CMS declares no change in payment policies for ESAs
This article was originally published in Scrip
In issuing its final decree, the US Centers for Medicare & Medicaid Services (CMS) stuck with the preliminary decision it issued in March to not impose payment restrictions on erythropoiesis stimulating agents (ESAs), like Amgen's Epogen (epoetin alfa) and Aranesp (darbepoetin alfa) and Centocor Ortho Biotech's Procrit's (epoetin alfa), for use in managing anemia in dialysis or non-dialysis chronic kidney disease (CKD) patients.
In a decision memo issued late on 16 June, CMS said that given the totality of the currently available evidence, the agency decided not to issue a national coverage determination at this time for ESAs – the same conclusion it came to in March (scripintelligence, 18 March 2011).
"It appears that Medicare is basically turfing the entire debate," said ISI Group analyst Dr Mark Schoenebaum.
While the decision was expected by Wall Street, it was "nonetheless, positive, in our view," said RW Barid analyst Christopher Raymond.
"While we anticipated as much, we did detect a certain amount of anxiety from investors related to this event. Therefore, tonight's final decision from CMS removes any such overhang, however minor," Mr Raymond said in a research report.
CMS currently does not have a national payment policy for the use of ESAs to treat anemia in CKD patients.
Medicare historically has made payments for ESAs for particular indications with specific conditions, with those payment decisions made through regional offices.
In March 2010, a CMS advisory panel concluded that the available evidence backed the use of ESAs as therapies to manage anemia in dialysis patients with chronic ESRD, but the panelists raised some concerns about the use of the medications in predialysis kidney disease patients. The Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) also said more clinical trials were needed to determine the proper dosing levels and whether hemoglobin was an appropriate target.
In addition, the MEDCAC said the available evidence demonstrated that ESAs reduced the need for risky blood transfusions and improved fatigue symptoms.
A second MEDCAC convened in January said evidence was insufficient to determine whether the use of ESAs improves the survival of adults with CKD with anemia who undergo renal transplants (scripintelligence, 24 January 2011).
In July 2007, CMS limited Medicare payments for ESA use in cancer patients after the US FDA imposed a black-box warning on the drugs' labeling alerting prescribers and patients about the risks of shortened overall survival and increased tumor progression or recurrence in cancer patients treated with the products.
While CMS made no change from its preliminary decision in March, "another shoe is yet to drop," Mr Raymond pointed out.
Indeed, Amgen in April indicated that the firm expected the US FDA to impose another ESA labeling change within the coming weeks.
"We believe this is likely imminent, but in our view, already well anticipated by the Street," Mr Raymond said.
But Deutsche Bank analyst Robyn Karnauskas said she was uncertain the FDA would actually act on a labeling change.
"We believe the key issue is impact to current dosing regimens," she said.
Ms Karnauskas pointed out that over the year-long coverage analysis, CMS commented numerous times about the totality of current available evidence and their intent to improve the body of evidence in order to improve decision-making.
"Based on our checks and the FDA having the same data set, we feel comfortable that a major change to the label is unlikely at this point," she said.
Last October, the FDA convened its Cardiovascular and Renal Drugs Advisory Committee to discuss the results of TREAT, a large, multicentre, randomized, controlled, double-blind trial of Aranesp in nondialysis CKD patients with Type II diabetes, which found that patients given the ESA were twice as likely to experience a stroke vs. those who got a placebo.
But the committee voted 15 to 1, with 1 abstention, that ESAs should continue to be used to treat anemia in patients with CKD not on dialysis (scripintelligence, 19 October 2011).
The panel also voted 9 to 5, with three abstentions, against changing Aranesp's labeling to indicate use of the ESA in nondialysis CKD patients when their hemoglobin concentration levels drop below 9 g/dL, discontinuing use in intervals when hemoglobin increases to 9 g/dL or greater. The panel also voted 13 to 2, with two abstentions, against making the same hemoglobin target changes for dialysis patients.