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Return to Facing the Challenges of "Off-Label" Usage Overview

More on Facing the Challenges of "Off-Label" Usage

Facing the Challenges of "Off-Label" Usage

New York (Mar 21, 2011)

Michael Vitale, MD
Michael Vitale, MD

The world has come a long way with medical breakthroughs regarding pediatric orthopaedics. Thanks to technological advancements, many children with musculoskeletal disorders are now able to improve and thrive as a result of new therapies and treatments.

Unfortunately, many of these new techniques require orthopaedic surgical devices that, although approved by the FDA for adult usage, are not approved for pediatric usage. Orthopaedic surgeons are often confronted by this "physician directed" or "off-label" issue, which includes not just devices, but drugs and new therapeutic agents. It isn't uncommon for some of these new technologies to become standards of care long before they receive formal regulatory approval.

In fact, off-label usage in pediatric orthopaedics has become standard acceptable practice. The American Academy of Orthopaedic Surgeons states, "If physicians use a product for an indication not in the approved or cleared labeling, they have the responsibility to be well informed about the product, to base its use on firm scientific rationale and on sound medical evidence, and to maintain awareness of the productís use and effects."

However, financial issues can come into play, since off-label usage can mean that health insurance will not cover costs. Physicians need to be aware of whether patients' insurance will reimburse for specific usage.

Michael Vitale, MD, Chief of the Pediatric Spine Service at NewYork-Presbyterian Morgan Stanley Children's Hospital/Columbia University Medical Center has been to Washington D.C. multiple times to advocate for the use of new surgical procedures and the need to find affordable regulatory pathways to FDA approval.

In 2009, Dr. Vitale served as a moderator for the orthopaedic panel at the FDA's Pediatric Clinical Trial Workshop, the first of its kind that addressed physicians' unmet needs, studies, safety and effectiveness of treatments, and outcomes. "The event was successful in that it opened lines of communication on issues and highlighted challenges in innovation," says Dr. Vitale. "One of the things we covered was how to deal with the fact that it's hard to spend prohibitive amounts of money on research and development on items that have such a small market."

There has been an increased necessity for surgeons to use off-label devices to receive the most successful results. Growing rods and pedicle screws are, at this time, off label. In order to get FDA approvals there needs to be device trials. However, the Institutional Review Board (IRB) won't approve device trials for devices that are not FDA approved. Pediatric orthopaedic surgeons from around the globe have realized the serious need for regulation reform.

"Once or twice a week, I use pedicle screws in skeletally immature patients," says Dr. Vitale. "Every pediatric spine surgeon uses them. Unfortunately, pedicle screws may not be optimal for use in kids but they're the best available implant available right now. I have to use the device off label. If I only used devices that were on label, I wouldn't be able to take care of the children we treat."

After several additional trips to Washington D.C., Dr. Vitale is noticing positive change. "We're realizing that we have the ability to do more for kids and intervene in ways that we didn't before. The market is growing and industry is aware that it has an opportunity to do a little more than it has in the past," says Dr. Vitale.

While Dr. Vitale would like the FDA approval process to speed up, he has no plans of slowing down in his quest for reform. "All parties need to realize that it's in the best interest of children to not only encourage innovation, but also to provide a smooth flowing approval process. We need to ensure these new technologies are safe and easily accessible for all kids with musculoskeletal disorders."

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