Regulatory actions on the off-label use of prescription drugs: ongoing controversy and contradiction in 2009 and 2010.

A letter by Zheng and Xu in this issue of JMCP spotlights a consensus statement on off-label medication use, written by 20 senior chief pharmacists from 17 hospitals in the Guangdong province of China in March 2010. The statement’s provocative recommendations include the imposition of requirements that (a) the off-label drug should be “irreplaceable for treatment of the patient,” that is, no drug labeled for the indication is available; (b) the off-label use must be “submitted and approved by the hospital’s pharmacotherapy committee and hospital ethics committee” except in emergency situations; and (c) informed consent for off-label use must be obtained from the patient or a legal representative. The letter comes on the heels of nearly 2 years of heightened debate in the United States over the use of and payment for drugs prescribed to treat indications for which they are not approved by the U.S. Food and Drug Administration (FDA). Triggered by several factors—including U.S. Department of Justice (DOJ) litigation against pharmaceutical companies for off-label promotion, new guidance for “Good Reprint Practices” promulgated by the FDA in January 2009 to clarify the types of medical literature deemed acceptable for distribution to physicians, and the passage of the Affordable Care Act of 2010—the controversy has drawn the attention of numerous stakeholders with different and sometimes sharply conflicting perspectives. In March 2009, we noted that positions on off-label drug use taken by different federal agencies appeared inconsistent, with the DOJ’s legal actions against off-label marketing becoming increasingly intense even as the FDA slightly relaxed its stance against the practice and the Centers for Medicare & Medicaid Services (CMS) expanded Medicare coverage for off-label use of chemotherapeutic drugs. Since that time, the controversy has intensified, with stakeholders adopting positions that seem to allow little or no ground for compromise.