American Conference Institute’s Definitive Legal, Regulatory, and Commercial Guide to

Orphan Drugs and Rare Diseases

Maximizing Opportunities and Overcoming Stumbling Blocks in the Designation and Development Process

Wednesday, November 28 to Thursday, November 29, 2012
Hyatt Regency, Boston, MA

Finally, a forum designed to give practitioners the complete picture of the orphan drugs and rare diseases landscape: the evolving laws, the regulations, the commercial implications, the patent considerations, and the international framework. Gain the competitive edge and capture your share of this burgeoning market as more competitors announce their intention to jump into the ring.

There are an estimated 6,000 rare diseases currently recognized in the U.S. alone; European authorities recognize as many as 8,000 rare diseases. With only approximately 150 treatments for these diseases approved so far according to the FDA’s orphan designation database, there is a unique opportunity for companies of all sizes to invest in research and development to try to capture this lucrative market share. Indeed, according to a leading industry report, the orphan disease therapeutics market will continue to see a marked increase in value through the decade, increasing from 2010’s $2.3 billion to an estimated $6 billion in 2018.1

Leave this conference with the knowledge necessary to realistically forecast the costs of orphan drug development balanced against the enormous potential for increased profit margins and attendant goodwill.

In-house experts, rare disease foundation representatives, and top regulatory and patent attorneys will help you analyze the strategic concerns which go into the decision to venture into this last frontier in drug development and to provide patients facing this unmet need access to a safe and effective therapy. Our faculty of expert practitioners, including leading scholar Dr. Christopher-Paul Milne, Associate Director, Center for the Study of Drug Development, will provide a nuanced look into the orphan drug market and help demystify the orphan drug designation process. In addition to hearing from the leading regulatory attorneys and experts who have successfully crafted an orphan drug designation, you will hear directly from keynote speaker, Timothy R. Cote, Director of Office of Orphan Products Development at the U.S. FDA from 2007-2011, who will give you an insider’s view of the criteria FDA looks for when determining the medically plausible legitimacy and will give real-world examples of what causes a designation rejection. Learn best practices on cooperatively working with the OOPD to get your designation request right the first time.


Devise strategies to minimize risk and capture the enormous market share at stake in this rapidly
burgeoning market.

Developing drugs and investing in research and development can be risky business in general, and it can be even more so for small populations with unique disease states. Our faculty of leading experts from Pfizer, Shire, Medicis, GSK, Emergent BioSolutions and many more will give you strategies to offset potential risks inherent to orphan drug development including:

  • Understanding and factoring in the unique incentives, including favorable exclusivity, pricing, and tax benefits, for companies who decide to pursue designation
  • Replenishing product pipelines in the face of the patent cliff through novel therapies
  • Preparing for eventual pharmacovigilance and labeling issues downstream as designation takes off
  • Designing clinical trials end points and proving safety and efficacy in a smaller population
  • Protecting orphan drug status and keeping competitors at bay post-Makena

Do not miss your chance to be part of the most in-depth and cutting edge conference designed to strengthen your chances for successful orphan drug designation and development by calling 888-224-2480, faxing your registration form to 877-927-1563 or registering online.

1 Orphan Disease Therapeutics Market to 2018 – Improved Understanding of Rare Diseases’ Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation, GBI Research, April 2012.