**New**: Expanded Access - The process for requesting use of a non-FDA-approved, investigational drug outside of a clinical trial setting has been formalized at the University of Rochester. This Expanded Access process has a REDCap Application to expedite the coordination of all groups which need to be involved.
The Office of Regulatory Support (ORS) provides a variety of services to support development of Investigational New Drug (IND) application submission, and provides guidance and assistance throughout the life cycle of IND-regulated studies.
An IND Training course, designed to provide local requirements for filing an IND application as the Regulatory Sponsor and to educate on FDA requirements, is offered. Students who need access to the IND Training course should contact Regulatory Support. Faculty and staff can access it through MyPath. This training will take less than an hour, and is required for any investigator who will be submitting (or who currently holds) an active IND and optional (but strongly recommended) for study coordinators and research staff involved with the submission and maintenance of an IND.
IND applications will be filed with the Center for Drug Evaluation and Research (CDER) if the product is a drug and to the Center for Biologics Evaluation and Research (CBER) if the product is a biologic, vaccine, blood product or a cell-based product.
University of Rochester Faculty and Staff Access to IND training through MyPath
In MyPath, use the search bar feature and the keywords, "Investigational New Drug."
Rochester students may request access by contacting the Office of Regulatory Support
Those not affiliated with the University of Rochester can view the IND training through the UR CTSI IND Training
Determination of Need
An IND application must be filed with the FDA for any clinical research that proposes the use of an unapproved drug or when clinical studies propose to use an approved drug for an off-label indication or at alternative doses.
ORS will assist in determining whether filing an IND is needed, and with the paperwork for requesting the exemption.
Some clinical trials are exempt from submitting an IND.
A study may be exempt if it complies with all of the following criteria:
- it is not intended to be reported to the FDA in support of a new indication for use or to support any other significant change in the labeling or advertising for the drug; and
- it does not involve a change in the route of administration or dosage level, use in a subject population, or other factor that significantly increases the risks (or decreases the acceptability of the risks) associated with the use of the drug product; and
- it is conducted in compliance with the requirements for IRB review and does not intend to invoke exemption from informed consent; and
- it is conducted in compliance with the requirements concerning the promotion and sale of drugs.
Guidance for Pre-IND Discussions with FDA
Often, the preparation of an IND application and the planning of a Study Protocol can be aided by some early advice from the FDA, in the form of a pre-IND consultation.
ORS assists in identifying any outstanding questions which would benefit from an audience with the FDA. ORS works with the researcher to assemble the pre-IND package and by participating in communications with the Regulatory Agency, as needed.
Submission of a request for a pre-IND consultation also provides an excellent opportunity to verify that the Division within the FDA selected for receipt of an IND application is the best choice for timely study review.
Assistance with Submission of IND to FDA
The FDA has specified a format that the IND application should follow. Templates and support for determining what content should be included in an IND package are available from ORS.
Areas where additional support and resources would be beneficial include preparation of the informed consent document and creating a Data Safety Monitoring Plan (DSMP).
The Office of Human Subjects Protection provides guidance on the Informed Consent Process. There is a 3-part series "Informed Consent Video Presentation" which can be found in the training materials sections of the OHSP Supplemental Training Materials page.
If you require assistance in developing your Informed Consent Process, or have a request for an expert to observe the administration of an informed consent, request a consultation through the Research Help Desk.
All clinical research protocols must include provisions for monitoring the safety of participants and for studies that require an IND, this section must be included in the IND application. The details of a DSMP are dependent upon many factors including the level of risk of the study as well as sponsor and regulatory requirements. The University of Rochester RSRB requires a DSMP for all research studies.
If you require assistance in developing your DSMP or wish to have your DSMP reviewed prior to IRB submission, request a consultation through the Research Help Desk.
Submission of IND materials to CDER through the FDA NextGen Portal
In 2020 the FDA has developed a system to allow INDs to be submitted electronically for drug studies. This is a slight change to how submissions are done. Work with the Office of Regulatory Support to create your IND submission, annual reports, amendments and withdrawals in the most efficient way.
Once the IND has been filed, the FDA acknowledges an official receipt date and assigns an IND number. The study cannot start until 30 days after the FDA receipt of the application, unless written notice from the FDA is given earlier that the study may begin.
The FDA may request that the study not begin until additional information has been submitted to the Agency. In some cases, if the FDA determines that significant safety concerns remain, they may place the study on "Clinical Hold" until all issues have been addressed.
Assistance with Filing Annual Reports and Amendments to the FDA
As the study progresses, the Regulatory Sponsor is responsible for seeing that the study is conducted according to the current approved protocol. Changes to the protocol can be made by submitting amendments to the FDA, and ORS can assist. This FDA amendment is in addition to any related IRB requirements.
Even if no protocol changes are made, regulations require that reports be submitted to the FDA on an annual basis, at a minimum. ORS will assist with both reminding Regulatory Sponsors about reporting milestones and also in the preparation of the reports.
Adverse Event reporting is also critical to the continued operation of any clinical trial and ORS will assist researchers in abiding by the proper reporting procedures. There are several categories of adverse events and they have varying timelines for submission of the reports, based on severity.
Expanded Access Guidance
The FDA has created the Expanded Access Program which allows a qualified physician to provide an investigational drug or device to a patient outside of a clinical trial. There are certain criteria which must be met for this Expanded Access use, and coordination with the company providing the therapeutic is critical.
If you have a patient who would benefit from the use of an investigational product that is not currently approved, and there is no clinical trial available for this patient, review Expanded Access information from the FDA for more details.
ORS provides guidance on completion of the required FDA forms and navigation of the entire process. To begin the process, complete an Expanded Access Application within REDCap.