The American Association of Orthopaedic Surgeons (AAOS) office of government relations (OGR) has been successful in getting language, which allows greater flexibility in the Food and Drug Administration’s (FDA) Custom Device Exemption, included in the Medical Device User Fee Act (MDUFA) bills in the House and Senate.

“Physicians need some leeway to do what’s best for the patient,” one orthopaedic surgeon said, “and that is to provide the patient with implants that fit properly and are based on established standards.” The AAOS believes that work on this draft language moves orthopaedic surgeons closer to this goal.

In May, both the U.S. House of Representatives and the U.S. Senate passed similar versions of the MDUFA, which reauthorizes user fees for medical devices charged by the FDA. The fees help fund device review programs. As of this writing, the bills are in conference committee to reconcile minor differences, and President Obama is expected to sign the legislation in early July.

Custom devices are medical devices that deviate from those under an approved premarket application (PMA) or 510(k) clearance and are manufactured in response to a specific request from a physician to meet the unique needs of an individual patient. Congress intended custom devices to be available for patients whose anatomy does not suit a standard device, but the AAOS believes that current statutes and regulations do not allow enough flexibility to serve the patient populations that require such devices.

The custom device exemption
Under the current law, which expires in September, only one unique device for a specific patient can be manufactured under the exemption. If a second patient requires a similar device, that device can no longer be considered a custom device, and the manufacturer must have FDA clearance or be evaluating the device in a clinical study. The clinical use of these devices, however, is so limited that it is unreasonable and/or overly burdensome to apply for 510(k) clearance or premarket approval.

The AAOS draft language seeks a broadening of the exemption to include devices that are modifications of existing devices and to allow for production of more than a single unit of a particular device. Rather than limiting the number of a particular device that can be made to one, the AAOS language raises the limit to five. It defines custom device more broadly to allow use of a “customized device” or the modification of a pre-approved device, as well as a device that is essentially made from scratch.

When Congress created the exemption, it was aware of the practice of creating devices for unusual needs of patients and healthcare practitioners and intended to create a statutory provision that would allow a limited number of custom devices to be produced for these cases. Unfortunately, the FDA’s current interpretation of the exemption does not afford patients and providers this flexibility.

This has had a significant impact on the availability of devices. According to John T. Gill, MD, “As physicians, our primary goal is to help our patients. We need to ensure that all patients who need custom medical devices can get them quickly because many of the cases are time-sensitive. Right now, that is not happening. There are delays, and in some cases the devices are no longer available.”

Laura L. Tosi, MD, a pediatric orthopaedic surgeon, voiced similar concerns. “For many years I have been able to turn to one of our vendors for the urgent manufacture of a custom rod in the rare circumstance that an off-the-shelf device would not work. Starting about 2 years ago, the company told me that they could no longer help me out. Initially I assumed that the cost accountants were demanding cuts. But, on investigation, it turned out that the company was afraid of the FDA.”

Unmet needs
There is clearly a need for custom implants and devices for patients with rare orthopaedic disorders. Treatment of these patients requires devices that are either existing products with modifications or new products to fit the unique anatomy of a particular patient.

Several groups of patients are particularly disadvantaged by the current exemption. For example, physicians who treat patients with juvenile inflammatory arthritis and skeletal dysplasia often have challenges fitting devices to the patient’s smaller anatomy. Standard implants will not fit, so it is necessary to make implants that are based on current designs or smaller versions of regular implants that may have unusual features.

For orthopaedic oncology patients, the custom device exemption provides the only timely method of obtaining an appropriate device. This time element is critical when treating these patients because often only a limited time window exists between cancer treatments when white blood cell count is high enough to do a procedure.

The compassionate use exemption does not provide a viable alternative pathway for patients who need devices quickly, because it actually takes more time than the custom device pathway. Moreover, devices that fall under the compassionate use exemption are labeled “experimental,” which may create further delays when insurance companies deny coverage.

One physician provided the following example. “A father of a child with Ewing’s sarcoma was in my office looking at one of the custom devices as an option for his young child. I hated to think of the additional hurdles that the FDA could place in front of him. He will have to worry about FDA approval of the compassionate use request, Institutional Review Board approval at the hospital, then insurance worries or denial of coverage—all while trying to get the device manufactured and delivered in the brief time window between his son’s chemo treatments when his small body would be able to withstand surgery.”

According to Stuart L. Weinstein, MD, the pediatric population is underserved by the development of devices to treat injury, deformity, tumors, and delayed limb development (Fig. 1). “When devices and implants are not available for pediatric orthopaedic surgeons to treat the various musculoskeletal problems that children face, we have few choices. We can use adult devices off-label or modify them, use implants designed for another purpose, use suboptimal implants designed decades ago, or order custom devices.”

Dr. Weinstein continued, “It is important that policy makers realize that children are not just small adults. From the orthopaedic standpoint, their conditions are very different from those of adults. Children are growing, which has to be taken into account, and they place high demands on their implants. Their bodies are constantly changing, and when they have an implant or device, they are generally long-term users and often require multiple staged procedures.”

The AAOS OGR staff will continue to follow developments closely leading up to enactment of the pending legislation and will keep AAOS members informed.

Richard Martin, JD, is the manager, federal regulatory affairs, in the AAOS office of government relations. Julia Williams is the senior manager, government relations, in the AAOS office of government relations. She can be reached at jwilliams@aaos.org