Leading U.S. pharmaceutical and medical device manufacturers have filed a citizen petition with the Food and Drug Administration (FDA) calling on the agency to clarify its regulations and policies on off-label uses of drugs and medical devices. Filed on July 5, 2011, the petition discusses the recognized value of off-label uses and notes how, while off-label use is legal, FDA has made it difficult for manufacturers to communicate off-label information because its policies on manufacturer communications are ambiguous, use undefined terms and must be pieced together from a variety of sources, including nonbinding letters and guidance.

Specifically, the petition seeks a rulemaking “embodying FDA’s current policy on [manufacturer] responses to unsolicited requests [about off-label uses].” The petition also seeks “a comprehensive, binding statement as to the contours of the safe harbor” provided by a rule that otherwise prohibits a drug manufacturer from promoting an investigational new drug as safe and effective. The safe harbor allows manufacturers to engage in a “scientific exchange” about investigational new drugs so they could be made available to “desperately ill patients.” The petition requests that whatever regulation is adopted on this point include medical devices.  

The companies further seek clarity from FDA on their communications about off-label uses with “formulary committees, payors, and similar entities,” and they request “formal FDA policies specifically relating to manufacturer dissemination of clinical guidelines that may discuss off-label uses.” According to the petition, clinical guidelines are created by medical professionals, academic institutions and government agencies to provide the latest information and data concerning diagnosis, management and treatment. To the extent that such guidance recommends off-label uses, the companies seek clarification on “whether, or to what extent, a manufacturer can disseminate such guidelines.”