NORD's
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 Check here to read about events on Capitol Hill, funding for rare-disease research, and other topics of interest from NORD's office in Washington, DC.
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Testimony before the United States Senate
Appropriations Committee
June 23, 2010
Mr. Chairman, thank you for the opportunity to testify before you today. I am
Diane Dorman and I am the vice president for public policy of the National
Organization for Rare Disorders, or NORD. We were founded in 1983 to advocate
for the enactment of the Orphan Drug Act, and we remain today the leading
advocate for the 30 million American patients with the estimated 7,000 known
rare diseases. In addition to our advocacy efforts, we also run patient
assistance and patient support programs and have compiled the largest database
of information about rare diseases in the world.
There are many federal and state agencies whose programs and policies affect
people with rare diseases, but none is more important than the Food and Drug
Administration. The FDA is the gatekeeper for the drugs, devices and medical
foods that are needed by patients with rare diseases. The FDA sets the standards
for studying new medical products and therefore plays a central role in research
as well as product approvals.
NORD advocates for full funding of the FDA. We were instrumental in founding an
organization now known as the Alliance for a Stronger FDA, which now includes
more than 180 members representing all of FDA's stakeholders and which has the
singular purpose of advocating for increased funding for the FDA. We have
witnessed what happens when FDA is underfunded: the agency cannot meet its
review times for new drugs and medical devices; cannot provide the guidance that
researchers so desperately seek; and, cannot maintain the public's confidence in
the regulatory system.
Delays in review times and lack of guidance affects our patient constituency
especially hard, because despite the great advances in medicine, there are
approved drugs for only about 200 of the estimated 7000 rare diseases. Many of
our patients are treated with approved products being used off-label, and many
are not being treated at all because there are no treatments.
NORD's top priority, in addition to the support services we provide to patients
with rare diseases, is to advance medical research and the development and
approval of new therapies for our patients.
It is with this perspective that we support in principle any steps that would
advance medical research or provide FDA with the resources it needs to carry out
its critical public health functions. We were encouraged by the creation earlier
this year of a new position within the FDA's Center for Drug Evaluation and
Research, a position dedicated exclusively to rare diseases. We advocate for
more training and support for FDA personnel who interface with the researchers
who develop orphan products, and we support more transparency in the regulatory
system so that investigators and drug and device manufacturers can make the
right decisions as they develop new products.
The current leadership of the FDA has demonstrated its sensitivity to the
vulnerability and special challenges of people with rare diseases. We understand
the constraints on the budgets of federal agencies, but at the same time the FDA
needs more resources if it is to fulfill the commitment it has made to the rare
disease community.
Mr. Chairman, I would be pleased to answer any questions you have. Again, thank
you for the opportunity to appear before you on behalf of the 30 million men,
women and children with rare diseases.
Respectfully Submitted,
Diane Edquist Dorman
National Organization for Rare Disorders (NORD)
1779 Massachusetts Avenue NW, Suite 500
Washington, DC 20036
(202) 588-5700
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