Regulatory Focus™ > News Articles > FDA Withdraws Proposed Rule on Public Disclosure of Info on Unapproved Gene Therapies

FDA Withdraws Proposed Rule on Public Disclosure of Info on Unapproved Gene Therapies

Posted 10 November 2016 | By Zachary Brennan 

FDA Withdraws Proposed Rule on Public Disclosure of Info on Unapproved Gene Therapies

The US Food and Drug Administration (FDA) on Thursday announced it is withdrawing a proposed rule from 2001 that would have required the public disclosure of summaries of safety and effectiveness data, in addition to other information, from pre-market clinical trials of gene therapies and transplanted non-human tissues to humans, known as xenotransplantation.

“FDA has reconsidered our position on this issue and deemed our concerns from 2001 outdated. We will continue to assess whether rulemaking in this area is necessary, and if so, we will proceed with a new proposed rule,” the agency said in a Federal Register notice.


Under the proposed rule, which was supported by Public Citizen at the time but opposed by industry group BIO, non-proprietary and non-trade secret information and data related to investigational new drug applications (INDs) for xenotransplantation and gene therapies would have been disclosed.

The categories of information that would have been disclosed included:

(1) product and patient safety data and related information, including results from preclinical and clinical studies and tests that demonstrate the safety and/or feasibility of the proposed procedures;
(2) the name and address of the sponsor;
(3) the clinical indications to be studied;
(4) the protocol for each planned study, including a scientific abstract and a nontechnical abstract, a statement of the objectives, purpose, and rationale of the study, the name and address of each investigator, the name and address of the official contacts of each local review body as appropriate (IRB, IBC) and dated copies of approval by each group, the criteria for patient selection and exclusion, an estimate of the number of patients to be studied, a description of the treatment that will be administered to patients, and the clinical procedures, laboratory tests, or other measures to be taken to monitor the safety and effects of the drug in human subjects and to minimize risk;
(5) written informed consent forms;
(6) identification of the biological product(s) and a general description of the method of production, including a description of product features that may affect patient safety;
(7) IND safety reports;
(8) information submitted to FDA in an annual report;
(9) the regulatory status of the investigation, the date of such action and the reason for such action;
(10) other relevant data and information that the director of the Center for Biologics Evaluation and Research determines as necessary for the appropriate consideration of the public health and scientific issues, including relevant ethical issues.

To facilitate public disclosure of this information, FDA also had proposed requiring sponsors of human gene therapy and xenotransplantation clinical trials to submit to FDA: The initial IND, any amendment documenting changes or additions to the IND, at the time the amendment goes into effect, IND safety reports and annual reports.

In announcing its withdrawal of the proposed rule (and another one on the regulation of crab meat) on Thursday, FDA added: “The withdrawal of these proposals identified in this document does not preclude the Agency from reinstituting rulemaking concerning the issues addressed in the proposals listed in the chart. Should we decide to undertake such rulemakings in the future, we will re-propose the actions and provide new opportunities for comment. Furthermore, this notice is only intended to address the specific actions identified in this document, and not any other pending proposals that the Agency has issued or is considering.”

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