Many Sponsors come out of a positive-response FDA meeting thinking they have gotten everything they wanted. Unfortunately, the FDA is not obligated to tell you what you missed. Did you get permission to conduct a trial, or did you get confirmation your drug discovery program is on track to receive marketing approval?
I’ve seen top tier consultancies, with ex-FDA personnel I know and respect, recommend meeting questions that don’t assure a path to market. For instance, in one case, the recommendation was to get the FDA to allow a clinical trial for a drug that had already been approved for another indication. Well, the Sponsor didn’t actually need FDA approval for that – this drug could be prescribed off-label by any M.D., and the results collected in a clinical trial without any Agency input. What the meeting should have been about, is whether the proposed clinical protocol would lead to marketing approval in the new indication.
Can you imagine the expense of conducting a clinical trial that didn’t contribute to meeting the Agency’s expectations, and didn’t check a single box in terms of safety or efficacy?
This is exactly what happens when you ask the wrong questions at an FDA meeting. You absolutely do NOT want to rush in and just get approval to conduct a clinical trial. You want to make sure you are identifying any gaps the Agency sees between the proposed protocol and the finish line, so you are not obligated to fund additional studies (or drop the entire program when your investors realize your mistake and lose confidence).
Review divisions are there to assist you, but not to direct your research. Many studies are considered natural history studies, or proof of concept studies, and it isn’t the Agency’s place to ask you what your intent is. This is why it is so important to confirm whether your plans line up with the FDA’s expectations for marketing approval. At KLA Breakthrough Consulting, we have years of experience asking the right questions. Please let us know if you would like a free consultation!