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Terminally ill patients want access to experimental drugs

Anna Tomalis and her mother, Elizabeth.
Anna Tomalis and her mother, Elizabeth. Andrew Councill / MCT

WASHINGTON — At the end of March, doctors told Anna Tomalis to stop the chemotherapy because it was no longer working, to go home and enjoy what time was left.

But the 12-year-old wanted no part of it. Choosing to continue the fight, she and her parents discovered 10 to 15 experimental drugs that have shown promise in treating Anna's rare form of liver cancer. There's one big problem, though: She can't get access to any of them because the drugs haven't been approved by the federal government.

It's all very perplexing to the seventh-grader from Maryland, who doesn't understand why she's being denied medicine that might help her live longer.

"I really don't want to stop and I don't think we should stop," she said in an interview Thursday. "I know there are other drugs out there for me. ... I'm not happy with it. I don't think it's right."

With more than 550,000 Americans dying from cancer each year, such pleas are gaining increased sympathy on Capitol Hill. In an unusual alliance, conservative Kansas Republican Sen. Sam Brownback and liberal California Democratic Rep. Diane Watson introduced legislation this week that would force the government to speed up its drug-approval process and make sure that terminally ill patients get quick access to investigative drugs.

"What we need is a system that looks at the patient and their life-or-death situation, not at a bureaucracy and its needs," said Brownback, a melanoma survivor. "This is deadly neglect and it can't continue."

Watson likened the new effort in Congress to the grass-roots movement in the gay community in the 1990s, when activists demanded experimental drugs for terminally ill patients with AIDS.

"Why wouldn't anyone whose diagnosis amounted to being handed a death sentence not want to see an opportunity to try these drugs?" asked Watson. "I am so pleased that we are finally recognizing what the activists knew many years ago."

Critics warn that it would be a mistake to allow patients to essentially demand a course of treatment before it has been approved by the Food and Drug Administration. And that argument has held sway with the courts.

In January, the Supreme Court refused to review a ruling that terminally ill patients have no constitutional right to be treated with experimental drugs, even if the patient could die as a result of the treatment being withheld. The high court let stand a ruling by a federal appeals court, which backed the FDA's argument that the government should deny access to unapproved investigational drugs. But the appellate court said Congress could change the law to broaden access to such drugs.

Backers of the Brownback-Watson legislation said the FDA has been far too cautious and moved too slowly in approving new drugs.

"It takes 10 years and $50 million to develop a new drug — that's insane," said Emil Freireich, an oncologist and researcher at the University of Texas M.D. Anderson Cancer Center. He said the FDA must balance its desire to protect public safety with risk-taking, but he added: "There's no progress without risk."

FDA spokeswoman Karen Riley declined to comment on the bill, saying the agency's policy is not to comment on pending legislation. But she said the FDA has a long history of approving the use of investigational therapies, dating back to the 1970s. Some of the larger programs, she said, have enabled tens of thousands of patients with cancer, cardiovascular diseases and HIV/AIDS to receive drugs before they were approved for marketing.

Riley said the FDA can refuse requests to use experimental drugs if they've not been proven safe or if there's not enough scientific evidence to show that the drug would work without exposing patients to more illness or injuries.

"We approve nearly all requests," she said. "What we can't do, though, is require companies to provide the product when a patient and his or her doctor asks for it."

Brownback said too many people with serious illnesses are being refused experimental drugs. He noted that only 650 people qualified for access to investigational drugs from 1997 to 2005, while the country recorded 4.8 million cancer deaths during the same period. And he said the decision to use experimental drugs "should be made by the patient and/or his physician, not a regulatory bureaucracy."

Under current law, Freireich said, the FDA is too powerful. The government's decision to deny experimental drugs to the dying is like "torturing our own citizens" and makes no sense as a policy, he said.

"Cancer patients have a sense of hopelessness," Freireich said. "That's something human beings cannot tolerate. Hopelessness is the worst disease in the world. These patients need some hope."

Anna Tomalis has plenty of hope.

She and her parents began their unsuccessful search for experimental drugs in December, when they learned that Anna had a recurrence of her tumor. And it was inoperable. She has undergone chemotherapy and radiation treatments since September 2005, when she was diagnosed with a rare cancer that affects only 10 to 20 people each year in the United States.

"Finding help for a sick child should be easy, but it is not," said Anna's mother, Liz Tomalis, 43, of Clarksville, Md. She said Anna has been turned away from drug studies because she is not healthy enough.

"For Anna, time is running out," her mother said. "She does not have years to wait for these drugs to become available. She does not have 10 to 20 years to fight for her own cause."

Freireich said that while many terminally ill patients are "crying out" for experimental drugs, it has become increasingly difficult for them to take part in drug studies because drug companies want to use healthier subjects.

"All of these trials are designed to maximize the chance of success," he said. "And so the eligible patients are Olympic athletes. They're completely healthy, they have no side effects, their organ function is normal. They're the right age. They live in the right town. Everything is perfect."

On Wednesday, Anna stood by her mother's side in a hearing room on Capitol Hill as Brownback and Watson announced the introduction of their bill. Her mother issued a plea to Congress on behalf of Anna: "Please help her. She wants to survive. She can survive. She should not have her hope taken away because of a system that does not work for patients."

Standing before the microphones and feeling a little intimidated, Anna said nothing at the news conference. But the little girl who likes horseback riding, reading and watching television in her spare time made her own case in an interview the following day. She said she hopes that members of Congress will let this piece of legislation rise to the top of the agenda and not let it get lost in a sea of bills.

"It would be a bill that would be saving so many people's lives," she said. "I know they must think of bills, you know, as just bills sometimes. But this isn't just any bill."

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