Issue 4: What Makes an FDA Meeting Work (It's Not Your Slides)

A mistake I saw repeatedly in FDA meetings: asking an open-ended question and expecting a specific answer.

I sat in many FDA meetings. I watched sponsors come in with a well-prepared meeting package, present their program clearly, and then ask questions so broadly framed that the only possible answer was a variation of "it depends." They left with that answer, and then spent weeks trying to work out what FDA meant.

The purpose of an FDA meeting is not to present your program. It is to resolve the specific uncertainties that are blocking your next decision. If you are not clear on them before the meeting, the meeting will not settle them.

Formal meetings for drugs and biologics come in several types. Each has its own purpose and its own clock, counted from the day FDA receives your request:

Two newer types round out the list: Type D (50 days), for a narrow set of issues, and INTERACT (75 days), for novel questions early in development, before a pre-IND meeting.

The End-of-Phase 2 meeting deserves special attention. It is one of the highest-leverage interactions in the entire development program — the chance to align with FDA on your pivotal trial before you run it. I write as a statistician, but the statistics come second. Study design comes first: the patient population and entry criteria, the primary endpoint and the study duration, to name a few. The statistical questions follow from the design: the estimand, the analysis, the sample size. And the agenda isn't limited to the trial. CMC and other development questions belong there too. Sponsors who use this meeting well may save themselves years of uncertainty.

FDA now calls it the meeting package. Many sponsors still call it the briefing document. Either way, it is not a summary of your program. The program summary belongs in your IND or your NDA or BLA. The meeting package is designed to let FDA answer your specific questions.

A strong meeting package opens with a short background section: a brief overview of the program, where development stands now, your earlier interactions with FDA, and anything reviewers should keep in mind as they read — safety signals from earlier studies, for example. Keep it focused on what FDA needs to answer your questions, not on retelling the full history of the program.

Then come the questions, grouped by FDA discipline. For each one:

The question itself is where many meeting packages fall short. A broad question gets a broad answer. Ask "Is our Phase 3 design acceptable?" and the answer will be some version of "it depends."

Compare that with: "Does FDA agree that our proposed primary endpoint, as defined in Table 3, is acceptable to support an NDA in [indication]?" FDA can give a direct answer to that question, and you can act on it.

The other answer sponsors often hear is "That is a review issue." This usually comes back when the question asks FDA to judge results it hasn't yet reviewed, such as "Do the data support our proposed indication?" FDA answers that question when it reviews your NDA or BLA, not in a meeting.

There is still a place for an open question. Ending with "Does FDA have any other concerns about our proposed approach?" is a good way to make sure you haven't overlooked anything. It works as a safety net, not as a substitute for specific questions.

Your meeting package is what FDA works from. Take the time to make sure it is well written and complete when it goes in. Don't count on filling gaps later.

FDA reviewers read the meeting package in advance. They have a position on your questions before you walk in. The meeting is not where they form that position — it is where they communicate it and where ambiguities get resolved.

That is why your slides should focus on your questions, not on a scientific presentation. The meeting will not be extended to make room for a presentation, and every minute you spend presenting is a minute not spent on the answers you came for. If the slides contain more than a small amount of new content, FDA staff may not be able to comment on it.

FDA's guidance on formal meetings goes further: presentations are usually unnecessary, because the information FDA needs should already be in the meeting package. If you do present, send the slides ahead of the meeting and keep them brief.

For most meetings, FDA sends preliminary responses a few days ahead. Read them as carefully as FDA read your package. If they fully answer your questions, you may not need the meeting at all. If they don't, use the meeting for what remains unclear, not to revisit every answer.

And don't respond to them with new data or a revised proposal. The same guidance is explicit: preliminary responses are not intended to generate new information or new questions. If you send them anyway, FDA may not be able to comment on them, or you may need to submit a new meeting request.

The meeting itself is most valuable when sponsors listen more than they present. When a reviewer asks a follow-up question, that question is information — it tells you what is on their mind. When they say "it depends," the follow-up should always be, "On what, specifically?"

Before the meeting ends, summarize the agreements and action items out loud. It is your last chance to catch a misunderstanding while everyone is still in the room.

FDA issues the official minutes within 30 days of the meeting. They are the record of what was discussed and agreed, and they are what reviewers will reference when your NDA or BLA comes in. Read them carefully. If something doesn't reflect what was said, raise it with the FDA project manager promptly.

If FDA agreed to something in the meeting that is not in the minutes, that agreement does not exist for regulatory purposes.

A successful FDA meeting is not one where FDA says yes to everything you proposed. It is one where you leave with a clear, documented answer to the question that was blocking you.

Sometimes that answer is no. A no is still valuable, because FDA explains it. The reasoning tells you what to do next — modify your approach, gather additional data, consider a different design. And a yes often comes with minor changes attached. Read those as carefully as the yes itself, and make them before you build on it.

Come in with specific questions. Get specific answers. Document them. Then execute.

If you have an FDA meeting on the calendar and want to walk in prepared, I'm happy to talk through where you stand. Book a free 30-minute call.

Thank you for reading!
Lisa