Food and Drug Administration - May 20, 2004
FDA published on May 20, 2004 a new Final Rule on donor
eligibility for human tissues and cells (Eligibility
Determination for Donors of Human Cells, Tissues, and Cellular
and Tissue-Based Products), and draft guidance (Guidance for
Industry Eligibility Determination for Donors of Human Cells,
Tissues, and Cellular and Tissue-Based Products) related to donor
screening and eligibility. These documents include HIV, among a
variety of conditions for which cell and tissue donor screening
would be required.
The final rule establishes donor eligibility criteria for donors
of human cells, tissues, and cellular and tissue-based products
(HCT/Ps) to help prevent the transmission of communicable disease
when these products are transplanted. This new rule is part of
the Agency's plan to regulate tissues and related products with a
comprehensive, risk-based approach. The requirements are
comprehensive, yet adequately flexible, and they provide needed
protections for patients without imposing unnecessary regulation.
Along with the potential for great benefit, products derived from
the human body such as HCT/Ps may pose risks of transmitting
communicable diseases, especially if donors are not properly
screened and tested. For this reason, this final rule requires
that, before the use of most HCT/Ps, the cell or tissue donor
must be found eligible, based on the results of screening for
risk factors and testing for relevant communicable diseases. In
most cases, a donor who tests positive for a particular disease
or who possesses clinical signs or risk factors for such a
disease would be considered ineligible, and cells and tissues
from that donor would not ordinarily be used.
The new rule on donor eligibility expands the types of
human-derived products that come under FDA regulation in an
effort to prevent the transmission of diseases through
transplantation. Some 350,000 transplants were performed in
1990. Approximately one million are expected to be performed in
2004. The rule pertains to donors of traditional tissues such as
musculoskeletal, skin and eye tissues that have been required
since 1993 to be screened and tested for HIV, Hepatitis B virus
(HBV) and Hepatitis C virus (HCV). Under this new rule, however,
reproductive tissue (semen, ova, and embryos), hematopoietic stem
cells derived from cord blood and peripheral blood sources
(circulating blood sources as opposed to bone marrow), cellular
therapies and other innovative products are also regulated.
In addition to including a broader range of tissues and cells,
the new rule extends the scope of protection against additional
communicable diseases that can be transmitted through
transplanted tissues and cells. The new regulation adds
requirements to screen for human transmissible spongiform
encephalopathies, including Creutzfeldt-Jakob disease (CJD), and
to screen and test for syphilis. Screening and testing for still
other relevant communicable disease agents (human T-lymphotropic
virus (HTLV) would be required for viable cells and tissue rich
in leukocytes such as semen and hematopoietic stem cells. For
reproductive tissues, Chlamydia trachomatis and Neisseria
gonorrhoeae also pose potential risks and are included.
The new rule also provides a framework for identifying emerging
diseases that may pose risks to recipients of transplanted HCT/Ps
and for which appropriate screening measures or testing are
available. Thus, this new regulation gives FDA the flexibility to
rapidly address new disease threats as they appear, providing
substantial new protections for patients receiving tissue
transplants. Examples of such diseases include West Nile virus,
Severe Acute Respiratory Syndrome (SARS) and sepsis.
The behavioral risk factors that are used to screen donors are
consistent with 1994 Centers for Disease Control and Prevention
(CDC) guidelines for preventing transmission of HIV through organ
and tissue transplantation and with the scientific literature as
reviewed by CDC in 2000. Professional groups, such as the
American Association of Tissue Banks, have adopted the
recommendations contained in the CDC guidelines.
The rule also contains requirements related to record-keeping,
quarantine, storage and labeling of the HCT/Ps, all important to
the prevention of disease transmission.
Certain exceptions from the requirements for donor eligibility
testing and screening exist. These tissues and cells include:
-autologous HCT/Ps (Cells or tissue removed from and transplanted
back into the same person)
-reproductive cells or tissues from a sexually intimate partner.
Other cells and tissues are subject to donor testing and
screening, but may be used with appropriate communication,
labeling and documentation of the relevant results even if the
donor is determined to be ineligible. These are:
-reproductive cells or tissues from a directed donor,
-those for use in first or second-degree blood relatives, and
-those that meet a documented urgent medical need.
The new framework does not include whole organs or
minimally-manipulated bone marrow, which are regulated by the
Health Resources and Services Administration (HRSA), another
agency of the Department of Health and Human Services. It also
does not cover blood products for transfusion or products derived
from animals, which FDA regulates under the biologics license
requirements and other applicable regulations.
The final rule becomes effective on May 25, 2005.
The new Final Rule is accompanied by draft guidance that provides
recommendations for complying with the requirements in the donor
eligibility rule. Comments on the draft guidance will be accepted
until August 23, 2004 (90 days from the publication date) for
consideration in the final guidance.
The Notice of Final Rule, and the draft guidance document are
available on FDA's website:
Federal Register Notice:
Draft Guidance Document:
In addition, a "Questions and Answers" document has been prepared
to address basic points about the Donor Eligibility Final Rule
and the Draft Guidance.
Questions and Answers
Richard Klein, HIV/AIDS Program Director
Office of Special Health Issues - Food and Drug Administration