Last updated on April 23, 2014 at 13:51 EDT

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

September 19, 2013

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

WORCESTER, Mass., Sept. 19, 2013 /PRNewswire-USNewswire/ — 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 a disease classified as rare.

(Photo: http://photos.prnewswire.com/prnh/20130919/DC82957-INFO)

To 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 1,330 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
    --  Even with available treatments, the impact of narcolepsy on daily life
        is profound.  83% percent 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 more detailed summary of survey responses including a sampling of respondents’ own comments can be read at http://www.unitenarcolepsy.org/wp-content/uploads/Interim-Survey-Analysis-v1.pdf.

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 combating narcolepsy.”

Life with a 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., 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 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.

SOURCE Wake Up Narcolepsy

Source: PR Newswire