The only way to get such a drug is through a government approved clinical trial. But now some patients, their families and doctors say that clinical trials in terminally ill patients may be killing more people than saving, reports CBS Sunday Morning medical correspondent Dr. Emily Senay reports.
Frank Burrough's daughter Abigail was diagnosed with head and neck cancer at age 18. She died three years later. He blames her death not only on her disease but the way modern medicine develops drugs.
When asked if his daughter realized how ill she was, Burroughs says, "Absolutely. She knew fairly early on that she was in big trouble."
But to Burroughs, it wasn't just neck and lung cancer that led to the death of his 21-year old daughter. He blames the way modern medicine develops drugs.
Abigail was a very open minded, inclusive person, Senay reports. A gifted student, active in her community and at church, Abigail's life was in full swing.
"She had lots of friends, honors in college. She was a very good student. She lived life to the fullest," Burroughs says. "She did all kinds of charity work."
Then the cancer diagnosis. But as Abigail's condition worsened, two promising experimental drugs -- drugs that might have made a difference -- were winding their way through testing, available only to patients who qualified for the clinical trials.
But Abigail didn't qualify. Her cancer didn't exactly fit the criteria for the trials. There was nothing her father could do.
"Cancer drugs that had a really good chance of saving or extending her life," Burroughs says, adding, "we didn't succeed."
Abigail's oncologist at Johns Hopkins University fought to get the medications, says her father.
Of course, no one knows if the drugs would have made a difference for Abigail. But every year, thousands of people with all kinds of diseases enroll in clinical trials for drugs pharmaceutical companies hope to have approved by the FDA.
To do this, researchers enroll a number of patients with the same disease, like certain cancer patients, to take part in a so-called clinical trial.
Typically, half the patients get the actual drug being tested -- the hoped for "cure" -- but half don't. Doctors then watch to see which group does better.
For the FDA, whose job is to protect Americans from drugs that are ineffective or unsafe, to distinguish drugs that work from those that don't, the clinical trial is the gold standard.
Eligibility criteria are strict as to patient age, stage of disease and other medical conditions. Getting an experimental drug outside a clinical trial through so called "compassionate use" programs, even for those at death's door, is in most cases impossible.
For Frank Burroughs, that was exactly the problem for Abigail.
"Before she died and she was battling her cancer she said, 'Dad, if I make it, I wanna devote my life to helping people with cancer and other life-threatening illnesses. Let's start working on ideas.' And little did we know that her struggle was the idea," Burroughs recalls.
If Abigail, her father and her doctor were willing to risk that an experimental drug might be ineffective, or worse, harmful, why should Abigail be denied a last chance?
"We wanna give them a chance at life," Burroughs says. "And they deserve that chance, you know, where there is some significant hope."
After Abigail died, Burroughs quit his job as an engineer and founded the Abigail Alliance For Better Access To Developmental Drugs, a lobbying group that wants to change the rules so that critically ill patients can get experimental drugs they and their doctors think might help.
And he has support. Steve Walker lost his wife to colon cancer. Vickie Doran, her husband Patrick to colon cancer as well. And John Rowe, his daughter Kianna to kidney cancer.
Rowe remains angry. "My kid died. I don't know if either of these drugs would have saved her life, but it sure would have been nice to find out. It sure would have been nice to have a chance. So yes, I'm am (angry)," Rowe says.
Dr. Alan Goldhammer is vice president of PhRMA, the lobbying group for the pharmaceutical giants. He says letting all comers have access to experimental drugs is not only bad science, it's unethical.
"I think by and large, the system does work," Goldhammer says.
Goldhammer adds, "We can't promise a cure for investigational new drugs. And I think there's a, there's very clearly an ethical issue. Are we raising hopes of patients who may be suffering from a terminal disease and they're saying, 'Well, there's a, there's a drug that's in clinical trials. I wanna take this. Maybe it will help me.' Well that maybe is just that, it's a maybe."
The FDA holds the same position and declined an on-camera interview with CBS News. But, the American Cancer Society and other leading groups agree as well: clinical trials are the way to go.
"As soon as we make investigational drugs available to people outside of clinical trials," Fran Visco, president of the National Breast Cancer Coalition says and adds that, "fewer people will want to enroll in trials, the focus will be on making certain more and more people have access to those investigational, unproven, drugs just based on false hope."
Hope, false or otherwise, was all Max Sonnevil had 23 years ago when he was diagnosed with leukemia.
Sonnevil remembers doctors telling him he had three to six months to live. "There was no treatment for my disease," Sonnevil says.
Waiting around for experimental drugs to be approved wasn't going to fly with this entrepreneur. Sonnevil searched for a cancer specialist who was willing to take a risk and wound up at the University of Texas' M.D. Anderson Cancer Center in Houston with Dr. Emil Freireich.
Dr. Freireich, a celebrated cancer researcher, says the FDA's policy is misguided when it comes to fatal diseases.
"The primary problem is that the FDA does not discriminate between the degrees of hopelessness and risk that the patients are willing to accept for potential benefit," Freireich.
As Freireich sees it, the FDA's job should be to protect healthy people with common ailments like headaches or high cholesterol from dangerous drugs.
"The FDA should not deny a desperate cancer patient a treatment that the patient, the physician scientist, the academic institution, the hospital where they work, have all agreed gives him the best chance of improving his lot," Freireich says. "That is something that the government should not intrude in."
What's more, says Freireich, denying half the patients a drug in a clinical trial by giving them a placebo is scientifically unnecessary. If patients do better than what doctors would typically expect, he argues, the drug works.
Freireich believes patients have died because of this practice in clinical trials. "Oh absolutely," he says.
To treat Sonnevil's leukemia, Freiriech used an unapproved drug, then kept on giving him newer ones each time his symptoms came back.
Sonnevil was asked whether he was capable of deciding he should take an experimental drug and if he was willing to take the risk.
"I mean a lot of people when they say, when they told me 'you got three to six months,' you know, get your life in order and a lot of people, that's what they did," Sonnevil says. "They took their three to six months and got their life in order and, and didn't fight. And there's a way to do it. I mean if, and you're not always successful, but you just can't quit."
And Sonnevil, diagnosed with incurable cancer 23 years ago, kept on fighting until he died two weeks ago from a massive infection while undergoing additional treatment for his cancer.
Most of the major institutions, like the American Cancer Society, even PhRMA, which could only benefit from getting more drugs on the market, says the idea of Abigail Alliance, of undoing or doing without clinical trials, could potentially hurt people, Senay reports.
It could delay the best drugs from getting on the market. It could have drugs get on the market that really are not effective at all.
"Life is not risk free," Freireich says. "There will be accidents. People will be hurt. We have to set up a system that prevents people from being hurt if we possibly can. But no insane regulations. We haven't made a law that you can't fly. We haven't made a law that you can't drive."
Goldhammer of PhRMA, disagrees.
"We absolutely cannot compromise the ongoing clinical trial programs," he says. "Because, you know, ultimately, the major benefits to cancer patients are very prompt completion of clinical trials, demonstration that the drugs do, in fact, work, and then getting them out to the patients that are suffering from this debilitating disease."
A lawsuit against the FDA to loosen the rules on clinical trials was thrown out by a federal court, but is now on appeal. Meanwhile, Abigail Alliance forges ahead, pushing for a new, more inclusive, system of drug trials they hope the FDA will adopt.
"It is a mission. Like them, I wish I were not here, but these are the cards I was handed and I hope other families do not have to go through the grief that we have," Rowe says.
Burroughs hopes to fulfill his promise to Abigail.
"What's interesting is that over the last four years of the Abigail Alliance every drug that we've pushed for earlier access to and earlier approval to has now been approved by the FDA," Burroughs says.