Off-Label Communications: What It Means for Stakeholders

Joseph Alvarnas, MD: Hello, and thank you for joining The American Journal of Managed Care^®’s Oncology Stakeholders Summit Spring 2017 Peer Exchange^®. Many changes have been made within the FDA surrounding off-label communication and the management of newer cancer agents and their costs. While innovative trial designs have led to the promise of a boost in emerging agents for the treatment of various cancers, provisions within the Affordable Care Act have had a significant impact on costs and patient care. In this Peer Exchange^®, our panel of experts will provide an overview of the FDA changes and address the promise of increased patient access and a more personalized approach to further ensure patient-centered care. The panelists will also address how the Affordable Care Act will continue to affect the delivery of patient care and coverage.

My name is Dr Joseph Alvarnas, and I’m the director of value based analytics and associate clinical professor in the Department of Hematology & Hematopoietic Cell Transplantation. I also serve as the clinical quality director of the ACT-1 clinic at the City of Hope Comprehensive Cancer Center in Duarte, California. I am also the editor-in-chief of AJMC^®’s Evidence-Based Oncology™.

Joining our distinguished panel to share their perspectives are: Dr Robert Carlson, chief executive officer for the National Comprehensive Cancer Network in Fort Washington, Pennsylvania; Elizabeth Carpenter, senior vice president of Avalere Health in Washington, DC; Dr John Fox, associate chief medical officer for Priority Health in Grand Rapids, Michigan; and Dr Bhuvana Sagar, national medical director at Cigna Healthcare in Houston, Texas. And joining us remotely for this panel will be Dr J. Leonard Lichtenfeld, deputy chief medical officer for the American Cancer Society Incorporated in Atlanta, Georgia. Thank you again for joining us. Let’s begin.

Let’s start talking about changes within the FDA. With respect to off-label communications, Bob, how does the FDA define the scope of off-label communications? What is the level of evidence that can be included in this communication, and how can payers use this information?

Robert Carlson, MD: Well, the FDA broadly defines, or looks at, off-label communication as healthcare economic information, and that may relate to the labeled indication, but the FDA also looks at economic analyses of the use of that drug in that labeled indication, often in comparison to other agents or potentially no treatment. That’s the information that may be of great interest to pharmacy and therapeutics committees and to payers—probably less so to practicing physicians or patients, as such.

And then, of course, there are the off-labeled indications. Those are the uses of the drug for which there is a label, but also using them in circumstances or situations where the drug is active, presumably, but not actually on the label. In doing that, the FDA does have a series of criteria that they look at in terms of what they think might be appropriate off-label communication between the pharmaceutical industry and the provider, payer, or patients. The information is required to be scientifically valid; it cannot be false or misleading. It’s an interesting area, both scientifically and legally, because there are the issues of drug companies’ first amendment rights—freedom of speech, freedom of communication with its stakeholders. And yet, the FDA wanted to be sure that that communication is appropriate—that it isn’t false or misleading—to help to protect the safety of the patient population and to be sure that the agents are used correctly.

John Fox, MD: Bob, how are they vetting? How is the FDA vetting that information to ensure the accuracy and validity of the data?

Robert Carlson, MD: I can’t tell you for sure. What I can tell you is that there’s enough concern among the pharmaceutical industry in that setting and in what is legal for them to do, that there isn’t a lot of that going on. I think there’s, for instance, very, very little information on the healthcare economic information that’s being provided to pharmacy and therapeutics committees by the industry. And that’s because they’re afraid they’re going to get sued by the FDA or by others. I think it’s the same for off-label indications. They’re very cautious about providing information off-label.

The FDA, in their defense, does provide some guidance. It’s primarily draft guidance in terms of what that communication should look like. Those communications, or things that they hand out, are things that should not have been derived primarily by the request of a company—so it can’t be an article that is written and funded by industry, and it also needs to be information that is readily available in the public domain and is the sort of information that you would normally buy or obtain where you get other peer-reviewed literature or textbooks. It can’t be a primary pharmacy drugstore or a bookstore, if you will. It needs to be more in the public domain.

John Fox, MD: I have a concern about the health economics data. I can’t imagine there’s a pharmaceutical company that exists today that doesn’t have a health economics and outcomes research department. They’re all trying to demonstrate the economic benefits of their drugs, and the most common claim is that they’ll reduce other costs. If everybody reduces the cost to the degree that they said, healthcare would be free, I think.

But I’m also concerned with the methodology that’s being used, especially if we have multiple manufacturers approaching us as a health plan making claims. Number 1: how can I compare the validity of the methodology of the findings? And number 2: given that I can’t do that, is there somebody that’s better to do that than a pharmaceutical manufacturer? Should it be the government or somebody else who’s unbiased, and who does that health economics outcomes research? The fact is that companies have these functions today, but I’m not sure that the loosened FDA rules on sharing ER data is going to make life any better or the information any more credible.

Joseph Alvarnas, MD: So, you think there’s an ethical dimension to what we’re talking about?

John Fox, MD: Look, I’ve never seen one of these analyses that didn’t support the drug that was being touted. Everybody is going to embellish the data in the way that makes them look the best, and I’m not saying they’re not people of integrity, but that’s everybody’s desire: to make us look better than, or as good as, we are or as good as we could be.