By Julie Flygare, J.D.
As a narcolepsy advocate, the FDA Patient Focused Drug Development Initiative (PFDDI) caught my attention right away. It’s not everyday that patients and high-level decision makers get together to discuss what matters most for patients. Here are the top five reasons why the FDA initiative matters.
The FDA initiative allows people with narcolepsy and their loved ones to actively shape the future of narcolepsy treatment. As a direct result of the narcolepsy meeting, FDA will produce a meeting report, reflecting the input obtained from the narcolepsy community. The report will be posted on FDA’s website and circulated to the agency’s key decision makers. This information may also inspire the creation of new patient-focused outcome measures qualified for use in drug development, to better capture patient experience more systematically in clinical trials.
2. Re-gaining control:
As a person with narcolepsy, you may feel out of control at times – with your symptoms holding the reigns and making demands on your time and energy. While participating in the FDA initiative will
not cure narcolepsy tomorrow, it will help you reclaim a small piece of control over by empowering yourself as an advocate and improving the future for all those with narcolepsy.
3. Collecting similarities and differences:
While we share many commonalities, no two cases of narcolepsy are exactly the same. The severity of symptoms, effectiveness of treatment options and tolerability of side effects varies greatly from person to person.
This is why it’s crucial for every individual with narcolepsy to get involved. FDA aims to better understand our collective experience – which includes my story, your story, his story and her story. We must each share our challenges, successes, hopes, and fears to ensure that FDA gains an accurate and full picture of our community.
4. Being Heard
At times, it may feel that no one is listening. When we mention narcolepsy, listeners sometimes look away or change the subject. In working with the FDA on this initiative for the past year, I am pleased to report that FDA’s representatives are listening. They are eager to learn about our symptoms, effects on our daily lives, experiences with current treatments and our priorities and concerns for future treatments. This validation will prove a unique, unforgettable experience.
5. Claiming the National Spotlight:
Narcolepsy is grossly misunderstood, underdiagnosed and rarely the main topic of discussion of conversation in Washington, DC. However, as one of the 20 conditions chosen for FDA’s initiative, and the first “rare” condition to be addressed, narcolepsy is now in the national spotlight, and boy, does it feel good!
Health policy experts, government leaders, sleep researchers, clinicians and health non-profit organizations will be watching to see how we respond to this opportunity. Strong participation will prove that narcolepsy advocates are organized and ready to collaborate with various stakeholders towards building a brighter future for narcolepsy.
Julie Flygare, JD, is a narcolepsy spokesperson, blogger, runner and the author of “Wide Awake and Dreaming: A Memoir of Narcolepsy.” She was diagnosed with narcolepsy with cataplexy in 2007, at age 24. After graduating from Boston College Law School in 2009, she set out to open people’s hearts to narcolepsy. Her story has been featured Marie Claire, ABC, NBC, Psychology Today and Discovery Channel. She is the author of the “REM Runner Blog” and recently, launched the NARCOLEPSY: NOT ALONE international awareness campaign.