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 tissues.

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 procedure.

* 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 potential biohazard.

* For most tissue transplanted from one person to another, FDA would require infectious disease testing, donor screening, and processing controls.

* 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 therapy.

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.