Economic evaluation of cemiplimab plus chemotherapy regimen for advanced non-small-cell lung cancer.

OBJECTIVE: Cemiplimab, a novel PD-1 inhibitor, exhibits significant antitumor activity against advanced non-small cell lung cancer (NSCLC). However, the cost-effectiveness of this drug for the treatment remains unclear. This study aimed to assess the cost-effectiveness of cemiplimab plus chemotherapy compared to chemotherapy for the treatment of advanced NSCLC, from the perspective of the United States payer. METHODS: A partitioned survival approach was developed to project the disease progression of NSCLC. Overall survival (OS) and progression-free survival (PFS) data were obtained from the EMPOWER lung 3 trial and extrapolated to estimate long-term survival outcomes. Direct medical costs and utility data were collected. The primary outcome measure, the incremental cost-utility ratio (ICUR), was used to evaluate the cost-effectiveness of cemiplimab plus chemotherapy regimen. One-way sensitivity analyses (OWSA) and probabilistic sensitivity analyses (PSA) were conducted to assess the robustness of the results. RESULTS: In the base-case analysis, the ICUR for cemiplimab plus chemotherapy versus chemotherapy alone was estimated to be $395,593.8 per quality-adjusted life year (QALY). OWSA revealed that the results were sensitive to Hazard ratio value, utility of PFS, and cost of cemiplimab. PSA demonstrated that cemiplimab plus chemotherapy exhibited 0% probability of cost-effectiveness.In hypothetical scenario analysis, the ICUR of two regimens was $188.803.3/QALY. OWSA revealed that the results were sensitive to the discount rate, utility, and cost of cemiplimab. PSA indicated that cemiplimab plus chemotherapy achieved at least an 11.5% probability of cost-effectiveness. CONCLUSION: Our cost-effectiveness analysis suggests that, at its current price, cemiplimab plus chemotherapy regimen is unlikely to be a cost-effective option compared with chemotherapy alone for advanced NSCLC patients, based on a threshold of $150,000 per QALY, from the perspective of the US payer.

Pembrolizumab alone or in combination with chemotherapy versus chemotherapy for advanced gastric cancer: A cost-effectiveness analysis.

PURPOSE: The KEYNOTE-062 trial demonstrated the efficacy and safety of pembrolizumab for advanced gastric cancer (GC). The current study evaluated the cost-effectiveness of pembrolizumab alone or in combination with chemotherapy versus chemotherapy for advanced GC from the perspective of the United States and China. And the results will provide evidence and data support for more drug selection-related decisions and research in the future. METHODS: A partitioned survival approach with three states was created for treatment of advanced GC. The survival data were derived from KEYNOTE-062 trial and the individual patient data were generated by a specific algorithm. We fitted 21 survival functions to each treatment arm and selected the most suitable distribution type for each one. Direct costs and utility values were collected from the published, available database. Cost, quality-adjusted life-years (QALYs), and incremental cost-utility ratios (ICURs) were considered as the primary measure outcomes. One-way and probabilistic sensitivity analyses were performed to assess the reliability of the analyses. RESULTS: In the base-case analysis of combined positive score (CPS) ≥1 patients, the ICUR of pembrolizumab plus chemotherapy versus chemotherapy in American and Chinese setting is $345,209/QALY and $186,802.6/QALY, respectively. And the ICUR of pembrolizumab versus chemotherapy is $473,650/QALY and $377,753/QALY in the context of the US and China, respectively. For CPS≥10 patients, the ICUR of pembrolizumab plus chemotherapy versus chemotherapy in American and Chinese setting is $483,742/QALY and $262,965/QALY, respectively. And that of pembrolizumab versus chemotherapy is $96,550/QALY and $67,896/QALY in the context of the US and China. CONCLUSION: Compared with chemotherapy, either pembrolizumab plus chemotherapy or pembrolizumab monotherapy is not regarded as a cost-effective strategy for patients with CPS≥1, advanced gastric cancer in the current American and Chinese setting. But pembrolizumab monotherapy for CPS≥10 patients would become a cost-effective option in the American setting.

The effective threshold dose of etanercept in patients with methotrexate-resistant rheumatoid arthritis.

INTRODUCTION: The therapy of rheumatoid arthritis (RA) was advanced by biological agents, yet costly. This study aims to identify the effective threshold dose of etanercept (ENT) and cost-effectiveness in methotrexate (MTX)-resistant RA in real world. METHODS: Eligible patients had an inadequate response (DAS28-ESR > 3.2) to initial MTX monotherapy, and subsequently received etanercept. The effective cut-off value of cumulative dose was identified to maintain remission response (DAS28-ESR < 2.6) at month 24 by using restricted cubic splines. Remission rate, low disease activity (LDA) rate, glucocorticoid exposure, safety, and cost-effectiveness were compared between the saturated and non-saturated dose groups divided by the cut-off dose. RESULTS: Seventy-eight (14.2%) of 549 enrolled patients were eligible, and 72 patients completed follow-up. The 2-year cumulative cut-off dose that maintained remission response at 24 months was 1975 mg. And the recommended threshold dosing strategy of etanercept was twice weekly (BIW) for the first 6 months, every week (QW) for the next 6 months, and every 2 weeks (Q2W) and every month (QM) for the second year. Greater net changes in DAS28-ESR score were observed in the ENT saturated dose group than in the non-saturated dose group (average change 0.569, 95%CI 0.236-0.901, p = 0.001). The proportion of patients achieving remission (27.8% vs 72.2%, p < 0.001) and LDA (58.3% vs 83.3%, p = 0.020) in the non-saturated group was both significantly lower than that in the saturated group at 24 months. The incremental cost-effectiveness ratio of the saturated group referred to the non-saturated group was 5791.2 $/QALY. CONCLUSIONS: In refractory RA patients, the effective cumulative cut-off dose of etanercept for sustained remission at 24 months was calculated as 1975 mg, and receiving saturated dose was more effective and cost-effective than with non-saturated dose. Key Points • The effective cumulative cut-off dose of etanercept for sustained remission at 24 months in RA patients is calculated as 1975 mg. • Receiving saturated dose of etanercept is more effective and cost-effective than with non-saturated dose in refractory RA patients.

Cost-effectiveness of sacituzumab govitecan versus chemotherapy in advanced or metastatic triple-negative breast cancer.

PURPOSE: The ASCENT trial demonstrated the efficacy of sacituzumab govitecan for the treatment of advanced or metastatic triple-negative breast cancer (TNBC). The current study evaluated the cost-effectiveness of receiving sacituzumab govitecan compared with standard of care chemotherapy from the United States payer perspective. METHODS: A partitioned survival approach was used to project the disease course of advanced or metastatic TNBC. Two survival modes were applied to analyze two groups of patients. The survival data were gathered from the ASCENT trial. Direct medical costs were derived from the data of Centers for Medicare & Medicaid Services. Utility data was collected from the published literature. The incremental cost-utility ratio (ICUR) was the primary outcome that measured the cost-effectiveness of therapy regimen. One-way sensitivity and probabilistic sensitivity analysis were implemented to explore the uncertainty and validate the stability of results. RESULTS: In the base-case, the ICUR of sacituzumab govitecan versus chemotherapy is $ 778,771.9/QALY and $ 702,281/QALY for full population group and brain metastatic-negative (BMN) group with the setting of classic survival mode. And in the setting of cure survival mode, the ICUR is $ 506,504.5/QALY for the full population group and $ 274,232.0/QALY for BMN population group. One-way sensitivity analyses revealed that the unit cost of sacituzumab govitecan and body weight were key roles that lower the ICUR value. Probabilistic sensitivity analyses also showed that reducing the unit price of sacituzumab govitecan can improve the likelihood of becoming cost-effective. CONCLUSION: The cost-effectiveness analysis suggested that from a US payer perspective, sacituzumab govitecan at current price is unlikely to be a preferred option for patients with advanced or metastatic TNBC at a threshold of $ 150,000/QALY.

Economic Evaluation of Trastuzumab Deruxtecan in Previously Treated HER2-Low Advanced Breast Cancer in the United States.

Purpose: Recently, the DESTINY-Breast04 trial revealed that significant benefits in both overall survival (OS) and progression-free survival (PFS) in patients with HER2-low advanced or metastatic breast cancer treated with trastuzumab deruxtecan (T-DXd) compared with chemotherapy. The current study assessed the cost-effectiveness of T-DXd from the perspective of the United States payer. Methods: We developed a partitioned survival model to project the disease course of breast cancer. The OS and PFS data were derived from the DESTINY-Breast04 trial. We extrapolate the survival data beyond the follow-up time to assess the long-term survival prognosis. Direct medical costs and utility data were collected. The incremental cost-utility ratio (ICUR) was the primary outcome that evaluated the cost-effectiveness of a therapy regimen. One-way sensitivity and probabilistic sensitivity analysis were implemented to explore the uncertainty of outputs. Results: In the base-case, the ICUR of T-DXd versus chemotherapy is $346,571.8/QALY and $337,789.4/QALY for all patients group and hormone-receptor-positive (HR+) subgroup, respectively. One-way sensitivity analyses revealed that the hazard ratio of OS, the unit cost of T-DXd, and body weight had a relatively large impact on the base-case result. Probabilistic sensitivity analyses showed that the likelihood that T-DXd was cost-effective is 14.5% and 12.6% for all patients group and HR+ subgroup, respectively. Conclusion: The cost-effectiveness analysis suggested that, at current price, trastuzumab deruxtecan is unlikely to be a preferred option for patients with HER2-low breast cancer at a threshold of $150,000/QALY from a US payer perspective.

Budget Impact Analysis of the Introduction of a Trastuzumab Biosimilar for HER2-Positive Breast Cancer in China.

BACKGROUND AND OBJECTIVE: Biosimilars provide the possibility to reduce the high expenditure on biologic drugs and expand access to effective but less expensive treatments. Biosimilars of trastuzumab showed significant cost savings from the payer's perspective in the USA and Europe. After 2020, with the first approval of a trastuzumab biosimilar in China, it became feasible for biosimilar switching for trastuzumab. However, the economic impact of switching to a trastuzumab biosimilar was not evaluated. A budget impact model was constructed from a payer's perspective of China to demonstrate the economic impact of the introduction of a biosimilar trastuzumab in the treatment of human epidermal growth factor receptor 2-positive breast cancer. METHODS: This budget impact model was based on disease incidence to estimate the net budget impact using epidemiological data from the literature, financial reports from manufacturers on the market shares of originator trastuzumab (Herceptin®) or the biosimilar, and localized direct costs. The budget impact was estimated for 5 years after the introduction of the first-approved trastuzumab biosimilar in China. Furthermore, two scenarios were simulated in this study to estimate the budget impact of biosimilars within: (1) real-world practice and (2) the policy of volume-based procurement. RESULTS: Analyses of the base-case and scenario results implied that adoption of a trastuzumab biosimilar would lead to an expenditure decrease. The average total cost savings over 5 years was estimated to be US$46,651,348, with a range from $10,306,611 in year 1 to $60,821,822 in year 5. The cost savings could benefit an additional 654-3858 patients with breast cancer. If utilizing costs from real-world practice, the introduction of a trastuzumab biosimilar could help an additional 2237-13,203 patients get access to human epidermal growth factor receptor 2-positive targeted therapy. When volume-based procurement was carried out after year 4, $672,366,180 could be saved annually. CONCLUSIONS: This budget impact analysis emphasized the positive effects of adopting a trastuzumab biosimilar in the healthcare system of China. However, cost savings still have a large potential to decrease by regulating pricing and by the procurement policy of biosimilars.

Atezolizumab plus platinum-based chemotherapy as first-line therapy for metastatic urothelial cancer: A cost-effectiveness analysis.

Purpose: According to the IMvigor130 trial, adding atezolizumab to platinum-based chemotherapy was effective in the treatment of metastatic urothelial cancer (mUC). Based on the perspective of the United States and China, the current study evaluated cost-effectiveness of atezolizumab plus chemotherapy for mUC patients in the first-line setting. Methods: A partitioned survival model was adopted for mUC patients. The survival data were derived from the IMvigor130 trial. Direct cost values were collected from the Centers for Medicare and Medicaid Services (CMS), Chinese Drug Bidding Database, and published literatures. The utility and toxicity data were gathered from related research studies and IMvigor130 trial. The incremental cost-utility ratios (ICURs) and incremental cost-effectiveness ratios (ICERs) were calculated and analyzed. Scenario analyses and sensitivity analyses were performed to observe the outputs and uncertainties. Results: The base-case analysis showed that the ICUR of atezolizumab plus chemotherapy versus chemotherapy in American and Chinese settings is $ 737,371 /QALY and $ 385,384 /QALY, respectively. One-way sensitivity analyses showed that the ICUR ranged from $ 555,372/QALY to $ 828,205/QALY for the United States. Also, the range was from $ 303,099/QALY to $ 433,849/QALY in the Chinese setting. A probabilistic sensitivity analysis showed the likelihood that atezolizumab plus chemotherapy becoming the preferred strategy was a little low even if the price reduction strategy was applied. Conclusion: Adding atezolizumab to chemotherapy improved survival time, but it is not a cost-saving option compared to chemotherapy for metastatic urothelial cancer patients in the American and Chinese settings.

αvß3 integrin-specific exosomes engineered with cyclopeptide for targeted delivery of triptolide against malignant melanoma.

BACKGROUND: Melanoma is the most malignant skin tumor and is difficult to cure with the alternative treatments of chemotherapy, biotherapy, and immunotherapy. Our previous study showed that triptolide (TP) exhibited powerful tumoricidal activity against melanoma. However, the clinical potential of TP is plagued by its poor aqueous solubility, short half-life, and biotoxicity. Therefore, developing an ideal vehicle to efficiently load TP and achieving targeted delivery to melanoma is a prospective approach for making full use of its antitumor efficacy. RESULTS: We applied exosome (Exo) derived from human umbilical cord mesenchymal stromal cells (hUCMSCs) and engineered them exogenously with a cyclic peptide, arginine-glycine-aspartate (cRGD), to encapsulate TP to establish a bionic-targeted drug delivery system (cRGD-Exo/TP), achieving synergism and toxicity reduction. The average size of cRGD-Exo/TP was 157.34 ± 6.21 nm, with a high drug loading of 10.76 ± 1.21%. The in vitro antitumor results showed that the designed Exo delivery platform could be effectively taken up by targeted cells and performed significantly in antiproliferation, anti-invasion, and proapoptotic activities in A375 cells via the caspase cascade and mitochondrial pathways and cell cycle alteration. Furthermore, the biodistribution and pharmacokinetics results demonstrated that cRGD-Exo/TP possessed superior tumor targetability and prolonged the half-life of TP. Notably, cRGD-Exo/TP significantly inhibited tumor growth and extended survival time with negligible systemic toxicity in tumor-bearing mice. CONCLUSION: The results indicated that the functionalized Exo platform provides a promising strategy for targeted therapy of malignant melanoma.

Patient Preference for Biologic Treatments of Psoriasis in the Chinese Setting.

Objective: Assessments of patients' preferences can support in clinical decision-making regarding biologic therapies for psoriasis. Our objective was to investigate patient preference for biologic treatments in patients with psoriasis in China. Methods: From October 2020 to January 2021, psoriasis patients were recruited for a survey that included demographic and disease-related questions, as well as a discrete choice experiment to measure their preferences for biologic therapy. A discrete-choice experiment was used in which respondents selected psoriasis treatments based on benefits (ie, early onset of efficacy, long-term efficacy, sustained efficacy) and treatment costs. We analyzed choice data using conditional logit model. Results: This study included 236 patients with moderate-to-severe psoriasis. The relative importance of the cost of biologic treatments, probability of keeping PASI100 at 5 years, probability of achieving PASI100 at 3 months and time to achieve PASI50 after initiation the biologic treatment were 0.593, 0.137, 0.185 and 0.085. Over 50% of patients regarded the cost of biologic treatments as the most important attribute. High-income and low-income subgroups had higher preference weight in probability of achieving PASI100 at 3-month and monthly cost. Conclusion: The cost of biologic treatments was found as the most important attribute for Chinese patients with psoriasis. Among efficacy attributes, the probability of achieving PASI100 at 3 months showed most sensitive. These results may be helpful to understand patient preference for biologic treatments used for psoriasis in China.

What Is Value in Health and Healthcare? A Systematic Literature Review of Value Assessment Frameworks.

OBJECTIVES: This study aimed to investigate how value is defined and measured in existing value assessment frameworks (VAFs) in healthcare. METHODS: We searched PubMed, Embase, the Cochrane Library, and Centre for Reviews and Dissemination from 2008 to 2019. We also performed backward citation chaining of included studies and previously published systematic reviews. Studies reporting the development of a VAF in healthcare were included. For each included framework, we extracted and compared the context, target users, intended use, methods used to identify value attributes, description of the attributes, and attribute scoring approaches. RESULTS: Of the 8151 articles screened, 57 VAFs were included. The value attributes included in 55 VAFs were grouped into 9 categories: health benefits (n = 53, 96%), affordability (n = 45, 82%), societal impact (n = 42, 76%), burden of disease (n = 36, 65%), quality of evidence (n = 32, 58%), cost-effectiveness (n = 31, 56%), ethics and equity (n = 27, 49%), unmet needs (n = 21, 38%), and innovation (n = 15, 27%). The remaining 2 VAFs used broad attributes or user-defined attributes. Literature review was the main approach to identify value attributes in 36 VAFs. Patient or public was engaged through the development of only 11 VAFs. Weighting has been used to score 29 VAFs, of which 19 used the methods of multicriteria decision analysis. CONCLUSIONS: There are substantial variations in defining and measuring value. A noticeable weakness of existing VAFs is that patient or public engagement was generally very limited or missing in framework development process. Existing VAFs tend to aggregate multiple value attributes into a single index for decision making.

Atezolizumab Plus Chemotherapy vs. Chemotherapy in Advanced or Metastatic Triple-Negative Breast Cancer: A Cost-Effectiveness Analysis.

Purpose: The IMpassion130 trial demonstrated the efficacy of adding atezolizumab to paclitaxel for advanced or metastatic triple-negative breast cancer (TNBC). The current study evaluated the cost-effectiveness of adding atezolizumab to nab-paclitaxel for TNBC from the perspective of Chinese health sector. Methods: A partitioned survival model was implemented for patients with TNBC. The survival data were derived from IMpassion130 trial. Direct costs and utility values were collected from the Chinese Drug Bidding Database and published literatures. The primary analysis outcomes were quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were performed to observe model stability. Results: In the base-case analysis, the ICER of atezolizumab plus nab-paclitaxel vs. nab-paclitaxel is respectively, $176,056/QALY, $118,146/QALY, and $323,077/QALY in the ITT, PD-L1(+) and PD-L1(-) group. Conclusion: Adding atezolizumab to nab-paclitaxel could improve survival time significantly in the PD-L1-positive group, but it is not a cost-effective strategy compared to nab-paclitaxel monotherapy for Chinese patients with advanced or metastatic triple-negative breast cancer in the current economic context of China.

Naringin attenuates renal interstitial fibrosis by regulating the TGF-ß/Smad signaling pathway and inflammation.

Interstitial fibrosis is a typical feature of all progressive renal diseases. The process of fibrosis is frequently coupled with the presence of pro-fibrotic factors and inflammation. Naringin is a dihydroflavone compound that has been previously reported to exhibit anti-fibrotic effects in the liver, where it prevents oxidative damage. In the present study, a rat model of renal interstitial fibrosis and fibrosis cell model were established to evaluate the effects of naringin on inflammatory proteins and fibrosis markers in kidney of rats and NRK-52E cells, and to elucidate the role of the TGF-ß/Smad signaling pathway in this mechanism. Compared with those in fibrotic NRK-52E cells that were stimulated by transforming growth factor-ß (TGF-ß), gene expression levels of α-smooth muscle actin (α-SMA), collagen 1 (COL1A1), collagen 3 (COL3A1), interleukin (IL)-1ß, IL-6 and tumor necrosis factor-α (TNF-α) were all found to be significantly decreased in fibrotic NRK-52E cells following treatment with naringin (50, 100 and 200 ng/ml). Results from the histopathological studies showed that naringin treatment preserved the renal tissue structure and reduced the degree of fibrosis in the kidney tissues of rats that underwent unilateral ureteral obstruction (UUO). In addition, naringin administration reduced the expression of α-SMA, COL1A1, COL3A1, IL-1ß, IL-6 and TNF-α in the kidneys of rats following UUO. The current study, using western blot analysis, indicated that naringin also downregulated the activation of Smad2/3 and the expression of Smad4, high-mobility group protein B1, activator protein-1, NF-κB and cyclooxygenase-2 whilst upregulating the expression of Smad7 in fibrotic NRK-52E cells and rats in the UUO group. In conclusion, naringin could antagonize renal interstitial fibrosis by regulating the TGF-ß/Smad pathway and the expression of inflammatory factors.

Methotrexate (MTX) Plus Hydroxychloroquine versus MTX Plus Leflunomide in Patients with MTX-Resistant Active Rheumatoid Arthritis: A 2-Year Cohort Study in Real World.

PURPOSE: To compare the efficacy, safety, and cost-effectiveness of methotrexate (MTX) plus hydroxychloroquine (HCQ) vs MTX plus leflunomide (LEF) in established rheumatoid arthritis (RA) with inadequate response to MTX monotherapy in a real-world Chinese cohort. PATIENTS AND METHODS: A prospective RA cohort (n=549) was screened with eligible patients who had inadequate response (disease activity score in 28 joints using erythrocyte sedimentation rate, DAS28-ESR>3.2) to initial MTX monotherapy and subsequently received either MTX+HCQ or MTX+LEF. Propensity score matching (PSM) was applied to adjust the possible baseline confounders between two groups. The primary outcome was the proportion of patients achieving first remission (DAS28-ESR<2.6) during follow-up by log rank test. Secondary outcomes were changes of DAS28, glucocorticoids (GCs) exposure, safety, cost-effectiveness, sustained remission, and low disease activity (LDA) rate after 24-month follow-up. RESULTS: Overall, 222 eligible patients were subjected to the aforementioned two treatment protocols (MTX+HCQ, n=102; MTX+LEF, n=120). After PSM adjustment, 97 patients in each group were analyzed. A higher remission rate was observed in the MTX+HCQ group than in the MTX+LEF group (70.1% vs 56.7%, P=0.048). The median time to remission was 11 and 16 months in the two groups, respectively. At the endpoint, more patients achieved remission (46.8% vs 32.5%, P=0.063) and maintained sustained LDA in the HCQ group (53.2% vs 38.6%, P=0.062) and also more patients withdrew GCs in this group (32% vs 16.7%, P=0.053) than those in the LEF group. Safety profiles were non-alarming, with no significant difference between the two groups. The incremental cost-effectiveness ratio yielded by MTX+HCQ over MTX+LEF was $1,111.8 per quality-adjusted life-year (QALY), within the cost-effective threshold set as the per capita gross domestic product (GDP) of China. CONCLUSION: The MTX+HCQ combination was seemingly superior to MTX+LEF in a real-world cohort of Chinese RA patients with inadequate response to methotrexate monotherapy in respect of the efficacy and cost-effectiveness.

Cetuximab Plus Chemotherapy versus Chemotherapy Alone in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma: A Cost-Effectiveness Analysis.

OBJECTIVE: The EXTREME clinical trial revealed that cetuximab plus chemotherapy improved the overall survival time of patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) versus chemotherapy alone. The current study examined the cost-effectiveness of cetuximab plus chemotherapy compared with chemotherapy alone in HNSCC patients from the perspective of China. MATERIALS AND METHODS: A partitioned survival model was implemented for R/M HNSCC patients. Survival information was derived from the CHANGE-2 trial. The model was designed as a ten-year time horizon, a 3-week cycle, and a 3% discount rate for costs and utilities. An incremental cost-effectiveness ratio (ICER) value is less than $30,201/quality-adjusted life-year (QALY) was considered cost-effective in China. We analyzed the uncertainty by performing one-way and probabilistic sensitivity analyses. RESULTS: In the base-case analysis, we found that the ICER of cetuximab plus chemotherapy compared with chemotherapy alone is $172,702/QALY. The results of one-way sensitivity analysis and probabilistic analysis showed that the fluctuations of each variable in its ranges do not cause ICERs to reach acceptable thresholds. CONCLUSION: The current observations suggested that treatment with cetuximab plus chemotherapy is not a cost-effective strategy for R/M HNSCC patients in China at a $30,201 willingness to pay threshold.

Pembrolizumab vs the EXTREME Regimen in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma: A Cost-Effectiveness Analysis.

BACKGROUND AND OBJECTIVE: The KEYNOTE-048 clinical trial revealed that pembrolizumab improved the overall survival time of patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) vs cetuximab plus chemotherapy (EXTREME regimen). The current study examined the cost effectiveness of pembrolizumab monotherapy and pembrolizumab plus chemotherapy compared with the EXTREME regimen in patients with HNSCC from the perspectives of USA and China. METHODS: A partitioned survival model was implemented for patients with R/M HNSCC, and the cost effectiveness of pembrolizumab monotherapy and pembrolizumab plus chemotherapy compared with the EXTREME regimen was compared. Survival information was derived from the KEYNOTE-048 trial. The model was designed as a 20-year time horizon, a 3-week cycle, and a 3% discount rate for costs and utilities. An incremental cost-effectiveness ratio (ICER) value less than $100,000/quality-adjusted life-year (QALY) was considered cost effective in the USA and $27,538/QALY in China. We analyzed the uncertainty by performing one-way and probabilistic sensitivity analyses. RESULTS: From the base-case analysis, we found that the pembrolizumab monotherapy scheme had a lower cost and better efficacy compared with the EXTREME regimen in the USA. In China, the ICER of the comparison was $62,401/QALY. The ICER of pembrolizumab plus chemotherapy vs the EXTREME regimen was $66,630/QALY in the USA and $90,538/QALY in China. CONCLUSIONS: The observations suggested that treatment with pembrolizumab monotherapy or pembrolizumab plus chemotherapy is a cost-effective strategy for patients with R/M HNSCC in the USA. However, the conclusion is the opposite for China: the EXTREME regimen is still a cost-effective choice.