Republicans: FDA Should Clarify Position on Off-Label Uses

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By Michael D. Williamson

May 27 — The FDA should clarify its position on what drug and device companies can tell doctors about unapproved or off-label uses, according to two House Republicans.

Recent court cases have raised doubts about the Food and Drug Administration's ability to restrict manufacturers from sharing truthful information about the off-label uses of drugs and medical devices, Reps. Fred Upton (R-Mich.), chairman of the Energy and Commerce Committee, and Joe Pitts (R-Pa.), chairman of the Energy and Commerce health subcommittee, said.

Despite these setbacks for the FDA, the agency hasn't released updated policies in the form of guidance or rules that would clarify its thinking on off-label uses, the lawmakers wrote in a May 26 letter to Health and Human Services Secretary Sylvia Mathews Burwell.

Off-label refers to uses that aren't approved or cleared by the FDA. Under long-standing policy, companies can be subject to criminal prosecution and civil liability if they promote their products for uses for which the FDA hasn’t specifically approved them.

The recent setbacks for the FDA include settlements of cases challenging the agency's off-label restrictions, and involved drugmakers Amarin (14 PLIR 357, 3/11/16) and Pacira (13 PLIR 1759, 12/18/15).

In addition, a federal jury in Texas returned a unanimous not guilty verdict in a criminal case against medical device company Vascular Solutions Inc. and its CEO in connection with alleged off-label promotion of a product (14 PLIR 319, 3/4/16).

The FDA's setbacks in the courts show the agency's policies on off-label enforcement are no longer tenable, James Czaban, a Washington-based attorney at DLA Piper LLP, told Bloomberg BNA May 27.


The two lawmakers said the Energy and Commerce Committee has learned that the FDA's inability to release an updated off-label policy could be the result of a disagreement between the FDA and HHS leadership, which may be harming patient health.

Upton and Pitts also said if the agency continues to remain silent, “settlement agreements will be the only means by which policy is formulated—and it will be in an ad hoc manner lacking any semblance of consistency and cohesiveness.”

Czaban told Bloomberg BNA that it's possible the HHS, which oversees the FDA, is delaying new policies on off-label promotion because agency leaders are worried that issuing updated rules or guidance documents could reduce the federal government's ability to recover monetary damages from False Claims Act cases.

False Claims Act settlements with medical product manufacturers are “a huge cash cow for the government,” Czaban said. The idea of losing this tremendous source of income and the power it gives the government over industry may be giving the HHS pause over changes to its off-label policies, according to Czaban.

In response to a Bloomberg BNA request for comment, Kevin Griffis, the assistant secretary for public affairs at the HHS, said May 27 the department will respond directly to the lawmakers about the concerns they raised in the letter.

Guidance Not Enough?

New FDA guidance on off-label promotion would be welcome, the lawmakers wrote, but added that they doubt whether non-binding policy statements would address due process concerns.

Upton and Pitts pointed to a February report from the Duke-Margolis Center for Health Policy at Duke University as a guide for developing new off-label policies. The report calls on the FDA to clarify the definition of labeling, for it to develop a working definition of “scientific exchange” and to provide more information around the term intended use. In that report, the Duke center said the scientific exchange principle has been viewed as permitting manufacturers to provide off-label information to certain audiences.

A draft bill that would clarify what industry can say about its products that Upton and Pitts attached to their letter would mandate similar changes.

That draft bill was first circulated in January 2015, Czaban told Bloomberg BNA.

Members of the Energy and Commerce Committee are open to considering alternative approaches to addressing the off-label issue, Upton and Pitts said. However, the agency's lack of willingness to make changes “shows why it is becoming increasing apparent that Congress must act,” they added.

Consumer Group's Response

Public Citizen, a consumer advocacy group, May 27 blasted the letter and said in a statement that changing the FDA's off-label policies would put patients in danger.

“The letter from Representatives Upton and Pitts shows a lack of appreciation for the role of the FDA in protecting patient health and a naive view of industry marketing practices,” Public Citizen said. Moreover, the threat to patient health posed by the draft bill attached to their letter is tremendous, according to the group.

For example, Public Citizen said the FDA's approval of a product for one use (say, to treat cancer) “tells us nothing about whether it is safe and effective for another use (say, to treat migraines). Yet this bill would allow drug and medical device manufacturers to advertise and promote their products—both to doctors and directly to consumers—for uses never approved by the FDA.”

To contact the reporter on this story: Michael D. Williamson in Washington at

To contact the editor responsible for this story: Brian Broderick at

For More Information

Upton and Pitts's letter is at

The report from Duke-Margolis Center for Health Policy is at