The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) have directed the care of patients with cancer for >20 years.1 In recent years, these guidelines have been further refined to include a category of “preferred” guideline treatment regimens to specify recommendations.2 Previous evaluations of NCCN Guidelines have focused on the use of specific, individual medications considered off-label or nonconcordant. In breast cancer, >10% of patients receive a medication not listed within the guidelines.3,4 These studies likely underestimate true levels of discordance because they do not capture concordance for regimens, which include specific single agents or combinations of hormone therapy, chemotherapy, and targeted therapies.
Use of nonconcordant treatments is known to contribute to the increasing cost of cancer care.5 The top 10 nonconcordant chemotherapy agents contribute to an estimated $2.5 billion in US cancer spending.6 Patient factors are known to contribute to nonconcordant care,4 but the role of provider factors is less clear. In an effort to minimize provider-based treatment variability, payers and health systems are implementing pathway programs restricting reimbursement for off-pathway regimens.7–10 Although these programs are showing early promise, few data exist about the outcomes for nonconcordant treatment regimens, particularly in metastatic breast cancer (MBC), in which treatment is heterogeneous and complex due to the large number of available guideline-based options. To ensure optimal patient care, it is essential to understand the types of commonly prescribed nonconcordant treatments and their impact on mortality, total Medicare costs, and rates of healthcare utilization.
The objectives of this study were to examine regimens concordant with NCCN Guidelines for MBC; describe categories of nonconcordant treatments; evaluate patient- and provider-level factors associated with nonconcordance; and determine the impact of nonconcordance on mortality, Medicare spending, and rates of healthcare utilization.
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