What Does the FDA’s New Off-label Guidance Mean for Pharma?

The Food and Drug Administration (FDA) primarily functions under the auspices of the Federal Food, Drug and Cosmetic Act. The Act allows the FDA to ensure that drugs are neither adulterated nor misbranded. The FDA has traditionally held the view that the off-label promotion of a drug constitutes misbranding of that drug. “Off-label” uses are those that have not been previously approved by the FDA.

Some argue that non-fraudulent, non-misleading claims by a manufacturer regarding the off-label uses of a drug should not constitute misbranding of the drug since it prevents contributions to the “marketplace of ideas.” The Supreme Court has consistently held that the cure to “bad information” is not less information, but in fact more information. Accordingly, while the FDA hopes to prevent the spread of bad information regarding the uses and appropriate treatments to diseases by preventing pharmaceutical companies from discussing these uses, the Supreme Court may disagree with such an assessment.

Considering this disconnect between the FDA and various state and federal jurisdictions, the FDA released a guidance in March 2014 to address this issue. However, despite the guidance, the metes and bounds of off-label discussions remain unanswered and have had a particularly chilling effect on conversations via social media.

The Off-label Argument

According to the FDA’s own website, the agency is “responsible for protecting the public health by assuring the safety, effectiveness, quality, and security of human and veterinary drugs, vaccines and other biological products, and medical devices.” In the interest of protecting public health they hope to prevent undue influence on prescribers by Pharmaceutical Companies. They hence limit these Pharmaceutical Companies to only promoting their products based on indications that have been previously approved by the FDA. However, it is still legal for healthcare professionals to prescribe pharmaceuticals for uses that have not been approved by the FDA as it is simply deemed to be the practice of medicine.

Pharmaceutical Companies have, for over a decade, argued that speaker-based restrictions to off-label discussions constitute an infringement on First Amendment rights and is unlikely to withstand constitutional scrutiny. They argue that since small molecule pharmaceutical companies and biopharmaceutical companies (collectively, “Pharmaceutical Companies”) typically fund trials for multiple indications of their product, they are uniquely positioned to best communicate the uses of the product, but are limited in the ways to convey that information without serious legal, regulatory and administrative repercussions.

Lack of Guidance

In light of court decisions protecting “pure speech,” the FDA has provided safe harbor to Pharmaceutical Companies that engage in “scientific discussion.” Scientific discussion may include the discussion of off-label indications as well as unsolicited requests regarding off-label questions. However, the fuzzy boundaries surrounding exactly what constitutes scientific exchange and what is deemed to be unsolicited off-label information is currently amorphous. As a result, manufacturers are afraid to overstep their bounds for fear of incurring massive fines, civil and criminal penalties and/or negative publicity—a chilling effect on free speech.

Due to the lack of guidance, Pharmaceutical Companies have effectively been working in the dark. Efforts to abide by the vague FDA guidelines have resulted in repeated incidences of violations where the broken rule was evident only after a misstep had been made. In light of several Supreme Court and appellate court cases, the Medical Information Working Group (MIWG), an industry interest group initially comprising seven members and later growing to 11 companies, submitted citizen’s petitions in 2009 and 2011 and asked for more specific guidance to aid in compliance (Figure 1).


FDA Response

In 2009, the FDA issued a guidance titled Good Reprint Practices for the Distribution of Medical Journal Articles and Medical or Scientific Reference Publications on Unapproved New Uses of Approved Drugs and Approved or Cleared Medical Devices. The FDA attempted to provide its “current thinking regarding ‘Good Reprint Practices’ with regard to the distribution by a drug or medical device manufacturer (or representative) of medical journal articles and scientific or medical reference publications (referred to generally as medical and scientific information) that discuss unapproved new uses for approved drugs or approved or cleared medical devices marketed in the United States to healthcare professionals and healthcare entities.”  However, since 2009, there have been a slew of legal challenges that have since upended the FDA’s narrow and amorphous view on the issue of sharing off-label information. In light of Supreme Court and appellate court decisions—and the aforementioned MWIG citizen’s petition—in February 2014 the FDA provided an updated guidance called Distributing Scientific and Medical Publications on Unapproved New Uses—Recommended Practices. The new guidance is intended to clarify the agency’s position on manufacturer dissemination of scientific or medical reference texts and clinical practice guidelines (CPGs) that include information on unapproved new uses of the manufacturer’s products.

What Does the Guidance Say?

The guidance indicates that information must be distributed independently of any type of promotional material and should come with a “prominently affixed and clearly displayed label” stating that the off-label indication is not FDA-approved and an explanation of the currently approved indication. Figure 2 below describes the characteristics of the types of publications where the FDA will allow information on unapproved uses of products.


Impact on Social Media

Social media has been gaining in importance. However, despite a 2014 IMS study indicating that half of the top 50 pharma companies have a social media presence, pharmaceutical companies are hesitant to engage patients and caregivers on product claim conversations via social media. One of the reasons associated with this reluctance to communicate using social media is the fear of inappropriately engaging in off-label discussions, considering the corresponding lack of guidance on the topic.

The FDA has recognized the paucity of guidance and has recently tried to provide additional draft guidances. The recent spate of guidances indicate that the FDA is willing to expand its definition of scientific exchange in light of the Supreme Court’s consistent position that blanket bans on communication are a hindrance to public discourse. However, the FDA still has a per se ban on any off-label promotion to protect the public health.

FDASIA requires that the FDA release its social media guidance(s) by August 2014. As a result, the pharmaceutical industry should expect more detailed guidelines on social media to emerge over the next several months. Interestingly, the FDA has asserted that it has already met FDASIA’s requirements, according to Regulatory Focus.

Crystal Ball

Ultimately, Pharmaceutical Companies will continue to push the FDA and the court system for clarification on the issues. However, early indications are that courts will consistently abide by the standard that product claims that are neither “false nor misleading” should not be banned. Accordingly, claims that are neither “false nor misleading” may eventually be advertisable via channels including social media.

  • McKenzie Noblitt

    McKenzie Noblitt received a Bachelor German Language and Literature from UNC-Chapel Hill in 2012. She will receive her MS in Biomedical Writing from the University of the Sciences in Philadelphia in May.


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