Mitochondrial Disease Externally-led Patient-Focused Drug Development Meeting

March 29, 2019

COLLEGE PARK MARRIOTT HOTEL & CONFERENCE CENTER – HYATTSVILLE, MD

How to Participate

Watch the Live Stream!

Join us for this groundbreaking meeting to share YOUR experiences living with mitochondrial disease with the Food and Drug Administration (FDA) and other drug development stakeholders. 

Through this EL-PFDD, YOU can help educate the FDA 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

Support for the
Mitochondrial Disease

Externally-led
Patient-Focused Drug Development
meeting 
is generously provided
in part by:

 

EDITH L. TREES CHARITABLE TRUST

 

Count down to the EL-PFDD – Get Ready to Participate!

Whether you are attending in person or by live stream, let’s get ready for the meeting. 

Try to complete the following prior to March 29:

1. Print out AgendaDownload PDF (Program books with agenda will be provided for attendees)

2. Get set up for Poll Everywhere: 

On a MOBILE DEVICE or SMART PHONE
Download the Poll Everywhere App (on Google Play or Apple App Store) to your device (smart phone) prior to the meeting and create your account. When the event is taking place you will need to enter EnergyInAction. For those wishing to text responses without downloading the app, you will text EnergyInAction to 22333 once to join. We encourage everyone to use the mobile apps rather than texting.

If using TABLET, iPad or COMPUTER
Visit PollEv.com/EnergyInAction prior to the meeting, login to create an account. (If you create one on your smartphone, you can use the same email and password to join by computer.)

NOTE: To create an account — click on the 3 bars in upper right hand corner and select login or signup.  You’ll add your email and then create a password.  That’s it!  Once created, you can use this same login on your mobile device, Tablet, iPad or Computer. Go ahead and enter EnergyInAction where it says “Join a Presentation.” Now just wait til the polling begins on March 29th.
 

3. Print out the Polling Questions

Note: It will be much easier to participate during the live polling on Friday if you have a printed copy of the polling questions in front of you ahead of time.

RENEO PHARMACEUTICALS

PhRMA

EVERYLIFE FOUNDATION
FOR RARE DISEASES

NOTE:  After March 29th, a post meeting survey link will be posted at www.umdf.org/pfddmeeting2019 and sent to meeting “patient/caregiver” participants.  For a broader patient representation in our Voice of the Patient Report, we ask that every patient or caregiver complete the survey (one survey submission per patient).  The survey will be very similar to the polling questions but with more response options to properly represent our community.  Whether you attended in person, remotely or not at all, this survey is open to everyone!

Past Webinars

ALL PATIENTS, FAMILIES, AND CAREGIVERS ARE INVITED TO PARTICIPATE!

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.

What is an Externally-led Patient-Focused Drug Development Meeting?

The patient perspective is critical in helping FDA understand the context in which regulatory decisions are made for new drugs. Externally-led PFDD meetings give FDA an important opportunity to hear directly from patients, patient advocates, and caretakers about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during review of a marketing application.  UMDF, in collaboration with the Muscular Dystrophy Association and MitoAction, is excited to host this Externally-led Patient-Focused Drug Development meeting for the mitochondrial disease community on March 29, 2019! 

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

This Externally-led Patient Focused Drug Development meeting will use 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 invited members of the community that reflect the diversity of mitochondrial disease experiences to be a panelist or a speaker.  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.  Select representatives of associated drug development stakeholders (researchers, clinicians, industry) will be invited to attend as well or may join the streamed presentation.   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 Externally Led Patient Focused Drug Development meeting important for OUR community?

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 to 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. This input can inform FDA’s decisions and oversight both during drug development and during review of a marketing application. We need YOUR participation to make sure our collective community voice makes a positive impact.