2) What is breakthrough therapy designation?
Breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A breakthrough therapy designation conveys all of the fast track program features (see below for more details on fast track designation), as well as more intensive FDA guidance on an efficient drug development program. The FDA also has an organizational commitment to involve senior management in such guidance. Section 902 of FDASIA requires the following actions, as appropriate:
holding meetings with the sponsor and the review team throughout the development of the drug
providing timely advice to, and interactive communication with, the sponsor regarding the development of the drug to ensure that the development program to gather the nonclinical and clinical data necessary for approval is as efficient as practicable
taking steps to ensure that the design of the clinical trials is as efficient as practicable, when scientifically appropriate, such as by minimizing the number of patients exposed to a potentially less efficacious treatment
assigning a cross-disciplinary project lead for the FDA review team to facilitate an efficient review of the development program and to serve as a scientific liaison between the cross-discipline members of the review team (i.e., clinical, pharmacology-toxicology, chemistry, manufacturing and control (CMC), compliance) for coordinated internal interactions and communications with the sponsor through the review division’s Regulatory Health Project Manager
involving senior managers and experienced review staff, as appropriate, in a collaborative, cross-disciplinary review
4) What are the differences between the breakthrough therapy designation and the fast track designation?
Although breakthrough therapy and fast track designation programs have similarities, as they both are intended to expedite the development and review of drugs for serious or life-threatening conditions, there are differences in what needs to be demonstrated to qualify for the programs. 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
Also well worth revisiting a Bloomberg News article from February last year -
One of the most tantalizing new programs now being rolled out at the FDA is its brand new category for "breakthrough" drugs, offering a select number of companies a chance at a shortcut to the market based on early-stage data for transformational new therapies.
So when Janet Woodcock, the influential director of the FDA's Center for Drug Evaluation and Research, started outlining how the program will work--indicating that a company can move from an expanded Phase I directly to commercialization, Bloomberg reporters were paying close attention.
According to the business news wire, Woodcock says that companies which earn breakthrough status will have the ear of the agency.
"We expect many of these would come available very quickly with Phase I data," she said.
So far, we know that Vertex ($VRTX) has won breakthrough status for two drugs for cystic fibrosis, the approved drug Kalydeco and the experimental VX-809, now being studied as a combination therapy.
According to Woodcock, a third drug has been anointed with the special status. And developers have submitted 18 for review, most of which are for cancer.
Biotechs have been paying particularly close attention to this new, infinitely shorter path to the marketplace.
A breakthrough designation would revolutionize their commercial prospects, upending some well known methods for building value through long-term partnerships with Big Pharma--which can easily take years to complete.
Whether the FDA will allow smaller companies with less experience dealing with the FDA on the ultimate inside track, though, will be interesting to see.