COMMENTS SUBMITTED TO THE

CENTERS FOR MEDICARE AND MEDCAID SERVICES (CMS)

REGARDING CHANGES IN CLINICAL TRIAL POLICY

May 10, 2007

Steve Phurrough, M.D., M.P.A

Director, Coverage and Analysis Group

Centers for Medicare and Medicaid Services

7500 Security Boulevard

Room C1-13-18

Baltimore MD 21244

RE:  Proposed Decision Memo for Clinical Trial Policy (CAG-00071R)

Dear Dr. Phurrough:

Thank you for the opportunity to provide comments to the Center for Medicare and Medicaid Services (CMS) on the proposed revisions to the Medicare National Clinical Research Policy (CRP).  I am submitting comments on behalf of ASBMT and the NMDP.

ASBMT is the American Society for Blood and Marrow Transplantation, an association of 1,500 clinicians and investigators promoting research, education and clinical practice in the field of blood and marrow transplantation and related cellular therapy.

NMDP is the National Marrow Donor Program, established in 1987 to facilitate unrelated donor allogeneic transplants.  The NMDP maintains a registry of more than 6 million adult volunteer donors in the United States and a listing of more than 50,000 cord blood units.  It has coordinated more than 25,000 allogeneic transplants and conducts research as a partner of the Center for International Blood and Marrow Transplant Research (CIBMTR) and The Blood and Marrow Transplant Clinical Trials Network (BMT CTN).

ASBMT and NMDP strongly endorse CMS interest in re-examining support of clinical trials.  We view clinical trials as fundamental to advancing stem cell transplantation as effective and often curative therapy for a variety of hematologic malignancies, inherited diseases and blood disorders.  Improvements in outcomes resulting from clinical trials in transplantation have positioned transplantation therapies as part of the armamentarium in treating Medicare beneficiaries with certain diseases.  Investigation of the utility of stem cell transplantation in a variety of other diseases has been hampered, however, by the present policies which limit funding of clinical trials.

ASBMT and NMDP jointly support most of the proposed policy changes, but have concerns about several.  Those concerns are addressed below.

PROPOSAL 4 – REGARDING MEDICARE-SPECIFIC STANDARDS

ASBMT and NMDP believe that research studies must be vetted through a peer review process, and only scientifically sound data should be offered to the public.  Peer review has been an effective method for verifying the authenticity as well as outcomes of clinical research.  Premature release of data (whether encouraging or disappointing) that has not been reviewed could be misleading for both the public and the scientific community.

For research that is not published through peer review, an alternative might be to require a summary paragraph in the federal registry of clinical trials addressing certain elements listed in the proposal.  These should include accrual information as well as any unanticipated toxicity.

We applaud the mention of the health care disparities among many subpopulations.  Transplant services are needed within all groups.  However, we are concerned that the proposed policy changes would exclude from clinical research studies Medicare beneficiaries younger than 65 years of age.  We believe that all Medicare beneficiaries should be allowed to participate in clinical research studies. Younger beneficiaries may have diseases that are not common in the general Medicare population, but inclusion in clinical trials may benefit the general population.

Furthermore, some older beneficiaries may meet criteria for therapies such as reduced intensity allogeneic transplantation, autologous transplantation or newer applications of cellular therapy.  Medicare recipients should not be denied the opportunity to participate in trials that advance therapeutic outcomes addressed by these treatment modalities.  A focus that is limited to those 65 years of age and older would undermine the intent of the clinical research policy to address health care disparities among all Medicare beneficiaries.

PROPOSAL 6 – REGARDING DEEMING AND SELF-CERTIFICATION FOR QUALITY CLINICAL TRIALS

ASBMT and NMDP are concerned that removal of the self certification process may result in a disincentive for patients to enroll in non-qualified trials.  In addition the proposal to remove IND-exempt studies is alarming.  A majority of the advances in hematopoietic stem cell transplantation have occurred in IND-exempt studies.  Such studies have examined questions regarding new uses of drugs in therapy, supportive care, transplant indications or even patient selection.  Exclusion of IND-exempt studies would serve as a major obstacle to clinical research and patient access to state-of-the-art transplant care.  An alternative would be to develop criteria for self- certification and establish local review committees that can determine if a study meets the criteria of both the General Standards for a Scientifically and Technically Sound Research Study and the Medicare-specific standards.

The IDE issue has an additional dimension that needs attention.  In the case of cellular therapies such as hematopoietic stem cell transplantation and immunotherapy, the FDA in some instances considers cellular processing procedures and products a biologic – for example, the infusion of Natural Killer (NK) cells – and these fall under IND review.  For other indications, the agency considers the processing procedure and products a device – for example, CD3 depletion of an allogeneic cell product – and therefore these fall under IDE review.  The FDA subjects both INDs and IDEs to rigorous review.  Medicare beneficiaries should not be denied coverage for trials involving IDEs.

Attention also should be given to appropriate “grandfathering” of patients currently enrolled in clinical trials. 

We would be pleased to assist CMS in the development of criteria for the inclusion of IND-exempt studies.

Thank you again for the opportunity to submit these comments.  Please let us know if you have any questions or need clarification.