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Join the Dialogue at the NORD Rare Summit!

4.0 from 1 vote
Wednesday, October 11, 2017
Topics that have dominated the headlines this year will be featured at the 2017 NORD Rare Diseases and Orphan Products Breakthrough Summit to take place October 16-17 in Washington DC. With more than 80 experts in public policy, regulatory affairs, clinical trial design and patient advocacy on the program, the event promises to generate a lively exchange of ideas.

For the first time this year, the Summit will be CME-accredited. In addition, an interactive, CME-accredited half-day symposium will take place on the day before the Summit at the same location to focus on diagnostic hurdles, tools and resources.

FDA Commissioner Scott Gottlieb MD will present a keynote address on the first day of the Summit that is expected to address topics that he has spoken and written about since his appointment in May, including the implementation of the Orphan Drug Modernization Plan announced by FDA in June.

The program will begin with a community keynote address by Mike Porath, founder and CEO of, who will speak about his experiences as the parent of a child with a rare disease, a member of the board of a nonprofit patient organization and the founder of a media platform to amplify the patient voice.

Medical ethicist Arthur Caplan PhD, will moderate a fireside chat on "Ethical Guidelines for Patient Organizations and Industry to Collaborate" with panelists from the pharmaceutical industry and patient organizations in the U.S. and Europe.

This will be followed by a panel on "Assuring Patient Access: Future Outlook for Patient Assistance Programs". Experts from industry and patient advocacy organizations will discuss the future of these programs, which currently play an important role in assuring patient access to treatments.
Other topics on the agenda that have been in the news this year include: Promoting Earlier Diagnosis, The Power of Data-Sharing, Right-to-Try legislation, and Developing a Global Strategy for Rare Diseases and Orphan Products.

Steve Usdin, Washington editor of BioCentury, will moderate a panel on "The Challenge of Healthcare Costs & Treatment Prices." Another panel chaired by David Scheer, president of Scheer & Company Inc., will address "The Ourlook for Investment in Orphan Products. FDA staff will play a significant role in the program with plenary and breakout sessions in which more than xx FDA staff will speak.

Staff of the National Institutes of Health (NIH) who will be speaking include Carrie Wolinetz PhD, Associate Director for Science Policy at NIH; and Petra Kaufmann MD, Director of Clinical Innovation in the NIH National Center for Advancing Translational Sciences.
Opportunities to network and engage with others working with rare diseases or orphan products include Lunch & Learn Roundtable Discussions that will be offered on both days on topics such as "Gene Therapy Policies at CBER", "What's Ahead for Personalized Medicine" and "Creative Protocol Designs to Accelerate Regulatory Approvals."

In addition, there will be networking receptions and opportunities to set up one-on-one meetings with a mobile app provided beforehand by NORD. The Summit will also feature its largest-ever poster session with nearly 60 posters.
Each year, the NORD Summit attracts approximately 600 participants from patient organizations, government agencies, the medical community and the pharmaceutical industry. The program will take place at the Marriott Wardman Park Hotel in downtown Washington DC near the Washington Zoo.


Source: NORD
4.0 from 1 vote
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