Abstract
Background:
Tyrosine kinase inhibitors (TKIs) such as imatinib mesylate have revolutionized the treatment of primary unresectable and/or metastatic gastrointestinal stromal tumors (GISTs), providing durable disease control and extended survival. Although most patients eventually progress on therapy, dose escalation has been shown to benefit some patients. Sunitinib, a multitargeted kinase inhibitor is effective against imatinib-resistant or intolerant GIST patients. Although the cost of TKI therapy in GIST is high, no other effective systemic treatment options exist.
Objective:
Review pharmacoeconomic studies to determine the cost effectiveness (CE) of 1st- and 2nd-line TKI therapies in GIST.
Methods:
A literature review using Medline and PubMed databases was conducted to identify published economic analyses of TKI therapy in GIST. Key results from these studies were analyzed.
Results:
Six pharmacoeconomic studies were identified, including three analyses of 1st-line imatinib and three analyses of 2nd-line sunitinib. These studies employed various time horizons and discount rates and modeled CE from a number of different perspectives. Most of the pharmacoeconomic studies reviewed used survival as their efficacy endpoint, projecting outcomes beyond available data to model CE. Analyses of 2nd-line sunitinib using survival additionally faced the challenge of adjusting for the effect of placebo crossover to active treatment in the pivotal phase III study. Most studies used Markov techniques with a range of transition probabilities.
Conclusions:
Published pharmacoeconomic studies of 1st- and 2nd-line TKI therapy for advanced GIST employ various time horizons, discount rates, and different CE models. Consequently, these differences make comparisons between studies difficult. Studies of 1st-line imatinib concluded that imatinib was cost effective in advanced, metastatic GIST. Likewise, based on data reviewed here, 2nd-line sunitinib appears to be cost effective in patients with advanced GIST who are intolerant/resistant to imatinib. Key limitations of this review included inconsistency among the studies evaluated with regard to methodologies, countries of origination (currency and healthcare systems), and patient demographics.
Transparency
Declaration of funding
The authors have not received financial support for the research reported in this article.
Declaration of financial/other relationships
All authors have completed the disclosure declaration. C.D.B. is chief of the Division of Medical Oncology at the University of British Columbia and has been a consultant/advisor for both Novartis Pharmaceuticals and Pfizer Pharmaceuticals. D.M.H. is vice president of the healthcare division at Thomson Reuters and has no relationships to declare.
Acknowledgments
Financial support for medical editorial assistance was provided by Novartis Pharmaceuticals. The authors thank Angelo Russo, PhD, for his medical editorial assistance with this manuscript.
Notes
* Glivec is a registered trade name of Novartis Pharmaceuticals, Basel, Switzerland.
† Sutent, is a registered tradename of Pfizer, New York, NY, USA.
‡ Tasigna is a registered tradename of Novartis Pharmaceuticals, Basel, Switzerland.
§ Nexavar is a registered tradename of Bayer HealthCare Pharmaceuticals, Montville, NJ, USA.