Orphan Diseases and the Future of BioPharma, the Focus of the 2012 Orphan Disease Forum

<em>Centric Health Resources and the National Organization for Rare Disorders (NORD), Co-organizers</em>Alnylam Pharmaceuticals, Genzyme, a Sanofi Company and Millennium: The Takeda Oncology Company<em>, Co-sponsors</em></p>

WASHINGTON, D.C.(April 5, 2012)— Thought leaders from dozens of biotech and pharmaceutical corporations from across the globe will gather June 19 – 20 to discuss innovations and regulations pertaining to Orphan Diseases, at the 2012 Orphan Disease Forum, as part of the 2012 BIO International Conventionin Boston, MA.

The forum is co-organized by Centric Health Resources and the National Organization for Rare Disorders (NORD), and is hosted by the Biotechnology Industry Organization.  

“We are pleased to partner with NORD and Centric on this two-day forum to raise awareness for rare diseases and to improve the health of patients with debilitating and life-threatening diseases,” said Jim Greenwood, President and CEO of BIO. “Thought leaders at the forefront of the international drug marketplace use this forum to advance therapies and cures for impacted patients.”

Industry consultants, biotech, pharmaceutical and venture capital companies and health benefits managers attend thistwo-day forum, as part of the BIO International Convention, the largest global gathering for the biotechnology industry.  The forum and the Convention offer networking and partnering opportunities with policymakers, scientists, CEOs and newsmakers, and hundreds of sessions covering biotech trends, policy issues and technological innovations.

Forum agenda highlights include:

Are We Heading into a Pharmaceutical and Biotechnology Market of Orphan and Narrow Indications?

Industry experts will discuss the changing orphan drug environment. Perhaps the conditions are right for a new strategy: getting early market FDA approval with efficient initial funding, then later broadening the indications.

Tuesday, June 19, 8:30 a.m. – 9:45 a.m.

Speakers: Emil D. Kakkis, PhD, President and Chief Executive Officer, Ultragenyx, Inc., Edward Mathers, Partner, New Enterprise Associates, Inc., David Savello, PhD, Senior Vice President, Development Operations, XenoPort, Inc. and Ron Smith, Vice President, Strategic Development, Blue Cross Blue Shield of North Carolina

Innovations in Supply Chain Economics and Value Creation in U.S. Orphan Markets

With the high per patient cost of many orphan products, demonstrating value to all constituents is particularly challenging considering the current and future state of the healthcare industry. The panel will explore ways manufactures can maximize shareholder value and meet the healthcare reform goals of lower costs and better care.

Tuesday, June 19, 2:00 p.m. – 3:30 p.m.

Moderator:  Doug Paul, Partner, Medical Marketing Economics (MME), LLC

Speakers: Craig Kephart, President, Centric Health Resources,Rita Numerof, PhD, President, Numerof & Associates, Inc.,  and Bill Zabel, Vice President and General Manager, Grifols

Considerations for Novel Approval Pathways for Rare and Orphan Diseases

Are there better ways for the industry to answer the unmet medical needs of people with often devastating conditions, and get drugs for them to market faster? The session will consider how biomarkers, novel clinical trial designs, the use of appropriate controls, and the attitudes of regulatory authorities all affect the goal of getting therapies to patients more quickly.

Tuesday, June 19, 2012, 3:45 p.m. – 5:30 p.m.

Speakers: Timothy Cote, MD, MPH, Chief Medical Officer, National Organization for Rare Disorders (NORD), John Crowley, President and CEO, Amicus Therapeutics, Inc., Sara Nochur, Vice President, Regulatory Affairs, Alnylam Pharmaceuticals and Anne Pariser, Associate Director, Rare Diseases, Center for Drug Evaluation and Research, Food and Drug Administration (FDA)

Specialized Marketing & Sales: A Vision for Orphan and Targeted Therapies

Attendees can expect to gain a critical understanding of how companies are employing specialized marketing and sales approaches that are patient-centric, especially in light of the heightened interest among bio-pharma companies.

Wednesday, June 20, 8:30 a.m. – 9:45 a.m.

Moderator: Donna L. LaVoie, President & CEO, LaVoie Strategic Communications Group Inc

Speakers: Kyle Brown, Founder & CEO, Innolyst, Inc.,Jayne Gershkowitz, Senior Director, Patient Advocacy & Public Policy, Amicus Therapeutics, Inc, Mark Rothera, Global President, Aegerion, and Rogerio Vivaldi, Senior Vice President and Head of Rare Diseases Business Unit, Genzyme, a Sanofi Company

Is Big Pharma the Preferred Partner for Orphan Drug Biotechs?

This session will focus on challenges facing large companies and their need to align their processes and structures with the requirements of orphan markets, including the need for much closer collaboration and openness with patient groups. Additionally, orphan drug commercialization often requires pharma’s broad international reach to capture the limited available patients.

Tuesday, June 19, 2:00 p.m. – 3:30 p.m.

Moderator: Andrew Funderburk, Vice President, Health Advances, LLC

Speakers: Bradley Campbell, Chief Business Officer, Amicus Therapeutics, Daniel Curran, Vice President, Business and Corporate Development, Millennium: The Takeda Oncology Company, Daniel Karp, Senior Director, Worldwide Business Development, Pfizer Inc., Andre Turenne, Vice President, Strategy and Business Development, Genzyme, a Sanofi Company, Jeffrey Walsh, Chief Operating Officer, bluebird bio

The Convention also features the BIO Business Forum, a unique platform for biotechnology and pharmaceutical companies, academic research institutions, and investors from around the world to gather and discuss strategic opportunities. For registration, conference agenda and exhibitor information, visit 2012 BIO International Convention.

BIO is pleased to recognize the leadership provided by the 2012 Orphan Disease Forum sponsors.  The sponsors include: Alnylam Pharmaceuticals, Genzyme, a Sanofi Company and Millennium: The Takeda Oncology Company.

Visit the most up-to-date program and speaker information.

Upcoming BIO Events 
April 16 – 18, 2012
Austin, TX
May 15-16, 2012
Frankfurt, Germany
2012 BIO International Convention
June 18-21, 2012
Boston, MA
June 18 – 21, 2012
Boston, MA


About the National Organization for Rare Disorders (NORD)

The National Organization for Rare Disorders (NORD)is a unique federation of voluntary health organizations dedicated to helping people with rare "orphan" diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service.

About Centric Health Resources

Centric Health Resources, a Dohmen Company, is a nationwide, Patient-Centered Health Management® organization serving patients with rare, ultra-orphan disorders. Centric helps drug innovators better support the patients they serve by providing outsourced services that include reimbursement expertise, dispensing of specialty pharmaceuticals, peer health coaching and clinical support from registered nurses. Like all Dohmen companies, Centric helps reduce cost and complexity in the healthcare supply system by connecting physicians, nurses, health benefit payers, patient advocacy groups, biotech/pharmaceutical companies and families in partnership for improved service and health outcomes for these special patient populations. Visit