FDA legislation changes pediatric research and development

In late September 2007, President Bush signed into law the Food and Drug Administration Amendments Act of 2007 (FDAAA) into law. The expansive legislation covered most aspects of the FDA’s charges and was a “must-pass” bill, meaning that approximately 2,000 employees would be laid off if the legislation was not enacted. In addition to updates on device user fees and drug safety, the bill included a section on pediatric medical device and development.

The law requires the Secretary of Health and Human Services to submit a plan to Congress based on consultations with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) of the National Institutes of Health (NIH), the FDA, and the Agency for Healthcare Research and Quality. The federal agencies convened a meeting on July 23, 2008, to solicit input from pediatric device stakeholders and to gain a broader perspective on developing the required plan.

A physician’s overview
Laurel C. Blakemore, MD, chief of orthopaedics and sports medicine at Children’s National Medical Center, represented the AAOS. In her presentation, she noted that the dynamics of developing devices for pediatric patients are fundamentally different than those for adults.

Pediatric device developers face the challenges of small markets, weak financial incentives, increased liability concerns, and the methodologic challenges of premarket clinical trials and postmarket surveillance. Clinicians treat pediatric patients by using or modifying adult devices, adapting implants designed for other purposes (such as using arm plates in the lower extremities of children), and using suboptimal implants designed decades ago.

Because devices are being used “off-label,” said Dr. Blakemore, surgeons are not able to teach or gather data on their effectiveness. She recommended the creation of a safe harbor to permit data collection on instances in which an off-label use is the standard of care. Other physicians noted that many pediatric conditions are rare and suggested that perhaps the FDA could relax its current interpretation of manufacturing custom devices.

Federal efforts
Representatives of the federal agencies briefed attendees on ongoing federal efforts. Altogether, six federal agencies sponsor 20 small business and other grants that may provide some level of funding for pediatric device development. Additionally, the NIH representatives distributed an overview of existing networks that are supported by government agencies and are related to pediatric device development.

Orthopaedic survey
Breakout discussions covered needs identification, safety monitoring, mechanisms to adapt adult devices for pediatric use, and challenges to device development.

Dr. Blakemore provided a recap on the 2005 pediatric device development survey conducted by the AAOS, the Pediatric Orthopaedic Society of North America (POSNA), and the Scoliosis Research Society (SRS). One third of respondents to that survey said they used adult-sized devices, and one quarter ordered custom devices to meet the needs of their patients. Robert M. Campbell Jr., MD, who was instrumental in creating the survey, led the device development session and called for mentorship during clinical trials.

More work is needed
Venture capitalists, inventors, and representatives from small market firms provided perspectives on steps needed to bring products to market. Angel investment firms were said to provide more funding than venture capital firms in 2007—up to $2.6 billion on medical devices alone.

The AAOS has been actively working with members of Congress, governmental officials, and others to find solutions to off-label use, the adaptation of adult devices, and other issues in pediatric device development. To present the orthopaedic view, the Academy, POSNA, and the SRS filed a joint response to the NICHD request for information.

Although the pediatric provisions in the FDAAA may not generate a significant number of new devices, they have raised awareness of a problem known to pediatric surgeons for years.

“The pediatric device development meeting was a good first step in bringing interested stakeholders together, but a lot of work needs to be done so that we can treat our pediatric patients with appropriate therapies” said Dr. Blakemore.

Jeanie Kennedy is manager, regulatory affairs, in the AAOS office of government relations. She can be reached at kennedy@aaos.org

View the final report of the survey and the NICHD response.