Bills would boost research funding for rare disorders through NIH and
FDA
Physicians envision a future with better treatment options for people
with these "orphan" diseases.
By
Michael J. Bernstein, AMNews correspondent. Oct. 21, 2002.
Additional information
Washington -- Legislation poised to pass in Congress could bring new
and much-needed energy to the search for treatment for rare diseases,
according to physicians.
Two bills, which already have passed the House, would double funding
for the Food and Drug Administration's orphan products research grant
program and formally establish the National Institutes of Health Office of
Rare Diseases.
"Doctors like myself and the patients we serve have been waiting for
something like this for a very long time," said Richard Barohn, MD, chair
of the Dept. of Neurology at the University of Kansas Medical Center in
Kansas City. "Hopefully, this legislation will encourage more doctors to
devote a career to finding an answer to a cure for these rare diseases. We
desperately need more money to attract more young investigators into the
field."
One of the House bills would authorize $25 million a year for four
years for the FDA orphan drug research program, which currently has annual
funding of $12 million.
The other bill would authorize $20 million a year for the NIH rare
disease office for the same four years.
The NIH bill also would create centers of excellence designed to offer
training grounds where new investigators could conduct more organized
research into the causes and treatment of rare diseases, commonly called
orphan diseases. The centers, to be located in existing academic research
facilities and hospitals, also would carry out clinical studies and accept
referrals from doctors who need help with diagnoses or treatments. Funding
would come through grants offered by the NIH rare diseases office.
Since 1982, 200 drugs and biological products have been developed
to treat rare diseases.
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These centers of excellence would help doctors provide better care in
their communities, said Hugo Moser, MD, professor of neurology and
pediatrics at Baltimore's Johns Hopkins University Medical Center.
Dr. Moser predicted that when more research is funded, "we will find
that these rare diseases are not as rare as we think." Additional study of
rare diseases would be helpful in developing improved diagnosis and
treatment of more common disorders, he added.
At press time, the Senate was expected to approve a bill that includes
language identical to both House measures. President Bush is expected to
sign the legislation.
A history of small budgets
Physicians and advocates for people with rare diseases say the new
funding in the bills couldn't come soon enough.
When the FDA set up its Office of Orphan Products Development in 1982,
the budget was just $500,000. That figure and the budgets for the next 20
years have been woefully inadequate, according to the National
Organization for Rare Disorders and many doctors treating these patients.
Through the years, supporters of the bills said, patients with some
rare diseases have suffered because there just hasn't been enough
research, funding and emphasis on these little-known disorders.
25 million people in the U.S. have one of 6,000 rare diseases.
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The legislation notes that the FDA has been able to support only small
clinical trials through its orphan products research grants.
These grants have led to partnerships between government and industry
and the development of 23 drugs and four medical devices for rare diseases
and disorders. Unfortunately, the bill language says, "the appropriations
in fiscal year 2001 for such grants were less than in fiscal year 1995."
Since the FDA orphan products office was created in 1982, more than 200
drugs and biological products have been developed to treat rare diseases.
Fewer than 10 were developed before that. The Orphan Drug Act created
financial incentives for research and production of drugs for rare
diseases.
Backers of increased funding for this research said it's remarkable
that many drugs and devices have been produced with so little research
money. They predict that the doubling of funding for the FDA orphan drugs
effort will have the potential to bring about significant improvements in
treatment.
"It's taken a lot of letters to Congress to convince members what it's
like to live without hope," said Abbey Meyers, president of NORD, which
has provided services and lobbied for patients with uncommon diseases
since the 1970s. "It's taken 20 years and a lot of educating. We're
delighted to see this bipartisan, unanimous support in Congress."
Historically, Congress has responded to the most powerful groups that
lobby the most, Meyers said. The problem has been that patients with a
rare disease, by definition, don't have the numbers to create the large
advocacy groups that those with breast cancer or prostate cancer have, she
said.
Meyers added that the $25 million Congress is poised to authorize,
though welcome, represents only $1 for each of the 25 million people in
the nation who have one or more of a total of 6,000 rare diseases.
But many physicians and patient advocacy groups see reason for hope.
Rup Tandan, MD, who treats patients with rare neurological disorders,
said the increased research funds would lead to many more drugs being
screened more quickly and efficiently by the FDA, the NIH and other
agencies.
"There will be more clinical trials, and we will get more answers about
treating and diagnosing patients with rare diseases," added Dr. Tandan,
professor and vice chair of neurology at University of Vermont College of
Medicine in Burlington.
The Tourette Syndrome Assn. also applauded the increased funding for
the FDA orphan drug program.
"There's probably no other group of patients more underserved -- in
regard to both the development of treatments and research -- than those
with rare diseases," said Sue Levi-Pearl, TSA's vice president for medical
and scientific programs.
A major problem to date has been that there has been so little funding
to support preliminary rare disease research by the federal government
that drug companies have resisted getting involved, researchers said.
Because the number of patients with rare diseases is small, the companies
are reluctant to spend the large sums needed to develop drugs if no
scientific groundwork has been laid.
Now, with added funding and the potential for added research by NIH and
other agencies, drug firms may be more receptive to investing time and
money, advocates said.
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ADDITIONAL INFORMATION:
Orphan aid
Two House bills aim to encourage research of rare diseases. A companion
Senate measure contains the same language. The legislation would:
- Authorize $25 million a year in Food and Drug Administration grants
and contracts for research to enhance the development of diagnosis and
treatment of rare diseases. The funding would continue through fiscal
2006.
- Make the National Institutes of Health Office of Rare Diseases
permanent. For several years the office has existed without being
codified.
- Authorize the NIH to create and operate centers of excellence for
rare diseases with a budget of $20 million for each of fiscal years 2003
through 2006. Funding would cover clinical training, patient care costs,
clinical research studies, demonstration programs and continuing medical
education.
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Weblink
FDA Office of Orphan Products
Development page (http://www.fda.gov/orphan/)
NIH Office of Rare Diseases
page (http://rarediseases.info.nih.gov/)
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Copyright 2002 American Medical Association. All
rights reserved.
