August 22, 2011

1st Annual Conference on Rare Diseases & Orphan Products

Stellar Lineup of Speakers Announced for U.S. Conference On Rare Diseases And Orphan Products

Join all stakeholders – patient advocates, researchers, government, industry, investors – in this 1st annual summit featuring thought leaders and focusing on collaboration!

You are invited to the 1st Annual U.S. Conference on Rare Diseases and Orphan Products to be hosted by the National Organization for Rare Disorders (NORD) and the Drug Information Association (DIA). Additional support for this conference is being provided by the Food and Drug Administration (FDA), National Institutes of Health (NIH), European Rare Diseases Organization (EURORDIS) and Duke University School of Medicine.

There will be separate tracks for patient advocates and organizations, researchers/drug and device companies, and investors. The conference will incorporate the well-received rare disease investigators training course offered by FDA for the first time last year.

NORD is the voice of the nearly 30 million Americans who have rare diseases. DIA is a nonprofit, global professional association of more than 18,000 members who are engaged in the discovery, development, and management of pharmaceuticals, biotechnology, medical devices, and related health care products.

The event will take place October 11-13 at the Omni Shoreham Hotel, Washington DC.

Speakers will include:

  • NIH Director Francis S. Collins, MD, PhD
  • Social Security Commissioner Michael J. Astrue, JD
  • John F. Crowley, Chairman and CEO, Amicus Therapeutics
  • Fred Hassan, MBA, Partner, Warburg Pincus, Chairman of the Board, Bausch and Lomb
  • Mark B. McClellan, MD, PhD, Director, Engelberg Center for Health Care Reform, The Brookings Institution
  • Frank J. Sasinowski, MPH, JD, Partner, Hyman, Phelps & McNamara
  • Jeffrey Shuren, MD, JD, Director, Center for Devices and Radiological Health, FDA
  • Janet Woodcock, MD, Center for Drug Evaluation and Research, FDA
  • Margaret Anderson, Executive Director, FasterCures
  • Kelly C. Slone, Director, Medical Industry Group, National Venture Capital Association
  • Suzanne L. Bruhn, PhD, Senior Vice President, Strategic Planning & Program Management, Shire Human Genetic Therapies

For patient advocates and patient organizations, there will be a separate track of presentations focused on strategic planning, growth, and effective advocacy for the patient community. In addition to the three tracks, all stakeholders will join together in high-level plenary sessions to discuss how the various interests can collaborate more effectively.

If you can only attend one rare disease/orphan product conference this year, this should be the one! It will provide a unique opportunity for thought leaders from all perspectives to address the issues and seek ways to drive progress toward safe, effective treatments for patients.


Limited scholarship assistance may be available for patient representatives

Online registration for this unique event is now open on the DIA website. For representatives of patient organizations, limited scholarship assistance may be available. Patient representatives interested in applying for scholarships may write to Audrey Ashley at NORD ( and request an application form.

There will also be limited exhibiting space for companies and organizations. Inquiries about this may be directed to Shannon Lewis at DIA ( 215 442-6149.


Attendees will benefit from discussions on the FDA and NIH processes, health care reform, changes in insurance reimbursement, off-label issues, and de-risking investments. Specific topics for the three tracks will include:

For Researchers, Drug and Device Companies:

  • Senior medical officials from FDA and NIH will discuss product development, how to develop a new product, and how to comply with NIH grant and FDA approval requirements.

  • The conference will provide a platform for addressing the unique challenges faced by companies in the development of orphan products and how to avoid common pitfalls.

  • Industry leaders and senior agency officials will discuss how to work with federal agencies on everything from grants to orphan designation and clinical development to product approvals.

For Patient Advocates and Patient Organizations

  • Learn how you can become more effective advocates for the patients you represent.

  • Learn about the new and emerging federal policies that affect patients with rare diseases.

  • Learn how the government and private sector are addressing the special challenges faced by patients in the new health care environment.

  • Meet individuals who run patient organizations efficiently and creatively to advance the interests of patients and how best to organize for effective advocacy.

For Investors:

  • Explore the new concept of venture philanthropy and how it will affect research funding.

  • Gain a better understanding of how the FDA and NIH work and how they interact with the drug and device development processes.

  • Learn ways to de-risk your investments in orphan products and understand better the timelines for research and regulatory reviews.

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