New for 2018

World Orphan Drug Congress USA 2018

World Orphan Drug Congress USA 2018

World Orphan Drug Congress USA 2018


  • We are moving to the Gaylord National Resort & Convention Center ;a "spectacular waterfront resort, with a stunning 19-story glass atrium and sweeping views of the Potomac River"

  • Stellar conference facilities and rooms

  • All in one floor


Along with the current Payers, KOL, Next Generation Therapies and Personalized Medicine programs

  • Data & Digital Health

  • Genomic Sequencing & Diagnostics​




  • There’s no doubt that gene therapy is a booming field, especially when it comes to developing therapies for rare diseases, which in most cases are genetic disorders. Though only two gene therapy drugs have received approval and marketing authorization to this day, the increasing number of players in this field is a sign that the major hurdles preventing these therapies to be brought to market are finally being overcome.
  • Diagnostics is another key challenge in this industry, especially when it comes to patients receiving accurate diagnosis faster. Not only this would help patients get access to available therapies faster, but emerging diagnostics solutions can help pharmaceutical and biotechs companies collect, aggregate and utilize genetic information that can result in developing more effective therapies and expediting rare disease R&D.
  • In 2018, as we see these areas further develop in front of our eyes, we address the key challenges to bringing more gene therapies to the market and making inaccurate and late diagnostics an issue of the past.


  • While access to orphan drugs improved in the US, Canadian, European, and Japanese markets, in part because of established regulatory and policy frameworks, that is still a major issue in other markets like Latin America, Africa, Middle East, Eastern Europe, Asia, and Australasia.
  • Not all countries have universal healthcare, a reimbursement pathway or recognize orphan drugs in similar terms to those set for US, Europe and Japan. So while these discussions evolve internally on each country we will be bringing government and regulatory authorities, industry, patient organizations and solution providers who have been working to facilitate access from a market penetration perspective as well as patient access to those therapies.