THERE'S A BETTER WAY
My Life's Not FDA-Approved
Why do I have to die for the sake of government rules?
I'm a mouse running in a maze. That's what it's like to be a cancer patient today. I've been one for nine years, so I know. Most cancer patients die, or get better and go away. Few of us hang around inside the Food and Drug Administration's death factory long enough to go up its blind alleys and learn firsthand that America's search for cancer cures has more to do with "research" than the patients' health.
It's up or out in the cancer world, and only a few stick around to tell stories. I call myself a "lab rat" and my doctors do not disagree. Here's my story.
I have something called metastatic soft tissue sarcoma. It's rare, but every oncologist in the civilized world knows I'm a goner if I don't get lucky. A few thousand cases are diagnosed each year; an equal number die. There's no cure. For about a third of us chemotherapy can "buy some time," but at a terrible price. Generally, regular surgery is the way to stay alive. The cancer grows slowly, and the tumors can be "resected" as they appear. Eventually, you have so many that surgery is no longer possible.
That's where I am. My only hope is a drug that might shrink them.
The good news is that there is one out there called ET 743 that might help me. The bad news is that I'm told I can't get it without spending the next six months making weekly trips from Boston to San Antonio, where an FDA-approved clinical trial is being held.
When I think of the number of trips to and from San Antonio I feel like an out-of-shape climber standing in front of Mount Everest. I'm tired: 50% of my lung capacity has been removed. It's hard to breathe. I have a husband and five kids to love and take care of. Two days of travel every week is going to take every ounce of strength I've got left. The financial cost of the travel would be breathtaking even if I had three lungs. Excellent doctors here in Boston have experience administering this drug. Why can't they give it to me? Why must I lay down what's left of my health, my treasure, and my family life to get it?
Johnson & Johnson has the U.S. marketing rights to ET 743. It's been approved by the FDA for trials in cancer patients who have failed other therapies. It's been given to hundreds of patients in U.S. clinical trials over the past few years. It's been shown to be safe. It's the only drug in the world that has had significant success with sarcoma patients.
Legally, the company could obtain a "compassionate use" waiver for me from the FDA so I would not have to commute or move to San Antonio. It costs some money and takes some time, and I've offered to pay the cost. Pharma Mar, the tiny Cambridge, Mass., company that had the original U.S. rights to ET 743, used to allow compassionate use of the drug. Compassionate use is also available in Europe. Johnson & Johnson just says "no."
Why? FDA rules. ET 743 is now being tested in San Antonio. Any outside use of the drug that cannot be monitored directly by the doctors in charge of the test could "taint" the whole test, if the patient were to experience an unpredicted symptom. Even if the symptom were minor and seemingly unrelated, like feeling dizzy in an overheated car, questions could be raised about the drug, jeopardizing its approval. The FDA could require more tests, costing Johnson & Johnson millions more in testing costs and delays and hurting its reputation with the FDA.
It's not hard to understand the company's problem: They're running numerous drug trials and they don't need trouble with the FDA. But if you're me, you wonder what the FDA is thinking. I'm dying here; I'm a citizen and a taxpayer. Why must Johnson & Johnson be paralyzed by the prospect of getting in trouble with the government if it gives its drug to dying people? The drug has already made it through several phase one trials and shown itself to be safe.
The government wants proof of efficacy before it will allow me to take this drug outside of an approved trial. But the "proof" is years away and I need the drug now. It's safe. It might work. Johnson & Johnson would let me have it if they could do so without the threat of a government hassle. But they're so caught up in the FDA web that the life of an individual patient has no importance whatsoever.
Without ET 743, I'm a dead woman walking. Five kids are going to wonder why they're left without a mother.
Won't somebody help me get this drug?
Mrs. Bacon lives in Massachusetts.
