The Food and Drug Administration has laid out a drug development continuum that includes three milestones, or Type B meetings. Earliest is the Pre-IND Meeting, the second is the End of Phase 2 (EOP2) Meeting, and the third milestone is the Pre-NDA Meeting.
Each meeting has a different agenda. This is because each meeting occurs at a different point in the development continuum. Nevertheless, all of the meetings share the same ultimate goal, which is to gain clarity in order to be able to proceed successfully to the next step in the development process.
There’s quite a bit of preparation involved with planning and carrying out a successful meeting with the FDA. It starts with determining what questions to ask. It’s important that you are not afraid to ask certain questions just because you don’t want to get negative answers to those questions. Getting a firm “no” after you’ve submitted the NDA hurts a lot more than getting that “no” earlier in the process.
Whether the feedback is positive or negative in nature, attending each of these meetings take away the unknown. If the feedback is positive, that’s a good thing. If the feedback is negative, that’s also a good thing. This is because the sponsor is now able to deal with the issue(s) that received negative feedback before the next submission or meeting.
The Pre-IND Meeting
The Pre-IND Meeting marks your first formal interaction with the FDA. In this meeting, you are introducing your product concept as well as a general plan outlining how you intend to turn that concept into reality. The overarching purpose of this meeting is to get in front of the FDA so that you can validate your clinical development program before you begin work.
As with each of the meetings, it is up to the sponsor to set the agenda for the meeting. Although the FDA has a standard meeting “template” that is designed to gain essential information, the sponsor decides which questions to ask. Consequently, the discussions that take place in this meeting are extremely strategic in nature.
Each question should be formulated so that you will receive a “yes” or “no” answer to a question that is important to the development process. The questions should be presented in paragraph format, providing context and statements, and ending with the question, “Does the agency agree?”
How do you know if you’ve had a successful meeting? You’ll know you’ve had a successful meeting when you’ve received the guidance you are looking for. You may not have received the answers you wanted, but what you should be focused on is whether you received the clarity you need in order to proceed.
End of Phase 2 Meeting
With a typical program, the End of Phase 2 Meeting will take place several years after the Pre-IND Meeting. By now, you will have conducted clinical trials, so in this meeting, you are presenting clinical data to the FDA in pursuit of enrolling into your Phase 3 pivotal trials. Think of this part of the discussion as providing proof of concept that your product works. Your goal is to get the FDA’s agreement that you have proven your concept and that the product shows promise.
You will present your Phase 3 program to the FDA during this meeting, which outlines the clinical trials that you plan to conduct. Your goal in this part of the meeting is to ask the question: “If we conduct these trials and the data are positive, will this warrant submission and approval of our NDA?” Again, you’re looking for “Yes” or “No” answers because Phase 3 of any clinical program can cost tens to hundreds of millions of dollars. As such, you want to make sure that you plan to do what the FDA wants you to do, making it crucial that you get clarity on all aspects of the planned trials.
The Pre-NDA Meeting (or in the biologic space, the Pre-BLA Meeting) is the meeting where you are presenting your entire package of data to the FDA in a meeting setting. You’ve done all of the work. You’ve had multiple formal and informal interactions with the FDA. Now, during this meeting, by summarizing how many trials you completed and the outcomes of those trials, you are presenting a high-level view of what you have accomplished.
At this point, your goal is to get confirmation from the FDA that you have a data package that warrants submission of an NDA (or BLA). In essence, you want their agreement to this fact. Their positive response is a way of saying that they don’t see anything that rises to the level that clearly indicates that your NDA is not ready to be submitted.