Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Myelodysplastic Syndrome

June 5, 2010

Louis B. Jacques, MD
Director, Coverage and Analysis Group
Centers for Medicare & Medicaid Services
Mail Stop S3-02-01
7500 Security Boulevard
Baltimore, MD 21244

Re: Proposed Decision Memorandum for Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Myelodysplastic Syndrome (MDS) (CAG-00415N)

Dear Dr. Jacques,

This letter is formal public comment by the National Marrow Donor Program (NMDP) and American Society for Blood and Marrow Transplantation (ASBMT) on the Centers for Medicare & Medicaid Services (CMS) proposed decision memorandum for allogeneic HSCT for MDS. We have reviewed the proposed national coverage determination (NCD) and appreciated the opportunity to meet with CMS representatives to understand better the proposed decision’s objectives and implications.

While we believe that the body of evidence supports CMS issuing a coverage policy that ensures that Medicare beneficiaries diagnosed with MDS have direct access to allogeneic HSCT, we look forward to working collaboratively with CMS on the proposed coverage with evidence development (CED) policy. As requestors of this NCD, we strongly agree that any CED approach must provide Medicare beneficiaries with access to allogeneic HSCT while making certain that additional data is collected to further demonstrate the curative potential of allogeneic HSCT and the health benefits for Medicare beneficiaries with MDS.

We welcome the opportunity to provide CMS with comments that will assist CMS in finalizing the NCD and are committed to designing a prospective clinical study that fulfills CED requirements. This CED can supply providers and beneficiaries with the additional data to make the most informed treatment choice. We would like to emphasize the following comments for your consideration:

1. If CMS finalizes its proposal, the Center for International Blood and Marrow Transplant Research (CIBMTR) has the capability to conduct and publicly disseminate the data from a prospective clinical study that meets CMS requirements. Through the CIBMTR, a worldwide network of HSCT centers currently share data on HSCT outcomes and maintain a clinical database with information on more than 300,000 HSCT recipients. In September 2006, the Health Resources and Serviic outcomes, donor registry and cord blood inventory size, and patient access to HSCT. Under the CIBMTR, centers provide a standard dataset for all consecutive transplant recipients pre-transplant and post-transplant at 100-day, 6-month, and annual intervals. This dataset is an internationally agreed upon set of information referred to as Transplant Essential Data (TED). TED.level data is also the dataset required for transplant center accreditation by the Foundation for the Accreditation of Cellular Therapy and the Joint Accreditation Committee – ISCT (International Society for Cellular Therapy) and EBMT (European Group for Blood and Marrow Transplantation), known as FACT-JACIE. Through use of TED forms and other data submission tools, researchers can use CIBMTR data to study a wide spectrum of treatment.related issues, including patient outcomes. Furthermore, CIBMTR provides broad database access to researchers, enablices Administration (HRSA) awarded a contract to CIBMTR to administer the Stem Cell Therapeutic Outcomes Database (SCTOD). SCTOD collects data on all allogeneic HSCTs performed in the United States with the purpose of increasing the safety, efficacy, and availability of HSCT. SCTOD data collection enables analysis of administrative program use, center-specifng a robust data enterprise. We believe that the federally mandated data collection efforts of CIBMTR and SCTOD represent ideal platforms upon which to design a prospective study to capture longitudinal outcomes data for Medicare beneficiaries undergoing allogeneic HSCT for MDS (and controls receiving non-transplant therapy) under the CED requirements outlined in the CMS proposed decision.
2. To meet the proposed CMS CED requirements, CIBMTR intends to conduct a prospective, observational, non-blinded study based upon the CIBMTR data elements, supplemented by additional data collection on non-transplant patients who are receiving other therapies for MDS. The study population will include all patients 55 years of age or older referred to qualified transplant centers for transplant. The patients assigned to the non-transplant arm would be those referred to a participating transplant center but for whom a donor could not be located. Survival will be the primary outcome measure for determining effectiveness with overall survival at 3 years after therapy (adjusted for waiting time until HSCT to compare with the patients not undergoing HSCT) tested in each group. International Prognostic Scoring System (IPSS) and other potential prognostic indicators will be analyzed to identify patients most likely to benefit from a particular therapy. We also intend to report other data on the transplant and non-transplant arms of the study. Existing data collection instruments and systems will be enhanced to capture data on both the transplant and non-transplant arms of the study. Such an approach would not preclude participation of patients in other clinical studies designed to improve outcome of either HSCT or non-HSCT therapy. As noted above, reporting HSCT outcomes is mandated by current law. Absent of a similar mandate to report outcomes of non-transplant therapies, prospective observational strategies and data collection processes will need to be created to collect the necessary information to make a credible comparative assessment. Efforts will need to be made to ease the reporting and analysis burden for participating centers. Supplemental funding will have to be sought to support this prospective data collection and analysis activity.
3. All centers that report data to CIBMTR will be included in the study to ensure an inclusive study approach with meaningful patient accrual. Per the Stem Cell Therapeutic and Research Act of 2005 (U.S. Public Law 109.129), a standard dataset for all U.S. allograft recipients must be submitted to the CIBMTR. Since all US HSCT centers must submit data to CIBMTR which will be used for this study, all US HSCT centers conducting HSCTs for Medicare beneficiaries should be covered under this proposed policy once finalized. We believe this approach will avoid possible referral bias and ensure that the data being collected for the study is as inclusive as possible and that results will be generalizable to future Medicare beneficiaries. It will also allow us to assess whether there are subgroups of patients better served by one or the other therapeutic approaches.
4. As CMS finalizes its decision, NMDP and ASBMT will work with CMS to ensure that our prospective clinical study meets the CED requirements. The proposed NCD does not specify a mechanism to ensure that prospective clinical studies meet CMS requirements. The proposed NCD states:

   "Medicare payment for these beneficiaries will be restricted to patients enrolled in a prospective comparative clinical study. In accordance with the law, a standard dataset is collected for all allogeneic transplant patients in the United States by the CIBTMR. The elements in this dataset, comprised of two mandatory forms plus one additional form, encompass the information we require for a study under CED."

   NMDP and ASBMT intend to work with CMS to ensure that the CIBMTR study meets the CMS NCD and CED requirements. CIBMTR plans to submit its study protocol CMS for review to ensure compliance. NMDP and ASBMT will ask for CMS to confirm in writing that the proposed study protocol will meet the CMS requirements. NMDP and ASBMT are committed to ensuring that the clinical study allows for beneficiary access to allogeneic HSCT for MDS and welcomes to the opportunity to discuss with CMS the appropriate study design and characteristics. We believe that CMS confirmation of the proposed CIBMTR protocol is vital to a successful CED implementation.

We support CMS issuing a national coverage policy that ensures that Medicare beneficiaries diagnosed with MDS have access to allogeneic HCT. As proposed, the Agency will cover allogeneic HSCT in the context of a prospective clinical study that must address how Medicare beneficiaries receiving HSCT experience improved outcomes. Such a policy can ensure appropriate beneficiary access through collaboration on the implementation of a prospective clinical study based upon the capabilities of the CIBMTR. As required by law, CIBMTR collects and analyzes data on the benefits of allogeneic HSCT. If finalized, the CMS decision will build upon the infrastructure of the CIBMTR to conduct research on the benefits of allogeneic HSCT for Medicare beneficiaries diagnosed with MDS.

We look forward to continuing to work closely with CMS throughout the NCD process and through implementation of this important policy. We would be pleased to provide any additional information that CMS may require. For further assistance, please contact Michael Boo, Chief Strategy Officer, National Marrow Donor Program, at 612.627.5855 or mboo@nmdp.org.

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June 4, 2010

Ms. Marilyn Tavenner
Acting Administrator
Centers for Medicare and Medicaid Services
7500 Security Boulevard
Room C5-26-16
Baltimore, MD 21244

Dear Ms. Tavenner:

Thank you for the opportunity to submit thispublic comment letter on the decision by theCenters for Medicare and Medicaid Services (CMS)to cover allogeneic hematopoietic stem celltransplants (HSCT) for patients withmyelodysplastic syndromes (MDS) who are enrolledin approved clinical trials. I appreciate youragency’s commitment to expanding Medicare coveragefor this deadly disease, because reducing theburden that MDS imposes on our society is one ofmy highest priorities in Congress.

I was pleased to see that CMS responded to therequest for a national coverage decision on HSCTfor MDS patients, which was made by a number oforganizations interested in bone marrowtransplantation for cancer treatment. CMS haschosen to limit coverage of HSCT to MDS patientsenrolled in a controlled clinical trial underCMS’s Coverage with Evidence Development (CED)policy. This decision is a step in the rightdirection because there are so few clinicaltreatments that have proven effective for MDSpatients.

I am committed to working with my colleagues inCongress and with CMS to increase the number ofeffective treatments—including HSCT—available topatients who suffer from MDS. I stronglyencourage CMS to work closely with relevantorganizations and with MDS experts to develop theclinical trials called for under the proposednational coverage decision, so that patient accessto this important treatment improves. It is myunderstanding that several of the organizationswhich made the initial coverage request to CMS arealready outlining the parameters for a clinicaltrial, with the intention of meeting your agency’srequirements as laid out in the proposed nationalcoverage decision.

I urge CMS to give prompt attention to the reviewand approval of this study design as soon as it isformally presented. I also urge CMS to berealistic in its expectations for this design,given the nature of MDS and the fact that most MDSpatients not receiving HSCT will continue toreceive those treatments that are currentlyavailable to combat their illness. As a result,it will be important to keep in mind that arandomized clinical trial involving a controlgroup receiving “placebo HSCT treatment” may notbe feasible.

Thank you for your prompt attention to this issue. Expanding Medicare coverage of treatments for MDSis critically important to me and to many of mycolleagues in Congress. I look forward to workingwith you in the future on this topic.

Sincerely,

DORIS O. MATSUI
Member of Congress

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Tamara S. Syrek Jensen, J.D.
Acting Director, Coverage and Analysis Group
Centers for Medicare & Medicaid Services
Mail Stop C1-09-06
7500 Security Boulevard
Baltimore, MD 21244

RE: Proposed Decision Memo for Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Myelodysplastic Syndrome (CAG-00415N)

Dear Ms. Jensen:

The Alliance of Dedicated Cancer Centers (the “Cancer Centers”) submit the following comments on the proposed decision memo for coverage of allogeneic hematopoietic stem cell transplantation (HSCT) for Myelodysplastic Syndrome (MDS). As you can see, the Cancer Centers’ 11 member organizations are nationally recognized institutions that focus exclusively on the care of cancer patients. Our institutions are at the forefront of the war on cancer and our physician leaders are some of the most knowledgeable in the country about MDS and this horrible disease’s treatment options (or lack thereof).

The Cancer Centers are pleased that CMS reviewed the information and comments submitted on this issue by the Cancer Centers and others. We are disappointed, however, by CMS’ belief that the current evidence does not demonstrate that the use of HSCT improves outcomes for Medicare patients with MDS. We disagree with CMS’ finding that national coverage of HSCT for MDS is not reasonable and necessary for its beneficiary population at this time and believe the agency’s analysis was overly conservative.

The Cancer Centers encourage CMS to consider the evidence again and revise its decision to allow for national coverage.

MDS is a group of bone marrow malignancies that is incurable with conventional therapies and for which allogeneic stem cell transplantation is the only treatment with a known curative potential – certainly any of the FDA approved therapeutics for MDS would be considered to offer “health benefit”. Allogeneic hematopoietic transplantation has been documented in numerous clinical trials to produce long term disease-free survival in this category of disease in which there is no other definitive treatment. MDS commonly evolves into Acute Myeloid Leukemia (AML); AML arising out of MDS (also called secondary AML) is extremely difficult to treat. Given that Medicare currently covers allogeneic HSCT for patients with AML — the end-stage of disease evolution for patients with higher-risk MDS — we believe that CMS should expand allogeneic HSCT coverage to MDS patients as those patients have no treatment options left beyond truly experimental drug therapies. Doing so would eliminate the existing and unjustified disparity caused by the fact that patients with MDS who progress to leukemia have HSCT coverage yet they have a much lower chance for success, compared to patients who are able to receive a transplant during the MDS phase yet do not have coverage. Providing coverage for a treatment only after the patient has progressed to a more advanced state (i.e., AML) reduces the chances for a beneficial outcome compared to providing the transplant treatment while the patient is better able to tolerate the procedure.

Allogeneic HSCT is the only treatment that leads to long-term, progression-free survival for those suffering from MDS. In recent years, allogeneic HSCT has shown success rates in MDS patients that are comparable to those seen in de novo AML patients. The American Society for Blood and Marrow Transplantation (ASBMT) expert panel reviewed the evidence and found that the procedure demonstrates long-term, curative outcomes for MDS. We believe there is clear evidence that patients with MDS experience improved health outcomes with, allogeneic hematopoietic transplantation compared to other forms of treatment. On page 21 of the decision memo, CMS itself notes that, “since most patients of Medicare age will be ineligible for intensive chemotherapy due to co-morbidities and/or generally poor health status, HSCT may be their only potential option.” The Cancer Centers would like to understand whether the agency believes that there is a subset of MDS patients that would benefit from SCT and where coverage would be appropriate – for example, high-risk patients or those that have failed conventional therapies?

Because of MDS’ prevalence among the elderly, it is important to note that age is no longer a barrier to successful HSCT and that the older population can benefit from the procedure. HSCT is no longer restricted to the younger MDS population. Medical advances now result in older MDS patients who receive allogeneic stem cell transplantation experiencing successful outcomes that are comparable to those seen in younger patients. Allogeneic HSCT is now routinely performed in those over age 65, and recent data also specifically indicate that the procedure is effective for elderly MDS patients; results are virtually identical to those for patients with AML.

For these reasons, the Cancer Centers respectfully disagree with CMS’ conclusion that there is insufficient evidence supporting the use allogeneic HSCT in MDS patients. We believe that sufficient clinical evidence already exists and that national coverage should be granted for HSCT for MDS patients.

While we are disheartened that CMS is not granting national coverage at this time, we are pleased that the agency proposes moving forward with the “Coverage with Evidence Development” (CED) process. This is the next best step, although one that is less desirable than national coverage. The CED process will at least gather additional data in the context of clinical care, and provide coverage for transplant patients with MDS who are enrolled in the controlled clinical study. We are concerned, however, that the CED process described in CMS’ coverage decision memo will create an administrative burden for providers — since enrolling each patient into the study and completing the pre- and post-procedure paperwork will be labor-intensive. Therefore, we request that CMS simplify and streamline the study enrollment process by creating a mechanism to notify transplant centers within 48 hours when patients are accepted into the study. This notification is critical for patients to know they have coverage and for providers to know they will be reimbursed prior to providing the transplantation procedure. Any determination of non-coverage after study enrollment would be significantly detrimental to both patients and providers. The Cancer Centers also propose CMS create a means to facilitate identification of enrolled patients through the claims submission process by using form locator 63 on the UB-04, which is an authorization field that enables centers to enter the patient’s study registration number. This will allow CMS to quickly and easily identify patients in the study and minimize transplant centers’ reporting burdens. In summary, the Cancer Centers concur with the evidence previously submitted by the following organizations as part of their formal request for a National Coverage Determination on allogeneic HSCT for MDS patients in the Medicare population: National Marrow Donor Program (NMDP), ASBMT, American Association of Blood Banks (AABB), American Cancer Society (ACS), American Cancer Society Cancer Action Network (ACS CAN), American Society of Hematology (ASH), American Society of Clinical Oncology (ASCO), Aplastic Anemia and MDS International Foundation, the Blood & Marrow Transplant Information Network, National Bone Marrow Transplant Link, the Bone Marrow Foundation, and the Leukemia & Lymphoma Society.We continue to believe that expanding Medicare coverage for MDS would ensure that beneficiaries who desperately need transplantation have access to the best and most effective treatment for their condition in a timely manner, regardless of their personal finances or state of residence. Expanding coverage would facilitate efforts to cure MDS, an otherwise fatal illness, without waiting for patients to become much sicker and progress to AML before being treated with covered HSCT. Coverage expansion would acknowledge scientific developments that make such transplants feasible and successful in this vulnerable population and enhance facilities’ ability to provide timely and life-saving services to their patients. In short, national coverage expansion would positively impact Medicare beneficiaries’ health and longevity while minimizing administrative burden for the provider community. To that end, the Cancer Centers request that CMS moves towards national HSCT coverage for MDS as quickly as possible. The Cancer Centers appreciate CMS’ consideration of our comments and hope the agency will review the evidence again and revise its current proposed decision. We feel strongly that HSCT is the best, most effective, option for treating Medicare patients with MDS and should not be denied to Medicare beneficiaries.

Sincerely,

R. Donald Leedy
Executive Director
Alliance of Dedicated Cancer Centers

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My education and expertise is not in the medical field. However, after absorbing any and all published information, scientific developments and progress pertaining to MDS and its cure, a Bone Marrow Transplant/Allogeneic Hematopoietic Stem Cell Transplant, there is no doubt that MDS is a malignant cancer and deserves to be treated as such. The position CMS has taken with regard to treatment of MDS patients is based on obsolete and outdated facts. The scientific community has unequivocally demonstrated that MDS IS a malignant bone marrow cancer and is NOT a form of benign anemia.

It is not only the scientific/medical community who is knowledgeable about the appalling refusal by CMS and to provide covered treatment for older MDS patients. Through word of mouth and media exposure, the lay community is also becoming aware that CMS and has chosen to underserve its senior MDS patient population by disallowing coverage for the only known cure.

The WHO and the US National Cancer Institute recognize MDS as forms of cancer. According to Dr. Mikkael Sekeres, Cleveland Clinic, and author of CLINICAL MALIGNANT HEMATOLOGY, "there is no question that (your) bone marrow disorder is not simply a benign form of anemia, MDS IS A CANCER". Dr. David Steensma, Harvard Medical School edited a book in 2009, MYELODYSPLASTIC SYNDROMES; PATHOBIOLOGY AND CLINICAL MANAGEMENT, in which (15) MDS experts worldwide refer to MDS as hemopoietic neoplasm/malignancy/cancer.

Clearly, MDS is known to be a malignant cancer as clearly as is breast cancer, colon cancer, prostate cancer, etc. and patients with MDS diagnosis need equal coverage for treatment. The bias that currently exists regarding CMS''s hit or miss coverage for MDS cancer patients needs to change, and quickly so. Older patients with MDS should be given every opportunity and the best odds to survive a BMT/HSCT while they are still in otherwise good health. Let''s not sleep on this, the proof has been provided that MDS is a malignant cancer. Many lives can be saved with CMS coverge approval for Bone Marrow Transplants/Allogeneic Hematopoietic Stem Cell Transplantation.

The potential opportunity to receive a CMS covered BMT/HSCT through an approved clinical study is HUGE!! I wish to express my gratitude to the doctors/scientists who have spent years studying this disease to provide the facts and data to prove to the world that MDS should be afforded as much equal consideration, treatment, and CMS COVERED care, as do all other malignant cancers.

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Charlene Frizzera
Acting Administrator
Centers for Medicare & Medicaid Services
Department of Health and Human Services
7500 Security Boulevard
Baltimore, MD 21244-1850

RE: CAG -00415N

Dear Ms. Frizzera:

We applaud the action by the Centers for Medicare & Medicaid Services (CMS) to initiate and proceed promptly with a national coverage analysis of allogeneic hematopoietic stem cell transplant (HSCT) for patients diagnosed with myelodysplastic syndrome (MDS). The analysis was launched in response to the formal request from The Leukemia & Lymphoma Society (LLS) and a number of other stakeholders. We are disappointed that the proposed decision memo concludes that the evidence does not demonstrate that HSCT for MDS is reasonable and necessary, but we are pleased that CMS proposes coverage for HSCT for MDS pursuant to Coverage with Evidence Development (CED) standards.

We understand that CMS has agreed to meet with stakeholders to discuss design of the CED process and trust this can be a productive dialogue. Our hope is that an effective study can be designed that will produce the requisite data to support Medicare coverage for allogeneic HSCT for patients with MDS. The Leukemia & Lymphoma Society looks forward to continued interaction with CMS regarding the CED process.

There is a clear need for rapid conclusion of studies through the CED process and a final decision regarding Medicare coverage for allogeneic HSCT, as measured by the significant burden of MDS among Medicare beneficiaries. MDS, a cancer of the blood and the marrow, occurs in approximately 4 per 100,000 persons and most commonly strikes men aged 70 and above. In fact, approximately 80% of all patients diagnosed with MDS are above the age of 65 and therefore are Medicare beneficiaries. There is a pressing need to determine the role of allogeneic HSCT for MDS, as MDS progresses to acute myelogenous leukemia (AML) in approximately 30 percent of patients.

LLS would like to express its appreciation for the work of CMS staff on the coverage analysis to date and looks forward to additional work on the CED process. Should you have any questions about this, please feel free to contact me.

Sincerely,

Mark Pascu
Director of Federal Affairs
The Leukemia & Lymphoma Society
66 Canal Center Plaza
Suite 520
Alexandria, VA 22314
703-535-6650 x 5815
mark.pascu@lls.org

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June 4, 2010

Louis B. Jacques, MD
Director, Coverage and Analysis Group
Centers for Medicare and Medicaid Services
Mail Stop C1-09-06
7500 Security Boulevard
Baltimore, Maryland 21244-1850

Dear Dr. Jacques:

Thank you for the opportunity to comment on the Centers for Medicare and Medicaid Services' (CMS's) proposed national coverage decision (NCD) Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Myelodysplastic Syndrome (CAG-00415N). America's Health Insurance Plans (AHIP) is the national association representing nearly 1,300 health insurance plans providing coverage to more than 200 million Americans. We are pleased to submit these comments on behalf of our members.

General Comments

AHIP and our member plans support CMS's efforts to review all current and available clinical evidence to determine appropriate coverage for procedures and treatments that are best shown to be safe, effective, and improve the health outcomes of Medicare beneficiaries. As we stated in our previous comments submitted to CMS on this issue in December 2009, our members are supportive of providing coverage to Medicare beneficiaries for HSCT for myelodysplastic syndrome, as this treatment has proven to be curative for this condition within certain patient populations. We also agree with CMS that there is a lack of evidence on the efficacy of this treatment for myelodysplastic syndrome for patients 65 years and older, so that there remain questions regarding appropriate populations (e.g., age appropriateness) and treatment approach.

Due to current gaps in evidence for this treatment, our members are supportive of providing this treatment through the Coverage with Evidence Development (CED) mechanism if the data collection is part of a well structured evidence development process -such as clinical trials sponsored by the National Institutes of Health (NIH) or the Department of Veterans Affairs (VA) - and the evidence collected is publicly reported upon conclusion of the trials. We also believe that the requirements set forth in the Stem Cell Therapeutic and Research Act of 2005 (U.S. Public Law 109-129), which establish the Stem Cell Therapeutic Outcomes Database (SCTOD), will enable CMS to better track beneficiary outcomes and provide valuable evidence on appropriate populations and treatment approaches for this indication. The Stem Cell Therapeutic and Research Act of 2005 requires "the Secretary…(to) establish and maintain a scientific database of information relating to patients who have been recipients of a stem cell therapeutics product (including bone marrow, cord blood, or other such product) from a donor."¹ Therefore, in light of the current evidence base and the requirements of HSCT outcomes to be tracked through the SCTOD, our members support the coverage of HSCT for myelodysplastic syndrome through CED.

Thank you for the opportunity to provide these comments.

Sincerely,

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I support the idea that doctors, not blanket policy, should be making the decisions as to which Medicare MDS patients should be considered for BMT/HSCT. Age alone (or being a Medicare patient) is not a valid criterion for exclusion from transplantation if the physician believes the patient is a good risk. Lim, ZY, et al, Journal of Clincal Oncology, 2010: "overall survival at four years was significantly adversely affected by advanced disease at transplantation. Advanced age per se should not be a contraindication for allografting in MDS." Waiting until a Medicare patient suffers from AML (i.e., advanced disease)before going to transplant flies in the face of Lim''s data. Also, National Comprehensive Cancer Network (NCCN) states that "Lower risk patients do well with allogeneic HSCT with five-year overall survival of 80% whereas those with 5-20% marrow blasts have only 25-28% five-year overall survival." It appears clear that, if transplantation is going to be considered, it should be done earlier rather than later. CMS policy does not currently support this earlier intervention (because CMS supports transplantation only for AML and not for MDS per se.) Our physicians are the ones to best decide the treatment path. If BMT/HSCT is indicated for a patient, then waiting until advanced disease has evolved will lead to more failures than successes.

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I have read the CMS draft proposal for Medicare coverage of BMT for patients w/MDS (w/o overt lymphoma). From the perspective of my general rural clinical practice, I believe that this decision will adversely affect my patients. Coverage (decisions)regarding BMT for MDS should be individualized on a case-by-case basis selected by qualified/experienced Oncologists (& approved by CMS) based on established clinical factors that protend good outcome. Several (otherwise healthy) elder (MDS) patient in our small community could essentially be cured of their MDS with successful BMT (undertaken before crisis) under the supervision of experienced Oncologist. Most of or patients DO NOT have access to (or cannot realistically participate) in clinical trials due to geographic isolation & legitamate transportation issues. Therefore, for the benefit our (& other rural) community''s patients I would hope this determination to restrict coverage to Clinic Trials be respectfully re-considered.

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[PHI Redacted] is 65 and was diagnosed with MDS in 2007. He is healthy in every way other than RARS MDS. He is now very anemic, but he feels good and has the body of a 40-yr-old. He might never progress to AML, thus becoming eligible for a transplant, yet HSCT done soon could provide a cure and allow him to live out his full life span. Waiting to become more ill before being considered for HSCT will make him a worse risk for the procedure. Offer a cure while he is still healthy and young enough to make it through!

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One of my dear family members has MDS diagnosed in 2007. Aside from this condition, he is extremely healthy (age 65) with parents who are currently healthy and well in their mid-90''s. His disease may never transform to AML (making him eligible for HSCT.) Should he be robbed of his next 30 years of life because he does not have AML to qualify for HSCT? He has been given only a few years to live, but it is unlikely that he will ever develop AML; MDS might kill him eventually due to anemia and organ failure. HSCT could provide a cure for his MDS thus allowing him to live out his genetically pre-programmed lifespan. This is not a person with co-morbidities who would die soon from other causes. HSCT could offer a gift of life and an actual cure for his MDS! His case should be able to be evaluated by his physicians without waiting to see if he ever contracts AML. Medicare should not hold the power of life or death by with-holding the potentially life-saving treatment of HSCT. Please approve this procedure for Medicare patients recommended by their physicians!

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Although the Proposed new policy on Allogeneic HSCT for Myelodysplasitc Syndrome is a baby step in the right direction, it does not go far enough. We know the prognosis for MDS once it transforms into AML is decreased. Yet, Medicare agrees to cover transplants for AML. It makes little sense to cover one and not the other. Providing transplants for appropriate patients with MDS can be cost effective as treating MDS patients on a long term basis can be quite expensive. Furthermore, once the patient transforms to AML, they may be a candidate for transplant. Therefore, Medicare has now paid for MDS treatment as well as transplant.

With new therapies emerging, transplant patient outcomes can improve. Decisions made based on older research that doesn''t include newer therapies need to be reviewed and reconsidered. One must look at the whole picture and look to the future. With this decision, answers to the proposed studies won''t be known for 5 years from now and thousands of patients will be affected, many of whom will not have the funds and ability to go to a center that has a study. Many will loose the chance for a possible cure and potentially better quality of life.

I ask that you reconsider this decision and allow patients diagnosed with MDS to have allogeneic transplants without the protocol restrictions.

Sincerely,

Judith Luck, MSN, ARNP, CPNP, CHTC

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Hello

[PHI Redacted]

Currently the only cure for MDS is a successful bone marrow transplant. We were shocked to learn that insurance (MEDICARE) won''t pay for a transplant until MDS has progressed to AML. acute myeloid Leukemia, full blown cancer. At this point in the disease progression there are many medical complications that accompany a bone marrow transplant, and excessively high health care costs and loss of life that could have been saved had there been an earlier intervention,,,,,,before AML.

Medicare was designed especially for seniors, and we as Americans have been promised health care for all so it''s incomprehensible that a SENIOR HEALTH CARE PLAN would deny a cure for a SENIOR DISEASE and choose to wait for the onset of full on cancer before authorizing a PREVENTIVE treatment for AML and a known cure for MDS.

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