Making Decisions on When to Use Off-Label Drugs

Reimbursement

A further complication with off-label usage is physician compensation.

Dr. Detwiller said that insurance companies will often point to conflicting research when looking to deny an off-label use. She has run into that when it came to using Avastin to treat hereditary hemorrhagic telangiectasia (HHT). “It’s getting harder to do because I think there was a study that showed no benefit with it. Now, the insurance companies say, ‘Well, there’s this one study that shows no benefit.’ And the medication is so expensive, it’s understandable. We only have so many healthcare dollars to go around, you want to give the patient the treatment that is most efficacious. We do run into that. Apart from writing letters back saying, ‘Well, that study shows one thing, but there are other studies that show something else,’ there’s not a lot we could do.”

In the case of HHT, Dr. Detwiller said the medication is so expensive that when insurance companies won’t pay, she would recommend other treatments. When an insurance company won’t agree to cover using budesonide to treat chronic rhinosinusitis, Dr. Detwiller’s workaround is to use compounding pharmacies to make it at a reasonable cost.

Dr. Gillespie said his nurses sometimes spend an hour of their time fighting with insurance companies over the use of Botox for off-label clinical purposes. Sometimes, they’re not reimbursed.

“There is a cost-benefit analysis,” he said. “I think physicians routinely do things realizing that they’re not going to be reimbursed, but their hope, at the end of the day, is that they don’t lose money. As long as you’re not losing money, you’re still in the game. Once you start losing money repeatedly, then you can’t keep your lights on. You can’t employ your people … if you’re getting repeated denials and losing money repeatedly, you’re actually wrong to continue to offer the service because if you have to close your office, you aren’t serving anyone.”

Dr. Detwiller added that, reimbursable or not, off-label uses will continue as long as otolaryngologists keep hunting for novel ways to help patients. “Ideally, I wish we lived in a world where we had these perfect algorithms for treating disease,” she said. But “you’re going to have patients where you do all of what’s in the guidelines, and you try everything, and they’re not getting better. They’re still having problems, and you do go to these more off-label uses.” 

Richard Quinn is a freelance medical writer in New Jersey.

Key Points

  • Otolaryngology is a small field, and doesn’t always have the volume of patients or the market for industry to conduct RCTs to obtain FDA approval.
  • Physicians are careful to ensure there is clinical experience and basic science to balance the risk–reward of off-label use.

Talking to Patients about Off-Label Use

When it comes to off-label use, Dr. Detwiller thinks informed consent is the best approach. And not just because it’s an ethical issue; it also makes patients more comfortable. “As long as you and the patient have had an informed consent, and you’ve really weighed the potential benefit against the risk at what is happening, it helps,” said Dr. Detwiller.