Food and Drug Administration, U.S. Department of Health and
The Food and Drug Administration today announced a plan for a
new, comprehensive regulatory framework for products derived
from cells and tissues that would protect the public health
through innovative common-sense government oversight. Today's
proposal is the FDA's sixth reinvention reform as part of the
Clinton Administration's Reinventing Government Initiative.
"This new regulatory framework, developed after discussions
with industry, academics and professional groups, will allow
greater flexibility and innovation in this promising field of
medicine," said Vice President Al Gore. "At the same time,
safeguards are maintained to protect the public health."
"Human tissues have wide uses in medicine, including skin
replacement after severe burns, tendons and ligaments to repair
injuries, and corneas to restore eyesight," said HHS Secretary
Donna E. Shalala. "Now that science is providing even more new
ways of using tissues, FDA has developed an innovative
regulatory approach to allow these novel products to benefit
patients as soon as possible."
In recent years, scientists have developed innovative methods
of manipulating and using human cells and tissues for
therapeutic purposes. For example, using somatic cell therapy,
scientists are studying how to manipulate and use human cells
to treat viral infections (including HIV), Parkinson�s disease,
and diabetes. Other tissue research includes the treatment of
diseases and medical conditions by using "cord blood" (from the
placenta and umbilical cord) and processed structural cells and
This new regulatory framework, which provides a tiered approach
with the level of regulation proportionate to the degree of
risk, focuses on three general goals:
* preventing the unwitting use of contaminated tissues with
the potential for transmitting infectious diseases;
* preventing improper handling or processing that might
contaminate or damage tissues;
* and ensuring that clinical safety and effectiveness are
demonstrated for certain cells and tissues. These include
highly processed tissues, as well as those that are used for
other than their normal purposes, those that are combined with
non-tissue components, and many that are used for metabolic
purposes (such as to treat diabetes).
This tiered approach will impose little or no regulation for
some products, with the degree of oversight increasing with the
potential risk, so that extensively processed and novel
products would require FDA�s approval before they could be
marketed. All tissue processing facilities would be required
to register with the FDA and to list their products, and all
labeling and promotion of these products would have to be
clear, accurate, balanced, and non-misleading.
In designing this new approach, FDA focused on five broad
public health and regulatory questions: (1) How can the spread
of communicable diseases be prevented? (2) What processing
controls are needed to prevent contamination and preserve the
integrity of cells and tissues? (3) How can clinical safety
and effectiveness be assured? (4) What labeling is necessary,
and what kind of promotion is permissible, so that the product
may be used properly? and (5) How can FDA best monitor and
communicate effectively with the cell and tissue industry?
The following examples show how the tiered approach would work:
* FDA would not regulate cells and tissues removed from and
transplanted into the same person in a single surgical
* For most conventional and reproductive tissues that are
minimally processed and used for their normal functions, FDA's
oversight would focus on proper handling and on ensuring that
the products are not infectious.
Registration, product listing, and adverse event reports would
be the only submissions required.
* FDA would require that all tissues (except those removed and
transplanted back into the same patient in one surgical
procedure) be handled according to �good tissue practices.� FDA
would also prescribe procedures for testing the tissue for
infectious agents and screening the donor about potential
exposure to disease agents.
* For tissue stored for use in the same person from whom it
was obtained (or in a sexually intimate partner of a
reproductive- tissue donor), FDA would recommend but not
require that similar testing and screening procedures be
followed. To protect health care workers, FDA would also
require labeling according to whether the tissue poses a
* For most tissue transplanted from one person to another, FDA
would require infectious disease testing, donor screening, and
* For some tissues, FDA would require controlled clinical
trials and pre-market approval to demonstrate safety and
effectiveness. This requirement would apply to tissues and
cells processed to alter their biological or functional
characteristics; tissues and cells used to perform other than
their normal functions; many tissues and cells used for
metabolic purposes; and tissues and cells that are combined
with medical devices, drugs, or other biological products.
Somatic cell therapy and gene therapy would be covered by this
category of regulation, as would many forms of stem cell
FDA's current regulation of unmodified or minimally modified
tissues that traditionally have been used for replacement
purposes (such as bone replacement) focuses on preventing the
spread of communicable disease. In December 1993, FDA issued
an interim final rule that required the testing of tissue
donors for certain transmissible disease such as HIV infection
and hepatitis, as well as the screening of donors for
behavioral risk factors. FDA intends to finalize these
requirements soon. The requirements for this new regulatory
framework would be phased in over the next two to three years.
The new regulatory framework does not include whole organs or
minimally-manipulated bone marrow, which are both regulated by
the Health Resources and Services Administration. It does not
cover blood products -- such as whole blood, red blood cells,
platelet and plasma -- for transfusion or animal-derived
tissues, which FDA regulates under existing authorities.
Additional products covered by other regulations and standards
include human milk, collagen, and growth factors.