Targeted oral anticancer medications (TOAMs) have been the focus of oncology drug development in the past two decades. Although these novel agents contribute to survival improvement in several cancers and are convenient to administer, high drug prices have raised concerns on their affordability and possibly low treatment adherence driven by high out-of-pocket expenses. Current knowledge on the utilization and adherence of TOAMs is largely built upon studies of patients with chronic myelogenous leukemia. It remains unclear whether such knowledge is directly transferrable to other cancers, especially those with both oral and intravenous anticancer medications as viable treatment options.
The specific aims of the study are: (1) to examine the utilization patterns of TOAMs as indicated vs. off-label use; (2) to estimate the impact of TOAM use on the overall costs of cancer care, costs of non-hormonal systemic therapies, as well as out-of-pocket payment; (3) to investigate the factors associated with the initiation of a TOAM and time to treatment initiation; and (4) to explore patterns of treatment switches and discontinuation and the associated factors among patients who started their systemic therapy with a TOAM. We will address our research questions using various cohorts of cancer patients extracted from three large claims databases: the SEER-Medicare, MarketScan?, and Health Care Cost Institution data. We will conduct a series of studies to better understand the role of TOAMs in modern cancer care delivery by systematically studying the utilization pattern and economic impact of TOAMs as well as factors affecting the initiation and discontinuation of these novel therapies, paying special attention to financial factors for patients and providers. The findings from our study will inform patients, clinicians, and policy makers of the extent to which off-label prescription of TOAM is practiced in the real-world and the long-term economic impact of TOAM use, and provide critical information to guide reform or re-design of insurance policies to avoid creating financial disincentive to prescribe or use TOAM. Ultimately, the goal of our research is to improve the accessibility, affordability, effectiveness, and safety of TOAMs.

Public Health Relevance

STATEMENT Scientific advances have made it possible for patients to receive cancer drugs orally. Although convenient to administer, most oral chemotherapy drugs are expensive and patients need to continue taking these drugs as long as their cancers do not progress. This research studies the utilization pattern of oral chemotherapy in common and rare cancers, estimates the long-term economic impact of continuously taking oral chemotherapy drugs, and examines whether some patients may decide not to use these drugs at all or to discontinue taking them because of high out-of-pocket costs.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Research Project (R01)
Project #
Application #
Study Section
Dissemination and Implementation Research in Health Study Section (DIRH)
Program Officer
Castro, Kathleen M
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
University of Texas MD Anderson Cancer Center
United States
Zip Code