Human Tissue Banks:

FDA Taking Steps to Improve Safety, but Some Concerns Remain

HEHS-98-25: Published: Dec 5, 1997. Publicly Released: Dec 22, 1997.

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Pursuant to a congressional request, GAO evaluated the Food and Drug Administration's (FDA) oversight of transplanted human tissue as well as potential safety problems, focusing on: (1) gaps in the current regulation; and (2) whether and how FDA plans to address them in the approach it has proposed for regulating tissue banking.

GAO noted that: (1) FDA is just now expanding its oversight to improve tissue-banking safeguards in the growing field of tissue-based therapies; (2) FDA has proposed a regulatory approach that is much broader in scope than the current regulation; (3) still in its formative stages, the regulatory approach describes a risk-based approach that industry has generally received well; (4) FDA's proposed approach would alleviate three safety problems not covered by the current regulation: (a) FDA has no universal registry of tissue facilities currently operating in the United States; (b) the current regulation does not cover reproductive tissue or stem cell facilities; and (c) there are numerous instances of misleading and false advertising in private cord blood banking; (5) two problems that FDA does not address in the current regulation or in the proposed approach are: (a) some facilities obtain informed consent after infant delivery, raising safety and ethical concerns about cord blood; and (b) FDA has no current or proposed requirements for disclosing to recipients whether genetic tests have been performed on reproductive tissues and stem cells; and (6) four instances in which FDA's proposed approach inadequately addresses potential safety problems not covered in the current regulation are: (a) facilities would be required to record and investigate errors and accidents but not to report them to FDA; (b) the proposed approach only minimally mentions tracking tissues as good tissue practices and then only in respect to controlling disease transmission; (c) FDA will not require premarket submissions when cells or tissues are to be used in the person from whom they are obtained or in a close blood relative; and (d) FDA has proposed that certain tissue products be subject to premarket approval and that it evaluate processing techniques used on those products, but, for minimally manipulated tissue products, would require only that the validation of the procedure be documented and available when FDA inspects facilities.

Recommendations for Executive Action

  1. Status: Closed - Implemented

    Comments: FDA intends to require IND applications for unrelated allogeneic cord blood stem cells if the agency determines that adequate safety and efficacy data are not available to license such products. A notice requesting recommended standards along with supporting clinical and laboratory data for peripheral and placental/umbilical cord blood stem cell products was published in the Federal Register on January 20, 1998. However, the proposed regulations will not specifically require that the good tissue practices and donor suitability criteria developed for unrelated allogeneic cord blood be applied to family-related allogeneic cord blood collection.

    Recommendation: FDA should add to its oversight plans provisions that would require tissue facilities that collect, store, process, or distribute allogeneic peripheral stem cells or any cord blood stem cells to make premarket submissions if FDA determines that adequate safety and efficacy data are not available to license such products.

    Agency Affected: Department of Health and Human Services: Public Health Service: Food and Drug Administration

  2. Status: Closed - Not Implemented

    Comments: The Final Rule for Human Tissue Intended for Transplantation, published July 29, 1997, requires that there be documentation of the receipt and distribution of human tissue. FDA also plans to publish a proposed rule in the Federal Register to address good tissue practices. This rule would include requirements for tracking tissues from donor to recipient and from recipient to donor. Currently, FDA does not have authority to implement recommendation.

    Recommendation: FDA should add to its oversight plans provisions that would require facilities that collect, store, process, distribute, or transplant human tissues to establish validated systems to track tissues to consignees and recipients.

    Agency Affected: Department of Health and Human Services: Public Health Service: Food and Drug Administration

  3. Status: Closed - Not Implemented

    Comments: The Department of Health and Human Services Secretary's Advisory Committee on Genetic Testing is actively taking Steps to evaluate this recommendation. Currently, FDA does not have authority to implement recommendation.

    Recommendation: FDA should add to its oversight plans provisions that would require disclosure of genetic tests that have been performed on donated reproductive tissues.

    Agency Affected: Department of Health and Human Services: Public Health Service: Food and Drug Administration

  4. Status: Closed - Implemented

    Comments: FDA plans to promulgate, through notice and comment rulemaking procedures, requirements for adverse event reporting. However, FDA feels that reporting of errors and accidents would be resource-intensive for the agency and for industry. FDA believes that budgeting constraints would not allow FDA to review and evaluate these reports. All facilities would be required to maintain records of errors and accidents, and to make such records available for FDA review during inspections.

    Recommendation: FDA should add to its oversight plans provisions that would require tissue facilities to report serious error and accidents to FDA (serious to be defined in consultation with industry representatives).

    Agency Affected: Department of Health and Human Services: Public Health Service: Food and Drug Administration

  5. Status: Closed - Not Implemented

    Comments: FDA does not believe that the timing of informed consent affects the safety of the tissue, especially in regard to procurement of umbilical cord blood. At this time, FDA does not concur with this recommendation, but may modify its position if the agency learns that the timing of informed consent affects the safety of tissue products.

    Recommendation: FDA should add to its oversight plans provisions that would require tissue facilities to obtain informed consent before procuring any tissues intended for transplantation from living donors.

    Agency Affected: Department of Health and Human Services: Public Health Service: Food and Drug Administration

  6. Status: Closed - Not Implemented

    Comments: FDA plans to require that labeling and promotion for tissue products be truthful, clear, balanced, accurate, and non-misleading. FDA plans to publish a proposed rule in the Federal Register to address good tissue practices. This rule would address labeling and claims issues as well as labeling controls. The draft proposed rule is under review. Currently, FDA does not have authority to implement recommendation.

    Recommendation: The Secretary of Health and Human Services should direct FDA to take action in several areas to improve the safety and efficacy of donated human tissue and to increase FDA's ability to regulate tissue facility activities. FDA should move ahead with its plan to require facilities that collect and store cord blood to provide accurate oral and written communication to consumers with regard to the state of knowledge of collection, processing, and storage techniques, as well as the likelihood of requiring cord blood transplantation, and to portray the risks and benefits relative to other therapies.

    Agency Affected: Department of Health and Human Services

  7. Status: Closed - Not Implemented

    Comments: FDA's proposal that manufacturers of human cellular and tissue-based products register with the FDA and list their products (see Federal Register, May 14, 1998) would include stem cell and reproductive facilities. FDA also plans to publish two proposed rules in the Federal Register to address donor suitability, good tissue practices, and enforcement. The first, "Suitability Determination for Donors of Human Cellular and Tissue-Based Products," was signed out of FDA on February 23, 1999. The second, a draft proposed rule describing current good tissue practice, inspection, and enforcement issues, is under review. These proposals will discuss requirements for stem cell and reproductive tissue facilities. Currently, FDA does not have authority to implement recommendation.

    Recommendation: The Secretary of Health and Human Services should direct FDA to take action in several areas to improve the safety and efficacy of donated human tissue and to increase FDA's ability to regulate tissue facility activities. FDA should move ahead with its plan to require reproductive and stem cell facilities to adhere to all requirements of the current regulation.

    Agency Affected: Department of Health and Human Services

  8. Status: Closed - Implemented

    Comments: FDA has proposed that manufacturers of human cellular and tissue-based products should register with the FDA and list their products. This proposal was published in the Federal Register on May 14, 1998 ("Establishment Registration and Listing for Manufacturers of Human Cellular and Tissue-Based Products").

    Recommendation: The Secretary of Health and Human Services should direct FDA to take action in several areas to improve the safety and efficacy of donated human tissue and to increase FDA's ability to regulate tissue facility activities. FDA should move ahead with its plan to require tissue facilities, including reproductive and stem cell facilities, to register with FDA.

    Agency Affected: Department of Health and Human Services

  9. Status: Closed - Not Implemented

    Comments: With respect to those tissue products that pose potentially higher risks (e.g., more than minimally manipulated, not homologous, etc.), FDA intends to require premarket review, which would include evaluation of the processes used in manufacturing the product. FDA has noted, however, that because of limited resources, it is necessary to make a distinction between procedures that minimally manipulate the tissue and procedures that manipulate the tissue to a greater degree. For the former procedures, FDA will require only that validation be documented and available when FDA inspects the facility. In addition, FDA is evaluating and developing strategies for the regulation of "ancillary products" used in the manufacture of cellular and tissue-based products. Currently, FDA does not have authority to implement recommendation.

    Recommendation: FDA should add to its oversight plans provisions that would require tissue facilities to inform FDA of the types of processing techniques used on tissues and to supply information on the safety and efficacy of these techniques.

    Agency Affected: Department of Health and Human Services: Public Health Service: Food and Drug Administration

 

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