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1st Annual Rare Diseases/Orphan Products Summit

Read about the highlights from the 1st Annual Rare Diseases and Orphan Products Summit hosted in Washington, DC in 2011.

Updates from Day 1:

Hightlights of the first day of the U.S. Conference on Rare Diseases and Orphan Products included the release of a landmark study of FDA orphan drug approvals from January 1983 through June 2010. Frank Sasinowski, chair of the NORD board of directors, presented the findings in a morning plenary session and at an afternoon press briefing. View Frank Sasinowski's slides.

Other highlights included the following:

From Yann Le Cam, CEO of EURORDIS (European Rare Disease Organization):

This conference is part of a partnership between EURORDIS and NORD to "beat the drums on both sides of the Atlantic." EURORDIS will host a companion conference in May 2012 in Brussels.

From John Crowley, father of two children with Pompe disease and CEO of Amicus Therapeutics:

Under the current paradigm, it will take almost 200 years to develop medications for just half of the known rare diseases.

From Janet Woodcock, MD, Director of FDA's Center for Drug Evaluation and Research:

Drug development for rare diseases is different and requires flexible regulatory standards. We need uniform articulation and application of these policies.

From Jeffrey Shuren, MD, JD, Director of FDA's Center for Devices and Radiological Health:

Collaboration with patients is important to us. In 2010, we started a series of town halls and we're working with patient advocacy groups, under the auspices of NORD, to figure out how better to get patient input on issues like benefit-risk.

From Mark McClellan, MD, PhD, Director, Engelberg Center for Health Care Reform, Brookings Institution:

There will be intense pressure to reduce costs in health care. It will be important to have good performance measures but that's very hard to do with rare diseases so input from patients will be especially important.

From Francis Collins, MD, PhD, Director of NIH:

The science of rare diseases has never moved faster than it is right now. But, at the same time, we're seeing a decrease in resources.

The U.S. Conference on Rare Diseases and Orphan Products continues through Thursday at the Omni Shoreham Hotel in Washington DC. It is co-sponsored by NORD and the Drug Information Association (DIA), with collaboration from EURORDIS, NIH, FDA, and the Duke University School of Medicine.

Updates from Day 2:

Topics included the following:

For Patient Organization Leaders

  • Pathways to Funding of Research: Opportunities for Patient Organizations with Stephen C. Groft, PharmD, Director of NIH Office of Rare Diseass Research; Katherine Needleman, Director, FDA Orphan Products Grants Program; and Matthew Alsante, Executive Director, Sarcoma Foundation of America
  • How Academic Investigators and Patient Organizations Can Collaborate to Support and Advance Research with Margaret Anderson, Executive Director, FasterCures, and James Cloyd, PharmD, Director of the University of Minnesota Center for Orphan Drug Research, moderated by E. Michael Scott, Vox Medica
  • From Research to Market: How the FDA Process Works with senior FDA representatives
  • Developing a Strong Patient Advocacy Organization: Taking a Leadership Role in Research with Kari Luther Rosbeck, President and CEO, Tuberous Sclerosis Alliance; Ronald Bartek, President & Co-Founder, Friedreich's Ataxia Research Alliance; and Ann Brazeau, VP of Development, MPN Research Foundation

For Industry

  • Landscape for New Product Development, chaired by Gregg Lapointe, CEO, Sigma-Tau Pharmaceuticals; Christopher Austin, MD, PhD, Scientific Director, NIH Center for Translational Therapeutics; Lynne McGrath, PhD, Vice President and North American Head, Drug Regulatory Affairs, Novartis Oncology Corporation; and Christopher-Paul Milne, DVM, MPH, JD, Associate Director, Tufts Center for the Study of Drug Development
  • Understanding the Regulatory Environment for Investors: Predictability and Consistency with Jeffrey Siegel, MD, Senior Group Medical Director, Genentech; David Meeker, MD, Chief Operating Officer, Genzyme Corporation; and Jonathan Leff, MBA, Managing Director, Warburg Pincus
  • The Economics of Investing in Orphan Products with David Scheer, President, Scheer & Company; Andrew Callos, VP, Commercial Development, Pfizer Specialty Care; and Sanf Patel, President and CEO, Synageva BioPharma Corporation
  • How to Select/Vet Candidate Diseases and Products with Suzanne Bruhn, PhD, Senior VP, Shire Human Genetic Therapies
  • International Markets and Product Development with Yann Le Cam, CEO EURORDIS, and Carlo Incerti, MD, Head, Global Medical Affairs, Genzyme Corporation in a session chaired by Dennis Jackman, Senior VP, Public Affairs, CSL Behring

For Rare Disease Investigators:

  • Regulatory Requirements for Marketing Applications, with a panel moderated by Andrew Mulberg, MD, Division Deputy Director, Gastroenterology and Inborn Errors Products, Office of New Drugs, CDER, FDA
  • Clinical Endpoint Identification and Development with a panel chaired by John McKew, PhD, Chief, Therapeutic Development Branch, NIH Center for Translational Therapeutics, with Petra Kaufmann, MD, Director, Office of Clinical Research, NIH NINDS, and Laurie Burke, RPh, Associate Director for Study Endpoints and Labeling, Office of New Drugs, CDER, FDA
  • Biomarkers and Surrogates with Robert Temple, MD, Deputy Director, CDER, FDA; Marc Walton, MD, PhD, Associate Director for Translational Medicine, CDER, FDA; and Thomas Fleming, PhD, Professor of Biostatistics, University of Washington

The conference continues through today. It is co-sponsored by NORD and DIA (the Drug Information Association), with collaboration from EURORDIS, FDA, NIH, and the Duke University School of Medicine.

Updates from Day 3:

A surprise announcement by Social Security Commissioner Michael Astrue headlined Day 3 of the U.S. Conference on Rare Diseases and Orphan Products, sponsored by NORD and DIA.

Speaking in a luncheon address, Commissioner Astrue announced that 13 additional conditions, all of which are rare, have just been added to the list of Compassionate Allowances. The Compassionate Allowances Program fast-tracks disability decisions to ensure that Americans with the most serious disabilities receive their benefit decisions within days instead of months or years.

"I have good news for the people in this room," Commissioner Astrue told the approximately 400 participants in the NORD conference. "Today, we are adding 13 more conditions to the Compassionate Allowances list."

In July, at a Capitol Hill event hosted by the Social Security Administration, Mr. Astrue, NORD President and CEO Peter L. Saltonstall and others celebrated the milestone of 100 medical conditions on the list. Yesterday's announcement brings the total to 113.

"This year, from 3.2 to 3.3 million people will apply for benefits," Commissioner Astrue said. "Approximately 150,000 people have received Compassionate Allowances this year, typically receiving disability benefits in two weeks or less."

Read the SSA press release, which includes the list of the 13 medical diagnoses.

Other highlights from Day 3 of the conference included the following:

Fred Hassan, Chairman of the Board of Bausch and Lomb, delivered a keynote address on the economic outlook for orphan product development and investment.

Steve Usdin, Washington Editor of BioCentury and co-host of BioCentury This Week, moderated a "roving microphone" session with audience discussion of economic and ethical issues related to rare diseases and orphan products.

John Jenkins, MD, Director of the Office of New Drugs, CDER, FDA, spoke on efforts to accelerate drug development.

Anne Pariser, MD, Associate Director for Rare Diseases in the Office of New Drugs, CDER, FDA, moderated a session on innovations in product development.

Panelists Liz Moench, President & CEO, MediciGlobal; Pamela Gavin, Senior VP, NORD; and Wendy White, President, Siren Interactive, spoke on using technology to advance research and patient recruitment and retention.

The training program for rare disease investigators developed by Dr. Pariser and her FDA colleagues last year continued throughout the day with speakers from NIH and FDA addressing specific topics related to rare disease research and product development.

Thursday was the concluding day of the conference. The U.S. Conference on Rare Diseases and Orphan Products was co-sponsored by NORD and DIA (Drug Information Association) with collaboration from EURORDIS (the European Rare Disease Organization), the National Institutes of Health (NIH), the Food and Drug Administration (FDA), and the Duke University School of Medicine.

View images from the conference here.

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