Sue Ducat
Director of Communications
(301) 841-9962

Melissa Blair
Robert Wood Johnson Foundation
(609) 627-5937

Off-Label Drug Promotion

A new policy brief from Health Affairs and the Robert Wood Johnson Foundation looks at a renewed debate over whether and how the Food and Drug Administration (FDA) should regulate the pharmaceutical industry's communication to physicians about off-label uses of drugs. When a drug is prescribed "off label," that means it is being given to a patient for indications that have not been specifically approved by the FDA. According to information issued by the Agency for Healthcare Research and Quality (AHRQ) in 2015, an estimated one in five prescriptions written are for off-label use. In some cases, such use is the accepted standard of care and recommended in clinical practice guidelines. The recent debate was spurred in part by high-profile recent court cases and shifts in the health care landscape. 

Topics covered by this policy brief include: 

  • What's the background? This section of the brief explains why providers might choose to prescribe off label—and lists some of the studies that have determined how frequent a practice this is. It also details FDA guidelines regulating how manufacturers are allowed to communicate with health care professionals about unapproved uses and highlights several key US Supreme Court decisions, which served to weaken the FDA's historic approach to regulating off-label communication between manufacturers and providers. 
  • What's the debate? At the center of the debate over off-label communication is the question of how the FDA can best safeguard public health while also supporting fully informed decision making by providers and patients. As the brief points out, the official mechanism for a drug manufacturer to expand a drug's use, a New Drug Application, requires new clinical trials and other costly and time-consuming efforts. Manufacturers frequently argue that additional uses of new drugs could help advance medical knowledge and practice. However, as the brief notes, weakening off-label regulations could further intensify problems such as the lack of high-quality evidence, and pose greater risks to patients. 
  • What's next? The brief summarizes upcoming guidance documents planned for release later this year by the FDA as well as how congress and the courts might ask the FDA for further regulatory clarity. As the brief concludes, it still remains to be seen how the FDA's regulatory approach will evolve over time and what the implications will be for patients. 

About Health Policy Briefs

Health Policy Briefs are aimed at policy makers, congressional staffers, and others needing short, jargon-free explanations of health policy basics. The briefs, which are reviewed by experts in the field, include competing arguments on policy proposals and the relevant research supporting each perspective.

Previous Health Policy Briefs have addressed:

  • Ambulance Diversion. Efforts to mitigate ambulance diversion have been effective, but questions remain for future progress.

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About Health Affairs

Health Affairs is the leading journal at the intersection of health, health care, and policy. Published by Project HOPE, the peer-reviewed journal appears each month in print and online, with additional Web First papers and health policy briefs published regularly at Read daily perspectives on Health Affairs Blog and customize the content you want to see in Health Affairs Alerts.

About the Robert Wood Johnson Foundation

For more than 40 years the Robert Wood Johnson Foundation has worked to improve health and health care. We are striving to build a national Culture of Health that will enable all to live longer, healthier lives now and for generations to come. For more information, visit Follow the Foundation on Twitter at or on Facebook at