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Unprecedented Collection of Duchenne Experts Developing Guidance for FDA

The process for writing a Draft Guidance on Duchenne for the FDA and industry is in full swing.

As you know, the purpose of this guidance is to assist sponsors in the clinical development of medical products (i.e., human drugs and therapeutic biological products) for the treatment of Duchenne over the entire spectrum of the disease (ambulatory and non ambulatory). It is the result of the first collaboration between the FDA and a rare disease community to produce clinical guidance in their respective disease area.

Where We Are in Developing This Critical Document

The Steering Committee and Working Groups have each met three times (or more) to discuss the outline and begin to produce guidance content. The Community Advisory Board has held two virtual meetings; members have submitted comments and questions on all sections of the guidance.  

Next week, the Draft Guidance will be posted online and made available for full public comment by you, the community at large. Stay tuned for your chance to voice your comments on the guidance. 

Over 80 Amazing Expert Stakeholders Working Together with One Goal –
End Duchenne

We have an unprecedented collaboration of experts from all facets of the Duchenne community currently working on this project. 

Steering Committee

  • Neera Gulati, MD Patient Representative
  • John Bridges - John's Hopkins University
  • Kevin Flanigan, MD - Nationwide Children's Hospital
  • Craig McDonald, MD – UC-Davis
  • Justin Fallon, PhD - Brown University
  • Lee Sweeney, PhD University of Pennsylvania
  • Lawrence Charnas, MD, PhD – Shire
  • Pat Furlong - Parent Project Muscular Dystrophy
  • Tim Franson, MD – FaegreBD Consulting

 

  • Professional writer- Theo Smart
  • Project Management- Mark Krueger and Associates

Working Groups

Working Group 1 - Benefit Risk

Co- chair: John Bridges - John's Hopkins University
 

Members:

  • Patrick Denger - Parent/Patient advocate
  • Susan DosReis - Department of Pharmacy, UMD
  • Reed Johnson -  RTI
  • Peter Pitts - Center for Medicine in the Public Interest
  • Erin Barnett - Parent/Patient advocate
  • Ellen Wagner - Parent/Patient advocate
  • Richard Hermann, MD, MPH AstraZeneca
  • Bennett Levitan - Johnson & Johnson
  • Marilyn Metcalf, PhD - GSK
  • Rebecca Noel, DrPH,MPH - Eli Lilly and Company


Working Group 2 - Diagnosis

Chair:  Kevin Flanigan, MD - Nationwide Children's Hospital


Members:

  • Annemike Aartsma-Rus - Department of Human Genetics, UMC
  • Madhuri Hegde, PhD, - Emory University
  • Ann Martin, MS, CGC - DuchenneConnect
  • Kathy Matthews, MD - University of Iowa
  • Vanessa Miller, MS, CGC - PatientCrossroads
  • Todd Morrow - Parent/Patient advocate
  • Stan Nelson, MD, PhD - UCLA, Parent/Patient advocate
  • Bob Weiss, PhD - University of Utah, Department of Human Genetics


Working Group 3 – Natural History

Chair:  Craig McDonald, MD - UCDavis

 

Members

  • Doug Biggar, MD - Holland Bloorview Kids Rehabilitation
  • Katie Bushby, MD - Newcastle University 
  • Avital Cnaan, PhD - George Washington University
  • David Cox, PhD - Eli Lilly
  • Kevin Flanigan, MD - Nationwide Children's Hospital
  • Nathalie Goemans - Universitair Ziekenhuis Leuven
  • Mohamed Haider - Patient
  • Eugenio Mercuri - The Agostino Gemelli Hospital, Italy
  • Marissa Penrod - Parent/Patient advocate
  • Allen Reha, MS - PTC Therapeutics

 

Working Group 4 Biomarkers 1 (dystrophin/utrophin)

Chair: Justin Fallon, PhD - Brown University

 

Members

  • Annemieke Aartsma-Rus - Department of Human Genetics of the LUMC
  • Mindy Cameron - Parent/Patient advocate
  • James Ervasti, PhD - University of Minnesota
  • Justin Fallon, PhD - Brown University
  • Kevin Flanigan, MD - Nationwide Children's Hospital
  • Sharon Hesterlee, PhD - Parent Project Muscular Dystrophy
  • Eric Hoffman, PhD - Children's National Medical Center
  • Afrodite Lourbakos, PhD - Prosensa Therapeutics
  • Steve Moore, PhD - Wellstone Center at the University of Iowa
  • Peter Sazani, PhD - Sarepta Therapeutics

 

Working Group 5  - Biomarkers 2 (MRI/blood/urine)

Chair:  Lee Sweeney, PhD University of Pennsylvania

 

Members

  • Terri Ellsworth - Parent/Patient advocate
  • Larry Gold, PhD - SomaLogic
  • Glen Nuckolls, PhD - National Institutes of Health (NIH)
  • Stu Peltz, PhD - PTC Therapeutics
  • Bill Rooney, PhD - Oregon Health & Science University
  • Krista Vandenborne, PhD - University of Florida
  • Glenn Walter, PhD - University of Florid

  

Working Group 6 - Clinical Trial Design & Outcome Measure

Chair: Lawrence Charnas, MD, PhD Shire

 

Members:

  • Giles Campion, MD - Prosensa
  • Catherine Collins - Parent/Patient advocate
  • Linda Cripe, MD - Nationwide Children's Hospital
  • Nathalie Goemans, MD., PhD Kinderneurologic-NMRC
  • Ed Kaye, MD - Sarepta Therapeutics
  • Craig McDonald, MD - UC Davis Health System
  • Eugenio Mercuri, MD The Agostino Gemelli, Italy
  • Elizabeth Vroom, MD - UPPMD

 

Working Group 7 – Imperatives

Chair:  Pat Furlong - Parent Project Muscular Dystrophy

 

Members:

  • Margaret Anderson - Faster Cures
  • Steve Dreher - Parent/Patient advocate
  • Tim Franson, MD - FaegreBD Consulting
  • Stephen Groft - National Institutes of Health (NIH)
  • Marlene Haffner, MPH - Haffner Associates, LLC
  • Annie Kennedy - Muscular Dystrophy Association (MDA)
  • Jeff Watkins - Parent/Patient advocate
  • Julia Jenkins- Everylife Foundation    

Community Advisory Board

Managed by: Ryan Fischer (PPMD)

Foundation Representatives

  • Steve Dreher - Hope for Gus
  • Neera Gulati, MD -Suneel's Light
  • Cath Jayasuriya – Coalition Duchenne
  • Alex Johnson- Joining Jack
  • Jenn Mcnary – The Jett Foundation 
  • Christine McSherry – The Jett Foundation
  • Robert Meadowcroft - Muscular Dystrophy Campaign
  • Debra Miller - CureDuchenne
  • Marissa Penrod - Team Joseph
  • Jen Portnoy - Hope for Javier
  • James Raffone- Jar of Hope
  • Diana Ribeiro - Action Duchenne
  • Tracy Seckler - Charley's Fund
  • Alex Smith - Harrison's Fund
  • Elizabeth Vroom - UPPMD
  • Allison Willis-  Two Smiles One Hope Foundation

 

Patient and Parent Reps

  • Elliot Barnett
  • Lynnette Bartels 
  • Karen Burch
  • Sarah Burgess
  • Mindy Cameron
  • Catherine Collins 
  • Brian Denger
  • Debbie Dupree
  • Terri Ellsworth
  • Anessa Fehsenfeld
  • Julie Garcia
  • Mohamed Haider  
  • Joanna Johnson
  • Grace Lightcap
  • Elizabeth Longmire
  • Jessica May
  • Todd Morrow
  • Stan Nelson, MD, PhD
  • Christine Piacentino
  • Elliot Barnett
  • Regina Reidenberg
  • Aparna Surampudi
  • Tayjus Surampudi
  • Ellen Wagner
  • Jeff Watkins 

We look forward to sharing this guidance with the entire community next week, and hope to hear your comments on the document! 

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