In an online article, Peter Pitts, a former FDA associate commissioner, discussed the topic of off-label product claims by pharmaceutical companies. Off-label use in this case refers to using a medication for an intention other than that which the FDA approved it for. This is in fact a common occurrence, particularly in the case of cancer drugs, which are often targeted at specific mutations, even though they may be approved for a disease in general. The article did not address off-label use specifically in oncology, but instead took a more general approach. While doctors, particularly in the US, may in fact prescribe drugs for an unapproved condition (i.e. “off label”) if they feel the benefits outweigh the risks, pharmaceutical companies have shied away from promoting this, perhaps for fear of incurring fines or other regulatory actions from the FDA.

Views are changing

Based on Pitts’ claims, it appears this is beginning to change, and the FDA seems ready to more thoroughly examine this topic. While not a complete authorization of off-label use, the FDA recently offered a proposal that would allow “pharmaceutical companies to present research published in peer-reviewed journals that goes beyond the information provided in the FDA label.” This information could be shared with both physicians and patients, and is intended to enable them to make a more informed decision by making data available that might not otherwise be.

A point the article makes is the distinct difference between “off-label communications” (dissemination of scientific data) and “off-label marketing” (sharing information with the intention to impact sales). The FDA’s proposal is explicitly aimed at the former. Marketing would still be restricted as always; the intention is to allow increased sharing of scientific data.

It is unclear what effect if any this proposed policy might have on the use of off-label drugs, particularly in oncology, where it is already somewhat widespread. The hope is a twofold one: first, that by releasing more information, both in a timely and responsible manner, clinicians and patients will be better equipped to make informed treatment decisions, and second, that drugs that have proved to be beneficial “off-label” will be able to make it “on label” that much faster.

The full article can be accessed here.