Why Otolaryngologists Should Embrace Off-Label Drugs, Devices

Steven Handler, MD, associate director of otolaryngology at The Children’s Hospital of Philadelphia, said it’s “incumbent” on doctors to use the equally effective, less expensive alternative. In pediatric respiratory papilloma disease (RPP), Dr. Derkay’s area of expertise, there’s little choice in the matter. “There’s nothing in terms of our armamentarium that is currently FDA approved,” Dr. Derkay said.

The quadrivalent human papilloma virus (HPV) vaccine, Gardasil, is a testament to the frustrating FDA system. Research has determined that if 80 percent of women or 70 percent of both women and men were vaccinated against strains of HPV that cause RPP, there would be enough herd immunity to basically wipe out the disease. Cost evaluations have found that administering HPV vaccines would mean 13 cents spent for every dollar of treatment cost saved, Dr. Derkay said. “There are few things that are that big of a bargain in medicine,” he added.

But Merck, the Gardasil manufacturer, is not allowed to promote the vaccine for that purpose, which wasn’t part of its initial approval. Pediatric otolaryngologists and head and neck surgeons are “really limited in our ability to speak to our pediatric colleagues” about the RPP benefits, said Dr. Derkay. “The system kind of works against us.”

Only approximately 54 percent of eligible girls and 10 percent of eligible boys are receiving the vaccine, he said. It will take 15 years to gather the evidence the FDA needs to approve the vaccine for RPP, he said.

Robotic Surgery

Bert O’Malley, MD, Gabriel Tucker Professor of otorhinolaryngology at the University of Pennsylvania in Philadelphia, reviewed the long process of seeking approval from the FDA for use of the da Vinci robotic system for minimally invasive head and neck surgery.

Beginning clinical studies for the safety and efficacy data to give to the FDA first required obtaining independent review board approval. The process ended up not requiring the further step of getting an “investigational device exemption,” he said. He and colleagues made the case that there was not “significant risk”—because it is manual, image-guided surgery, they said, the operation stops once the surgeon’s hands leave the console. The independent review board gave approval, and data was collected to show that the system was at least as equally effective as standard techniques.

At first, though, the FDA reviewers were fearful that there were not enough data to justify safety, even though the same system had been approved for use by cardiac surgeons. “The otolaryngologists on the review panel were not head and neck surgeons who do minimally invasive or transoral or highly complex head and neck surgery, so they were fearful of this device and the nature of the surgery, said Dr. O’Malley. “So it shows you a whole other factor that’s involved with getting FDA approval.”