The Powerful and Evolving Role of Patient Advocacy Groups in Orphan Drug Development

Life Sciences, Pharmaceutical Regulation, Pharmaceutical,
  • Wednesday, September 14, 2016

A major goal of patient advocacy groups is to address the unmet needs of patients with cancer or rare diseases by providing access to the most effective drugs. In recent years, patient advocacy groups have expanded their influence over the drug development continuum from discovery to approval to market entry. Many groups directly fund, help design, or recruit patients to participate in clinical trials.

This webinar reviews the history of patient advocacy groups in advancing clinical research and examines the evolution of their role in light of recent and pending legislative and regulatory changes in the United States and European Union. Included is a discussion of how patient advocacy groups and industry can join to respond most effectively to these anticipated changes. Presenters are Susan Stein, MPH, a member of the Board of Directors of worldwide patient advocate umbrella organization Global Genes, and Juliet Moritz, MPH, Executive Director of Strategic Drug Development for Rare Diseases at Premier Research.


Juliet M. Moritz, Executive Director, Strategic Development, Rare Diseases, Premier Research

Juliet Moritz has worked in clinical research for more than 25 years, and her extensive background covers the spectrum from single-site studies to large, multinational trials. She joined Premier Research in 2016 to specialize in rare diseases, supporting the strategic development of products that address unmet medical needs associated with rare and orphan afflictions.

Prior to joining Premier Research, Ms. Moritz was Associate Director of Global Project Management at PPD Inc., overseeing infectious and respiratory disease research, and prior to that was Associate Director of Clinical Research at Knopp Biosciences. She held senior positions at Wyeth, Theravance, and PPD and began her career as a clinical research associate.

Ms. Moritz holds a Master of Public Health degree from the Drexel University School of Public Health and a bachelor’s degree in biology from the University of Pennsylvania. She is a member of the National Organization for Rare Disorders and the Drug Information Association.

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Susan Stein, MPH, CEO & President, Connexion Healthcare, and Board Member, Global Genes

CEO/President Susan founded Connexion Healthcare in 1999 based on her commitment to clearly and concisely communicate evidence-based, relevant scientific information to educate healthcare providers, support clinical decision making, and improve patient outcomes. All of her decisions are based on the notion of ultimately furthering patient care while nurturing a culture of scientific discourse among employees.

Susan draws on more than 2 decades of experience to offer invaluable service to her clients. She began her career on the Pharmaceutical side of the healthcare industry at Johnson & Johnson then Pfizer, which allows her to instinctively understand client needs. Susan then began her career on the agency side and worked her way to vice president before forming Connexion Healthcare.

In 2010, Susan completed her Master’s degree in Public Health at Drexel University in Philadelphia, where she gained tremendous insight on the healthcare landscape. She continues to foster her passion for knowledge while serving on the prestigious Dean’s Advisory Council at Drexel. Susan’s varied background allows her to partner with clinical experts and clients in a meaningful and effective manner.

Susan’s ability to lead and inspire has received media attention. Her career has been highlighted in various trade journals, including Philadelphia Business Journal and PharmaVoice. Susan is also a repeat interviewee on Executive Leaders Radio.

As a philanthropist, Susan has been active in charity work throughout her life. Because patient care is at the core of Connexion, she formed Connexion Cares™ as a vehicle to support healthcare research and foster patient advocacy. Through this program, a portion of the proceeds from each Connexion project is donated to charities in the areas of oncology and rare disease.

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Who Should Attend?

VPs, Directors, Managers, Department Heads, Scientists and Researchers working within:

  • Clinical Affairs
  • Clinical R&D
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Operations
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

from pharmaceutical, biotechnology and medical device companies.

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Premier Research

Premier Research is a leading CRO serving highly innovative biotech, Pharmaceutical and medical device companies. The company has a wealth of experience in rare disease and pediatric research having managed about 100 projects in each area in the last five years alone. Its services include clinical research and regulatory outsourcing in the areas of analgesia; neurology; infectious, cardiovascular, and respiratory disease; dermatology; oncology; and medical devices.

Premier Research and operates in 84 countries. It employs 1,000+ clinical professionals dedicated first and foremost to fulfilling each customer’s requirements in a timely, accurate, and cost-effective manner. This includes a strong international network of monitors and project management professionals combined with regulatory, data management, statistical, scientific, and medical experts, and staff at its well-established network of dedicated clinical sites.

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