Quality-adjusted Life Year Gain Research Articles

Patients on vitamin K treatment: is switching to direct-acting oral anticoagulation cost-effective? A target trial on a prospective cohort

AimsDirect-acting oral anticoagulants (DOACs) have, to a substantial degree, replaced vitamin K antagonists (VKA) as treatments for stroke prevention in atrial fibrillation (AF) patients. However, evidence on the real-world causal...

Cost-utility analysis of proton beam therapy for locally advanced esophageal cancer in Japan.

Proton beam therapy (PBT) has recently been included in Japan's health insurance benefit package for certain cancer types. This study aimed to determine the cost-effectiveness of PBT as a replacement for conventional three-dimensional conformal radiotherapy (3D-CRT) for locally advanced esophageal cancer (LAEC) that is not covered by social insurance. We estimated the incremental cost-effectiveness ratio (ICER) of PBT as a replacement for 3D-CRT, using clinical evidence from the literature and expert opinions. We used an economic model, decision tree, and Markov model to illustrate the courses followed by patients with LAEC. Effectiveness was estimated as quality-adjusted life years (QALY) using utility weights for the health state. Social insurance fees were calculated as costs. We assumed two base cases depending on the two existing levels of fees for PBT in social insurance: 2,735,000 Japanese yen (US$20,652) or 1,600,000 yen (US$13,913). The stability of the ICER against these assumptions was appraised using sensitivity analysis. The effectiveness of PBT and 3D-CRT was 2.62 and 2.51 QALY, respectively. The estimated ICER was 14,025,268 yen (US$121,958) per QALY for the higher fee level and 7,026,402 yen (US$61,099) for the lower fee level. According to the Japanese threshold for cost-effectiveness of anticancer therapy of 7,500,000 yen (US$65,217) per QALY gain, the inclusion of PBT for LAEC in the benefit package of social insurance is cost-effective if a lower fee is applied. PBT is a cost-effective alternative to 3D-CRT for LAEC and making it available to patients under social insurance could be justifiable.

Cost-effectiveness analysis of upadacitinib as a treatment option for patients with rheumatoid arthritis in the Kingdom of Saudi Arabia

Aim To evaluate cost-effectiveness of upadacitinib (targeted synthetic-disease modifying anti-rheumatic drug [ts-DMARD]) as first-line (1 L) treatment versus current treatment among patients with rheumatoid arthritis (RA) in the Kingdom of Saudi Arabia (KSA), who had an inadequate response to prior conventional-synthetic (csDMARDs) and/or biologic-DMARDs (bDMARDs). Methods This Excel-based model included patients with moderate (Disease Activity Score [DAS28]: >3.2 to ≤5.1) or severe RA (DAS28 > 5.1). Cost-effectiveness of current treatment (1 L: adalimumab-originator/biosimilar; second-line (2 L): other bDMARDs/tofacitinib) was compared against a new treatment involving two scenarios (1 L: upadacitinib, 2 L: adalimumab-biosimilar [scenario-1]/adalimumab-originator [scenario-2]) for a 10-year time-horizon from societal perspective. Model outcomes included direct and indirect costs, quality-adjusted life-years (QALYs), hospitalization days, number of orthopedic surgeries, and incremental cost-utility ratio (ICUR) per QALY. Results With the current pathway, estimated total societal costs for 100 RA patients over 10-year period were Saudi Riyal (SAR) 50,450,354 (United States dollars [USD] 13,453,428) (moderate RA) and SAR50,013,945 (USD13,337,052) (severe RA). New pathway (scenario-1) showed that in patients with moderate-to-severe RA, upadacitinib led to higher QALY gain (+8.99 and +15.63) at lower societal cost (cost difference: -SAR2,023,522 [-USD539,606] and -SAR3,373,029 [-USD899,474], respectively). Thus, as 1 L, upadacitinib projects “dominant” ICUR per QALY over current pathway. Moreover, in alternate pathway (scenario-2), upadacitinib also projects “dominant” ICUR per QALY for patient with severe RA (QALY gain: +15.63; cost difference: -SAR 164,536 [-USD43,876]). However, moderate RA was associated with additional cost of SAR1,255,696 (USD334,852) for improved QALY (+8.99) over current pathway (ICUR per QALY: SAR139,742 [USD37,264]). Both scenarios resulted in reduced hospitalization days (scenario-1: −14.83 days; scenario-2: −11.41 days) and number of orthopedic surgeries (scenario-1: −8.36; scenario-2: −6.54) for moderate-to-severe RA over the current treatment pathway. Conclusion Upadacitinib as 1 L treatment in moderate-to-severe RA can considerably reduce healthcare resource burden in KSA, majorly due to reduced drug administration/monitoring/hospitalization/surgical and indirect costs.

Adding a Gene Expression Profile Test to Aid Differential Diagnosis and Treatment in Aggressive Large B-Cell Lymphoma: An Early Exploratory Economic Evaluation.

Adding gene expression profiles (GEPs) to the current diagnostic work-up of aggressive large B-cell lymphomas may lead to the reclassification of patients, treatment changes and improved outcomes. A GEP test is in development using TempO-Seq® technology to distinguish Burkitt lymphoma (BL) and primary mediastinal large B-cell lymphoma (PMBCL) from diffuse large B-cell lymphoma (DLBCL), and to classify patients with DLBLC and to predict the benefit of (e.g.) adding bortezomib to R-CHOP therapy (RB-CHOP). This study aims to estimate the potential impact of a GEP test on costs and health outcomes to inform pricing and evidence generation strategies. Three decision models were developed comparing diagnostic strategies with and without GEP signatures over a lifetime horizon using a UK health and social care perspective. Inputs were taken from a recent clinical trial, literature and expert opinion. We estimated the maximum price of the test using a threshold of Great Britain Pound (GBP) 30,000 per quality-adjusted life-year (QALY). Sensitivity analyses were conducted. The estimated maximum threshold price for a combined test to be cost effective is GBP 15,352. At base-case values, the BL signature delivers QALY gains of 0.054 at an additional cost of GBP 275. This results in a net monetary benefit at a threshold of GBP 30,000 per QALY of GBP 1345. For PMBCL, the QALY gain was 0.0011 at a cost saving of GBP 406 and the net monetary benefit was GBP 437. The hazard ratio for the impact of treating BL less intensively must be at least 1.2 for a positive net monetary benefit. For identifying patients with the DLBCL subtype responsive to bortezomib, QALY gain was 0.2465 at a cost saving of GBP 6175, resulting in a net monetary benefit of GBP 13,570. In a probabilistic sensitivity analysis using 1000 simulations, a testing strategy was superior to a treat all with R-CHOP strategy in 81% of the simulations and with a cost saving in 92% assuming a cost price of zero. Our estimates show that the combined test has a high probability of being cost effective. There is good quality evidence for the benefit of subtyping DLBCL but the evidence on the number of patients reclassified to or from BL and PMBCL and the impact of a more precise diagnosis and the cost of treatment is weak. The developers can use the price estimate to inform a return on investment calculations. Evidence will be required of how well the TempO-Seq® technology performs compared to the testing GEP technology used for subtyping in the recent clinical trial. For BL and PMBCL elements of the test, evidence would be required of the number of patients reclassified and improved costing information would be useful. The diagnostic and therapeutic environment in haematological malignancies is fast moving, which increases the risk for developers of diagnostic tests.

Cost-effectiveness analysis of secukinumab versus standard of care in the treatment of psoriatic arthritis: A Vietnam National health insurance perspective

Psoriatic arthritis (PsA) is a chronic, systemic inflammatory disease. This study assessed the cost-effectiveness of secukinumab, an interleukin-17A inhibitor, versus standard of care (SoC) from Vietnam’s National Health Insurance (NHI) perspective. Model-based cost-effectiveness analysis, the structure was a 3-month decision tree leading into a Markov model. Clinical parameters, including responses at 3 months, were from the FUTURE 2 trial. Cost parameters were from real data at the National Hospital of Dermatology and Venereology and a research period. The outcome included total costs and quality-adjusted life years (QALYs) over a life-time horizon (3.5% annual discount for both outcomes and cost), and incremental cost-effectiveness ratios (ICER). The robustness of the study findings was evaluated via sensitivity analysis. Patients treated with secukinumab achieved with QALY gain 1.16 versus SoC, but associated with higher total costs was 211.3 million VND over lifetime horizon. ICER for secukinumab vs SoC was 181.6 million VND lower than a willingness to pay threshold recommended in Vietnam (3GDP per capita equal to 286.8 million VND). Deterministic sensitivity analysis indicated that parameters related to Health Assessment Questionnaire scores were most influential. At the willingness to pay threshold of three times the GDPpc, the probability of secukinumab being cost-effective was 90.3% in the probabilistic sensitivity analysis. Secukinumab is a cost-effectiveness treatment versus SoC for PsA patients from the National health insurance’s perspective of Vietnam.

Cost-utility analysis of robotic arm-assisted medial compartment knee arthroplasty.

To perform an incremental cost-utility analysis and assess the impact of differential costs and case volume on the cost-effectiveness of robotic arm-assisted unicompartmental knee arthroplasty (rUKA) compared to manual (mUKA). This was a five-year follow-up study of patients who were randomized to rUKA (n = 64) or mUKA (n = 65). Patients completed the EuroQol five-dimension questionnaire (EQ-5D) preoperatively, and at three months and one, two, and five years postoperatively, which was used to calculate quality-adjusted life years (QALYs) gained. Costs for the primary and additional surgery and healthcare costs were calculated. rUKA was associated with a relative 0.012 QALY gain at five years, which was associated with an incremental cost per QALY of £13,078 for a unit undertaking 400 cases per year. A cost per QALY of less than £20,000 was achieved when ≥ 300 cases were performed per year. However, on removal of the cost for a revision for presumed infection (mUKA group, n = 1) the cost per QALY was greater than £38,000, which was in part due to the increased intraoperative consumable costs associated with rUKA (£626 per patient). When the absolute cost difference (operative and revision costs) was less than £240, a cost per QALY of less than £20,000 was achieved. On removing the cost of the revision for infection, rUKA was cost-neutral when more than 900 cases per year were undertaken and when the consumable costs were zero. rUKA was a cost-effective intervention with an incremental cost per QALY of £13,078 at five years, however when removing the revision for presumed infection, which was arguably a random event, this was no longer the case. The absolute cost difference had to be less than £240 to be cost-effective, which could be achieved by reducing the perioperative costs of rUKA or if there were increased revision costs associated with mUKA with longer follow-up.

Cost-Utility Analysis of the Port Delivery System with Ranibizumab for Neovascular Age-Related Macular Degeneration

To quantify the cost-utility ratio of the ranibizumab Port Delivery System (PDS; SUSVIMO) versus intravitreal ranibizumab injections for treating neovascular age-related macular degeneration (nAMD) based upon Archway Phase 3 Trial data. Cost-utility analysis. Archway Phase 3 Clinical Trial nAMD participants previously responsive to anti-VEGF therapy were randomized 3:2. Two hundred forty received PDS refills q 24 weeks and 162 received ranibizumab injections. Ophthalmic patient, time tradeoff utilities, direct medical and societal cost perspectives, 12-year, 1-year, and 5-year timelines, United States 2022 real dollars, and a 3% annual discount rate were employed. Utilities were adjusted for nAMD conversion in fellow eyes during the 12-year, mean participant life expectancy. Premature death associated with severe vision loss was integrated as per the population-based Salisbury Eye Evaluation Study. Quality-adjusted life-year (QALY) accruals, costs, and incremental and average cost-utility ratios in $/QALY (dollars expended per QALY gained). Versus no therapy, the 1-year PDS QALY gain was 0.0156 (6.8%) versus 0.0063 (0.82%) for intravitreal injections (P < 0.001), whereas the respective 12-year QALY gains were 1.714 (28%) and 1.639 (26.8%) (P= 0.99). One-year direct PDS ophthalmic costs totaled $21 825 with 2 ranibizumab fills, whereas ranibizumab injection therapy totaled $18 405 with 11.8 injections. The 1-year incremental PDS $/QALY versus injections was cost effective at $75 497/QALY. Five-year PDS therapy was not incrementally cost effective at $304 108/QALY, nor was the 12-year therapy at $761 646/QALY. Average 12-year cost-utility ratios were $78773/QALY for the PDS and $47 917/QALY for injection therapy. Adding-$476 442 12-year offsetting societal costs netted $314 521 to society per PDS participant versus $370 958 per participant for injection therapy. Ranibizumab PDS therapy was not incrementally cost effective versus ranibizumab injection therapy at 12 or 5 years but was at 1 year. Injection therapy had a more favorable 12-year average cost-utility ratio. Vision gain was the major determinant of participant value gain and was the same for both interventions. Both interventions were highly cost effective utilizing average cost-utility analysis with the societal cost perspective. Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

Early lowering of LDL-C is a more cost-effective strategy than lowering LDL-C later in life: a cost-effectiveness analysis using Mendelian Randomisation

Abstract Background Early control of low-density lipoprotein-cholesterol (LDL-C) is an effective way to reduce the risk of coronary heart disease (CHD). However, a comprehensive assessment of cost-effectiveness of different lipid-lowering strategies (LLS) for primary prevention of CHD across different ages is lacking. Objective To assess the cost-effectiveness of four different LLS initiated at ages 30, 40, 50, and 60 years on the primary prevention of CHD from the UK National Health Service perspective. Design, setting and participants: We developed a microsimulation model, based on the causal effect of LDL-C on myocardial infarction (MI) or coronary death derived from Mendelian randomisation, comparing initiation of an LLS to current standard of care (control). Data were primarily drawn from 458,727 participants of the UK Biobank study with available data on date of birth, LDL-C, and who were free of MI at first assessment (between 2006 and 2010), with follow-up until 2021. Interventions: The four LLS were: 1) low/moderate intensity statins; 2) high intensity statins; 3) low/moderate intensity statins and ezetimibe; and 4) inclisiran. Main outcomes measures: The incremental cost-effectiveness ratio (ICER), defined as the incremental healthcare costs divided by the incremental quality-adjusted life years (QALYs) for each LLS compared to control, with 3.5% annual discounting. Results The most effective intervention, low/moderate intensity statins and ezetimibe, was projected to lead to a gain in QALYs of 0.068 per person at age 30 and 0.026 at age 60 compared to control (Figure). The age of intervention with the lowest ICER was age 40, with ICERs of £2,524 (95% uncertainty interval: 1,383, 3,796), £4,466 (3,163, 6,134) £11,118 (8,632, 14,177), and £1,405,123 (1,117,1113, 1,789,012) per QALY gained for strategies 1-4 from age 40 years, respectively (Table). Conclusions LDL-C lowering from early ages is a more cost-effective strategy than late intervention. The approach to primary prevention of CHD may improve with a shift to early and sustained lowering of LDL-C. Figure – Results of 1,000 probabilistic sensitivity analyses presented in a common cost-effectiveness plane, by age of intervention. Inclisiran excluded due to very high costs. Solid line: £20,000 per quality-adjusted life year (QALY) willingness-to-pay threshold; dashed line: £30,000 per QALY willingness-to-pay threshold. Any dot to the right of these lines is considered cost-effective. Table – Summary of LLS interventions by age of intervention. Abbreviations: MI – Myocardial infarction; YLL – Years of life lived; QALYs – Quality-adjusted life-years; ICER – Incremental cost-effectiveness ratio.FigureTable

Cost-utility analysis of venetoclax in combination with obinutuzumab as first-line treatment of chronic lymphocytic leukaemia in Spain

ObjectiveVenetoclax in combination with obinutuzumab has significantly improved efficacy versus immunochemotherapy (progression-free survival) in patients with chronic lymphocytic leukaemia who have not received prior treatment. The objective of this study was to evaluate its efficiency in Spain using a cost-utility analysis. MethodUsing a partitioned-survival analysis model adapted to the Spanish context and based on three health states (progression-free survival, survival after progression, and death), a simulation of the evolution of patients who were candidates for initiating first-line treatment was conducted for a lifetime time horizon. Venetoclax in combination with obinutuzumab was compared to the most commonly used therapeutic options for these patients at the time of study design: chlorambucil in combination with obinutuzumab, ibrutinib, fludarabine in combination with cyclophosphamide and rituximab, and bendamustine in combination with rituximab. In order to estimate survival curves, efficacy data were derived from the CLL14 trial and a network meta-analysis. The analysis was conducted from the perspective of the Spanish National Healthcare System and included direct healthcare costs (i.e. pharmacological costs and their administration), and those associated with the management of the disease and adverse events. The resource use was validated by an expert group. Quality of life data were used to estimate the quality-adjusted life years obtained for each alternative. A threshold of €25,000/quality-adjusted life years was used. The robustness of the model was evaluated using deterministic and probabilistic sensitivity analyses. ResultsVenetoclax in combination with obinutuzumab was shown to be a dominant alternative compared to the rest of the treatment alternatives, with a lower cost per patient (€-67,869 compared to chlorambucil in combination with obinutuzumab, €-375,952 compared to ibrutinib, €-61,996 compared to fludarabine in combination with cyclophosphamide and rituximab, and €-77,398 compared to bendamustine in combination with rituximab). It also had a greater gain in quality-adjusted life years (0.551 quality-adjusted life years gained compared to chlorambucil in combination with obinutuzumab and ibrutinib, 1.639 quality-adjusted life years gained compared to fludarabine in combination with cyclophosphamide and rituximab, and 1.186 quality-adjusted life years gained compared to bendamustine in combination with rituximab). Between 68% and 85% of the simulations performed in the sensitivity analysis showed that venetoclax in combination with obinutuzumab had lower costs and more quality-adjusted life years gained. ConclusionsVenetoclax in combination with obinutuzumab is an efficient and dominant alternative for treating previously untreated patients with chronic lymphocytic leukaemia compared to the available alternatives and from the perspective of the Spanish National Health System.

Estimating the cost-effectiveness threshold of advanced non-small cell lung cancer in China using mean opportunity cost and contingent valuation method

ObjectivesMonetizing health has sparked controversy and has implications for pricing strategies of emerging health technologies. Medical insurance payers typically set up thresholds for quality-adjusted life years (QALY) gains based on health productivity and budget affordability, but they rarely consider patient willingness-to-pay (WTP). Our study aims to compare Chinese payer threshold and patient WTP toward QALY gain of advanced non-small cell lung cancer (NSCLC) and to inform a potential inclusion of patient WTP under more complex decision-making scenarios.MethodsA regression model was constructed with cost as the independent variable and QALY as the dependent variable, where the regression coefficients reflect mean opportunity cost, and by transforming these coefficients, the payer threshold can be obtained. Patient WTP was elicited through a contingent valuation method survey. The robustness of the findings was examined through sensitivity analyses of model parameters and patient heterogeneity.ResultsThe payer mean threshold in the base-case was estimated at 150,962 yuan (1.86 times per capita GDP, 95% CI 144,041–159,204). The two scenarios analysis generated by different utility inputs yielded thresholds of 112,324 yuan (1.39 times per capita GDP) and 111,824 yuan (1.38 times per capita GDP), respectively. The survey included 85 patients, with a mean WTP of 148,443 yuan (1.83 times per capita GDP, 95% CI 120,994–175,893) and median value was 106,667 yuan (1.32 times the GDP per capita). Due to the substantial degree of dispersion, the median was more representative. The payer threshold was found to have a high probability (98.5%) of falling within the range of 1–2 times per capita GDP, while the robustness of patient WTP was relatively weak.ConclusionsIn China, a country with a copayment system, payer threshold was higher than patient WTP, indicating that medical insurance holds significant decision-making authority, thus temporarily negating the need to consider patient WTP.

Cost-Effectiveness of Carbidopa-Levodopa Enteral Suspension for Advanced Parkinson's Disease in the United States.

Carbidopa/levodopa enteral suspension (CLES) is indicated for the treatment of advanced Parkinson's disease (aPD) with severe motor fluctuations. To determine the cost, quality-adjusted life years (QALY), and cost-effectiveness of CLES compared to the standard-of-care (SoC) for aPD patients in the United States (US), using real-world data. A published Markov model, comprising of 25 health states and a death state, (defined by a combination of the Hoehn and Yahr scale and waking time spent in OFF-time) was adapted to estimate the benefits for CLES versus oral SoC over a patient's lifetime in the US healthcare setting. Clinical inputs were based on a clinical trial and a registry study; utility inputs were sourced from the Adelphi-Disease Specific Programmes. CLES compared to SoC was associated with incremental costs ($1,031,791 vs. $1,025,180) and QALY gain (4.61 vs. 3.76), resulting in an incremental cost-effectiveness ratio of $7711/QALY. CLES is a cost-effective treatment for aPD patients with medication resistant motor fluctuations. © 2023 AbbVie, Inc and The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Which Patients gain more from short-term planned hospitalization?

Abstract Background The measurement of health-related quality of life (HRQoL) has become one of the most important components in the evaluation of healthcare outcomes. The EQ-5D-5L is an econometric tool to transform HRQoL into Quality Adjusted Life Year (QALY). The study aims to measure the health gain, expressed in QALY, provided by an Italian clinic. Methods A retrospective study was conducted on 137 geriatric patients admitted, for short-term planned rehabilitation hospitalization, from March 2018 to March 2020. Patients completed the EQ-5D-5L questionnaire at both admission and discharge. We calculated QALY from the difference between EQ-5D-5L at discharge and admission, assuming that health gains (discounted at the rate of 3.5%) accumulate up to one year after discharge. A risk adjustment has been done through the Cumulative Illness Rating Scale (CIRS). We stratified our sample by gender, age, BMI and clinical severity. Descriptive (median and interquartile range) and statistical (Mann-Whitney and Dunn's Test) analysis was carried out using STATA. Results In our sample (62.8% female) the median age was 82[15] years old. The median length of stay was 16[17] days. Patients gained 0.14[0.19] QALY: this was greater (p &amp;lt; 0.05) for female (0,15[0,14]) than males (0,12[0,14]). According to age groups, patients &amp;gt;75 years old had a lower QALY (0.12[0.26]) than both patients ≤65 years (0.16[0.15]) and those between 65 and 75 years (0.15[0.12]), while patients younger than 65 years had a higher QALY gain than the other two groups (p &amp;gt; 0.05). The obese class I (0.31[0.46]) gained more QALY than the other BMI classes (p &amp;lt; 0,05), except that obese class II. Patients with a Severity Index of CIRS higher than the median of our sample had a greater health gain (0.18[0.20] vs. 0.12[0.20]) (p &amp;gt; 0.05). Conclusions The EQ-5D-5L allowed us to quantify the ‘health gain’ obtained by patients through its transformation into QALY. The best results were obtained in female and obese class I patients. Key messages • The outcome assessment is essential when performing healthcare. • The QALY allows us to assess quantitative and qualitative life expectancy's increases provided by healthcare.

Public preferences in healthcare resource allocation: A discrete choice experiment in South Korea

ObjectivesThis study aimed to explore the public view on priority-setting criteria for healthcare resource allocation. Specifically, it investigates how the value of a quality-adjusted life year (QALY) varies depending on patient characteristics. MethodsA discrete choice experiment was conducted using an online sample of the general South Korean population. Respondents were presented with two competing treatment scenarios. The attributes of the scenarios were age at disease onset, life expectancy without treatment, life-years gain with treatment, health-related quality of life (HRQoL) without treatment, and HRQoL gains with treatment.Two hundred choice sets were generated and randomly allocated into 20 blocks. A conditional logit model was used to estimate the factors affecting the respondents' choices. ResultsA total of 3,482 respondents completed the survey. The larger the QALY gain, the more likely it was that the scenario would be chosen but with a diminishing marginal value. Respondents prioritized 40-year-old patients over 5-year-olds and 5-year-olds over 70-year-olds and prioritized baseline HRQoL of 40% and 60% over 20%. Patients at the end of life were not preferred to those with a longer life expectancy. ConclusionOverall, respondents preferred health-maximizing options without explicit consideration for end-of-life patients or those with poor health. In addition, they revealed a kinked preference for patient age, prioritizing middle-aged patients over children and older people

Estimating the cost-effectiveness of intermittently scanned continuous glucose monitoring in adults with type 1 diabetes in England.

We previously showed that intermittently scanned continuous glucose monitoring (isCGM) reduces HbA1c at 24 weeks compared with self-monitoring of blood glucose with finger pricking (SMBG) in adults with type 1 diabetes and high HbA1c levels (58-97 mmol/mol [7.5%-11%]). We aim to assess the economic impact of isCGM compared with SMBG. Participant-level baseline and follow-up health status (EQ-5D-5L) and within-trial healthcare resource-use data were collected. Quality-adjusted life-years (QALYs) were derived at 24 weeks, adjusting for baseline EQ-5D-5L. Participant-level costs were generated. Using the IQVIA CORE Diabetes Model, economic analysis was performed from the National Health Service perspective over a lifetime horizon, discounted at 3.5%. Within-trial EQ-5D-5L showed non-significant adjusted incremental QALY gain of 0.006 (95% CI: -0.007 to 0.019) for isCGM compared with SMBG and an adjusted cost increase of £548 (95% CI: 381-714) per participant. The lifetime projected incremental cost (95% CI) of isCGM was £1954 (-5108 to 8904) with an incremental QALY (95% CI) gain of 0.436 (0.195-0.652) resulting in an incremental cost-per-QALY of £4477. In all subgroups, isCGM had an incremental cost-per-QALY better than £20,000 compared with SMBG; for people with baseline HbA1c >75 mmol/mol (9.0%), it was cost-saving. Sensitivity analysis suggested that isCGM remains cost-effective if its effectiveness lasts for at least 7 years. While isCGM is associated with increased short-term costs, compared with SMBG, its benefits in lowering HbA1c will lead to sufficient long-term health-gains and cost-savings to justify costs, so long as the effect lasts into the medium term.

Analysis of the cost-effectiveness of proton beam therapy for unresectable pancreatic cancer in Japan.

Proton beam therapy (PBT) has recently been included in Japan's social health insurance benefits package. This study aimed to determine the cost-effectiveness of PBT for unresectable, locally advanced pancreatic cancer (LAPC) as a replacement for conventional photon radiotherapy (RT). We estimated the incremental cost-effectiveness ratio (ICER) of PBT as a replacement for three-dimensional conformal RT (3DCRT), a conventional photon RT, using clinical evidence in the literature and expense complemented by expert opinions. We used a decision tree and an economic and Markov model to illustrate the disease courses followed by LAPC patients. Effectiveness was estimated as quality-adjusted life years (QALY) using utility weights for the health state. Social insurance fees were calculated as the costs. The stability of the ICER against the assumptions made was appraised using sensitivity analyses. The effectiveness of PBT and 3DCRT was 1.67610615 and 0.97181271 QALY, respectively. The ICER was estimated to be ¥5,376,915 (US$46,756) per QALY. According to the suggested threshold for anti-cancer therapy from the Japanese authority of ¥7,500,000 (US$65,217) per QALY gain, such a replacement would be considered cost-effective. The one-way and probabilistic sensitivity analyses demonstrated stability of the base-case ICER. PBT, as a replacement for conventional photon radiotherapy, is cost-effective and justifiable as an efficient use of finite healthcare resources. Making it a standard treatment option and available to every patient in Japan is socially acceptable from the perspective of health economics.

Cost-effectiveness of somatrogon in the Spanish pediatric population with growth hormone deficiency

ABSTRACT Objective To analyze the cost-effectiveness of weekly somatrogon compared to daily growth hormones (GH-d) in the pediatric population of Spain with growth hormone deficiency (GHD). Methods Markov model with two states (patients with or without GH-d or somatrogon treatment) in prepubertal children (3 to 11 years and 3 to 12 years in girls and boys, respectively) with GHD in isolation or as part of multiple pituitary hormone deficiency and without previous treatment, from the perspective of the National Health System. The simulation of the economic model ends at the age of 18. The costs of hormones and monitoring were obtained from Spanish sources. The utilities were obtained from the literature. Spanish clinical experts validated the assumptions of the model. Results In the deterministic analysis, somatrogon would be cost-effective, compared to GH-d, with a cost per QALY (quality-adjusted life year) gained of €19,259 and a clinically relevant QALY gain (0.336). This result was confirmed in deterministic sensitivity analyses. According to the probabilistic analysis, somatrogon would be the dominant treatment, with a 61% probability of a willingness to pay of €25,000 per QALY gained. Conclusion Compared to GH-d, somatrogon is cost-effective in the Spanish pediatric population with GHD.

Medico-economic impact of thoracoscopy versus thoracotomy in lung cancer: multicentre randomised controlled trial (Lungsco01)

BackgroundLungsco01 is the first study assessing the real benefits and the medico-economic impact of video-thoracoscopy versus open thoracotomy for non-small cell lung cancer in the French context.MethodsTwo hundred and fifty nine adult patients from 10 French centres were randomised in this prospective multicentre randomised controlled trial, between July 29, 2016, and November 24, 2020. Survival from surgical intervention to day 30 and later was compared with the log-rank test. Total quality-adjusted-life-years (QALYs) were calculated using the EQ-5D-3L®. For medico-economic analyses at 30 days and at 3 months after surgery, resources consumed were valorised (€ 2018) from a hospital perspective. First, since mortality was infrequent and not different between the two arms, cost-minimisation analyses were performed considering only the cost differential. Second, based on complete cases on QALYs, cost-utility analyses were performed taking into account cost and QALY differential. Acceptability curves and the 95% confidence intervals for the incremental ratios were then obtained using the non-parametric bootstrap method (10,000 replications). Sensitivity analyses were performed using multiple imputations with the chained equation method.ResultsThe average cumulative costs of thoracotomy were lower than those of video-thoracoscopy at 30 days (€9,730 (SD = 3,597) vs. €11,290 (SD = 4,729)) and at 3 months (€9,863 (SD = 3,508) vs. €11,912 (SD = 5,159)). In the cost-utility analyses, the incremental cost-utility ratio was €19,162 per additional QALY gained at 30 days (€36,733 at 3 months). The acceptability curve revealed a 64% probability of efficiency at 30 days for video-thoracoscopy, at a widely-accepted willingness-to-pay threshold of €25,000 (34% at 3 months). Ratios increased after multiple imputations, implying a higher cost for video-thoracoscopy for an additional QALY gain (ratios: €26,015 at 30 days, €42,779 at 3 months).ConclusionsGiven our results, the economic efficiency of video-thoracoscopy at 30 days remains fragile at a willingness-to-pay threshold of €25,000/QALY. The economic efficiency is not established beyond that time horizon. The acceptability curves given will allow decision-makers to judge the probability of efficiency of this technology at other willingness-to-pay thresholds.Trial registrationNCT02502318.

An economic evaluation of radiotherapy for patients with symptomatic Ledderhose disease

BackgroundEvidence for effectiveness of radiotherapy for Ledderhose disease was demonstrated in the LedRad-study. However, the health economic impact of Ledderhose disease is unclear. Therefore, an economic evaluation alongside the LedRad-study was planned. MethodsThe economic evaluation was performed as a cost-effectiveness and cost-utility analysis from the societal perspective. Primary outcome parameters were pain burden and Quality Adjusted Life Years (QALY), until 12 months after the end of treatment. Secondary analyses were performed with outcomes until 18 months. Incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) were calculated to express costs per unit improvement in pain burden and costs per QALY gained, for radiotherapy compared to sham-radiotherapy. Bootstrap replication was used to assess uncertainty surrounding the ratios and to construct cost-effectiveness acceptability curves for QALY gain. ResultsPrevious analysis showed a statistically significant improvement in pain- and QoL scores in favour of radiotherapy at 12 and 18 months. At these timepoints and excluding treatment costs, cumulative total costs were considerably lower in the radiotherapy group. The ICER until 12 months after treatment was 4987 euro per unit of pain burden reduction. The ICUR was 14249 euro per QALY gained. Most of the bootstrap replications were in the upper right quadrant, indicating that health gain can be achieved at higher costs. At increasing levels of willingness to pay for a gain in QALY, the probability of cost-utility gradually increased to approximately 85%. ConclusionsIn patients with symptomatic Ledderhose disease, radiotherapy, at a moderate threshold for willingness to pay, is cost-effective in terms of QoL gain.

Economic burden of nonoperative treatment of adult spinal deformity.

The purpose of this study was to investigate the cost utility of nonoperative treatment for adult spinal deformity (ASD). Nonoperatively and operatively treated patients who met database criteria for ASD and in whom complete radiographic and health-related quality of life data at baseline and at 2 years were available were included. A cost analysis was completed on the PearlDiver database assessing the average cost of nonoperative treatment prior to surgical intervention based on previously published treatments (NSAIDs, narcotics, muscle relaxants, epidural steroid injections, physical therapy, and chiropractor). Utility data were calculated using the Oswestry Disability Index (ODI) converted to SF-6D with published conversion methods. Quality-adjusted life years (QALYs) used a 3% discount rate to account for residual decline in life expectancy (78.7 years). Minor and major comorbidities and complications were assessed according to the CMS.gov manual's definitions. Successful nonoperative treatment was defined as a gain in the minimum clinically importance difference (MCID) in both ODI and Scoliosis Research Society (SRS)-pain scores, and failure was defined as a loss in MCID or conversion to operative treatment. Patients with baseline ODI ≤ 20 and continued ODI of ≤ 20 at 2 years were considered nonoperative successful maintenance. The average utilization of nonoperative treatment and cost were applied to the ASD cohort. A total of 824 patients were included (mean age 58.24 years, 81% female, mean body mass index 27.2 kg/m2). Overall, 75.5% of patients were in the operative and 24.5% were in the nonoperative cohort. At baseline patients in the operative cohort were significantly older, had a greater body mass index, increased pelvic tilt, and increased pelvic incidence-lumbar lordosis mismatch (all p < 0.05). With respect to deformity, patients in the operative group had higher rates of severe (i.e., ++) sagittal deformity according to SRS-Schwab modifiers for pelvic tilt, sagittal vertical axis, and pelvic incidence-lumbar lordosis mismatch (p < 0.05). At 2 years, patients in the operative cohort showed significantly increased rates of a gain in MCID for physical component summary of SF-36, ODI, and SRS-activity, SRS-pain, SRS-appearance, and SRS-mental scores. Cost analysis showed the average cost of nonoperative treatment 2 years prior to surgical intervention to be $2041. Overall, at 2 years patients in the nonoperative cohort had again in ODI of 0.36, did not show a gain in QALYs, and nonoperative treatment was determined to be cost-ineffective. However, a subset of patients in this cohort underwent successful maintenance treatment and had a decrease in ODI of 1.1 and a gain in utility of 0.006 at 2 years. If utility gained for this cohort was sustained to full life expectancy, patients' cost per QALY was $18,934 compared to a cost per QALY gained of $70,690.79 for posterior-only and $48,273.49 for combined approach in patients in the operative cohort. Patients with ASD undergoing operative treatment at baseline had greater sagittal deformity and greater improvement in health-related quality of life postoperatively compared to patients treated nonoperatively. Additionally, patients in the nonoperative cohort overall had an increase in ODI and did not show improvement in utility gained. Patients in the nonoperative cohort who had low disability and sagittal deformity underwent successful maintenance and cost-effective treatment.

Lipid-Lowering Strategies for Primary Prevention of Coronary Heart Disease in the UK: A Cost-Effectiveness Analysis.

We aimed to assess the cost effectiveness of four different lipid-lowering strategies for primary prevention of coronary heart disease initiated at ages 30, 40, 50, and 60 years from the UK National Health Service perspective. We developed a microsimulation model comparing the initiation of a lipid-lowering strategy to current standard of care (control). We included 458,692 participants of the UK Biobank study. The four lipid-lowering strategies were: (1) low/moderate-intensity statins; (2) high-intensity statins; (3) low/moderate-intensity statins and ezetimibe; and (4) inclisiran. The main outcome was the incremental cost-effectiveness ratio for each lipid-lowering strategy compared to the control, with 3.5% annual discounting using 2021 GBP (£); incremental cost-effectiveness ratios were compared to the UK willingness-to-pay threshold of £20,000-£30,000 per quality-adjusted life-year. The most effective intervention, low/moderate-intensity statins and ezetimibe, was projected to lead to a gain in quality-adjusted life-years of 0.067 per person initiated at 30 and 0.026 at age 60 years. Initiating therapy at 40 years of age was the most cost effective for all lipid-lowering strategies, with incremental cost-effectiveness ratios of £2553 (95% uncertainty interval: 1270, 3969), £4511 (3138, 6401), £11,107 (8655, 14,508), and £1,406,296 (1,121,775, 1,796,281) per quality-adjusted life-year gained for strategies 1-4, respectively. Incremental cost-effectiveness ratios were lower for male individuals (vs female individuals) and for people with higher (vs lower) low-density lipoprotein-cholesterol. For example, low/moderate-intensity statin use initiated from age 40 years had an incremental cost-effectiveness ratio of £5891 (3822, 9348), £2174 (772, 4216), and was dominant (i.e. cost saving; -2,760, 350) in female individuals with a low-density lipoprotein-cholesterol of ≥ 3.0, ≥ 4.0 and ≥ 5.0 mmol/L, respectively. Inclisiran was not cost effective in any sub-group at its current price. Low-density lipoprotein-cholesterol lowering from early ages is a more cost-effective strategy than late intervention and cost effectiveness increased with the increasing lifetime risk of coronary heart disease.