Panel III: Lived Experience of the Family - The Duchenne Patient-Focused Compass Meeting

18 May 2018 52:53 0
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The Duchenne Patient-Focused Compass Meeting

As part of this year's Advocacy Conference (March 4-6, 2018), PPMD will host the Duchenne Patient-Focused Compass Meeting: Navigating our Pathway Forward. The meeting will be held on Monday, March 5, 2018 at the Mayflower Hotel in Washington, DC from 10 AM - 4 PM. The Duchenne Patient-Focused Compass meeting will be comprised of panels and presentations with Duchenne community members (patients and caregivers) to explore 'living with Duchenne,' clinical trial and therapeutic experiences, and access to services, supports, and approved therapies across the Duchenne spectrum.

Following a discussion with key FDA personnel last fall, our PPMD team has been working on developing a meeting format that utilizes the "FDA PFDD Workshop" framework, while also broadening the day's scope to apply to federal partners and stakeholders beyond FDA. We believe that this will allow us to build upon our community's work with the Duchenne Guidance, Patient Preferences studies, and formalized data collection. Invited members of FDA, NIH, CDC, CMS, and other federal agencies, along with relevant policy stakeholders will be present. Live polling of Duchenne families present and those viewing the meeting online will provide real time data about patient and caregiver perspectives and experiences.

In preparing for this meeting we have invited all of our Duchenne community foundation partners to join the Community Advisory Board (CAB) for this meeting. We currently have 15 Duchenne foundations signed onto the CAB.

CAB members include:

Best Day Ever Foundation
Coalition Duchenne
Foundation for a Future
Foundation to Eradicate Duchenne (FED)
Hope for Gus
Hope for Javier
JB's Keys
Jett Foundation
Little Hercules Foundation
Powers Promise
Ryan's Quest
Team Joseph
Walking Strong
Zach Heger Foundation

PPMD is grateful to all foundations participating in this effort!

In preparation for this important meeting, PPMD published a White Paper intended to reflect our current Duchenne landscape and create context for the March meeting. The White Paper was led by PPMD with the collaboration of the CAB. A meeting report will be published following the March 5 meeting.

We urge ALL members of the Duchenne/Becker community – parents and teens and adults to join us for this historic meeting.

Why this Meeting? Why Now?

Since the passage of PDUFA V (during the reauthorization of and the launch of the Patient Focused Drug Development (PFDD) Initiative at the FDA, the agency has been holding a series of disease specific “Voice of the Patient” meetings with different patient communities. These meetings have followed a set “PFDD workshop formula” in order to elicit an understanding of a given disease area and to collect high-level information on burden of disease and symptom management.

In 2017, PPMD’s Duchenne Drug Development Roundtable (DDDR) partners worked together to evaluate our research and ‘regulatory learnings’ throughout 2015 and 2016. PPMD also convened meetings that explored the role of inflammation in Duchenne and the burden of clinical trial participation.

In addition, as a result of our community’s efforts around the MD-CARE Act Amendments of 2014 and a strong collaboration between the Centers for Disease Control and Prevention (CDC) and the Duchenne community, the Duchenne/Becker Care Considerations have been updated to reflect changing care standards. The DBMD Care Considerations were published last month (January 2018).

And just last week, the FDA published the finalized “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” Guidance for Industry (click here to learn more and access the document). The publication was the result of years of work from multiple stakeholders in this community, including all of you who contributed your voice, your story to the initial draft guidance PPMD submitted in 2014.

We now want to ensure that we bring our patient community’s voice, perspective, and current experience of Duchenne into the center of this conversation. That is the purpose of the March 5 meeting.

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