WASHINGTON -- Actor Jack Klugman, the television pathologist 'Quincy,' assumed the role of a congressional witness Monday to urge help for Americans suffering from rare diseases for which medicines are not available.
With Klugman was Adam Seligman, 19, Los Angeles, a victim of Tourette syndrome whose real life story prompted a recent 'Quincy' episode that tuned Klugman into the 'orphan drug' problem.
Orphan drugs are those not widely produced because of profit or regulatory barriers, yet which could be of help to people suffering from rare disorders such as those that killed folk singer Woody Guthrie and baseball star Lou Gehrig.
Klugman noted he was testifying at a time of budget cuts and waning support for federal spending. 'It shows you why I'm such a lousy handicapper,' he said. 'But I'm also an eternal optomist.'
Klugman said there are no villains or heros in the drug situation. 'We are not talking about orphan drugs. We're talking about orphan people.'
Chairman Henry Waxman, D-Calif. of the House health subcommittee, said, 'It is not acceptable for people to continue to suffer and die because they have a disease that only affects a small number of people.
'The fact that there are only 100,000 people suffering from Tourette syndrome, 14,000 from Huntington's disease, 9,000 from Lou Gehrig's disease and 27,500 from spina bifida does not make the victims less important than people with more common ailments.'
Rep. Ted Weiss, D-N.Y., has proposed legislation to establish an office in the National Institutes of Health to deal with drugs intended for the victims of rare diseases.
Klugman said his recent show, prompted by an earlier subcommittee hearing, raised questions about the profit motive in drug development as well as why extensive safety tests are needed for drugs intended for a relatively small patient population.
He quoted a character in his show who asked why 'the drug companies are tripping over each other trying to come up with a better remedy for athlete's feet' while thousands of victims of rare disease go without medication.
Dr. J. Richard Crout, head of the FDA's Bureau of Drugs, acknowledged that the road to drug approval often is slow and frustrating because advocacy for such drugs comes from patients and their doctors -- not from industry.
