Investigational Drugs

Features

During a consultation, we can help you:

• Determine if an IND is needed
• Provide guidance for pre-IND discussion with the FDA
• Assist with IND applications or exemption requests to the FDA
• Assist with submission of IND materials to the Center for Drug Evaluation and Research
• Provide guidance on the expanded access process

Get Started

To get started, request an IND consultation with the ORS.

Determination of Need

ORS will assist in determining whether filing an IND is needed, and with the paperwork for requesting an exemption.

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.

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.
• 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.
• It is conducted in compliance with the requirements for IRB review and does not intend to invoke exemption from informed consent.
• It is conducted in compliance with the requirements concerning the promotion and sale of drugs.

Pre-IND Discussion Guidance

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.

IND Submission

The FDA has specified a format that the IND application should follow. The ORS can provide templates and support for determining what content should be included in an IND package, including guidance on the new IND application 1571 Form. ORS can also help prepare data safety monitoring plans (DSMP) and informed consent documents and can provide an expert to observe the administration of an informed consent.

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 a study as well as sponsor and regulatory requirements. The University of Rochester RSRB requires a DSMP for all research studies.

Electronic IND Submission

In 2020, the FDA developed the NextGen portal to allow INDs to be submitted electronically to Center for Drug Evaluation and Research (CDER) for drug studies. Work with the ORS 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.

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. If any changes to the protocol are needed, ORS can assist with submitting the necessary amendments to the FDA, which must be file 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's Expanded Access Program 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.

Learn more about expanded access guidance provided by ORS.

Get Training

ORS offers an IND Training course, designed to provide local requirements for filing an IND application as the Regulatory Sponsor and to educate on FDA requirements. 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.

• Faculty and staff can access it through MyPath (search for "Investigational New Drug").
• University of Rochester students who need access to the IND Training course should contact Office of Regulatory Support.
• Those not affiliated with the University of Rochester can access the IND training after taking a brief survey.

If you are interested in learning a bit more about what happens during an FDA audit and how you can be prepared for one, watch this short Preparing for an FDA Audit training video.

Resources

ORS can provide templates for many of the FDA submissions during the lifetime of an IND. Contact the Research Help Desk if you need assistance with drafting communications to the FDA or for templates for any of the following items:

• Request for IND Exemption
• Pre-IND meeting request and briefing package
• IND Application
• IND Annual Report
• IND Withdrawal
• IND Letter of Cross-Reference/Letter of Authorization
• Cover Letters
• Investigator Brochures

Contacts

Joan E. Adamo, Ph.D., Director, Regulatory Support
Joan_Adamo@urmc.rochester.edu, (585) 275-0742

Reshma Ravilla, Pharm.D., Regulatory Affairs and Compliance Specialist
Reshma_Ravilla@urmc.rochester.edu

Carrie Dykes, Ph.D., Research Engagement Specialist
Carrie_Dykes@urmc.rochester.edu,  (585) 275-0736

Shelby Johnson, Administrative Support
Shelby_Johnson2@urmc.rochester.edu, (585) 275-5327