Fact Sheet: Breakthrough Therapies
The Food and Drug Administration Safety and Innovation Act (FDASIA) includes a provision that allows sponsors to request that their drug be designated as a Breakthrough Therapy. FDA is in the process of developing guidance related to this designation. Until guidance is developed, requests for Breakthrough Therapy designation should follow the criteria outlined below. A request for Breakthrough Therapy designation should be submitted concurrently with, or as an amendment to an Investigational New Drug Application (IND) with a cover letter, a completed form 1571, and the following information:
Identification in the cover letter of the submission as a REQUEST FOR BREAKTHROUGH THERAPY DESIGNATION in bold, uppercase letters.
The IND application number, if applicable.
The proprietary and/or generic name and established name for drug products; proper name and trade name for biological products.
The division or office where the IND is being submitted to or is active.
The proposed indication(s) for your product.
A brief, but comprehensive, summary of information that justifies why your product qualifies for a Breakthrough Therapy designation for the indication being studied, including:
Evidence that the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition,
Preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
If applicable, a list of documents previously submitted to the IND that are deemed relevant to the designation request, with reference to submission dates so the documents can be readily located. Copies of such documents can be resubmitted to the FDA as appendices to the designation request (if not too voluminous).
In most cases, this information could be captured in an approximately 10-20 page document.
No later than 60 days after receipt of the submission, a determination will be made to either grant or deny the request for Breakthrough Therapy designation in the form of a designation letter (for requests granted) and a non-designation letter (for requests denied).
The Food and Drug Administration Safety and Innovation Act (FDASIA) includes a provision that allows sponsors to request that their drug be designated as a Breakthrough Therapy. FDA is in the process of developing guidance related to this designation. Until guidance is developed, requests for Breakthrough Therapy designation should follow the criteria outlined below. A request for Breakthrough Therapy designation should be submitted concurrently with, or as an amendment to an Investigational New Drug Application (IND) with a cover letter, a completed form 1571, and the following information:
Identification in the cover letter of the submission as a REQUEST FOR BREAKTHROUGH THERAPY DESIGNATION in bold, uppercase letters.
The IND application number, if applicable.
The proprietary and/or generic name and established name for drug products; proper name and trade name for biological products.
The division or office where the IND is being submitted to or is active.
The proposed indication(s) for your product.
A brief, but comprehensive, summary of information that justifies why your product qualifies for a Breakthrough Therapy designation for the indication being studied, including:
Evidence that the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition,
Preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
If applicable, a list of documents previously submitted to the IND that are deemed relevant to the designation request, with reference to submission dates so the documents can be readily located. Copies of such documents can be resubmitted to the FDA as appendices to the designation request (if not too voluminous).
In most cases, this information could be captured in an approximately 10-20 page document.
No later than 60 days after receipt of the submission, a determination will be made to either grant or deny the request for Breakthrough Therapy designation in the form of a designation letter (for requests granted) and a non-designation letter (for requests denied).
