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Article: Cost-effectiveness of Pembrolizumab as a Second-Line Therapy for Hepatocellular Carcinoma
Title | Cost-effectiveness of Pembrolizumab as a Second-Line Therapy for Hepatocellular Carcinoma |
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Authors | |
Issue Date | 2021 |
Publisher | American Medical Association: JAMA Network Open. The Journal's web site is located at https://jamanetwork.com/journals/jamanetworkopen |
Citation | JAMA Network Open, 2021, v. 4 n. 1, article no. e2033761 How to Cite? |
Abstract | Importance Immune checkpoint inhibitors have been approved for use as a second-line therapy for hepatocellular carcinoma (HCC) in patients who previously received sorafenib. Pembrolizumab has shown substantial antitumor activity and a favorable toxicity profile as a second-line treatment of HCC. However, considering the high cost of pembrolizumab, there is a need to assess its value by considering both the clinical efficacy and cost.
Objective To evaluate the cost-effectiveness of pembrolizumab vs placebo as second-line therapy in patients with HCC from the US payer perspective.
Design, Setting, and Participants A Markov model was developed to compare the lifetime cost and efficacy of pembrolizumab as a second-line treatment of HCC with those of placebo using outcome data from the KEYNOTE-240 randomized placebo-controlled trial, which included 413 patients with advanced HCC previously treated with sorafenib and randomized patients to receive pembrolizumab plus best supportive care or placebo plus best supportive care in a 2:1 ratio.
Main Outcomes and Measures Life-years, quality-adjusted life-years (QALYs), lifetime costs, and incremental cost-effectiveness ratio (ICER) were estimated at a willingness-to-pay threshold of $150 000 per QALY. One-way and probabilistic sensitivity analyses were performed to account for the parameter of uncertainty. A cost-threshold analysis was also performed. The study was conducted from January 31 to July 29, 2020.
Results The base-case model found that treatment with pembrolizumab was associated with increased overall cost by $47 057 and improved effectiveness by 0.138 QALYs compared with placebo, leading to an ICER of $340 409 per QALY. The model was most sensitive to the hazard ratio of overall survival (range, 0.61-1.00), health utility of placebo (range, 0.59-0.93), price of pembrolizumab (range, $5531-$8297), and price of postprogression therapies (range, $5596-$7944 for pembrolizumab and $4770-$7156 for placebo). The ICER of pembrolizumab was larger than $150 000 per QALY in most of the sensitivity and subgroup analyses. The price of pembrolizumab needed to be reduced by 57.7% to $2925 per cycle to achieve cost-effectiveness.
Conclusions and Relevance The findings of this cost-effectiveness analysis suggest that, at its current price, pembrolizumab is not a cost-effective second-line therapy for HCC in the US, with a willingness-to-pay threshold of $150 000 per QALY. |
Persistent Identifier | http://hdl.handle.net/10722/302116 |
ISSN | 2023 Impact Factor: 10.5 2023 SCImago Journal Rankings: 3.478 |
PubMed Central ID | |
ISI Accession Number ID |
DC Field | Value | Language |
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dc.contributor.author | Chiang, CL | - |
dc.contributor.author | Chan, SK | - |
dc.contributor.author | Lee, SF | - |
dc.contributor.author | Wong, IOL | - |
dc.contributor.author | Choi, HCW | - |
dc.date.accessioned | 2021-08-21T03:31:48Z | - |
dc.date.available | 2021-08-21T03:31:48Z | - |
dc.date.issued | 2021 | - |
dc.identifier.citation | JAMA Network Open, 2021, v. 4 n. 1, article no. e2033761 | - |
dc.identifier.issn | 2574-3805 | - |
dc.identifier.uri | http://hdl.handle.net/10722/302116 | - |
dc.description.abstract | Importance Immune checkpoint inhibitors have been approved for use as a second-line therapy for hepatocellular carcinoma (HCC) in patients who previously received sorafenib. Pembrolizumab has shown substantial antitumor activity and a favorable toxicity profile as a second-line treatment of HCC. However, considering the high cost of pembrolizumab, there is a need to assess its value by considering both the clinical efficacy and cost. Objective To evaluate the cost-effectiveness of pembrolizumab vs placebo as second-line therapy in patients with HCC from the US payer perspective. Design, Setting, and Participants A Markov model was developed to compare the lifetime cost and efficacy of pembrolizumab as a second-line treatment of HCC with those of placebo using outcome data from the KEYNOTE-240 randomized placebo-controlled trial, which included 413 patients with advanced HCC previously treated with sorafenib and randomized patients to receive pembrolizumab plus best supportive care or placebo plus best supportive care in a 2:1 ratio. Main Outcomes and Measures Life-years, quality-adjusted life-years (QALYs), lifetime costs, and incremental cost-effectiveness ratio (ICER) were estimated at a willingness-to-pay threshold of $150 000 per QALY. One-way and probabilistic sensitivity analyses were performed to account for the parameter of uncertainty. A cost-threshold analysis was also performed. The study was conducted from January 31 to July 29, 2020. Results The base-case model found that treatment with pembrolizumab was associated with increased overall cost by $47 057 and improved effectiveness by 0.138 QALYs compared with placebo, leading to an ICER of $340 409 per QALY. The model was most sensitive to the hazard ratio of overall survival (range, 0.61-1.00), health utility of placebo (range, 0.59-0.93), price of pembrolizumab (range, $5531-$8297), and price of postprogression therapies (range, $5596-$7944 for pembrolizumab and $4770-$7156 for placebo). The ICER of pembrolizumab was larger than $150 000 per QALY in most of the sensitivity and subgroup analyses. The price of pembrolizumab needed to be reduced by 57.7% to $2925 per cycle to achieve cost-effectiveness. Conclusions and Relevance The findings of this cost-effectiveness analysis suggest that, at its current price, pembrolizumab is not a cost-effective second-line therapy for HCC in the US, with a willingness-to-pay threshold of $150 000 per QALY. | - |
dc.language | eng | - |
dc.publisher | American Medical Association: JAMA Network Open. The Journal's web site is located at https://jamanetwork.com/journals/jamanetworkopen | - |
dc.relation.ispartof | JAMA Network Open | - |
dc.rights | This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License. | - |
dc.title | Cost-effectiveness of Pembrolizumab as a Second-Line Therapy for Hepatocellular Carcinoma | - |
dc.type | Article | - |
dc.identifier.email | Chiang, CL: chiangcl@hku.hk | - |
dc.identifier.email | Choi, HCW: hcchoi@hku.hk | - |
dc.identifier.authority | Chiang, CL=rp02241 | - |
dc.identifier.authority | Choi, HCW=rp02815 | - |
dc.description.nature | published_or_final_version | - |
dc.identifier.doi | 10.1001/jamanetworkopen.2020.33761 | - |
dc.identifier.pmid | 33464318 | - |
dc.identifier.pmcid | PMC7816108 | - |
dc.identifier.scopus | eid_2-s2.0-85100226360 | - |
dc.identifier.hkuros | 324351 | - |
dc.identifier.volume | 4 | - |
dc.identifier.issue | 1 | - |
dc.identifier.spage | article no. e2033761 | - |
dc.identifier.epage | article no. e2033761 | - |
dc.identifier.isi | WOS:000612967700003 | - |
dc.publisher.place | United States | - |