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FSHD Society to Hold Landmark “Voice of the Patient” Meeting

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Input from patients can inform the FDA’s decisions and oversight both during drug development and during its review of a marketing application.


LEXINGTON, Mass. (PRWEB)

The FSHD Society announced today that the U.S. Food and Drug Administration (FDA) has approved its application to hold an externally led Patient-Focused Drug Development (EL-PFDD) meeting on facioscapulohumeral muscular dystrophy. This meeting, a key component of the Society’s Therapeutic Accelerator initiative, has been scheduled for April 21, 2020, in College Park, Maryland. The Muscular Dystrophy Association and Friends of FSH Research will be supporting and participating as well.

The PFDD meetings, started in 2012 by the FDA, are used to obtain patient perspectives on specific diseases and their treatments. The demand from disease advocacy groups to have the meetings quickly exceeded the agency’s capacity, and so the FDA set up a mechanism for groups to apply to organize “externally led” PFDD meetings.

According to the FDA, “PFDD meetings give FDA and other key stakeholders, including medical product developers, health care providers, federal 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, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during our review of a marketing application.”

The FSHD Society’s EL-PFDD meeting will provide individuals with FSHD, caregivers, and other stakeholders an opportunity to share their experiences and preferences so that the FDA and the biopharmaceutical industry can:


  • understand the FSHD patient journey, and recognize patient preferences and risk tolerance so these may be translated into improved clinical trial designs, selection criteria, and development of outcome measures relevant to FSHD drug development;

  • demonstrate the complexity and heterogeneity of FSHD, with the end goal of development programs and trial designs that will reflect these aspects of the disease;

  • create a practical, scientifically rigorous framework that incorporates patient preferences and patient-reported outcomes into FSHD clinical research to ensure that trials are measuring not only statistical success, but also demonstrating meaningful benefit to individuals with FSHD;

  • better understand patient and caregiver perceptions about the treatments they are currently using, which treatments are most beneficial, and which ones may have side effects;

  • encourage identification and use of approaches and best practices to facilitate patient enrollment and minimize the burden of patient participation in clinical trials; and

  • ensure that people with FSHD understand the value of their participation in clinical trials, and how their input impacts FDA and biopharmaceutical industry decision-making and outcomes at all levels to both improve their own health-related quality of life as well as that of others with FSHD, now and in the future.

Prior to the meeting, the FSHD Society plans to distribute online surveys to some 3,500 patients and family members in its contact database, as well as more than 5,000 members of various FSHD-focused Facebook groups. In addition, the Society will collect patient comments over its social media channels and conduct in-person focus group discussions through its national chapter program and Family Day conferences.

“Data from these surveys and other methods will be used to shape the content and selection of panelists for the April meeting,” explained June Kinoshita, Chief Strategic Programs Officer of the FSHD Society. “We urge patients and caregivers who want their voices to be included in this process to make sure they are on the Society’s mailing list.”

To be included on the FSHD Society’s mailing list, please sign up here.

About the FSHD Society

The FSHD Society is the world’s largest research-focused patient organization for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy. The Society has catalyzed major advancements and is accelerating the development of treatments and a cure to end the pain, disability, and suffering endured by one million people worldwide who live with FSHD. The FSHD Society has transformed the landscape for FSHD research and is committed to making sure that no one faces this disease alone. The Society offers a community of support, news, and information through its website at https://www.fshdsociety.org.

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