FDA Meeting FAQs
Questions and Answers about the FDA’s public meeting on narcolepsy drug development
Q: What is the Food and Drug Administration?
A: The United States Food and Drug Administration, also known as the FDA, is a government agency that is in charge of protecting the public health by assuring the safety, effectiveness and security of medications, biological products, medical devices, food, cosmetics, and products that emit radiation.
FDA also improves the public health by helping to speed innovations that make medicines more effective, safer, and more affordable, and by providing accurate, science-based information about health, medicine and food to the public.
Q: Why is the FDA holding a meeting?
A: The FDA is holding public meetings and gathering public comment on narcolepsy and 19 other diseases as part of its Patient-Focused Drug Development Initiative (PFDDI). PFDDI is part of the fifth Prescription Drug User Fee Act (PDUFA V), which was passed by congress and signed into law by the President in July 2012.
The FDA has committed to obtain the patient perspective in 20 disease areas during the course of PDUFA V. For each disease area, the agency will conduct a public meeting to discuss the disease and its impact on patients’ daily lives, the types of treatment benefit that matter most to patients, and patients’ perspectives on the adequacy of the available therapies.
As part of Patient-Focused Drug Development, the FDA will gather patient and patient stakeholder input on symptoms of narcolepsy that matter most to patients and on current approaches to treating narcolepsy.
Q: Why is it important that PWNs attend?
A: The FDA is interested in gaining a better understanding of patients’ perspectives on the severity of narcolepsy and assessments of available therapies. This is a rare opportunity for PWNs to directly and personally address the agency that guides the development of new treatments for narcolepsy.
To attend the meeting or the webcast, you must register by September 13. To register, visit the FDA’s meeting registration page.
Q: How was narcolepsy chosen for this initiative?
A: In the fall of 2012, the FDA released a list of 39 diseases nominated for PFDDI and requested comments from the public, patient organizations and the medical and research communities. PWNs like you took the time to send your comments to the FDA. In fact, narcolepsy was the disease with the third highest number of comments. After reviewing the comments, the FDA released the final 20 diseases chosen, including narcolepsy. In short, narcolepsy was chosen for this initiative through the work of people with narcolepsy!
Q: What will the FDA do with the information it gets from this meeting and the public docket?
A: The FDA is developing a more systematic way of gathering information from the patient perspective. After the meeting, a short meeting report will be shared with FDA reviewers and posted on the FDA website. Patient points of view included in these reports will provide FDA reviewers with helpful insights when conducting benefit-risk assessments for new drugs to treat narcolepsy. Patient responses could also show the FDA that they need new ways to measure the effectiveness of treatments being reviewed.
Q: If I’m not selected to be on the panel, will there still be a chance for me to speak?
A: Yes, there will be lots of opportunities to speak during the facilitated discussion. The FDA wants to hear from as many individuals as possible in their own words and voices.
Q: How long do I have to make comments at the meeting?
A: Comments will not be timed, but those commenting are asked to keep their remarks to 2-3 minutes so that as many people as possible will have the chance to speak.
Q: Do I have to answer specific questions, or can I talk about whatever issue I want?
A: The FDA has specific questions and topics it would like to focus on. See the questions here. During the discussion, the facilitator will periodically ask if anyone has a different experience or opinion to offer, making sure that a wide range of voices are heard.
Q: Can people attending via the webcast offer comments?
A: Yes. Webcast attendees will be able to submit comments during the discussion by typing in comments into a discussion box on the webcast screen. During the discussion period, a moderator will read summaries of comments. At past meeting, moderators have attempted to summarize most of the comments submitted over the web in real-time. With the large number of people expected for the narcolepsy webcast, it may not be possible to summarize every comment.
At meetings for other diseases, the FDA has emphasized during the discussion periods that they would like to hear from patients on the web who might have a different perspective from what has already been offered by other participants. This lets them make sure to cover many perspectives, while managing the large volume of comments that come in through the web.
The FDA acknowledges that they will probably not be able to get to all comments during the meeting. Those whose comments are not included in the meeting are encouraged to share their points of view through the public docket (link to docket). The docket will be open for comments until November 25, 2013.
Q: Do I have to give my name when I offer comments?
A: No. During the facilitated discussion, people attending the meeting in-person do not have to state their name before speaking if they aren’t comfortable doing so.
On the webcast, when participants sign in, they will be asked to provide a name. If they would prefer not to give their full name, attendees may participate using only a first name, initials, or could even type “Anonymous” if they’d like. In addition, when participants ask questions or submit comments, their name and comment/question is only shown to the FDA staff moderating the webcast. The moderator will not disclose any names when reading web comments.
Q: Is it possible that this meeting could actually set narcolepsy treatment back by calling the FDA’s attention to off-label treatments, leading to a ban on those uses?
A. No. The FDA understands the off-label prescribing is necessary in many cases. The FDA’s role is to ensure the safety of new medications and treatments. They do not dictate doctor’s individual prescribing decisions.
Q: Will Idiopathic Hypersomnia (IH) be included in the discussion?
A: Yes. The FDA would like to include IH in the conversation.
Q: I want to attend the meeting in person, but it’s really long. Is the FDA making any accommodations for PWNs?
A: Yes. Those attending are welcome to move around the room, stand in the back, or leave the room and return during the meeting. There will also be a nap room available.
There will be coffee available at the meeting. Those attending will be allowed to bring coffee and food though the security checkpoint, and eating and drinking is permitted in the meeting room.
A: Yes. You can submit comments to the FDA’s public docket until November 25, 2013. All comments should include the docket number FDA–2013–N–0815. Written or videotaped comments can be submitted at http://www.regulations.gov/#!submitComment;D=FDA-2013-N-0815-0001. Written comments can also be mailed to the Division of Dockets Management (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.