AAOS Now

Published 12/20/2023
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Caleb Grote, MD, PhD; Jonathan Dubin, MD, FAAOS; Norman Otsuka, MD

Mind the Gap: Innovation in Pediatric Orthopaedics Is Lagging

Pediatric patients have unique anatomy, physiology, and pathology that must be considered by the surgeons treating them. An “innovation gap” is widely recognized in the field of pediatric orthopaedics. The FDA estimates that pediatric medical device development lags 5 to 10 years behind that for adults.

In the United States, 25 percent of the population is under the age of 18, yet only 10 percent of all healthcare spending and less than 12 percent of all National Institutes of Health (NIH) funding are devoted to pediatrics. The burden of disease in children and adolescents is likely less than that of adults; however, clinical experience combined with the value, vulnerability, and longevity of this population makes these numbers concerning. Although the entirety of pediatric medicine is affected, the impact of this gap is compounded for musculoskeletal conditions. Despite musculoskeletal disorders accounting for $380.9 billion in healthcare costs annually in the United States, less than 2 percent of NIH funding is dedicated to these conditions. Pediatric orthopaedics lies at the intersection of these disparities.

Regulatory issues
Throughout society, innovation is exploding, and the number of novel medical devices being brought to market has grown tremendously over recent years. However, several recent studies have demonstrated that children are being left behind in this boon. Pathak et al. demonstrated that of the 124 Class 3 (high-risk/highly innovative) devices approved by the FDA from 2016 to 2021, only two were dedicated specifically for pediatric use, whereas another 23 included pediatric and adult labeling. Lee et al showed that across all specialties, only 42 high-risk devices have been approved for use in patients <17 years old since inception of fda device regulation in 1976. it is important to point out that in both of those studies, cardiovascular and ophthalmology devices constituted the majority of the approved pediatric indications.>

Interestingly, by law, the upper age limit to be considered pediatric by the FDA is 21 years. Furthermore, the FDA arbitrarily defines neonates (0 through 28 days of life), infants (29 days to <2 years), children (aged 2 to><12 years), and adolescents (aged 12 to 21 years). most approved devices are for older “pediatric” patients (ages 18 to 21 years). when considering younger patients only, there were five (9 percent) devices directed toward infants and neonates, according to an fda report to congress in 2017.>

The authors recently conducted a similar study of Class 3 devices that identified only three that had pediatric orthopaedic indications, all of which were approved via the Humanitarian Device Exemption FDA regulatory pathway. To facilitate development of devices that treat rare diseases (<8,000 cases per year), the fda allows these devices to be approved in this less stringent regulatory process. as many rare diseases manifest in children, congress further encouraged development by lifting previous profitability restrictions for humanitarian device exemption devices with pediatric indications.>

In contrast to these findings in pediatrics, adult patients with orthopaedic conditions benefit immensely from innovation. More than 600 orthopaedic devices are authorized by the FDA annually, representing nearly 20 percent of all devices.

The FDA recently surveyed multiple physician groups with relevant interests and expertise in device development, and among key findings was that 66 percent of respondents identified the need for additional devices that grow with children. Surgeons with experience in fixating pediatric fractures are keenly aware of the shortage of adequately designed implants, which forces surgeons into off-label use by repurposing adult-designed implants. This usage frequently leads to poor implant fit, which can cause not only prominence for the patient but also inferior reduction quality and fracture stability—in addition to the moral and ethical burden the surgeon carries with off-label use of medical devices.

For example, the authors treated an 11-year-old skeletally immature female with a severely comminuted, open tibia fracture with a large zone of injury. Unfortunately, without any physeal-sparing distal tibia plates available on the market long enough for this injury, a distal femoral locking plate was utilized instead. Although acceptable reduction was achieved and the patient went on to heal, the implant proved to be prominent, necessitating a second surgery for removal. Some examples of other unmet needs that the authors have encountered include implants designed to withstand the increased physiologic forces seen in morbidly obese children and adolescents, improved cartilage restoration techniques, and higher tensile grafts in ligament reconstruction that can reduce bone tunnel size.

Innovation roadblocks
Barriers to innovation in pediatric orthopaedics are multifactorial, ranging from clinical considerations and technical difficulties to financial incentives. Implant designers for this population must account for changing physiology and patient growth, unique daily activities of children, and the impact of developmental age on the patient’s ability to interact with devices. Research and ethical considerations specific to minors must be recognized too, including their heightened vulnerability and the assent/consent process.

Importantly, the costs of bringing a novel device to market can be inhibitory for many developers, with the mean expenditure on Class 3 devices that require clinical trials estimated at $54 million dollars, although it can balloon to more than four times that amount in some cases. Considering the relatively smaller market and lower reimbursement rates in pediatrics, this cost is a major contributing factor to the lack of innovation. At the 2012 AAOS ‘‘Industry for Kids’’ forum, manufacturers reported the costs and poor prospects of profitability as major impediments to novel pediatric device production.

Facilitating medical device innovation for pediatric patients has been identified as a key initiative by the FDA. In 2007, Congress passed the Pediatric Medical Device Safety and Improvement Act to dedicate funding to development in this area. Out of this came the FDA’s Pediatric Device Consortium, which focuses on fostering the development of pediatric medical devices. The FDA and the NIH are also collaborating with a spectrum of stakeholders via the National Evaluation System for Health Technology to improve methods to safely employ real-world evidence for labeling of pediatric devices. The most recent development has been the System of Hospitals for Innovation in Pediatrics–Medical Devices, which is a network of specialized pediatric hospitals and healthcare professionals with whom device manufactures can interface. This infrastructure is designed to help streamline product development via centralization of regulatory bodies, collaborative trial initiatives, and engagement with reimbursement pathways.

Ultimately, some of the burden falls to pediatric orthopaedic surgeons and other clinicians to continue to advocate for the development of appropriate devices to care for pediatric patients and not to continually “make do” with the implants available. Reducing the innovation gap will improve care but will require significant buy-in at multiple levels. Improving the landscape for pediatric orthopaedic innovation will require focusing on increasing market compensation, limiting burdensome regulatory pathways, and improving collaborations between healthcare and industry.

Caleb Grote, MD, PhD, is a pediatric orthopaedic surgeon at Children’s Mercy Hospital in Kansas City, Mo. He is an assistant professor of orthopaedics at the University of Missouri–Kansas City School of Medicine and the University of Kansas School of Medicine. He is the director of the Adolescent Hip Preservation Program at Children’s Mercy Hospital.

Jonathan Dubin, MD, FAAOS, is an associate professor of orthopaedics at the University of Missouri–Kansas City Medical School and serves as chief of orthopaedic trauma at University Health–Truman Medical Center. He is a member of the AAOS Committee on Devices, Biologics, and Technology.

Norman Otsuka, MD, is chair of the Department of Orthopaedic Surgery at Children’s Mercy Hospital in Kansas City, Mo.

References

  1. Espinoza JC: The scarcity of approved pediatric high-risk medical devices. JAMA Netw Open 2021;4(6):e2112760.
  2. McConaghy K, Klika AK, Apte SS, et al: A call to action for musculoskeletal research funding: the growing economic and disease burden of musculoskeletal conditions in the United States is not reflected in musculoskeletal research funding. J Bone Joint Surg Am 2023;105(6):492-8.
  3. S. FDA: Report to Congress: Annual Report Premarket Approval of Pediatric Uses of Devices FY 2017. Available at: https://www.fda.gov/media/128659/download. Accessed Dec. 4, 2024.
  4. Sheha ED, Hammouri Q, Snyder BD, et al; POSNA/SRS Task Force for Pediatric Medical Devices: Off-label use of pediatric orthopaedic devices: important issues for the future. J Bone Joint Surg Am 2014;96(3):e21.