scholarly journals Sequencing Systemic Therapy Pathways for Advanced Hepatocellular Carcinoma: A Cost Effectiveness Analysis

Liver Cancer ◽  
2020 ◽  
Vol 9 (5) ◽  
pp. 549-562
Author(s):  
Christopher Sherrow ◽  
Kristopher Attwood ◽  
Kehua Zhou ◽  
Sarbajit Mukherjee ◽  
Renuka Iyer ◽  
...  

Introduction: Hepatocellular carcinoma (HCC) is the most common form of liver cancer worldwide and carries a poor prognosis. Historically, sorafenib was the only available systemic treatment for advanced HCC. However, in recent years, 6 new treatments have been approved by the US Food and Drug Administration (FDA): regorafenib, lenvatinib, cabozantinib, pembrolizumab, ramucirumab, and nivolumab. Data are lacking regarding the most appropriate sequencing pathway for these agents. Our objective was to conduct a comprehensive cost effectiveness analysis (CEA) of different 1st- and 2nd-line treatment pathways for HCC reflecting all new drug approvals, and then use our data to provide guidance for clinicians on which pathway is the most cost-effective. Materials and Methods: Markov models were used to evaluate the cost effectiveness of 8 different 1st- and 2nd-line treatment sequences. The model allowed for 9 possible states. Cost effectiveness ratios (CER) and incremental CER (ICER) were calculated to compare costs between different pathways and against a willingness-to-pay (WTP) threshold. Efficacy and toxicity data were extracted from the landmark trials for each agent. All agents except ramucirumab were included. The cost of each agent was based on the wholesale acquisition cost (WAC) in USD as of June 2019. Monte-Carlo methods were used to simulate the experience of 1,000,000 patients per treatment sequence for a 12-month period. Results: The pathway with the lowest CER was sorafenib, followed by pembrolizumab (USD 227,741.03/quality-adjusted life year [QALY]). ICER analysis supported implementing 2nd-line pembrolizumab-based pathways at a higher WTP threshold of 300,000/quality-adjusted life year. Sensitivity analysis did not substantially change these results. Conclusions: The most cost-effective strategy was 1st-line tyrosine kinase inhibitor therapy followed by 2nd-line immunotherapy. All pathways exceeded a commonly accepted WTP of USD 100–150,000/QALY. Our preliminary results warrant further studies to best inform real-world practices.

2019 ◽  
Vol 17 (6) ◽  
pp. 669-675 ◽  
Author(s):  
Enrique Soto-Perez-de-Celis ◽  
Pedro N. Aguiar ◽  
Mónica L. Cordón ◽  
Yanin Chavarri-Guerra ◽  
Gilberto de Lima Lopes

Background: Treatment options are limited for patients with advanced hepatocellular carcinoma (HCC) that progresses after treatment with sorafenib. Cabozantinib, an oral small molecule inhibitor of multiple tyrosine kinase receptors, recently showed improved overall survival (OS) compared with placebo in sorafenib-pretreated patients with advanced HCC in the CELESTIAL trial. This study assessed the cost-effectiveness of cabozantinib for second-line treatment of patients with advanced HCC from a US healthcare system perspective. Patients and Methods: Cost and utility data were extracted from the CELESTIAL trial and used to determine the cost-effectiveness of cabozantinib compared with placebo plus best supportive care. The main outcome of this study was the incremental cost-effectiveness ratio (ICER), expressed as cost per quality-adjusted life year (QALY) gained by using cabozantinib compared with placebo plus best supportive care in sorafenib-pretreated HCC. Results: In the base-case analysis using data from the CELESTIAL trial, the incremental QALY and ICER were 0.067 and $1,040,675 for cabozantinib compared with placebo and best supportive care. OS reported in the CELESTIAL trial (hazard ratio, 0.76; 95% CI, 0.63–0.92) had the strongest association with the ICER. In one-way sensitivity analyses, there were no scenarios in which cabozantinib was cost-effective. In a cost-threshold analysis, cabozantinib would have to be priced at least $50 per pill to be cost-effective considering a willingness to pay of $100,000 per QALY. Although the CELESTIAL trial demonstrated that cabozantinib improves OS compared with placebo in patients with HCC that progresses after treatment with sorafenib, our analysis shows that cabozantinib is not a cost-effective therapy in this scenario. Conclusions: At current costs, cabozantinib is not cost-effective for second-line therapy of HCC in the United States.


2021 ◽  
pp. 019459982110268
Author(s):  
Joseph R. Acevedo ◽  
Ashley C. Hsu ◽  
Jeffrey C. Yu ◽  
Dale H. Rice ◽  
Daniel I. Kwon ◽  
...  

Objective To compare the cost-effectiveness of sialendoscopy with gland excision for the management of submandibular gland sialolithiasis. Study Design Cost-effectiveness analysis. Setting Outpatient surgery centers. Methods A Markov decision model compared the cost-effectiveness of sialendoscopy versus gland excision for managing submandibular gland sialolithiasis. Surgical outcome probabilities were found in the primary literature. The quality of life of patients was represented by health utilities, and costs were estimated from a third-party payer’s perspective. The effectiveness of each intervention was measured in quality-adjusted life-years (QALYs). The incremental costs and effectiveness of each intervention were compared, and a willingness-to-pay ratio of $150,000 per QALY was considered cost-effective. One-way, multivariate, and probabilistic sensitivity analyses were performed to challenge model conclusions. Results Over 10 years, sialendoscopy yielded 9.00 QALYs at an average cost of $8306, while gland excision produced 8.94 QALYs at an average cost of $6103. The ICER for sialendoscopy was $36,717 per QALY gained, making sialendoscopy cost-effective by our best estimates. The model was sensitive to the probability of success and the cost of sialendoscopy. Sialendoscopy must meet a probability-of-success threshold of 0.61 (61%) and cost ≤$11,996 to remain cost-effective. A Monte Carlo simulation revealed sialendoscopy to be cost-effective 60% of the time. Conclusion Sialendoscopy appears to be a cost-effective management strategy for sialolithiasis of the submandibular gland when certain thresholds are maintained. Further studies elucidating the clinical factors that determine successful sialendoscopy may be aided by these thresholds as well as future comparisons of novel technology.


2021 ◽  
Vol 104 (5) ◽  
pp. 818-824

Background: Diabetic retinopathy (DR) causes blindness of the population in many countries worldwide. Early detection and treatment of this disease via a DR screening program is the best way to secure the vision. An annual screening program using pharmacological pupil dilatation becomes the standard method. Recently, non-mydriatic ultrawide-field fundus photography (UWF) has been proposed as a choice for DR screening. However, there was no cost-effectiveness study between the standard DR screening and this UWF approach. Objective: To compare the cost-effectiveness between UWF and pharmacological pupil dilatation in terms of hospital and societal perspectives. Materials and Methods: Patients with type 2 diabetes mellitus that visited the ophthalmology clinic at Chulabhorn Hospital for DR screening were randomized using simple randomization method. The patients were interviewed by a trained interviewer for general and economic information. The clinical characteristics of DR and staging were recorded. Direct medical costs, direct non-medical costs, and informal care costs due to DR screening were recorded. Cost analyses were calculated for the hospital and societal perspectives. Results: The present study presented the cost-effectiveness analyses of UWF versus pharmacological pupil dilatation. Cost-effectiveness analysis from the hospital perspective showed the incremental cost-effectiveness ratio (ICER) of UWF to be –13.87. UWF was a cost-effective mean in DR screening in the societal perspective when compared with pharmacologically pupil dilatation with the ICER of 76.46, under the threshold of willingness to pay. Conclusion: The UWF was a cost-effective mean in DR screening. It can reduce screening duration and bypass post-screening blurred vision. The results suggested that UWF could be a viable option for DR screening. Keywords: Diabetic retinopathy, Diabetic retinopathy screening, Non-mydriatic ultrawide-field fundus photography, Cost-effectiveness analysis


Immunotherapy ◽  
2021 ◽  
Author(s):  
Wei Jiang ◽  
Zhichao He ◽  
Tiantian Zhang ◽  
Chongchong Guo ◽  
Jianli Zhao ◽  
...  

Aim: To evaluate the cost–effectiveness of ribociclib plus fulvestrant versus fulvestrant in hormone receptor-positive/human EGF receptor 2-negative advanced breast cancer. Materials & methods: A three-state Markov model was developed to evaluate the costs and effectiveness over 10 years. Direct costs and utility values were obtained from previously published studies. We calculated incremental cost–effectiveness ratio to evaluate the cost–effectiveness at a willingness-to-pay threshold of $150,000 per additional quality-adjusted life year. Results: The incremental cost–effectiveness ratio was $1,073,526 per quality-adjusted life year of ribociclib plus fulvestrant versus fulvestrant. Conclusions: Ribociclib plus fulvestrant is not cost-effective versus fulvestrant in the treatment of advanced hormone receptor-positive/human EGF receptor 2-negative breast cancer. When ribociclib is at 10% of the full price, ribociclib plus fulvestrant could be cost-effective.


2020 ◽  
Vol 9 (8) ◽  
pp. 553-562
Author(s):  
Hongfu Cai ◽  
Longfeng Zhang ◽  
Na Li ◽  
Bin Zheng ◽  
Maobai Liu

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.


2018 ◽  
Vol 41 (2) ◽  
pp. 391-398
Author(s):  
Monica Teng ◽  
Hui Jun Zhou ◽  
Liang Lin ◽  
Pang Hung Lim ◽  
Doreen Yeo ◽  
...  

Abstract Background The study evaluated the cost-effectiveness of hydrotherapy versus land-based therapy in patients with musculoskeletal disorders (MSDs) in Singapore. Methods A decision-analytic model was constructed to compare the cost-effectiveness of hydrotherapy to land-based therapy over 3 months from societal perspective. Target population comprised patients with low back pain (LBP), osteoarthritis (OA), rheumatoid arthritis (RA), total hip replacement (THR) and total knee replacement (TKR). Subgroup analyses were carried out to determine the cost-effectiveness of hydrotherapy in individual MSDs. Relative treatment effects were obtained through a systematic review of published data. Results Compared to land-based therapy, hydrotherapy was associated with an incremental cost-effectiveness ratio (ICER) of SGD 27 471 per quality-adjusted life-year (QALY) gained, which was below the willingness-to-pay threshold of SGD 70 000 per QALY (one gross domestic product per capita in Singapore in 2015). For the respective MSDs, hydrotherapy were dominant (more effective and less costly) in THR and TKR, cost-effective for LBP and RA, and not cost-effective for OA. Treatment adherence and cost of hydrotherapy were key drivers to the ICER values. Conclusions Hydrotherapy was a cost-effective rehabilitation compared to land-based therapy for a population with MSDs in Singapore. However, the benefit of hydrotherapy was not observed in patients with OA.


2020 ◽  
Vol 30 (Supplement_5) ◽  
Author(s):  
M Fujita ◽  
S Kusumoto ◽  
M Sugiyama ◽  
T Fujisawa ◽  
M Mizokami ◽  
...  

Abstract Background There is no worldwide standard recommendation for preventing hepatitis B virus (HBV) reactivation for patients with resolved infection treated with an anti-CD20 antibody for B-cell non-Hodgkin lymphoma. This study aims to compare the cost-effectiveness between two commonly used strategies to prevent HBV reactivation-related death. Methods The two strategies compared were prophylactic antiviral therapy (Pro NAT) and HBV DNA monitoring followed by on-demand antiviral therapy (HBV DNA monitoring) using entecavir (Entecavir, a generic drug for Baraclude). Effectiveness was defined as the prevention of death due to HBV reactivation and costs were calculated under the health insurance system of Japan as of April 2018 using Markov model. A cost-minimization analysis, one of the cost-effectiveness analyses, was applied, since the effectiveness was the same between the two strategies according to a meta-analysis. To consider the effect of uncertainty for each parameter, probabilistic sensitivity analysis (PSA) was performed. In the scenario analysis, costs were calculated using lamivudine (Zefix) or tenofovir alafenamide (Vemlidy) instead of entecavir. All analyses were done using TreeAge Pro 2019 (TreeAge Software, Inc., MA, USA). Results Estimated costs per patient during the 30 months after initiation of chemotherapy for lymphoma were 1,513 USD with Pro NAT and 1,265 USD with HBV DNA monitoring. A PSA revealed that HBV DNA monitoring was more consistently cost-effective compared with Pro NAT when some parameters were set randomly according to probability distributions. In our scenario analysis, costs of Pro NAT and HBV DNA monitoring were calculated as 2,762 and 1,401 USD using lamivudine, 4,857 and 1,629 USD using tenofovir alafenamide. Conclusions Our cost-effectiveness analysis shows that an HBV DNA monitoring strategy using entecavir should be recommended for preventing HBV reactivation-related death in Japan. Key messages Cost-effectiveness analysis demonstrated that HBV DNA monitoring was more cost-effective compared to Pro NAT; this result was consistent with PSA. HBV DNA monitoring strategy should be recommended to prevent HBV reactivation-related death for the patients with resolved HBV infection in Japan.


2020 ◽  
Vol 3 ◽  
pp. 23
Author(s):  
James F. O'Mahony

Callender et al. recently published a model-based cost-effectiveness analysis of a risk-tailored approach to prostate cancer screening. It considers the costs and effects of prostate cancer screening offered to all men aged 55-69 without any risk selection and, alternatively, over a range of risk-tailored strategies in which screen eligibility is determined by a varying threshold of disease risk. The analysis finds that the strategy of screening men once they reach a 10-year absolute risk of disease of 5% or more is cost-effective in a UK context. I believe there are several problems with the study, mostly stemming from an incorrect interpretation of the cost-effectiveness estimates. I show that one reinterpretation of their results indicates that screening is much less cost-effective than the original analysis suggests, indicating that screening should be restricted to a much smaller group of higher risk men. More broadly, I explain the challenges of attempting to meaningfully reinterpret the originally published results due to the simulation of non-mutually exclusive intervention strategies. Finally, I consider the relevance of considering sufficient alternative screening intensities. This critique highlights the need for appropriate interpretation of cost-effectiveness results for policymakers, especially as risk stratification within screening becomes increasingly feasible.


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