Jun 08 2019
Narcolepsy Finds Its Public Voice

By Monica Gow
Executive Director & Co-founder
Wake Up Narcolepsy

On, Tuesday, September 24, I had the honor of attending the Food And Drug Administration (FDA) public meeting on narcolepsy.  This was the fourth disease and first rare disease to have patient testimony heard by the FDA under the Prescription Drug User Fee Act (PDUFA V) passed in 2012.

The response to this meeting was unprecedented! The large meeting room filled to capacity, with 150 people present, including 10 people with narcolepsy or loved ones giving direct testimony. Another 700 viewed the webcast. These 10 panelists, chosen by the FDA, brought a unique perspective on how narcolepsy has affected their lives. They ranged in age from a 15-year-old boy to a women of retirement age.

As we gathered at the FDA White Oaks Campus, in Silver Spring, MD, nervous anticipation and excitement continued to build. This important and powerful meeting exceeded my every expectation and gave narcolepsy the long-overdue public voice it so abundantly deserves.

Following opening remarks by FDA officials, the panelists from the first of two groups each spoke from the heart for a few minutes about their narcolepsy symptoms and how the disease most significantly affects their daily lives.  One of the five panelists, for example, spoke emotionally of the 19 years of living secretly with hypnagogic hallucinations for fear of losing her child. If she had revealed her hallucinations, which she said made her feel like she was going crazy, she thought her son would be taken from her. Hypnagogic hallucinations, one of the several symptoms of narcolepsy, are vivid, often frightening, dream-like images and sounds experienced at the onset of REM sleep.

After these panelists gave their testimony, the FDA moderator solicited audience and webcast feedback to specific questions about narcolepsy symptoms.  Meeting participants felt the raw emotion continue to build throughout the day as people described lives lost to narcolepsy. Clearly, the complexity of the disease continued to unfold with each person’s gripping testimony.

The second panel discussion addressed patients’ perspectives on current approaches to treating narcolepsy. This testimony exposed the fact that there is not one medication solution for narcolepsy; rather it becomes a trial-and-error process of finding the medication or medications that work best. Four drugs on the market have FDA approval, but panelists reported a high incidence of off-label drug use for narcolepsy. Many sufferers try multiple drugs to combat the various symptoms of narcolepsy.

The narcolepsy community is extremely grateful for the opportunity created by the FDAs meeting. This was a huge step forward toward educating the FDA and the public-at-large about the gravity of narcolepsy and erasing some of the many misconceptions about the disease.

As the creator of Unite Narcolepsy, Wake Up Narcolepsy is grateful for the outpouring of responses and participation in the FDA initiative. There is still time to take our survey and also enter comments in the federal docket. We deeply appreciate the commitment made by all who have participated. If you have not been involved, please make your voice heard by participating in this landmark opportunity for narcolepsy.