Narcolepsy selected for FDA Patient Focused Drug Development Initiative (PFDDI)

by julie on April 12, 2013

I’m thrilled to announce that NARCOLEPSY has been selected for FDA’s Patient-focused Drug Development Initiative (PFDDI).  Narcolepsy has been selected as one of the initial 12 disease areas for individual evaluation to be addressed in FY 2013 – FY 2015.

We did it!  

I testified in person and led the campaign for online comments. And did you help me?

“Over half of the comments received concerned lung cancer, narcolepsy, and interstitial lung disease,” FDA’s official announcement states here

Thank you for supporting my efforts. Together, we raised our voices and FDA heard us!

Now what? 

FDA 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 available therapies. These meetings will include participation of FDA review divisions, the relevant patient community, and other interested stakeholders.

Narcolepsy is scheduled to be addressed later this year starting in Sept 2013.  This initiative will allow FDA to better understand the patient perspective in evaluating any upcoming narcolepsy treatments for FDA approval. As hypocretin agonists become more of a reality for future narcolepsy treatment, this is an exciting opportunity for patients to aid FDA in understanding what matters most to us. For full details, visit the FDA website.

IMPORTANT:

Subscribe to my mailing list (right-side panel) for future participation opportunities!  

For updated posts about the FDA Initiative, click here.

{ 35 comments… read them below or add one }

Sharron April 12, 2013 at 3:53 pm

Thank you for all of your hard work on our behalf, Julie.

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julie April 12, 2013 at 4:08 pm

You’re welcome, Sharron. Thank you for supporting my efforts.

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Julie Ann Funk April 12, 2013 at 3:59 pm

So incredible!! I know I got my comments in there, and a number of my friends did, as well. You truly are amazing with the work you do to bring narcolepsy to the forefront, and I can’t thank you enough for it! <3

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julie April 12, 2013 at 4:09 pm

Thank you, Julie Ann for submitting comments and rallying your friends to submit comments too. We did it!! Jumping for joy today.

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Elaine Garza April 12, 2013 at 4:16 pm

This is incredible news! Great job, Julie! I provided comments online and also sent the link to friends and family to contribute! Way to go!

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julie April 12, 2013 at 5:59 pm

Thank you so much, Elaine. Your efforts made an important impact. Every letter counted towards drawing FDA’s attention to our condition.

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Evan Callicoat April 12, 2013 at 4:27 pm

Awesome!! This is fantastic news. So glad to have contributed in a small way to this accomplishment.

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julie April 12, 2013 at 5:58 pm

Thank you so much for supporting this effort, Evan! We did it!!

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Anna aka Sleepymoon April 12, 2013 at 5:59 pm

Julie, you ROCK, and this is AWESOME NEWS!!! So exciting!! Way to make a difference. Hopefully we will all sleep better as more of society starts to recognize and respect our need for sleep instead of calling us lazy and crazy. Doctors included!!

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julie April 12, 2013 at 6:53 pm

Thank you, Anna! I am hopeful there’s a bright future for narcolepsy, with people respecting our disorder and better treatments to improve quality of life. With gratitude and smiles, Julie

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elizabeth April 12, 2013 at 7:14 pm

Julie!!! This is awesome! Thank you for spreading the word and enabling us to participate in the effort. And thank you for your advocacy; I am so grateful that you are out there representing us!

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julie April 14, 2013 at 5:16 pm

Thank you, Elizabeth. It’s an honor and joy to represent the community. Each and every person that submitted a comment to FDA made this possible. :-)

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Jennifer Miller April 12, 2013 at 7:37 pm

I am so glad that you are able to do these things. I hope people will listen and respond. Where I live they hand you the results and let you know you can now get medication and that is all. No follow up, no additional tests, just cope best you can. I am a 3rd generation sufferer, which seems to not be the norm. There are four diagnosed cases that I know of, my grandmother, (out of her four kids) my mother and my sister and myself (my mothers only kids). I have 3 girls my oldest behaves like my sister when she was young and my middle child reminds me of myself, this causes concern that they may have it as well but I have been told “It dose not present until mid 20′s. ” This I believe to be hog wash! I had suspected and found more reason to believe since I have found a group to commiserate with which led to my learning of you and reading what you have wrote, that I have been fighting this from at least the 5th grade. Ignorance is not bliss whether the doctor or the sufferer. So I want to thank you for all that you do and may productive sleep bless your rest. Jennifer

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julie April 14, 2013 at 5:14 pm

Hi Jennifer, Thank you for your comment. I’m sorry to hear you are a 3rd generation person with narcolepsy. I agree that it’s a myth that narcolepsy doesn’t present until mid-20s. Top specialists recognize that narcolepsy development in children is increasingly common, but we’re still getting the word out to all doctors. I believe narcolepsy will gain more accurate attention in the future, at least that’s my goal! Sending wakefulness and many thanks your way, Julie

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JudePNP April 12, 2013 at 7:41 pm

Fantastic! I was thrilled to see this news. What a wonderful improvement in the quality of life and opportunities this will make for my 4 daughters, myself and my grandson….all narcoleptic. I’ll help in any way I can.

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julie April 14, 2013 at 5:18 pm

Thank you for your support, Jude! I’m so sorry to hear so many members of your family are affected. Please sign up for my mailing list for future participation opportunities.

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Susan April 12, 2013 at 11:21 pm

This is great news! Thank you for testifying and leading the effort to get people to submit comments. I submitted my comments and tried to spread the word. Your advocacy work has led to some amazing accomplishments.

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julie April 14, 2013 at 5:09 pm

Thank you, Susan for submitting comments and spreading the word! I’m so proud of our community coming together to support our common goal of a brighter future.

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TheDreamer April 12, 2013 at 11:57 pm

Yes! I know my comment is on there…. wonder what it’ll look like when it appears?

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julie April 14, 2013 at 5:08 pm

Thank you so much for posting a comment for the FDA initiative, Dreamer! Every comment counted toward our selection.

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Jennifer Beetz April 13, 2013 at 12:22 am

I have a couple (two) friends with narcolepsy. I’m so very, very happy for them and I know a great deal of effort went into making this initiative happen- effort from those who suffer from narcolepsy and also their families and friends…

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julie April 14, 2013 at 5:07 pm

Thank you, Jennifer for supporting your friends’ experience with narcolepsy and our efforts to build a brighter future. We will keep fighting together as a community for change!

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Mike April 13, 2013 at 12:36 am

Congrats on all your hard work paying off Julie! I look forward to the public hearings in the fall. I will be keeping an eye out for the announcements and hope to be able to attend.

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julie April 14, 2013 at 5:06 pm

Thank you so much, Mike! I will keep folks posted via my blog and mailing list.

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Gail Monte April 13, 2013 at 12:39 am

Such amazing news!! Thanks for everything!!

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julie April 14, 2013 at 5:06 pm

You’re so welcome, Gail! Great news, indeed.

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Liz April 13, 2013 at 12:56 am

Julie,
This disease is new to my daughter and me, so we were not involved when you all were fighting for us. Thank you so much for everything you have done!

Liz

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julie April 14, 2013 at 5:05 pm

Liz, I’m so sorry to hear your daughter was recently diagnosed. Thank you for supporting my efforts now. -Julie

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Stacy April 13, 2013 at 5:04 pm

Thank you, thank you, thank you for being our “leader”, our activist! Without you Julie, none of this would be possible!! I’m forever grateful in hopes that one day soon all of us can be freed of this horrible disease!

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julie April 14, 2013 at 5:02 pm

Thank you so much, Stacy! I knew in my heart I had to take this project on, it was the right thing to do, and I’m so glad I took the time and effort. The community supported me and I couldn’t have done it without each and every person that sent in a comment! Big smiles and many thanks, Julie

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Susan April 27, 2013 at 12:27 am

I was given your name by a friend of my daughter who has narcolepsy. I bought yur book & am reading it. I started a blog years ago, but too tired most nights to write in it – A Narcoleptic Tale. I started it for my kids & grandchildren. I was diagnosed when I was in Nursing school at 20 years old. I am now 62 & drs are just as ignorant as they were in the 70′s. I shouldn’t say drs I mean the whole medical community. Because of what the ritalin has done to my heart I need to go on a newer drug. Insurance won’t approve it because I never had a sleep study done. Had one study – they are telling me I have sleep apnea. I’ve had cataplexy for 40 yearss! And as a matter of clarification, the illness doesn’t seem to progress, unfortunately as we get older, we get more tired! I thought you might find this interesting. When I was young – maybe 5 I has scarlet fever. Then at about 8 or 9 I hit my head while ice skating had a concussion. Next at 12 I got the Hong Kong flu (the name of the strain in the 60′s) Finally in college, celebrating my birthday & drinking too much I had a severe head injury, shortly after I started having symptoms. My family didn’t understand at all. Before my mom died in 2004 she apologized for not understanding what I was going through. I told her there was nothing she could do, I had to learn my way on my own. I am the only 1 of 6 with narcolepsy but I worry about my hhildren & grandchildren. My older sister joined the National Geographic genome project. She sent in a DNA swab & they sent her a mapping of our family’s mitochondrial Eve. She sent in another swab & they analyzed it. My dad has heart disease & diabetes on his side of the family. The only genetic illness they found was Narcolepsy!!That was exciting!
I am here to tell you I appreciate what you are doing so my descendents don’t have to go through what I went through & to volunteer if you need help!

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julie April 27, 2013 at 8:14 pm

Susan:

Thank you so much for your kind comment. I’m so sorry to hear you’ve had narcolepsy for so many years and dealt with so much misunderstanding. It’s my greatest hope to change public perceptions and raise awareness. We are stronger together – thank you for your support.

Sending wakefulness your way, Julie

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Shelley Bailey May 10, 2013 at 7:45 pm

Hi Susan,
My daughter was diagnosed with Narcolepsy/Cataplexy at age 15 and my co-workers sometimes joke that I’m slightly narcoleptic because of how fast I can go to sleep at my desk. I found your comment about scarlet fever interesting because my daughter also had it around age 3 or 4. When she was about five she said “I wonder if when you’re asleep you’re really awake and when you’re awake you’re really dreaming.” In retrospect I realize she had the disease for much longer than we thought. For many years whenever she got a fever she would get blisters on the palms of her hands too. We just submitted our DNA to 23andme, along with my Mom and Dad’s. We are curious to see if there is a genetic link. Sadly, like your family, most of my family views it as malingering and we’re both still trying to climb out of the depression it brought.
On a final note, do any of your family members have an inner ear disorder called Menier’s disease? I’ve run across a few narcoleptics on various boards with it and am wondering if there is a connection. Take care and know that the generations behind you are getting better at spotting this! Shelley Bailey
Fremont CA

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doug dietrich May 17, 2013 at 1:09 pm

amphetamines are scarcely understood yet are highly effective in promoting wakefulness. as patent rights have expired, no profit motive exists to further research this proven, yet largely not understood, treatment option.

today, there is new hope on the horizon for myself and others who benefit from this drug, and especially those who could benefit if it were better understood.

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julie May 21, 2013 at 1:56 pm

Great point, Doug! Thanks for your comment.

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