Help the FDA Review Potential DM Therapies

Published on Tue, 04/19/2016

The FDA – Why It Matters

You may think you don’t care about the Food and Drug Administration (FDA). It’s hard to get excited about regulations and the Washington, DC bureaucracy. But the FDA is a critical federal agency when it comes to drug development (click here to watch a short MDF webinar with an overview of the FDA). A major responsibility of the FDA is the review and approval of drugs and medical devices for use in the US. The FDA will have the final say on whether therapies developed to treat myotonic dystrophy (DM) are approved for US patients, and its decisions will significantly influence the availability of DM treatments in other countries around the world.

PFDD: Bringing Patients Into Drug Development

In 2015 the FDA expanded and deepened its approach to therapy review. It began to seek input from patients to help ensure that eventual treatments are responsive to actual patients needs. It launched the Patient-Focused Drug Development (PFDD) Initiative, a series of carefully designed public meetings, to systematically gather input from patients regarding the experience of living with specific diseases, the preferred impact of potential treatments, and the risks patients were willing to accept when taking targeted medications. To date the FDA has held 16 of the meetings identified in the 20 meeting initial series.

We Need Your Voice

Myotonic dystrophy wasn’t included in the original series of disease-focused meetings, so MDF submitted a comprehensive Letter of Inquiry (LOI) in late 2015 to request one. The FDA responded positively, encouraging MDF to sponsor a DM PFDD meeting and providing input on FDA invitees, meeting agenda and format. It also created a new PFDD program component, Externally-Led PFDD, to engage the voices of patients with diseases not included in the initial series. MDF will host the first-ever FDA Externally-Led PFDD meeting in September 2016, and you are invited.

The Myotonic Dystrophy Patient-Focused Drug Development Meeting (DM PFDD), which will occur in conjunction with the 2016 MDF Annual Conference in WA, DC, will be your opportunity to tell the FDA what it is like to live with myotonic dystrophy, how it has impacted you and your family, and what you want from potential therapies. The information we capture at the DM PFDD meeting will be published and submitted to the FDA’s Division of Neurology Products, Office of New Drugs, for inclusion in the framework used to evaluate DM therapies.

The PFDD Meeting – How It Works

PFDD meetings are all designed with input and feedback from the FDA, and follow a proscribed format, which typically includes two patient panels focused on very specific questions the FDA would like answered, and additional live feedback from meeting attendees on those questions and others the FDA may ask during the meeting. The DM PFDD meeting will run from 1 PM to 4:30 PM on Thursday, September 15, 2016. The final meeting agenda is still under development, but is expected to begin with introductions, a short film and a clinical presentation on the disease from a leading DM investigator, followed by the first panel.

Panel #1 will focus on the experience of living with DM from the patient perspective, and will feature several MDF community members providing prepared remarks on the following questions:

  • What 1-3 symptoms of DM have the most significant impact on your life?
  • How do they affect your life on a typical day? On your worst day?
  • Are there specific activities that are important to you that you can no longer do or do as fully because of your condition?
  • How have your symptoms changed over time?

Audience members will then be invited to provide their answers to these questions, and others FDA meeting participants may ask. MDF will also have live polling using handheld devices so all audience members can weigh in on the importance and prevalence of different symptoms and impacts.

This panel will be followed by a second panel of different patients providing prepared remarks on current and future treatments. Panel #2 will focus on the following questions:

  • What current treatments or therapies do you use for symptom management?
  • How well are these therapies or treatments working?
  • What are the downsides, if any, to these treatments or therapies?
  • What do you want from an ideal treatment?

Panel # 2 will be followed by a second round of live audience feedback on the treatment-focused questions and any other questions the FDA may have. The meeting will conclude at 4:30 PM ET.

The DM PFDD Meeting: MDF Conference Attendees Invited to Attend and Speak Up

MDF will provide private bus service to and from the 2016 MDF Annual Conference hotel, the Crystal Gateway Marriott, to the DM PFDD meeting venue free of charge. You must have a 2016 MDF Annual Conference registration to attend the meeting. Buses will leave from the conference hotel at noon on Thursday, September 15, and return to the hotel about 5:30 PM the same day. MDF anticipates welcoming several hundred community members, industry representatives, academic researchers, clinicians and FDA professionals to the meeting.

Building On MDF’s Earlier Regulatory Efforts

The upcoming DM PFDD meeting builds on other regulatory meetings MDF has sponsored since 2014 to ensure that FDA professionals understand myotonic dystrophy, in terms of the genetic mutation, disease symptoms and the complexities of designing clinical trials (for slow-progressing, multi-systemic and variable diseases like DM). It is also critical for us to help the FDA understand what people living with DM think of the disease burden and how they define effective therapies. MDF created a strong foundation for the upcoming DM PFDD meeting with our 2014 science workshop on the status of biomarkers and endpoints (two critical components of clinical trials for therapies), our 2015 all-day meeting on clinical trial design, endpoint identification and biomarker development and validation for DM clinical trials, and the Benefit/Risk Study MDF commissioned from Silicon Valley Research Group in the summer of 2015 at the FDA’s request.

The Most Important Voice? Yours

The B/R Study results, reports from prior meetings and other data available through patient registries and care studies will be included in the appendix of the final DM PFDD publication. However, the most important voices to the FDA regarding therapy assessment will be those of the patients and families attending the DM PFDD meeting this September. We need MDF community participation to make sure the DM community voice makes an impact. If you haven’t already registered for the 2016 MDF Annual Conference, click here to learn more. Questions? Contact MDF at