Drugmakers await FDA guidance on 'off-label' uses of medicines

Drugmakers await FDA guidance on 'off-label' uses of medicines
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This story previously appeared in The Hill Extra.  


Doctors commonly prescribe drugs to treat conditions the medications weren't designed for, but for drugmakers, discussing such "off-label" uses can lead to trouble. 

As a result, drugmakers have been pushing both the Food and Drug Administration and lawmakers to draw clearer lines around communicating about unapproved uses of their products, particularly since it affects what companies can tell insurers. Such information is used to negotiate prices for covered medical products and their place in certain payment models. 

“We’ve been begging for it — for years — we’ve been begging for it and yet we’re not given in it, and then when somebody goes awry in any way, there’s huge enforcement and huge risks for every company,” Paula Taylor Whitfield, senior director and assistant general counsel for biopharmaceutical manufacturer Eli Lilly, said at the FDA/CMS Summit.


The FDA has been working on a proposal to address such off-label guidance, but on Dec. 14 announced that it has extended its deadline for comments by 90 days to April 10. 

The delay comes as some academics and consumer groups voice concerns about the risks of unapproved uses that have not been vetted by the FDA's gold standard of randomized clinical trials. 

Adriane Fugh-Berman, an associate professor in the Department of Pharmacology and Physiology at the Georgetown University Medical Center, said doctors can best determine when off-label uses are warranted. But most of drugmakers’ communications with providers promote uses that are not scientifically supported, and they do not discuss competitive options, like generics, she said.

“I would argue that the information is always misleading because it’s always a piece,” Fugh-Berman said.

A question of truth.

FDA has issued “safe harbor” policies that allow the sharing of scientific and medical journal articles and other materials describing off-label use if certain conditions are met. The agency also drafted guidance in 2014 to clarify what it considers to be legitimate and scientific communication.

Doctors can prescribe drugs and devices for off-label use if it’s in the best interest of their patient, but the FDA prohibits companies from promoting unapproved uses without including risk information.

Courts have in recent years ruled that discussions involving truthful and scientific information are allowed, which could affect who is responsible for rooting out falsehood, said Christopher Robertson, a law professor at New York University and the University of Arizona.

The FDA believes it sets the standard and the company has to prove effectiveness, Robertson said, but recent litigation is challenging that burden, saying it’s on the FDA to prove falsehood and giving courts the final say. 

Better partners.

Whitfield said liability laws and payer limits already keep drugmakers from promoting risky off-label uses, in addition to their concern for credibility and reputation. 

But drug companies could be better partners with the FDA, offering the agency a look at what information manufacturers have in “gray areas of science” and allowing officials to give feedback on what more may be needed, she said.

“That might be something that could benefit everybody and not have FDA or others thinking that this is the wild, wild West and companies are out there saying anything and everything,” Whitfield said.

But if the FDA looks at what drug companies intend to communicate, they’re effectively asking for soft consent to a new use that could otherwise be submitted for federal approval, he said. 

Expanding safe harbor policies is one approach, Robertson said, but the agency can go only so far before it becomes absurd because there’s clearly an intended use that the company has not proven is safe and effective.

“And that starts to erode the very logic of the FDA’s regulatory process,” he said.

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