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Frequently Asked Questions: Breakthrough Therapies

What are the benefits of a 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), more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review. 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, 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

What are the differences between the criteria for breakthrough therapy designation and fast track designation?
Breakthrough therapy and fast track designation programs both are intended to expedite the development and review of drugs for serious or life-threatening conditions, but there are differences in what needs to be demonstrated to qualify for the programs. A breakthrough therapy designation 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 designation is for a drug that treats a serious or life-threatening condition, and nonclinical or clinical data demonstrate the potential to address unmet medical needs for the serious condition.

Can a sponsor submit a request for breakthrough therapy designation for multiple indications of the same drug?
A breakthrough therapy designation applies to a combination of a drug (either alone or in combination with other drugs) and the specific use for which it is being studied. A separate breakthrough designation request must be submitted for each proposed development program (i.e., each indication for a drug (or drug combination)).



This is also useful posted by another HC poster couple of days ago...

FDA’s New Breakthrough Therapy Designation: What does it mean for Pricing and Market Access?