Abstract
Background
The incidence of hepatocellular carcinoma (HCC) is increasing in the USA and worldwide. Several treatments are available for patients diagnosed at any disease stage. It remains unclear how medical expenditures vary across patients who remain untreated or undergo different modes of therapy. We evaluate the comparative and cost effectiveness of treatment modalities for HCC from a Medicare perspective.
Methods
The Surveillance, Epidemiology, and End Results (SEER) registries and linked Medicare database with claims from Parts A/B were used to identify Medicare enrollees with initial diagnosis of HCC between 2000 and 2007 and followed through 2009. Patients were assigned to treatment modalities based on HCC staging systems: transplant, resection, liver directed, radiation, chemotherapy or no treatment. Survival benefits and cumulative Medicare expenditures were estimated in multivariate models, stratified by initial disease stage, to control for confounding. Cost-effectiveness ratios compared costs and benefits of the modalities across initial stages.
Results
Cancer stages I, II, III, IV and unstaged represented 24, 9, 14, 17 and 37 % of 11,047 patients, respectively. Fewer than 40 % received any treatment. Relative to no treatment, transplant was most effective in reducing mortality, followed by resection, liver directed, and radiation or chemotherapy. Resection tended to be most cost effective in early staged and unstaged patients; transplant was least cost effective. In stage IV patients, liver directed therapy was more cost effective than chemotherapy or radiation.
Conclusions
Survival benefit was attributable to all treatment modalities. More effective treatments incurred greater Medicare expenditures, but resection patients incurred the least expenditures per year of life gained.
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Notes
Estimates may be somewhat biased if unidentified sorafenib use was systematically distributed across treatment modalities or no treatment within stages, and it had a significant impact on survival and costs. However, such bias is likely to be minimal given that sorafenib was approved for use by the FDA in December 2005 and our observation period spans the period from 2000 to 2009.
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Acknowledgments
The authors would like to acknowledge the staff of Pharmaceutical Research Computing (PRC), University of Maryland Baltimore for their analytical and programming support. A list of the current staff is available on the PRC web site at http://www.pharmacy.umaryland.edu/PRC/staff.htm. This work was sponsored by Bayer HealthCare Pharmaceuticals, Inc., Wayne, NJ, USA. While not contingent on the sponsor’s approval or censorship of any of its contents, the sponsor did approve submission of this work for publication. Dr. Brian Seal is Director at the Division of Health Economics and Outcomes Research at Bayer HealthCare Pharmaceuticals, Inc. He oversaw the satisfactory completion of the research grant funding the project. Dr. C. Daniel Mullins receives grant support from Bayer and Pfizer; consulting income from Bayer, Amgen, BMS, Janssen/J&J, Novartis and Pfizer; and payment for lectures from Celgene. Drs Fadia Shaya, Ian Breunig, C. Daniel Mullins and Nader Hanna, and Viktor Chirikov, have no financial interests to disclose directly or indirectly related to the research in the manuscript. Dr. Shaya coordinated and led the conceptual design of the overall study, contributed to the design of the statistical models, interpretation of the results, and writing of the manuscript, and acts as overall guarantor. Dr. Breunig led the writing of the manuscript, contributed to the conceptual design, conducted all statistical programming, and led the design of the statistical models and interpretation of the results. Drs Seal, Mullins and Hanna, and Viktor Chirikov, contributed to the conceptual design, the design of statistical models, and interpretation of results. Dr. Mullins and Viktor Chirikov also contributed to the writing of the manuscript.
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Shaya, F.T., Breunig, I.M., Seal, B. et al. Comparative and Cost Effectiveness of Treatment Modalities for Hepatocellular Carcinoma in SEER-Medicare. PharmacoEconomics 32, 63–74 (2014). https://doi.org/10.1007/s40273-013-0109-7
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DOI: https://doi.org/10.1007/s40273-013-0109-7