Unite Narcolepsy Wrap Up

First slideThe recording of the final webinar in our Unite Narcolepsy series is now available for on-demand viewing.

Date Recorded: Thursday, Oct. 10 at 2 p.m. (Eastern)

Speakers: Monica Gow, Julie Flygare & Kim McCleary

Description: The FDA’s patient-focused meeting on narcolepsy held Sept. 24 was a great success by all measures. We recap what happened at the event, review ways that patients and other stakeholders can offer their perspective to the agency, and look ahead at ways to sustain the positive momentum.

Watch recording: http://www.youtube.com/watch?v=PcTPe83NSR8

Here are links to resources mentioned to during the webinar:

Instructions for submitting your perspective to the FDA’s docket (in writing or by video): http://www.unitenarcolepsy.org/fdas-questions-narcolepsy-community/

Complete the patient-focused narcolepsy survey: https://www.surveymonkey.com/s/unitenarcolepsy

View the archived recording of the 4-hour FDA meeting: http://www.unitenarcolepsy.org/fda-meeting-video-now-available/

Read Unite Narcolepsy’s report on the FDA meeting: http://www.unitenarcolepsy.org/fda-hears-narcolepsy-realities/

Read the first of FDA’s “Voice of the Patient” reports from the patient-focused meeting on chronic fatigue syndrome (CFS, also known as ME/CFS) in April 2013: http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM368806.pdf

FDA Meeting Video Now Available

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In spite of a government shutdown that has challenged operations at many federal agencies, the U.S. Food and Drug Administration has posted the videotapes of the Sept. 24, 2013 Patient-Focused Drug Development Initiative meeting on narcolepsy. The recording includes viewing windows that show the speaker, the slides used by the speaker and a transcript.

View the recording at:

  • Part 1: https://collaboration.fda.gov/p2phbazfvia/?launcher=false&fcsContent=true&pbMode=normal
  • Part 2: https://collaboration.fda.gov/p3b3qbgldex/?launcher=false&fcsContent=true&pbMode=normal

All FDA links related to the narcolepsy meeting are available at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm359018.htm

We will post a detailed meeting summary in the next day or two.

There’s still time to make sure your voice is heard. Here are two ways you can share your experience and perspectives:

  • Complete the Survey: Take 15-20 minutes to respond to the Unite Narcolepsy Patient-Focused Survey based on FDA’s questions and other issues important to narcolepsy patients: https://www.surveymonkey.com/s/unitenarcolepsy All survey responses received by November 15, 2013 will be submitted to FDA before the November 25, 2013 deadline. We will also update the summary of the first 1,350 responses to reflect all responses. We’re also exploring ways to make the valuable data set available to researchers.
  • Respond to the FDA Docket: We’ve made it easy to provide input directly to FDA. Read the step-by-step instructions here or visit the docket site directly.

Press Release from Wake Up Narcolepsy

Wake Up NarcolepsyContact:
Ken McDonnell

Narcolepsy Patient Community Prepares to Share
Life Impacts, Treatment Perspectives with FDA Regulators

Comprehensive patient survey documents delayed diagnosis and
unmet medical needs for this devastating disease

Worcester, MA – September 19, 2013 – On September 24, 2013, patients with narcolepsy and other stakeholders will gather at the Food & Drug Administration to provide direct input on drug development issues affecting nearly 200,000 Americans with narcolepsy. The meeting, fourth in a series of 20 mandated under a 2012 law, will be the first session dedicated to disease classified as rare.

Age at onsetTo prepare the patient community for this opportunity to speak directly with FDA regulators, Wake Up Narcolepsy, Inc., launched Unite Narcolepsy, a special patient education and engagement initiative. A survey based on questions about narcolepsy posed by FDA has attracted more than 1,350 responses, including more than 1,000 from people diagnosed with narcolepsy by a physician. Key survey findings include:

  • Cataplexy, a striking and sudden episode of muscle weakness often triggered by strong emotion, was reported by 65% of respondents. The three symptoms rated as having the most significant impact on patients’ lives were excessive daytime sleepiness (77%), difficulty thinking, remembering, concentrating or paying attention (50%), and general fatigue/never feeling rested (45%).
  • Nearly 95% of survey respondents reported having been prescribed one or more of the four medications approved by FDA for treatment of narcolepsy or its key symptoms (Adderall, Nuvigil, Provigil, and XYREM® [sodium oxybate] oral solution). 70% use other prescription medications (including stimulants, anticataplectics, and hypnotics/sedatives), and 80% use other therapies (such as lifestyle modifications, nutritional supplements, and diet) to help manage their condition. Fewer than 8% reported that they currently pursue no form of treatment for the condition.
  • SeverityEven with available treatments, the impact of narcolepsy on daily life is profound. 83% report not being able to perform as they wish at work or in school. 76% indicate that they have difficulty interacting with family or friends, and 70% can’t get through the day without falling asleep.
  • Nearly 51% of survey respondents reported that it took 6 years or longer to get properly diagnosed.

A  detailed summary of survey responses, including a sampling of respondents’ own comments, can be read at: Interim Survey Analysis.

Monica Gow, Wake Up Narcolepsy co-founder and executive director, is gratified by the community’s candor in sharing their experiences and perspectives. “The thousands of comments submitted tell a heart-breaking story of dreams deferred, career and educational plans put on hold, independence lost, and families torn apart,” she said. “Simple things we take for granted are lost to people combatting narcolepsy.”

Narcolepsy, a disease that is often the butt of unkind jokes, can be very isolating. Unite Narcolepsy organizers reached across the community to educate patients about this unique invitation from the FDA. Julie Flygare, J.D., a popular narcolepsy blogger and book author, helped launch and sustain the effort via social media. Jazz Pharmaceuticals, Inc., whose medication XYREM is the first FDA-approved treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy, will make an unrestricted grant to Wake Up Narcolepsy in support of the Unite Narcolepsy community-building initiative.

Gow, Flygare, and other representatives from the Unite Narcolepsy initiative will attend the FDA meeting, which will also be webcast live; and hundreds of people have registered to participate in person and online. FDA staff members have called the strong response “unprecedented” for this meeting series.

Narcolepsy is a neurological sleep disorder that affects some 200,000 Americans and 3 million adults and children worldwide. Wake Up Narcolepsy, Inc., is a nonprofit organization working to speed diagnosis and help in the search for a cure. To learn more about narcolepsy, other interim results of the patient survey, and the upcoming FDA meeting, please visit www.UniteNarcolepsy.org.

A Parent’s Perspective on Narcolepsy

By Kim Grady

Ben is one of the “lucky” ones. He was diagnosed at the young age of 7 with narcolepsy and cataplexy. I say lucky because it only took two months for him to be diagnosed. You see, it’s not uncommon for many years to pass before this dreadful sleep disorder is diagnosed. We knew something wasn’t right when we watched as he’d repeatedly fall asleep playing with Matchbox cars and friends, at dinner, and in school.

Since Ben’s diagnosis, 9 years ago, I have been on a mission to raise awareness amongst doctors, insurance companies and anyone who “controls” the treatment for people with narcolepsy. That is why I am so thrilled that we at last have the opportunity to tell the FDA how narcolepsy with cataplexy affects Ben’s life and the lives of his loved ones.

KG quoteThe FDA’s Patient-Focused Drug Development Initiative is vital for helping all those with narcolepsy foster better treatment options.  At best, the medications approved for treatment of narcolepsy offer only temporary relief from its debilitating symptoms. For many patients, multiple treatments are required including medications and rigid lifestyle adjustments. For others, available therapies don’t work well or are out of reach because of medical complications or financial limitations. Those with narcolepsy deserve better options so they can achieve their full potential.

Sleeping half your life, as so many people with narcolepsy do, is no way to live. The myths and misunderstanding by doctors and the public adds to the burden patients and their loved ones carry. By sharing our stories with the FDA and helping them understand just how challenging it is to live with narcolepsy,  we will increase our chances of one day getting treatment that  will be more effective and cause fewer side effects.

As Ben has said, “I know there are worse things to have, but narcolepsy is pretty bad.  There are certain jobs I will never be able to do, like serve in the military – which I really want to do. Driving a car will be difficult, and I love cars!  Having narcolepsy does restrict my life.” As his mother, every day I see the social embarrassment and his struggle to keep up academically. And his cataplexy withholds him from laughing for fear of falling down during an attack.

The medications Ben has to take are many, and because research has been limited – especially on children with narcolepsy – we don’t know what long-term effects of the disease may affect him later in life. It’s time our government and the pharmaceutical companies understand how difficult it is to live with narcolepsy and cataplexy. We must do what we can to raise awareness and understanding.  For Ben and the other 200,000 Americans with narcolepsy, please help Unite Narcolepsy, so better treatments will become available, and one day they will be cured of this devastating disease.

Kim Grady is an experienced financial sales professional with over 10 years experience. She currently works with several non-profits on fundraising and development projects. Working with Wake Up Narcolepsy as the organization’s Community Outreach Coordinator, Kim has implemented a new program to raise awareness in the Massachusetts school community called, “Narcolepsy Goes to School.” She has also been active in launching the Unite Narcolepsy initiative to educate, engage and empower the narcolepsy community to participate effectively in the FDA’s Patient-Focused Drug Development Initiative.

Get Involved

Narcolepsy affects every person differently, so we need many voices to capture our community’s full experience. This is a unique opportunity for people with narcolepsy and those who love them to help shape the future of narcolepsy treatment. Learn more about how you can participate!

Community Voices

We’re rallying people across the narcolepsy community to share their experiences about what it’s like to live with narcolepsy with FDA decision-makers who are listening. Hear from some of your fellow community members on our “Voices” blog.

What’s It All About?

Narcolepsy is in the spotlight! The FDA wants to hear from patients, their caregivers and loved ones about what it’s like to live with narcolepsy. Unite Narcolepsy has been created to inform and empower our community to make the most of this unique opportunity.