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IND approved - question on protocol amendment

  • 1.  IND approved - question on protocol amendment

    Posted 31-Oct-2022 21:55
    Edited by May Litt 01-Nov-2022 02:07
    I have the following scenario and am not sure how to proceed. For a drug study, the FDA issued an authorization to proceed letter for our IND. However, there were a few non-hold comments. We would like to immediately update the protocol with these comments. Would this require a protocol amendment or can we simply reply to the FDA confirming the changes and reply? I have honestly never seen this scenario. Unfortunately, the FDA had a few comments, which were addressed prior to IND approval, so these somewhat minor non-hold comments were a surprise, but we want to get the protocol updated now, before enrollment. Thank you!

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    May Litt
    Clinical Operations Consultant
    Weirsdale FL
    United States
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  • 2.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 02:42
    Hi May,
    I have seen this scenario many times, especially for first-in-human or Phase 3 studies. Since the comments are non-hold, they are obviously not deemed critical for the safety of the study. So you could proceed without inplementing any changes to your protocol.
    In my experience, however the comments would be beneficial for the study itsself or the overall development program. And, while there may not be an obligation to follow them, it makes for a lot better dialog with the Agency down the road if you can point to the fact that you incorporated their feedback. Of course there may be good reasons not to incoporate feedback, and then you should make sure you have good arguements for that. Because generally the Agency will look back at their own prior comments when the assess the study results later on.
    So, in most cases we are prepared to incorporate any non-hold comments into a protocol amendment. As much as possible, we plan for a fast turn-around after the Agency provides the feedback, so that the study starts with the issues addressed. There may, of course, be non-hold comments that could be incorporated in a later amendment (e.g., how to analyze samples collected in the study), so that you could start the study prior to amending the protocol and collect the samples, as long as the amendment occurs prior to the analysis of the samples. But many times there are multiple comments that we address in the first amendment. 
    As long as the changes made in the amendment are basically restricted to the non-hold comments, then your can feel confident in initiating the study once you've re-submitted the protocol to the IND and gotten IRB approval. While not obligatory, many companies still wait another 30-days after re-submission to allow the Agency the time for a safety assessment prior to dosing first patient. And you can check in with your regulatory project manager as well to confirm.
    And if the amendment includes more fundamental changes or beyond the non-hold comments the team may choose to wait for the full FDA review of the amended protocol (60-days per FDA's MAPP 6030.0).
    Good luck!
    Anna

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    Anna Sedello, PhD, RAC-Drugs
    Senior Director, Regulatory Affairs
    Palo Alto CA
    United States
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  • 3.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 13:16
    Thank you, Anna!

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    May Litt
    Clinical Operations Consultant
    Weirsdale FL
    United States
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  • 4.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 03:18
    Edited by Jan Peterson 01-Nov-2022 03:20
    Hello, May - 

    Yes, tell your reviewing division contact (an email to the project management person is fine for this) that you are responding to their non-hold comments with protocol amendments and that you will submit the resulting version in due course as you begin enrollment. Now that your IND is "in effect," go ahead amend your protocol just as the non-hold FDA comments suggest (assuming you can do what is suggested and you, i.e., the sponsor, agree with those suggestions). Good time to fix those other typos, too.

    The next key step is independent of FDA at that point, and that is to obtain IRB/Ethics Committee approvals for the amended protocol version, get that distributed to your investigators and provide any needed training, changes to CRFs, etc., and proceed with your study enrollment. You could have proceeded with the initial protocol first, but if the timing is OK, you're best to do as you planned and get the revision processed first so you can start enrollment with the updated version. You don't have to do this next step before implementation, and by regulation it may not even qualify as being required, but I recommend that in a timely manner, submit a copy of the amended, IRB-approved protocol you started enrollment with to your IND as an information amendment. Include a cover letter detailed how you responded to the non-hold items, point by point [quote one item from their correspondence exactly and follow it with your response], and describe how you altered the earlier protocol version. Including a red-line/strike-out version along with a clean copy of the update protocol with your submission is a good idea, so the precise changes can be easily observed. You may even include in that cover letter the date of the new IRB approval (at least the first one, if you have it) and of course, be sure to comply with requirements of letting FDA know about added investigators (use of Form FDA 1572 is important) that may come along and were not previously listed under your IND.

    Depending on the phase of your investigation, many (perhaps most?) minor changes to your protocol after this will not need any pre-submission or pre-approval by FDA under your "in effect" IND. But any further revisions will of course require IRB approval and at least, as before, you might consider a subsequent submission to FDA so they have a current copy on file. Some/many sponsors do not do any IND protocol amendment submissions with every minor protocol revision during a year (unless required by regulation) and accumulate minor protocol changes as one annotated, omnibus update in their IND Annual Report submission. You can think of this tactic as not adding unnecessarily to the burden of your review staff at FDA-for which they might find you a more collegial and agreeable sponsor. This is especially so when you end up with lots of minor amendments during a year, which seem to arise when investigators are finally faced with actually trying to operate under the protocol you so carefully wrote for them and now you are finally getting some useful feedback. Accumulating protocol changes during the year depends on whether the sponsor considers (or how you feel FDA staff might consider) the changes being made would meet the criteria for a required submission (e.g., new protocols, "significant" changes likely to affect risks, like increase in dosages, overall design changes including a much larger enrollment population, adding new testing or the omission of previously planned testing, and of course adding new investigators). Lots of other changes don't meet those regulatory requirements, but you have to at least keep careful track and still get IRB approvals for each one.

    As with any controlled document in the study, be sure your protocol versions are clearly marked and you can document when each site placed the later versions into effect and that this aligns with relevant IRB approval dates. Be wary of methods like "amendments by memo," which are difficult to track when the protocol document is not re-issued and doesn't provide investigators and their staff with a single current, authoritative protocol document. If you have a separate study manual that provides additional details outside the protocol to manage study conduct, be aware that some IRBs want to review/approved those details as well, and that may even include the CRFs, by considering them a part of the "investigational plan." Some sponsors like to make changes to the non-protocol support documents (i.e., manuals) specifically because they might therefore avoid IRB review, which could be time consuming and possibly costly, when the changes in some cases really should have been included in the formal study protocol proper. Good luck on your study, and I hope your protocol is so well written that few if any amendments are needed during the year. 

    Best regards,

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    Jan S. Peterson, MS, CCRA, RAC, ASQ CBA, ACRP-CP
    Consultant
    United States
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  • 5.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 13:15
    Thank you for the thoughtful response, much appreciated.

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    May Litt
    Clinical Operations Consultant
    Weirsdale FL
    United States
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  • 6.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 06:13
    Hi May,

    Go ahead and amend the protocol, submit the protocol amendment to the IRB and to the IND.  As soon as the IRB approves the protocol amendment, you can start to use the amended protocol to enroll subjects.  Pretty straight forward.

    In the cover letter when submitting the protocol amendment to the IND, I typically like to indicate that the amendment is in response to Agency comments which have been addressed in the amended protocol.  But this is not required as this should be evident in the redlined version of the protocol amendment and the summary of changes document, both of which are submitted to the IND with the amended protocol.


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    Mark De Rosch FRAPS
    Chief Operating Officer, Aura Biosciences
    Laconia NH
    United States
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  • 7.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 13:15
    Thank you!

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    May Litt
    Clinical Operations Consultant
    Weirsdale FL
    United States
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  • 8.  RE: IND approved - question on protocol amendment

    This message was posted by a user wishing to remain anonymous
    Posted 01-Nov-2022 08:43
    This message was posted by a user wishing to remain anonymous

    Protocol amendment would be the appropriate pathway from RA perspective!


  • 9.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 14:47
    Hello,
    I agree with the other posted comments.   My organization is in the same situation.  We are incorporating FDA reviewer comments prior to receiving authorization to proceed, as well as non-mandatory comments received afterwards, into our IND and submitting as a protocol amendment. 
    Adam


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    Adam Armstrong
    Regulatory Program Manager
    HeartWorks, Inc.
    Rochester MN
    United States
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  • 10.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 17:24
    In a similar situation, we did both. We replied to confirm the changes and followed up with a protocol revision.





  • 11.  RE: IND approved - question on protocol amendment

    Posted 01-Nov-2022 19:36
    Seems simple enough to make the changes (protocol amendment) and send to FDA as your first supplement with cover letter and 1571. Maybe you want to wait until you have any additional changes to the protocol and ICF from the IRB/site so you have fewer supplemental filings. Maybe I'm missing something here?

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    Joy Frestedt PHD, CPI, RAC, FRAPS, FACRP
    President and CEO
    Frestedt Incorporated (www.frestedt.com)
    Saint Louis Park MN
    United States
    612-219-9982
    jf@frestedt.com
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