Cost-effectiveness analysis of PD-1 inhibitors as second-line therapy for advanced or metastatic esophageal squamous cell carcinoma in China: an economic evaluation based on network meta-analysis.

BACKGROUND: Several economic studies have assessed the cost-effectiveness of programmed cell death protein-1 (PD-1) inhibitors compared to second-line chemotherapy in treating esophageal squamous cell carcinoma (ESCC). However, there is a lack of economic comparisons among the different PD-1 inhibitors. AIM: This study aimed to assess the cost-effectiveness of PD-1 inhibitors (nivolumab, pembrolizumab, camrelizumab, and tislelizumab) in second-line treatment for advanced or metastatic ESCC within the Chinese healthcare system. METHOD: The clinical trials were systematically retrieved from PubMed, Embase, Web of Science, and the Cochrane Library. We established a fractional polynomials model to conduct a network meta-analysis, enabling the calculation of hazard ratios and expected survival rates. Economic outcomes were estimated using a partitioned survival model. The costs and utilities were gathered from published sources. The threshold for willingness-to-pay (WTP) for a quality-adjusted life year (QALY) was set at three times China's per capita gross domestic product in 2022. Sensitivity analyses (SA) were performed to address uncertainties in the model. RESULTS: Four phase III randomized controlled trials were included, evaluating the cost-effectiveness of four PD-1 inhibitors, camrelizumab, nivolumab, tislelizumab, and pembrolizumab, compared to chemotherapy for the second-line treatment of advanced or metastatic ESCC. For camrelizumab, nivolumab, tislelizumab, and pembrolizumab, the corresponding incremental cost-effectiveness ratios were $27,375.43/QALY, $205,312.19/QALY, $9,266.73/QALY, and $220,368.10/QALY, respectively. The SA results indicated the robustness of the base analysis findings. CONCLUSION: From the Chinese healthcare system, under the WTP of $38,253.48/QALY, tislelizumab is a cost-effective treatment option for the second-line treatment of advanced or metastatic ESCC.

Impacts of adjustment of National Reimbursement Drug List on orphan drugs volume and spending in China: an interrupted time series analysis.

OBJECTIVE: To evaluate the impacts of the 2017 adjustment of National Reimbursement Drug List (NRDL) on orphan drugs hospital procurement volumes and spending in China. DESIGN: We used an interrupted time series design covering the period from 2016 to 2018 to analyse changes in hospital procurement volumes and spending of orphan drugs for which were included in the 2017 NRDL. SETTING AND DATA: The study was conducted in China. Orphan drug procurement data of 789 public hospitals (594 tertiary hospitals and 195 secondary hospitals) were derived from the Chinese Medical Economic Information (CMEI). OUTCOME MEASURES: Monthly orphan drugs hospital procurement volumes and spending. RESULTS: Nine orphan drugs were included in the 2017 NRDL (seven were directly included, and two were included after price negotiation). Comparing to orphan drugs not included in the NRDL, hospital procurement volumes ([Formula: see text] =43 312, p<0.001) and spending ([Formula: see text] =6 48 927, p<0.001) of the nine included drugs showed significant upward trends after implementation of the 2017 NRDL adjustment. CONCLUSIONS: Our results suggest that the 2017 adjustment of NRDL significantly changed the usage and spending on certain orphan drugs. The increase in orphan drug hospital procurement volumes should improve rare disease patients' access to these orphan drugs.

Adebrelimab plus chemotherapy vs. chemotherapy for treatment of extensive-stage small-cell lung cancer from the US and Chinese healthcare sector perspectives: a cost-effectiveness analysis to inform drug pricing.

Purpose: The aim of this study was to evaluate the cost-effectiveness of a recently approved first-line therapy (adebrelimab plus chemotherapy vs. chemotherapy alone) for patients with extensive-stage small-cell lung cancer (ES-SCLC) in the US and China, and to estimate the reasonable range of adebrelimab price from the decision-makers. Methods: Several partitioned survival models were built to compare the cost and effectiveness of adebrelimab plus chemotherapy vs. chemotherapy alone over a 10-year time horizon. Clinical efficacy and safety data were extracted from the CAPSTONE-1 trial. Costs and utilities were obtained from previously published studies. Sensitivity, scenario and subgroup analyses were performed to explore the uncertainty of the model outcomes. Price simulation was conducted at three thresholds of willingness-to-pay (WTP), including WTP of $100,000 in the US and of $37,422 in China, 0.5WTP of $50,000 in the US and of $18,711 in China, and 1.5WTP of 150,000 in the US and of $56,133 in China. Findings: Base-case analysis at $1382.82/600 mg of adebrelimab price indicated that adebrelimab plus chemotherapy would be cost-effective in the US at the WTP threshold of $100,000, but not in China at the WTP threshold of $37,422. If PAP was taken into account, the regimen would be cost-effective in China at the given WTP. The results of price simulation indicated that adebrelimab plus chemotherapy was completely favored in the US if adebrelimab price was less than $8894.98/600 mg (total quality-adjusted life years [QALYs] were calculated with progression-based utility [PB-utility]) or $8912.51/600 mg (total QALYs were calculated with time-to-death utility [TTD-utility]) at the WTP threshold of $100,000; if adebrelimab price was reduced by at least $202.03/600 mg (total QALYs were calculated with PB-utility) or $103.06/600 mg (total QALYs were calculated with TTD-utility), the regimen was also cost-effective in China without PAP at the WTP threshold of $37,422. The above results were stable in the sensitivity analyses. Subgroup analysis found that the subgroup with better survival benefits tended to have a higher probability of cost-effectiveness, which was also associated with adebrelimab price. Implications: First-line adebrelimab plus chemotherapy represented a dominant treatment strategy comparing with chemotherapy alone in the US and also did in China with PAP at $1382.82/600 mg of adebrelimab price. Decision-makers could benefit from pricing strategy provided by this study in making optimal decisions. More evidences were needed to verify and improve the results.

First-line treatments for advanced hepatocellular carcinoma: a network meta-analysis and cost-effectiveness analysis in China and the United States.

Background: Various therapeutic strategies are available for the first-line treatment of patients with advanced hepatocellular carcinoma (aHCC). But which approach is the most cost-effective remains uncertain. Objectives: This study aims to evaluate the cost-effectiveness of first-line strategies in aHCC patients from the perspective of Chinese and US payers. Design: A network meta-analysis (NMA) and cost-effectiveness study. Data sources and methods: A NMA was conducted to collect all first-line strategies with aHCC from 1 October 1 2018 until 1 January 2022. The relevant randomized controlled trial literature in PubMed, Embase, and Cochrane Library for the last 3 years were searched. The abstracts of meetings of the American Society of Clinical Oncology, European Society of Medical Oncology, and American Association for Cancer Research were also reviewed. A Markov model that included three states was developed. One-way sensitivity and probabilistic sensitivity analysis were performed to investigate the uncertainty of the economic evaluation. Scenario analysis was conducted to explore the economic benefits of treatment strategies in low-income populations. Results: Base-case analysis in China included 1712 patients showed that atezolizumab combined with bevacizumab, sintilimab combined with bevacizumab, lenvatinib (LEVA), and sorafenib (SORA) added 0.46, 1.25, 0.77, and -1.08 quality-adjusted life-years (QALYs), respectively, compared with donafenib, resulting in an incremental cost-effective ratio of $85607.88, $12109.27, and $1651.47 per QALY at a willingness-to-pay (WTP) of $11101.70/QALY. In the United States, only the incremental cost-effectiveness ratios (ICERs) of SORA was higher that were lower than the WTP threshold ($69375/QALY), and LEVA was the most cost-effective strategy with the ICERs were 25022.13/QALY. Conclusion: The NMA and cost-effectiveness analysis revealed that LEVA is the favorite choice in the first-line treatment of Chinese aHCC patients and US payers' perspective when the WTP was $11101.70/QALY in China and $69375.0/QALY in the United States. Registration: This study has been registered on the PROSPERO database with the registration number CRD42021286575.

Economic evaluation of tislelizumab versus chemotherapy as second-line treatment for advanced or metastatic esophageal squamous cell carcinoma in China.

Background and purpose: The latest RATIONALE-302 trial (NCT03430843) showed that tislelizumab therapy significantly improved overall survival benefits for patients with advanced or metastatic esophageal squamous cell carcinoma (ESCC) compared with traditional chemotherapy. This study aimed to compare the cost-effectiveness of tislelizumab versus chemotherapy as a second-line treatment for advanced or metastatic ESCC in China. Methods: A partitioned survival model was developed to predict patients' lifetime quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER) from the Chinese healthcare payers' perspective. We extracted efficacy and safety data from the RATIONALE-302 trial and the local cost and resource use data from online databases and published studies. One-way sensitivity analysis (OWSA) and probabilistic sensitivity analysis (PSA) were performed to explore model uncertainty. Results: Compared with chemotherapy, tislelizumab generated a higher cost (US$ 10211.78 vs. US$ 7294.72) but yielded more QALY (0.78 vs. 0.51 QALYs). The ICER for tislelizumab was US$11073.85 per QALY gained. The PSA results indicated that the probability of tislelizumab being economical was 76% under a willingness-to-pay (WTP) threshold of 1.5 times per capita GDP ($17915) in China. Conclusion: Tislelizumab could be a promising cost-effective strategy as the second-line treatment for patients with ESCC compared with chemotherapy in the Chinese setting.

Cost-effectiveness of edaravone dexborneol versus edaravone for the treatment of acute ischemic stroke in China: Based on the TASTE study.

Background and purpose: The TASTE trial indicated that patients with acute ischemic stroke (AIS) using edaravone dexborneol have a significantly higher proportion of 90-day good functional outcomes (mRS 0-1) than those using edaravone. This study compared the cost-effectiveness of the aforementioned interventions in treating AIS in the Chinese setting, aiming to inform treatment decisions in clinical practice. Methods: A model combining a decision tree and a Markov model was developed to assess the cost-effectiveness of edaravone dexborneol versus edaravone for AIS over a 30-year time horizon from the Chinese healthcare system's perspective. Both efficacy and safety data were extracted from the TASTE study. Local costs and utilities were derived from publications and open-access databases; both cost and effectiveness were discounted at a rate of 5% per year. Sensitivity analyses were conducted to ensure robustness and identify the main drivers of the result. Results: Compared with edaravone, edaravone dexborneol for AIS was found to be cost-effective in the first year and highly cost-effective as the study time horizons extended. In the long term (30 years), edaravone dexborneol yielded a lifetime gain of 0.25 (0.07-0.45) quality-adjusted life years (QALYs) at an additional cost of CNY 2201.07 (-3,445.24-6,637.23), yielding an ICER of CNY 8823.41 per QALY gained under the willingness-to-pay (WTP) of 1.5 times per capita GDP (121,464 CNY). The result is robust in both deterministic and probabilistic sensitivity analysis (PSA) methods, with the advantage of the edaravone dexborneol strategy increasing over time. Specifically, the probability of edaravone dexborneol dominant dexborneol is 76.30%, 98.90%, and 99.50% over 1-, 5-, and 30-year time horizons. Conclusion: Both short- and long-term economic analyses suggest that edaravone dexborneol is highly likely to be a cost-effective alternative to treat AIS compared with edaravone in China.

A Systematic Review of Methods and Study Quality of Economic Evaluations for the Treatment of Schizophrenia.

Background: Schizophrenia is a severe and complex disease with substantial economic and social burdens. Despite multiple treatment choices, adverse events, and impaired social functions are still challenges in clinical therapy. Pharmacoeconomic evaluations could provide evidence to help decision makers improve the utilization of scarce resources. However, there remains some challenges especially in modeling due to uncertainties in progression of schizophrenia. There are limited summaries about the overall methodologies of schizophrenia economic evaluations. Objective: The aim of this study is to review the existing economic evaluations of antipsychotics for the treatment of schizophrenia and summarize the evidence and methods applied. Methods: An electronic literature search was performed in PubMed, Web of Science, EBSCO host, The Cochrane Library and ScienceDirect from January 2014 to December 2020. Search terms included "schizophrenia," "schizophrenic," "pharmacoeconomic," "economic evaluation," "cost-effectiveness," and "cost-utility." The Literature was screened and extracted by two researchers independently and assessed with the Quality of Health Economic Studies (QHES) List and Consolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement. Results: A total of 25 studies were included in the review. The regions included Europe, North America, Asia and Africa. Most of the studies chose second-generation antipsychotics as comparators and integrated treatment sequences. Time horizons varied from 1 year to lifetime. The healthcare sector was the most common perspective, accordingly, most of the evaluations considered only direct medical costs. The Markov model and decision tree model were the most common choices. Adverse events, compliance and persistence were considered important parameters. Quality-adjusted life-years were the major outcomes applied to the economic evaluations. All utilities for health states and adverse events were collected from published literature. All of the studies applied uncertainty analysis to explore the robustness of the results. The quality of the studies was generally satisfactory. However, improvements were needed in the choice of time horizons, the measurements of outcomes and the descriptions of assumptions. Conclusions: This study highlights the methodology of economic evaluation of schizophrenia. Recommendations for modeling method and future study are provided.

Economic Evaluation of Cisplatin Plus Gemcitabine Versus Paclitaxel Plus Gemcitabine for the Treatment of First-Line Advanced Metastatic Triple-Negative Breast Cancer in China: Using Markov Model and Partitioned Survival Model.

INTRODUCTION: This study aimed to evaluate the cost-effectiveness of cisplatin plus gemcitabine vs. paclitaxel plus gemcitabine as a first-line treatment for metastatic triple-negative breast cancer in China. METHODS: The Markov model and partitioned survival (PS) model were used, and the study included three health states over the period of a lifetime. Transition probabilities and safety data were derived from the CBCSG006 trial (cisplatin plus gemcitabine vs. paclitaxel plus gemcitabine in patients who had acquired metastatic triple-negative breast cancer). Cost and utility values were derived from previous studies, the Chinese Drug Bidding Database, and healthcare documents. Sensitivity analyses were performed to observe model stability. RESULTS: In the Markov model, compared with paclitaxel plus gemcitabine, cisplatin plus gemcitabine yielded an additional 0.15 QALYs, with an incremental cost of 1976.33 USD. The incremental cost-utility ratio (ICUR) was 12,826.98 USD/QALY (quality-adjusted life year). In the PS model, cisplatin plus gemcitabine yielded an additional 0.17 QALYs with an incremental cost of 2384.63 USD; the incremental cost-utility ratio (ICUR) was 13,867.7 USD/QALY. In the first scenario analysis, in which the 3-year time horizon was used in both arms, the total QALYs in the cisplatin plus gemcitabine group were larger and the costs were lower, indicating that cisplatin plus gemcitabine was superior to paclitaxel plus gemcitabine. In the second scenario, in which the progression-free (PF) utility (during chemotherapy) was 0.76, the PF utility was 0.96, and the post-progression (PP) utility was 0.55, the result obtained with the Markov model showed that the ICUR was 11,063.68 USD/QALY. In the probabilistic sensitivity analysis (PSA) on the Markov model, the probabilities that cisplatin plus gemcitabine would be cost-effective were 48.94-78.72% if the willingness-to-pay threshold was 9776.8 to 29,330.4 USD/QALY. CONCLUSIONS: The findings of the present analysis suggest that cisplatin plus gemcitabine might be much more cost-effective than paclitaxel plus gemcitabine in patients receiving first-line treatment for metastatic triple-negative breast cancer in China.