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Concerns exist for off-label ESA indications

Issue: July 10, 2010

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An increasing number of patients with anemia have myelodysplastic syndrome. In those patients, ESAs are prescribed off-label and are not part of the Risk Evaluation and Mitigation Strategy. However, there are still issues for these patients that fall under the discussion between Amgen, Centocor Ortho Biotech and the FDA.

For instance, patients receiving ESAs are supposed to receive the Medication Guide on a monthly basis. However, many patients with myelodysplastic syndrome do not require monthly physician visits. Many of the patients I care for with myelodysplastic syndrome I only see once every 3 to 6 months. These patients take ESAs off-label to avoid transfusions. How do physicians give these patients their Medication Guide? This is just one of a number of questions that are still unanswered.

Oncologists are using ESAs much less frequently for patients with cancer, but continue to prescribe these drugs for patients with myelodysplastic syndrome. In fact, a higher dollar volume of the prescription costs of ESAs are probably from off-label use of ESAs rather than on-label use because of the increased length of time the ESAs are used in myelodysplastic syndrome as compared to chemotherapy-related anemia.

There are also questions regarding cancer patients assigned to ESAs because they have chronic renal insufficiency. That is an on-label diagnosis. How are these patients managed under the REMS program?

I’m not saying these patients should be covered under the REMS — APPRISE apparently only deals with on-label issues — but there a number of patients with comorbidities who are on ESAs for other diagnoses who can certainly get cancer. What do we do with them? What do we do with the burgeoning population of patients who have myelodysplastic syndrome who benefit from ESAs?

We are going to see how APPRISE rolls out, determine what issues there are and hopefully bring those issues to the FDA, Amgen and Centocor Ortho Biotech. But to reiterate, the major issue for ASH, dealing as we do with hematologic diseases, is patients with myelodysplastic syndrome.

– Samuel Silver, MD, PhD

HemOnc Today Editorial Board member