June 20, 2001 (Washington) -- "How can you ignore a dying mother?"
Fred Santino posed his poignant question during a governmental hearing Wednesday, as he spoke of his frustration with a growing problem -- getting limited supplies of experimental drugs to dying patients who might benefit by them. It's a program known as "compassionate use."
Santino's wife, Ruth-Ann, had colorectal cancer. The mother of two teens died on May 5 after a desperate attempt to get a new treatment known as C225. According to Santino, his wife wrote the drug's manufacturer, ImClone, three times but got no response to her pleas for help.
Generally speaking, when a patient needs an unapproved treatment, he or she is enrolled in a clinical trial. But that isn't always possible. In such cases, the FDA allows the manufacturer to make the medication available under a "compassionate-use" waiver.
However, the lack of a coordinated system often leaves patients frustrated and puts drug companies on the defensive, according to testimony provided at a hearing of the House Committee on Government Reform Wednesday.
"If someone with a ... terminal illness wants to seek treatments that may offer [a] cure or slow down the progression of disease, federal agencies and red tape should not stand in their way," says Committee Chairman Rep. Dan Burton (R-Ind.).
Burton says some companies might be afraid to give experimental drugs to extremely sick patients for fear of contaminating their trials and compromising the medication's chances for approval. One solution, says Burton, would be to "wall off" drug studies so that those in compassionate-use programs wouldn't be included in clinical trials.
But even firms with compassionate-use programs may operate under extreme restrictions. For instance, Frank Burroughs of Arlington, Va., says his 21-year-old daughter Abigail died of lung and neck cancer earlier this month. Burroughs says Abigail was a compassionate case, but AstraZeneca and ImClone were not compassionate toward her. They failed to provide drugs that, Burroughs says, could have saved his daughter's life.
"ImClone Systems has no compassionate-use program. Multibillion dollar AstraZeneca has a very small and very narrowly defined compassionate use program that young Abigail did not qualify for ... We did not get a chance to try to save dear, sweet, talented, compassionate Abigail," said Burroughs.
So who does get experimental drugs under compassionate use programs?
Shannon Kellum, for one. Diagnosed with metastatic colon cancer in 1998, all conventional treatments had failed by 1999. Then with her doctor's help, she got ImClone's C225, and her tumor virtually disappeared.
"I thank God for every additional day that I have, and it's very difficult for me being up here, and in some ways I do feel guilty, because I'm still here ... But I also feel that this is a chance for me to help get these drugs approved quicker," Kellum testified.
Samuel Waksal, PhD, president and CEO of ImClone Systems, Inc., said his company had been overwhelmed by 8,000 requests for compassionate use of C225.
Waksal says there's hasn't been enough manufacturing capacity to make the drug for clinical trials as well as for those who need it as a last resort. Waksal is hoping for FDA approval next year and greater of availability of C225.
Meanwhile, Robert Temple, MD, director of the FDA's office of medical policy, told the committee there's "no chance" a drug company's application for approval would be jeopardized by the safe use of unapproved drugs. However, he does see fairness problems under the current system.