Evidence From Drug Manufacturers and Patient Access

Joseph Alvarnas, MD: So, what does that mean? We talked about how these were not necessarily designed for physicians or for patients, but it seems to have some implications for physicians and for patients. How do we navigate that? How do we reconcile that tension?

John Fox, MD: It’s interesting. I think that years ago, we relied heavily on manufacturers to provide that information. I’ll call out Bob. Bob’s organization, the NCCN, has done an incredible job of making information readily accessible, even to people like me at a health plan company that has been vetted by experts. I don’t know if we need that information directly from a manufacturer anymore. We might need some nuances about safety and drug administration—that’s not coming from a sales person—but I think there are enough sources of information today that we don’t have to rely on the industry for that information.

Joseph Alvarnas, MD: You’ve actually raised a different question, which is what level of evidence should the manufacturers share with the physician to help them make a more informed decision about using a new drug?

John Fox, MD: Bob, that might be a better question for you, because I don’t see patients.

Robert Carlson, MD: If we stay within the off-label versus on-label sort of scenario, there are a lot of concerns about what level of information should be available. And by that, I mean that if you’re talking about a new drug—you had an on-label indication—there is going to be a fair amount of information there, and there is a lot of reason that the company will have good data and good information and that’s how they got the label. On the other hand, there are uses of those same products off-label for which the level of evidence is every bit as good. So, unapproved does not mean unproven and you could have levels of evidence that are every bit as good that are off-label.

In fact, I’ve heard officials from the FDA state recently that there are a number of agents—especially older agents that are off-patent for which they believe that the current label is inadequate—and that there are data supporting the off-label indications that are at the level and quality that, if there was someone who had a vested interest in getting a labeled indication, they could do so. The challenge is, with the older drugs that are less expensive off-patent and so on, that there’s really no one that has a strong motive to provide the high-level data that would then support the use of those drugs. So, there is a lot of bias here in terms of what agents, and for what indications, are going to have high levels of information. It’s even collated, put together, and then put forward to the medical community or to the Food and Drug Administration.

Joseph Alvarnas, MD: So, you’ve raised some interesting considerations, because we have a motive gap—we have a label gap. How does this affect patient access?

John Fox, MD: Well, I can speak for our health plan.

Joseph Alvarnas, MD: I think that would be helpful.

John Fox, MD: It varies state-by-state. In our state, we’re required to cover FDA-approved drugs, even drugs that are off-label or that would be considered off-label, as long as they’re listed in a compendium or the manufacturer or the physician can produce 2 peer-reviewed articles supporting that use. I think the fact that this is not on-label, at least in our state, hasn’t precluded access to a drug. What precludes access to a drug is non—evidence-based applications. I’m not sure that this will change anything because at least we’ve never been in a position to deny access for a therapy simply because it’s not on a label. And I think that’s where the NCCN has helped level set things, and most health plans today will cover that as long as it’s in the compendium—as will Medicare.

Robert Carlson, MD: One of the challenges here is peer-reviewed literature. And we all know that there’s good peer-reviewed literature, there’s awful peer-reviewed literature, and there’s everything in between. So, having a therapy in the peer-reviewed literature does not necessarily mean it’s an appropriate therapy, even if that study shows advantage, because it may be poorly conducted, poorly analyzed, not have a meaningful endpoint—all kinds of reasons that that peer-reviewed literature could be poor. And that’s why I think we really do need to look at the totality of evidence and understand how people who are really experts within that specific domain or disease interpret the quality of the peer-reviewed literature.

John Fox, MD: Having said that, I don’t know that anybody has ever used that route to get access to a drug. If they can’t find something in the NCCN guidelines to use, they’re not trying hard enough. I don’t know that that’s a legislative requirement, and I don’t know that it’s ever been successfully applied.

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