May 5, 2010

ACBSCT recommends to the Secretary that an expert panel be convened to review and determine indications for stem cell transplantation.

Background

Allogeneic hematopoietic transplantation is an effective, potentially curative treatment for a broad range of hematologic, malignant, immunologic and genetic diseases. It is important that patients have access to this treatment modality. Public and private medical insurance providers have independently determined which diagnoses should be covered. There is no authoritative body which determines the accepted indications for hematopoietic transplantation, and there is considerable variability in coverage policies and covered diagnoses. Some payers have different criteria for related and unrelated donor transplants, degrees of HLA matching and different hematopoietic cell sources.

The ACBSCT recommends that an expert panel be convened, including experts regarding hematopoietic transplantation, experts in the candidate diseases, and representatives of the medical insurance industry, public payers and patient advocates, to develop a consensus, evidence-based list of diagnoses for which hematopoietic transplantation is an accepted standard of care. The expert panel should also consider whether there should be different indications for related and unrelated donor transplants, degrees of HLA match and mismatch, and different hematopoietic cell sources (bone marrow, peripheral blood progenitor cell, cord blood transplants). This panel will publish its conclusions and meet from time to time to update this listing. The panel will report to the ACBSCT Council, which will make recommendations to the Secretary.

ACBSCT recommends that the Secretary mandate that Medicare and Medicaid cover patient participation in clinical trials involving hematopoietic transplantation.

Background

Hematopoietic transplantation is a rapidly evolving area where incremental changes in treatment and supportive care have progressively improved safety and treatment outcomes. This has occurred through clinical trials. Improved approaches for hematopoietic transplantation are needed. New and promising treatments are being rapidly designed to improve safety and effectiveness.

It is important that patients have access to participate in clinical trials. Many insurance carriers do not cover standard patient care costs for patients participating in clinical trials. This deprives patient access to the most promising new treatments and limits the further vital improvements in the standards of care for hematopoietic transplantation.

Some years ago, a Presidential Executive Order mandated that Medicare cover patient care costs for clinical trials, but this has not been effectively implemented for studies of hematopoietic transplantation. Many States have laws requiring insurance carriers to cover standard patient care costs while participating in clinical trials, and this is included in the recent federal health care reform legislation, but only becomes active in 2014. This remains a major problem for patients.

The ACBSCT recommends that the Secretary mandate that Medicare and Medicaid cover the standard patient care costs for patients who are participating in clinical trials involving the use of hematopoietic transplantation and supported by or conducted under the auspices of the National Institutes of Health or National Cancer Institute (NCI), NCI-designated cancer centers and cooperative groups, the Department of Veterans Affairs, or a Food and Drug Administration Investigational New Drug (IND)/Investigational Device Exception (IDE). The ACBSCT recommends that the Secretary encourage private insurance carriers to immediately provide coverage for standard patient care costs for patients participating in clinical trials involving hematopoietic transplantation.

ACBSCT recommends to the Secretary that cord blood collections be increased and improved.

Awareness of cord blood donation and the procurement of high quality products is critical to providing life saving cord blood grafts for patients in need of stem cell therapies. Collection practices yielding larger cord blood units, particularly from ethnically and racially diverse donors, will increase access to curative treatments for patients in need.

1. Awareness: The collection of high quality products depends on the commitment of the medical community and the motivation of families to positively impact the life of an unrelated individual. Private banking has had greater visibility compared to public cord blood donations in large part because millions of dollars are spent for marketing by private banks. The materials advertising private storage are typically a parent’s first exposure to the concept of cord blood banking and influence their decisions for managing the disposition of their baby’s cord blood. The ACBSCT recommends that the Secretary promote education about public donation as another front-line option in regions where cord blood banking is available.
    
2. Parent Education: Tools have been created to educate parents about the potential disposition of their baby’s cord blood. The ACBSCT recommends that the Secretary guide expectant parents to the HRSA Program’s website for accurate, unbiased information and refer expectant parents to position statements from professional societies which are accessible. In addition, as state-based educational websites are developed, similar information about public donation and private banking should be posted. Packets presenting this information, including the Pregnancy Passport, could be provided through HRSA and the C.W. Bill Young Cell Transplantation Program.
    
3. Medical Professional Education: To support an efficient collection process which maximizes effort and resources, physicians must be educated. Cord blood collection programs and federally funded websites are sources for information regarding donation options and current applications for cord blood products. The ACBSCT recommends that the Secretary encourage medical school and residency programs, particularly obstetrical training, introduce cord blood banking concepts, and provide updates on clinical and research applications of cord blood therapies.
    
4. Compensation: Many physicians support the concept of cord blood donation. When obstetricians collect cord blood, the donation opportunities are enhanced for most delivering mothers. Although compensating medical professionals for cord blood collection may increase the operational costs of public cord blood banks, it may confer a greater value for this resource and service. The ACBSCT recommends that, if offered, compensation must not incentivize medical professionals to alter safe delivery practices or to include ineligible donors. The ACBSCT further recommends that banks take all feasible steps to ensure that mothers or their insurance companies are not charged for costs associated with cord blood collection.
    
5. Legislation: Cord Blood Legislation has been enacted in at least 18 states to date and is initiated by private cord blood bank marketing rather than the incentive to inform expectant parents about cord blood banking options. The ACBSCT recommends that the Secretary encourage the creation of harmonized legislation that asserts accurate and balanced language regarding public donation and private banking.
    
6. Licensing: As the industry proceeds to obtain product licensure, cord blood banks may infer a requirement to strictly standardize practices and forego novel approaches to procure and manufacture safe, quality products. The ACBSCT recommends that FDA consider innovative collection alternatives, particularly for public donation, as the industry pursues licensure of unrelated allogeneic cord blood as a biologic product.
    
7. Resources: With an annual rate of four million births per year, it appears that collection opportunities would be sufficient to create an inventory that adequately addresses diversity and patient access. However, processing, testing and storage costs limit the number of products that can be banked. Over the next few years, facilities that obtain licensure from the FDA will be able to support manufacturing through commercial charging mechanisms, but this revenue will be limited by what the market will bear. The ACBSCT recommends that the Secretary support the establishment of best practice guidelines for industry, to exploit economies of scale, and encourage efficient, cost effective practices.