Some BTD tidbits I was curious how the BTD process work and was reading a bit on the FDA sites. I thought it was a submit and hope process but it has a bit more nuance.
"A sponsor can contact the regulatory project manager (RPM) in the division to which the active IND is assigned and request the “Preliminary Breakthrough Therapy Designation (BTD) Advice Request” template. This template should then be submitted as a formal amendment to the IND and a subsequent teleconference between the sponsor and the review division will be set-up by the RPM. The review division will make a recommendation as to whether a request for a BTD is appropriate, may be too preliminary, or does not currently meet the criteria for a BTD. The Agency’s recommendation is advisory and is not to be interpreted to predict the Agency’s decision on the BTD request."
This is helpful. It means that TLT can get advice on how the total numbers of percents are looking and what threshold is best met for the BTD application. This would be especially useful as we don't know how the FDA would interpret the 12 under-treated patients. Or how they would factor in the 3 phase 1 b patients in the total numbers.
Good to know that the process allows for a pre-check in for advice before submitting for BTD.