CMS to Again Scrutinize ESAs

Rachel Shaffer
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The Centers for Medicare and Medicaid Services (CMS) will meet again on Jan. 19 to study “Erythropoiesis Stimulating Agents (ESAs) for Treatment of Anemia in Adults with CKD Including Patients on Dialysis and Patients not on Dialysis: The Impact of ESA Use on Renal Transplant Graft Survival.”As in all previous CMS meetings on ESA use, the American Society of Nephrology (ASN) will again present testimony to the Medicare Evidence Development and Coverage Advisory Committee (MedCAC) panel.

Medicare currently does not have a national coverage determination (NCD) for the use of ESAs for anemia in patients who have CKD. However, over the past year the agency has devoted increased attention to this issue. This month’s meeting is the third in a series of Medicare reviews of evidence for ESA use in kidney patients. The Food and Drug Administration (FDA) has also heightened scrutiny of ESAs in recent months (see Table 1).


In March 2010, MedCAC convened a meeting to review the available evidence on the use of ESAs to manage anemia in patients who have CKD. MedCAC’s advice can precipitate coverage changes. ASN Public Policy Board member Wolfgang Winkelmayer, MD, ScD, FASN, testified at the meeting that the available evidence shows that current ESAs may be dangerous if used for overly aggressive treatment targets compared with practices that are compatible with current treatment guidelines. Continued access to these medications is required to give patients with CKD a fair chance at receiving and then maintaining a kidney transplant, the society stated, and comparative effectiveness research that closes the evidence gap in the optimal role of ESAs is needed.

CMS did not issue any coverage changes following the March meeting, but in June 2010 initiated a national coverage analysis (NCA) examining evidence regarding the effects of ESAs on health outcomes in adult CKD patients, both pre-dialysis and on dialysis. The purpose of an NCA is to gather input from Medicare, experts, and the public that may influence changes to coverage. NCAs themselves do not change existing policy, but information collected during the course of an NCA could bring about an NCD, depending on the findings. ASN stressed in written testimony for the NCA that any decision the FDA makes regarding ESA treatment for anemia must differentiate among patients with CKD on dialysis and those not on dialysis. Such a decision should also protect patient access to necessary therapies, recognizing the variations in appropriate anemia care in a diverse patient population.

FDA considers ESAs

Meanwhile, the FDA also took a closer look at ESAs. Having instituted a “black box” warning for ESAs in 2009, citing greater risks for death, serious cardiovascular events, and stroke in some ESRD populations, FDA convened a meeting of its Cardiovascular and Renal Drugs Advisory Committee (CRDAC) to discuss the risks and benefits of ESAs in the treatment of anemia in patients with CKD based on the results from the recent Trial to Reduce Cardiovascular Events with Aranesp® Therapy (TREAT), and to potentially consider labeling changes for this patient population in October 2010. ASN again presented testimony, stating that TREAT outcomes support the current label, which is grounded in the best evidence currently available and has been adequate to support individualized treatment decisions among patients and their physicians.

So, what does all this increased attention mean for nephrologists and their patients? One of the primary rationales ASN and others have presented to CMS for preserving CKD patient access to ESAs is the drug’s effectiveness in preventing blood transfusions, since transfusions decrease patients’ likelihood of receiving or maintaining a transplant due to immune sensitization. On Jan. 19, CMS will take a closer look at the evidence available to support a portion of that position—whether or not ESAs affect transplant survival. The agency appears to be assembling evidence that might allow it to come to a conclusion on the effects and most appropriate use of ESAs for CKD patients, and is methodically narrowing its focus to subsets of that population.

Whether such a conclusion would alter existing labeling and coverage is unknown. For the time being no labeling or coverage changes for ESAs are imminent. The results of the January 2011 meeting will likely provide further insight into CMS’ next steps, and CMS is also expected to release a “Decision Memo” for the NCA on March 16, 2011. ASN will continue to advocate for nephrologists and their patients this coming January and in subsequent CMS and FDA examinations of ESAs and other renal care drugs.