Allowing Off-Label Drug Promotion Would Hurt Patients and Payers, Experts Warn

Recent court decisions have endangered the US Food and Drug Administration’s (FDA’s) long-standing prohibition against the off-label marketing of pharmaceuticals in favor of free speech protections, according to opinion columns in the New England Journal of Medicine and JAMA Internal Medicine. 

Commentators in both journals have a consistent message for the FDA: For the best interest of patients, payers, and the future of health care, stand strong.

“The existing regulatory regime has protected the public and helped provide physicians with rigorous evidence over many decades,” write Jeanie Kim, JD, and Amy Kapczynski, JD, both of Yale Law School, in JAMA Internal Medicine. “Drug companies’ disputed First Amendment rights should not catalyze a major shift in the agency’s enforcement authority; any changes should be approached with an abundance of caution.” 

In United States v Caronia, the US Court of Appeals for the Second Circuit in New York ruled in 2012 that off-label pharmaceutical sales pitches were protected speech under the First Amendment. Three years later, a US district court in Manhattan ruled that drug companies have the right to market off-label uses to physicians as long as their statements are not misleading or false. Pointing to these decisions, 2 ranking Republicans on the House Energy and Commerce Committee—Fred Upton of Michigan and Joseph Pitts of Pennsylvania—are urging the FDA to begin to allow pharmaceutical companies to promote off-label uses of approved drugs. 

Doing so, however, would be “substituting marketing for science,” warn Christopher Robertson, JD, PhD, of the University of Arizona and New York University School of Law, and Aaron S Kesselheim, MD, JD, MPH, Brigham and Women’s Hospital and Harvard Medical School, in a New England Journal of Medicine column.

“Manufacturers, could secure FDA approval of products for very narrow indications on the basis of highly limited data and then widely promote case series, poorly designed trials, and inadequately controlled observational ‘real-world’ evidence to support additional uses, to the potential detriment of patients and payers,” Dr Robertson and Dr Kesselheim wrote. —Jolynn Tumolo