Our study aimed to assess the cost-effectiveness of molecular subtyping in the management of MIBC. METHODS: A two-dimensional Markov microsimulation model was developed using TreeAge Pro comparing three strategies: NAC at current usage rates (36%), universal NAC usage, and molecular subtype-directed care. Model probabilities and utilities were derived from the published literature. Four distinct subtypes of MIBC were modelled based on the Seiler classification. Cost of each phase of care was obtained from primary data and the Canadian Institute for Health Information patient cost estimator. The primary outcomes were quality-adjusted life years (QALYs), cost (CAD), overall survival (OS) and the incremental cost-effectiveness ratio (ICER). RESULTS: The predicted QALYs were 8.34, 8.73, and 9.14 with costs of $62,478, $76,962, and $62,579 for NAC at current usage rates, universal NAC usage, and subtype-directed care, respectively. Probabilistic sensitivity analysis shown in Figure 1. OS at 10 years was 39.2%, 40.8% and 42.8% for NAC at current usage rates, universal NAC usage, and subtype-directed care, respectively. When comparing subtype-directed care to current rates of NAC usage the ICER was $127/QALY. Subtype-directed care dominated universal NAC usage. CONCLUSIONS: We demonstrated that in patients with MIBC a molecular subtype directed approach to the administration of NAC results in improved OS and greater QALYs; moreover, it is cost-effective within a single payer healthcare system. A push for the universal use of NAC will result in improved survival compared with what our current rates of use achieve but it is likely not the best approach considering the drawbacks of chemotherapy including toxicity and unequal response. This model is built upon the available literature and as such requires validation prior to clinical implementation. Source of Funding: This project was funded by a grant from the Canadian Urologic Oncology Group © 2021 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 206Issue Supplement 3September 2021Page: e997-e997 Advertisement Copyright & Permissions© 2021 by American Urological Association Education and Research, Inc.MetricsAuthor Information Diana Magee More articles by this author Douglas Cheung More articles by this author Beate Sander More articles by this author Girish Kulkarni More articles by this author Expand All Advertisement Loading ...

Optimizing Costs and Outcomes for Carpal Tunnel Release Surgery: A Cost-Effectiveness Analysis from Societal and Health-Care System Perspectives.

It is unclear which carpal tunnel release (CTR) strategy (i.e., which combination of surgical technique and setting) is most cost-effective. A cost-effectiveness analysis was performed to compare (1) open CTR in the procedure room (OCTR/PR), (2) OCTR in the operating room (OCTR/OR), and (3) endoscopic CTR in the operating room (ECTR/OR). A decision analytic model was used to compare costs and health utilities between treatment strategies. Utility and probability parameters were identified from the literature. Medical costs were estimated with Medicare ambulatory surgical payment data. Indirect costs were related to days out of work due to surgical recovery and complications. The effectiveness outcome was quality-adjusted life years (QALYs). Probabilistic sensitivity analyses and one-way sensitivity analyses were performed. Cost-effectiveness was assessed from the societal and health-care system perspectives with use of a willingness-to-pay threshold of $100,000/QALY. In the base-case analysis, OCTR/PR was more cost-effective than OCTR/OR and ECTR/OR from the societal perspective. The mean total costs and QALYs per patient were $29,738 ± $4,098 and 0.88 ± 0.08 for OCTR/PR, $30,002 ± $4,098 and 0.88 ± 0.08 for OCTR/OR, and $41,311 ± $4,833 and 0.87 ± 0.08 for ECTR/OR. OCTR/PR was also the most cost-effective strategy from the health-care system perspective. These findings were robust in the probabilistic sensitivity analyses: OCTR/PR was the dominant strategy (greater QALYs at a lower cost) in 55% and 61% of iterations from societal and health-care system perspectives, respectively. One-way sensitivity analysis demonstrated that OCTR/PR and OCTR/OR remained more cost-effective than ECTR/OR from a societal perspective under the following conditions: $0 surgical cost of ECTR, 0% revision rate following ECTR, equalization of the return-to-work rate between OCTR and ECTR, or 0 days out of work following ECTR. OCTR/OR became more cost-effective than OCTR/PR with the median nerve injury rate tripling and doubling from societal and health-care system perspectives, respectively, or if surgical direct costs in the PR exceeded those in the OR. Compared with OCTR/OR and ECTR/OR, OCTR/PR minimizes costs to the health-care system and society while providing favorable outcomes. Economic and Decision Analysis Level III. See Instructions for Authors for a complete description of levels of evidence.

Cost-effectiveness of zanubrutinib versus ibrutinib in adult patients with Waldenström macroglobulinemia in the United States.

e18856 Background: The efficacy of zanubrutinib and ibrutinib was examined in the randomized ASPEN trial (NCT03053440) in adult patients with Waldenström macroglobulinemia (WM). This analysis assessed the cost-effectiveness (CE) of zanubrutinib vs ibrutinib in this population from a US payer perspective. Methods: A 3-state (pre-progression, post-progression, and death) partitioned survival model was used to estimate the life years (LYs), quality-adjusted life years (QALYs), and costs for each treatment over a 30-year lifetime horizon. Overall survival (OS), progression-free survival (PFS), and time-to-discontinuation (TTD) curves were fitted using parametric distributions to extrapolate long-term outcomes. Selection of the parametric models for each outcome and treatment was based on assessments of 1) the proportional hazard assumption, 2) goodness-of-fit, and 3) clinical plausibility of extrapolated mean OS and associated hazard patterns (based on literature and US clinical expert input) and the alignment between PFS and TTD. Background US mortality was accounted for in the model. Utilities were based on the ASPEN EQ-5D data and literature. Costs (2020 US$) included drug (wholesale acquisition cost from RED BOOK) and adverse event management (Healthcare Cost and Utilization Project) for zanubrutinib and ibrutinib, routine care, and terminal care. Sensitivity analyses were conducted to evaluate the impact of parameter uncertainty. All outcomes were discounted at 3% annually. Results: In the base case analyses using the dependent exponential model for all outcomes over a 30-year time horizon, zanubrutinib led to 0.94 LY and 0.84 QALY gained with an additional total drug cost of $11,132. This additional cost was primarily driven by patients staying on zanubrutinib treatment longer as zanubrutinib has longer time to treatment failure. However, this is partially offset by zanubrutinib’s lower monthly drug acquisition, reduced cost of routine care (–$2,935) and terminal care (–$2,964) than ibrutinib. The incremental cost-effectiveness ratio (ICER) of zanubrutinib is $13,205 per QALY gained. The deterministic sensitivity analyses showed that ICER was most sensitive to the monthly costs of routine care. The probabilistic sensitivity analyses showed that the mean probabilistic ICER was $16,804, and that the probability of zanubrutinib being cost-effective was 61% at a willingness-to-pay threshold of $100,000 per QALY gained. Varying the time horizon to 5, 10, or 15 years consistently led to zanubrutinib being dominant (i.e., greater QALYs but lower costs). Conclusions: Zanubrutinib appears to be cost-effective compared with ibrutinib for the treatment of patients with WM in the US.[Table: see text]

Cost-Effectiveness Analysis of Gemtuzumab Ozogamicin for First-Line Treatment of Patients with Cd-33 Positive Acute Myeloid Leukaemia in Spain.

ObjectiveTo assess the incremental cost–utility ratio (ICUR) of gemtuzumab ozogamicin (GO) + standard of care (SOC) vs SOC alone for treatment of patients with de novo AML from a Spanish Health Service perspective.MethodsA cohort state-transition model, with 12 health-states, was used to estimate the lifetime accumulated cost and benefits in terms of quality-adjusted-life-years (QALYs) in AML patients with favourable, intermediate, and unknown cytogenetic profiles. Patient profile was defined based on the ALFA-0701 trial. Therapeutic regimens were defined by 5 haematologists. SOC was assumed to be idarubicin and cytarabine, the combination most used in Spain. QALYs were estimated by applying utilities for the time spent by the cohort in each health-state and utility decrements associated with adverse events (AE). Total cost (€,2020) included drug-acquisition, hematologic stem-cell transplantation, disease management, AE management and end-of-life costs. Unit costs were derived from local databases. All parameters were validated by haematologist. Costs and outcomes were discounted (3%/year).ResultsHigher cost/patient (€177,618 vs €151,434) and greater QALYs (5,70 vs 4,62) were obtained with GO+SOC vs SOC. The ICUR was €24,203/QALY gained.ConclusionThis simulation suggests that GO + SOC could be a cost-effective option for treatment of patients with de novo AML in first line.

High-Expenditure Disease in the EU-28: Does Drug Spend Correspond to Clinical and Economic Burden in Oncology, Autoimmune Disease and Diabetes?

BackgroundDrug costs are increasing in Europe, and there is a heightened need to reduce pressure on healthcare systems. In 2017, oncology, autoimmune disease, and diabetes featured as the three highest therapy areas for drug spend in the EU-28. However, the absolute 1-year drug spend growth for diabetes did not feature within the ten fastest growing therapy areas.ObjectiveThis study explores the association between drug spend and disease burden in oncology, autoimmune disease, and diabetes in the EU-28.MethodsOncology, autoimmune disease and diabetes therapeutic areas were investigated using four methodologies. Historical and forecasted drug spend was analysed using the IQVIA MIDAS® drug sales database. Clinical and economic burden was estimated from targeted literature reviews. Trend analyses compared changes in drug spend with clinical burden using the Global Burden of Disease tool as the epidemiological reference. Cost per quality-adjusted life-years (QALYs) from UK health technology assessments were compared to interpret the health economic value.ResultsOncology had the highest historical drug spend and growth compared with autoimmune disease and diabetes. Total drug spend and growth in oncology is forecasted to exceed diabetes by twofold. Increasing oncology drug spend historically did not correspond with reductions in mortality and morbidity. Diabetes had the lowest drug spend and greatest QALY/€1000 spent benefit.ConclusionThis study indicates that drug spend may not correlate to clinical burden across diseases. Future research could stimulate debate on whether more equitable drug funding may improve disease management.Supplementary InformationThe online version contains supplementary material available at 10.1007/s41669-020-00253-4.

Timing of parathyroidectomy for tertiary hyperparathyroidism with end-stage renal disease: A cost-effectiveness analysis

BackgroundTertiary hyperparathyroidism associated with end-stage renal disease is characterized by progression from secondary hyperparathyroidism to an autonomous overproduction of parathyroid hormone that leads to adverse health outcomes. Rates of parathyroidectomy (PTX) have decreased with the use of calcimimetics. Optimal timing of PTX in relation to kidney transplant remains controversial. We aimed to identify the most cost-effective strategy for patients with tertiary hyperparathyroidism undergoing kidney transplant. MethodsWe constructed a patient level state transition microsimulation to compare 3 management schemes: cinacalcet with kidney transplant, cinacalcet with PTX before kidney transplant, or cinacalcet with PTX after kidney transplant. Our base case was a 55-year-old on dialysis with tertiary hyperparathyroidism awaiting kidney transplant. Outcomes, including quality-adjusted life years, surgical complications, and mortality, were extracted from the literature, and costs were estimated using Medicare reimbursement data. ResultsOur base case analysis demonstrated that cinacalcet with PTX before kidney transplant was dominant, with a lesser cost of $399,287 and greater quality-adjusted life years of 10.3 vs $497,813 for cinacalcet with PTX after kidney transplant (quality-adjusted life years 9.4) and $643,929 for cinacalcet with kidney transplant (quality-adjusted life years 7.4). ConclusionCinacalcet alone with kidney transplant is the least cost-effective strategy. Patients with end-stage renal disease-related tertiary hyperparathyroidism should be referred for PTX, and it is most cost-effective if performed prior to kidney transplant.

Cost-effectiveness analyses demonstrate that observation is superior to sentinel lymph node biopsy for postmenopausal women with HR + breast cancer and negative axillary ultrasound.

To evaluate the cost-effectiveness of axillary observation versus sentinel lymph node biopsy (SLNB) after negative axillary ultrasound (AUS). In patients with clinical T1-T2 N0 breast cancer and negative AUS, SLNB is the current standard of care for axillary staging. However, SLNB is costly, invasive, decreasing in importance for medical decision-making, and is not considered therapeutic. Observation alone is currently being evaluated in randomized clinical trials, and is thought to be non-inferior to SLNBfor patients with negative AUS. We performed cost-effectiveness analyses of observation versus SLNB after negative AUS in postmenopausal women with clinical T1-T2 N0, HR+/HER2- breast cancer. Costs at the 2016 price level were evaluated from a third-party commercial payer perspective using the MarketScan® Database. We compared cost, quality-adjusted life years (QALYs), and net monetary benefit (NMB). Multiple sensitivity analyses varying baseline probabilities, costs, utilities, and willingness-to-pay thresholds were performed. Observation was superior to SLNB for patients with N0 and N1 disease, and for the entire patient population (NMB in US$: $655,659 for observation versus $641,778 for SLNB for the entire patient population). In the N0 and N1 groups, observation incurred lower cost and was associated with greater QALYs. SLNB was superior for patients with > 3 positive lymph nodes, representing approximately 5% of the population. Sensitivity analyses consistently demonstrated that observation is the optimal strategy for AUS-negative patients. Considering both cost and effectiveness, observation is superior to SLNB in postmenopausal women with cT1-T2 N0, HR+/HER2- breast cancer and negative AUS.

Cost-effectiveness of empagliflozin compared with liraglutide based on cardiovascular outcome trials in Type II diabetes.

Aim: Cost-effectiveness (CE) analysis of empagliflozin+standard of care (SoC) compared with SoC and liraglutide+SoC, in patients with Type II diabetes and established cardiovascular disease, was conducted using evidence from cardiovascular outcomes trials. Methods: The IQVIA Core Diabetes Model was calibrated to predict same outcomes observed in EMPA-REG OUTCOME and LEADER trials. Three-year observed cardiovascular events of SoC, empagliflozin+SoC and liraglutide+SoC were derived from EMPA-REG OUTCOME trial and an indirect comparison. Time horizon was 50 years and the UK payer perspective was taken. Results: Empagliflozin+SoC dominated liraglutide+SoC with greater quality-adjusted life years and reduced costs. Base-case incremental CE ratio of 6428 GBP/QALY was observed for empagliflozin+SoC versus SoC. Conclusion: Results suggest that empagliflozin+SoC is costeffective versus SoC and liraglutide+SoC.

PGI26 THE COST-EFFECTIVENESS OF PHARMACOLOGIC VS SURGICAL INTERVENTION FOR PATIENTS HOSPITALIZED WITH ACUTE SEVERE ULCERATIVE COLITIS IN THE UNITED STATES

The American College of Gastroenterology (ACG) recently released guidelines for the management of acute severe ulcerative colitis (ASUC) that recommend infliximab, cyclosporine, or surgical intervention (colectomy) as primary treatment options. While the cost-effectiveness of induction and maintenance therapy for UC has been investigated in many studies, there is limited information for ASUC. We evaluated the cost-effectiveness of pharmacologic treatments compared to surgery for patients hospitalized due to ASUC. A Markov model was developed from a US payer perspective with a one-week cycle length over a one-year time horizon. Interventions included infliximab, infliximab biosimilars (infliximab-abda and infliximab-dyyb), and cyclosporine compared to surgery. Patients treated with pharmacologic treatments could transition to remission, response without remission, or surgery. Health state utilities were included for active UC, response without remission, remission, post-surgical remission, and post-surgical complications. Costs included drug wholesale acquisition cost, administration, inpatient stay for ASUC, surgery, post-surgical complications, and endoscopy. We calculated incremental cost per quality-adjusted life year (QALY) gained, cost per life year (LY) in remission, and cost per surgery avoided. All pharmacologic treatments were more costly than surgery (infliximab: $61,602; infliximab-abda: $48,259; infliximab-dyyb: $52,970; cyclosporine: $4,293; surgery: $38,411) with greater QALYs (infliximab: 0.7825; infliximab-abda: 0.7825; infliximab-dyyb: 0.7825; cyclosporine: 0.7466; surgery: 0.723) Cyclosporine was the most cost-effective at $61,415/QALY, followed by infliximab-abda ($122,860/QALY), infliximab-dyyb ($181,630), and infliximab ($289,312). Pharmacologic treatments were dominated by surgery for the outcome of cost per LY in remission. Cost per surgery avoided ranged from $7,901 for cyclosporine to $41,259 for infliximab. Cyclosporine and infliximab biosimilars may present cost-effective treatment options compared to elective colectomy for patients hospitalized ASUC from the payer perspective over a 1-year time horizon.

Impact of mental disorders and chronic physical conditions on quality-adjusted life years in Singapore

The current study aims to evaluate the burden of disease in Singapore by estimating the quality-adjusted life years (QALYs) lost due to mental disorders and chronic physical conditions. The second Singapore Mental Health Study (SMHS-2016) was conducted in 2016 among 6126 respondents aged 18 years and above. The World Health Organization Composite International Diagnostic Interview version 3.0 (WHO-CIDI 3.0) and a modified version of the CIDI chronic medical disorders checklist were used to assess the 12-month diagnoses of mental and chronic physical disorders while the SF-6D scores derived from the 12-item Short Form Health Survey instrument was used to estimate the QALYs lost. The mean SF-6D score in this population was 0.87. The largest reduction in SF-6D scores among people with mental disorders was observed in Generalized Anxiety Disorder (GAD), followed by Major Depressive Disorder (MDD), alcohol abuse, bipolar disorder and Obsessive Compulsive Disorder (OCD) while the largest reduction in SF-6D score among people with chronic physical conditions was observed in ulcer, followed by lung disease, chronic pain and cardiovascular disease. At the population level, chronic pain was associated with the greatest QALY loss followed by MDD (14,204 and 6,889 respectively). Lung disease was associated with the smallest QALY loss (376). These findings highlight chronic pain, MDD, OCD, cardiovascular disease and GAD as the five leading contributors of QALYs lost in the general population which deserve prioritisation in public health prevention programmes.