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CMS May Limit Off-Label Coverage For Cancer Drugs To Clinical Trials

CMS is considering whether to limit off-label coverage for certain expensive cancer treatments to trials under the auspices of the National Cancer Institute, according to an oncology trade group.

CMS' consideration of a trial-only coverage policy for off-label uses of cancer drugs was revealed in an Aug. 30 meeting between representatives of the Association of Community Cancer Centers and senior CMS officials, including CMS Medical director Sean Tunis and Scott Gottlieb, an advisor to CMS Administrator Mark McClellan, according to the ACCC newsletter.

“CMS' intent is to develop a model for studies to assess the clinical evidence on off-label uses of drugs that are not included in a compendia so that the agency will have the necessary information to determine whether Medicare should cover such uses. CMS officials indicated that they are considering studies that are less rigorous than they usually utilize for coverage decisions -- e.g., the trials would not have to be randomized,” the ACCC newsletter reports. “ACCC also learned that as CMS contemplates a national coverage determination, a specified off-label use would not be covered unless the service is furnished under the auspices of an NCI trial.”

However, CMS has subsequently backpedaled, emphasizing that the coverage criteria are still under review, according to one informed source.

While CMS is contemplating first imposing limits on four colorectal cancer drugs now under review, the model could also be used for radioimmunotherapy for Non-Hodgkin's lymphoma, according to ACCC. The ACCC report is consistent with remarks by Tunis, who indicated the agency was looking at the pending coverage decision on colorectal cancer drugs as means of gathering further clinical evidence on off-label uses (Inside CMS, Sept. 9).

CMS officials also said that a coverage model designed to develop clinical evidence could be employed for additional uses of PET scans, according to ACCC. CMS last week put into effect a coverage call that limits PET scans for Alzheimer's to a clinical trial or to cases where other diagnostic techniques had failed. The agency also has a pending coverage decision on use of PET to diagnose seven rare cancers. Under questioning at the Senate Finance Committee last week, McClellan told Sen. Bob Graham (D-FL) that the agency would try to have a coverage decision by the end of September on the use of PET for diagnosis of  multiple myeloma, one of the seven treatments under review. Last spring, a bipartisan group of senators urged CMS to leave coverage decisions on PET use for rare cancers up to local contractors (Inside CMS, May 6).

CMS met last week with the NCI to discuss the evidence requirements in the trials for the colorectal cancer drugs. The evidence requirements were among the issues ACCC representatives raised with CMS in the August meeting, according to an informed source. The group also questioned who would be responsible for gathering and compiling the data and whether off-label treatments for rare diseases would ever have sufficient breadth to meet the evidentiary thresholds for the trials. In addition, ACCC also asked CMS whether variations in clinical practice -- oncologists often vary the chemotherapy “cocktail” given different patients -- would impede the ability to get good results for a trial.

The ACCC report also says that CMS is “exploring the extent to which it could pay for the trials, including the costs of providing service and data collection costs.” That prospect led one drug manufacturer representative to call the proposal a win for both manufacturers, who would be off-the-hook for the cost of the trials, and CMS, which would have an ability to restrain off-label use of very expensive new drugs. But an oncology source said the proposed restriction could slow down oncologists' ability to find the most-effective new cancer treatments. -- Paul Precht

Read the American Society of Clinical Oncology's response to a New York Times article on this topic:

January 30, 2004: New York Times article on Off-Label Use of Chemotherapy Drugs

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