Narcolepsy Network Joins Narcolepsy Community at Historic FDA Public Meeting

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Historic FDA meeting on narcolepsy drug development brings narcolepsy community to FDA to offer candid and emotional remarks on how narcolepsy affects their lives, and on current treatment options.

Narcolepsy Network is a national patient support organization founded in 1986

At a certain point, I had to give up control of my life to narcolepsy. It controls how long I can shop, how long I can read, how long I can work on my computer.

Narcolepsy Network, a national nonprofit organization supporting people with narcolepsy, joined dozens of others from the narcolepsy community at the FDA’s public meeting on narcolepsy drug development. The meeting was held on September 24 at FDA headquarters is Silver Spring, MD.

The public meeting was held as part of the FDA’s Patient-Focused Drug Development Initiative (PFDDI). PFDDI is part of the fifth Prescription Drug User Fee Act (PDUFA V), which was passed by congress and signed into law by the President in July 2012.

The FDA has committed to gather patient perspectives in 20 disease areas through 2015. For each disease area, the agency will conduct a public meeting to discuss the disease and its impact on patients’ daily lives, the types of treatment benefits that matter most to patients, and patients’ perspectives on the adequacy of the available therapies.

Patient points of view shared at the public meeting and through a public docket will provide FDA reviewers with helpful insights when conducting benefit-risk assessments for new drugs to treat narcolepsy. Patient responses could also show the FDA that they need new ways to measure the effectiveness of treatments being reviewed.

“This meeting is quite an historic event,” said Narcolepsy Network Executive Director Eveline Honig, MD, MPH. “This is one of the first times that the FDA has sought patient input on living with a disease and the available treatments. We are so fortunate that narcolepsy was one of the diseases chosen for this initiative, and we are hopeful that patient input will have a positive influence on the development of new treatments for narcolepsy.”

Narcolepsy is a sleep disorder that impacts one in approximately 2,000 people in the United States. Despite causing excessive daytime sleepiness, which makes it difficult to stay alert on the job, at school or at home, many people are unaware of they have the condition and remain undiagnosed. The disease may also include cataplexy, which causes muscle weakness and can result in momentary paralysis and collapse. While the cause of narcolepsy is not completely understood, current research points to a combination of genetic and environmental factors that influence the immune system.

Approximately 150 people attended the meeting in person with nearly 700 more attending via an Internet webcast. Narcolepsy Network rallied support for the meeting though communication with its extensive network of members, people with narcolepsy, their families and health care professionals. FDA officials have described the strong response to the narcolepsy meeting as unprecedented.

There were two main areas of discussion at the meeting: “disease symptoms and daily impacts that matter most to patients,” and “patients’ perspectives on current approaches to treating narcolepsy.” For each topic, a panel of five patients made prepared statements on the topic, followed by a facilitated discussion with the live and online audiences.

Narcolepsy Network member Fran Rosen, 62, traveled from Florida to attend and was a panelist for the first topic, symptoms and impacts that matter most to patients. Like many speakers at the meeting, Rosen identified Excessive Daytime Sleepiness (EDS) as the most problematic symptom for her.

“At a certain point, I had to give up control of my life to narcolepsy. It controls how long I can shop, how long I can read, how long I can work on my computer,” Rosen said. “Can I go to the party, will I be able to stay up during the meeting, am I able to drive today?”

Fifteen-year-old Brandon Coonrod said that EDS could cause him “to fall asleep anywhere (he is), like sitting quietly in class or at lunch in the cafeteria.” He and other students said that their behavior and need to sleep is not understood by their peers and makes them more isolated.

Other symptoms that were frequently cited by speakers were cataplexy (the sudden loss of muscle tone brought on by strong emotion), and despite EDS, many people spoke of difficulty sleeping at night. Other patients described the “brain fog” that affects them and told how it creates difficulty in staying focused and completing tasks at school, at work and at home.

Speakers on the second panel, addressing patients’ perspectives on current approaches to treating narcolepsy, described the great benefits of approved medications but also their limitations – and for some the great expense.

The mother of a 9 year old who was diagnosed with narcolepsy at 5 said she is worried that her daughter is already taking three medications, one or more of which have never been tested in children. Without these medications, she said her daughter would not be able to go to school. Even so, when she returns home, she is falling over from extreme sleepiness and every day they have to determine which need is greater – to go to sleep immediately or to eat first.

Many speakers reported having dealt with years, even decades of incorrect diagnoses and being treated with drugs for diseases they didn’t have. Other common themes addressed included general ignorance about narcolepsy, and the difficulties of staying in school, holding a job and keeping up homemaking and family responsibilities.

Narcolepsy Network member Sharon Link said that sleepiness is no excuse because, “employers won’t put up with missed deadlines or tardiness.”

Justin Greene of Leesburg, VA told how narcolepsy had interrupted a promising college education and that he had dropped out because he couldn’t keep up the high grades he had always achieved before he developed narcolepsy symptoms. “I want to be the last of the children to have no choice but to sleep through a bright future,” he said.

Nancy Ackerman of Olney, MD, said she has had to reluctantly accept that, “my life will never be as I want it to be.”

Narcolepsy Network board member Patricia Higgins, BSN, a nurse, recounted how, “as a result of narcolepsy and cataplexy, I was labeled as a drunk, became severely depressed and considered suicide.”

FDA officials were highly attentive throughout the meeting and through question and answers were clearly learning more about the disease. For instance, speakers helped officials understand that napping is a primary effect of narcolepsy and not just a way of coping with it. However, patients also said that napping boxes out the productive and enjoyable times of their lives. Other discussions with FDA officials focused on the pharmaceutical, behavioral, dietary and lifestyle alternatives that patients try and that their doctors recommend in order to cope with limited medication benefits, progressive aspects of the disease, drug side effects or gradual tolerance to the medications.

“All the panelists and speakers did an outstanding job of representing the many facets of narcolepsy and sharing the challenges that people with narcolepsy face,” said Honig. “This has been such a wonderful opportunity for people with narcolepsy to be heard and to influence the future of narcolepsy treatment. We are very, very pleased with the meeting.”

The public docket remains open for comments until November 25, 2013 at!submitComment;D=FDA-2013-N-0815-0001.

Video of the meeting will be posted on the FDA website at

Narcolepsy Network is a national nonprofit organization dedicated to increasing early diagnosis of narcolepsy, advocating for and supporting persons with narcolepsy and their families, and promoting critical research for treatment and a cure.

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