Lower Quality-adjusted Life-years Research Articles

Cost-effectiveness of proton beam therapy vs. conventional radiotherapy for patients with brain tumors in Sweden: results from a non-randomized prospective multicenter study

BackgroundThis study assessed the cost-effectiveness of proton beam therapy (PBT) compared to conventional radiotherapy (CRT) for treating patients with brain tumors in Sweden.MethodsData from a longitudinal non-randomized study performed between 2015 and 2020 was used, and included adult patients with brain tumors, followed during treatment and through a one-year follow-up. Clinical and demographic data were sourced from the longitudinal study and linked to Swedish national registers to get information on healthcare resource use. A cost-utility framework was used to evaluate the cost-effectiveness of PBT vs. CRT. Patients in PBT group (n = 310) were matched with patients in CRT group (n = 40) on relevant observables using propensity score matching with replacement. Costs were estimated from a healthcare perspective and included costs related to inpatient and specialized outpatient care, and prescribed medications. The health outcome was quality-adjusted life-years (QALYs), derived from the EORTC-QLQ-C30. Generalized linear models (GLM) and two-part models were used to estimate differences in costs and QALYs.ResultsPBT yielded higher total costs, 14,639 US$, than CRT, 13,308 US$, with a difference of 1,372 US$ (95% CI, -4,914–7,659) over a 58 weeks’ time horizon. Further, PBT resulted in non-significantly lower QALYs, 0.746 compared to CRT, 0.774, with a difference of -0.049 (95% CI, -0.195–0.097). The probability of PBT being cost-effective was < 30% at any willingness to pay.ConclusionsThese results suggest that PBT cannot be considered a cost-effective treatment for brain tumours, compared to CRT.Trial registrationNot applicable.

Informing optimal treatment (tx) sequences for platinum-eligible patients with locally advanced or metastatic urothelial carcinoma (la/mUC) in the United States: Results of a clinical simulation modeling exercise.

e13514 Background: Platinum-based chemotherapy (PBT) followed by avelumab (AVE) as first-line (1L) maintenance for patients with nonprogressive disease (PBT ± AVE) is the standard of care in la/mUC. This study compared the overall survival (OS) and cost-effectiveness of the tx sequence of PBT ± AVE followed by enfortumab vedotin (EV) or other therapies, including immuno-oncology (IO), in second line (2L; ie, PBT + 2L EV/IO or PBT + AVE + 2L EV/IO) vs 1L EV + pembrolizumab (PEM) for PBT-eligible patients (PBTp) from a US commercial payer perspective. Methods: A partitioned survival model compared outcomes with PBT ± AVE and EV + PEM among PBTp. Inputs included progression-free survival and OS, time to tx discontinuation, adverse event (AE) incidence, and health utilities and costs. Efficacy inputs were sourced and extrapolated from patient data in JAVELIN Bladder 100 (data cut: Jun 4, 2021), AVENANCE real-world study (1), and published data (EV-302 trial). Safety data were sourced from US prescribing information. Drug, subsequent tx, disease management, and AE management costs (2023 USD) were calculated. The optimal tx sequencing scenario assumed 80% of patients starting PBT were eligible to receive AVE (2023 Flatiron data cut; (2)), 70% of patients progressing on PBT ± AVE would start 2L, and 70% of those would be eligible for EV. Outcomes included life-years (LYs), quality-adjusted LYs (QALYs), costs, and incremental cost-effectiveness ratio (ICER). Scenarios and probabilistic sensitivity analyses (PSAs) were conducted to assess uncertainty. Results: LY, QALY, costs, and ICER modeling outcomes are shown in the Table. Use of EV in 2L after PBT ± AVE is associated with 0.76 lower QALYs, but given the large cost saving, it represents a cost-effective use of resources at the $150,000/QALY willingness to pay (WTP) threshold. PSAs showed that PBT ± AVE is likely to be more cost-effective over a broad and relevant WTP threshold range ($0-500,000/QALY). Results were sensitive to drug acquisition costs and eligibility to receive AVE and 2L EV. Conclusions: Based on this simulation exercise, the use of 2L EV after PBT ± AVE achieves considerable survival benefit at substantially lower costs vs 1L EV + PEM in PBTp with la/mUC. Given the shift toward value-based care in oncology, such analyses are becoming increasingly relevant to guide clinical decision-making. There is a need to assess tx alternatives that balance healthcare costs while optimizing outcomes in patients with la/mUC. 1. Barthélémy et al, J Clin Oncol 2024;42[suppl 4]: Abstract 561. 2. Moon et al, J Clin Oncol 2023;41[suppl 16]: Abstract 4567. [Table: see text]

Immune checkpoint inhibitors as the second-line treatment for advanced esophageal squamous cell carcinoma: a cost-effectiveness analysis based on network meta-analysis

BackgroundImmune checkpoint inhibitors (ICIs) have demonstrated superior clinical efficacy in prolonging overall survival (OS) as the second-line treatment for advanced or metastatic esophageal squamous cell carcinoma (ESCC), and were recommended by the guidelines. However, it remains uncertain which ICI is the most cost-effective. This study assessed the cost-effectiveness of ICIs as the second-line treatment for ESCC based on the perspective of the Chinese healthcare system.MethodsA network meta-analysis (NMA) was performed to obtain the Hazard ratios (HRs) for indirect comparisons. A three-state Markov model with a 10-year time horizon was conducted to assess the cost-effectiveness. The state transition probabilities were calculated with Kaplan-Meier (KM) curves data from clinical trial and HRs from the NMA. Utilities and costs were derived from local charges or previously published studies. Univariate and probabilistic sensitivity analyses (PSA) were performed to examine model robustness. The results were assessed based on the total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs).ResultsFive clinical trials (ATTRACTION-3, ESCORT, KEYNOTE-181, ORIENT-2, RATIONALE-302) with a total of 1797 patients were included in the NMA. The NMA showed that both camrelizumab and tislelizumab received relatively high rankings for progression-free survival (PFS) and OS. Compared with sintilimab, treatment with tislelizumab and camrelizumab gained 0.018 and 0.034 additional QALYs, resulting in incremental ICERs of $75,472.65/QALY and $175,681.9/QALY, respectively. Nivolumab and pembrolizumab produced lower QALYs and greater costs, suggesting that both were dominated in comparison to sintilimab. HRs and health state utilities were the most influential parameters in most univariate sensitivity analyses of paired comparisons. PSA results suggested that sintilimab had an 84.4% chance of being the most cost-effective treatment regimen at the WTP threshold of $38,223.34/QALY. In the scenario analysis, sintilimab would no longer be cost-effective, if the price of camrelizumab was assumed to decrease by 64.6% or the price of tislelizumab was assumed to decrease by 16.9%.Conclusions and relevanceAmong the five potential competing ICIs, sintilimab was likely to be the most cost-effective regimen as the second-line treatment for locally advanced or metastatic ESCC in China.

Cost-effectiveness analysis of routine computed tomography angiography (CTA) for lower extremity penetrating trauma

BackgroundRoutine evaluation with CTA for patients with isolated lower extremity penetrating trauma and normal ankle-brachial-indices (ABI) remains controversial. While prior literature has found normal ABI's (≥0.9) and a normal clinical examination to be adequate for safe discharge, there remains concern for missed injuries which could lead to delayed surgical intervention and unnecessary morbidity. Our hypothesis was that routine CTA after isolated lower extremity penetrating trauma with normal ABIs and clinical examination is not cost-effective. MethodsWe performed a decision-analytic model to evaluate the cost-effectiveness of obtaining a CTA routinely compared to clinical observation and ABI evaluation in hemodynamically normal patients with isolated penetrating lower extremity trauma. Our base case was a patient that sustained penetrating lower extremity trauma with normal ABIs that received a CTA in the trauma bay. Costs, probability, and Quality-Adjusted Life Years (QALYs) were generated from published literature. ResultsClinical evaluation only (no CTA) was cost-effective with a cost of $2056.13 and 0.98 QALYs gained compared to routine CTA which had increased costs of $7449.91 and lower QALYs 0.92. Using one-way sensitivity analysis, routine CTA does not become the cost-effective strategy until the cost of a missed injury reaches $210,075.83. ConclusionsPatients with isolated, penetrating lower extremity trauma with normal ABIs and clinical examination do not warrant routine CTA as there is no benefit with increased costs.

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

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

Two-year follow-up of a clustered randomised controlled trial of a multicomponent general practice intervention for people at risk of poor health outcomes

BackgroundThis study was a two-year follow-up evaluation of health service use and the cost-effectiveness of a multicomponent general practice intervention targeted at people at high risk of poor health outcomes.MethodsA two-year follow-up study of a clustered randomised controlled trial was conducted in South Australia during 2018–19, recruiting 1044 patients from three cohorts: children; adults (aged 18–64 years with two or more chronic diseases); and older adults (aged ≥ 65 years). Intervention group practices (n = 10) provided a multicomponent general practice intervention for 12 months. The intervention comprised patient enrolment to a preferred general practitioner (GP), access to longer GP appointments and timely general practice follow-up after episodes of hospital care. Health service outcomes included hospital use, specialist services and pharmaceuticals. The economic evaluation was based on quality-adjusted life years (QALYs) calculated from EuroQoL 5 dimensions, 5 level utility scores and used an A$50,000 per QALY gained threshold for determining cost-effectiveness.ResultsOver the two years, there were no statistically significant intervention effects for health service use. In the total sample, the mean total cost per patient was greater for the intervention than control group, but the number of QALYs gained in the intervention group was higher. The estimated incremental cost-effectiveness ratio (ICER) was A$18,211 per QALY gained, which is lower than the A$50,000 per QALY gained threshold used in Australia. However, the intervention's cost-effectiveness was shown to differ by cohort. For the adult cohort, the intervention was associated with higher costs and lower QALYs gained (vs the total cohort) and was not cost-effective. For the older adults cohort, the intervention was associated with lower costs (A$540 per patient), due primarily to lower hospital costs, and was more effective than usual care.ConclusionsThe positive cost-effectiveness results from the 24-month follow-up warrant replication in a study appropriately powered for outcomes such as hospital use, with an intervention period of at least two years, and targeted to older people at high risk of poor health outcomes.

Cost-effectiveness analysis of expectant vs active management for treatment of persistent pregnancies of unknown location

BackgroundPersistent pregnancies of unknown location (PUL) are defined by abnormally trending serum human chorionic gonadotropin with nondiagnostic ultrasound. There is no consensus on optimal management. ObjectiveThis study was designed to assess the cost-effectiveness between three primary management strategies for persistent pregnancies of unknown location: (1) expectant management, (2) empirical two-dose methotrexate, (3) uterine evacuation followed by methotrexate, if indicated. Study DesignWe conducted a prospective economic evaluation performed concurrently with the Expectant versus Active Management for Treatment of Persistent Pregnancies of Unknown Location (ACT or NOT) multicenter randomized trial conducted from July 2014 to June 2019. Participants were randomized 1:1:1 to expectant management, two-dose methotrexate, or uterine evacuation. The analysis was from the healthcare sector perspective with a 6-week time horizon following randomization. Costs were expressed in 2018 U.S. dollars. Effectiveness was measured in quality-adjusted life-years (QALYs) and the rate of salpingectomy. Incremental cost-effectiveness ratios (ICERs) and cost-effectiveness acceptability curves were generated. Sensitivity analyses were performed to assess the robustness of the analysis. ResultsMethotrexate had the lowest mean cost, $875, followed by expectant management $1085, and uterine evacuation $1902 (p=0.001). Expectant management had the highest mean QALY (0.1043) followed by methotrexate (0.1031) and uterine evacuation (0.0992) (p=0.0001). Salpingectomy rate was higher for expectant management compared to methotrexate (9.4% vs 1.2%; p=0.02) and expectant management compared to uterine evacuation (9.4% vs 8.1%; p=0.04). Uterine evacuation, with highest costs and lowest QALYs, was dominated by both expectant management and methotrexate. In the base case analysis, expectant management was not cost-effective compared to methotrexate at a willingness-to-pay of $150,000/QALY given an ICER of $175,083/QALY gained (95% CI, -$1,666,825-2,676,375). Threshold analysis demonstrated methotrexate administration would have to cost $214 (an increase of $16 or 8%) to favor expectant management. Expectant management would also be favorable in lower risk patient populations with rates of laparoscopic surgical management for ectopic pregnancy not exceeding 4% of pregnancies of unknown location. Based on the cost-effectiveness acceptability curves, the probability expectant management was cost-effective compared to methotrexate at a willingness-to-pay of $150,000/QALY gained was 50%. Results were dependent on the cost of surgical intervention, and the expected rate of methotrexate failure. ConclusionManagement of pregnancies of unknown location with a two-dose methotrexate protocol may be cost-effective compared to expectant management and uterine evacuation. While uterine evacuation was dominated, expectant management versus methotrexate results were sensitive to modest changes in treatment costs of both methotrexate and surgical management.

Cost-effectiveness of latent tuberculosis infection testing and treatment with 6-week regimen among key population in rural communities in China: a decision analysis study.

Several model studies suggested the implementation of latent tuberculosis infection (LTBI) testing and treatment could greatly reduce the incidence of tuberculosis (TB) and achieve the 2035 target of the "End TB" Strategy in China. The present study aimed to evaluate the cost-effectiveness of LTBI testing and TB preventive treatment among key population (≥ 50 years old) susceptible to TB at community level in China. A Markov model was developed to investigate the cost-effectiveness of LTBI testing using interferon gamma release assay (IGRA) and subsequent treatment with 6-month daily isoniazid regimen (6H) (as a standard regimen for comparison) or 6-week twice-weekly rifapentine and isoniazid regimen (6-week H2P2) in a cohort of 10,000 adults with an average initial age of 50 years. In the base-case analysis, LTBI testing and treatment with 6H was dominated (i.e., more expensive with a lower quality-adjusted life year (QALY)) by LTBI testing and treatment with 6-week H2P2. LTBI testing and treatment with 6-week H2P2 was more effective than no intervention at a cost of $20,943.81 per QALY gained, which was below the willingness-to-pay (WTP) threshold of $24,211.84 per QALY gained in China. The one-way sensitivity analysis showed the change of LTBI prevalence was the parameter that most influenced the results of the incremental cost-effectiveness ratios (ICERs). As estimated by a Markov model, LTBI testing and treatment with 6-week H2P2 was cost-saving compared with LTBI testing and treatment with 6H, and it was considered to be a cost-effective option for TB control in rural China.

Exploring the Cost Effectiveness of a Whole-Genome Sequencing-Based Biomarker for Treatment Selection in Patients with Advanced Lung Cancer Ineligible for Targeted Therapy.

We aimed to perform an early cost-effectiveness analysis of using a whole-genome sequencing-based tumor mutation burden (WGS-TMB), instead of programmed death-ligand 1 (PD-L1), for immunotherapy treatment selection in patients with non-squamous advanced/metastatic non-small cell lung cancer ineligible for targeted therapy, from a Dutch healthcare perspective. A decision-model simulating individual patients with metastatic non-small cell lung cancer was used to evaluate diagnostic strategies to select first-line immunotherapy only or the immunotherapy plus chemotherapy combination. Treatment was selected using PD-L1 [A, current practice], WGS-TMB [B], and both PD-L1 and WGS-TMB [C]. Strategies D, E, and F take into account a patient's disease burden, in addition to PD-L1, WGS-TMB, andboth PD-L1 andWGS-TMB, respectively. Disease burden was defined as a fast-growing tumor, a high number of metastases, and/or weight loss. A threshold of 10 mutations per mega-base was used to classify patients into TMB-high and TMB-low groups. Outcomes were discounted quality-adjusted life-years (QALYs) and healthcare costs measured from the start of first-line treatment to death. Healthcare costs includes drug acquisition, follow-up costs, and molecular diagnostic tests (i.e., standard diagnostic techniques and/or WGS for strategies involving TMB). Results were reported using the net monetary benefit at a willingness-to-pay threshold of €80,000/QALY. Additional scenario and threshold analyses were performed. Strategy B had the lowest QALYs (1.84) and lowest healthcare costs (€120,800). The highest QALYs and healthcare costs were 2.00 and €140,400 in strategy F. In the base-case analysis, strategy A was cost effective with the highest net monetary benefit (€27,300), followed by strategy B (€26,700). Strategy B was cost effective when the cost of WGS testing was decreased by at least 24% or when immunotherapy results in an additional 0.5 year of life gained or more for TMB high compared with TMB low. Strategies C and F, which combined TMB and PD-L1 had the highest net monetary benefit (≥ €76,900) when the cost of WGS testing, immunotherapy, and chemotherapy acquisition were simultaneously reduced by at least 47%, 39%, and 43%, respectively. Furthermore, strategy C resulted in the highest net monetary benefit (≥ €39,900) in a scenario where patients with both PD-L1 low and TMB low were treated with chemotherapy instead of immunotherapy plus chemotherapy. The use of WGS-TMB is not cost effective compared to PD-L1 for immunotherapy treatment selection in non-squamous metastatic non-small cell lung cancer in the Netherlands. WGS-TMB could become cost effective provided there is a reduction in the cost of WGS testing or there is an increase in the predictive value of WGS-TMB for immunotherapy effectiveness. Alternatively, a combination strategy of PD-L1 testing with WGS-TMB would be cost effective if used to support the choice to withhold immunotherapy in patients with a low expected benefit of immunotherapy.

Impact of Educational Background on Preoperative Disease Severity and Postoperative Outcomes Among Patients With Cervical Spondylotic Myelopathy.

Retrospective review of a prospectively maintained database. Assess differences in preoperative status and postoperative outcomes among patients of different educational backgrounds undergoing surgical management of cervical spondylotic myelopathy (CSM). Patient education level (EL) has been suggested to correlate with health literacy, disease perception, socioeconomic status (SES), and access to health care. The CSM data set of the Quality Outcomes Database (QOD) was queried for patients undergoing surgical management of CSM. EL was grouped as high school or below, graduate-level, and postgraduate level. The association of EL with baseline disease severity (per patient-reported outcome measures), symptoms >3 or ≤3 months, and 24-month patient-reported outcome measures were evaluated. Among 1141 patients with CSM, 509 (44.6%) had an EL of high school or below, 471 (41.3%) had a graduate degree, and 161 (14.1%) had obtained postgraduate education. Lower EL was statistically significantly associated with symptom duration of >3 months (odds ratio=1.68), higher arm pain numeric rating scale (NRS) (coefficient=0.5), and higher neck pain NRS (coefficient=0.79). Patients with postgraduate education had statistically significantly lower Neck Disability Index (NDI) scores (coefficient=-7.17), lower arm pain scores (coefficient=-1), and higher quality-adjusted life-years (QALY) scores (coefficient=0.06). Twenty-four months after surgery, patients of lower EL had higher NDI scores, higher pain NRS scores, and lower QALY scores ( P <0.05 in all analyses). Among patients undergoing surgical management for CSM, those reporting a lower educational level tended to present with longer symptom duration, more disease-inflicted disability and pain, and lower QALY scores. As such, patients of a lower EL are a potentially vulnerable subpopulation, and their health literacy and access to care should be prioritized.

Estimating the value of repositioning timing to streamline pressure injury prevention efforts in nursing homes: A cost-effectiveness analysis of the 'TEAM-UP' clinical trial.

Pressure injury (PrI) prevention guidelines recommend 2-h repositioning intervals in healthcare settings, requiring significant nursing time investment. We analysed the cost-effectiveness of PrI prevention protocols with 2-, 3- and 4-h repositioning intervals in US nursing homes according to 'Turn Everyone and Move for Ulcer Prevention' (TEAM-UP) randomized controlled trial findings. Markov modelling compared 2-, 3- and 4-h repositioning intervals, controlling for other practice guidelines, to prevent PrIs in nursing home residents from a US health sector perspective over one year using TEAM-UP trial data for model structure, sampling and parameterization. Costs, captured in 2020 US dollars, and quality-adjusted life years (QALYs) were used to derive an incremental cost-effectiveness ratio and net monetary benefit (NMB) at $50 000/QALY-$150 000/QALY cost-effectiveness thresholds. Sensitivity analyses tested model uncertainty. Repositioning intervals between 3 and 4 h were cost-effective based on reduced costs at slightly lower QALYs than 2 h at a $50 000/QALY threshold, and the NMB of 4-h repositioning was also more efficient than at 3 h ($9610). Repositioning labour cost and prevention routines were among the most sensitive parameters. Sensitivity analyses demonstrated that 3- and 4-h intervals were cost-effective in over 65% of simulations at any cost-effectiveness threshold. Repositioning intervals of 3 to 4 h have potential to reduce nursing time costs without significant decrements in clinical benefits to nursing home residents. Clinical guidelines for PrI prevention should be updated to reflect TEAM-UP clinical and economic findings. Facilities can use cost-savings recuperated from nursing time to deploy to other patient safety priorities without seriously jeopardizing PrI safety.

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.

Cost-effectiveness of budesonide-formoterol vs inhaled epinephrine in US adults with mild asthma

BackgroundThe management of mild asthma has lacked an over-the-counter (OTC) option aside from inhaled epinephrine, which is available in the United States. However, inhaled epinephrine use without an inhaled corticosteroid may increase the risk of asthma death. ObjectiveTo compare the cost-effectiveness of OTC as-needed budesonide-formoterol as a plausible alternative to inhaled epinephrine. MethodsWe developed a probabilistic Markov model to compare OTC as-needed budesonide-formoterol inhaler use vs inhaled epinephrine use in adults with mild asthma from a US societal perspective over a lifetime horizon, with a 3% annual discount rate (2022 US dollars). Inputs were derived from the SYmbicort Given as-needed in Mild Asthma (SYGMA) trials, published literature, and commercial costs. Outcomes were quality-adjusted life-years (QALY), costs, incremental net monetary benefit (INMB), severe asthma exacerbations, well-controlled asthma days, and asthma-related deaths. Microsimulation was used to evaluate underinsured Americans living with mild asthma (n = 5,250,000). ResultsInhaled epinephrine was dominated (with lower QALYs gains at a higher cost) by both as-needed budesonide-formoterol (INMB, $15,541 at a willingness-to-pay of $100,000 per QALY) and the no-OTC inhaler option (INMB, $1023). Adults using as-needed budesonide-formoterol had 145 more well-controlled asthma days, 2.79 fewer severe exacerbations, and an absolute risk reduction of 0.23% for asthma-related death compared with inhaled epinephrine over a patient lifetime. As-needed budesonide-formoterol remained dominant in all sensitivity and scenario analyses, with a 100% probability of being cost-effective compared with inhaled epinephrine in probabilistic sensitivity analysis. ConclusionIf made available, OTC as-needed budesonide-formoterol for treating mild asthma in underinsured adults without HCP management improves asthma outcomes, prevents fatalities, and is cost-saving.

Cost-effectiveness of alternative first- and second-line treatments for benign prostatic hyperplasia in Singapore

BackgroundMinimally invasive surgical therapies, such as water vapor thermal therapy (WVTT) and prostatic urethral lift (PUL), are typically second-line options for patients in whom medical management (MM) failed but who are unwilling or unsuitable to undergo invasive transurethral resection of the prostate (TURP). However, the incremental cost-effectiveness of WVTT or PUL as first- or second-line therapy is unknown. We evaluated the incremental cost-effectiveness of alternative first- and second-line treatments for patients with moderate-to-severe benign prostatic hyperplasia (BPH) in Singapore to help policymakers make subsidy decisions based on value for money.MethodsWe considered six stepped-up treatment strategies, beginning with MM, WVTT, PUL or TURP. In each strategy, patients requiring retreatment advance to a more invasive treatment until TURP, which may be undergone twice. A Markov cohort model was used to simulate transitions between BPH severity states and retreatment, accruing costs and quality-adjusted life-years (QALYs) over a lifetime horizon.ResultsIn moderate patients, strategies beginning with MM had similar cost and effectiveness, and first-line WVTT was incrementally cost-effective to first-line MM (33,307 SGD/QALY). First-line TURP was not incrementally cost-effective to first-line WVTT (159,361 SGD/QALY). For severe patients, WVTT was incrementally cost-effective to MM as a first-line treatment (30,133 SGD/QALY) and to TURP as a second-line treatment following MM (6877 SGD/QALY). TURP was incrementally cost-effective to WVTT as a first-line treatment (48,209 SGD/QALY) in severe patients only. All pathways involving PUL were dominated (higher costs and lower QALYs).ConclusionBased on the common willingness-to-pay threshold of SGD 50,000/QALY, this study demonstrates the cost-effectiveness of WVTT over MM as first-line treatment for patients with moderate or severe BPH, suggesting it represents good value for money and should be considered for subsidy. PUL is not cost-effective as a first- nor second-line treatment. For patients with severe BPH, TURP as first-line is also cost-effective.

FRI682 Cost Effectiveness Of Four Treatment Approaches For US Women And Men At Very High Risk Of Osteoporotic Fractures

Abstract Disclosure: M. Hiligsmann: Grant Recipient; Self; Radius Health, Inc. S. Silverman: Consulting Fee; Self; Amgen Inc, Radius Health, Inc. Grant Recipient; Self; Amgen Inc, Radius Health, Inc. A.J. Singer: Consulting Fee; Self; Agnovos, Amgen Inc, Radius Health, Inc, UCB. Speaker; Self; Amgen Inc, Radius Health, Inc. L. Pearman: Employee; Self; Radius Health, Inc. Stock Owner; Self; Radius Health, Inc. Y. Wang: Employee; Self; Radius Health, Inc. Stock Owner; Self; Radius Health, Inc. J.N. Caminis: Employee; Self; Radius Health, Inc. Stock Owner; Self; Radius Health, Inc. J. Reginster: Advisory Board Member; Self; Pierre Fabre. Consulting Fee; Self; IBSA-Genevrier, Mylan, Teva Pharmaceutical Industries Ltd. Speaker; Self; IBSA-Genevrier, Mylan, CNIEL, Dairy Research Council, Teva Pharmaceutical Industries Ltd. Background: Cost-effectiveness analyses are becoming increasingly important for efficiency in healthcare resource allocation. This study aims to assess the cost-effectiveness of four common treatment approaches of US men and women at very high risk of fracture: bisphosphonate (alendronate [ALN]) monotherapy, sequential treatment with an anabolic first (either abaloparatide [ABL] or teriparatide [TPTD]) followed by a bisphosphonate, and no treatment, as many patients at very high fracture risk do not receive an osteoporosis medication. Methods: A microsimulation-based Markov model was used to assess the cost-effectiveness of four treatment approaches in US women and men aged 50 to 80 years who had a recent fracture and had densitometry-confirmed osteoporosis (bone mineral density T-score ≤−2.5): ALN monotherapy, sequential ABL/ALN, sequential unbranded TPTD/ALN, and no treatment. Analyses were conducted from the US healthcare decision-maker perspective using $2022 costs, and health outcomes were expressed in quality-adjusted life years (QALYs). Deterministic and probabilistic sensitivity analyses were performed to characterize uncertainty. Results: In both sexes and over the full age range, no treatment and sequential unbranded TPTD/ALN were dominated (ie, they had lower QALYs for more costs than a comparator treatment), and should be excluded. Sequential ABL/ALN was associated with more QALYs and more costs than ALN monotherapy. At the US threshold of $150,000 per QALY gained, sequential ABL/ALN was cost-effective compared to ALN monotherapy in men aged ≥50 years and in women aged ≥65 years. Sensitivity analyses suggested that sequential ABL/ALN was also cost-effective in women with a recent hip or vertebral fracture aged ≥55 years. Conslusion: In both US men (aged ≥50 years) and women (aged ≥65 years) at very high risk of fracture, sequential ABL/ALN is the most cost-effective intervention among the four treatment approaches. Sequential unbranded TPTD/ALN and no treatment were dominated, and are thus not efficient treatment approaches. Treating US men and women at very high fracture risk with sequential ABL/ALN leads to an efficient resource allocation. Presentation: Friday, June 16, 2023

A Cost-Effectiveness Analysis of Azithromycin for the Prevention of Acute Exacerbations of Chronic Obstructive Pulmonary Disease.

Rationale: Daily oral azithromycin therapy can reduce the risk of acute exacerbations of chronic obstructive pulmonary disease (COPD). However, given its adverse events and additional costs, it is not known whether adding long-term azithromycin as an adjunct therapy to inhaled pharmacotherapy is cost effective. Objectives: The objective of this study was to evaluate the cost-effectiveness of add-on azithromycin therapy in COPD as recommended by contemporary COPD management guidelines. Methods: We extended a previously validated Canadian COPD policy model to include azithromycin-related inputs and outcomes. The cost-effectiveness of azithromycin was evaluated over a 20-year time horizon in patients who continue to exacerbate despite receiving maximal inhaled therapies. The benefit of azithromycin was modeled as a reduction in exacerbation rates. Adverse events included cardiovascular death, hearing loss, gastrointestinal symptoms, and antimicrobial resistance. The incremental cost-effectiveness ratio (ICER) was calculated with costs in 2020 Canadian dollars ($) and quality-adjusted life-years (QALYs) discounted at 1.5% per year. The analysis was stratified among patient subgroups based on exacerbation histories. Results: In patients with a positive exacerbation history (one or more events in the previous 12 mo), azithromycin was associated with $49,732 costs, 7.65 QALYs, and 10.95 exacerbations per patient over 20 years. The corresponding values were $48,436, 7.62, and 11.86 for the reference group, resulting in an ICER of $43,200 per QALY gained. In patients defined as frequent exacerbators (two or more moderate or one or more severe events in the past 12 mo), the ICER was reduced to $8,862 per QALY gained. In patients with no history of exacerbation, azithromycin had lower QALYs and higher costs than the reference group. Conclusions: Add-on azithromycin is cost effective in patients with a recent history of exacerbations at commonly accepted willingness-to-pay thresholds of $50,000-$100,000/QALY. Guidelines should consider recommending add-on azithromycin for patients who had at least one moderate or severe exacerbation in the past year, albeit more information about treatment efficacy would strengthen this recommendation.

Cost-utility analysis of total knee arthroplasty using 10-year data from a randomised controlled trial: Implant design influences quality-adjusted life year gain

BackgroundThe aim of this study was to perform a cost-utility analysis for total knee arthroplasty (TKA) over a 10-year follow up period. MethodsTwo-hundred and twelve patients were randomised to receive either a Kinemax or a Triathlon TKA. Patients were assessed pre-operatively, and at 6 months, and 1, 3, 7 and 10 years postoperatively. The costs of the primary and revision surgery were accounted for. One-year quality-adjusted life year (QALY) gain was used to estimate 10-year gains using the established annual health gain discounts. ResultsForty-eight patients died and eight were revised during the follow up period. Overall QALY gain per patient over the 10-year period was 2.594 and the cost per patient was £6559, which resulted in a cost per QALY of £2761 at 10 years. The Triathlon group had a significantly greater QALY gain compared with the Kinemax (mean difference (MD) 0.53, 95% CI 0.03–1.03, P = 0.02), which resulted in a cost per QALY for the Triathlon group of £2521 compared with £3107 for the Kinemax group at 10 years. The 5% annual discount resulted in a significantly lower QALY gain (MD 0.135, 95% CI 0.201–0.354, P = 0.002), whereas the 3.5% annual discount resulted in non-significant difference in QALY gain compared with the actual gain (MD 0.021, 95% CI −0.084 to 0.077, P = 0.292). ConclusionsTKA was a cost-effective intervention, and the Triathlon was associated with a greater cost effectiveness at 10 years. The 3.5% annual discounts for QALY gain would seem to be the most accurate, with an underestimation being observed with the 5% discount.

An Alternative Measure of Health for Value Assessment: The Equal Value Life-Year.

The quality-adjusted life-year (QALY) is an international standard in cost-effectiveness analysis. A known concern arises from the relatively lower QALY gains attributed to treatments that extend the life of individuals with chronic disability. We analyze here the advantages and disadvantages of the equal value life-year (evLY) as an alternative or a complementary measure to the QALY, and share learned experiences from using this measure in health technology assessments. We present the conceptual rationale for the evLY, describe how it is estimated, and assess the differences in results between analyses based on the evLY and the QALY. We share a how-to guide in estimating the evLY using a downloadable tool and summarize our empirical experience using this measure. Incremental evLYs are feasible and address concerns regarding the risk for a cost-effectiveness analysis to undervalue treatments for people with chronic disabilities. Based on our set of analyses using the evLY, a threshold of $84,000 per evLY gained would be needed to maintain alignment with a threshold of $100,000 per added QALY. The evLY is a measure of health gain that can be used as an alternative or a complement to the QALY to address concerns related to undervaluing treatments that extend the life of individuals with serious illness or chronic disability. We recommend that it be reported within all cost-effectiveness analyses but may have special relevance in the current political environment in the USA, where use of the QALY is often challenged or prohibited.

The impact of cognitive impairment on disease burden in Chinese patients with multiple sclerosis: A model simulation study

Cognitive impairment (CI) is prevalent in Chinese patients with relapsing-remitting multiple sclerosis (RRMS). A decision analytic model was constructed to simulate Chinese patients with newly diagnosed RRMS and their matched control cohort without MS for the risks of developing CI, developing secondary progressive MS (SPMS), and mortality. Both English and Chinese bibliographic databases were searched for evidence to estimate model inputs. Base case analysis and sensitivity analysis were conducted for the point estimations and uncertainty of the measured burden outcomes. Model simulations estimated that the lifetime cumulative risk of CI in newly diagnosed RRMS patients was 85.2%. Relative to the matched control cohort, newly diagnosed RRMS patients were associated with a lower life expectancy (33.2 years vs. 41.7 years, difference: -8.5 years), lower quality-adjusted life years (QALY) (18.4 QALY vs. 38.4 QALY, difference: -19.9 QALY), and higher lifetime medical costs (¥613,883 vs. ¥202,726, difference: ¥411,157) and indirect costs (¥1,099,021 vs. ¥94,612, difference: ¥1,004,410). Patients who developed CI accounted for at least half of the measured burden. The disease burden outcomes were mainly driven by the risk of developing CI, progression risk from RRMS to SPMS, hazard ratios of mortality associated with CI relative to no CI, utility of patients with RRMS, annual relapse risk, and annual costs of personal care. Most Chinese patients with newly diagnosed RRMS are likely to develop CI in their lifetime, and such patients that develop CI could significantly contribute to the disease burden of RRMS.

Cost-effectiveness of testing for Mycoplasma genitalium among men who have sex with men in Australia

ObjectivesMycoplasma genitalium (MG) disproportionately affects men who have sex with men (MSM). We determined the cost-effectiveness of different testing strategies for MG in MSM, taking a healthcare provider perspective.MethodsWe used...