FDA Listening Session on Narcolepsy and Idiopathic Hypersomnia

On August 8th, 2022, from 2:00 – 3:30 p.m. EDT, a patient-led Listening Session took place online between the Food and Drug Administration (FDA), Project Sleep, and patient advocates in the narcolepsy and idiopathic hypersomnia community. This 90-minute meeting featured seven patient or caregiver advocate speakers, along with a Project Sleep representative and a clinician. Twenty-four FDA offices or divisions were represented in attendance at the Listening Session.

Today, Project Sleep is proud to share a Summary Report highlighting key takeaways from the meeting.

Objectives of the Listening Session

  • Build upon and update FDA regarding the topics covered in the 2013 Patient-Focused Drug Development (PFDD) Meeting on Narcolepsy
  • Focus on the first-hand experience of patients and caregivers managing narcolepsy and idiopathic hypersomnia
  • Convey a sense of urgency and enthusiasm for partnering with FDA to advance new treatment options

Summary of Topics Discussed

  • Impacts of symptoms on daily functioning and quality of life
  • Underrecognized and invisible aspects of living with narcolepsy or idiopathic hypersomnia
  • Challenges with current therapies
  • Patient and caregiver concerns (including symptom severity, quality of life, disruption of daily activities, impacts on employment and parenting, and related considerations that are self-reported and often difficult to track clinically)

Building Upon the FDA’s 2014 Narcolepsy Report

In June 2014, the FDA published an in-depth “The Voice of the Patient” Narcolepsy Report, based on the FDA’s 2013 Patient-Focused Drug Development (PFDD) Narcolepsy Meeting. In hosting a 90-minute Patient Listening Session with the FDA in 2022, Project Sleep aimed to build upon the 2014 Report by highlighting additional underrecognized impacts while also providing new perspectives on current therapeutic options. Read the FDA’s 2014 “The Voice of the Patient” Narcolepsy Report.

Key Takeaways

We encourage you to read the full report. Some key takeaways included:

  • There is a spectrum of symptoms across NT1, NT2, and IH, and people’s experiences vary widely.
  • Sleepiness is serious and has significant impacts on daily functioning and real-life activities.
  • Brain fog is an underappreciated and deeply challenging part of many people’s experiences with narcolepsy and IH.
  • Wakefulness is not one state. Many people with narcolepsy or IH experience automatic behavior, i.e. continuation of routine activities with little conscious awareness and often no memory of them.
  • Nighttime symptoms such as terrifying hallucinations around sleep, sleep paralysis, and interrupted sleep blur the lines between sleeping and waking, and are underappreciated aspects of patient experiences.
  • People must manage narcolepsy and IH symptoms 24 hours a day. Many plan their lives around symptoms, medications, side effects, napping, etc.
  • There is no “one size fits all” approach to treatment or management of symptoms. Finding a medication regimen that adequately manages symptoms can be challenging. Treating narcolepsy over a lifetime requires adjustments and many options. No treatment will work for every person at every stage of life.
  • Current clinical measures (e.g., the Epworth Sleepiness Scale) are inadequate to assess meaningful impacts of treatment on patients’ lives. Validated patient-reported outcomes measures are needed along with additional diagnostic tools.
  • The patient community is eager to be consulted and engaged in shaping future development of outcome measures and clinical trial designs.

Special thanks to the FDA’s Office of Patient Affairs, the patient advocates and clinician speaker, and our staff for making important contributions to this effort. Learn more about Patient Engagement at the FDA

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