The proposal states that trade secret and confidential information should be removed from the adverse event report sent to NIH. It also requires that patient identifiers be stripped. We appreciate the NIH’s acknowledgement that adverse event reports contain information that cannot lawfully be publicly disclosed. We also appreciate its recognition that disclosure of patient information would violate that individual’s privacy rights and hurt sponsors’ and investigators’ ability to recruit patients into clinical trials.
However, the data and information that would be submitted under the proposal are, by definition, trade secrets and confidential commercial information. Virtually every detail about the design, size, or status of a clinical trial is of potential competitive value. Indeed, FDA explicitly recognizes that adverse events are confidential commercial information. Therefore, the proposal’s requirement of deletion of this information is illogical.
The same argument applies to patient data. BIO is concerned that confidential patient information will still be disclosed because other information in the report will be made public and this information could be used to identify a patient in a clinical trial.
Therefore, the proposal would require reporting of data that would provide the RAC with confidential information that cannot be disclosed. It is well settled law that three federal confidentiality statutes – the confidentiality provisions of the Freedom of Information Act, the federal Trade Secrets Act, and the Federal Food, Drug, and Cosmetic Act – preclude the FDA from publicly disclosing clinical protocols, adverse events, and other confidential commercial and trade secret information in the drug development process. The first two statutes also apply to the NIH. Since the NIH cannot legally apply the same federal statutes to the identical scientific data and information in a completely inconsistent way, if the RAC were to obtain data as contemplated by the proposal, it would not be allowed to disclose them to the public.
Consequently, the proposal has the ironic effect of providing the RAC with data it cannot use to further its mission of public discussion. Thus, the RAC proposal is internally inconsistent. It requires reporting of data for use in a public forum, while acknowledging by its own terms that this data must remain confidential.
BIO Recommendations for Gene Therapy Oversight
Gene therapy is a highly promising technology that has the potential to cure disease and improve the quality of life for thousands of patients. In addition, like other new drugs or biologics under investigation, gene therapy is already strictly regulated. Sponsors of gene therapy clinical trials must comply with federal regulations governing the development of all new drug products. Hundreds of gene therapy clinical trials have been approved over the past decade and thousands of patients have safely received investigational gene therapies.
The FDA regulates clinical trials of gene therapy and has the statutory authority to permit an IND to go into effect or, if necessary, place an IND on clinical hold to ensure safety of human subjects. Under existing federal regulations, serious adverse events are reported in a timely fashion to the FDA. This regulatory oversight role is critical to the FDA’s mission and provides effective protection for the public. BIO believes that only FDA has the scientific expertise needed to review and assess adverse events and that this should remain a statutory responsibility of the FDA. To protect patient confidentiality and because the information in an adverse event report may be of a proprietary nature for the sponsor, the FDA, by law, is not allowed to publicly disclose this information.
BIO continues to support the RAC’s role to publicly discuss the social and ethical implications of new technologies, such as gene therapy. The RAC can and should continue to play a critical role in publicly debating novel scientific and ethical issues associated with certain types of clinical gene therapy research. In this way, the RAC complements the FDA’s regulatory responsibility to oversee the development of individual drug products. However, the RAC should not, and legally may not, publicly disclose confidential commercial and trade secret data and personal patient information relating to gene therapy drugs and clinical trials submitted to it.
As an alternative to the NIH proposal, BIO companies propose the following structure for the future oversight of gene therapy:
The NIH does not have the legal authority to compel adverse event reporting by sponsors of clinical trials using gene therapy. Nonetheless, sponsors agree to voluntarily provide serious, related and unexpected adverse event reports (serious adverse events or SAEs) that are currently sent on an expedited basis to the FDA to the NIH/OBA. Sponsors would also send to RAC the safety data summarized in the IND annual progress report currently provided to FDA. That report contains summaries of all adverse events, whether or not the event was related to the drug in development and an overview of all other adverse events. In this way, OBA and the RAC would have access to adverse event reports at the same time as the FDA.