ODAC Recommends ESAs Continue to Be Indicated for Treatment of Chemotherapy-Induced Anemia but With New Restrictions

March 13, 2008 – The Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) recommended to continue the erythropoiesis-stimulating agent (ESA) indication for treatment of chemotherapy-induced anemia in patients with cancer; however, the panel also recommended several changes to the safety labels on the drugs (Aranesp and Procrit) that will place significant restrictions on their use for cancer patients.

By a vote of 13 to 1, ODAC recommended that ESAs continue to be marketed for ESA 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 and for patients with metastatic disease of the breast and/or head and neck. 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 for these patients, but the panel rejected a recommendation calling for FDA to mandate a restricted distribution system for oncology patients receiving ESAs.

Dr. Samuel Silver presented the views 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, Dr. Silver 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.

The "ASH/ASCO 2007 Clinical Practice Guideline Update on the Use of Epoetin and Darbepoetin" can be found on the Blood site.

OUTLOOK: ODAC’s recommendations are non-binding. The FDA will review ODAC’s recommendations and make a decision on how to revise the drug labeling. ASH will continue to meet with FDA and Congress about its concerns on the impact these potential restrictions could have on patients with hematologic diseases. Specifically, it is not clear what ODAC’s recommendations would mean for treating patients with stage IV lymphoma. In addition, while ODAC’s recommendations did not address ESA therapy for patients with myelodypslasia, ASH will continue to work to ensure that insurers allow coverage. ASH will keep the practice community apprised of all developments.