Background: Medicare and third-party payers may be reluctant to pay for investigational (protocol) therapy for patients with cancer on the premise that such treatment is more expensive than standard therapy. However, prior studies that have attempted to compare protocol therapy with standard therapy have been difficult to interpret because of the assortment of malignancies studied and the lack of suitable control groups of patients who received standard therapy.
Methods: In the current study, the authors conducted a retrospective review of the financial charges associated with protocol or nonprotocol (standard) chemotherapy in patients with a single malignancy, newly diagnosed acute myelogenous leukemia (AML), who received their initial course of chemotherapy ("induction") at the Memorial Sloan-Kettering Cancer Center (MSKCC) between 1996 and 1999. Protocol and nonprotocol groups were analyzed according to clinical characteristics and standard prognostic features to determine whether the two groups were comparable. Median charges for all patients were determined using a database that linked clinical information, financial data, and clinical outcomes.
Results: A total of 353 patients with newly diagnosed AML were registered at MSKCC during the time period studied; of these, 79 patients (22%) received all of their care at the institution. Thirty patients (38%) received treatment on an investigational protocol. Forty-nine patients (62%) did not receive protocol therapy for the following reasons: 10 patients (20%) did not meet eligibility criteria, 4 patients (8%) were eligible for protocol therapy but declined, and 35 patients (71%) met protocol criteria but were not offered protocol therapy based on the judgment of their primary oncologist. The groups were not comparable because patients treated with standard therapy were older and had a poorer initial Eastern Cooperative Oncology Group (ECOG) performance status. Overall median charges for patients in the nonprotocol group were higher than for patients treated on a protocol although charges were not related to age, initial ECOG performance status, or cytogenetic risk group.
Conclusions: Although charges for the nonprotocol group were higher, specific factors responsible for this difference were not identified. This study emphasizes the problems inherent in assembling suitable groups of patients for comparison.
Copyright 2002 American Cancer Society.