Patient Advocacy Summit 2014: The Must-Have Collaborations for Successful Drug Development

August 29, 2014

Ready to hone your skills of collaboration and develop a better understanding of the drug development process? At this year’s Patient Advocacy Summit moderators of this session hope to develop a white paper from the group’s participation. As they lead you through the step-by-step process, which involves participation of the biopharmaceutical industry, academia, government and patient groups, you’ll learn ways to help get drugs to market as quickly as possible.

This hands-on workshop will bring representatives from each side of the equation to discuss what it is that they’re group brings to the table and what makes them a successful part of the process.

After the presentation groups will have twenty minutes to develop a “quick pitch” in a role-playing exercise that allows participants to take on the role of one of these stakeholders.

Barbara H. Wuebbels, RN, MS

Screen Shot 2014-08-28 at 10.28.19 AMVice President of Patient Advocacy and Medical Affairs, Audentes Therapeutics

Barbara has been involved in patient advocacy activities for patients with rare diseases for the past 15 years at Ucyclyd Pharma and BioMarin. Recently, she joined Audentes Therapeutics as Vice President of Patient Advocacy and Medical Affairs. She has been actively involved with the National Organization of Rare Diseases, International Conference of Rare Disorders, and Global Genes. Currently she serves on the Boards of the International Conference on Rare Disorders and the Batten’s Disease Support and Research Association. She holds Bachelor’s and Master’s degrees in nursing. Twitter: @AudentesTx

Wendy S. White

Screen Shot 2014-08-28 at 10.29.29 AMFounder and CEO, Siren Interactive

Wendy founded Siren Interactive in 1999. For more than 14 years across more than 30 disease states, Siren Interactive has had one focus: finding rare disease patients and connecting them to appropriate therapies. Wendy approaches the business from her unique perspective of being an entrepreneur and digital marketing expert, as well as being the mother of a daughter with a rare disorder that was diagnosed as a direct result of Wendy becoming an empowered caregiver. Wendy’s personal journey with her daughter, which began in 2001, was the impetus for Siren to focus solely on marketing rare disorder therapies. In 2011, Wendy led the collaboration with 12 fellow rare disease caregivers to co-author Uncommon Challenges; Shared Journeys: Stories of Love, Hope, and Community by Rare Disease Caregivers. In 2012 she was recognized as one of the 100 most inspiring people in pharma. Twitter: @sirenwendy

Stephen C. Groft, PharmD

Screen Shot 2014-08-28 at 10.30.15 AMSenior Advisor to the Director, National National Center for Advancing Translational Sciences at the National Institutes of Health (NIH)

His current major emphasis at NCATS/NIH is on patient and community engagement in rare diseases research and medical devices as orphan products. Steve served as Director of the Office of Rare Diseases Research (ORDR) at the NIH prior to his retirement in 2014 after 44 years in government service. His major focus for the previous 32 years was on stimulating research with rare diseases and developing information about rare diseases and conditions for researchers, biopharmaceutical industry, healthcare providers, and the public. Twitter: @NIH

Matt Wilsey

President, Grace Wilsey Foundation

Matt Wilsey is a Silicon Valley entrepreneur, investor, and advisor. Beyond consumer products and services, Matt advocates for and invests in biomedical research, drug development, and genetic sequencing technologies. After starting his career at the White House and the Department of Defense, Matt spent numerous years as a frontline operator at Zazzle, KKR, Howcast Media, and CardSpring before becoming a “rare disease hunter” and moving to the investment side. Matt holds a BA from Stanford University and a MBA from Stanford’s Graduate School of Business. Twitter: @gracewilseyorg

Hudson Freeze, PhD

Professor and Director, Human Genetics Program at Sanford Children’s Health Research Center, Sanford-Burnham Medical Research Institute

Dr. Freeze has worked on rare diseases for over 35 years, specializing in the discovery of Congenital Disorders of Glycosylation (CDG) and their treatments. He is  Professor of Glycobiology and the Director of the Human Genetics Program as Sanford-Burnham Medical Research Institute. His current interests are in defining new types of CDG and in  NGLY1, the first Congenital Disorder of Deglycosylation. He is the Vice President for Science Policy at FASEB, a 120,000 member alliance of biomedical researchers.

Session Participants:

  • Dr. Steve Groft, former Director, NIH Office of Rare Diseases Research
  • Hudson Freeze PhD, Professor and Director, Human Genetics Program

Sanford Children’s Health Research Center, Sanford-Burnham Medical Research Institute

  • Matt Wilsey, President, Grace Wilsey Foundation
  • Jonathan Jacoby, Hide & Seek Foundation, SOAR-NPC
  • Katherine Rauen, MD, PhD, University of California Davis MIND Institute


Can’t attend in person? You can’t afford to miss this! Our new Livestream component will allow up to 5,000 interested advocates from around the globe to attend virtually. The event will be broadcast live with opportunities for patients to participate from afar using social media, including twitter and Facebook. Follow using #2014GGSummit

To view the full schedule of events click here.

To register to attend the summit in person, click here.

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