“We are a bunch of deeply motivated people
doing everything in our power to live full lives.”
– Saraiah, Narcolepsy Advocate
Leading up to the FDA Narcolepsy Meeting on Sept. 24, 2013, I traveled to FDA’s campus four times, wrote 15 blog posts, co-led the Unite Narcolepsy initiative, co-presented webinars, wrote FAQs, and coached panelists – yet nothing could have prepared me for this day.
Just before 1:00 p.m., the Great Room at FDA buzzed with excitement and nerves, as 150 narcolepsy patients, caregivers, and stakeholders filled the room to capacity. At 1:00 p.m., the room fell silent as FDA representatives welcomed the narcolepsy community and provided introductory remarks. Another 700 narcolepsy advocates joined us via webcast from around the world. I smiled from ear-to-ear, proud to have helped secure this opportunity for our community.
Narcolepsy is the fourth disease area addressed by FDA’s ground-breaking Patient Focused Drug Development Initiative and the first rare disease to go through the process. The initiative aims to better understand patients’ perspectives on symptoms, severity, and current treatment options – to help FDA make more informed regulatory decisions when assessing the risks and benefits of future therapies.
The first five patient panelists took their seats at the front of the room to kick off the Topic 1 discussion on narcolepsy’s symptoms and daily life impact. Each panelist eloquently described his or her journey with narcolepsy.
One young woman, Kerry visited 20 – 25 specialists before finding an accurate diagnosis. In the process, she was misdiagnosed with epilepsy and put on heavy drugs for a condition that she did not have. Doctors encouraged her to leave school, but she has persisted.
Another panelist, Carrie poignantly stated, “The limitations on my time and energy affect my ability to give to society, to my kids, and to finish projects. There is so much inside that I want to do and create.”
My throat clenched and eyes welled with tears. I did not expect these emotions, but the discussion hit a nerve – reminding me of the frustrations of delayed diagnosis and the serious life-changing nature of narcolepsy. After the panelists’ testimony, the group discussion began including audience and webcast participation.
The FDA representatives did an amazing job facilitating this highly emotional discussion with kindness, respect and professionalism. One fellow patient, Saraiah recalls, “I was deeply impressed with the skill that the FDA staff brought to facilitating that meeting and helping to communicate back to us that we were heard.”
After a short break, Topic 2 panelists provided testimony on current treatment options. Panelists addressed a wide range of medications, alternative treatments, side effects and complications. No two people’s experience was exactly the same, but our collective determination to feel better and make the most of our lives was very apparent.
A lively discussion followed with one patient clearly stating, “A nap is not a treatment, it’s a symptom. It’s uncontrollable.” The audience broke into applause. FDA listened closely and took copious notes.
For future treatments, the aspects of narcolepsy that patients would MOST like addressed are EDS, brain fog, sleep architecture and hypocretin deficiency. The meeting closed with an open-mic portion and summary from FDA.
Afterwards, Unite Narcolepsy hosted a reception to celebrate our community’s accomplishments. It was wonderful to see many familiar faces and make new friends.
Thank you to the panelists, attendees and webcast participants who gave their time and energy to this meeting. We “wowed” the FDA and the rare disease community with our record-setting participation.
Personally, I will never forget this day. Fellow advocate, Saraiah said it best, “The overall message of the meeting was that we are a bunch of deeply motivated people who are doing *everything* in our power to live full lives.”
- View FDA’s Webcast Recording from meeting
- Take the survey (Deadline: Nov. 11, 2013)
- Provide Comments to the FDA Docket (Deadline: Nov. 25, 2013)
- FDA will publish a “The Voices of the Patients” Report a few months after the docket closes on Nov. 25, 2013.