By Samuel M. Silver, MD, PhD, FACP

2008-05-01

Dr. Silver is Professor of Medicine in the Division of Hematology/Oncology and Assistant Medical Director of the Faculty Group Practice at the University of Michigan. He is also Chair of ASH's Reimbursement Subcommittee.

Dr. Silver consults for Gerson Lehrman Group, Lehman Brothers, and BlueCross BlueShield of MI.

Editor's Note: Dr. Silver presented testimony to the FDA on behalf of ASH.

On March 13, the U.S. Food & Drug Administration's (FDA) Oncologic Drugs Advisory Committee (ODAC) met to review the cumulative data on the risks of erythropoiesis-stimulating agents (ESAs) when administered to patients with cancer. The advisory committee recommended to continue the ESA indication for treatment of chemotherapy-induced anemia; however, the panel also recommended several changes to the safety labels on the drugs Aranesp, Epogen, Procrit, and Mircera. These changes will place significant restrictions on their use for patients with cancer.

By a vote of 13 to 1, ODAC recommended that ESAs continue to be marketed for use for treatment of chemotherapy-induced anemia in patients with cancer. While the panel rejected a proposal to modify the current indication to restrict use only to patients with small-cell lung cancer, it supported recommendations to amend the label and include statements that ESA usage is not indicated for patients receiving "potentially curative treatments," patients with head and neck cancer, or patients with metastatic disease of the breast. The panel did not reach consensus on when to initiate ESA therapy. The panel also supported requiring implementation of an informed consent/patient agreement for the treatment of anemia in these patients, but the panel rejected a recommendation calling for the FDA to mandate a restricted distribution system for oncology patients receiving ESAs.

As this issue of The Hematologist was going to press, it was unclear what the impact of the ODAC meeting will be. ODAC's recommendations are non-binding. The FDA will review the recommendations and make its own decision on how to revise the drug labeling. Specifically, it is not clear what ODAC's recommendations would mean for treating patients with hematologic malignancies. For example, if adopted, does ODAC's recommendation to exclude potentially curative treatments mean that ESAs could only be used for palliative care? Would limited-stage, low-grade lymphoma be considered potentially curable? Most significantly, perhaps, at this time it is unclear how the Centers for Medicare & Medicaid Services may interpret this and whether Medicare's current coverage policy may be altered as a result. In addition, ASH will continue to work with local Medicare carriers to ensure their policies regarding coverage for patients with MDS are not adversely affected by ODAC's recommendations.

I testified before the advocacy panel on behalf of ASH and the American Society of Clinical Oncology (ASCO) regarding the safety and appropriate use of ESAs. The testimony emphasized that pending the publication of more definitive and peer-reviewed data on safety signals in the target population of the ASH-ASCO guideline, our organizations do not see sufficient evidence of harm to support recommending complete cessation of the use of ESAs across all patients with malignancies. Further, I reminded the advisory committee that there is compelling evidence to support safe use of ESAs in anemic patients with low-risk myelodysplasia. The testimony also addressed the need to better inform patients about the risks and benefits of ESA therapy and that additional studies are necessary to address lingering safety questions.

ASH will continue to meet with the FDA and Congress about its concerns regarding the impact these potential restrictions could have on patients with hematologic diseases and will keep the membership apprised of all developments.

back to top