Citing improved health outcomes, CMS will cover pancreas only transplants in type I diabetics.

After several years of study, the Department of Health and Human Services (HHS) Centers for Medicare and Medicaid Services (CMS) finally agreed to cover pancreas alone (PA) transplants in patients with type I diabetes who do not have kidney disease severe enough to warrant a kidney transplant.

CMS said in a "decision memo" issued on April 26 it made the decision after determining that the evidence of improved health outcomes in type I diabetic patients who receive pancreas-only transplants was sufficient to warrant coverage.

Previously, Medicare only covered patients with kidney disease who were to receive a kidney transplant concurrently with the pancreas transplant (simultaneous kidney-pancreas (SPK)) or who would receive the pancreas transplant some time after receiving the kidney transplant (pancreas-after - kidney transplant (PAK)).

Coverage will be limited to Medicare beneficiaries under the following "limited circumstances:

*PA will be limited to those facilities that are Medicare-approved for kidney transplantation;

*Patients must have a diagnosis of type I diabetes;

*The patient with diabetes must be beta cell autoantibody positive, or

*The patient must demonstrate insulinopenia defined as a fasting C-peptide level that is less than or equal to 110% of the lower limit of normal of the laboratory's measurement method. Fasting C-peptide levels will only be considered valid with a concurrently obtained fasting glucose <225 mg/dl;

*Patients must have a history of medically-uncontrollable labile (brittle) insulin-dependent diabetes mellitus with documented recurrent, severe, acutely life-threatening metabolic complications that require hospitalization. Aforementioned complications include frequent hypoglycemia unawareness or recurring severe ketoacidosis, or recurring severe hypoglycemic attacks;

*Patients must have been optimally and intensively managed by an endocrinologist for at least 12 months with the most medically-recognized advanced insulin formulations and delivery systems;

*Patients must have the emotional and mental capacity to understand the significant risks associated with surgery and to effectively manage the lifelong need for immunosuppression; and

*Patients must otherwise be a suitable candidate for transplantation."

The 20-page decision memo contains a history of non-coverage decisions made by the agency since 1999 and a section on why it was finally covering PA transplants. The major roadblock in the past had been CMS concerns about high mortality and high graft survival rates. However, several more recent studies convinced the agency their concerns had finally been "ameliorated."

"Mortality and graft survival rates are consistent among the three procedures" and "mortality from PA has improved to the point that long term survival is comparable to that for patients with type I diabetes on the PA waiting list that did not get a transplant. We believe these data are sufficient to assuage our concerns about the increased risk of the PA procedure," CMS said in the memo.

In addition, although specific quality of life data for PA transplants doesn't exist, CMS concluded that it does for SPK/PAK transplants and there is no reason to expect those results to differ.

Finally, CMS lays out what new clinical trials need to be performed in the future.

"The ability of pancreas transplantation to halt or reverse the long term complications of diabetes is unknown and continues to be debated within the professional community. Similarly, data are not available to determine whether performing pancreas transplantation earlier in the course of the disease would prevent complications. Whether or to what extent pancreas transplantation alters the course of autonomic neuropathy is still controversial. The clinical improvement of autonomic neuropathy is known to be variable from patient to patient. Hence the magnitude of improvement may be a function of time after resumption of normoglycemia. There is some preliminary evidence that a functioning pancreas graft can improve the ability of a patient with autonomic neuropathy to recognize the symptoms of hypoglycemia and to respond appropriately. A controlled clinical trial that focuses on this outcome needs to be conducted to confirm these preliminary results. We encourage and expect those trials to be done. We will be monitoring evidence development through trials, claims data and the UNOS (United Network for Organ Sharing) database to determine the need for future modification of this policy."

The CMS decision memo does not address how the PA's will be reimbursed at least in the short term. Fred Forsthoffer, MBA, a financial analyst at Benner Good Samaritan Transplant Services in Phoenix, AZ, surmised that because there is no DRG for PA yet, it will "probably be paid under DRG 513 which covers PAK, presumably the most appropriate in terms of RVU's and dollars."

Forsthoffer also pointed out that since Medicare is now covering PA, other insurance companies should follow suit.

The entire CMS decision memo can be found online at: https://www.cms.hhs.gov/mcd/viewdecisionmemo.asp?id=166