This is the second entry in a running series about U.S. Food and Drug Administration guidance documents. View the first entry, “What is an FDA guidance document?”, here.
“I’m from the government, and I’m here to help you.” This short phrase instills fear in some who hear it, but for drug developers it is often essential to be able to sit down with government officials. Meeting with officials of the United States Food and Drug Administration (FDA) early in a product’s development cycle is of utmost importance, so that both parties may come to a mutual understanding of the best way to proceed. Firms regularly request meetings with the FDA, and for this reason, a draft guidance, Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products, was among the bolus of guidance documents released by the Agency in December 2017.
Let’s start with a definition: PDUFA is an acronym for the Prescription Drug User Fee Act, a law passed by Congress in 1992 which allowed FDA to begin collecting user fees from drug firms in return for the Agency’s commitment to meet particular timelines in the drug review process. FDA uses a portion of the money collected to hire additional review staff to speed the process.
Three types of formal meetings with FDA
There are three basic types of meetings with FDA: Types A, B, and C:
- Type A meetings serve the purpose of finding a path forward when a product’s development is stalled or dealing with a significant safety issue. A typical reason for a Type A meeting is when an Investigational New Drug (IND) has been put on clinical hold.
- Type B meetings are the most common type of meeting with FDA. They tend to be check-ins at various points in the drug development process, such as pre-IND, end of Phase 2, pre-NDA/BLA (New Drug Application or Biologic Licenses Application)
- Type C meetings are all meetings that are neither Type A or B. An example the guidance provides is the use of a biomarker as new surrogate endpoint for a key clinical study.
Initiating a formal meeting request
The process begins with a Meeting Request in which you, the drug developer, tell the Agency what the purpose of the meeting will be, including the questions to which you would like responses. A proposed range of dates for the meeting must also be provided. You can request a face to face meeting, a teleconference, or just written responses to your questions. FDA reserves the right to grant or not grant the meeting based on the state of development of your drug, the medical need, and their available resources.
If FDA grants your meeting, you must then provide a Briefing Document with the questions proposed in the original meeting request and any necessary background information/data to provide context for the questions.
Framing your questions
The most important thing to understand is how to frame your questions. The guidance doesn’t tell you this, but you must resist the temptation to state a question like this, “If we take this approach (e.g. to a clinical trial or chemical synthesis), will you approve it?” FDA’s answer to that type of question will always be, “That’s a review issue,” and, as a result, your question won’t be answered. One best practice is to frame a question like this: “If we take this approach (e.g. to a clinical trial or chemical synthesis), will that provide you with an appropriate data set to enable you to make a scientifically sound public health decision?” Questions framed in this manner are far more likely to receive useful answers from FDA.
Knowing how to set up meetings with regulators and understanding the art of framing questions are part of the services offered by Pearl Pathways. We can help you find your way through the regulatory pitfalls to speed the development of your drug and deliver it to patients sooner. That’s why we are here. Contact us today for a free 30-minute consultation.