Not enough evidence exists to say whether using erythropoiesis-stimulating agents (ESAs) for anemia management improves transplant graft survival, according to findings from a Centers for Medicare and Medicaid Services (CMS) Medicare Evidence Development and Coverage Advisory Committee (MedCAC) .
At a January meeting CMS asked MedCAC panelists to rate not only the evidence regarding the effect of ESAs on transplant survival but also the evidence regarding pretransplant donor-specific transfusions on graft survival. The meeting was titled “The Impact of ESA Use on Renal Transplant Graft Survival.”
“This is just a reminder how many data there aren’t,” said MedCAC Panel Chair Clifford Goodman, PhD.
The ASN was one of a handful of professional societies and patient organizations selected to present testimony at the meeting. Public Policy Board member William E. Harmon, MD, a noted transplant nephrologist, spoke on the society’s behalf.
In his comments, Harmon focused on the well demonstrated utility of ESAs for the treatment of anemia, emphasizing their ability to prevent blood transfusions that would likely otherwise be necessary—transfusions that place patients at risk for sensitization because of exposure to foreign human antigens. Sensitization is associated with a longer time on the wait list, faster and higher rates of rejection, delayed graft function, and longer term complications. Patients who are highly sensitized often never receive an organ because no compatible graft becomes available.
National Coverage Analysis and National Coverage Decision 101
The purpose of a National Coverage Analysis (NCA) is to gather input and information from CMS staff, independent experts, and the public about a certain item or service for which Medicare provides reimbursement.
MedCAC panels are one component of the NCA evidence-gathering and analysis effort. NCAs themselves do not necessarily bring about changes to existing policy, but information collected during the course of an NCA can help bring about a National Coverage Decision (NCD), depending on the findings. An NCD is the official ruling that grants, limits, or excludes coverage.
Currently, no NCD exists for ESAs. However, anyone can request that the CMS initiate the NCD process, which typically involves an NCA. The CMS received a formal request for a national coverage determination for recombinant human erythropoietin for the treatment of CKD and dialysis-related anemia in March 2010.
Owing to the development of modern immunosuppressive therapies and tests that identify specific antigens to which a transplant recipient is sensitized, donor-specific transfusion protocols have not been used for more than 20 years. Consequently, Harmon stated, “the balance of data suggests that it is most appropriate to avoid sensitization prior to or subsequent to kidney transplantation.”
Besides hearing testimony from public speakers, including Harmon, and from CMS staff, MedCAC relied heavily on a Technical Assessment prepared by a contracted team. The CMS asked the Technical Assessment team to study and present a review of the available evidence on the effect of donor-specific transfusions on renal graft outcomes, but notably did not ask the team to address the effect of ESAs on the same outcomes.
The CMS convened the MedCAC meeting as part of an ongoing National Coverage Analysis (NCA) examining evidence regarding the effects of ESAs on health outcomes for patients with chronic kidney disease, both those receiving and those not receiving dialysis (see timeline). The CMS is expected to issue a final memo on the NCA this month, which could lead to a change in existing ESA coverage policy—a so-called National Coverage Determination (NCD) (see sidebar).
MedCAC is an independent advisory committee created to help CMS weigh clinical questions related to the safety and efficacy of medications and treatments. While not binding, MedCAC’s recommendations often factor heavily into the CMS’s decision-making regarding NCDs.
Fourteen panelists participated in the MedCAC meeting, including an industry representative and a liaison from CMS. CMS selected 12 of these panelists from a MedCAC pool of approximately 90 individuals with expertise in a diversity of medical specialties, and invited two participants with expertise in nephrology. Consequently, the majority of panelists were not nephrologists.
Given the complexity of the questions the CMS asked the panel to consider, the MedCAC panel could have benefited from additional nephrology expertise, Harmon said. “While the panel did have the representation of one nephrologist, additional representation might have helped to frame the scope of evidence available on ESAs, putting the immediate questions regarding ESAs and graft survival in the proper context,” Harmon said.
After the January meeting, CMS requested public comment on the testimony heard at the MedCAC meeting as well as on the Technical Assessment.
The ASN responded to this request and followed up Harmon’s testimony with a written letter to CMS reiterating that ESAs are a cornerstone of chronic kidney disease care—specifically for anemia management—and have been proved effective for that purpose. Because ESAs are not indicated for the purpose of improving renal transplant graft survival, the ASN urged CMS not to issue an NCD or make any other changes to existing policies based on considerations of the evidence of the effect of ESAs on renal transplant graft survival.
The ASN continues to provide evidence-based information about ESAs and kidney patients and to advocate for policies that promote the highest quality of care as the CMS progresses in its deliberations regarding the NCA on ESAs.
To read Harmon’s testimony or ASN’s commentary on the Technical Assessment, or to learn more about advocacy efforts related to patient care, please visit ASN Policy online at www.asn-online.org/policy_and_public_affairs.
