Ann: Type C Meeting granted by FDA for Phelan-McDermid syndrome, page-35

I've been looking through some sites today for information on how the FDA approves the trial design. My basic understanding is if the FDA is happy with the trial design, they would just say so, no need for a seperate formal meeting. The Type C meeting is a mechanism available to sponsors to hold a formal meeting in person with the FDA for those issues outside of the criteria for the other Types of meetings. Considering the trial design is nearly complete (many things agreed on already with the FDA), and if you also consider that a Type D meeting is for sponsors with more than one pressing issue (perhaps due to the complexity of a given drug and trial), then a Type C meeting in my view is for one issue that still needs to be sorted (assuming primary endpoints).

I read also that the FDA makes suggestions, but a drug developer doesn't have to follow them. I'm not clear on what this means, but I assume if there wasn't alignment on all the key aspects of a trial design then theoretically a drug developer could go ahead with a trial but it would be fraught with danger because you didn't get that alignment in the first place. (For example a trial would be completed, the drug developer goes to the FDA and they reject the results on the basis it didn't cover what they wanted in a statistically sufficient manner across the key sticking points.)

Happy to corrected on all of the above, but I just think we would've heard about the possibility of a Type C meeting earlier following the disclosure the communication between Neuren and the FDA was simply a matter of email correspondence.