Mitochondrial Disease Patient-Focused Drug Development Meeting 2018-06-15T09:08:34+00:00





Mitochondrial Disease Patient-Focused Drug Development Meeting

March 29, 2019


Join us for this groundbreaking meeting to share YOUR experiences living with mitochondrial disease with the Food and Drug Administration (FDA).   The FDA is responsible for ushering through the development of new therapies, and they want YOU to help them with those decisions by educating them about:

  • What it is like to live with a mitochondrial disease
  • Your concerns as the disease progresses
  • How you currently manage your mitochondrial disease
  • What meaningful treatments look like

This is an opportunity to have YOUR VOICES heard.  The FDA approves all treatments for mitochondrial diseases and needs to know what is important for you.  This knowledge will inform their decision-making, and lead to better treatments and faster approvals for mitochondrial diseases!


Individuals living with mitochondrial disease and their caregivers and family members are invited to participate.  Everyone who attends the meeting will have a chance to share his or her experiences and perspectives.  If you cannot attend in person, you can stream the meeting online where you can participate in live polling throughout the day.  Your input, both in-person or online, will be heard by FDA officials attending the meeting and will also be summarized in a report to the FDA.   We will also send you surveys and other communications so that we can help you contribute your thoughts.   Sign up now to receive these monthly e-communications!

What is a Patient-Focused Drug Development Meeting?

A few years ago, the FDA began a new initiative to expand the way it looks at therapies, recognizing that people living with a condition are uniquely positioned to inform the agency about a specific disease.  This is important because the FDA wanted to hear directly from as many people as possible in a given patient community about their experiences and needs.    To help them do this in a systematic way, the FDA launched the Patient-Focused Drug Development (PFDD) Initiative where they hosted a series of public meetings to hear directly from patients and their caregivers.  Under the PFDD initiative, FDA conducted 25 PFDD meetings that have been extremely successful.   The FDA has now moved to a new model where PFDD meetings are led by patient groups, with the FDA attending and participating.  In 2018 UMDF submitted a request for a PFDD meeting on behalf of the entire mitochondrial disease community.  We have the green light from FDA and are excited to host this meeting on March 29, 2019! 

How is this meeting designed for YOU to be heard by FDA?

This PFDD meeting uses multiple methods for soliciting YOUR thoughts and experiences about living with mitochondrial disease.  EVERYONE who attends will have a chance to be part of the conversation.  To allow us to hear as many different perspectives as possible, we have four main areas of focus for our panels – Burdens of the Disease for Pediatric Mitochondrial Disease Patients with Neurologic Manifestations and Adult Patients with Mitochondrial Myopathies and Management of Care for Pediatric Mitochondrial Disease Patients with Neurologic Manifestations and Adult Patients with Mitochondrial Myopathies.  Following opening remarks by FDA officials and others, each session will include panel discussions made up of patients and family members.   Mitochondrial disease patient advocacy groups will invite members of the community to be a panelist or a speaker.  Panelists and speakers will be selected that reflect the diversity of mitochondrial disease experiences.  After the panels, everyone in the audience in-person or online will be able to answer live polling questions and we will be able to see the results in real time.  In addition, all of YOU that can attend in-person will be invited to be part of a live discussion in the room where you can share your own experiences.  The meeting will be ‘live streamed’ online, too, for those who cannot attend in person.   After the meeting, all of this input, along with information collected prior to and after the meeting, will be summarized as a “Voice of the Patient” report, which our community will be submitting to FDA.

Why is this PFDD meeting important for OUR community?

The FDA is the government agency that regulates all new therapies (drugs and medical devices) as they move through development to commercially available medications. FDA scientists decide if a potential new drug is safe enough to be tested in humans, if it can continue development after early trials, and ultimately if there is enough evidence that it is both safe and effective so it can be approved for use in the United States.  For the mitochondrial disease community, this meeting is the first opportunity for a group of patients, families and other caregivers to speak directly to the FDA, and share their experiences in their own words. It is also the first time a report on the patient experience will be completed and shared with the FDA.  We need YOUR participation to make sure our collective community voice makes a positive impact.