How should FDA revamp its off-label marketing rules? An expert panel backs big changes

After a series of court decisions put its regulatory approach in question, the FDA promised new guidance on off-label drug promotions. That was more than a year ago. Now, a group of health policy wonks and legal experts has come out with some suggestions of their own.

The recommendations from the Duke-Margolis Center for Health Policy include one big change that might simplify the free-speech debate over off-label marketing. The agency could revamp its approach to official labeling to include clinical data on off-label uses, graded for evidence quality, and even post-marketing data generated through the agency's own Sentinel surveillance system.

But before the FDA tries to get creative, it needs to take care of the basics, the report suggests.

The FDA needs to speak out with a set of new rules that address some specific outstanding questions, and quickly. Clarify some loose terms like "intended use," for instance.

Second, the FDA needs to make its current off-label rules easily accessible online. Right now, the information is scattered about in documents and filings, forcing a read-the-tea-leaves approach to discerning what the rules really are. Put it all together, the paper suggests.

Setting up a website sounds like a no-brainer, but anyone who's visited the FDA website knows how Byzantine its site map can be--and how difficult it is to find specific information at first pass.

"To address this, FDA could develop a web page that links all existing guidance (final or otherwise) in one easy-to-access place, regularly updated and augmented by any subsequent documents as they may be released," the group advises.

Changing official FDA labeling would be a more involved affair, and the Duke-Margolis Center suggests using pilot programs to test the idea. One might use the Sentinel system. Another might allow provisional off-label use claims based on evidence that's not enough for a new indication, contingent on Sentinel keeping watch on the new use. Overall, the idea would be to develop a tiered labeling system, with the strongest supporting data on the highest tier, and lesser quality evidence farther down.

An even more radical suggestion? Setting up a third-party data-review organization with the FDA's involvement. The review body would assess new off-label promotional material and give it a quality-control score based on the quality of evidence backing it up. Some of the materials would be approved for broad distribution, others wouldn't.

Peter Pitts

The third-party review idea didn't get backing from the entire committee, though some members were strongly in favor, including former FDA associate commissioner Peter Pitts, who touted the idea in the DrugWonks blog Monday. It's not unprecedented, as Pitts points out. Canada's Pharmaceutical Advertising Advisory Board (PAAB) assesses the accuracy and evidentiary basis for biopharma's promotional information.

"The PAAB process works within the Canadian regulatory framework with Health Canada as an ex-officio member of board leadership, conferring "approval" of advertising materials through a logo incorporated on cleared materials," Pitts says.

The committee also included University of Michigan law professor Nicholas Bagley, Ropes & Gray partner Joy Liu, CEO of the Food and Drug Law Institute Amy Comstock Rick, Alston & Bird partner Marc Scheineson and Sidley Austin partner Coleen Klasmeier. ASCO CMO Richard Schilsky also served, as well as four officials from the Duke-Margolis Center.

The FDA clearly wants to roll out its own guidance, rather than letting the courts decide. In December, the agency settled an off-label marketing dust-up with Pacira over its Exparel marketing, and it's on the verge of a deal in Amarin's ($AMRN) off-label lawsuit involving its cardiovascular med Vascepa.

- check out the report here
- see the DrugWonks post

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