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Comments to CMS on Clinical Trial Coverage Proposal

May 9, 2007

Steve Phurrough, MD, MPA
Coverage and Analysis Group
Center for Medicare and Medicaid Services
Hubert H. Humphrey Building, Room 445-G
200 Independence Avenue, SW
Washington, DC 20201

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

Dear Dr. Phurrough:

AABB (formerly known as the American Association of Blood Banks) appreciates the opportunity to comment on the Center for Medicare and Medicaid Services’ (CMS) proposed revisions to the Medicare National Clinical Research Policy. AABB is a professional association dedicated to advancing transfusion medicine and cellular therapies. AABB’s members include approximately 1,800 institutions, including hospital-based blood banks and laboratories, transfusion services and blood and bone marrow collection facilities, as well as approximately 8,000 individuals involved in blood, bone marrow, cord blood and peripheral blood stem cell collection, processing, storage and infusion.

Clinical research plays a critical role in promoting quality care for patients requiring transfusions as well as those battling certain cancers and other life-threatening conditions treated with hematopoietic stem cell transplants. It is important that CMS’ clinical research policy be designed and implemented in a manner so as not to discourage patients from participating in high quality clinical trials. Although AABB supports the overall goals of CMS’ proposed policy as well as many of the proposed edits to this policy, the association has several significant concerns regarding the latest proposal. Prior to its implementation, AABB urges CMS to amend and/or clarify the revised policy to address the following issues.

Release of Study Results

AABB agrees that it is extremely important that the public have access to critical outcomes information. However, the association is concerned that if outcomes are released without going through an appropriate peer-review process, the released information may mislead the public and compromise good research. Long-accepted peer-review practice offers the best means of verifying the scientific legitimacy of outcomes information. Therefore, AABB recommends that CMS amend its proposal to state that research studies should undergo a peer review process and all data found to be scientifically sound must be made available to the public. This requirement should only apply to the principal organization in a multi-site trial, not to each individual site.

Medicare Populations

AABB believes that CMS’ proposal requiring the protocol to discuss how results will generalize to the Medicare population is problematic and may deter Medicare beneficiaries from participating in potentially life-saving clinical trials. First, this proposal appears to focus almost exclusively on elderly Medicare beneficiaries, and not the population of Medicare beneficiaries who are under age 65, but are disabled. These individuals – a population that includes ill patients requiring hematopoietic stem cell transplants and/or blood transfusions – must not be shut out of Medicare-covered clinical trials.

In addition, CMS should be careful not to deny Medicare beneficiary access to trials that even though they are not easily generalizable to the 65-year or older Medicare population offer these patients the best possible care. For example, studies involving hematopoietic stem cell transplantation may be restricted to patients under 65 or 67 due to related toxicities to older patients.

AABB requests that CMS amend its proposal to allow the researcher to state the reasons why a particular study may not be generalizable to the entire Medicare population.

Routine Costs

AABB recommends that CMS further refine its definition of routine costs to distinguish between events that are research-related and those that are due merely to the underlying disease or standard treatment. AABB urges CMS to add language stating that a research-related injury is defined as “something that would not reasonably be expected outside of participation in the study.”

Approval Processes

AABB is concerned that CMS’s proposal to no longer allow for coverage of IND-exempt studies could significantly deter Medicare beneficiaries from participating in valuable clinical trials. Such investigator-initiated trials help answer discrete, yet important, questions involving new combinations or dosages of potentially life-saving drugs. Over the next few years, there will be several cellular therapy products licensed as biologicals. As more such products are approved and this practice of medicine continues to evolve rapidly, a growing number of patients will be treated with IND-exempt off-label use of cellular therapy products. Medicare beneficiaries, including those battling cancer and other non-malignant life-threatening conditions, should have access to these promising new treatments. AABB requests that CMS not eliminate this important deemed coverage for IND-exempt studies.

Rather than eliminating self certification, AABB recommends that CMS allow for self-certification based on the following set criteria. These criteria should include establishing local Protocol Review Committees consisting of clinical investigators, biostatisticians and basic scientists. This process would determine if the study met both the “General Standards for a Scientifically and Technically Sound Research Study” and the “Medicare-Specific Standards.”

In addition, AABB recommends that CMS amend its proposed language prohibiting coverage for studies conducted under an IND “when the FDA has reviewed the protocol and the IND has not been put on hold.” This language should be edited to instead read “when the FDA has reviewed the protocol and the IND is currently not on hold.” It is not uncommon for FDA to put an IND on hold pending receipt of requested information and then release the hold. Once an IND is no longer on hold, the trial should be eligible for Medicare coverage.

Finally, AABB is very concerned about the need for Medicare coverage of trials under an investigational device exemption (IDE). Although we recognize that this issue may not be covered under this particular clinical research policy, AABB believes that the IDE issue requires attention. In the case of cellular therapy (bone marrow/stem cell transplant and immunotherapies), FDA in certain instances considers cellular product processing procedures and the resultant product as a biologic (i.e., the infusion of Natural Killer (NK) cells) and therefore these fall under the IND review mechanism. For other indications, the agency considers the processing procedure and the resultant product a device (i.e., CD3 depletion of an allogeneic cell product) and therefore these fall under the IDE review mechanism. FDA subjects both INDs and IDEs to rigorous review. Medicare beneficiaries, therefore, should not be denied coverage for trials involving IDEs.

Thank you for the opportunity to comment on this important coverage policy that will significantly impact Medicare beneficiary access to high quality cellular therapy and transfusion medicine related care. If you have any questions or require further information, please contact Theresa Wiegmann, Director, Public Policy at 301-215-6554 or


Karen Shoos Lipton, JD
Chief Executive Officer