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After years of controversy, the Food and Drug Administration has issued a proposed rule to clarify when manufacturers would have to update product labeling to reflect unapproved uses of their medicines.

In doing so, the agency has left intact a decades-old rule that stated drug makers must update labeling if there is evidence indicating a company intended its medicine to be used off-label, or for an unapproved use. Doctors are free to prescribe a medicine for any purpose, but court rulings have determined drug makers can make statements about off-label uses only if information provided is truthful and not misleading.


At issue, though, has been a regulatory term called “intended use,” which the agency relies upon for determining when a company intentionally markets a drug for an off-label use. Why? In 2015, the FDA proposed a revision that deleted language indicating a company could be liable for having knowledge of an off-label use. That move followed years of concern by drug makers over the threat of prosecution.

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  • Clear as mud, right? I don’t understand. A drug like Alecensa is approved to treatment non-small cell lung cancer patients where the tumor contains the ALK mutation. I have a patient who has a non-small cell lung cancer patient where the tumor as the ROS1 mutation. I know, like every other oncologist knows that ALK inhibitors work well for ROS1 mutations. But it is not FDA approved for ROS1. I do this (it gets paid for) but I am suppose to keep that information a secret from my pharma rep. If my pharm rep learns out my off label use, they are suddenly responsible for….. for what? For collecting data? For providing more data to the FDA? Seems strange. I mean, every expert in ROS1 mutations in lung cancer will tell you that these drugs work and work well. Why can’t the package insert allow use for ROS1 mutated lung cancer? Why is the rep not allowed to talk about it? Or even worse…. why can’t the rep even hear that I am using it?

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