FDA Issues Off-Label Information Guidelines for Industry

Wayne J. Guglielmo

December 27, 2011

December 27, 2011 — The US Food and Drug Administration (FDA) today issued draft guidelines that would permit drug and device makers to provide information on off-label uses, but only if the request for information is unsolicited. An unsolicited request is one not initiated in any form by the drug or device maker or distributor itself.

As a rule, manufacturers and distributors cannot promote a drug or medical device for uses other than those approved or cleared by FDA. But once given the FDA seal of approval, products can be used by healthcare professionals for purposes that are not included in the products approved labeling or statement of intended uses. Such off-label uses or treatment regimens, says the FDA, "may be important therapeutic options and may even constitute a medically recognized standard of care."

The draft guidance covers not only nonpublic unsolicited requests — that is, requests "directed to firms privately through one-on-one communications" — but also public unsolicited requests. According to the FDA, this latter category of request has proliferated in recent years because of the communication revolution brought about by the Internet. But as consumers move increasingly online for publicly available answers to their medical questions, they don't always access sites and electronic forums controlled by makers and distributors, which the FDA says it trusts to respond in "a truthful, non-misleading, and accurate manner."

Instead, says the agency, consumers sometimes access Web sites, discussion boards, and chat rooms that are created and maintained by people other than the manufacturer or intellectual property owner who don't always publish online content that's accurate.

In response to each type of request for off-label information — nonpublic and public — the FDA has set out a separate list of recommendations. For firms' response to nonpublic unsolicited requests, the agency has made the following 7 recommendations:

  • Response information should be limited to the requestor and treated as private, one-on-one communication.

  • Information should be limited to the scope of the initial question.

  • Information should be "truthful, non-misleading, accurate, and balanced."

  • Information should be scientific in nature.

  • Information should be generated "by medical or scientific personnel independent from sales or marketing departments."

  • Information should be accompanied by several documents, including a statement notifying the recipient that FDA has not approved the product for the specific off-label use.

  • The responding firm should maintain required records, including any follow-up questions from the requestor.

For firms responding in public to public unsolicited requests, FDA makes the following 4 recommendations:

  • A firm should respond only to a request about its own named product and is not solely about a competitor's product.

  • The firm's response "should be limited to providing [its] contact information and should not include any off-label information."

  • Those responding on behalf of the firm should clearly disclose their specific involvement with that firm.

  • Responses "should not be promotional in nature or tone."

FDA has given those wishing to comment on the draft guidance 90 days from the date of its publication to do so.