We’re all used to state attorneys general suing drug companies for inappropriate promotion of off-label indications (the pain medication Neurontin comes to mind—among others). But now there’s an interesting new wrinkle. Some 16 state attorneys general have written to Abby Black, director of the Center for Drug and Health Plan Choice at the Centers for Medicare & Medicaid Services, to complain that Part D participating insurance companies are requiring that their customers fail on a variety of off-label therapies before they will be reimbursed for medicines that have the more appropriate, on-label indication relevant to their particular conditions.

“Just as it is inappropriate for pharmaceutical companies to market drugs for off-label uses, it is equally inappropriate for health insurance companies to refuse to reimburse for physician-prescribed medications unless a patient first undergoes treatment with drugs that are off-label,” write the AGs. They argue this practice of requiring treatment with an off-label drug before reimbursing a patient for using a drug approved by the FDA for that specific condition “subverts the legislatively mandated approval process for drug indications by substituting the judgment of health insurance companies for that of the FDA. It undermines the doctor-patient relationship by empowering health insurance companies to make broadly applicable medical decisions best left to a physician considering the needs of a specific patient. This ‘one-size-fits-all’ insurance-company mandate is inappropriate and dangerous.”

 

There are many issues raised here, not the least of which is the validity of the FDA label for anything following the recent Supreme Court decision in Wyeth v. Levine that shot down the idea that FDA approval of a drug provided protection to drugmakers against state liability laws. But an equally important question is when is off-label not off-label? At present, the answer seems to be when it’s on-formulary and off-patent.

 

Consider Iowa Medicaid and pregabalin (Lyrica), which is FDA-approved for fibromyalgia among other conditions. Iowa Medicaid requires preauthorization for pregabalin. A patient in that state with a diagnosis of fibromyalgia must first fail on at least two of the hawkeye state’s ”preferred” agents—trycyclic anti-depressants, topical lidocaine, or gabapentin. None of these three agents are approved by the FDA for the treatment of fibromyalgia, but they are less expensive than the on-patent, on-indication product. 

So what we’ve got here is step-therapy based on off-label usage. That’s not unheard of, but it does start sending some interesting policy messages about the appropriateness of off-label use in various circumstances. And it’s more than a little bizarre when you consider that Pfizer, the manufacturer of pregabalin, had to pay a $430-million fine for off-label promotion of gabapentin.

The actions of the Iowa State Department of Human Services are even more peculiar considering that Senator Grassley, a Republican from Iowa, asked the U.S. Government Accounting Office to investigate off-label prescribing—and not because he thought it was a valuable tool for patient care.

So here's where we stand: Off-label use of on-patent medications is bad, but off-label use for generics is good. In other word: off-label use is good when it saves the payers money.

“Insurance company requirements that patients utilize off-label treatments before being reimbursed for FDA-approved treatments are dangerous and should not be permitted,” the 16 state AGs write in their letter. “The same policy considerations that support a ban on off-label marketing by pharmaceutical companies support the prohibition of this insurance company practice.”

 

Are you paying attention Mr. Waxman?

To view the AG's letter, click here.

Peter J. Pitts is President of the Center for Medicine in the Public Interest and a former FDA Associate Commissioner. He is also Director of Global Healthcare for Porter Novelli.