Xenotransplantation Action Plan

Regulatory Policy and Guidance Development

Xenotransplantation products are subject to regulation by the FDA (e.g., under section 351 of the Public Health Service Act (42 U.S.C. 262) and the Federal Food, Drug and Cosmetic Act (21 U.S.C. 321 et. seq.)). In accordance with the statutory provisions governing premarket development, xenotransplantation products are subject to FDA review and approval. Investigators of such products should obtain FDA review of proposed xenotransplantation clinical trials before proceeding.

FDA intends to publish guidance documents to assist sponsors and investigators interested in conducting clinical trials in the field of xenotransplantation. These documents will provide reasonably detailed and timely pragmatic guidance to sponsors regarding xenotransplantation product safety and clinical trial development, including specific recommendations for the procurement and screening qualification of source animals, the manufacture and testing of xenotransplantation products, preclinical testing, clinical trial design, and post-transplant monitoring/surveillance of recipients of xenotransplantation products. FDA will provide notice and invite public comment on these draft documents.

Application Review

Currently, several xenotransplantation clinical trials are under FDA oversight. In order to respond efficiently to the data submitted to the agency in xenotransplantation product applications, CBER has developed a mechanism for the systematic and regular evaluation of the scientific and clinical literature relevant to xenotransplantation and submissions to xenotransplantation product files. A Xenotransplantation Product Reviewer Working Group, consisting of the review staff responsible for the review of xenotransplantation submissions (clinical, product, and pharm-tox reviewers, and veterinary staff) meets regularly to:

  • discuss application of the principles set forth in relevant FDA regulations,
  • review and discuss current scientific and medical data and literature relevant to xenotransplantation
  • review and discuss the current status of xenotransplantation applications submitted to the agency
  • discuss the unique issues that these products may present
  • highlight areas of concern where further expert advice may be needed

This provides for a consistent review of xenotransplantation applications and should facilitate the recognition of patterns, trends, and/or common problems that may be associated with xenotransplantation products. This data evaluation and management process is linked to the regulatory process and is applied during regulatory decision making and policy design.

Scientific Investigations and Research

Research conducted at CBER has been instrumental to the understanding of safety issues associated with xenotransplantation. CBER is engaged in scientific investigations of known and emerging infectious agents and immunological hurdles that will need to be overcome for the safe and effective use of xenotransplantation products. The results of these studies have helped CBER in its safety assessment including assessment of risk and the development of diagnostic methods and standards. CBER researchers continue to develop assays appropriate for safety monitoring and are working with sponsors and collaborating with other government scientists in this development.

These research scientists apply their unique expertise in performing regulatory review and to the development of regulatory policy for xenotransplantation. Working groups analyze data and events, develop and propose strategies for appropriate studies, risk assessment, prevention, communication and agency response or regulatory action (e.g., request for more data from sponsors, request for particular product assays, placement of clinical hold) and discuss these proposals and strategies with advisory committees when needed or at public meetings as appropriate.

This process provides improved coordination of efforts to address the potential safety issues associated with xenotransplantation and provides a mechanism whereby infectious agents and diseases that may be associated with xenotransplantation can be rapidly recognized and an appropriate and timely regulatory response generated.

Public Discussion and Consultation

FDA has sponsored, planned or participated in numerous open public meetings and workshops, both domestic and international that focused in whole or in part on xenotransplantation. These activities are essential for both sharing information and receiving public input on issues relevant to xenotransplantation.

In 1997, FDA formed a Xenotransplantation Subcommittee of the Biological Response Modifiers Advisory Committee (BRMAC) as an ongoing mechanism for open discussions of the scientific, medical, social, and ethical issues and the public health concerns raised by xenotransplantation, as well as specific ongoing and proposed protocols. Open public meetings update the committee and the general public on the issues associated with xenotransplantation and the development of FDA policy regarding the regulation of xenotransplantation products.

FDA will continue to promote an open public process to discuss the issues related to xenotransplantation.

DHHS Xenotransplantation Committee

The DHHS Xenotransplantation Committee oversees departmental initiatives to address the public health issues raised by xenotransplantation. It is comprised of members from FDA, CDC, NIH and HRSA and is administered through the Office of the Assistant Secretary for Planning and Evaluation/Office of Science Policy.

DHHS has established the Secretary's Advisory Committee on Xenotransplantation (SACX), which will consider the full range of complex scientific, medical, social, ethical, and public health concerns raised by xenotransplantation, and make recommendations to the Secretary on policy and procedures.

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Updated: December 17, 2001