Controversial 'Compassionate Use'

Some Patients Get Experimental Drugs

As if battling colon cancer weren't enough, two women also fought desperately for a scarce experimental drug each believed could help them. But only one of them got the drug, and she believes it was her personal plea to the drug maker's president that made the difference.

Lesley Stahl reports on the elusive practice called "compassionate use" - a controversial way for some patients to obtain experimental drugs before they've been FDA-approved.

"It just makes me angry," says Ruth-Ann Santino, who did not get the cancer drug C-255. "There's no written criteria…available to say why they are getting it and [the drug company] can't tell you why…To me, that's a true injustice," she tells Stahl. Santino, 51 and the mother of two teens, made phone calls and wrote letters for months to anyone who could help, including President Bush, but it didn't help her get the drug. Santino was told when she began her campaign that 2,000 patients were on a waiting list to get C-225 through compassionate use. Family attorney and friend, Alex Moschella, tells that Ruth-Ann unfortunately lost her battle with colon cancer late Friday night.

Amy Cohen tried just as hard and was also turned down for C-225, but the 36-year-old mother tracked down the president of ImClone, the maker of C-225, in a desperate plea. "I called at a very early hour…'Please, I need two minutes of your time, sir,'" she said. Cohen says she was told by the president that he would ask his brother, the firm's medical director, about her case. After much paperwork and three months, Cohen got her C-225. "I do believe [the phone call] opened the door for me, to lend a voice, a name…versus just being a patient," she says.

ImClone declined to be interviewed and would not reveal its criteria for compassionate use, but says that its focus must be on clinical trials for C-225, a process it must complete for the FDA to approve the drug for mass use.

The FDA has no authority to compel drug companies to provide drugs under compassionate use. At least one company provides what it considers to be a fair program. AstraZeneca is testing a lung cancer drug called Iressa and runs a weekly lottery which randomly selects patients for compassionate use. This program has cost Astrazeneca close to $10 million, a sum many companies would probably balk at.

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