Economic evaluations of oral medications for breast cancer treatment in the U.S.: a systematic review with a focus on cost-effectiveness threshold

ABSTRACT

Introduction: With the advent of targeted therapy, the U.S. Food and Drug Administration has recently approved several oral anticancer medications (OAMs) for breast cancer (BC). Despite the improved effectiveness of those OAMs, the high financial burden is an issue. Evidence from cost-effectiveness analysis (CEA) can provide valuable information for decision-makers when deciding whether to use these high-priced medications. Many CEAs on OAMs have been conducted using various analytical approaches and cost-effectiveness thresholds (CETs). However, there is no comprehensive systematic review of CEAs across all OAMs.

Area covered: PubMed and Cochrane library were used to select for CEAs of OAM for BC in the U.S. published by May 2019. Among the 25 included studies, studies published between 1993 and 2011 analyzed either early BC (n = 11) or advanced/metastatic BC (n = 5), those between 2012–2019 analyzed advanced/metastatic BC (n = 9). Studies including targeted therapies were published after 2009. The CETs tended to increase over time and were higher in the studies for advanced/metastatic BC (median = $125,000) than those for early BC (median = $50,000).

Expert commentary: The target population and medications of interest have changed and the methods of articles have evolved. The range of CETs tends to differ by study setting with an increase over time.

KEYWORDS:

• Breast cancer
• cost-effectiveness
• oral anticancer medication
• systematic review: cost-effectiveness threshold
• targeted therapy

Article Highlights

• With the advent of targeted therapy, the U.S. Food and Drug Administration has recently approved several oral anticancer medications (OAMs) for breast cancer. Because of a financial issue for newly approved OAMs, evidence from cost-effectiveness analysis (CEA) can be useful for decision-makers when deciding whether to use these high-priced medications. Many CEAs on OAMs have been conducted using various analytical approaches and cost-effectiveness (CE) thresholds, which are the criterion for the conclusion of CE. However, no study has comprehensively reviewed CEAs across all OAMs and examined how different CE thresholds were used to elicit conclusions in each CEA.

• As the targeted population changed from early breast cancer to advanced/metastatic breast cancer, the characteristics of analytical framework also evolved. A wide range of values for the CE threshold was used in the CEA studies, although the role of threshold is important in determining CE. Moreover, the range of CE threshold tends to differ by cancer status (early or advanced/metastatic) with an increase over time.

• The overview of study characteristics summarized in this review would be helpful to conduct further research using generally accepted analytical approaches. In addition, the summarized CE threshold information would be useful and meaningful to provide a consistent and appropriate standard to conclude decisions on the CE of anticancer medications.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Supplementary material

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Additional information

Funding

This paper was not funded.