Working with ORAQ on Drug/Biologic Projects

Frequently Asked Questions (FAQs)

Does my study need an IND?

When an investigational drug (not FDA-approved) is being used in a clinical study, an IND is required.  When an FDA-approved drug is being used in an investigational manner (“off-label”--different patient population, dose, route of administration, etc.), an IND may be required depending on whether the study meets the IND exemption criteria. The IRB will determine if an IND is needed during the study protocol’s full board review, but ORAQ can provide a preliminary assessment when requested.

 

Who puts together and submits the IND?

A regulatory coordinator, the sponsor, another study team member, or an ORAQ Regulatory Affairs Scientist can take the lead on putting the Duke-sponsored IND together; it’s up to the study team. It will be a collaborative process regardless of who leads the effort. However, even if ORAQ is not involved in drafting the IND, per Duke School of Medicine policy, ORAQ must review and submit all Duke-sponsored INDs and subsequent amendments.

 

What is ORAQ’s role in drug/biologic manufacturing and oversight?

ORAQ has a Regulatory Affairs team and a Good Manufacturing Practices (GMP) Quality team. At this time, all ORAQ Quality staff are assigned to specific manufacturing facilities on campus. Therefore, for products made outside of these facilities, the IND sponsor is responsible for ensuring that the drug or biologic product is made in accordance with phase-appropriate GMP. The IND sponsor or a qualified delegate should review and store all documentation from the manufacturing process and any analytical testing. Please share manufacturing and testing information with the ORAQ Regulatory Affairs Scientist so that required information can be submitted to the IND.

 

Should I submit to the FDA or IRB first?

Either way can work. If the initial IND is submitted ahead of the IRB submission, the “Study May Proceed” letter from the FDA can be provided to the IRB in the initial submission.  It is very common for FDA to have edits to the protocol during initial IND review, and this allows them to be incorporated prior to IRB submission.

There are some teams at Duke that choose to submit the study to the IRB ahead of the initial IND submission—for example, they prefer to see all the IRB modifications to protocol/consent before providing to the FDA.  However, in this situation, the IRB may hold up full approval of the study until they receive confirmation of an effective IND.

 

What happens during FDA’s review of the initial IND?

The initial IND has a 30-day review clock. The FDA will likely send information requests with a short turnaround time by email to the IND sponsor during the review period, so the IND Sponsor needs to be available and attentive to their email during this time. Please share any correspondence from the FDA with the ORAQ Regulatory Affairs Scientist supporting the project.

Note that having an IND number, completing the IND submission, and/or having an IND Acknowledgement Letter is not sufficient to say the study is “FDA approved”—you either need to wait 30 days after submission or have the Study May Proceed letter from the FDA.

 

How are changes to the study or IND handled?

Protocol amendments:

The IND regulations in 21 CFR 312.30(b) outline when you are required to submit a protocol amendment to the IND in “real time” (i.e. before actually making the change).  This includes any change in a Phase 1 protocol that significantly affects the safety of subjects or any change in a Phase 2 or 3 protocol that significantly affects the safety of subjects, the scope of the investigation, or the scientific quality of the study.  For amendments that meet these criteria, the changes to the protocol should not be implemented until both the FDA submission is made and the IRB has approved the amendment.  For amendments that don’t meet the criteria, it could be acceptable to submit the amendment to the FDA in the next annual report.  However, it is recommended that you make ORAQ aware of the amendment before IRB submission so that everyone is on the same page with the assessment. As a best practice, ORAQ prefers to submit protocol amendments to both the FDA and IRB in “real time” to keep versions aligned, even in situations where the changes in the protocol may not meet the regulatory criteria.

Review timeline: Unlike the initial IND, there is no 30-day review clock for amendments, and you will generally not receive any confirmation from FDA that they “approved” the amendment.  For higher risk changes, ORAQ recommends giving the FDA at least 30 days to review before implementing the change. ORAQ can e-mail the regulatory project manager at FDA to confirm that they had no concerns. Another option is noting clearly in the IND cover letter when the protocol amendment will be implemented or stating a time when the team needs FDA feedback by.  Please discuss any time constraints with ORAQ ahead of submission. 

Updated Form FDA 1572s:

The Form FDA 1572 has to be updated with changes to sub-investigators, facilities, labs, and/or the IRB.  Most often the updates are related to changes in sub-investigators. Please provide ORAQ with an updated Form FDA 1572 or make them aware of any changes. ORAQ will save the updated Form FDA 1572 in the IND files to include in the next IND submission.

Each time you update a Form FDA 1572, please check the FDA’s Forms site to make sure you are using the current version of the form. Each FDA form has an expiration date in the top right hand corner of p.1 and a version (Month/Year) in the bottom left hand corner on all pages. You may find that the expiration date has passed. If that is the case, the form is still okay to use if it is the one located on the Forms website. It just means that the FDA has not yet issued a newer version.

Changes and updates to drug manufacturing information:

Please let the ORAQ Regulatory Affairs Scientist know about any manufacturing process changes or when new analytical data (e.g. stability data) are available. Please also let ORAQ know when new lots of a drug substance or drug product are manufactured. The ORAQ Regulatory Affairs Scientist can help determine what information needs to be submitted to the IND.

 

Do I need to submit a protocol deviation to my IND?

If a deviation significantly affects the safety of subjects, the scope of the investigation, or the scientific quality of the study, ORAQ may recommend submitting a description of the deviation in an IND amendment. In general, we recommend making the assigned ORAQ Regulatory Affairs Scientist aware of any protocol deviation that meets the criteria to be reported to the IRB. In these situations, ORAQ can work with the IND sponsor and study team to discuss whether to report the deviation to the IND (and whether to submit in an amendment or annual report, if applicable). For protocol deviations planned ahead of time (e.g. for individual patients), it is recommended to make the assigned ORAQ Regulatory Affairs Scientist aware of the planned deviation as early as possible to discuss whether an IND amendment is appropriate. If the deviation is to eliminate an immediate hazard, it can be implemented immediately and reported to the IND later.

 

When do I have to report adverse events to the FDA?

Serious and unexpected suspected adverse reactions must be submitted to the IND in addition to the IRB.  If they are fatal or life-threatening, they must be submitted within 7 calendar days, and all other serious suspected adverse reactions must be submitted within 15 calendar days.  If an SAE occurs that could possibly be related to the investigational drug, loop in the ORAQ member ASAP for awareness.  ORAQ can help the team with assessing whether the adverse event is considered “unexpected”.  This assessment is required to be made based on the risk information provided to the FDA within the IND, protocol, and consent, not just based on the PI’s assessment on whether it is expected for the disease or drug.  An adverse event or suspected adverse reaction is considered “unexpected” if it is not listed in the investigator brochure or is not listed at the specificity or severity that has been observed; or, if an investigator brochure is not required or available, is not consistent with the risk information described in the general investigational plan or elsewhere in the current IND application.

 

What are my IND annual report obligations?

An annual report must be submitted to the IND within 60 days of the anniversary of the effective date of the IND. ORAQ’s REDCap database will send automatic annual report reminders starting 60 days before the IND annual report due date. ORAQ has an IND annual report template and the ORAQ Regulatory Affairs Scientist can populate the template with known study information and provide the draft report to the study team. The study team will need to populate information on enrollment and adverse events. Please review the template on our website for more information on what data are required in the report.
 

Can I remain the IND sponsor or an investigator during my extended leave?

If you are an IND sponsor and/or investigator, please contact your ORAQ Regulatory Affairs Scientist to make them aware of any extended periods of time out of the office (e.g. personal leave, Family Medical Leave Act (FMLA), maternity/paternity leave, sabbatical, etc.). Per FDA guidance, supervisory responsibility of a study site cannot be delegated to a subinvestigator. Therefore, when an investigator will take an extended leave, it is expected that a new investigator will be assigned for the extended leave period and an updated Form FDA 1572 will be submitted to the IND. Please also see the IRB’s FAQ on oversight of an FDA-regulated protocol when the PI is unavailable and the DUHS Principal Investigator Agreement

 

When can I withdraw my IND? 

The IND regulations state that an IND can be withdrawn once all clinical investigations under the IND have ended, all current investigators have been notified, and all stocks of the drug have been returned to the sponsor or otherwise disposed of at the request of the sponsor. We interpret “clinical investigations under the IND have ended” to mean that all data collection and participant interactions are complete, but not necessarily that all data analysis has been completed. Please note that IRB protocols should remain open for data analysis of identifiable private information for research purposes. Please see the IRB's policy on Expiration of IRB Approval and Subsequent Notice to Cease Study Activity for more information.

 

Can ORAQ help answer IRB regulatory questions or make IRB submissions?

ORAQ does not assist with IRB submissions or questions. For questions regarding IRB requirements, please contact your Duke IRB specialist.

 

Can ORAQ help submit INDs in electronic Common Technical Document (eCTD) format?

Yes, ORAQ has the capability to submit in eCTD format for certain projects; please talk to your Regulatory Affairs Scientist if you are interested in this option. eCTD submission documents must meet certain technical specifications and therefore undergo a “quality control” (QC) process within ORAQ before submission. Following QC of a clinical protocol or other relevant documents, ORAQ will e-mail the study team an updated version that incorporates any formatting changes made during QC. To avoid ORAQ having to make the same formatting updates for subsequent amendments, it is preferred to use the post-QC version as a starting point for subsequent amendments. Since it is common to use the IRB-approved protocol as a starting point for a subsequent amendment, it can be very useful to get the post-QC version from ORAQ prior to IRB submission.

 

Where can I find additional information on IND requirements?

  • ORAQ offers a Regulatory Affairs Training Program that gives an overview of premarket regulatory work related to drugs, biologics, and medical devices. 
  • ORAQ’s Education and Training page includes recordings of previous trainings like our IND and IDE workshops.
  • ORAQ also has a recorded training course in the LMS on the sponsor responsibilities for INDs.  While the SOM requires the IND holder to complete this, anyone at Duke can register and choose to take the course.
  • The ReGARDD website has some educational information and resources for academic investigators.