2013 BIO International Convention to Host Orphan Disease Forum

  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

2013 BIO International Convention

Business Wire

WASHINGTON -- 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 20^th anniversary, 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
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
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 theindustry'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 toinitiate business contacts in a
friendly environment and schedule meetings prior to theConvention.

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.

About BIO

BIO represents more than 1,100 biotechnology companies, academic institutions,
state biotechnology centers and related organizations across the United States
and in more than 30 other nations. BIO members are involved in the research
and development of innovative healthcare, agricultural, industrial and
environmental biotechnology products. BIO also produces the BIO International
Convention, the world’s largest gathering of the biotechnology industry, along
with industry-leading investor and partnering meetings held around the world.
BIO produces BIOtechNOW, an online portal and monthly newsletter chronicling
“innovations transforming our world.” Subscribe to BIOtechNOW.

Upcoming BIO Events

BIO IPCC Conference
March 25-27, 2013
San Diego, CA

BIO International Convention
April 22 – 25, 2013
Chicago, IL

World Congress on Industrial Biotechnology
June 16-19, 2013
Montreal, Canada


Biotechnology Industry Organization (BIO)
Abigail Hirsch, 202-962-9230
Web: www.bio.org
Blog: www.biotech-now.org
Twitter: @IAmBiotech
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