Message to the FDA on ESAs: REMS Is Not Enough, More Studies Are Needed

Since March 24, 2010, the Food and Drug Administration (FDA) has been requiring a “Risk Evaluation and Mitigation Strategy” (REMS) for all patients who are treated with erythropoiesis-stimulating agents (ESAs) (1). Although regulatory authorities elsewhere in the world have not as yet taken this approach, experience suggests that these agencies often take their cues from the FDA.

Most nephrologists are unaware that the FDA now requires distribution to both dialysis and nondialysis patients of a medication guide that explains the risks and benefits of ESAs. The FDA is also mandating that oncologists—but not nephrologists, at least thus far—register and participate in a program called Assisting Providers and Cancer Patients with Risk Information for the Safe Use of ESAs (APPRISE) (2); however, APPRISE for ESAs in cancer could herald a similar program for ESAs in the anemia of kidney disease. What is REMS? Should nephrologists be required to register in an APPRISE-like program? Should the FDA be doing more to address safety concerns with ESAs?

REMS was announced in February for all patients who receive ESAs, and notices have been sent to ESRD networks asking that medication guides be distributed to patients (3). In addition, for the ESA cancer indication, oncologists need to undergo training on the risks and benefits of ESAs to continue prescribing them. This involves discussion about ESAs with patients who have cancer before beginning a course of treatment. Oncologists have to document that this discussion took place. A health care provider enrollment form that was developed by Amgen and Centocor Ortho Biotech and approved by the FDA must be completed for oncologists to prescribe ESAs (4).

The FDA gained authority to require REMS under the Food and Drug Administration Amendments Act (FDAAA) of 2007 (5). The goal of REMS is to manage a known or potentially …