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:

• If the breakthrough therapy designation request is submitted to the sponsor’s IND as an amendment, the cover letter should indicate the submission as a REQUEST FOR BREAKTHROUGH THERAPY DESIGNATION in bold, uppercase letters. If the request is submitted with an initial IND, the cover letter should indicate the submission as both an INITIAL INVESTIGATIONAL NEW DRUG SUBMISSION and REQUEST FOR BREAKTHROUGH THERAPY DESIGNATION in bold, uppercase letters.

• In the cover letter of the submission, the name of the sponsor’s contact person, including the person’s address, email address, telephone number, and fax number.

• If applicable, the IND application number should be noted.

• If available, include, for drug products, the proprietary name and active ingredient and, for biological products, the proper name and trade name.

• The division or office to which the IND is being submitted or in which it is active.

• The proposed indications.

• A concise summary of information that supports the sponsor’s breakthrough therapy designation request for the indication being studied, including:
  • The basis for considering the drug to be one intended to treat a serious condition

  • The preliminary clinical evidence that the drug may demonstrate substantial improvement over available therapies. A sponsor should describe the preliminary clinical evidence, including, for example, justification for the clinical study endpoint used and a brief description of statistical analyses

• If applicable, include a list of documents previously submitted to the IND considered relevant to the designation request, with reference to submission dates. Paper submissions can be resubmitted to FDA as appendices to the designation request.