The controversial new Medicare policy restricting the use of erythropoiesis-stimulating agents (ESAs)—Medicare's single largest drug expense—continues to stir intense debate. Unless the Centers for Medicare and Medicaid Services responds to the oncology community's pleas to change the current policy, the only alternative may be legislative intervention.

Prompted by safety concerns, FDA and CMS have joined forces in the battle over the "proper" use of ESAs in oncology. The two agencies contend that the new CMS national coverage decision (NCD) is backed by sound scientific data, while the oncology community argues that the policy is too restrictive and stems as much from economic concerns as from concerns about patient safety.

Fearing a return to the "dark ages" when transfusions were the primary option for treating anemic cancer patients, oncology leaders are committed to reversing the NCD, Joseph S. Bailes, MD, chair of ASCO's Government Relations Council, told Oncology NEWS International.

"The new restrictions are inconsistent with both FDA-approved labeling and national guidelines, and we strongly disagree with this restriction," Dr. Bailes said. Although the new Medicare coverage determination has attracted congressional attention, Dr. Bailes conceded that given the current political climate, reversing the NCD will be a long uphill battle.

Safety concerns escalate

Safety concerns about ESAs were first raised in 2003 by two studies that some experts say were flawed and unconvincing. Since then, subsequent trials have produced equally debatable conclusions.

On March 9, 2007, based largely on the results of a phase III trial called the "103 study" and some preliminary data from a Danish trial called DAHANCA 10, FDA added a new "black box" warning to the package inserts of ESAs marketed in the United States. The new label recommended using the lowest ESA dose necessary to avoid the need for blood transfusions.

The warning label prompted the new CMS directive: In cancer patients with chemotherapy-related anemia, ESAs cannot be used until the patient's hemoglobin level falls below 10 g/dL and must be stopped if, after 4 weeks of therapy, the hemoglobin level rises to 10 g/dL or higher. Starting January 1, 2008, CMS will require physicians to submit data on a patient's hemoglobin levels along with any claim for reimbursement of ESAs. However, CMS expects physicians to institute the new regulations immediately.