The U.S. Food and Drug Administration (FDA) recently released two draft guidance documents and a memorandum related to off-label communications and the FDA’s power to regulate such communications. The publications are the FDA’s response to years of conflict between drug makers, which contend they have a first amendment right to communicate off label or information that is not contained on medication labels, and the FDA, which interprets current law to prohibit such communications under misbranding legal theories. This ongoing debate is grounded in the drug industry’s assertion that these communications are protected free speech versus the FDA’s duty to protect public health and well-being, which has arguably been impacted by the recent Amarin settlement.

The memorandum

Historically, the FDA has managed off-label use by regulating corporate marketing, and in the memorandum, the agency asserts its statutory authority to regulate off-label communications, regardless of whether they are truthful, misleading, or false, in order to protect consumers. Included in the memorandum is a section in which the FDA addresses why it has declined to adopt alternative approaches that include eliminating or capping off-label drug uses or communications, stating that these approaches do not “take into account the public health interests behind allowing health care providers and patients to work to determine the best treatment options for each patient in specific circumstances.” Read as a whole, the memorandum reinforces the FDA’s authority to regulate off-label communications while limiting the types of communications and types of speakers being regulated.

The FDA notes throughout the memorandum that it does not prohibit all communications about off-label uses. For example, the presentation of truthful and nonmisleading scientific information about unapproved uses at medical or scientific conferences is acceptable. On the other hand, all communications that are not supported by “objective and scientifically valid evidence” are deemed misleading and are subject to FDA regulation. Further, the FDA can accuse a firm of misbranding when its off-label communications provide evidence that a drug is intended for an off-label use. However, the FDA also notes that the scope of the regulation is narrow and is focused on “firms who actually control the distribution of the products,” not independent healthcare providers and researchers.

Consistent communications draft guidance

The first draft guidance addresses communications that contain information consistent with, but not found on medical labels required by the FDA. The FDA acknowledges that drug manufacturers have an interest in communicating data and information about the approved uses of products that are not contained in the products’ FDA-required labeling. However, the FDA warns that these communications must be truthful, nonmisleading, and grounded in fact and science. To address both of these interests, the FDA has identified three factors indicating that a communication is consistent with a product’s FDA-required labeling:

  • The communication does not make representations or suggestions relating to a different indication or patient population, or that conflict with the limitations and directions on the labeling.
  • The representations or suggestions in the communication do not increase the potential for harm relative to the information in the labeling.
  • The directions for use in the labeling enable the product to be safely and effectively used under the conditions represented/suggested in the communication.

Examples of communications that the FDA would consider to be consistent with labeling include information obtained directly from patients about the effects of a product when used for its approved indication and information about the long-term safety of products that are approved for chronic use.

Payor communications draft guidance

The second guidance addresses the communication of healthcare economic information (HCEI) to payors and similar entities. The FDA acknowledges that payors have an interest in receiving HCEI from drug and device makers about medical products not yet approved by the FDA. In an attempt to clarify the scope of permissible HCEI communications, the guidance states that where HCEI relates to an approved indication and is based on competent and reliable scientific evidence, the FDA will not consider such information to be false or misleading.

HCEI that relates to the disease or condition, manifestation of the disease or condition, or symptoms associated with the disease or condition in the patient population for which the drug is approved is deemed to “relate to an approved indication.” This includes information relating to duration of treatment and dosing. Conversely, HCEI does not relate to an approved indication if it addresses treatment of a disease where the drug is approved only to treat a symptom of the disease, or the information is derived from studies of patients not within the approved patient population. Last, HCEI will meet the standard of deriving from competent and reliable scientific evidence if it has been developed using generally-accepted scientific standards, is appropriate for the information being conveyed and yields accurate and reliable results.

What’s next?

The FDA is soliciting comments on this new guidance until April 19, 2017, while it makes its final determinations. After it has collected all necessary information, the FDA will release its final rules. In addition to the comments received, the final rules may also be shaped by the election of the new FDA commissioner, whom President Trump has not yet nominated.