Regulating Speech About a Drug’s Off-Label Uses

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Scholar argues that FDA should adopt an evidenced-based approach to regulating drug manufacturers’ speech.

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Sometimes the key to effective regulation is a little common sense.

In a new article, David A. Simon, an associate professor at the Northeastern University School of Law, argues that the U.S. Food and Drug Administration (FDA) should end its draconian regulations on drug manufacturers’ speech about unapproved uses for a drug. Simon proposes a sliding scale system where speech that is well-supported by evidence is subject to few restrictions, while less-supported statements are more scrutinized—as opposed to the current system, which bars all speech absent two narrow exceptions.

Using an FDA-approved drug in an unapproved manner is commonly known as an “off-label” use. Simon notes that speech by manufacturers that promotes off-label use has traditionally been condemned by FDA because of the fear that this speech will lead to a rise in riskier, unapproved drug use. In addition, Simon suggests that, if drug companies were freely allowed to advertise off-label uses, they would have little reason to conduct the expensive clinical trials and other research needed to gain full FDA approval.

On the other hand, Simon contends that off-label drug uses also fill a vital need in many circumstances. Simon explains that off-label use may be the only available treatment in some instances and can even represent the standard of care accepted by the medical community. Simon emphasizes that, in these situations, FDA’s stringent rules may decrease the chance that a doctor will prescribe a drug off-label to a patient who could benefit.

Simon notes that courts have also looked down on these rules. In the past, FDA had argued that statements about off-label uses, even if truthful, could still be seen as an attempt by drug manufacturers to falsely brand the drug as approved for that use. Simon explains that courts rejected this argument because FDA, in not banning off-label uses outright, had already acknowledged that these off-label uses could be beneficial.”

Simon argues that the two existing exceptions to the ban on off-label speech fall short. The first exception allows drug manufacturers to distribute information about a drug’s off-label uses only if the manufacturer receives an unsolicited request for information. Simon argues that this is too restrictive, noting that manufacturers also face stiff penalties if they solicit these requests.

The second exception allows for manufacturers to distribute information about off-label uses as long as this information is kept separate from promotional materials and complies with requirements surrounding the trustworthiness, content, and form of the information. Simon argues that these requirements are often too vague. Furthermore, he suggests that the requirements also force companies to provide information in a cumbersome manner that is not ideal for effectively reaching doctors.

To replace the current system, Simon proposes that FDA should partner with the Centers for Medicare and Medicaid Services (CMS) to use drug compendia, which are summaries of drug information. These compendia would assign a rating to different off-label uses based on how much evidence supports a particular use. The higher the rating, the more regulatory flexibility FDA should grant drug companies to discuss that off-label use.

Simon notes that this system would allow FDA to tailor regulations more closely to the risk presented by the drug manufacturer’s speech. He emphasizes that this proposed approach will place sufficient limits on drug manufacturers’ speech to still encourage them to conduct clinical trials and seek full FDA approval for popular off-label uses.

Simon contends that his proposed approach brings together the efficiency of private actors with the safety of public oversight. He notes that the drug compendia at the heart of the evidentiary ratings will typically be compiled by private experts but subject to public regulation regarding their accuracy. Simon argues that this approach represents a cost-efficient yet accurate way to ensure FDA regulations on speech about a drug’s off-label use are rooted in evidence of the use’s effectiveness.

Simon acknowledges, however, that drug compendia can at times be biased and unreliable. To solve this problem, Simon proposes FDA and CMS take a stronger regulatory approach to compendia. If the compendia publishers show that their systems for analyzing evidence and identifying potential biases meet FDA norms, CMS could grant the compendia “recognized” status.

As a result, this proposal will not only lead to more effective regulation of speech about off-label drug use, but it will also improve the quality of drug compendia, Simon concludes.