Considerations With Off-Label Communication

Leonard Lichtenfeld, MD: The question about off-label communication between manufacturers and physicians is certainly not new. It has been around for a long time, and there’s been a lot of discussion as to whether or not manufacturers should be able to educate physicians regarding indications that may not have been approved by the FDA. The FDA has been pretty tough on that rule. I’ve personally been in situations where I’ve gone to meetings, and I walked up to a pharmacy display at a booth at a meeting, and I asked a question and they said, “We can’t talk about that.” I said, “Why can’t you talk about it?” And they would say, “Because, the FDA has people walking around the floor of the meeting to see if we violate the rule, and there have been problems in the past with other companies.”

I happen to write a blog, and for one of my blogs a couple of years ago, I was trying to find something interesting to write, something off the beaten path, about ASCO: the oncology meetings. A couple of years ago, the blog was, “What goes on in the little room?” Because, all over the conference floor, there were these little rooms—sometimes with a guard or somebody else standing outside—and I, as a US licensed physician, couldn’t walk into the room. Well, the answer is, these were doctors, oncologists, from around the world who were not subject to that guidance, and they could walk in the room.

So, again, it’s not something that’s new. It has been a source of concern. And now, it’s coming to fruition. When I say fruition, it’s in the sense that more people are talking about it and that the FDA is discussing it and trying to figure out what the rules might be to allow that to happen. The obvious question is, “Is that a good thing or a bad thing?” I personally don’t know the answer to that, because you can’t have unfettered communication, at least in my personal opinion. You can’t have everybody out there doing everything they want whenever they want.

On the other hand, I come from an era where most of the indications in cancer treatment for those drugs were off-label—not so much today. The targeted therapies and immunotherapies are pretty restricted, and that’s because payers restrict them and there are other factors to consider—the cost, what have you.

But I think we need to start opening the door a little bit to allow some communications that might not exactly fit a label, but where there’s reasonable information that the company may have that could be provided. Of course, whenever that information is provided, it has to be done in a balanced way. It can’t be, “This is the greatest thing we’ve ever seen!” “It’s obviously a great new idea, why don’t you try it?” No, it has to be, “What do we really know?” “What can we really expect?”And “How are we going to implement that for practicing physicians and other healthcare professionals?”

If we talk about the ethical considerations regarding off-label use of medications or FDA-approving communication regarding off-label use, again, we have to be very careful that they be done in an appropriate way. I’m not so sure I can define that easily. That’s obviously a source of discussion, perhaps concern, and contention, because, like everything else, there’s a tendency to try to say, “Use this, try it, it’s good, it’s great.” But there also has to be that balance. You always have to know about the side effects, the harms, and the issues—how does it really work? What’s the evidence? What does the evidence show us? So, the ethical concerns are very real and have to be addressed.

The question about patient access is a very real one, and we’re facing that right now because, as our world expands, as we begin to understand about targeted therapies, and even immunotherapy as time goes on—we know something now, but we’re going to learn even more. We’re going to find patients who are candidates for drugs that don’t meet the label, and of course those drugs are very expensive and there are huge barriers, not only for the patients but also for the health professionals, the oncologists who want to prescribe those drugs. So, we do a genomic test. We get an abnormality. It may work—we don’t know if it works, but, unfortunately, when a patient failed other treatments, it may be their last opportunity. So, how do we get that drug to that patient?

The payers are not exactly opening their arms and saying, “It’s okay.” I’m not necessarily criticizing them for that, but in reality, this is a very real question. I personally think that we need to be more aware of these opportunities, of these situations. I think we have to be willing to share our data. I think the FDA is indicating an openness to evaluating that information—to giving us the clues and the guidance as to how we should go forward with some of these newer therapies when they’re clearly off-label, they’re clearly expensive—and we need everybody’s help to get this done the right way.