The Cost-Effectiveness of a Novel SIAscopic Diagnostic Aid for the Management of Pigmented Skin Lesions in Primary Care: A Decision-Analytic Model

ObjectivesPigmented skin lesions are commonly presented in primary care. Appropriate diagnosis and management is challenging because the vast majority are benign. The MoleMate system is a handheld SIAscopy scanner integrated with a primary care diagnostic algorithm aimed at improving the management of pigmented skin lesions in primary care.MethodsThis decision-model–based economic evaluation draws on the results of a randomized controlled trial of the MoleMate system versus best practice (ISRCTN79932379) to estimate the expected long-term cost and health gain of diagnosis with the MoleMate system versus best practice in an English primary care setting. The model combines trial results with data from the wider literature to inform long-term prognosis, health state utilities, and cost.ResultsResults are reported as mean and incremental cost and quality-adjusted life-years (QALYs) gained, incremental cost-effectiveness ratio with probabilistic sensitivity analysis, and value of information analysis. Over a lifetime horizon, the MoleMate system is expected to cost an extra £18 over best practice alone, and yield an extra 0.01 QALYs per patient examined. The incremental cost-effectiveness ratio is £1,896 per QALY gained, with a 66.1% probability of being below £30,000 per QALY gained. The expected value of perfect information is £43.1 million.ConclusionsGiven typical thresholds in the United Kingdom (£20,000–£30,000 per QALY), the MoleMate system may be cost-effective compared with best practice diagnosis alone in a primary care setting. However, there is considerable decision uncertainty, driven particularly by the sensitivity and specificity of MoleMate versus best practice, and the risk of disease progression in undiagnosed melanoma; future research should focus on reducing uncertainty in these parameters.     

Cost-Effectiveness of Cetuximab,Cetuximab Plus Irinotecan,and Panitumumab for Third and Further Lines of Treatment for KRAS Wild-Type Patients with Metastatic Colorectal Cancer

ObjectivesTo estimate the cost-effectiveness of cetuximab monotherapy, cetuximab plus irinotecan, and panitumumab monotherapy compared with best supportive care (BSC) for the third and subsequent lines of treatment of patients with Kirsten rat sarcoma wild-type metastatic colorectal cancer from the perspective of the UK National Health Service.MethodsAn “an area under the curve” cost-effectiveness model was developed. The clinical effectiveness evidence for both cetuximab and panitumumab was taken from a single randomized controlled trial (RCT) in each case and for cetuximab plus irinotecan from several sources.ResultsPatients are predicted to survive for approximately 6 months on BSC, 8.5 months on panitumumab, 10 months on cetuximab, and 16.5 months on cetuximab plus irinotecan.Panitumumab is dominated, and cetuximab is extended dominated. An incremental cost-effectiveness ratio (ICER) of £95,000 per quality-adjusted life-year (QALY) was estimated for cetuximab versus BSC and is likely to be relatively accurate, because the relevant clinical evidence is taken from a high-quality RCT. The estimated ICER for panitumumab versus BSC, at £187,000 per QALY, is less certain due to assumptions in the adjustment for the substantial crossing-over of patients in the RCT. The ICER for cetuximab plus irinotecan versus BSC, at £88,000 per QALY, is least certain due to substantial uncertainty about progression-free survival, treatment duration, and overall survival. Nonetheless, when key parameters are varied within plausible ranges, all three treatments always remain poor value for money.ConclusionsAll three treatments are highly unlikely to be considered cost-effective in this patient population in the United Kingdom. We explain how the reader can adapt the model for other countries.     

Trabectedin for the treatment of relapsed ovarian cancer

The paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of trabectedin for the treatment of relapsed platinum-sensitive ovarian cancer, based upon a review of the manufacturer's submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal process. The submission addressed only part of the decision problem and did not provide evidence to compare trabectedin (Yondelis?, PharmaMar) and pegylated liposomal doxorubicin hydrochloride (PLDH) (Caelyx?, Schering-Plough) with key comparators. The submission's direct comparison evidence came from one reasonable-quality randomised controlled trial (RCT) of trabectedin and PLDH versus PLDH alone (ET743-OVA-301). The results of the RCT were subdivided into the entire platinum-sensitive population (> 6-month relapse after initial platinum-based chemotherapy) and partially platinum-sensitive (≥ 6- to 12-month relapse) and fully platinum-sensitive (> 12-month relapse) populations. The outcomes included were overall survival, progression-free survival measured by three types of assessor, response rates, adverse effects of treatment, health-related quality of life and cost per quality-adjusted-life-year (QALY) gained. A mixed treatment comparison (MTC) meta-analysis comparing trabectedin and PLDH with single-agent PLDH within the entire platinum-sensitive population, with paclitaxel or with topotecan also formed part of the submission. The RCT data showed that trabectedin plus PLDH compared with PLDH monotherapy had a significant effect on overall survival only within the partially platinum-sensitive subgroup. PFS results reported by the independent radiologists showed significant effects in favour of the trabectedin and PLDH arm for the entire and partially platinum-sensitive populations only. Rates of grade 3 and 4 adverse events were mostly higher in the trabectedin and PLDH arm than in the PLDH alone arm. There were several issues regarding the undertaking of the MTC, and thus the data were not considered robust. Furthermore, the ERG did not believe the MTC to be necessary to answer the decision problem. The manufacturer submitted a de novo cost-effectiveness model. The main analysis compared trabectedin in combination with PLDH versus paclitaxel, topotecan and PLDH (each as monotherapy) in the entire platinum-sensitive population, using results estimated from the MTC. Additional analyses were presented comparing trabectedin in combination with PLDH versus PLDH monotherapy using direct evidence from the OVA-301 trial for the fully, partially and entire platinum-sensitive populations. The cost per QALY gained for trabectedin in combination with PLDH versus PLDH monotherapy was estimated to be ￡ 70,076 in the main analysis. In the additional analyses, the cost per QALY gained for trabectedin in combination with PLDH versus PLDH monotherapy was ￡ 94,832, ￡ 43,996 and ￡ 31,092 for the entire, partially and fully platinum-sensitive populations, respectively. Additional work was undertaken by the ERG using patient-level data and amending some assumptions to provide a better statistical fit to the Kaplan-Meier data than the exponential distribution assumed by the manufacturer. The ERG base-case estimate of the cost per QALY of trabectedin in combination with PLDH ranged from ￡46,503 to ￡54,607 in the partially platinum-sensitive population. At the time of writing, trabectedin in combination with PLDH for the treatment of women with relapsed platinum-sensitive ovarian cancer is not recommended by NICE in the final appraisal determination.     

High-Dose Hemodialysis versus Conventional In-Center Hemodialysis: A Cost-Utility Analysis from a UK Payer Perspective

ObjectiveTo investigate the cost-effectiveness of high-dose hemodialysis (HD) versus conventional in-center HD (ICHD), over a lifetime time horizon from the UK payer’s perspective.MethodsWe used a Markov modeling approach to compare high-dose HD (in-center or at home) with conventional ICHD using current and hypothetical home HD reimbursement tariffs in England. Sensitivity analyses tested the robustness of the results. The main outcome measure was the incremental cost-effectiveness ratio (ICER) expressed as a cost per quality-adjusted life-year (QALY).ResultsOver a lifetime, high-dose HD in-center (5 sessions/wk) is associated with higher per-patient costs and QALYs (increases of £108,713 and 0.862, respectively) versus conventional ICHD. The corresponding ICER (£126,106/QALY) indicates that high-dose HD in-center is not cost-effective versus conventional ICHD at a UK willingness-to-pay threshold of £20,000 to £30,000. High-dose HD at home is associated with lower total costs (£522 less per patient) and a per-patient QALY increase of 1.273 compared with ICHD under the current Payment-by Results reimbursement tariff (£456/wk). At an increased home HD tariff (£575/wk), the ICER for high-dose HD at home versus conventional ICHD is £17,404/QALY. High-dose HD at home had a 62% to 84% probability of being cost-effective at a willingness-to-pay threshold of £20,000 to £30,000/QALY.ConclusionsAlthough high-dose HD has the potential to offer improved clinical and quality-of-life outcomes over conventional ICHD, under the current UK Payment-by Results reimbursement scheme, it would be considered cost-effective from a UK payer perspective only if conducted at home.     

Cost-Effectiveness of Systemic Treatments for Metastatic Castration-Sensitive Prostate Cancer: An Economic Evaluation Based on Network Meta-Analysis

ObjectivesTo assess the cost-effectiveness of systemic treatments for metastatic castration-sensitive prostate cancer from the US healthcare sector perspective with a lifetime horizon.MethodsWe built a partitioned survival model based on a network meta-analysis of 7 clinical trials with 7287 patients aged 36 to 94 years between 2004 and 2018 to predict patient health trajectories by treatment. We tested parameter uncertainties with probabilistic sensitivity analyses. We estimated drug acquisition costs using the Federal Supply Schedule and adopted generic drug prices when available. We measured cost-effectiveness by an incremental cost-effectiveness ratio (ICER).ResultsThe mean costs were approximately $392 000 with androgen deprivation therapy (ADT) alone and approximately $415 000, $464 000, $597 000, and $959 000 with docetaxel, abiraterone acetate, enzalutamide, and apalutamide, added to ADT, respectively. The mean quality-adjusted life-years (QALYs) were 3.38 with ADT alone and 3.92, 4.76, 3.92, and 5.01 with docetaxel, abiraterone acetate, enzalutamide, and apalutamide, added to ADT, respectively. As add-on therapy to ADT, docetaxel had an ICER of $42 069 per QALY over ADT alone; abiraterone acetate had an ICER of $58 814 per QALY over docetaxel; apalutamide had an ICER of $1 979 676 per QALY over abiraterone acetate; enzalutamide was dominated. At a willingness to pay below $50 000 per QALY, docetaxel plus ADT is likely the most cost-effective treatment; at any willingness to pay between $50 000 and $200 000 per QALY, abiraterone acetate plus ADT is likely the most cost-effective treatment.ConclusionsThese findings underscore the value of abiraterone acetate plus ADT given its relative cost-effectiveness to other systemic treatments for metastatic castration-sensitive prostate cancer.     

Evaluating the Cost-Effectiveness of Changes to the Surveillance Intervals in the UK Abdominal Aortic Aneurysm Screening Programme

ObjectivesTo investigate the safety and cost-effectiveness of lengthening the time between surveillance ultrasound scans in the UK Abdominal Aortic Aneurysm (AAA) Screening Programme.MethodsA discrete event simulation model was used to evaluate the cost-effectiveness of AAA screening for men aged 65, comparing current surveillance intervals to 6 alternative surveillance interval strategies that lengthened the time between surveillance scans for 1 or more AAA size categories. The model considered clinical events and costs incurred over a 30-year time horizon and the cost per quality-adjusted life year (QALY). The model adopted the National Health Service perspective and discounted future costs and benefits at 3.5%.ResultsCompared with current practice, alternative surveillance strategies resulted in up to a 4% reduction in the number of elective AAA repairs but with an increase of up to 1.6% in the number of AAA ruptures and AAA-related deaths. Alternative strategies resulted in a small reduction in QALYs compared to current practice but with reduced costs. Two strategies that lengthened surveillance intervals in only very small AAAs (3.0-3.9 cm) provided, at a cost-effectiveness threshold of £20 000 per QALY, the highest positive incremental net benefit. There was negligible chance that current practice is the most cost-effective strategy at any threshold below £40 000 per QALY.ConclusionsLengthening surveillance intervals in the UK Abdominal Aortic Aneurysm Screening Programme, especially for small AAA, can marginally reduce the incremental cost per QALY of the program. Nevertheless, whether the cost savings from refining surveillance strategies justifies a change in clinical practice is unclear.     

Cost-effectiveness analysis for midostaurin versus standard of care in acute myeloid leukemia in the United Kingdom

Aims

Midostaurin (MIDO) has been proposed for the treatment of newly-diagnosed adult patients with FMS-like tyrosine kinase 3 mutation-positive (FLT3+) acute myeloid leukemia (AML) in combination with standard chemotherapy. The cost-effectiveness of MIDO and standard of care (SOC) followed by MIDO monotherapy was compared to SOC alone for newly-diagnosed FLT3+?AML in the UK.

Methods

A partitioned survival model was developed from a UK public healthcare system perspective to compare the cost-effectiveness of MIDO plus SOC and SOC over a lifetime horizon. The model included the following health states/partitions: induction, consolidation, monotherapy, complete remission (CR), relapse, stem cell transplantation (SCT), SCT recovery, and post-SCT recovery. Data on CR, overall survival, and adverse events were obtained from a Phase III clinical trial. Overall survival was extrapolated beyond the trial horizon using a ‘cure model’ approach and data from the Office for National Statistics. Utilities were identified via a systematic review. Routine care utilization was obtained from the National Institute for Health and Care Excellence single technology appraisal for azacitidine in AML (TA399). The costs of drugs and administration, adverse events, hospitalizations, physician visits, and end-of-life care were incorporated.

Results

Incremental life years (LYs) and quality-adjusted life years (QALYs) gained by patients on MIDO and SOC versus SOC were 1.67 and 1.47, respectively. At an incremental cost of £54,072 over a lifetime horizon, the ICER was £32,465 per LY and £36,826 per QALY. Sensitivity analyses were generally consistent with the base case findings.

Conclusions

With limited treatments in FLT3+?AML, MIDO represents a clinically significant advance in the management of newly-diagnosed AML. Using a threshold of £50,000 per QALY for end-of-life treatment, MIDO was shown to be a cost-effective option for newly-diagnosed FLT3+?AML.

     

The Cost-Effectiveness of Spinal Cord Stimulation for Complex Regional Pain Syndrome

ObjectivesHealth-care policymakers and payers require cost-effectiveness evidence to inform their treatment funding decisions. The aims of this study were to assess the cost-effectiveness of the addition of spinal cord stimulation (SCS) compared with conventional management alone (CMM) in patients with complex regional pain syndrome (CRPS), and to determine the cost-effectiveness of nonrechargeable versus rechargeable SCS implanted pulse generators (IPGs).MethodsA decision analytic model was used to synthesize data on CRPS patient outcomes and health-care costs over a 15-year time horizon from the perspective of the UK National Health Services. Data were sourced from two SCS randomized controlled trials. Results are expressed as an incremental cost per quality-adjusted life-year (QALY) in 2008 GBP.ResultsThe incremental cost-effectiveness of SCS compared with CMM was £3562 per QALY, a finding that was robust across sensitivity analyses with an 87% probability that SCS is cost-effective at a willingness to pay threshold of £30,000. When the longevity of an IPG is 4 years or less, a rechargeable (and initially more expensive) IPG is more cost-effective than a nonrechargeable IPG.ConclusionsIn selected patients with CRPS, SCS is cost-effective as an adjunct to CMM. Despite their initial increased expense, rechargeable IPGs should be considered when IPG longevity is likely to be short. These findings support policymakers to extend the use of SCS as a good value for money treatment for CRPS.     

Cost-Effectiveness of Temsirolimus for First Line Treatment of Advanced Renal Cell Carcinoma

ObjectivesTo estimate the cost-effectiveness of temsirolimus compared to interferon-α for first line treatment of patients with advanced, poor prognosis renal cell carcinoma, from the perspective of the UK National Health Service.MethodsA decision-analytic model was developed to estimate the cost-effectiveness of temsirolimus. The clinical effectiveness of temsirolimus compared with interferon-α and the utility values (using EQ-5D tariffs) were taken from a recent phase III randomized clinical trial. Cost data were obtained from published literature and based on current UK practice. The effect of parameter uncertainty on cost-effectiveness was explored through extensive one-way and probabilistic sensitivity analyses.ResultsCompared to interferon-α, temsirolimus treatment resulted in an incremental cost per QALY gained of £94,632; based on an estimated mean gain of 0.24 quality-adjusted life years (QALYs) per patient, at a mean additional cost of £22,331 (inflated to 2007/8). The cost per QALY for patient subgroups ranged from £74,369 to £154,752. The probability that temsirolimus is cost-effective compared to interferon-α at a willingness to pay threshold of £30,000 per QALY for all patient groups is expected to be close to zero. The cost per QALY was sensitive to the clinical effectiveness parameters, health state utilities, drug costs and the cost of administration of temsirolimus.ConclusionsTemsirolimus has been shown to be clinically effective compared to interferon-α offering additional health benefits, however, with a cost per QALY in excess of £90,000, it may not be regarded as a cost-effective use of resources in some health care settings.     

Cost-Effectiveness of Duloxetine: The Stress Urinary Incontinence Treatment (SUIT) Study

ObjectiveTo assess the cost-effectiveness of duloxetine compared with conservative therapy in women with stress urinary incontinence (SUI).MethodsCost and outcome data were taken from the Stress Urinary Incontinence Treatment (SUIT) study, a 12-month, prospective, observational, naturalistic, multicenter, multicountry study. Costs were assessed in UK £ and outcomes in quality adjusted life years using responses to the EuroQol (EQ-5D); numbers of urine leaks were also estimated. Potential selection bias was countered using multivariate regression and propensity score analysis.ResultsDuloxetine alone, duloxetine in combination with conservative treatment, and conservative treatment alone were associated with roughly two fewer leaks per week compared with no treatment. Duloxetine alone and with conservative treatment for SUI were associated with incremental quality-adjusted life-years (QALYs) of about 0.03 over a year compared with no treatment or with conservative treatment alone. Conservative treatment alone did not show an effect on QALYs. None of the interventions appeared to have marked impacts on costs over a year. Depending on the form of matching, duloxetine either dominated or had an incremental cost-effectiveness ratio (ICER) below £900 per QALY gained compared with no treatment and with conservative treatment alone. Duloxetine plus conservative therapy had an ICER below £5500 compared with no treatment or conservative treatment alone. Duloxetine compared with duloxetine plus conservative therapy showed similar outcomes but an additional cost for the combined intervention.ConclusionsAlthough the limitations of the use of SUIT's observational data for this purpose need to be acknowledged, the study suggests that initiating duloxetine therapy in SUI is a cost-effective treatment alternative.     

Assessing the Value of Cemiplimab for Adults With Advanced Cutaneous Squamous Cell Carcinoma: A Cost-Effectiveness Analysis

ObjectivesTo evaluate the cost-effectiveness of cemiplimab in patients with advanced cutaneous squamous cell carcinoma (CSCC) from a payer perspective in the United States.MethodsA partitioned survival model was developed to assess the cost-effectiveness of cemiplimab versus historical standard of care (SOC). All inputs were identified based on a systematic literature review, supplemented by expert opinion where necessary. Clinical inputs for cemiplimab were based on individual patient data from a cemiplimab phase 2 single-arm trial (NCT27060498). For SOC, analysis was based on a pooled analysis of single-arm clinical trials and retrospective studies evaluating chemotherapy and epidermal growth factor receptor inhibitors (cetuximab, erlotinib, and gefitinib) identified via a systematic literature review (6 of the 27 included studies). Overall survival and progression-free survival were extrapolated over a lifetime horizon. Costs were included for drug acquisition, drug administration, management of adverse events, subsequent therapy, disease management, and terminal care. Unit costs were based on published 2019 US list prices.ResultsIn the base case, cemiplimab versus SOC resulted in an incremental cost-effectiveness ratio of $99 447 per quality adjusted-life year (QALY), where incremental costs and QALYs were $372 108 and 3.74, respectively. At a willingness-to-pay threshold of $150 000/QALY, the probabilistic sensitivity analysis suggests a 90% probability that cemiplimab is cost-effective compared to SOC. Scenario analyses resulted in incremental cost-effectiveness ratios ranging from $90 590 to $148 738.ConclusionsCompared with historical SOC, cemiplimab is a cost-effective use of US payer resources for the treatment of advanced CSCC and is expected to provide value for money.     

Cost-Effectiveness of Ivacaftor Therapy for Treatment of Cystic Fibrosis Patients With the G551D Gating Mutation

ObjectivesCystic fibrosis (CF) is a rare genetic disease with no cure. Until recently, treatment has targeted symptoms of the disease and not the disease-causing genetic defect. Ivacaftor is included in a new class of breakthrough drugs targeting the genetic defects of CF. We sought to estimate the long-term cost-effectiveness of ivacaftor from a US payer perspective.MethodsWe developed an individual-level microsimulation model that followed a cohort of heterogeneous US CF patients over a lifetime. The primary outcome of interest was quality-adjusted life years (QALYs). We also compared unadjusted life years, count of acute pulmonary exacerbations, and count of lung transplants over a lifetime between patients treated with ivacaftor plus best supportive care and patients treated with best supportive care alone. We conducted one-way and probabilistic sensitivity analyses to test the impact of various model inputs and uncertainties.ResultsWe found a substantial increase in QALYs, life years, and treatment costs over a lifetime for patients treated with ivacaftor plus best supportive care versus best supportive care alone. Discounted results for ivacaftor were 22.92 QALYs and $8 797 840 in total lifetime costs compared to 16.12 QALYs and $2 336 366 lifetime costs for best supportive care alone. The incremental cost-effectiveness ratios (ICERs) were $950 217 per QALY. Results from the probabilistic sensitivity analysis indicated a 0% chance that ivacaftor was cost-effective at a willingness-to-pay (WTP) threshold of $500 000 per QALY.ConclusionsTreatment with ivacaftor plus best supportive care versus best supportive care alone is not cost-effective at or near commonly accepted WTP thresholds.     

Cost-Effectiveness Analysis of Rituximab Combined with CHOP for Treatment of Diffuse Large B-Cell Lymphoma

PURPOSE: To estimate the cost-effectiveness from a French payer perspective of CHOP (cyclophosphamide, doxorubicin, vincristine, and prednisone) alone compared with CHOP plus rituximab (R-CHOP) for treatment of patients with diffuse large B-cell lymphoma. METHODS: Mean patient survival, days of hospitalization, and chemotherapy costs during treatment were estimated from a Phase III trial in France, Belgium, and Switzerland. Survival during the trial was estimated using the Kaplan-Meier method; survival beyond the trial period was projected based on mortality rates from the Scottish and Newcastle Lymphoma Group database. French diagnosis-related group (DRG) payment schedules were applied to trial data to estimate cost of adverse events and drug administration. We estimated survival and cost-effectiveness [the incremental cost per quality-adjusted life-year (QALY) gained] from 4 years (median clinical trial follow-up period) to 15 years, discounted at a fixed annual rate of 3%. We used published patient preferences. We converted currency to euros, based on 2003 exchange rates. RESULTS: R-CHOP resulted in a 20.6% relative increase in complete response rate (absolute increase from 63% to 76%), and a 31% decrease in risk of death at 4 years (95% CI 8-49%). Over a 15-year time horizon, mean overall survival (OS) duration was estimated to be 6.90 years for R-CHOP and 5.74 years for CHOP, a mean increase in OS of 1.16 years (or 1.07 QALYs). Total direct medical costs were 13,170 euro higher with R-CHOP, with an incremental cost-effectiveness ratio of 12,259 euro per QALY gained. CONCLUSION: R-CHOP significantly increases mean OS up to 4 years compared with CHOP, and its cost-effectiveness ratio compares favorably with other oncology treatments in widespread use.     

An Evaluation of the Cost-Effectiveness of Rituximab in Combination with Chemotherapy for the First-Line Treatment of Follicular Non-Hodgkin's Lymphoma in the UK

ObjectivesIn this study, the cost-effectiveness of rituximab was evaluated in comparison with commonly used chemotherapy regimens for patients with advanced follicular lymphoma (FL), from the perspective of the UK National Health Service (NHS).MethodsResults from four randomized controlled trials comparing the addition of rituximab to chemotherapy regimens: mitoxantrone, chlorambucil, and prednisolone (MCP); cyclophosphamide, vincristine, and prednisolone (CVP); cyclophosphamide, doxorubicin, vincristine, and prednisolone (CHOP); or cyclophosphamide, etoposide, doxorubicin, prednisolone, and interferon alpha (CHVP + IFNα) versus chemotherapy alone were used to develop a Markov model. The rates of disease progression and the duration of treatment effect were obtained from the trial data. Treatments were compared in two ways: 1) an individual comparison of rituximab + chemotherapy versus chemotherapy and 2) a multiple treatment comparison using league tables. Economic and clinical outcomes (quality-adjusted life-years (QALYs)) were estimated over patient lifetimes and discounted at 3.5% per annum.ResultsIn the individual comparison, the addition of rituximab increased QALYs by (mean, 95% confidence interval) 1.174 (1.02–1.30), 0.909 (0.79–1.01), 0.823 (0.71–0.91), and 0.453 (0.40–0.50) for MCP, CVP, CHOP, and CHVP, respectively, compared with chemotherapy alone. The incremental costs per QALY gained were £7474, £8621, £10,732, and £8551, respectively. Sensitivity analyses indicated that rituximab plus chemotherapy was a cost-effective treatment option, with incremental cost-effectiveness ratios below a threshold of £30,000 per QALY gained. When compared across the chemotherapy regimens, rituximab plus MCP appeared to be the single most cost-effective treatment option, but further randomized trials are required to substantiate this.ConclusionsThe addition of rituximab to chemotherapy in advanced FL was found to be highly cost-effective in the UK.     

The Cost-Effectiveness of Remdesivir for Hospitalized Patients With COVID-19

ObjectivesThis study aimed to estimate the cost-effectiveness of remdesivir, the first novel therapeutic to receive Emergency Use Authorization for the treatment of hospitalized patients with COVID-19, and identify key drivers of value to guide future pricing and reimbursement efforts.MethodsA Markov model evaluated the cost-effectiveness of remdesivir in patients hospitalized with COVID-19 from a US healthcare sector perspective. A lifetime time horizon captured potential long-term costs and outcomes. Model outcomes included discounted total costs, life-years, and quality-adjusted life-years (QALYs). Remdesivir was modeled as an addition to standard of care and compared with standard of care alone, including dexamethasone for patients requiring respiratory support. COVID-19 hospitalizations were assumed to be reimbursed through a single payment based on the respiratory support received alongside a remdesivir carveout payment in the base case. Sensitivity and scenario analyses identified key drivers.ResultsAt a unit price of $520 per vial and assuming no survival benefit with remdesivir, the incremental cost-effectiveness was $298 200/QALY for patients with moderate to severe COVID-19 and $1 847 000/QALY for patients with mild COVID-19. Although current data do not support a survival benefit, if one was assumed, the cost-effectiveness estimate was $50 100/QALY for the moderate to severe population and $103 400/QALY for the mild population. Another key driver included the hospitalization payment structure (per diem vs bundled payment).ConclusionsWith the current evidence available, remdesivir’s price is too high to align with its expected health gains for hospitalized patients with COVID-19. Results from this study provide a rationale for iterative health technology assessment.     

Cost-effectiveness of roflumilast as an add-on treatment to long-acting bronchodilators in the treatment of COPD associated with chronic bronchitis in the United Kingdom

Objective

To estimate the cost-effectiveness of adding a selective phosphodiesterase-4 inhibitor, roflumilast, to a long-acting bronchodilator therapy (LABA) for the treatment of patients with severe-to-very severe chronic obstructive pulmonary disease (COPD) associated with chronic bronchitis with a history of frequent exacerbations from the UK payer perspective.

Methods

A Markov model was developed to predict the lifetime cost and outcomes [exacerbations rates, life expectancy, and quality-adjusted life years (QALY)] in patients treated with roflumilast, which showed a reduction in the exacerbation rates and lung function improvement in a pooled analysis from two clinical trials, M2-124 and M2-125. Sensitivity analyses were conducted to explore the impact of uncertainties on the cost-effectiveness.

Results

The addition of roflumilast to concomitant LABA reduced the number of exacerbations from 15.6 to 12.7 [2.9 (95 % CI 0.88–4.92) exacerbations avoided] and increased QALYs from 5.45 to 5.61 [0.16 (95 % CI 0.02–0.31) QALYs gained], at an incremental cost of £3,197 (95 % CI £2,135–£4,253). Cost in LABA alone and LABA + roflumilast were £16,161 and £19,358 respectively. The incremental cost-effectiveness ratios in the base case were £19,505 (95 % CI £364–£38,646) per quality-adjusted life-year gained and 18,219 (95 % CI £12,697–£49,135) per life-year gained. Sensitivity analyses suggest that among the main determinants of cost-effectiveness are the reduction of exacerbations and the case fatality rate due to hospital-treated exacerbations. Probabilistic sensitivity analysis suggests that the probability of roflumilast being cost-effective is 82 % at willingness-to-pay £30,000 per QALY.

Conclusions

The addition of roflumilast to LABA in the treatment of patients with severe-to-very severe COPD reduces the rate of exacerbations and can be cost-effective in the UK setting.     

Cost-Effectiveness and Economic Burden Analyses on All First-Line Treatments of Chronic Lymphocytic Leukemia

ObjectivesSeveral chemoimmunotherapy and targeted treatment regimens are approved as front-line therapies in chronic lymphocytic leukemia. We estimated for the 10-year cost-effectiveness of these treatment regimens and the economic burden of following the estimated risk-stratified 21 040 patients with chronic lymphocytic leukemia diagnosed in 2020 for 10 years.MethodsA Markov model with 7 exclusive health states was specified over a 10-year time horizon. Treatment effectiveness inputs were obtained from a novel network meta-analysis on the progression-free survival, overall survival curves, and time to next treatment. Costs and utilities inputs were included for each health state for each treatment and discounted at 3.0%/year. Life-years (LYs) and quality-adjusted LYs (QALYs) for each treatment were determined. Using the lowest cost regimen as reference, the incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratio (ICUR) were estimated. The 10-year per-patient cost was determined by risk status and by initial treatment.ResultsVenetoclax-plus-obinutuzumab was the lowest cost regimen, hence the reference. Superior in effectiveness to all chemoimmunotherapies, it was cost saving. With the highest effectiveness gains at 6.26 LYs and 5.01 QALYs and despite being the most expensive regimen ($1 298 638 per patient), acalabrutinib-plus-obinutuzumab yielded the best ICER ($409 343/LY gained) and ICUR ($501 236/QALY gained). The remaining ICERs of targeted therapies ranged from $512 101/LY gained to $793 236/LY gained and the ICURs from $579 737/QALY gained to $869 300/QALY gained. The 10-year postdiagnosis low/high (venetoclax-plus-obinutuzumab/acalabrutinib-plus-obinutuzumab) economic burden ranges were $42 690 to $98 665 for low-risk, $141 339 to $326 660 for intermediate-risk, and $273 650 to $632 453 for high-risk patients.ConclusionsCompared with venetoclax-plus-obinutuzumab, chemoimmunotherapies are associated with less health benefits at higher cost. The targeted therapies achieve greater benefits at higher cost.     

Evaluation of a Stratified National Breast Screening Program in the United Kingdom: An Early Model-Based Cost-Effectiveness Analysis

Objectives

To identify the incremental costs and consequences of stratified national breast screening programs (stratified NBSPs) and drivers of relative cost-effectiveness.

Impact of Non-Adherence and Flare Resolution on the Cost-Effectiveness of Treatments for Gout: Application of a Linked Pharmacometric/Pharmacoeconomic Model

Background

Dual urate-lowering therapy (ULT) with lesinurad in combination with either allopurinol or febuxostat is an option for patients with gout unsuccessfully treated on either monotherapy. Treatment failure is often a result of poor medication adherence. Imperfect adherence in clinical trials may lead to biased estimates of treatment effect and confound the results of cost-effectiveness analyses.

Cost–Utility Analysis of Topical Intranasal Steroids for Otitis Media with Effusion Based on Evidence from the GNOME Trial

ObjectivesTo estimate the cost-effectiveness of topical intranasal steroids for the treatment of otitis media with effusion (OME) in primary care from the perspective of the UK National Health Service.MethodsAn economic evaluation was conducted based on evidence from the double-blind, randomized, placebo-controlled GPRF [General Practice Research Framework] Nasal Steroids for Otitis Media with Effusion (GNOME) trial. Participants comprised 217 children aged 4–11 years who had at least one episode of otitis media or related ear problem in the previous 12 months and had tympanometrically confirmed bilateral OME. Children were randomly allocated to receive either mometasone furoate 50 µg or placebo spray once daily into each nostril for 3 months. The main outcome measure was the incremental cost per quality-adjusted life-year (QALY) gained for topical steroids compared with placebo. The nonparametric bootstrap method was used to present cost-effectiveness acceptability curves at alternative willingness to pay thresholds.ResultsChildren receiving topical steroids accrued nonsignificantly higher costs (incremental cost/child: £11, 95% confidence interval [CI]: ?£199 to £222) and nonsignificantly fewer QALYs (incremental QALY gain/child: ?0.0166, 95% CI: ?0.0652 to 0.0320) than those receiving placebo. Topical steroids had a 24.19% probability of being cost-effective at a £20,000 per QALY gained threshold, a 23.82% probability of being more effective and a 46.25% probability of being less costly. Sensitivity and subgroup analyses showed incremental costs and benefits to be highly sensitive to the methods used and the patient group considered, although differences between groups did not reach statistical significance in any analysis.ConclusionsTopical steroids are unlikely to be a cost-effective treatment for OME in general practice.