Externally-Led Patient-Focused

Meeting was held October 21st, 2022

Patient-focused drug development (PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. ... As experts in what it is like to live with their condition, patients are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation.”

Scientific Response meeting

November 18th from 11 am – 1 pm ET

“Integration of the patient perspective
into therapy development for MLD”

When was the MLD PFDD Meeting?

The live MLD community meeting with biopharma and the FDA was Friday, October 21st, 2022. Over 150 were registered to view the livestream.  Dozens of MLD families shared live and written comments representing all forms of MLD, all of the current therapies including no therapy, and from countries around the world.

You can still engage and participate

Submit a post-meeting Response.  All comments  will be come part of the final Voice of the MLD Patient report, a formal record of the MLD PFDD meeting.  Comments will be accepted for 30 days after the meeting.

Scroll past the videos to learn more about PFDD meetings

Watch the MLD EL-PFDD meeting below.  Download the transcript here.

Use the Chapter Selector in the lower right corner of the above video window to jump to specific points in the video.

Watch the MLD Research/Scientific Clinical response meeting meeting below. Download the transcript here.

Use the Chapter Selector in the lower right corner of the above video window to jump to specific points in the video.

What is an Externally-Led Patient-Focused Drug Development meeting?

Externally-led Patient Focused Drug (EL-PFDD) meetings give the Food and Drug Administration (FDA) and other key stakeholders — including researchers, biopharma companies, health care providers, and federal regulatory partners — an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, patients’ experiences with currently available treatments, and patient’s priorities for therapeutic outcomes. These inputs will also inform FDA’s decisions and oversight both during drug development and their reviews of a marketing applications for new therapies. Learn more from the FDA.

The FDA wants to hear YOUR experiences and desires

Sharing your experiences and desires is critical.  Your inputs will be come part of the Voice of the MLD Patient report prepared after the meeting and will become a formal submission to the FDA as well as guidance for ongoing and future research.

Your submissions should be personal and focused on these primary areas:

  • What symptoms of MLD are the most bothersome?
  • How does MLD impact activities of daily life?
  • What have you done to treat or manage MLD? Has it helped?
  • What are your hopes and desire for new therapies? i.e. what symptoms do you want to see addressed?
  • How much risk would you accept for a given therapeutic improvement?

We want and need to hear from families with all forms of MLD, with both living and deceased loved ones, both with and without therapy (transplant, gene therapy, ERT, etc.), and from all countries.

Learn More

Visit the Participate tab to view an informational video, see the day’s agenda, and more.

What happens during the meeting?

The meeting and participants, including the FDA, will be virtual, however, the hosts will be looking for the MLD community to participate in polls, by sending in comments, or calling in live! There will be a brief 10-15 minute overview of MLD, followed by two sessions with panelists, callers, reading of submitted comments, and live anonymous polling.  

Please plan on viewing and participating in the entire meeting, but if you can only attend part of the meeting, that would also be greatly appreciated.  The meeting recording will immediately replace the live feed after the meeting ends. 


What happens after the meeting?

The comments and information shared before, during, and after the meeting will be consolidated into a Voice of the MLD Patient report. The report will be provided to the FDA and to researchers developing therapies, and well as being made available to the public on this website.


FAQ - Frequently Asked Questions
  •  Is this meeting free?  Absolutely – we want all MLD community members to attend and participate.d and participate
  • Can families outside of the USA attend and participate?  Yes – the meeting is open to all MLD families.  
  • What about families with deceased loved ones? We want and need to hear and learn from you as well.  
  • Do I need a computer to attend?  No, you can attend and participate from your mobile device or tablet – if you can read this web page (and you are right now), you will see the meeting here.
  • What will be the primary meeting language? It will be English, however, if your primary language is not English, please consider submitting a written Response in advance, and we will attempt to translate it to add to our knowledge base.
  • Will the meeting be recorded? Yes – it will be available immediately after the meeting ends on this page (hosted by YouTube so everyone can easily access it). 

More FAQ questions will be coming.  What else do you want to know?  Click here to submit your question.

Who is organizing the EL-PFDD?

The MLD EL-PFDD meeting is being organized and hosted to benefit all MLD families by a collaboration of patient advocacy groups and researchers.

Our supporters

Our supporters are helping us to invite families from around the globe to participate

Our sponsors

These generous sponsors have made this meeting possible … thank you!

Please share this page with other MLD families