WASHINGTON, May 14 /PRNewswire-USNewswire/ -- A shortage of new pharmaceutical products in the pipeline combined with new scientific tools has created a climate of opportunity for the rare disease community, a senior Food and Drug Administration (FDA) official said today at a conference hosted by the National Organization for Rare Disorders (NORD).
The blockbuster model is no longer viable, said Janet Woodcock, MD, director of the Center for Drug Evaluation and Research at FDA. As a result, she said, companies are more willing to consider new options, including more products for rare diseases.
Former National Human Genome Research Institute Director Francis Collins, MD, PhD, agreed, adding that it's time to de-risk orphan drug development, making it a more viable business model for companies.
Collins and Woodcock were among several speakers at a Partners in Progress Summit hosted by NORD in Washington, DC. NORD represents the nearly 30 million Americans who have rare diseases, or ones affecting fewer than 200,000 people.
Peter L. Saltonstall, NORD's president and CEO, noted that most rare diseases have no FDA-approved treatment and many patients are denied insurance for unapproved treatments. NORD sponsored the summit, he said, to develop a policy agenda for action to spur development of new therapies and ensure that patients have access to them.
Former FDA Commissioner David Kessler, MD, moderated the event. Speakers included thought leaders in government and industry working with rare diseases and orphan products. A theme articulated by several speakers was that now is the time for rare disease patients and researchers to press their case.
The healthcare system we know today is going to be completely transformed over the next 12 months, said Tommy Thompson, former governor of Wisconsin and former Secretary of Health and Human Services. Now is the time for action.
NORD Board of Directors Vice Chairman Frank Sasinowski announced the formation of a Rare Disease Congressional Caucus to seek creative solutions to problems faced by patients and to serve as a forum for discussion of issues related to access, research, and increased innovation.
The Caucus will also seek to increase funding for two government agencies the National Institutes of Health Office of Rare Diseases Research and the FDA Office of Orphan Products Development. In addition, it will address regulatory issues facing the pharmaceutical, biotechnology, and medical device industries, Sasinowski said.
Social Security Commissioner Michael Astrue described efforts to expedite the process of applying for Social Security disability assistance for people with severely disabling rare diseases. Under Commissioner Astrue, the Social Security Administration last fall launched a new Compassionate Allowances initiative to reduce problems encountered by people with certain severe diseases when they apply for assistance.
Presentations from the Partners in Progress Summit will be posted soon on the NORD website at www.rarediseases.org.
|SOURCE National Organization for Rare Disorders (NORD)|
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