Jun 08 2019
Press Release: Narcolepsy Patient Community Prepares to Share Life Impacts, Treatment Perspectives with FDA Regulators
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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 a disease classified as rare.
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 condition.
- Even 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 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 combatting 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.