What do you think about when you hear the name Jack Klugman? The slovenly sportscaster Oscar Madison in The Odd Couple? The tenacious, irascible medical examiner on Quincy, M.E.? Or perhaps the throat cancer survivor who had to reteach himself to speak so that he could return to a career he loved?But there is another legacy that Jack Klugman left us that is much less well known. Klugman, who died on Christmas eve, left us something much more important and tangible. He played a vital role in the passage of the Orphan Drug Act of 1983.
An article in the Los Angeles Times about congressional hearings on the orphan drug issue caught the attention of Klugman's brother Maurice, a Hollywood writer and director who worked with him on Quincy.
"Orphan diseases" refers to rare ailments with relatively few afflicted. They were considered ignored or "orphaned" by the pharmaceutical industry, who did not see any financial return on investment for research on treatments for those illnesses.
In 1981, Maurice wrote an episode entitled "Seldom Silent, Never Heard" about a young woman with It immediately brought a lot of attention to the orphan drug issue, and the chief sponsor of the bill, Congressman Henry Waxman, asked Jack to .
During his testimony, Mr. Klugman made a very persuasive argument:
" I'm not trying to legislate morality, I'm just trying to encourage it."The Orphan Drug Act (ODA) offered pharmaceutical companies three main incentives for investing in orphan drug development:
- A lighter regulatory burden for developing new orphan drugs
- A seven-year monopoly
- A 90% tax credit for the cost of clinical trials
The bill passed easily in the House, but was up against stiff opposition from Chairman of the Senate Finance Committee Orrin Hatch. Hatch stripped the tax credit from the bill -- a main incentive for pharmaceutical companies.But Jack and Maurice came up with a novel idea: They wrote a second episode of Quincy, "Give Me Your Weak" where a "fictitious" Senator is holding up the Orphan Drug Act and Quincy goes to Washington to try to convince him to change his mind.
During the pivotal scene, Quincy tells the Senator to look out his window, where a large crowd was protesting and carrying signs saying: “We Want the Orphan Drug Act.” Most interestingly, the show hired 500 extras for the scene, all of whom were suffering from rare diseases themselves!
Hatch eventually gave in and the Waxman-Hatch Orphan Drug Act of 1983 was passed and signed by President Reagan.
Technically, The Orphan Drug Designation program provides orphan status to drugs and biologics that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
Since 1983, the program has successfully enabled the development and marketing of more than for rare diseases. More than a thousand are currently under development. One of the first drugs developed under the program was AZT -- for the treatment of HIV-AIDS.
The expansion by the FDA to include biologics (vaccines, blood or blood components, allergenics, somatic cells, gene therapy, tissue, recombinant therapeutic proteins, or living cells that are used as therapeutics to treat diseases) is credited with promoting growth of the biotech industry in the U.S.