Taking my kidney in my own hands

After almost 12 years on dialysis, I am ready to have a functioning kidney so I can have a more normal life.

But I have 100% antibodies resulting from more than 20 blood transfusions over the years and it is unlikely that I will ever find a match. Because I cannot be desensitized, Dr. Enrico Benedetti, a kidney transplant surgeon of the University of Illinois in Chicago, has suggested an alternative: use of the drug Soliris (eculizumab, Alexion Pharmaceuticals), at the time of my transplant to “block” my antibodies so I will not reject a kidney. This may be my only chance at having a kidney transplant and life without dialysis.

Soliris has been used successfully in clinical trials with other patients in the US and Europe using both live donors and deceased donors. Dr. Benedetti says, “… I believe this strategy deserves to be explored in selected highly sensitized patients like Ms. Schmidt, who have no suitable alternatives to getting a kidney transplant. My initial experience with this drug has been extremely positive and it holds great promise for many.”

Dr. Benedetti and I have been working hard in trying to get the funding for use of Soliris for this procedure, which is $110,000 for the nine doses needed.

Medicare turned us down

We first approached Medicare about covering the cost of this drug because Soliris is approved by the U.S. Food and Drug Administration and Medicare for a rare blood disease––paroxysmal nocturnal hemoglobinuria, and a rare kidney disease called aHUS (hemolytic uremic syndrome)––and thought getting it “repurposed” for a kidney transplant would not be difficult. “Among the US transplant centers that perform positive crossmatch transplants, eculizumab is gaining popularity due to encouraging outcomes reported in several abstracts and case reports,” wrote Maya Campara, PharmD, BCPS, on behalf of Dr. Benedetti in a May 9 letter to CMS. “Currently, there are multiple ongoing prospective, clinical trials exploring benefits of eculizumab for sensitized patients undergoing renal transplant and UIH is an active, participating center in these trials.”

Table 1

We worked through my Congressman’s office

The National Government Services Department responded, “Medicare does not pre-approve this type of service. The service(s) would need to be provided and a claim would need to be submitted to Medicare for consideration of coverage. Should the claim deny, the provider, beneficiary, or a representative can appeal the denial with additional information to justify the medical necessity of the service.”

After reviewing the case, Medicare said the drug Soliris is considered investigational for this purpose and could not be approved by Medicare at this time.

We appealed to the Kidney Caucus and drug company

After appealing to the Kidney Caucus, a group of Senators and House members who support kidney-related legislation, we were told that the “… the Kidney Caucus, like other Congressional caucuses, does not serve individuals in a casework role.” A representative from Alexion said when asked if their Foundation would be able to help with the cost of the drug, “…that the foundation is enabled to support patients with free drug only for FDA approved indications.” Blue Cross/Blue Shield and Humana have covered the cost of Soliris for their kidney patients, in that, they saw the successful clinical trials and economic benefit of getting the patients off dialysis. There is the possibility that over time Medicare will approve this drug for kidney transplants based on the success of the clinical trials.

In the meantime, with Medicare disability being my only source of income at just over $1,000/month for disability, I need to raise the funds for the drug so I can have a kidney transplant. Suggestions from NN&I readers are encouraged to assist in identifying resources to raise the funds and ways to help others like myself in this situation. -by Lara Schmidt; Patty Purcell