Deputy HHS Secretary, Investors and Entrepreneurs to Share Their Vision of Upcoming New Era in Health Care

How Will the Affordable Care Act, Globalization and Current Market Trends Affect Development of Therapies for People with Rare Diseases?

WASHINGTON, Sept. 18, 2013 /PRNewswire-USNewswire/ -- One of the hottest trends in the pharmaceutical and biotechnology industries in recent years has been the development of orphan products or treatments for people with rare diseases. 

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Now, industry-watchers are wondering how legislation such as the Affordable Care Act and the FDA Safety and Innovation Act of 2012 as well as current global market and investment trends may affect this rapidly growing field.

That topic will take center stage with an all-star platform of speakers Oct. 7-9 at the U.S. Conference on Rare Diseases and Orphan Products in Bethesda, MD.  Speakers will include Deputy HHS Secretary William Corr; entrepreneur David Scheer; and investors David Mott of New Enterprise Associates; Kris Jenner, Rock Springs Capital; Rajiv Kaul, Fidelity Investments; and Jean-Francois Formela, MD, Atlas Venture.

The conference will also provide an opportunity for approximately 100 representatives of rare disease patient organizations to share first-hand their needs and concerns.  In addition, senior Food and Drug Administration officials will lead sessions on FDA's new patient-focused initiative, accelerated regulatory pathways, and other current topics.

"We'll be focusing on the new era in health care," said Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), a nonprofit organization representing the 30 million Americans with rare diseases. 

"There have been tremendous recent advances in research, regulatory practices, level of industry/investor interest, and related legislation.  We want to pull it all together and address the key question:  What does this mean to patients and to those seeking to develop innovative new therapies? And what can we learn from each other to ensure that greater progress is made in the future?"

The conference is being sponsored by NORD and DIA (the Drug Information Association), in collaboration with FDA, NIH (the National Institutes of Health), and EURORDIS (Rare Diseases Europe).  It will take place at the Marriott North Hotel and Conference Center in Bethesda.  It is open to all, and online registration is available.

In addition to sessions on the investment environment, globalization, and research frontiers, other topics and speakers will include:

• William Shrank, MD, of Harvard Medical School and former head of research at the Centers for Medicare and Medicaid Services Innovation Center on "The Health Care System of the Future"
• Janet Woodcock, MD, Director of FDA's Center for Drug Evaluation and Research, on FDA Initiatives on Orphan Products
• A panel chaired by Pamela Gavin of NORD on "Assuring Patient Access to Treatments"
• A panel chaired by Kevin Lee, PhD, of Pfizer on "How Industry and Patients Can Partner in Rare Disease" and
• A panel of representatives of health care payers on "How Payers Make Decisions Related to Orphan Products" and
• A panel chaired by John C. McKew, PhD, of the National Institutes of Health on "FDA and NIH Perspectives on Drug Repurposing"

SOURCE National Organization for Rare Disorders (NORD)