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M-06: Analysis of FDA Guidance of Off – Label Medical Product Advertising

Poster Presenter

      Kirolous Makarious

      • PharmD Candidate
      • Touro College of Pharmacy
        United States


With the growing population who have inexplicable disease states, prescribers have sought off-label drug use. It is reported, 73% of medications prescribed for an off-label use had limited scientific data. Therefore, the objective of this study is the review and analysis of FDA off-label guidances.


For this study, we analyzed approximately 47 FDA guidances from 1998 - 2017 that pertained to the regulation of medical product communications for unapproved new uses of approved drugs detailing how they enforce these regulations, and who may discuss unapproved new uses of approved drugs.


From the 47 guidances, 5 specifically pertain to off-label advertisement. We critiqued these 5 guidances and have identified where further clarity is needed. Our results indicate that FDA currently restricts data outside the realm of the medical product’s approved labeling. Guidances address several topics ranging from first amendment rights to distribution of scientific articles that discuss off-label use. Within said guidances, there is a tone of ambiguity leading to miscommunication between firms, prescribers, and patients. The FDA suggests that off-label communication for medical products should not be “false or misleading,” and should be supported with strong scientific data. However, the FDA fails to identify what is meant by “false or misleading,” and what studies are acceptable as scientific evidence. Furthermore, the guidances express conditions under which a manufacturer can disseminate off-label information without being considered as evidence to market the product for an unapproved use. Thus, the definition of evidence of intent is unclear. Accordingly, the FDA states that any evidence of intent will be considered misbranding according to the FD&C Act. Due to the obscurity of information in the guidances, industry is left at a crossroads of what is lawful in regards to the dissemination of off-label medical information. The chief concern of the FDA is that public health may be compromised if a firm communicates an unapproved use that has not been deemed safe or effective. The FDA recognizes the value of drugs being used as off-label for patients with no other treatment options. Nonetheless, there is substantial evidence that depict off-label medication as having a direct correlation with patient harm, fraud, and waste of health care resources. On the other hand, manufacturers believe that public health may be compromised if a company is not permitted to communicate truthful, non-misleading and clinically proven information, even if unapproved.


While current FDA off - label advertising guidances detail regulatory compliances for pharmaceutical companies by stating medical product advertisement must be labeled as, “truthful, balanced, non-misleading, and non-promotional scientific or medical information,” the evaluated guidances adopt the tone of being ambiguous. Unfortunately, this ambiguity breeds miscommunication. Miscommunication then leads to additional risks and potential harms, which may lead to poor patient outcomes. Regrettably, there have been several court cases as well as rulings that hinder FDA’s path to regulate off-label communication which might deprive manufacturers from being accountable for off-label promotional expression, leading to harmful outcomes and more importantly, posing as a major health risk to the general public. The pharmaceutical industry has long awaited clarity from the FDA in regards to off-label advertising of approved medical products. The FDA held a meeting in November 2016 to re-examine its rules, and policies relating to manufacturer communication regarding unapproved uses of approved medical products. In January 2017 the FDA released a memorandum, in which they clarified that in order for a medication to achieve, “general recognition of safety and effectiveness, there must be the same quality and quantity of scientific data necessary to support the approval of an NDA, including substantial evidence consisting of adequate and well-controlled clinical investigations that establish the drug as safe and effective.” This was in response to a study published in 2015 that showed adverse drug events were substantially increased when medications were used for off-label purposes without the reinforcement of sound scientific data. While policy changes may not be immediate, the public meeting and memorandum show the willingness of the FDA and industry to put the best interest of the public as priority, and resolve the continuous concern of off-label advertising.