A funny man who plays an unfunny story yesterday -- about millions of people "unfortunate enough to have a disease that never made it to the top 40."
Drugs for these people often are never sought, never fully developed or never made commercially available because there are not enough patients to make sales profitable, actor-commedian Jack Klugman, television's "Quincy," told a House health subcommittee.
Klugman was the star witness in a hearing room packed with drug firm officials and a small crowd of men, women and children who are victims of rare diseases.
Among the diseases are: amyotropic lateral sclerosis ("Lou Gehrig's disease"), which affects only 9,000 currently; Huntington's disease (it killed singer Woody Guthrie), which affects 14,000, and myasthenia gravis, which affects 30,000. Also included, because the drugs used for them are not effective enough, are such diseases as epilepsy, multiple sclerosis, Parkinson's disease, cerebral palsy and others, which together affect millions.
Also included is Tourette's disease, the symptons of which are on often gruesome combination of facial distortions and involuntary vocal outbursts. Adam Seligman, a 19-year-old Tourette's patient from Los Angeles, sat alongside Klugman as he testified. Klugman starred in a "Quincy" episode that dealt with the plight of a boy with Tourette's. The TV role inspired patients' groups and Rep. Ted Weiss (D-N.Y.) sponsor of a bil to spur development fo so-called "orphan drugs," to ask Klugman to testify.
"I have many qualms because I come here as a celebrity rather than as an authority," Klugman said. "But imagine evaluating a person's suffering by whether or not his illness is profitable."
Repeating charges made by many critics, Klugman also blamed the long, costly regulatory process required by the Food and Drug Administration, following congressional mandates on drug safety and efficacy.
Drug firm officials said that, despite lack of profit, they have marketed 40 drugs sinced 1970 for rare diseases, and have distributed 39 more under "compassionate" permits from the Food and Drug Administration.
Dr. J. Richard Crout, head of the FDA Bureau of Drugs, said the FDA has devoted considerable effort to issuing such permits to qualified medical investigators and getting much-needed drugs on sale faster.
But Crout admitted "the plathway to [federal] drug approval is often slow, tortuous, frustrating [and] painful" to both researchers and patients.
And Lewis Engman, president of the Pharmaceutical Manufacturers Association, admitted that there is a lag in the development of "orphan" drugs. He said the industry in forming a new commission "to collect and distribute basic research leads" to inspire development.
Weiss' bill is one he took over from former New York Democratic representative Elizabeth Holtzman. It would have the National Institutes of Health collect information, then give subsidies, loans and grants to firms and researchers to develop drugs for which there is little demand.
But the main way to go in a time of tight budgets, Weiss said, might be tax breaks and other incentives for industry.
Reps. Henry A. Waxman (D-Calif.) and Edward R. Madigan (R-Ill.), subcommittee chairman and ranking minority member, agreed to try to develop some kind of orphan drug bill at this session. Neither was ready to back any one approach.
All who testified agreed that industry, government and agitating patients all must help. So far, as Klugman summed up, "there are no vilains" in this story "but there are also no heroes."