Safeguarding Patient Welfare: Who's In Charge?

Over the course of my career, I have been privileged to serve in a variety of roles, including that of oncologist, principal investigator, study manager, senior academic administrator, Commissioner of Food and Drugs at the U.S. Food and Drug Administration (FDA), and as board member for 3 Fortune 500 and Global 500 companies (AstraZeneca PLC, 2001 to present; AmerisourceBergen Corporation, 2002 to present; and Cigna Corporation, 2004 to present). I have been regulated and a regulator, and I have led institutions striving to develop and advance new investigational therapies within a heavily regulated environment. This experience has afforded me a unique view of the various controversies in drug marketing, particularly those related to off-label uses of drugs. Steinman and colleagues' article (1) on the marketing of gabapentin guides my discussion of off-label use, changing FDA regulatory power, the role of industry, the involvement of the medical profession in promoting off-label use, and our fundamental obligation to protect the best interests of our patients.

Off-label use is the common practice of prescribing a drug for an indication other than those approved by the FDA. The physician rationale for prescribing off-label is often based on the lack of FDA-approved effective treatments, reports of clinical effectiveness for the off-label use, or both. A recent study confirms that off-label prescribing continues in earnest, with 21% of drugs listed in a data set being prescribed for off-label uses, most with little scientific evidence of efficacy (2). Although the responsibility for seeking FDA approval for adding new uses to the product label resides solely with the manufacturer, companies are reluctant to invest the resources necessary to develop the evidence required for FDA review, particularly when the off-label uses are already profitable.

In this issue, Steinman and colleagues (1) examine the gabapentin marketing and promotion strategies …