Lighting the Way to a Cure

  • Financials
  • Patient Library
  • Blog
  • Calendar
en English
en Englishes Spanishfr Frenchde Germanit Italiannl Dutchru Russianzh-CN Chinese (Simplified)ja Japanesehi Hindiiw Hebrewpt Portuguese
FSHD Header Logo
mobile-logo
  • Who We Are
        • About Us
          • History
          • Mission
          • Our Team
        • Our Impact
          • Therapeutic Accelerator
          • Education
          • Advocacy
          • Our Partners
        • Extraordinary Measures
          • 2021 Donor Impact Report
          • 2020 Donor Impact Report
  • Understanding FSHD
        • What is FSHD
          • Symptoms
          • Diagnosis
          • Early-onset FSHD
          • Genetic Testing
        • Living with FSHD
          • Newly Diagnosed?
          • Finding Care
          • Symptom Management
          • Patient Library
        • FSHD University
          • Physical Health
          • Wellness
          • Research
  • Get Involved
        • Support Us
          • Ways to Give
          • Become a Torch Bearer
          • Raise Awareness
          • Volunteer
          • Fundraise
        • Advance Research
          • Patient Involvement
          • Join the True Cost of FSHD Study
          • Clinical Trials
          • Donate tissue
        • Connect
          • Find Your Local Chapter
          • Join the Community
          • World FSHD Alliance
          • Online Communities
        • Events
          • Walk & Roll to Cure FSHD
          • Events Calendar
          • FSHD 360 Regional Conferences
          • FSHD Connect Conference
  • Research
        • For Patients & Families
          • Advance Research
          • True Cost of FSHD Study
          • TestFSHD genetic testing
          • Clinical Trials
        • For Researchers
          • Apply for a Grant
          • Grants We Have Funded
          • Resources
          • Int’l Research Congress
        • For Clinicians
          • FSHD Masterclass
          • ICD-10
        • For Industry
          • Therapeutic Accelerator
          • Clinical Trial Research Network
          • Drug Development Pipeline
          • Voice of the Patient Report
  • News
    • Media Library
    • Blog
      • Living with FSHD
        • Health and Medicine
        • PT and Exercise
        • Tips and Tricks
        • Mental Health
      • FSHD Society News
      • FSHD Research
      • Fundraising & Events
      • Media & PR
    • FSHD Society Radio
    • The FSHD Advocate
  • JOIN US
  • DONATE

Patient-Focused Drug Development Meeting for FSHD

Home / FSHD Research / Patient-Focused Drug Development Meeting for FSHD

Posted on: Oct 23, 2019

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 “voice of the patient” 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 externally led Patient-Focused Drug Development 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.

Share

Filed Under: FSHD Research, FSHD Society News, Media & PR

Leave a Reply Cancel reply

Your email address will not be published. Required fields are marked *

SEARCH

Categories

  • FSHD Research
  • FSHD Society News
    • Extraordinary Measures
  • Fundraising & Events
  • General
  • Living with FSHD
    • Health and Medicine
    • Mental Health
    • PT and Exercise
    • Tips and Tricks
  • Media & PR

Sign up for Email Alerts

Stay up to date with the latest FSHD news and updates from FSHD Society.

CONNECT WITH US

Proud Member World FSHD Alliance (1)
Footer-Logo-min

Donation Address:

Department 960, P O Box 4106
Woburn, MA 01888    U.S.A.

General Mailing Address:

75 North Main Street, Suite 1073
Randolph, MA 02368 U.S.A.

(781) 301-6060

  • DONATE
  • Donate
  • Join Us
  • Inclusivity & Equity
  • Privacy Policy
  • Contact Us
  • Glossary Of Scientific Terms
Digital Marketing by