Sunil John and Jon Gonzales contributed to this post.shutterstock_171463343.jpg

To label or not to label? That is the question—or rather, one of the many questions that those invited to the FDA’s much anticipated public forum on off-label communication tried to come to grips with when tackling difficult questions about the topic. 

It would be easy if lives weren’t at stake, but they are. That point was quickly brought to light at the forum: The 50-plus speakers—including healthcare professionals, patient advocates, payers and insurers, and academics—delivered impassioned speeches to industry representatives during the two-day session, which started the day after the U.S. presidential election.


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At a glance, the labeling issue seems incredibly straightforward: If you believe that your product will be safe and efficacious in a specific patient population, you should run a label-enabling clinical trial so that you can promote this usage “on label” once it’s approved. That’s the ideal approach. Although that process works in many instances, in too many others, it’s neither straightforward nor fast enough. Here are a few of the most poignant examples from the forum:  

  • Pediatric cancer: According to the St. Baldrick's Foundation, only three new drugs have been approved in the last 20 years that were specifically developed to treat children with cancer. Part of this lack of drug development and label approval is due to the many challenges in completing label-enabling clinical trials in pediatric populations with relatively small numbers of patients and heightened safety implications and developmental considerations. In the meantime, nearly 2,000 children die from pediatric cancer every year, according to the FDA. 
  • Targeted cancer therapies: Many of the investigational and new therapies from the robust oncology pipeline are targeting key signaling pathways or growth mechanisms of cancer cells. Often, these therapies can provide efficacy across multiple distinct types of cancers; however, the feasibility of performing label-enabling therapies for each distinct disease type is unreasonable or can’t be performed in a timely manner. (This is especially true in small, hard-to-reach patient populations.)
  • Standard of care: In some cases, the standard of care or best treatment option is for an off-label use of an approved therapy. During the forum, Dr. Doyle Stulting from the American Society of Cataract and Refractive Surgery pointed out that treating his patients with an antibiotic is the standard of care after eye surgery, and he’d be sued for malpractice if he didn’t prescribe antibiotics for his patients, even though antibiotic use is not, nor will it be, an approved, on-label indication for treatment after eye surgery. Other speakers also pointed out that off-label treatments can often be found in treatment guidelines drafted by physician associations: According to the Journal of the National Comprehensive Cancer Network, 64% of all of the regimens in breast cancer patients were considered off label but are within the recommended guidelines by the National Comprehensive Cancer Network.

In these and many other examples, the timing is critical. With patient lives at stake, the healthcare industry is struggling to find a way to balance a rigorous regulatory pathway that keeps patients safe (“to label”) while also allowing for the influx of available scientific information that can potentially save lives (“not to label”). Wherever the FDA lands on this question, an important distinction that the industry and the public should keep in mind is that communicating off-label information is not promoting off-label usage. We believe that the former is necessary, and we know that the latter is illegal.

The FDA is accepting written public comments through Jan. 9, 2017. Here’s where you can find more information on the FDA forum or access the recorded webcasts.


Topics: Sarah Jarvis, FDA, off label, Sunil John, Jon Gonzales, on label, off-label communication