Appeal type: Experimental/Investigational
Decision: Overturned

Physician 1: The patient is an 81-year-old man diagnosed with Waldenstroms macroglobulinemia (Waldenstroms disease). He was apparently started on Velcade chemotherapy in August 2003 and received regular injections through March 2004. It is noted that his abnormal protein, IGM, was 4810 in August 2003 and though there were some fluctuations during the treatment period, his last value while on treatment was 3700 (March 2004). A request has been made for reimbursement for Velcade therapy. The Health Plan has denied this request on the basis that Velcade is considered investigational for treatment of the patients condition. Velcade is a proteasome inhibitor, a new and novel chemotherapeutic agent. There is sufficient data that Velcade is useful in the treatment of multiple myeloma. It is being studied in other plasma cell dyscrasias and lymphomas. Though there does appear to be some responsiveness to Velcade in Waldenstroms disease, the data is quite preliminary and does not clearly indicate real clinical significance. Indeed, the submitted evidence suggests the patients response has been fairly minimal and of questionable clinical significance. Thus, there is insufficient evidence at this time that Velcade is superior to standard therapies for treatment of patients such as this patient. Accordingly, I have determined that the therapy at issue was not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. Therefore, the Health Plans denial should be upheld. Physician 2: The patient is an 81-year-old male with Waldenstroms macroglobulinemia (Waldenstroms disease). He has been treated with Velcade for multiple cycles with documented paraprotein response. The Health Plan has denied coverage for Velcade therapy on the basis it is considered investigational in this clinical setting.Waldenstroms disease is a B cell neoplasm that shares features of multiple myeloma and lymphoma. There are chemotherapeutic agents that are active in both diseases, i.e., alkylating agents, fludarabine, cladribine, and prednisone. Velcade has widely documented activity in multiple myeloma and will likely move into its frontline management. There is a growing body of literature showing activity of Velcade in non-Hodgkins lymphoma. Articles addressing this issue are soon to be published in the Journal of Clinical Oncology. It is not surprising to find that Velcade has activity in non-Hodgkins lymphoma and thus, it is a very reasonable agent to use in patients with Waldenstroms disease who are treatable but rarely curable. Therefore, I have determined that the therapy at issue was likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be overturned.Physician 3: The patient is an 81-year-old male with Waldenstroms macroglobulinemia (Waldenstroms disease). The patient failed first-line Rituxan therapy, and then received Velcade. The submitted records indicate the patient responded to Velcade therapy. The Health Plan denied coverage for Velcade on the basis it is considered experimental for treatment of patients such as this patient.Velcade has been approved as a third-line therapy for a related malignancy, myeloma. The therapy at issue represents an active treatment for the patients disease and was an acceptable therapy for this patient. Furthermore, it appears the patient has responded to treatment with Velcade. Therefore, I have determined that the therapy at issue was likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be overturned.