Two weeks ago, the US Food and Drug Administration (FDA) announced the withdrawal of a proposed rule from 2001. The rule would have required the public disclosure of summaries of safety and effectiveness data from pre-market clinical trials of gene therapies and transplanted non-human tissues to humans.
In its notice of withdrawal, the FDA announced “[we have] reconsidered our position on the issue and deemed our concerns from 2001 outdated.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.”
RAPS explains that “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.” RAPS goes on to detail the categories of information that would have been disclosed under the rule:
- 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
- the name and address of the sponsor
- the clinical indications to be studied
- 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
- written informed consent forms
- 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
- IND safety reports
- information submitted to FDA in an annual report
- the regulatory status of the investigation, the date of such action and the reason for such action
- 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
The FDA closed their notice of withdrawal by stating “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 chard. Should we decide to undertake such rulemakings in the future, we will re-propose the actions and provide new opportunities for comment.” If your company needs help navigating the FDA’s ever-changing rules and the complex regulatory landscape, please contact us.