UK Payer Perspective Research Articles

Cost-Effectiveness of 9-Valent HPV Vaccination for Patients Treated for High-Grade Cervical Intraepithelial Neoplasia in the UK

Patients who have been treated for high-grade cervical intraepithelial neoplasia (CIN grade ≥2) are at a high risk for subsequent CIN and other cancers and diseases related to human papillomavirus (HPV). HPV vaccination can reduce the risk of subsequent disease in patients surgically treated for grade 2 or greater CIN; however, there is no formal recommendation for prophylactic HPV vaccination in this high-risk population, and the cost-effectiveness is unknown. To assess the incremental lifetime outcomes, costs, and cost-effectiveness of integrating peritreatment 9-valent HPV (9vHPV) vaccination in combination with posttreatment surveillance for the prevention of cervical cancer and other HPV-attributable diseases in patients surgically treated for grade 2 or greater CIN vs posttreatment surveillance alone from a UK payer perspective. This economic evaluation used 3 independent Markov model structures. Model inputs for vaccine efficacy, utilities, and costs were obtained from published sources, and cervical cancer screening data were obtained from the National Health Service Cervical Screening Program. Costs were adjusted to 2022 to 2023 reference years. Data were analyzed from October 2022 to September 2023. Peritreatment vaccination with 9vHPV in combination with posttreatment surveillance compared with posttreatment surveillance alone. Clinical outcomes included grade 1, 2, or 3 CIN; cervical cancer; vaginal cancer; vulvar cancer; anal cancer; head and neck cancer; genital warts; and recurrent respiratory papillomatosis. Incremental cost-effectiveness ratios (ICERs) using a willingness-to-pay threshold (WTP) of £20 000 (US $26 200) per quality-adjusted life-year (QALY) were estimated. Deterministic sensitivity analysis and probabilistic sensitivity analysis were performed. Vaccination with 9vHPV in conjunction with posttreatment surveillance was cost-effective, with a favorable ICER of £13 789.07 (US $18 064.68) per QALY gained (ie, below the WTP of £20 000 per QALY) vs posttreatment surveillance alone. The resulting ICER was £52 358.01 (US $68 588.99) per HPV-related cancer averted and £64 090 (US $83 958.18) per HPV-related cancer death averted. The ICER was most sensitive to discount rate, incidence of HPV infection, vaccine price, and age at initial treatment for grade 2 or greater CIN. Results of the probabilistic sensitivity analysis showed peritreatment 9vHPV vaccination was cost-effective at the WTP recommended by the UK's Joint Committee on Vaccination and Immunisation (90% of iterations <£30 000 [US $39 300] per QALY) in 100% of iterations. These findings suggest that peritreatment prophylactic 9vHPV vaccination is a cost-effective option for preventing subsequent HPV-attributable diseases in patients surgically treated for grade 2 or greater CIN.

Cost-Effectiveness of Gene-Specific Prevention Strategies for Ovarian and Breast Cancer

Pathogenic variants (PVs) in BRCA1, BRCA2, PALB2, RAD51C, RAD51D, and BRIP1 cancer susceptibility genes (CSGs) confer an increased ovarian cancer (OC) risk, with BRCA1, BRCA2, PALB2, RAD51C, and RAD51D PVs also conferring an elevated breast cancer (BC) risk. Risk-reducing surgery, medical prevention, and BC surveillance offer the opportunity to prevent cancers and deaths, but their cost-effectiveness for individual CSGs remains poorly addressed. To estimate the cost-effectiveness of prevention strategies for OC and BC among individuals carrying PVs in the previously listed CSGs. In this economic evaluation, a decision-analytic Markov model evaluated the cost-effectiveness of risk-reducing salpingo-oophorectomy (RRSO) and, where relevant, risk-reducing mastectomy (RRM) compared with nonsurgical interventions (including BC surveillance and medical prevention for increased BC risk) from December 1, 2022, to August 31, 2023. The analysis took a UK payer perspective with a lifetime horizon. The simulated cohort consisted of women aged 30 years who carried BRCA1, BRCA2, PALB2, RAD51C, RAD51D, or BRIP1 PVs. Appropriate sensitivity and scenario analyses were performed. CSG-specific interventions, including RRSO at age 35 to 50 years with or without BC surveillance and medical prevention (ie, tamoxifen or anastrozole) from age 30 or 40 years, RRM at age 30 to 40 years, both RRSO and RRM, BC surveillance and medical prevention, or no intervention. The incremental cost-effectiveness ratio (ICER) was calculated as incremental cost per quality-adjusted life-year (QALY) gained. OC and BC cases and deaths were estimated. In the simulated cohort of women aged 30 years with no cancer, undergoing both RRSO and RRM was most cost-effective for individuals carrying BRCA1 (RRM at age 30 years; RRSO at age 35 years), BRCA2 (RRM at age 35 years; RRSO at age 40 years), and PALB2 (RRM at age 40 years; RRSO at age 45 years) PVs. The corresponding ICERs were -£1942/QALY (-$2680/QALY), -£89/QALY (-$123/QALY), and £2381/QALY ($3286/QALY), respectively. RRSO at age 45 years was cost-effective for RAD51C, RAD51D, and BRIP1 PV carriers compared with nonsurgical strategies. The corresponding ICERs were £962/QALY ($1328/QALY), £771/QALY ($1064/QALY), and £2355/QALY ($3250/QALY), respectively. The most cost-effective preventive strategy per 1000 PV carriers could prevent 923 OC and BC cases and 302 deaths among those carrying BRCA1; 686 OC and BC cases and 170 deaths for BRCA2; 464 OC and BC cases and 130 deaths for PALB2; 102 OC cases and 64 deaths for RAD51C; 118 OC cases and 76 deaths for RAD51D; and 55 OC cases and 37 deaths for BRIP1. Probabilistic sensitivity analysis indicated both RRSO and RRM were most cost-effective in 96.5%, 89.2%, and 84.8% of simulations for BRCA1, BRCA2, and PALB2 PVs, respectively, while RRSO was cost-effective in approximately 100% of simulations for RAD51C, RAD51D, and BRIP1 PVs. In this cost-effectiveness study, RRSO with or without RRM at varying optimal ages was cost-effective compared with nonsurgical strategies for individuals who carried BRCA1, BRCA2, PALB2, RAD51C, RAD51D, or BRIP1 PVs. These findings support personalizing risk-reducing surgery and guideline recommendations for individual CSG-specific OC and BC risk management.

EE355 Budget Impact Model of a New rFVIIa (Eptacog Beta), for the On-Demand Treatment of Spontaneous Bleeding Events and Surgical Bleedings Among Hemophilia Patients with Inhibitors: A Payer Perspective From UK

To estimate, from a UK payer perspective, the budget impact (BI) of a new recombinant activated factor VII (rFVIIa), eptacog beta (EB) in the treatment of spontaneous bleeding events (BE) and surgical bleedings (SB) in hemophilia A (HA) and B (HB) adult patients with inhibitors (PWI).

PRS10 Economic Evaluation of Umeclidinium/Vilanterol Versus Umeclidinium or Salmeterol in a UK COPD Population at Low Risk of Exacerbations Using the GALAXY Model

The 24-week EMAX trial in symptomatic patients with chronic obstructive pulmonary disease (COPD) and low exacerbation risk not receiving inhaled corticosteroids demonstrated clinical benefits of once-daily umeclidinium/vilanterol (UMEC/VI) 62.5/25 mcg versus once-daily UMEC 62.5 mcg and twice-daily salmeterol (SAL) 50 mcg. This post hoc analysis assessed cost-effectiveness of UMEC/VI versus UMEC or SAL from a UK payer perspective in patients with COPD and no exacerbation history in the EMAX trial. The validated GALAXY risk equation model was populated with EMAX patient characteristics, treatment effects, rescue medication use data, and 2020 UK costs. Treatment effects included forced expiratory volume in 1 second (FEV1) and St George’s Respiratory Questionnaire (SGRQ); no treatment effect was included for exacerbations. Model outputs included estimated exacerbation rates, survival, costs, life years (LYs), and quality-adjusted LYs (QALYs); incremental cost-effectiveness ratio (ICER) was calculated for cost per QALY gained. The base case used a 10-year time horizon, excluded treatment discontinuation, and discounted future costs and QALYs by 3.5% annually. Sensitivity and scenario analyses assessed robustness of model results. Despite greater incremental drug costs, UMEC/VI was the dominant treatment versus UMEC and SAL, providing an additional 0.090 LYs (95% CI: 0.035, 0.158) and 0.055 QALYs (-0.059, 0.168) with cost savings of £690 (-£1306, -£231) versus UMEC, and 0.174 LYs (0.076, 0.286) and 0.204 QALYs (0.079, 0.326) with cost savings of £1336 (-£2032, -£1006) versus SAL. In scenario and sensitivity analyses, ICERs for UMEC/VI versus UMEC were sensitive to time horizon (5 years: ICER=£4) and SGRQ treatment effect (upper CI: ICER=£12,337), while UMEC/VI was consistently dominant versus SAL. Based on model predictions from a UK NHS perspective, symptomatic patients with COPD and no exacerbation history receiving UMEC/VI are expected to have better outcomes (QALYs and survival) at lower costs versus UMEC and SAL. FUNDING: GSK (study 209845; EMAX study 201749/NCT03034915).

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.

Early Economic Evaluation Demonstrates That Noncomputerized Tomography Robotic-Assisted Surgery Is Cost-Effective in Patients Undergoing Unicompartmental Knee Arthroplasty at High-Volume Orthopaedic Centres.

Background For over fifty years, unicompartmental knee arthroplasty (UKA) has been used to treat single-compartment osteoarthritis of the knee and is considered a safe alternative to total knee arthroplasty (TKA). The development and use of robotic-assisted surgery (r-UKA) have made the execution of the procedure more precise, and various studies have reported improved radiographic outcomes and implant survival rates; however, its cost-effectiveness is unknown. This study aimed at assessing the cost-effectiveness of noncomputerized tomography (non-CT) r-UKA compared to the traditional unicompartmental knee arthroplasty (t-UKA) method in patients with unicompartmental knee osteoarthritis from the UK payer's perspective. Methods We developed a 5-year four-state Markov model to evaluate the expected costs and outcomes of the two strategies in patients aged 65 years. Failure rates for t-UKA were taken from the British National Joint Registry while data for non-CT r-UKA were obtained from a 2-year observational study. Cost was obtained from the NHS reference cost valued at 2018/19 GBP£, and a discount rate of 3.5% was applied to both costs and benefits. Results For a high-volume orthopaedic centre that performs 100 UKA operations per year, non-CT r-UKA was more costly than t-UKA but offered better clinical outcomes, and the estimated cost per QALY was £2,831. The results were more favourable in younger patients aged less than 55 and sensitive to case volumes and follow-up period. Conclusion Non-CT r-UKA is cost-effective compared with t-UKA over a 5-year period. Results are dependent on case volumes and follow-up period and favour younger age groups.

Cost-effectiveness of dapagliflozin as an adjunct to insulin for the treatment of type 1 diabetes mellitus in the United Kingdom.

To assess the cost-effectiveness of dapagliflozin, a sodium-glucose co-transporter-2 (SGLT2) inhibitor, as an adjunct to insulin in adults with type 1 diabetes mellitus (T1DM) inadequately controlled by insulin alone in the UK setting. A cost-utility analysis was conducted to compare dapagliflozin (5 mg or 10 mg) added to insulin versus insulin monotherapy (standard of care) over a lifetime horizon. Treatment efficacy and safety data were obtained from 52-week results of the DEPICT-1 and DEPICT-2 trials and a network meta-analysis of SGLT2 inhibitors in T1DM. Direct healthcare costs, life-years, and quality-adjusted life-years (QALYs) were estimated from a UK payer perspective and discounted at 3.5% annually, using the Cardiff T1DM Model. Sensitivity analyses assessed uncertainty in estimated incremental cost-effectiveness ratios (ICERs). Dapagliflozin 5 mg was associated with gains of 0.23 life-years and 0.42 QALYs, at an additional cost of £4240 per person; corresponding to an ICER of £10 143 versus standard of care. For dapagliflozin 10 mg, incremental life-years, QALYs and costs were 0.24, 0.49 and £2964, respectively; corresponding to an ICER of £6103 versus standard of care. In probabilistic sensitivity analysis, ICER estimates fell below £20 000/QALY in 78% to 90% of simulations. Cost-effectiveness results were sensitive to changes in baseline patient characteristics and treatment effects on glycated haemoglobin; however, ICERs remained below £20 000. At cost-effectiveness thresholds conventionally applied in the UK, dapagliflozin as an adjunct to insulin appears to be a cost-effective treatment option for people with T1DM inadequately controlled by insulin alone.

Cost-effectiveness Analysis of Ribociclib plus Letrozole versus Palbociclib plus Letrozole in the United Kingdom

Objective: To evaluate the cost-effectiveness of ribociclib plus letrozole versus palbociclib plus letrozole in post-menopausal women with hormone receptor positive (HR+) and human epidermal growth receptor 2 negative (HER2-) advanced breast cancer from a UK payer perspective. Methods: A cohort-based partitioned survival model was developed to evaluate the cost-effectiveness of ribociclib plus letrozole versus palbociclib plus letrozole in post-menopausal women with HR+/HER2- advanced breast cancer over a lifetime horizon. The analysis was carried out from a National Health Services and Personal Social Services perspective, and results are presented in incremental costs per quality adjusted life years. Clinical data from three randomized controlled trials (MONALEESA-2, PALOMA-1 and PALOMA-2 studies) were used, and supplemented with available real world evidence. Costs categories comprised of drug acquisition, medical management, and treatment of adverse events. Healthcare resource utilization data were identified from literature and unit costs sourced from secondary sources. Utility values were derived from MONALEESA-2 study and were supported with values identified from literature. Both deterministic and probabilistic analyses were carried out to assess uncertainty. Results: In the base case, treatment with ribociclib plus letrozole increased mean progression free survival (PFS) by 4.1 months and overall survival by 5.0 months compared to palbociclib plus letrozole. Further, treatment with ribociclib plus letrozole resulted in cost-savings of £8464 and incremental QALYs of 0.261, demonstrating that treatment with ribociclib plus letrozole is dominant to treatment with palbociclib plus letrozole. The probabilistic analysis also yielded mean cost-savings of £7914 and mean QALY gain of 0.273. At willingness-to-pay threshold of £30 000 per QALY, treatment with ribociclib plus letrozole had a 92% probability of being cost-effective compared to palbociclib and letrozole. Conclusions: The results of the analysis demonstrate that ribociclib plus letrozole treatment is both cost-saving and a cost-effective option amongst the available cyclin dependent kinase 4/6 inhibitors for the treatment of post-menopausal women with advanced breast cancer. The biggest driver of the cost savings were the lower acquisition costs of ribociclib.

Evaluating the cost-effectiveness of percutaneous closure of a patent foramen ovale versus medical management in patients with a cryptogenic stroke: from the UK payer perspective

Aims: Percutaneous closure of a patent foramen ovale (PFO) is known to lower the risk of recurrent stroke in patients with a cryptogenic stroke. However, the economic implications of transcatheter PFO closure are less well known. From a UK payer perspective, a detailed economic appraisal of PFO closure was performed for prevention of recurrent ischemic stroke in patients with a PFO who had experienced a cryptogenic stroke.Materials and methods: A Markov cohort model was constructed using a 5-year time-horizon with a patient mean age of 45.2 years, reflecting the characteristics reported in the REDUCE trial. Transition probabilities, clinical inputs, costs, and utility values were ascertained from published and national costing sources. Total costs, incremental costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios were calculated, utilizing a discount rate of 3.5%. A range of univariate and probabilistic sensitivity analyses were also performed.Results: When applying a willingness-to-pay (WTP) threshold of £20,000/QALY in accordance with NICE guidelines, PFO closure compared with antiplatelet therapy alone showed a beneficial cost/QALY of £18,584, attained at 4 years. Applying discount rates of 0% and 6% had a negligible effect on the base-case model findings. PFO closure demonstrated a 76.9% probability of being cost-effective at a WTP threshold of £20,000/QALY at a 5-year time-horizon.Limitations: This model focused specifically on UK stroke patients and typically enrolled young (mean age <65 years old) patients. Hence, caution should be taken when comparing data vs non-UK populations, and it remains unclear how older patients might have affected cost-effectiveness findings, as the risk of paradoxical embolism can persist as patients age.Conclusion: Percutaneous closure of a PFO is cost-effective compared with antiplatelet therapy alone, underlining the economic benefits potentially afforded by this treatment in selected patients.

DB4 - COST-EFFECTIVENESS OF DAPAGLIFLOZIN IN ADULT PATIENTS WITH INADEQUATELY CONTROLLED TYPE 1 DIABETES: A HEALTH ECONOMIC ANALYSIS USING 24-WEEK RESULTS FROM THE DEPICT-1 RANDOMISED CONTROLLED TRIAL

The Dapagliflozin Evaluation in Patients With Inadequately Controlled Type 1 Diabetes (DEPICT-1) trial assessed the efficacy and safety of dapagliflozin as an adjunct to insulin in people with inadequately controlled type 1 diabetes mellitus (T1DM). The objective of this study was to estimate the cost-effectiveness of dapagliflozin (5mg or 10mg) versus placebo for people with T1DM in the UK setting. A cost-utility analysis was undertaken from the UK payer perspective using the Cardiff Type 1 Diabetes Model and 24-week data from the DEPICT-1 trial. Treatment effects (reduction in HbA1c, weight and incidence of hypoglycaemia) were maintained for a lifetime unless discontinuation occurred, after which all treatment effects were reversed. Cost and utility data were sourced from the published literature and discounted at 3.5% per annum to estimate the impact of intervention-based resource use and incidence of diabetes-related complications on lifetime costs and quality-adjusted life years (QALYs). Over a lifetime horizon, total costs associated with dapagliflozin (5mg), dapagliflozin (10mg) and placebo, were £47,330, £47,578 and £42,118 per person, respectively. The incremental cost of dapagliflozin treatment was partially offset by the avoidance of hypoglycaemia and microvascular complications. Dapagliflozin was associated with improved life expectancy of 15.95 years (5mg or 10mg) versus 15.75 years with placebo and improved QALYs of 11.76 (5mg) or 11.77 (10mg) versus 11.37 per person with placebo. Corresponding incremental cost-effectiveness ratios were £13,449 and £13,896 per QALY gained for dapagliflozin 5mg and 10mg, respectively, as an add-on to insulin versus placebo. This analysis of 24-week DEPICT-1 data suggests that at commonly applied willingness-to-pay thresholds in a UK setting, dapagliflozin in combination with insulin would be a cost-effective treatment for people with T1DM whose insulin regimen does not provide sufficient glycaemic control.

Development of a health economic model to evaluate the potential benefits of optimal serum potassium management in patients with heart failure

Aims: Patients with heart failure are at increased risk of hyperkalemia, particularly when treated with renin-angiotensin-aldosterone system inhibitor (RAASi) agents. This study developed a model to quantify the potential health and economic value associated with sustained potassium management and optimal RAASi therapy in heart failure patients.Materials and methods: A patient-level, fixed-time increment stochastic simulation model was designed to characterize the progression of heart failure through New York Heart Association functional classes, and predict associations between serum potassium levels, RAASi use, and consequent long-term outcomes. Following internal and external validation exercises, model analyses sought to quantify the health and economic benefits of optimizing both serum potassium levels and RAASi therapy in heart failure patients. Analyses were conducted using a UK payer perspective, independent of costs and utilities related to pharmacological potassium management.Results: Validation against multiple datasets demonstrated the predictive capability of the model. Compared to those who discontinued RAASi to manage serum potassium, patients with normokalemia and ongoing RAASi therapy benefited from longer life expectancy (+1.38 years), per-patient quality-adjusted life year gains (+0.53 QALYs), cost savings (£110), and associated net monetary benefit (£10,679 at £20,000 per QALY gained) over a lifetime horizon. The predicted value of sustained potassium management and ongoing RAASi treatment was largely driven by reduced mortality and hospitalization risks associated with optimal RAASi therapy.Limitations: Several modeling assumptions were made to account for a current paucity of published literature; however, ongoing refinement and validation of the model will ensure its continued accuracy as the clinical landscape of hyperkalemia evolves.Conclusions: Predictions generated by this novel modeling approach highlight the value of sustained potassium management to avoid hyperkalemia, enable RAASi therapy, and improve long-term health economic outcomes in patients with heart failure.

When does economic model type become a decisive factor in health technology appraisals? Learning from the expanding treatment options for relapsing–remitting multiple sclerosis

Objectives: Specific economic model types often become de facto standard for health technology appraisal over time. Markov and discrete event simulation (DES) models were compared to investigate the impact of innovative modeling on the cost-effectiveness of disease-modifying therapies (DMTs) in relapsing–remitting multiple sclerosis (RRMS). Fingolimod was compared to dimethyl fumarate (DMF; in highly active [HA] RRMS), alemtuzumab (in HA RRMS) and natalizumab (in rapidly evolving severe RRMS). Comparator DMTs were chosen to reflect different dosing regimens.Materials and methods: Markov and DES models used have been published previously. Inputs were aligned in all relevant respects, with differences in the modeling of event-triggered attributes, such as relapse-related retreatment, which is inherently difficult with a memoryless Markov approach. Outcomes were compared, with and without different attributes.Results: All results used list prices. For fingolimod and DMF, incremental cost-effectiveness ratios (ICERs) were comparable (Markov: £4206/quality-adjusted life year [QALY] gained versus DES: £3910/QALY gained). Deviations were observed when long-term adverse events (AEs) were incorporated in the DES (Markov: £25,412 saved/QALY lost, versus DES: £34,209 saved/QALY lost, fingolimod versus natalizumab; higher ICERs indicate greater cost-effectiveness). For fingolimod versus alemtuzumab, when relapse-triggered retreatment was included in the DES, large cost differences were observed (difference between incremental cost is £35,410 and QALY is 0.10).Limitations: UK payer perspective, therefore societal approach was not considered. Resource utilization and utilities for both models were not derived from the subpopulations; as the focus is on model type, input limitations that apply to both models are less relevant.Conclusions: Whilst no model can fully represent a disease, a DES allows an opportunity to include features excluded in a Markov structure. A DES may be more suitable for modeling in RRMS for health technology assessment purposes given the complexity of some DMTs. This analysis highlights the capabilities of different model structures to model event-triggered attributes.

The Economic Impact of Switching To Herceptin (TRASTUZUMAB) Biosimilar for The Treatment of Metastatic Breast Cancer From A UK Payer Perspective

Herceptin (trastuzumab) is indicated for the treatment of adult patients with HER2 positive metastatic breast cancer (MBC) as a monotherapy for the treatment of those patients who have received at least two chemotherapy regimens for their metastatic disease. Currently, there are four Herceptin biosimilars under review by the EMA. The aim of the study was to evaluate the economic impact of switching to the Herceptin biosimilar product in patients with MBC from a UK payer perspective. A one-year budget impact model was created in Microsoft Excel to calculate the number of patients in the UK who can receive the biosimilar for MBC. Incident and prevalent populations were both eligible to receive the biosimilar, with a 50% uptake among incident patients, and 25% uptake among prevalent patients. MBC incidence and prevalence rates, and population and treatment characteristics were taken from available literature. Only drug costs were included as the same efficacy and safety was assumed for trastuzumab and the biosimilar. Trastuzumab cost was obtained from the British National Formulary; a 20% discount was assumed with the biosimilar. Patients were assumed to be on treatment without breaks in the base-case scenario. Among the 25,809 patients estimated to be on treatment for MBC, 6,999 (25%) were predicted to receive the biosimilar, resulting in a cost reduction of £89,088,725 (5%).When additional patients who had stopped and restarted treatment with the biosimilar were also accounted for, similar savings were estimated. The results from this economic assessment suggest that the introduction of the biosimilar could lead to considerable savings in the UK. These findings should be evaluated in light of actual treatment and prescribing patterns, and patient preferences.

Cost-Effectiveness of Golimumab in Ankylosing Spondylitis from the UK Payer Perspective.

IntroductionGolimumab is a tumor necrosis factor-α (TNF-α) inhibitor for treatment of patients with severe, active ankylosing spondylitis. This study evaluated the cost-effectiveness of golimumab compared with conventional care and other TNF-α inhibitors in treatment of AS from the UK National Health Service perspective.MethodsA long-term Markov model (with initial decision tree) was developed to simulate the progression of a hypothetical cohort of patients with active AS over a lifetime. The effectiveness outcome was quality-adjusted life-years (QALYs). Utilities were estimated by mapping Bath Ankylosing Spondylitis Functional Index scores, and the primary response measure was ≥50% improvement on the Bath Ankylosing Spondylitis Disease Activity Index at 12 weeks. Direct, medication, and AS management costs were included. Costs and outcomes were discounted at 3.5%.ResultsAll TNF-α inhibitors were comparable to each other and superior to conventional care. The incremental cost-effectiveness ratios (ICERs) for TNF-α inhibitors were £19,070–42,532 per QALY gained compared with conventional care. Analyses of the ICERs for each TNF-α inhibitor compared with conventional care demonstrated that golimumab was the most cost-effective treatment, and that adalimumab and etanercept were dominated by golimumab. Sensitivity analyses confirmed the robustness of these analyses.ConclusionsGolimumab may be considered a cost-effective treatment alternative for patients with active AS. With comparable costs and efficacy among TNF-α inhibitors, the choice of TNF-α inhibitor to treat AS is likely to be driven by patient and physician choice.FundingMerck & Co., Inc.Electronic supplementary materialThe online version of this article (doi:10.1007/s40744-017-0083-1) contains supplementary material, which is available to authorized users.

Cost-effectiveness of osimertinib in the UK for advanced EGFR-T790M non-small cell lung cancer

Aim: This study presents the cost-utility analysis that was developed to inform the NICE health technology assessment of osimertinib vs platinum-based doublet chemotherapy (PDC) in patients with EGFR-T790M mutation-positive non-small cell lung cancer (NSCLC) who have progressed on epidermal growth factor receptor-tyrosine kinase inhibitor (EGFR-TKI) therapy.Methods and materials: A partitioned survival model with three health states (progression-free, progressed disease, and death) from a UK payer perspective and over lifetime (15 years) was developed. Direct costs included disease management, treatment-related (acquisition, administration, monitoring, adverse events), and T790M testing costs. Efficacy and safety data were taken from clinical trials AURA extension and AURA2 for osimertinib and IMPRESS for PDC. An adjusted indirect treatment comparison was applied to reduce the potential bias in the non-randomized comparison. Parametric functions were utilized to extrapolate survival beyond the observed period. Health state utility values were calculated from EQ-5D data collected in the trials and valued using UK tariffs. Resource use and costs were based on published sources.Results: Osimertinib was associated with a gain of 1.541 quality-adjusted life-years (QALYs) at an incremental cost of £64,283 vs PDC (incremental cost-effectiveness ratio [ICER]: £41,705/QALY gained). Scenario analyses showed that none of the plausible scenarios produced an ICER above £44,000 per QALY gained, and probabilistic sensitivity analyses demonstrated a 63.4% probability that osimertinib will be cost-effective at a willingness-to-pay threshold of £50,000.Limitations: The analysis is subject to some level of uncertainty inherent to phase 2 single-arm data and the immaturity of the currently available survival data for osimertinib.Conclusions: Osimertinib may be considered a cost-effective treatment option compared with PDC in the second-line setting in patients with EGFR-T790M mutation-positive NSCLC from a UK payer perspective. Further data from the ongoing AURA clinical trial program will reduce the inherent uncertainty in the analysis.

PSS15 - Budget Impact of Apremilast for Moderate to Severe Plaque Psoriasis in the UK

Apremilast is an orally administered, small-molecule phosphodiesterase-4 inhibitor. It has a novel mechanism of action, targeting multiple steps implicated in the pathogenesis of psoriasis. Apremilast was approved by the European Medicines Agency in 2015 for the treatment of moderate-to-severe chronic plaque psoriasis (PsO) in adult patients who failed to respond to or have a contraindication to, or are intolerant to other systemic therapies. We assessed the budget impact of introducing apremilast in the current treatment portfolio for psoriasis from the UK payer perspective. A 5-year budget impact model was developed using a prevalence-based approach. The target population was estimated based on the total UK population, annual population growth rate, prevalence of PsO, and proportion of patients with moderate-to-severe disease. The total psoriasis population was divided into 3 categories: untreated patients, patients receiving ≥1 conventional systemic non-biologic therapy (SNBT), and patients on biologics. Biologic treatments included adalimumab, etanercept, infliximab, secukinumab, and ustekinumab. Biosimilars for etanercept and infliximab were also considered. Drug costs were quoted from the British National Formulary (BNF) and the Monthly Index of Medical Specialties (MIMS). Administration costs were obtained from the National Clinical Guideline Centre (NCGC); physician visit and laboratory test costs were informed by National Health Service (NHS) reference costs, the NCGC, and literature values. Over a 5-year period, the number of patients treated with apremilast was estimated to increase from 69 to 1,980. Inclusion of apremilast decreased the total budget by 2.19% (£735,221,377 vs. £751,645,055) relative to the world without. In the final year, drug acquisition costs decreased by 4.43% (£124,099,644 vs. £129,856,022), administration costs by 18.67% (£671,543 vs. £825,667), and monitoring costs by 2.81% (£21,291,886 vs. £21,907,514), yielding a net cost saving of 4.28%. Inclusion of apremilast in the PsO treatment regimen in the UK was cost saving in all respects

AbobotulinumtoxinA in the management of cervical dystonia in the United Kingdom: a budget impact analysis.

BackgroundCervical dystonia (CD) can be effectively managed by a combination of botulinum neurotoxin A (BoNT-A) and conventional therapy (skeletal muscle relaxants and rehabilitative therapy), but the costs of different interventions in the UK vary.MethodsA budget impact model was developed from the UK payer perspective with a 5-year time horizon to evaluate the effects of changing market shares of abobotulinumtoxinA, onabotulinumtoxinA, and incobotulinumtoxinA, and best supportive care from the UK payer perspective. Epidemiological and resource use data were retrieved from the published literature and clinical expert opinion. Deterministic sensitivity analyses were performed to determine the parameters most influential on the budgetary findings under base case assumptions.ResultsUnder base case assumptions, an increased uptake of abobotulinumtoxinA showed an accumulated savings of £2,250,992 by year 5. Treatment per patient per year with onabotulinumtoxinA and incobotulinumtoxinA costs more when compared to treatment with abobotulinumtoxinA. One-way sensitivity analyses showed that the prevalence of CD, dose per injection of each of the BoNT-As, and time to reinjection of incobotulinumtoxinA and abobotulinumtoxinA influenced the base case findings most.ConclusionThere is potential for cost savings associated with the greater use of abobotulinumtoxinA rather than other BoNT-A treatments, permitting more patients to benefit more from effective BoNT-A treatment with a fixed budget.

Budget impact analysis of botulinum toxin A therapy for upper limb spasticity in the United Kingdom.

BackgroundBotulinum toxin A (BoNT-A) is an effective treatment for patients with upper limb spasticity (ULS), which is a debilitating feature of upper motor neuron lesions. BoNT-A preparations available in the UK are associated with different costs.MethodsWe developed a budget impact model to assess the effect of changing market shares of different BoNT-A formulations – abobotulinumtoxinA, onabotulinumtoxinA, and incobotulinumtoxinA – and best supportive care, from the UK payer perspective, over a 5-year time horizon. Epidemiological and resource use data were derived from published literature and clinical expert opinion. One-way sensitivity analyses were performed to determine parameters most influential on budget impact.ResultsBase-case assumptions showed that an increased uptake of abobotulinumtoxinA resulted in a 5-year savings of £6,283,829. Treatment with BoNT-A costs less than best supportive care per patient per year, although treating a patient with onabotulinumtoxinA (£20,861) and incobotulinumtoxinA (£20,717) cost more per patient annually than with abobotulinumtoxinA (£19,800). Sensitivity analyses showed that the most influential parameters on budget were percentage of cerebral palsy and stroke patients developing ULS, and the prevalence of stroke.ConclusionStudy findings suggest that increased use of abobotulinumtoxinA for ULS in the UK could potentially reduce total ULS cost for the health system and society.

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.

Comparison of the Cost-effectiveness of New Oral Anticoagulants for the Prevention of Stroke and Systemic Embolism in Atrial Fibrillation in a UK Setting

PurposeThree new oral anticoagulants (NOACs) have recently become available in the United Kingdom as an alternative to warfarin in the prevention of stroke and systemic embolism in atrial fibrillation. This study examines the relative cost-effectiveness of dabigatran (BID dosing of 150 mg or 110 mg based on patient age), rivaroxaban, and apixaban from a UK payer perspective. MethodsA previously published model that follows up patients through treatment of atrial fibrillation during a lifetime was adapted to allow comparison of the 3 NOACs and warfarin. Acute thromboembolic and bleeding events, as well as long-term consequences of stroke, intracranial hemorrhage, and acute myocardial infarction, were tracked. Relative efficacy was calculated from a formal indirect treatment comparison using data from the 3 key trials (Randomized Evaluation of Long-Term Anticoagulation Therapy, Rivaroxaban Once-daily oral direct factor Xa inhibition Compared with vitamin K antagonism for prevention of stroke and Embolism Trial in Atrial Fibrillation, and Apixaban for the Prevention of Stroke in Subjects With Atrial Fibrillation) of the NOACs. Data from the rivaroxaban trial were adjusted for the difference in international normalized ratio control among warfarin patients versus the other 2 trials. Model outputs included total costs, event rates, and quality-adjusted life-years. FindingsAmong the patients taking NOACs, those taking dabigatran had the highest total QALYs (7.68 QALYs), followed by apixaban (7.63 QALYs) and rivaroxaban (7.47 QALYs). Patients taking dabigatran had the lowest total lifetime costs (£23,342), followed by apixaban (£24,014) and rivaroxaban (£25,220). The differences between dabigatran and apixaban were modest but consistent in sensitivity analyses, with the directionality only changing at the limits of the CIs for the relative risks of ischemic stroke or intracranial hemorrhage or when assuming that both treatment discontinuation and post-event disability rates differ by drug. ImplicationsDabigatran was found to be economically dominant over rivaroxaban and apixaban in the UK setting. These economic findings are based on relative clinical efficacy from an indirect treatment comparison and would benefit from any data of direct comparisons of the NOACs in the future.