2013 BIO International Convention to Host Orphan Disease Forum

Forum to highlight new therapies and diagnosis for rare diseases, business models and partnering strategies for orphan drug products and commercialization of biologics for orphan indications and rare diseases</p>

Washington, DC (February 28, 2013) –The international orphan drug marketplace is expected to grow some 30 percent by 2014, fueled by the entrance of big pharmaceutical companies and U.S. legislation favoring development and coverage. This growth promises to improve the health of patients with debilitating and life-threatening disorders. The Orphan Disease Forum at the 2013 BIO International Convention will provide unique perspective on the orphan drug marketplace, regulatory considerations and much more. Hosted by the Biotechnology Industry Organization (BIO), the Convention, celebrating its 20thanniversary, will take place April 22-25, 2013 at McCormick Place in Chicago, Ill.

“According to the National Institutes of Health, there are approximately 6,800 rare diseases in the United States, affecting nearly 30 million Americans or almost 1 in 10 people,” said BIO President & CEO Jim Greenwood. “The Orphan Diseases Forum is an important program which fosters dialogue amongst industry and patients, ultimately benefiting an increasingly diverse population.”

Forum agenda highlights include:

Welcoming Remarks with Rogerio Vivaldi, Senior Vice President and Head of Rare Diseases Business Unit, Genzyme, a Sanofi Company

Tuesday, April 23, 8:45 am – 9:00 am

200 New Therapies and Means to Diagnose Most Rare Diseases by 2020: Collaboration Toward Ambitious Goals

The International Rare Diseases Research Consortium (IRDiRC) was launched in 2011 by the European Commission and the US National Institutes of Health with the objective to have 200 new therapies and means to diagnose most rare diseases by 2020. This panel will discuss potential pathways that support the infrastructure and innovation needs to accelerate the development of therapies and diagnosis for people with rare diseases. 

Tuesday, April 23, 9:00 – 10:15 a.m.

Organized by the European Commission

Moderated by Ruxandra Draghia-Akli, Director, Health Research, European Commission

Speakers: Luc Dochez, Chief Business Officer, Prosensa, Pamela Gavin, Senior Vice President, National Organization for Rare Disorders, and Lu Wang, PhD, Program Director, National Human Genome Research Institute, National Institutes of Health (NIH)

Orphan and Rare Diseases: Closing Distances in Understanding Larger Disease Mechanisms

Research on rare diseases can shed light on mechanisms and therapeutic opportunities for common diseases. Better tools and improved support models have helped mitigate the special risks of research for rare diseases. This panel aims to educate everyone along the continuum from basic research through clinical trials about the infrastructure and innovation needed to accelerate the development of therapies for people with rare diseases.

Tuesday, April 23, 10:30 – 11:45 a.m.

Organized by aTyr Pharma, Inc.

Speakers: Robert Beall, PhD, President and CEO, The Cystic Fibrosis Foundation, Jose Carlos Gutierrez-Ramos, SVP, Head of BioTherapeutics R&D, Pfizer Inc., John Mendlein, PhD, Executive Chairman and CEO, aTyr Pharma, Inc. and Rogerio Vivaldi, SVP and Head of Rare Diseases, Business Unit, Genzyme, a Sanofi company

Survival Strategies in the New Individual Patient-Centered Marketplace

Necessity has spawned innovation in drug development for rare diseases. Thanks to a shift from a product-focused approach to a patient-centered, outcomes-based approach, we are in a critical time of change. Innovative business models must rapidly meet the needs of our new market: the individual patient. This will require more efficient use of resources, transparency across the development and commercial continuum, better and faster access to data, and personalization of the customer experience.

Tuesday, April 23, 2:30 – 3:30 p.m.

Organized by Centric Health Resources, A Dohmen Company

Moderated by Catherin Sohn, President, Sohn Health Strategies

Speakers: Cynthia LaConte, CEO, Dohmen, John F. McDonald, Vice President Co-Head BD/M&A, Biogen Idec, Inc, John Walsh, President and CEO, Apha-1 Foundation and Eugene Williams, Chairman & CEO, DART Therapeutics, Inc.

Navigating the Special Challenges of Commercializing Biologics for Rare Diseases: Distribution, Partnership, Reimbursement

Manufacturers of biologics for rare diseases face unique commercialization challenges at odds with the model of low-cost, bulk product distribution. Information is key down the line: patients and advocates need guidance about the products’ therapeutic benefit and assistance programs; manufacturers need patient input for marketing and education campaigns; payers need notice about the products’ high cost and evidence supporting coverage, payment, and usage; physicians and staff need payment assurance from payers and knowledge of assistance programs so patients have product access. This panel will feature, biologic marketing and commercialization experts and a patient advocate sharing insights about navigating these challenges.

Tuesday, April 23, 3:45 – 5:00 p.m.

Organized by Xcenda

BIO is pleased to recognize the leadership provided by the 2013 Orphan Disease Forum sponsors.  The sponsors include: Genzyme, a Sanofi Company; Alexion Pharmaceuticals, Inc and CSL Behring.

The BIO International Convention also features the industry's largest partnering event, hosting a record breaking 25,000+partnering meetings in 2012.  More than 4,000 companies participated in One-on-One Partnering meetings in the BIO Business Forum™ and in the BIO Exhibition. These efficient meetings foster the opportunity for companies to initiate business contacts in a friendly environment and schedule meetings prior to the Convention.

Advance media registration is now available for qualified reporters working full time for print, broadcast and web publications with valid press credentials. To register, please visit here.



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March 25-27, 2013
San Diego, CA
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Chicago, IL
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