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.

Cost-effectiveness analysis of atezolizumab plus bevacizumab compared with sorafenib as first-line treatment in advanced hepatocellular carcinoma in Singapore.

OBJECTIVES: This study aims to explore the cost-effectiveness of atezolizumab plus bevacizumab against sorafenib for first-line treatment of locally advanced or metastatic hepatocellular carcinoma (HCC) in Singapore. METHODS: A partitioned survival model was developed from a healthcare system perspective, with a 10-year lifetime horizon. Clinical inputs and utilities were obtained from the IMbrave150 trial. Healthcare resource use costs were obtained from published local sources; drug costs reflected the most recent public hospital selling prices. Outcomes included life years, quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs). Deterministic and probabilistic sensitivity analyses were performed to assess the model's robustness. RESULTS: Atezolizumab plus bevacizumab offered an additional 1.42 life years and 1.09 QALYs, with an additional cost of S$111,847; the ICER was S$102,988/QALY. The World Health Organization considers interventions with ICERs <1 gross domestic product (GDP)/capita to be highly cost-effective. At a willingness-to-pay (WTP) threshold of S$114,165/QALY (Singapore's 2022 GDP/capita), atezolizumab plus bevacizumab is cost-effective compared with sorafenib. The ICER was most sensitive to variations in utilities, but all parameter variations had no significant impact on the model outcomes. CONCLUSION: At a WTP threshold of Singapore's GDP/capita, atezolizumab plus bevacizumab is cost-effective compared with sorafenib.

Economic evaluation of camrelizumab plus rivoceranib versus sorafenib as first-line therapy for unresectable hepatocellular carcinoma in the United States and China.

BACKGROUND: Camrelizumab combined with rivoceranib has been proven effective for treating unresectable hepatocellular carcinoma (uHCC). However, their higher prices than sorafenib could impose a substantial economic burden on patients. AIM: This study aimed to evaluate the relative cost-effectiveness of the combination of camrelizumab and rivoceranib versus sorafenib as first-line therapy for patients with uHCC from the perspective of the US and Chinese payers. METHOD: Using data from the CARES-310 trial, a partitioned survival model (PSM) was developed, considering the perspectives of the US and Chinese payers. The model employed a 15-year time horizon and a biweekly cycle. Direct medical costs and utility data were collected from previous studies and open-access databases. Primary outcomes included quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio (ICER). Price simulations, sensitivity analyses, and subgroup analyses were conducted. RESULTS: The ICER for the US and China was $122,388.62/QALY and $30,410.56/QALY, respectively, falling below the willingness-to-pay (WTP) thresholds of $150,000/QALY for the US and $35,898.87/QALY for China. Price simulations indicated the cost-effectiveness of camrelizumab plus rivoceranib when the price of camrelizumab (200 mg) remained below $6275.19 in the US and $558.09 in China. The primary determinant of cost-effectiveness in both regions was the cost of camrelizumab. CONCLUSION: The combination of camrelizumab and rivoceranib is a cost-effective first-line therapy for uHCC in both the US and China. Lowering their prices could significantly influence their cost-effectiveness and accessibility to patients. These findings will guide clinicians in treating uHCC and help decision-makers formulate value-based drug pricing strategies.

Cost-effectiveness of Atezolizumab Plus Bevacizumab vs Sorafenib for Patients With Unresectable or Metastatic Hepatocellular Carcinoma.

Importance: Atezolizumab plus bevacizumab as a first-line therapy for patients with unresectable or metastatic hepatocellular carcinoma has been shown to improve overall and progression-free survival compared with standard sorafenib treatment. However, because of the high cost of atezolizumab plus bevacizumab, assessment of its value by considering both efficacy and cost is needed. Objective: To evaluate the cost-effectiveness of atezolizumab plus bevacizumab vs sorafenib for patients with unresectable or metastatic hepatocellular carcinoma from a US payer perspective. Design, Setting, and Participants: This economic evaluation was performed from June through September 2020, with a 6-year investment time period. Hypothetical patients were male and female adults 18 years or older who had a diagnosis of locally advanced metastatic or unresectable hepatocellular carcinoma confirmed by histologic or clinical features. Main Outcomes and Measures: Health care costs (adjusted to 2020 US dollars), life-years, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) of atezolizumab plus bevacizumab vs sorafenib were examined using a partitioned survival model. One-way deterministic and probabilistic sensitivity analyses were used to examine model uncertainty. The model was also used to estimate price reductions of atezolizumab plus bevacizumab that would achieve more favorable cost-effectiveness. Results: In the base case analysis of a hypothetical sample of 424 patients, atezolizumab plus bevacizumab was associated with an increase of 0.623 life-years (1.840 vs 1.218 life-years) and 0.484 QALYs (1.412 vs 0.928 QALYs) and with an incremental cost of $156 210 per patient compared with sorafenib. The ICER was $322 500 per QALY (5th to 95th percentile, $149 364-$683 744 per QALY), with 0.6% and 5.1% chance of being cost-effective at willingness-to-pay thresholds of $100 000 and $150 000 per QALY, respectively. The ICER never decreased below $150 000 per QALY in the 1-way sensitivity analyses. To achieve more favorable cost-effectiveness under the thresholds of $150 000 to $100 000 per QALY, the prices of atezolizumab and bevacizumab would need to be reduced by 37% to 47%. Conclusions and Relevance: In this economic evaluation, atezolizumab plus bevacizumab was associated with clinical benefit but was not cost-effective compared with sorafenib for first-line treatment of unresectable or metastatic hepatocellular carcinoma from a US payer perspective. A substantial reduction in price for atezolizumab plus bevacizumab would be needed to achieve favorable cost-effectiveness for this new therapy.

Cost-effectiveness of Atezolizumab Plus Bevacizumab vs Sorafenib as First-Line Treatment of Unresectable Hepatocellular Carcinoma.

Importance: Treatment with atezolizumab plus bevacizumab may prolong overall survival among patients with unresectable hepatocellular carcinoma. However, to our knowledge, the cost-effectiveness of using this high-priced therapy for this indication is currently unknown. Objective: To evaluate the cost-effectiveness of atezolizumab plus bevacizumab to treat unresectable hepatocellular carcinoma from the US payer perspective. Design, Setting, and Participants: This economic evaluation used a partitioned survival model consisting of 3 discrete health states to assess the cost-effectiveness of treatment of hepatocellular carcinoma with atezolizumab plus bevacizumab vs sorafenib. The characteristics of patients in the model were similar to patients in a phase 3, open-label randomized clinical trial (IMbrave150) who had unresectable hepatocellular carcinoma and had not previously received systemic treatment. Key clinical data were generated from the IMbrave150 trial conducted between March 15, 2018, and January 30, 2019, and cost and health preference data were collected from the literature. Main Outcomes and Measures: Costs, quality-adjusted life-years (QALYs), incremental cost-utility ratios, incremental net health benefits, and incremental net monetary benefits were calculated for the 2 treatment strategies. Subgroup, 1-way sensitivity, and probabilistic sensitivity analyses were performed. Results: Treatment of hepatocellular carcinoma with atezolizumab plus bevacizumab added 0.530 QALYs and resulted in an incremental cost of $89 807 compared with sorafenib therapy, which had an incremental cost-utility ratio of $169 223 per QALY gained. The incremental net health benefit was -0.068 QALYs, and the incremental net monetary benefit was -$10 202 at a willingness-to-pay threshold of $150 000/QALY. The probabilistic sensitivity analysis indicated that treatment with atezolizumab plus bevacizumab achieved a 35% probability of cost-effectiveness at a threshold of $150 000/QALY. One-way sensitivity analysis revealed that the results were most sensitive to the hazard ratio of overall survival. The subgroup analysis found that treatment with atezolizumab plus bevacizumab was associated with preferred incremental net health benefits in several subgroups, including patients with hepatitis B and C. Conclusions and Relevance: Atezolizumab plus bevacizumab treatment is unlikely to be a cost-effective option compared with sorafenib for patients with unresectable hepatocellular carcinoma. Reducing the prices of atezolizumab and bevacizumab may improve cost-effectiveness. The economic outcomes also may be improved by tailoring treatments based on individual patient factors.

Cost-utility analysis of selective internal radiation therapy with Y-90 resin microspheres in hepatocellular carcinoma.

Background: The study assessed the cost-utility of selective internal radiation therapy (SIRT) with Y-90 resin microspheres versus sorafenib in UK patients with unresectable hepatocellular carcinoma ineligible for transarterial chemoembolization. Materials & methods: A lifetime partitioned survival model was developed for patients with low tumor burden (≤25%) and good liver function (albumin-bilirubin grade 1). Efficacy, safety and quality of life data were from a European Phase III randomized controlled trial and published studies. Resource use was from registries and clinical surveys. Results: Discounted quality-adjusted life-years were 1.982 and 1.381, and discounted total costs were £29,143 and 30,927, for SIRT and sorafenib, respectively. Conclusion: SIRT has the potential to be a dominant (more efficacious/less costly) or cost-effective alternative to sorafenib in patients with unresectable hepatocellular carcinoma.

Cost-Effectiveness of Lenvatinib Compared with Sorafenib for the First-Line Treatment of Advanced Hepatocellular Carcinoma in Australia.

BACKGROUND AND OBJECTIVE: In the REFLECT trial, lenvatinib showed superior clinical benefits to sorafenib in terms of progression-free survival and was non-inferior for overall survival in the treatment of advanced hepatocellular carcinoma (HCC). We assessed the cost-effectiveness of lenvatinib compared with sorafenib for patients with advanced HCC in Australia. METHOD: A partitioned-survival model was built to perform a cost-effectiveness analysis comparing lenvatinib and sorafenib from an Australian health-system perspective. Survival curves were obtained from the REFLECT trial and fitted with parametric survival functions for extrapolation purposes beyond the trial follow-up. Cost and quality-adjusted life-years (QALYs) were accrued over the 10-year time horizon of the model. Deterministic and probability sensitivity analysis (PSA) were carried out to verify the validity of the model. RESULTS: Lenvatinib incurred higher costs (A$96,325) and superior health outcomes (QALYs: 1.205), while sorafenib had lower costs (A$92,394) and inferior health outcomes (QALYs: 1.086). Thus, lenvatinib yielded an incremental cost-utility ratio of A$33,028/QALY gained. Further, the results of the PSA found that the probability of lenvatinib being cost-effective at a willingness-to-pay threshold of A$50,000/QALY was 64%. CONCLUSION: Our study found that, at current prices, lenvatinib is a cost-effective treatment option compared with sorafenib for the first-line treatment of patients with advanced HCC.

Treatment patterns and direct medical costs among patients with advanced hepatocellular carcinoma.

AIMS: This study assessed the real-world United States (US) treatment patterns and the associated economic burden in patients diagnosed with advanced hepatocellular carcinoma (HCC). METHODS: The MarketScan database was used to identify patients newly diagnosed with HCC who received systemic therapy between 2011 and 2018 and continuously enrolled for ≥6 months (baseline period) prior and ≥1 month following HCC diagnosis. Treatment patterns (systemic and locoregional therapy), healthcare resource utilization, and costs were reported during follow-up. RESULTS: The final sample included 1580 patients (median age, 61; 78% male; median follow up, 8.7 months). The most common first line of therapy (LOT) was sorafenib (78%). The median time from HCC diagnosis to start of sorafenib was 43 days, and the median duration of sorafenib therapy was 60 days. Only 17% of patients received second LOT, and non-sorafenib treatment use increased to 66% (mostly chemotherapy combination). Transarterial chemoembolization was the most commonly observed locoregional therapy prior to the first LOT. The multivariable-adjusted average all-cause total cost among sorafenib treated patients was $17,642 (95% CI: $16,711-$18,558) per-patient per-month), of which $11,393 were HCC-specific. CONCLUSIONS: In patients who received first-line therapy for HCC, the duration of therapy was short (potentially due to progression or tolerability). Most patients did not continue to second-line therapy. Despite the short duration of therapy, HCC patients still incur a high economic burden, and there is a need for more effective and tolerable treatments.

Demographic and Clinicopathologic Factors of Patients With Hepatocellular Carcinoma in a Safety Net Hospital.

BACKGROUND: Safety net hospitals have historically cared for a disproportionate number of patients of low socioeconomic status, racial and ethnic minorities, and patients with cancer. These innate challenges make safety net hospitals important in understanding how to improve access to cancer care in order to fit the needs of vulnerable patients and ultimately improve their outcomes. The purpose of this study is to characterize the current state and treatment of hepatocellular carcinoma (HCC) at Ben Taub Hospital, a safety net hospital in Houston, Texas. MATERIALS AND METHODS: A retrospective chart review was performed to review the demographic characteristics, clinicopathologic data, treatment strategies, and outcomes of HCC patients at Ben Taub Hospital between January 2012 and December 2014. RESULTS: Two-hundred twenty-six men and 78 women with a mean age of 58 y underwent evaluation. Most (87%) were either uninsured or covered by Medicaid. The majority (69%) of patients presented with advanced (stage 2 or more) disease, with 58% of patients presenting with multiple lesions. Of the 40% that presented with a solitary lesion, the average size was 4.97 cm. Transarterial chemoembolization was used in 37% of patients and sorafenib was given to 26% of patients. Five patients underwent successful transplant. One hundred seventeen (38%) patients died of their disease, 25 patients are alive with no evidence of disease, and 159 patients have been lost to follow-up. CONCLUSIONS: Most patients with HCC presented to this safety net hospital with advanced disease; however, multiple local and systemic treatments were offered. Screening programs to detect HCC at an earlier stage are essential for successful long-term outcomes in a resource-strapped hospital with limited access to liver transplantation.

New insights on sorafenib resistance in liver cancer with correlation of individualized therapy.

Liver cancer is highly malignant and insensitive to cytotoxic chemotherapy and is associated with very poor patient prognosis. In 2007, the small-molecule targeted drug sorafenib was approved for the treatment of advanced liver cancer. In the subsequent ten years, sorafenib has been the only first-line therapeutic targeted drug for advanced hepatocellular carcinoma (HCC). However, a number of clinical studies show that a considerable percentage of patients with liver cancer are insensitive to sorafenib. The number of patients who actually benefit significantly from sorafenib treatment is very limited, and the overall efficacy of sorafenib is far from satisfactory, which has attracted the attention of researchers. Based on previous studies and reports, this article reviews the potential mechanisms of sorafenib resistance (SR) and summarizes the biomarkers and clinicopathological indicators that might be used for predicting sorafenib response and developing personalized therapy.

Lenvatinib versus sorafenib for unresectable hepatocellular carcinoma: a cost-effectiveness analysis.

Aim: To investigate the cost-effectiveness of lenvatinib and sorafenib in the treatment of patients with nonresected hepatocellular carcinoma in China. Materials & methods: Markov model was used to simulate the direct medical cost and quality-adjusted life years (QALY) of patients with hepatocellular carcinoma. Clinical data were derived from the Phase 3 randomized clinical trial in a Chinese population. Results: Sorafenib treatment resulted in 1.794 QALYs at a cost of $43,780.73. Lenvatinib treatment resulted in 2.916 QALYs for patients weighing <60 and ≥60 kg at a cost of $57,049.43 and $75,900.36, The incremental cost-effectiveness ratio to the sorafenib treatment group was $11,825.94/QALY and $28,627.12/QALY, respectively. Conclusion: According to WHO's triple GDP per capita, the use of lenvatinib by providing drugs is a cost-effective strategy.

Treatment Patterns and Economic Burden by Lines of Therapy Among Patients with Advanced Hepatocellular Carcinoma Treated with Systemic Cancer Therapy.

PURPOSE: This study examined clinical and economic outcomes among patients with advanced hepatocellular carcinoma (HCC) treated with systemic agents by line of therapy. METHODS: Adults with ≥ 2 medical claims for primary diagnosed HCC (from January 1, 2008, through September 30, 2015) and ≥ 1 claim for systemic HCC-related therapy were identified in the IBM MarketScan® Research Databases. Continuous enrollment was required 6 months before and 1 month after diagnosis. Patients were categorized into first- (1L) and second-line (2L) treatment cohorts; those receiving sorafenib as 1L were evaluated. Treatment patterns, healthcare resource utilization, costs, and survival during 1L and 2L therapy were measured. Survival was assessed for patients linked to the Social Security Administration Master Death File. RESULTS: 1459 patients, 758 with death data, met the 1L cohort criteria; 163 patients, 87 with death data, later received 2L therapy. 77.1% had 1L sorafenib, alone or in combination. Median 1L treatment duration was 3.0 months; median survival time from start of 1L to death or censor was 6.8 months. There was no predominant 2L agent. Median 2L treatment duration was 3.0 months; median survival time from start of 2L was 9.3 months. Median total healthcare costs per patient per month were $13,297 for 1L (all), $13,471 for 1L (sorafenib), and $11,786 for 2L. CONCLUSIONS: Findings confirm high 1-year mortality for advanced HCC, suggesting a high cost burden. While no 2L therapy was available during this analysis, recently approved 2L agents have the potential to improve survival after sorafenib failure or intolerance.

Economic Implications of Hepatocellular Carcinoma Surveillance and Treatment: A Guide for Clinicians.

The incidence of hepatocellular carcinoma (HCC) is increasing worldwide, with significant morbidity and associated costs. Treatment allocation depends on the stage of diagnosis; however, resource utilization can be significant across all stages. We aimed to summarize the available data on the cost effectiveness of surveillance of and treatments for HCC in the context of current treatment guidelines. We performed a focused review of studies investigating the economic burden and cost effectiveness of HCC surveillance treatment modalities published between January 2000 and January 2019. The overall economic burden of HCC is increasing in the USA and in several countries worldwide due to its rising incidence and the proliferation of therapies. Liver transplantation is a cost-effective strategy for early-stage HCC treatment in selected patients. In settings where liver transplantation is not available or in patients awaiting transplant, ablative or locoregional therapies are cost effective with increases in quality-adjusted life-years. First-line therapy with sorafenib for advanced stage HCC is cost effective in the treatment of compensated cirrhosis. The cost effectiveness of recently approved systemic therapies for advanced HCC require further investigation. Existing studies have shown that guideline-recommended surveillance techniques and several available therapies for the treatment of HCC are cost effective; however, there are limitations in the literature, including reliance on suboptimal modeling with incomplete/simplified model structure or inadequate inputs. With increasing therapeutic options in patients with HCC, understanding their relative value is critical in designing HCC treatment algorithms.

How patent law reform can improve affordability and accessibility of medicines in South Africa: Four medicine case studies.

South Africa (SA) is in the process of amending its patent laws. Since its 2011 inception, Fix the Patent Laws, a coalition of 40 patient groups, has advocated for reform of SA's patent laws to improve affordability of medicines in the country. Building on two draft policies (2013, 2017) and a consultative framework (2016) for reform of SA's patent laws, Cabinet approved phase 1 of the Intellectual Property Policy of the Republic of South Africa on 23 May 2018. Fix the Patent Laws welcomed the policy, but highlighted concerns regarding the absence of important technical details, as well as the urgent need for government to develop bills, regulations and guidelines to provide technical detail and to codify and implement patent law reform in the country. In this article, we explore how reforms proposed in SA's new intellectual property policy could improve access to medicine through four medicine case studies.

Cost-effectiveness analysis of lenvatinib treatment for patients with unresectable hepatocellular carcinoma (uHCC) compared with sorafenib in Japan.

BACKGROUND: Lenvatinib demonstrated a treatment effect on overall survival by the statistical confirmation of non-inferiority to sorafenib for the first-line treatment of uHCC. The objective of this study was to evaluate the cost-effectiveness of lenvatinib compared with sorafenib for patients with uHCC in Japan. METHODS: A partitioned-survival model was developed to estimate the cost-effectiveness of lenvatinib versus sorafenib when treating uHCC patients over a lifetime horizon and considering total public healthcare expenditure. Efficacy and safety data were extracted from the REFLECT trial. Utility values were derived from the European Quality-of-Life 5-Dimension Questionnaire, conducted with patients enrolled in the REFLECT trial. Direct medical costs, such as primary drug therapy, outpatient visits, diagnostic tests, hospitalization, post-progression therapy, and adverse-event treatments, were included. Cost parameters unavailable in the clinical trial or publications were obtained based on the consolidated clinical standards from a Delphi panel of four Japanese medical experts. RESULTS: For lenvatinib versus sorafenib, the incremental cost was - 406,307 Japanese Yen (JPY), and the incremental life years and quality-adjusted life years (QALYs) were 0.27 and 0.23, respectively. Thus, lenvatinib dominated sorafenib, due to the mean incremental cost-effectiveness ratio falling in the fourth quadrant, conferring more benefit at lower costs compared with sorafenib. The probabilistic sensitivity analysis showed that 81.3% of the simulations were favorable to lenvatinib compared with sorafenib, with a payer's willingness-to-pay-per-QALY of 5 million JPY. CONCLUSIONS: Lenvatinib was cost-effective compared with sorafenib for the first-line treatment of uHCC in Japan.

Cost-effectiveness of sorafenib versus best supportive care in advanced hepatocellular carcinoma in Egypt.

BACKGROUND: In light of constrained budgets and the need to fund efficient treatment options, this study set out to assess the cost-effectiveness of sorafenib as a first-line treatment of hepatocellular carcinoma (HCC) compared to best supportive care (BSC) from the military hospital perspective in Egypt. METHODS: A decision analytic Markov model simulated disease progression with clinical parameters and utility values derived from published data. Data on direct medical costs were collected from the local healthcare system or payer. Costs and effects were discounted at 3.5% annually and reported in USD using purchasing power parity adjustments. Deterministic and probabilistic sensitivity analyses were conducted. RESULTS: Mortality occurred less frequently in the sorafenib group (sorafenib group: 99.96%, BSC group: 99.99%). The total quality-adjusted life years (QALYs) of the sorafenib cohort were estimated to be 46.24 compared with 42.27 for the BSC cohort, which resulted in an incremental gain of 3.96 QALYs. The total costs for the sorafenib and BSC cohorts were USD 4,229,940 and USD 3,092,886, respectively (incremental cost = $1,137,054), resulting in an incremental cost-effectiveness ratio (ICER) of USD 286,776 per QALY gained for the sorafenib cohort. One-way sensitivity analyses that addressed the uncertainty of the BSC estimates indicated that the progression-free survival for BSC and utility value of progression had the greatest effects on the results. CONCLUSION: This study concluded that sorafenib does offer increased survival and quality-of-life at an increased cost but at an ICER that exceeds the nationally accepted cost-effectiveness threshold. The findings support healthcare decision-making of the efficient allocation of healthcare system resources to improve the health of the Egyptian population. Whether sorafenib is cost-effective in specific sub-groups with additional risk factors needs to be addressed in future studies.

Regorafenib treatment for patients with hepatocellular carcinoma who progressed on sorafenib-A cost-effectiveness analysis.

BACKGROUND AND AIMS: Hepatocellular carcinoma (HCC) is one of the leading causes of cancer related deaths. Patients with advanced HCC are treated with sorafenib. A recent randomized controlled trial demonstrated a survival benefit for regorafenib treatment in patients with advanced HCC who had progressed on sorafenib. We aimed to evaluate the cost-effectiveness of this approach. METHODS: To evaluate the cost effectiveness of regorafenib, we used a Markov model that incorporates health outcomes, measured by life-years and quality-adjusted life-years (QALYs). Drug costs were based on 2017 discounted prices. Model robustness was validated by probabilistic sensitivity analyses using Monte Carlo simulations. RESULTS: The use of regorafenib results in a gain of 19.76 weeks of life (0.38 Life Years) as compared to placebo. When adjusted for quality of life, using regorafenib produced a gain of 0.25 quality adjusted life years (QALYs). The incremental cost-effectiveness ratio for regorafenib compared with best supportive care was between $201,797 and $268,506 per QALY. CONCLUSION: The modest incremental benefit at a relatively high incremental cost of regorafenib treatment suggests that it is not cost-effective at commonly accepted willingness to pay thresholds.

Cost analysis for different sequential treatment regimens for metastatic renal cell carcinoma in China.

PURPOSE: Targeted therapies, including sunitinib, sorafenib, axitinib, and everolimus, have recently become the mainstay for the treatment of metastatic renal cell carcinoma (mRCC). The objective of this study was to estimate the costs of sequential treatment regimens for mRCC and associated adverse events (AEs) from the Chinese payers' perspective. METHODS: Key inputs included in the calculation were patient population, dosing information, incidence rates and associated costs of Grade 3/4 AEs, treatment costs (including drug discount programs), and patients' progression-free survival (PFS) as a proxy for length of treatment. To calculate PFS, this study identified pivotal clinical trials and generated a reconstructed individual patient data set from the published Kaplan-Meier survival curves. The median PFS from the pooled estimates were used in the calculation. In the base-case scenario, sunitinib was used as first line and the other three therapies were used as second line. Sensitivity analyses were conducted where (1) sorafenib was used as first line, or (2) a third-line therapy was added to the base-case scenario. RESULTS: In the base case, the cost per patient per treatment month (PPPM) cost was the lowest for sunitinib + axitinib among all sequential regimens (¥14,898) and was the highest for sunitinib + sorafenib (¥20,103). If sorafenib is used as first line, everolimus had lower per patient per months (PPPM) cost than axitinib (¥17,046 vs ¥23,337), but also had shorter PFS (13.5 months vs 15 months). Second sensitivity analysis with an additional third-line therapy showed consistent results with the base-case scenario; axitinib as second line was the least costly. CONCLUSIONS: This study demonstrates that, for mRCC sequential treatment, sunitinib followed by axitinib generates the highest cost savings from the Chinese payers' perspective. Future studies are warranted to examine the cost-effectiveness of various mRCC treatment regimens in Chinese populations.

Disparities in Access to Sorafenib in Communities with Low Socioeconomic Status.

OBJECTIVE: In the United States, hepatocellular carcinoma (HCC) is more common among communities with low socioeconomic status (SES), and these groups tend to be diagnosed with later-stage cancers. Sorafenib is the primary treatment for advanced HCC, however its substantial cost raises concern for access to treatment. METHODS: The newly developed Case-Background method was used to estimate odds ratios for the impacts of various sociodemographic factors on sorafenib access in clinically eligible patients. Socioeconomic status was defined as a factor of median income and education level based on ZIP code of residence. RESULTS: There was a strong association between sorafenib prescription and residence in an area of higher SES. While controlling for age, race/ethnicity, and insurance status, high SES residence doubled the odds of sorafenib prescription (OR=2.05, p<.01). CONCLUSIONS: Low socioeconomic status communities appear to have a reduced chance of receiving the only effective treatment for advanced HCC.

Cost-effectiveness analysis of treatments involving radioembolization in intermediate-stage hepatocellular carcinoma.

AIM: We evaluated two treatment sequences, transarterial radioembolization followed by transarterial chemoembolization and possibly sorafenib (=TTS) versus transarterial radioembolization followed by sorafenib alone (=TS), to identify the most cost-effective pathway to treat intermediate-stage hepatocellular carcinoma from the Italian healthcare system perspective. MATERIALS & METHODS: A Markov model was developed to project costs and health outcomes for TTS and TS over a lifetime horizon. Data available at three hospitals in Italy were collected. Healthcare resource utilization was derived from standard clinical protocols. Costs were obtained from official regional tariffs. RESULTS & CONCLUSION: Taking into consideration 16 patients for TTS and 22 patients for TS pathways, the TTS sequence provided a dominant strategy in comparison to TS. Further evidence is desirable to confirm these results.