Autologous stem cell transplantation (AuSCT) is a process in which stem cells are harvested from a patient’s bone marrow or peripheral blood, stored, and then transplanted back into the patient following high dose chemotherapy. This process used to treat various malignancies and is one of the leading therapies for AL Amyloidosis.
The Centers for Medicare and Medicaid Services (CMS) in it’s March 15, 2005 decision, has determined that the evidence is adequate to conclude that when recognized clinical risk factors are employed to select patients for transplantation, high dose melphalan together with autologous stem cell transplantation (HDM/AuSCT) can provide a net health benefit for Medicare beneficiaries of any age group with primary AL amyloidosis. HDM/AuSCT is reasonable and necessary for patients of any age with primary AL amyloidosis who meet the following criteria:
- amyloid deposition in 2 or fewer organs, and
- cardiac left ventricular ejection fraction (EF) of greater than 45%.
For details, go to:
http://www.cms.hhs.gov/mcd/viewdecisionmemo.asp?id=126
Prior to this, section 110.8.1 of the National Coverage Determination (NCD) manual (CIM §35-30) states that this procedure is covered via contractor discretion for patients 63 years of age and under. As of October 1, 2000, primary AL amyloidosis has a national non-coverage determination for Medicare beneficiaries 64 years of age or older.
The Centers for Medicare & Medicaid Services (CMS) agreed to reconsider this coverage determination if new evidence became available. Requestors have submitted new data relating to this transplant procedure that was not available at the time of the original determination. The Amyloidosis Support Network also submitted its comments and are available for viewing at http://www.cms.hhs.gov/mcd/viewpubliccomments.asp?nca_id=126
For additional status information click on the following link:
Tracking Sheet for AuSCT for Amyloidosis (CAG-00050R)
The comments we submitted are as follows:
To whom it may concern:
I am writing on behalf of the Amyloidosis Support Network (ASN), a not-for-profit 501(c)(3) all volunteer organization. We are the world’s only organization dedicated to amyloidosis patient advocacy, support and medical community awareness.
Currently section 110.8.1 of the National Coverage Determination (NCD) manual (CIM §35-30) states that the Autologous stem cell transplantation (AuSCT) procedure for primary AL amyloidosis has a national non-coverage determination for Medicare beneficiaries 64 years of age or older.
The ASN is in strong agreement with the March 30, 2004, formal request for reconsideration provided by Legacy Good Samaritan Hospital and the July 26, 2004 comments from the Dana Farber Cancer Institute in support of coverage for coverage of AuSCT for AL Amyloidosis. Data presented by Boston University School of Medicine, Mayo Clinic, Washington University, and the ABMTR conclude that AuSCT offers improvement in overall survival for patients age 65 and over and therefore would provide significant life-prolonging benefit to applicable patients in the Medicare population.
Moreover, the ASN would to add two additional points as background for our comments:
- We agree with one of President Bush’s health care policies where he stated on various occasions “Give patients and doctors – not government bureaucrats – more control over health care decisions”.
- Also, the AuSCT procedure for Multiple Myeloma, a closely related “sister disease” to AL Amyloidosis, is covered for Medicare beneficiaries 64 years of age or older. We do not understand how Medicare justifies this discriminatory practice.
In reference to the December 15, 2004, Draft Decision Memo for AuSCT for Amyloidosis (CAG-00050R), the ASN believes it is directionally correct. As we see it, the draft contains 2 operative components; the criteria for coverage and the registry.
The ASN fully supports the registry concept and would be willing to provide what we can do as a patient advocacy organization to make it a reality. This might include infrastructure and/or procedures. We understand that the registry would not be a requirement for coverage, but we believe the CMS has an opportunity to help improve, refine and contribute to the body of knowledge on the diagnosis, treatment and management of AL Amyloidosis. In addition, we recommend the CMS expand the registry concept to incent and otherwise encourage patients to participate in clinical trials. This inevitably will serve to provide the following benefits:
- Increase the rate of early detection
- Improve the quality of care
- Enhance patient quality of life
To the extent these benefits are realized, costs associated with pre and post treatment supportive care would decrease.
However, the criteria itemized by CMS in the draft decision memo we believe are unnecessary. Patients receiving transplants for amyloidosis are already a highly selected group by virtue of age, performance status, number of organs involved, and the absence of class IV heart failure. The Mayo Clinic published [ref. Blood Reviews (2004) 18, 17–37] its development of a risk-adapted strategy for transplantation. Memorial Sloan-Kettering [Blood, 15 June 2002, Volume 99, Number 12] and Boston University Medical Center have also published material on this approach. Doctors, and even the patients, understand the importance of patient selection cannot be overemphasized. Again, we strongly believe AL Amyloidosis treatment decisions should made between patients and doctors – especially at the main amyloidosis treatment centers.
We would ask that the CMS incorporate the risk-adapted approach as a guideline (not a requirement for coverage) in its decision memo. This, coupled with the registry, would serve to give patients and doctors more control over health care decisions. Therefore, it would ensure the maximum number of patients get the optimum treatment as dictated by their condition.
To be sure, the cost of medical treatment is expensive. The expense of diagnosing, treating and recovering from amyloidosis is well beyond the means of most of us. It is at this critical time that we most need financial support. Amyloidosis is an aggressive, insidious disease and patients and families must be equally aggressive in obtaining care. We expect the healthcare system would be ready, willing and able to provide that help. We are in daily contact with many of those affected by AL Amyloidosis. Situations exist where patients and families have had to sell their home or substantially draw down their savings. Worse yet, they may have had to accept less than optimal treatment because the cost was too great. And we only see a small percentage of patients. How many patients , and their doctors, give up because of financial barriers and the lack of treatment guidelines?
The CMS has an opportunity to make an improved difference in the lives of all patients through the registry and allowing the AuSCT to be included in the treatment arsenal for those on Medicare. We, along with our doctors, realize the AuSCT is not for everyone. However, the reason for not being treated with an AuSCT should be a medical one, not a financial decision.
The Amyloidosis Support Network Inc., organized exclusively for charitable purposes under Section 501(c)(3) of the Internal Revenue Code, is committed to increasing the rate of early detection of amyloidosis, thereby improving survivability and quality of life. Our approach is to increase awareness among the public and professional communities, as well as, linking patients/families to further experienced medical and emotional support resources. We intend to sponsor advocacy programs and services, worldwide, through the leadership of our knowledgeable and dedicated staff and volunteers.
Sincerely,
Dennis Krysmalski
President, Amyloidosis Support Network, Inc.
www.amyloidosis.org
dkrysmalski@amyloidosis.org
