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.
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