NORD Wants $1 For Each Person Suffering With a Rare 'Orphan' Disease
NORD: Funding for
Orphan Research Program Inadequate
17 Apr 17:44
NEW FAIRFIELD, Conn., April 17 /U.S. Newswire/ -- The National Organization for Rare Disorders (NORD) voiced its concern today over the inadequate funding of the Orphan Drug Research Program at the Food and Drug Administration (FDA). Funding for this critically important program has remained static for many years, as evidenced by the FDA's budget request of only $12.5 million, nearly the identical amount appropriated for FY 2001.
In testimony submitted to the Senate and House Appropriations subcommittees with jurisdiction over FDA funding, NORD is requesting that just one dollar for each rare disease patient in America, rather than the current funding level of only fifty cents per patient, be appropriated to the Orphan Drug Research Program.
Because there are many diseases and conditions too rare to attract private investment by large pharmaceutical companies, Congress created the research grants program in 1983 to provide funding for pivotal clinical trials on new orphan drugs, medical devices, and medical foods for rare diseases. The investment necessary for research and development of new drugs and devices is too large in comparison to the size of the potential market for a rare disease.
The funds made available through the grants program encourage academic scientists and small companies to conduct clinical trials at academic institutions throughout the nation to develop the preliminary evidence necessary to attract commercial sponsors.
Since 1983, the FDA has approved over 218 orphan drugs for marketing, and more than 800 additional drugs are in the research pipeline. Of those products approved for marketing, 27 (23 drugs and 4 medical devices) were developed with funding from the orphan product grants.
"These 27 treatments and devices would not be on the American market today, saving the lives of thousands of Americans, enabling them to return to school or work, if Congress had not created this small but critically important pool of research funds," said Abbey Meyers, president of NORD.
NORD is a federation
of approximately 140 voluntary health organizations and over 70,000 individual
patients, healthcare providers and clinical researchers dedicated to helping
people with rare "orphan" diseases. There are 25 million Americans suffering
with the known 6,000 rare "orphan" diseases in the United States. An orphan
disease is defined by the Orphan Drug Act of 1983 as any disease or condition
impacting fewer than 200,000 Americans. NORD is committed to the identification,
treatment and cure of rare disorders through programs of education, advocacy,
research and service.
Copyright 2001, U.S. Newswire