Dr. Mitchell Seymour, November 20, 2013

Seymour head shotWhen I mention off-label drug use to my non-clinical colleagues or to the public, I often face confusion, winces, or leery looks. “Isn't that dangerous?” “How can that be allowed?” Misperceptions exist that off-label use is risky and deceptive. One goal of Cures Within Reach is to educate on the prevalence, impact, and value of repurposing “old” or approved drugs for new uses. Our audience is broad, but we accept that challenge with both passion and purpose.


Among health care providers, there is broad understanding that off-label use is part of innovative clinical care. Insurance companies and other payers like Medicare/Medicaid may also reimburse for this off-label use, even if this use is not FDA-approved. In these cases, the payers have decided, like the treating physician, that the off-label use could be clinically beneficial and fiscally sound by limiting disease severity or duration.

So, if doctors can prescribe drugs for off-label use, and insurance sometimes pays for off-label use, how can drug companies get in trouble for off-label promotion? This has certainly been a hot topic. Recently, the US Government took a powerful stance against Johnson & Johnson (J&J) and Janssen Pharmaceuticals, a J&J subsidiary. J&J’s Risperdal® was initially FDA-approved to treat schizophrenia. However, according to criminal charges, sales reps for Janssen Pharmaceuticals urged physicians to prescribe Risperdal® to treat symptoms such as agitation, hostility, and confusion in elderly dementia patients. Also, sales materials apparently emphasized these off-label indications, but lacked detail on the FDA-approved use for schizophrenia. Furthermore, company representatives received incentives for promoting the drug's off-label uses. This November, The US Justice Department revealed that J&J has agreed to pay more than $2.2 billion to settle criminal and civil claims related to these practices – a landmark settlement, indeed.  

Through these events, both industry and FDA learned vital lessons that strengthened education and enforcement of federal regulations on drug labeling and promotion. However, these events can erode public and patient trust and further cloud public understanding of repurposing and its role in biomedical innovation.


The knowledge that doctors use to prescribe off-label can be based upon accumulated clinical experiences. Ideally, this knowledge comes from well-designed clinical trials that comprehensively assess safety and efficacy. Cures Within Reach knows that the drug/biotech industry (including J&J!) can be vital partners in this repurposing research, providing free drug or other resources to physician investigators for use in their own repurposing trials. Also, companies can grant FDA access to their Drug Master File to support the review of Investigational New Drug applications for repurposing clinical trials. While an investigator can perform a repurposing clinical trial without industry help, this partnership can be a great asset on many levels.


The recent headlines with J&J can add to public misperception that off-label use is deceptive. However, these off-label promotional practices are now the exception rather than the norm, and FDA, industry, and physicians have learned vital lessons. The recent headlines provide Cures Within Reach an opportunity to clarify what is valuable about repurposing research, and to clarify its distinctions from industry-driven off-label promotion. With patient safety in mind, the alliances between industry, physicians, and physician investigators are improved by greater oversight and scrutiny, and these partnerships are vital to advancing our understanding of the full value of FDA-approved drugs.  

Read all Regulatory Roundup posts by Dr. Mitchell Seymour


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