March 13, 2013

$PVCT & Breakthrough Therapy Designation

A friend of the blog provided this link from the FDA's website: FAQs about the designation. Thank you. Updated statistics about application numbers, receipts and denials can be found here (as the FDA website is updated as of 12/31/12, and will be updated quarterly). I selected portions of the FAQs and provided them below in italics (bold emphasis is mine).

"Breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions."

"A breakthrough therapy designation conveys all of the fast track program features..., as well as more intensive FDA guidance on an efficient drug development program."

"A breakthrough therapy program is for a drug that treats a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. In contrast, a fast track program is for a drug that treats a serious or life-threatening condition, and nonclinical or clinical data demonstrate the potential to address unmet medical need."

"After a development program is designated as a breakthrough therapy, the FDA will work closely with the sponsor to ensure that the design of the clinical trials is as efficient as practicable, when scientifically appropriate. An efficient trial design typically would help minimize the number of patients exposed to a potentially less efficacious treatment, which could translate to fewer patients enrolled in clinical trials that support marketing approval. Regulatory standards to demonstrate safety and efficacy must still be met."

"A breakthrough therapy designation and SPA are two independent regulatory pathways. Therefore, a drug designated as a breakthrough therapy is eligible for SPA, if the protocol meets the criteria for SPA. Conversely, a drug for which an SPA is under review may be considered for breakthrough therapy designation, if the breakthrough therapy designation criteria are met."

"[T]he FDA may suggest that the sponsor consider submitting a request for breakthrough therapy designation if: (1) after reviewing submitted data and information (including preliminary clinical evidence), the Agency thinks the drug development program may meet the criteria for breakthrough therapy designation and (2) the remaining drug development program can benefit from the designation."

The FDA knows Provectus always wanted an expedited regulatory path for PV-10. The company's MM Phase 2 trial was designed for accelerated approval. How does the regulatory path potential get clarified? Moffitt data at the annual meeting of the American Association for Cancer Research in April.

Management has long spoken of a very collaborative relationship with the FDA. Did the Agency suggest to them that Provectus submit a request for breakthrough therapy designation for PV-10...?

No comments:

Post a Comment