Probabilistic Sensitivity Analysis Research Articles

Modeling the population health impact of incorporating a multi-cancer early detection (MCED) test to existing cancer screening among immunocompromised individuals

Objective To assess the screening efficiency of an multi-cancer early detection (MCED) test added to standard of care (SoC) screening, compared to SoC screening alone, among immunocompromised individuals, and to estimate the diagnostic workup costs associated with positive screening results. Methods We estimated the potential impact of cancer screening among immunocompromised individuals aged 50–79 years within the University of Utah Health system who underwent a stem cell/solid organ transplant or were diagnosed with a primary or secondary immunodeficiency disorder between January 2000 and February 2018. We derived cancer incidence rates from the Huntsman Cancer Institute Tumor Registry, and screening performance of SoC screening and an MCED test from published literature. Outcomes of screening efficiency included the true-positive to false-positive (TP:FP) ratio, diagnostic yield (DY), and cancer detection rate (CDR) for SoC screening alone and an incremental MCED test. Scenario and probabilistic sensitivity analyses were conducted. Results Among 4932 immunocompromised individuals aged 50–79 years, we estimated that 2595 tests would be done under SoC screening and assumed that all individuals received an additional MCED test. Adding an MCED test to SoC screening substantially improved screening efficiency (TP:FP = 1:1, DY = 5.15/1000 tests, CDR = 42.0%), compared to SoC screening alone (TP:FP = 1:99, DY = 1.23/1000 tests, CDR = 5.3%), assuming an MCED test with 100% uptake. Our findings were also robust to parameter uncertainty. Conclusion Adding an MCED test to complement existing screening may be a highly efficient strategy to increase the detection of cancers among immunocompromised individuals. These results could help to improve cancer prevention and detection efforts among individuals with multiple cancer risk factors.

Cost-effectiveness analysis of budesonide/formoterol SMART therapy versus salmeterol/fluticasone plus as-needed SABA among patients ≥12 years with moderate asthma from the Chinese societal perspective

Objectives To evaluate the cost-effectiveness of budesonide/formoterol reliever and maintenance therapy compared with salmeterol/fluticasone plus salbutamol as reliever therapy for asthma patients ≥12 years from the societal perspective in China. Methods A Markov model was developed with three health states (non-exacerbation, exacerbation, and death) with a lifetime horizon. The exacerbation rates were obtained from a prospective cohort study conducted in Chinese asthma patients. Healthcare resources utilization data were estimated based on current clinical asthma management guidelines. Asthma-related mortality, cost input and utility values were derived from public database and literature. Model robustness was assessed with one-way sensitivity and probabilistic sensitivity analyses. Results Compared with salmeterol/fluticasone plus salbutamol, budesonide/formoterol reliever and maintenance therapy led to fewer exacerbation events (13.6 vs. 15.9) and 0.0077 quality-adjusted life years (QALY) gain at an additional cost of ¥196.38 over lifetime. The base case incremental cost-effectiveness ratio (ICER) was ¥25,409.98 per QALY gained. The variables that had most impact on the model output included drug costs and medication adherence. At a willingness-to-pay threshold of ¥257,094/QALY (3 times of gross domestic product per capita in China in 2022), the probability of budesonide/formoterol maintenance and reliever therapy being cost-effective versus salmeterol/fluticasone plus as-needed salbutamol was 83.00%. Conclusion From the societal perspective, budesonide/formoterol reliever and maintenance therapy is likely to be a cost-effective option compared with salmeterol/fluticasone plus as-needed salbutamol for Chinese asthma patients ≥12 years.

Methods to Quantify the Importance of Parameters for Model Updating and Distributional Adaptation.

Decision models are time-consuming to develop; therefore, adapting previously developed models for new purposes may be advantageous. We provide methods to prioritize efforts to 1) update parameter values in existing models and 2) adapt existing models for distributional cost-effectiveness analysis (DCEA). Methods exist to assess the influence of different input parameters on the results of a decision models, including value of information (VOI) and 1-way sensitivity analysis (OWSA). We apply 1) VOI to prioritize searches for additional information to update parameter values and 2) OWSA to prioritize searches for parameters that may vary by socioeconomic characteristics. We highlight the assumptions required and propose metrics that quantify the extent to which parameters in a model have been updated or adapted. We provide R code to quickly carry out the analysis given inputs from a probabilistic sensitivity analysis (PSA) and demonstrate our methods using an oncology case study. In our case study, updating 2 of 21 probabilistic model parameters addressed 71.5% of the total VOI and updating 3 addressed approximately 100% of the uncertainty. Our proposed approach suggests that these are the 3 parameters that should be prioritized. For model adaptation for DCEA, 46.3% of the total OWSA variation came from a single parameter, while the top 10 input parameters were found to account for more than 95% of the total variation, suggesting efforts should be aimed toward these. These methods offer a systematic approach to guide research efforts in updating models with new data or adapting models to undertake DCEA. The case study demonstrated only very small gains from updating more than 3 parameters or adapting more than 10 parameters. It can require considerable analyst time to search for evidence to update a model or to adapt a model to take account of equity concerns.In this article, we provide a quantitative method to prioritze parameters to 1) update existing models to reflect potential new evidence and 2) adapt existing models to estimate distributional outcomes.We define metrics that quantify the extent to which the parameters in a model have been updated or adapted.We provide R code that can quickly rank parameter importance and calculate quality metrics using only the results of a standard probabilistic sensitivity analysis.

Cost-effectiveness analysis of a digital diabetes-prevention programme versus an in-person diabetes-prevention programme in people with prediabetes in the United States.

To assess the cost-effectiveness of a digital diabetes prevention programme (d-DPP) compared with a diabetes prevention programme (DPP) for preventing type 2 diabetes (T2D) in individuals with prediabetes in the United States. A Markov cohort model was constructed, simulating a 10-year period starting at the age of 45 years, with a societal and healthcare sector perspective. The effectiveness of the d-DPP intervention was evaluated using a meta-analysis, with that of the DPP as the comparator. The initial cycle represented the treatment period, and transition probabilities for the post-treatment period were derived from a long-term lifestyle intervention meta-analysis. The onset of T2D complications was estimated using microsimulation. Quality-adjusted life years (QALYs) were calculated based on health utility measured by short form (SF)-12 scores, and a willingness-to-pay threshold of $100 000 per QALY gained was applied. The d-DPP intervention resulted in cost savings of $3,672 from a societal perspective and $2,990 from a healthcare sector perspectiveand a gain of 0.08 QALYs compared with the DPP. The dropout rate was identified as a significant factor influencing the results. Probabilistic sensitivity analysis showed that the d-DPP intervention was preferred in 85.8% in the societal perspective and 85.2% in the healthcare sector perspective. The d-DPP is a cost-effective alternative to in-person lifestyle interventions for preventing the development of T2D among individuals with prediabetes in the United States.

Cost-Utility Analysis of Bilateral Cochlear Implants for Children With Severe-to-Profound Sensorineural Hearing Loss in Taiwan.

Cochlear implants are an option for children with sensorineural hearing loss who do not benefit from hearing aids. Although bilateral cochlear implantation (CI) has been shown to enhance hearing performance and quality of life, its cost-effectiveness remains unclear. This study aimed to evaluate the cost-effectiveness of bilateral CI compared with bimodal hearing for children with sensorineural hearing loss in Taiwan from both the perspectives of patients and Taiwan's National Health Insurance Administration (TNHIA). A four-state Markov model was utilized in the study, including "use the first internal device," "use the second internal device," "use the third internal device," and "death." Health utility values were obtained from a local survey of health professionals and then adjusted by a scale to reflect both the negative impact of aging on hearing and the time needed to develop the full benefit of treatment in the earliest years of life. The cost data were derived from a caregiver survey, hospital databases, clinical experts, and the TNHIA. The incremental cost-effectiveness ratio (ICER) was calculated over the lifetime horizon and presented as cost per quality-adjusted life year (QALY) to evaluate the cost-effectiveness of simultaneous bilateral CI, sequential bilateral CI, and bimodal hearing. In addition, one-way sensitivity analyses and probabilistic sensitivity analyses were conducted to investigate the impact of uncertainty and the robustness of the model. The base-case analysis showed that children with bilateral CI gained more QALYs while incurring more costs when compared with those with bimodal hearing. From the TNHIA perspective, compared with bimodal hearing, the ICER of simultaneous bilateral CI was New Taiwan Dollars 232,662 per QALY whereas from the patient perspective, the ICER was New Taiwan Dollars 1,006,965 per QALY. Moreover, simultaneous bilateral CI dominated sequential bilateral CI from both perspectives. Compared with bimodal hearing, the ICER of sequential bilateral CI did not exceed twice the gross domestic product per capita in Taiwan from either perspective. One-way sensitivity analysis demonstrated that the utility gain of bilateral CI compared with bimodal hearing was the most impactful parameter from both perspectives. Probabilistic sensitivity analysis confirmed the robustness of the base-case analysis results. Our findings reveal that bilateral CI was cost-effective when using the threshold of one to three times the 2022 gross domestic product per capita in Taiwan from both the TNHIA and patient perspectives. Future research incorporating cost and effectiveness data from other dimensions is needed to help decision-makers assess the cost-effectiveness of bilateral CI more comprehensively.

Optimal malarial screening strategy in Australian blood donors: A cost-effectiveness analysis.

The risk of transfusion-transmitted malaria (TTM) infections is extremely low in Australia, and the cost-effectiveness of the current screening strategy has not been assessed. This study aims to conduct a cost-effectiveness analysis of different malaria screening strategies in blood donors as part of the risk-based decision-making framework. A decision tree model was developed to assess the cost-effectiveness of five alternative malaria screening strategies from a healthcare sector perspective. Screening strategies combining total or partial removal of malaria testing with different deferral periods were considered. The probabilities of developing severe and uncomplicated TTM were based on a literature review of cases in non-endemic areas since 2000. The health outcomes were quantified using disability-adjusted life years. The costs of non-returning donors due to deferral were also included. Deterministic and probabilistic sensitivity analyses were conducted to account for data uncertainty. The residual risks for all strategies were so low that the costs, mortality and morbidity associated with TTM are almost negligible. The overall costs were predominantly influenced by the costs of non-returning blood donors. As a result, removal of malaria testing and applying a 28-day deferral for at-risk donors were the least costly and most cost-effective of all the options considered. The current screening strategy for malaria in blood donors in Australia is not an efficient use of healthcare resources. Partial or total removal of malaria testing would bring significant cost savings without significantly compromising blood safety.

Cost-effectiveness of Pembrolizumab in Combination with Chemotherapy as Neoadjuvant Treatment and Continued as a Single Agent Adjuvant Treatment for High-Risk Early-Stage Triple-Negative Breast Cancer in Hong Kong.

The phaseIII randomized KEYNOTE-522 trial demonstrated that pembrolizumab in combination with chemotherapy as neoadjuvant treatment followed by adjuvant pembrolizumab (pembrolizumab + chemotherapy) provided significant improvements in event-free survival (EFS) and overall survival (OS) for patients with high-risk early-stage triple-negative breast cancer (eTNBC). The objective was to assess the cost-effectiveness of pembrolizumab + chemotherapy compared to neoadjuvant chemotherapy alone (chemotherapy) in patients with high-risk eTNBC from a Hong Kong third-party payer perspective. A multistate transition model with four health states (event-free), locoregional recurrence, distant metastases, and death) was developed to assess the lifetime medical costs and health outcomes (3% annual discount), along with incremental cost-effectiveness ratios (ICERs) using efficacy and safety data from the KEYNOTE-522 trial. The health state utilities were derived from KEYNOTE-522 Euro-QoL-five-dimension five-level questionnaire (EQ-5D-5L) data. Costs were expressed in 2022 Hong Kong dollars (HKD).Scenario and sensitivity analyses were performed to assess the robustness of results. Over a 32-year time horizon, base case results showed that pembrolizumab + chemotherapy was associated with a 3.42year longer EFS and expected gains of 3.05 lifeyears (LYs) and 2.45 quality-adjusted lifeyears (QALYs) compared to chemotherapy. The resultant ICERs were HKD135,200 per QALY gained and HKD108,463 per LY gained, which were lower than the World Health Organization (WHO) cost-effectiveness threshold of three times gross domestic product (GDP) per capita for Hong Kong of HKD1,171,308 per QALY. The one-way sensitivity analyses (OWSA) and probabilistic sensitivity analysis (PSA) showed the results were robust across various inputs and alternative scenarios. On the basis of the analysis conducted for a 56-year-old cohort with high-risk eTNBC and assumptions in the model, pembrolizumab + chemotherapy represents a cost-effective proposition (as the ICER is approximately 35% of the GDP per capita in Hong Kong) for patients with high-risk eTNBC in Hong Kong.

Cost-Effectiveness Analysis of Hepatic Arterial Chemotherapy for Advanced Hepatocellular Carcinoma in China: A Comparative Analysis of HAIC-FO and Sorafenib.

BACKGROUND The FOHAIC-1 trial showed hepatic arterial infusion chemotherapy with infusional fluorouracil, leucovorin, and oxaliplatin (HAIC-FO) improved survival, compared with sorafenib, in patients with advanced hepatocellular carcinoma (HCC). The aim of this study was to conduct a cost-effectiveness comparison between HAIC-FO and sorafenib from the perspective of the Chinese healthcare system. MATERIAL AND METHODS The economic evaluation was conducted between July 2023 and February 2024, spanning a 10-year investment horizon. A Markov model was developed to perform a cost-effectiveness analysis of HAIC-FO vs sorafenib. Health states incorporated in the model comprised progression-free disease, progressed disease, and death. Transition probabilities were derived from data obtained from the FOHAIC-1 trial. Incremental cost-effectiveness ratio (ICER) was calculated to evaluate cost-effectiveness. Additionally, one-way and probabilistic sensitivity analyses assessed the model's robustness. RESULTS The HAIC-FO group accrued a total cost of $22,781, whereas the sorafenib group totaled $18,795. In terms of effectiveness, the HAIC-FO group achieved 1.06 quality-adjusted life years (QALYs), whereas the sorafenib group attained 0.65 QALYs. Compared with sorafenib, HAIC-FO yielded an additional 0.41 QALYs at a cost of additional $3,985, resulting in an incremental cost of $9,720 per QALY gained. The one-way sensitivity analysis revealed the final ICER remained below the willingness-to-pay (WTP) threshold of $30,492 per QALY, when considering parameter fluctuations. Additionally, probabilistic sensitivity analysis indicated a 99.8% probability that the ICER for HAIC-FO compared with sorafenib would fall below the WTP threshold. CONCLUSIONS Compared with sorafenib, HAIC-FO emerged as a cost-effective first-line treatment option for patients facing advanced HCC in China.

Probabilistic typhoon hazard and sensitivity analysis for nuclear power plant sites in Korea using logic tree

There has been an increase in typhoon intensity, and high-intensity typhoons can affect the safety of nuclear power plants (NPPs). Typhoons can damage the structures, systems, and components (SSCs) of NPPs, causing core damage and the release of radioactive materials into the environment. Therefore, design criteria for the effects of natural phenomena are demanded to ensure the safety-critical SSC functions of NPPs. Also, there is a need for safety assessments of typhoons in operational NPPs, which require typhoon hazard analysis. In this study, we derived typhoon hazards for NPPs in Korea using Logic Tree and Monte Carlo simulations. We performed sensitivity analysis on the typhoon hazards based on the distances (up to 100 km) from the NPP sites. Among other NPP sites, the typhoon hazard of the Kori site showed the highest wind speed, and the Hanul site showed the lowest wind speed. The uncertainty of the wind speed increased as the recurrence interval of the typhoon hazard increased. We compared the typhoon hazard in the areas of interest to that within 100 km. The range of the distance deviation for the Hanbit site's typhoon hazard was within μ±σ up to 75 km, while for the other sites, it was within μ±σ up to 100 km.

Cost-effectiveness analysis of a ‘Termination of Resuscitation’ protocol for the management of out-of-hospital cardiac arrest

BackgroundHistorically in Singapore, all out-of-hospital cardiac arrests (OHCA) were transported to hospital for pronouncement of death. A ‘Termination of Resuscitation’ (TOR) protocol, implemented from 2019 onwards, enables emergency responders to pronounce death at-scene in Singapore. This study aims to evaluate the cost-effectiveness of the TOR protocol for OHCA management. MethodsAdopting a healthcare provider’s perspective, a Markov model was developed to evaluate three competing options: No TOR, Observed TOR reflecting existing practice, and Full TOR if TOR is exercised fully. The model had a cycle duration of 30 days after the initial state of having a cardiac arrest, and was evaluated over a 10-year time horizon. Probabilistic sensitivity analysis was performed to account for uncertainties. The costs per quality adjusted life years (QALY) was calculated. ResultsA total of 3,695 OHCA cases eligible for the TOR protocol were analysed; mean age of 73.0 ± 15.5 years. For every 10,000 hypothetical patients, Observed TOR and Full TOR had more deaths by approximately 19 and 31 patients, respectively, compared to No TOR. Full TOR had the least costs and QALYs at $19,633,369 (95% Uncertainty Interval (UI) 19,469,973 to 19,796,764) and 0 QALYs. If TOR is exercised for every eligible case, it could expect to save approximately $400,440 per QALY loss compared to No TOR, and $821,151 per QALY loss compared to Observed TOR. ConclusionThe application of the TOR protocol for the management of OHCA was found to be cost-effective within acceptable willingness-to-pay thresholds, providing some justification for sustainable adoption.

Direct oral anticoagulants versus low-molecular-weight heparin in patients with cancer-associated venous thrombosis: a cost-effectiveness analysis.

Direct oral anticoagulants (DOACs) have demonstrated clinical benefits and better patient adherence over low-molecular-weight heparin (LMWH) in treating patients with cancer-associated venous thrombosis (CAT). We aimed to compare the cost-effectiveness of DOACs against LMWH in patients with CAT from the perspective of the Hong Kong healthcare system. A Markov state-transition model was performed to estimate the incremental cost-effectiveness ratio (ICER) per quality-adjusted life years (QALYs) for DOACs and LMWH in a hypothetical cohort of 10,000 patients with CAT over a 5-year lifetime horizon. The model was primarily based on the health states of no event, recurrent venous thromboembolism, bleeding, and death. Transition probabilities, relative risks, and utilities were derived from the literature. Resource cost data were obtained from the Hong Kong Hospital Authority. Deterministic and probabilistic sensitivity analyses tested the robustness of the results. Relative to LMWH, DOACs were associated with increased QALYs (1.52 versus 1.50) at a lower medical cost of USD 2,232 versus 8,224 in five years. The cost of LMWH was the main contributor to the outcome. Out of 10,000 simulated cases, DOACs were dominant in 15.8% and cost-effective in 42.1%, at the willingness-to-pay threshold of USD 148,392 per additional QALY. DOACs were associated with greater QALY improvements and lower overall costs compared to LMWH. Accounting for uncertainty, DOACs were between cost-effective and dominant in 57.9% of cases. DOACs are a cost-effective alternative to LMWH in the management of CAT in Hong Kong.

Cost-effectiveness analysis of first-line serplulimab plus chemotherapy for advanced squamous non-small-cell lung cancer in China: based on the ASTRUM-004 trial

ABSTRACT Objective In the ASTRUM-004 trial, serplulimab plus chemotherapy demonstrated significantly improved survival and controllable safety. This study assessed the cost-effectiveness of serplulimab plus chemotherapy in advanced squamous non‐small cell lung cancer (sqNSCLC), considering the perspective of the Chinese healthcare system. Methods A decision tree and a Markov model were constructed to simulate the treatment. The interesting results included total cost, life-years (LYs), quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs). Scenario, one-way and probabilistic sensitivity analyses were used to examine model instability. Results Compared with placebo plus chemotherapy, serplulimab plus chemotherapy had an ICER of $55,539.46/QALY ($47,278.84/LY). The ICERs were estimated to be $58,706.03/QALY, $48,978.34/QALY and $59,709.54/QALY inpatients with programmed death-ligand 1 expression level of tumor proportion score (TPS) < 1%, 1% ≤ TPS < 50%, and TPS ≥ 50%. The cost-effective prices of serplulimab were $168.276/100 mg, $349.157/100 mg, and $530.039/100 mg at the willingness-to-pay threshold of $12,574.30/QALY, $25,148.60/QALY, and $37,722.90/QALY. Patient weight and price of serplulimab created the most significant impact. Presently, the probability of serplulimab plus chemotherapy being cost-effective was 14.15%. Conclusion Compared with placebo plus chemotherapy, serplulimab plus chemotherapy might not be cost-effective in the first-line treatment for advanced sqNSCLC.

Cost-effectiveness analysis of pembrolizumab plus chemotherapy versus placebo plus chemotherapy for HER2-negative advanced gastric/gastroesophageal junction cancer in the Chinese healthcare system

ABSTRACT Background This study compares first-line pembrolizumab plus chemotherapy with chemotherapy alone for patients with HER2-negative advanced gastric cancer (GC) and gastroesophageal junction cancer (GEJC) in China. Methods A Markov state-transition model was developed based on the phase 3 randomized KEYNOTE-859 clinical trial data. The health state utility values and direct medical costs were derived from the KEYNOTE-859 clinical trial, the relevant literature, and local charges. The measured outcomes included quality-adjusted life-years (QALYs) and the incremental cost-effectiveness ratio (ICER). Probabilistic and one-way sensitivity analyses (OWSA) were performed to assess the uncertainty of the model. Results In the base analysis, the incremental effectiveness and cost of pembrolizumab plus chemotherapy versus chemotherapy alone were 0.22 QALYs and $16,627.31, respectively, resulting in an ICER of $76,936.60/QALY, which is higher than the willingness-to-pay threshold in China ($35,864.61/QALY). Subgroup analyses revealed that the ICERs of pembrolizumab plus chemotherapy versus chemotherapy alone were $72,762.68 and $34,813.70 in the populations with PD-L1 CPS of 1 or higher (CPS ≥ 1) and PD-L1 CPS ≥ 10 (CPS ≥ 10), respectively. Conclusions As first-line therapy for patients with locally advanced or metastatic HER2-negative GC/GEJC in China, pembrolizumab plus chemotherapy is less cost-effective than chemotherapy alone, however, in the CPS ≥ 10 subgroup is more.

Cost-effectiveness analysis of rezvilutamide versus bicalutamide in the treatment of metastatic hormone-sensitive prostate cancer

ObjectivesThe economic implications of combining rezvilutamide with androgen deprivation therapy (ADT) remain uncertain, despite the observed survival advantages compared with bicalutamide plus ADT. Therefore, this study evaluates the cost-effectiveness of...

Pembrolizumab combined with chemotherapy versus placebo combined with chemotherapy for HER2-negative advanced gastric cancer in China: a cost-effectiveness analysis

ABSTRACT Objective This study aims to conduct a cost-effectiveness analysis of pembrolizumab in combination with chemotherapy for HER2-negative advanced gastric cancer in China. Methods A partitioned survival approach model was constructed to simulate the progression of HER2-negative advanced gastric cancer and evaluate the outcomes of different treatment strategies. We calculated incremental cost-effectiveness ratios (ICER) to assess the cost associated with each quality-adjusted life-year (QALY) gained. One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess robustness and reliability. Results The analysis conducted in the base case demonstrated that the ICER associated with pembrolizumab was $177405.83/QALY gained in all population. In the subgroup analysis, it was found that individuals with a PD-L1 CPS ≥ 1 and those with a PD-L1 CPS ≥ 10 had ICERs of $152397.06/QALY and $109534.13/QALY, respectively. All ICER values for both the all population groups and the subgroups exceeded the WTP threshold in China. Our analysis shows the robustness of these results, as they remained consistent when input parameters were varied within a ± 25% range. Conclusion The findings of this cost-effectiveness analysis suggest that pembrolizumab in combination with chemotherapy is not a cost-effective treatment option for HER2-negative advanced gastric cancer in China.

Cost-effectiveness analysis of amivantamab plus chemotherapy for non-small cell lung cancer patients with epidermal growth factor receptor exon 20 insertions in the UnitedStates.

Although amivantamab has shown clinical benefits for non-small cell lung cancer (NSCLC) patients with epidermal growth factor receptor (EGFR) exon 20 insertions, its cost-effectiveness requires further investigation. To evaluate the cost-effectiveness of amivantamab plus chemotherapy for the treatment of NSCLC patients with EGFR exon 20 insertions from the UnitedStates payer perspective. A partitioned survival model was developed based on the data from the PAPILLON trial. Costs were derived from the pricing files of Medicare and Medicaid Services and published literature, and utility values were derived from previous studies. A 3% annual discount rate was applied to both costs and outcomes. The primary outcome was the incremental cost-effectiveness ratio (ICER). One-way sensitivity analysis, probabilistic sensitivity analysis and scenario analysis, were conducted to test the model stability. Amivantamab plus chemotherapy yielded an additional 1.12 quality-adjusted life years (QALYs) while increasing costs by $483,769.50 relative to the chemotherapy regimen, leading to an ICER of $432,401.16/QALY. The combination of amivantamab with chemotherapy was not cost effective at a threshold of $150,000/QALY. In the scenario analysis, the results showed that the ICERs were $263,680.69/QALY and $418,416.35/QALY when different utility values and 10-year time horizons were adopted, respectively. For PSA, the probability that amivantamab plus chemotherapy would be cost-effective was 0% if the willingness-to-pay (WTP) threshold was $150,000/QALY. Amivantamab plus chemotherapy is unlikely to be a cost-effective option for NSCLC patients with EGFR exon 20 insertions. Reducing the cost of amivantamab may produce favorable economic outcomes.

Cost-Effectiveness of Laparoscopic Sleeve Gastrectomy for Chinese Patients.

Laparoscopic sleeve gastrectomy (LSG) is the most popular bariatric surgery procedure in China. However, its cost-effectiveness in Chinese patients is currently unknown. This study aims to assess the cost-effectiveness of LSG vs no surgery in Chinese patients with severe and complex obesity, taking into account both healthcare expenses and the potential improvement in health-related quality of life (HRQoL). A retrospective cohort study was conducted, encompassing 135 Chinese patients who underwent LSG between January 3, 2022 and December 29, 2022, at a major bariatric center. The study evaluated the cost-effectiveness from a healthcare service perspective, employing the incremental cost-effectiveness ratio (ICER) for quality-adjusted life years (QALYs) gained. The analyses compared LSG with the alternative of not undergoing surgery over a 1-year period, using actual data, and extended to a lifetime horizon by projecting costs and utilities at an annual discount rate of 3.0%. Subgroup analyses were undertaken to explore cost-effectiveness variations across different sex, age and BMI categories, and diabetes status, employing a one-way analysis of variance (ANOVA). To ensure the reliability of the findings, one-way and probabilistic sensitivity analyses were executed. The results indicated that 1-year post-LSG, patients achieved an average total weight loss (TWL) of (32.7 ± 7.3)% and an excess weight loss (EWL) of (97.8 ± 23.1)%. The ICER for LSG compared to no surgery over a lifetime was $4,327/QALY, significantly below the willingness-to-pay (WTP) threshold for Chinese patients with severe and complex obesity. From a lifetime perspective, LSG proved to be cost-effective for all sex and age groups, across all BMI categories, and for both patients with and without diabetes. Notably, it was more cost-effective for younger patients, patients with higher BMI, and patients with diabetes. LSG is a highly cost-effective intervention for managing obesity in Chinese patients, delivering substantial benefits in terms of HRQoL improvement at a low cost. Its cost-effectiveness is particularly pronounced among younger individuals, those with higher BMI, and patients with diabetes.

Quantifying societal burden of radiation-induced small bowel toxicity in patients with rectal cancer.

Advancements in rectal cancer (RC) treatment not only led to an increase in lives saved but also improved quality of life (QoL). Notwithstanding these benefits, RC treatment comes at the price of gastrointestinal morbidity in many patients. Health economic modelling poses an opportunity to explore the societal burden of such side-effects. This study aims to quantify radiation-induced late small bowel (SB) toxicity in survivors of RC for Three-Dimensional Conformal Radiation Therapy (3D-CRT), Intensity Modulated Radiation Therapy (IMRT) and Intensity Modulated Radiation Therapy - Image Guided Radiation Therapy (IMRT/IGRT). Materials and A model-based health economic evaluation was performed. The theoretical cohort consists of a case-mix of survivors of RC aged 25-99 years according to Belgian age-specific incidence rates. A societal perspective was adopted. The base case analysis was complemented with one-way deterministic analyses, deterministic scenario analyses and probabilistic sensitivity analysis (1,000 iterations). Results were presented as mean lifetime incremental cost (€) and utility (QALYs) per patient. The analyses showed that the use of innovative radiotherapy (RT) improves lifetime QoL in survivors of RC by 0.11 QALYs and 0.05 QALYs by preferring IMRT/IGRT and IMRT over 3D-CRT, respectively. The use of IMRT/IGRT and IMRT results in an incremental cost-saving of €3,820 and €1,863 per patient, solely by radiation-induced SB toxicity, compared to 3D-CRT. It is important to consider late toxicity effects in decisions regarding investments and reimbursement as our analysis highlighted the potential long-term cost-savings and improved QoL of novel RT techniques in patients with rectal cancer.

Cost-effectiveness of single-pill and separate-pill administration of antihypertensive triple combination therapy: a population-based microsimulation study

BackgroundSingle-pill combination (SPC) of three antihypertensive drugs has been shown to improve adherence to therapy compared with free combinations, but little is known about its long-term costs and health consequences. This study aimed to evaluate the lifetime cost-effectiveness profile of a three-drug SPC of an angiotensin-converting enzyme inhibitor, a calcium-channel blocker, and a diuretic vs the corresponding two-pill administration (a two-drug SPC plus a third drug separately) from the Italian payer perspective.MethodsA cost-effectiveness analysis was conducted using multi-state semi-Markov modeling and microsimulation. Using the healthcare utilization database of the Lombardy Region (Italy), 30,172 and 65,817 patients aged ≥ 40 years who initiated SPC and two-pill combination, respectively, between 2015 and 2018 were identified. The observation period extended from the date of the first drug dispensation until death, emigration, or December 31, 2019. Disease and cost models were parametrized using the study cohort, and a lifetime microsimulation was applied to project costs and life expectancy for the compared strategies, assigning each of them to each cohort member. Costs and life-years gained were discounted by 3%. Probabilistic sensitivity analysis with 1,000 samples was performed to address parameter uncertainty.ResultsCompared with the two-pill combination, the SPC increased life expectancy by 0.86 years (95% confidence interval [CI] 0.61–1.14), with a mean cost differential of -€12 (95% CI -9,719–8,131), making it the dominant strategy (ICER = -14, 95% CI -€15,871–€7,113). The cost reduction associated with the SPC was primarily driven by savings in hospitalization costs, amounting to €1,850 (95% CI 17–7,813) and €2,027 (95% CI 19–8,603) for patients treated with the SPC and two-pill combination, respectively. Conversely, drug costs were higher for the SPC (€3,848, 95% CI 574–10,640 vs. €3,710, 95% CI 263–11,955). The cost-effectiveness profile did not significantly change according to age, sex, and clinical status.ConclusionsThe SPC was projected to be cost-effective compared with the two-pill combination at almost all reasonable willingness-to-pay thresholds. As it is currently prescribed to only a few patients, the widespread use of this strategy could result in benefits for both patients and the healthcare system.

A Cost-Effectiveness Analysis of Adjuvant Nivolumab for Patients with Resected Esophageal Cancer or Gastroesophageal Junction Cancer in France.

Esophageal and gastroesophageal junction cancer (EC/GEJC) is a poor prognosis disease with a high risk of recurrence even in patients curatively resected. Adjuvant nivolumab is currently used for patients with completely resected (R0) EC/GEJC who have residual pathologic disease following prior neoadjuvant chemoradiotherapy. This study aimed to determine the cost effectiveness of nivolumab in this indication in France according to the collective perspective excluding indirect costs. A simplified four-health-state semi-Markov model was developed to model EC/GEJC patients who have residual disease after neoadjuvant chemoradiotherapy followed by R0 over a 15-year time horizon, comparing adjuvant nivolumab versus surveillance, which was the recommended French clinical practice before immunotherapy arrival. Time-to-recurrence (TTR) from CheckMate 577 was used to inform transition from disease-free to post-recurrence health state; patients who recurred were split according to the distribution of type of recurrence observed during the trial. Post-recurrence survival (PRS) according to the type of recurrence was derived from a real-world registry. Adjuvant treatment with nivolumab led to an incremental survival gain of 1.19 years (+34%), mostly in the disease-free state, an incremental cost of €48,634 and QALY of 0.98 resulting in an incremental cost-utility ratio (ICUR) of €49,572/QALY with limited uncertainty. 'Cure assumption' at 5years had an important impact on the results (€41,115/QALY; -17%), as that tends to increase life-years and QALYs while costs remain the same. Probabilistic sensitivity analyses confirmed reference ICUR (€52,542/QALY) with 80% probability of nivolumab being cost effective at a willingness-to-pay threshold of €75,000/QALY. Our analysis suggests that adjuvant nivolumab is cost effective in the treatment of EC/GEJC patients who have residual disease after neoadjuvant CRT followed by R0 resection. Compared with previously evaluated cost-effectiveness analyses for other immune-checkpoint inhibitors indicated in metastatic settings, ICUR appears particularly low in this early setting thanks to the important impact on health outcomes and capped treatment duration.