Tags: fda

Narcolepsy Network Conference 2013 in parts ... 2


So, I got up a bit before 6am, leisurely got ready since breakfast wasn’t until 7:30am. But, I was pretty much ready to go down at 6:30am, as I hadn’t gotten out my computer and resisted the need to use it. I did neglect some of the things I need to do regularly, like my voluntary role as moderator for a couple of mailing lists. But, when I finally returned to the real world, it was clear that somebody else had taken care of it. It has always felt like there was another person moderating as well, though usually the spam just piles up until I get rid of it. But, everything was clear this time. So, I guess no worries when I got on future vacations.

Anyways…I went down and wandered back and forth watching people set up for the day, along with the various other people that had also come down early to wait for breakfast. A little after 7am, I found myself back at the breakfast area and there were other people already getting food and finding tables to occupy. So, I got breakfast too. I picked an empty table by the door, which gradually filled up during breakfast and I met people from past conferences, as well as first timers. Eventually, I got up for seconds, and came back to find that my spot had been cleared…so I had to get another set of utensils.

Breakfast was kind of interesting in setup and layout….the tables were empty and you had to pickup your own napkin/utensil set outside, as well as all drinks. Which was okay, but what was odd was that the space was split across the hall. So, it would make it difficult if you were looking to connect with specific friends during breakfast, etc. Then I made my way to the room where the opening speakers would be. Things started late. Since there was to be a Welcome and announcements bit at 8:45am, before the two main opening speakers at 9:00am and 10:00am. But, the Welcome/announcements were rushed through a little after 9am. Probably having breakfast scheduled to 9am was a factor, though there were also some technical issues.

Keynote Speaker

This was a “Narcolepsy Update” by Dr. Michael Thorpy. Which I recall touched on the recent developments in Narcolepsy in the last few years, including that most of the understanding and work has been in the last few decades, despite it being first described in the 1800’s. He made a note to mention other speakers at the conference that would be speaking more in depth on some of the areas he touched on.

Following this, was Dr. Jerry Siegel, who spoke of “A New Development in Our Understanding of Human Narcolepsy". Which like all speakers start with a summary of what is Narcolepsy, and then a review of hypocretin cell loss associated with Narcolepy. And, then got into the new development. A major increase in Histamine cells in Narcolepsy, yet a low concentration of histamine in CSF.

Not sure what the development means for Narcolepsy though…. since Histamine is wake promoting (which is why anti-histamines make you drowsy), would they be looking to increase Histamine levels as wake to increase wakefulness. Where an obvious downside is that it might make you more allergic or have strong allergic responses to allergens. Or, is it because this is the first time they’ve seen an increase of a certain type of brain cell in people. Where the general understanding has been you are born with a certain amount, which would otherwise stay constant throughout your lifetime if you didn’t have events, conditions, or such that result in the loss of brain cells. And, such losses are irreversible.

After this, was the first breakout session.

I went to “Narcolepsy: Is there an environmental trigger?” by Dr. Todd Swick. After a few minutes, it dawned on me that the answer is definitely yes. Given that its considered an autoimmune disease, where large portions of the population have the HLA(s) that have been linked to Narcolepsy, but not everybody develops Narcolepsy. Including a note that if there is Narcolepsy with an identical twin, the other doesn’t develop it. Even though they obviously have the same genes, and had mostly the same exposures in early development.

But, while the big focus seems to have moved from streptoccocus to H1N1 as the trigger, he talked about other things that have been associated with the onset of Narcolepsy. Like pneumonia (which I do know I had when I was a child), or heavy metal exposure (where I know I’ve had exposure to mercury….)

This session was followed by Lunch, which included the Membership meeting…where things got quite heated. And, eventually adjourned to sometime on Sunday, so that the rest of the conference can try to keep to its tight schedule.

That was probably another weird thing with the Hotel this year, we didn’t have any space that was ours for the entire weekend, not even most of the day. It was we had to end at 5pm, because they needed to reset the space for another group/event in the space.

Anyways…the first session I caught after lunch was “Narcolepsy Advocacy Update on the FDA Patient Focused Initiative” by Dr. Mark Patterson and Julie Flygare, JD…hmm, just noticed they have her name as “July Flygare” in the schedule :DD

Seemed it was to go back and forth between Mark and Julie, though Julie soon found that she was doing most of the talking. Which was fine with me &#59;D Didn’t really learn a whole lot that I didn’t know, having heard about it early on during the dinner cruise last year which led to lots of attention to get involvement following last year’s conference, and continued attention up to the meeting on September 24, 2013. Though it was interesting to hear a bit more in depth of the panelists, and connecting that someone I had met on Friday was one of the panelists, etc.

This was then followed by a “Narcolepsy and Hypersomnia Research Update” by Dr. Lynn Marie Trotti, which I was really interested in hear, since I had caught her presentation during my first NN Conference in DC and had found it interesting, despite not know what I was going into. And, I had wondered about things over the years. Her update was more interesting in that during the presentation she asked how she was doing for time, and learning that there might not be enough time to cover everything. She started talking faster. But, I could still follow along, and in fact it seemed to make it easier to maintain my focus. And, she would still respond to questions, etc.

There wasn’t much of a break, and I then made my way to the “How to Cope in the Work Place Professional Panel"….which turned out to not really be what I had thought it would be, but it was still kind of interesting. And, it was certain interesting to find out some of the things a person with narcolepsy does for work. Namely Mee who is a trader on wall street. Generally, the main theme was that we work in something that we are interested and/or passionate about. With the exception of Sara K., who is working in something really boring and survives by doing Narcolepsy Network business to break up the monotony of her work.

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Prescription Drug User Fee Act -- Patient Focused Drug-Development -- Docket Number FDA-2012-N-0967

Link: http://www.gpo.gov/fdsys/pkg/FR-2013-04-11/pdf/2013-08441.pdf

Last year the FDA soliciting public comments to narrow down the diseases to focus on for its Patient-Focused Drug Development Initiative, which has the goal of obtaining the patient's perspective on certain disease areas during the five period of the PDUFA (Prescription Drug User Fee Act) V. To assess a product's benefits and risks involves an analysis of the severity of the condition treated and the current treatment options available for the given disease. This information is a critical aspect of FDA's decision-making as it establishes the context in which the regulatory decisions are makde. The FDA believes that drug development and their review process could benefit from a more systematic and expansive approach by obtaining the perspective of patients on disease severity and current available options in a therapeutic area.

They had nominated 39 diseases, where narcolepsy was one of them, and were looking to narrow the list down to 20.

And, the result is Narcolepsy got selected! :cool:

Even though there were directions on how to make the comment anonymously, I choose to include my name and I had gone with greater emphasis for Narcolepsy since it was my most recent diagnosis, and since it was first brought to my attention during the after dinner conversation aboard the Nautica Queen....though we Narcoleptics tend to have more than one thing wrong...and perhaps someday I'll get to know what my current problem is called (something I've had over over 10 years now, but only started getting really bad the shortly after the conference.)

The conference was October 19-21, 2012...I got back on the 23rd, and wrote the letter on the 24th. Since I had book Wednesday to Wednesday off from work for the conference....

The resulting list for FY 2013-2015 are: Alpha-1 antitrypsin deficiency; breast cancer; chronic Chagas disease; female sexual dysfunction; fibromyalgia; heomphilia A, hemophilia B, von Willebrand disease, and other heritable bleeding disorders; HIV; idiopathic pulmonary fibrosis; irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors; lung cancer; myalgic encephalomyelitis/chronic fatigue syndrome; narcolepsy; neurological manifestations of inborn errors of metabolism; Parkinson's diseases and Huntington's disease; pulmonary arterial hypertension; and sickle cell disease.

There will be another public process to determine the list of disease areas for FY 2016-2017.

Wonder what the future meetings will be....

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