There is a recurring question I simply cannot get out of my mind as I am flying back from ASCO’s annual meeting in Chicago: “What the heck goes on in ‘the little room?’”
The little rooms, my friends, are the sound-proofed spaces that are an intrinsic part of many of the drug companies’ booths on the ASCO convention floor.
From what I can tell, they must be part of another sovereign country.
As a physician licensed in the United States, I am forbidden from going into the little room. And I do mean forbidden. Very pleasant young women and men will forcibly prevent me from entering if I don’t have a convention badge that designates me as a physician from a country other than the United States.
What goes on in these little rooms?
That’s where the pharmaceutical companies can have discussions about off-label and new uses of drugs for the treatment of cancer.
Bottom line, my international physician colleagues can hear about these new drugs for cancer treatment, but I cannot. I am forbidden to have access to that information.
This might give you pause as well:
I was told by one source that the United States Food and Drug Administration (FDA) actually has “secret shoppers” that roam the floor of medical conventions such as ASCO in an effort to trap the drug company representatives into making a mistake and talking about off-label drug use within hearing range of an American oncologist or saying anything that should not be said.
If I ask them a question about an off-label drug, the pharmaceutical representative can ask me if I am an FDA gumshoe. If I was an FDA representative looking for bad behavior, I would have to answer yes. That would call off the “sting.” Sounds to me like a different kind of undercover drug information request.
I kid you not. You simply cannot make this stuff up.
What makes this even more problematic is that I would guess many of the treatments given to cancer patients today by their medical oncologists are actually “off-label.” For the most part, cancer treatment in the United States is indeed “off-label.” But you won’t get much help from anyone outside the little room.
What does “off-label” mean?
When a new drug comes to market, it does so after extensive research in the lab and the clinic. If the drug successfully treats a cancer, the company applies to the FDA to market the drug in the United States. If the FDA approves the drug, then it does so for the specific cancer that was studied, and at the doses and regimens studied in the trial. That then becomes the “labeled indication.”
When a drug is approved, the drug company can then market that drug to doctors, but they must do so only for the approved indication. They are free to discuss how the drug works, how to administer the drug, what the side effects of the drug may be, and so on but only for that specific disease.
But many of these drugs are actively and appropriately used in the treatment of other cancers.
Doctors continue to research the use of the drug beyond its original indication, and they may find the drug works in different cancers or maybe at different doses than originally approved. Then they publish their results in medical journals. But these do not necessarily become “approved uses” since the drug companies don’t or can’t afford to make the investment to go through the FDA process to get approval for that specific new indication supported by published research in peer reviewed journals.
Take Avastin (bevacizumab) for example. This drug is approved for the treatment of colon cancer, lung cancer and most recently breast cancer. Those are “approved indications.”
But Avastin appears to be effective in other cancers as well.
Given the current prominence of brain tumors in the media, I would offer the treatment of brain cancers as another form of cancer where Avastin appears to be effective. In fact, the National Comprehensive Cancer Network’s Drug and Biologics Compendium, which assesses off-label and on-label use of drugs includes brain cancer in its list of appropriate off-label indications for Avastin.
So let’s say I am a doctor who is interested in using Avastin for the treatment of my patients with recurrent glioblastoma multiforme, a very aggressive and lethal form of brain cancer with a very poor outlook.
I come to the ASCO meeting, and go over to the manufacturer’s booth where there are clinical experts who know something about the research on that drug.
I ask the experts from the company, “Tell me about using Avastin in brain cancer. My patient is dying and I would like to help them.”
The quick answer: they can’t answer my question.
So I rephrase my question, “Do you have any information about current clinical trials using Avastin for brain cancer?”
Now the expert can tell me about the trial and the doses of Avastin in the trial, and what other drugs are being used in those protocols.
So, I ask for some scientific literature, perhaps an article that has been published in a leading medical journal.
Absolutely not. Can’t do it. Go find it yourself. That is an off-label indication and we can’t give you any literature on this drug’s use in brain cancer to help you and your patient, even though they are dying.
And, by the way, if I ask the specific question above, the people who talk to me—by law/regulation—have to answer my question out of earshot of any other United States licensed physician, lest they too might hear information about using Avastin in the treatment of their dying patient with brain cancer.
Get the picture????
However, if I am a physician from, let’s say for example, Iran or Iraq, they can take that doctor into the little room and safely tell them all sorts of things they can’t tell me.
Sounds like that little room is part of a sovereign country on United States soil, sort of like an embassy. I’m surprised they don’t have the Marines guarding the entrance. But they’ll just have to rely on the secret FDA police circulating the convention floor to protect us doctors from ourselves.
My government is happy, the foreign doctors are happy, the drug companies are following the rules, and my patient is dying.
I know there are reasons for these rules, and I know there is some effort afoot to try to make them more reasonable. But the scenarios I outlined above are real, and multiplied many, many times over during the course of this recent ASCO meeting and any other medical meeting that occurs on United States soil.
A couple of weeks ago I participated on a program panel hosted by the American Enterprise Institute (AEI) in Washington, DC. The topic was off-label drugs, with an emphasis on oncologists and how they prescribe off-label.
The AEI is a well-known conservative think tank, and they are very concerned about the effects of off-label issues, especially regarding cancer care.
One of their staff, Dr. Scott Gottlieb, has written on this subject and also spoke at this meeting. He described the issues faced by women and their doctors when Herceptin was reported to be so effective in reducing the recurrence of breast cancer in women at high risk.
Dr. Gottlieb believes that the off-label prohibition about talking to doctors prevented many women from receiving this truly life-saving therapy as quickly as possible. He maintains that if the data was so compelling, and if the drug companies had been permitted to ethically spread the word around the country, then lives that have been lost would have otherwise been saved.
I don’t know that I completely agree with Dr. Gottlieb’s contention regarding Herceptin, since there was so much publicity about the drug that an oncologist would have had to be under a rock not to have heard about the stunning results. In fact, I blogged about some of the off-label issues surrounding Herceptin and its use in the United States compared to Europe back in May 2006.
But Dr. Gottlieb has a point, and the little room is one more extension of how the best-intended rules and regulations can result in bizarre situations.
We need to take a very careful look at how we deal with off-label indications and drug promotion through pharmaceutical companies.
Yes, they are not innocent participants. They have drugs they want to sell, and they want to make profits.
But no one should have to face jail time or have the very viability of their company threatened because I asked the wrong question and someone cared enough to provide me information which may save the life of my patient.
With all of the new drugs coming down the pipeline, and the inevitable fact that many of these drugs will have research reports published in highly-regarded scientific journals long before the FDA can approve them, it’s time we figured out how to take the conversation out of the little room and move it into the mainstream.
After all, I don’t like being treated like a second-class, lawbreaking citizen in my own country just because I want to help my patients.