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.

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. [...]