The US Food and Drug Administration (FDA) has further clarified and updated its guidance on the system for resolving complicated medical and scientific disagreements between drug sponsors and the agency.
The draft guidance is meant for cases when a sponsor may disagree with FDA and a dispute arises. The disputes often involve complex scientific and/or medical matters, so FDA deems it critical to have procedures in place to help ensure an open, prompt discussion.
The revisions of the previous guidance – issued in March 2013 – offer more clarity, particularly on when a formal dispute resolution requests (FDRRs) are necessary, and more details on supporting background information. The draft also clarifies cases when the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) intend to manage formal requests for appeals of disputes.
FDA also updated its guidance so that biosimilar sponsors can have the same formal process for raising a dispute with the agency as other companies filing investigational new drug applications (INDs), new drug applications (NDAs), biologics license application (BLAs) and abbreviated new drug applications (ANDAs). In all cases, CDER and CBER set a goal of responding to an appeal of a dispute within 30 calendar days.
"The procedures and policies described in this guidance are intended to promote rapid resolution of scientific and/or medical disputes between sponsors and CDER or CBER," the guidance says.
For applications not covered by the Prescription Drug User Fee Act of 1992 (PDUFA), the Biosimilar User Fee Act of 2012 (BsUFA), or the Generic Drug User Fee Amendments of 2012 (GDUFA), and applications for CBER- regulated medical devices (covered by the Medical Device User Fee Act), the procedures described in the guidance "generally will be applied and the time frames will be met as resources permit," FDA says.
FDA notes that its communications, including meeting minutes or general advice letters, typically include recommendations and/or advice for a sponsor that generally convey CDER’s or CBER’s current thinking on a particular topic. However, such advice is not a regulatory action so it would not be an appropriate subject for an FDRR.
In addition, when a scientific and/or medical dispute arises, the sponsor should initially seek to resolve the matter with the original deciding official before making an appeal to the next higher management level.
FDA lists the following examples of regulatory actions appropriate for an FDRR:
- complete response (CR) letter
- IND clinical hold (partial or full)
- request for breakthrough therapy designation denied
- request for proprietary name review denied
- refuse to receive for an ANDA
In addition, after a sponsor has decided to submit an FDRR, as part of its appeal it can also request a meeting with the deciding official for the appeal.
The draft guidance also includes procedures for submitting a request for a formal dispute resolution and information on responses to an appeal.