Preliminary Breakthrough Therapy Designation (BTD) AdviceThe
FDA BTD FAQ site makes it clear that
preliminary BTD advice is advisory and does not predict the actual
BTD decision.
Frequently Asked Questions: Breakthrough Therapies Question 22. What are the timelines for FDA to respond to a breakthrough therapy designation request?
FDA will respond to breakthrough therapy designation requests within 60 days of receipt of the request.
Question 23. Can a sponsor get preliminary breakthrough therapy designation (BTD) advice from the review division prior to the submission of a formal BTD request?
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. NOTE: A Preliminary BTD Advice Request may be submitted to an active PIND, although a formal BTD request may not be submitted until the IND is opened.