FDAAA included provisions to support and track  pediatric device development. According to FDA officials, the agency defined an additional  subpopulation, transitional adolescents, for its internal application review  purposes. FDAAA further required FDA to annually report to congressional  committees on the number of PMA and HDE devices approved in the  preceding year (1) for which there is a pediatric subpopulation that suffers  from the disease or condition that the device is intended to treat,  diagnose, or cure; (2) labeled for use in pediatric patients; and (3) exempt from user fees because the device was intended solely for pediatric use. To help connect individuals with ideas for pediatric medical devices with  experienced professionals to assist them through the medical device  development process, FDAAA also authorized demonstration grants for  pediatric device consortia to promote pediatric device development. Characteristics of the Population and Limited Economic Incentives Cited as Barriers to Pediatric Device Development

Certain characteristics unique to pediatric populations—including  physiological differences in pediatric patients and challenges with  recruiting pediatric participants for clinical trials—are barriers to  developing pediatric medical devices that were cited by stakeholders. Given these unique characteristics of the pediatric population, as well as  the fact that the market for pediatric devices is generally smaller than the  market for adult devices, there are limited economic incentives for  manufacturers to develop pediatric medical devices. Pediatric Device Consortia Have Assisted over 100 Device Projects; Many Were in Early Development

The pediatric device consortia reported assisting 107 pediatric device  projects—that is, projects to develop new pediatric devices—in the first   2 years of the demonstration grant program. In order to facilitate the  development and approval of pediatric medical devices, FDA awarded  grants totaling about $5 million to four pediatric device consortia in fiscal  years 2009 and 2010 (see table 2). For the device projects assisted by the consortia, the phase of  development varied. FDA Lacks Reliable Information to Readily Identify All Pediatric PMA and HDE Devices

		FDA Has Not Consistently Collected Reliable and Timely Information Needed to Report on Pediatric Devices

Although FDAAA required FDA to annually report to congressional  committees on the number of PMA and HDE devices approved in the  preceding year that were labeled for use in pediatric patients, the agency  lacks reliable information to readily identify all such pediatric devices. Fifteen PMA and 3 HDE Devices Have Been Explicitly Indicated for Use in Pediatric Patients since FDAAA Was Enacted

While the indication for use statement for most approved PMA and HDE  devices did not identify the age of the intended population, our review  identified 18 devices approved since FDAAA was enacted that were  explicitly indicated for use in pediatric patients.found that 15 PMA and 3 HDE devices approved since FDAAA was  enacted have indication for use statements explicitly stating that the  devices are for patients that include patients age 21 or younger, pediatric  patients, or skeletally immature patients. Specifically, FDA  should take steps to consistently collect information using its existing  pediatric electronic flag or otherwise develop internal controls to readily  and reliably collect and report information on devices for pediatric use. We are sending copies of this report to the Secretary of Health and  Human Services, the Commissioner of FDA, and other interested parties. Food and Drug Administration: Opportunities Exist to Better Address  Management Challenges.
Why GAO Did This Study

Medical devices can significantly improve, and save, the lives of children. Yet according to the Department of Health and Human Services (HHS) Food and Drug Administration (FDA), the development of pediatric devices lags years behind the development of devices for adults. The FDA Amendments Act of 2007 (FDAAA) provided incentives to develop devices for children, particularly devices that receive FDAs humanitarian device exemption (HDE), a process for devices that treat or diagnose rare diseases or conditions. FDAAA also authorized demonstration grants for nonprofit consortia to facilitate pediatric device development and required FDA to annually report the number of approved devices labeled for use in pediatric patients. Finally, FDAAA required GAO to report on pediatric device development. This report (1) describes barriers to developing pediatric devices, (2) describes how pediatric device consortia have contributed to the development of pediatric devices, and (3) examines FDA data on the number of pediatric devices approved since FDAAA was enacted. GAO examined FDA data and documents related to device approvals, reviewed relevant laws and regulations, and interviewed and reviewed documents from stakeholders and FDA officials.

What GAO Found

Certain characteristics unique to pediatric populationsincluding physiological differences from adult patients and challenges with recruiting pediatric participants for clinical trialsare barriers to developing medical devices for pediatric use cited by stakeholders. Given the unique characteristics of the pediatric population, and because the market for pediatric devices is smaller than the market for adult devices, there are limited economic incentives for manufacturers to develop pediatric medical devices.
FDAAA provisions authorized demonstration grants for pediatric device consortiathese consortia reported assisting over 100 pediatric device projects in the first 2 years of the program, fiscal years 2009 and 2010. FDA awarded grants totaling about $5 million to four pediatric device consortia in these 2 years. Of the device projects assisted by the consortia, many projects were in early stages of development, such as the creation of a prototype and the preclinical testing of that prototype. Activities performed by the consortia included regularly scheduled forums where innovators could discuss new device ideas with a group of experts.
FDA lacks reliable internal data with which to identify all pediatric devices. FDAAA required FDA to annually report to congressional committees on the number of devices approved in the preceding year that were labeled for use in pediatric patients. While FDA created an electronic flag in its internal tracking system to identify such devices, FDA has not consistently used this flag and therefore lacks reliable and timely information needed for reporting. GAOs review of the indication for use statements of devices approved since FDAAA was enacted identified 18 devicesincluding 3 HDE devices and 15 premarket approval (PMA) devicesthat were explicitly indicated for use in pediatric patients (patients age 21 or younger). However, the approved indication for use statements for most (72 percent) of the devices approved through the HDE or PMA processes did not specify the age of the intended patient population, so additional pediatric devices could exist.

What GAO Recommends

GAO recommends that FDA collect reliable information to report data on pediatric medical devices by consistently using its existing pediatric electronic flag in its tracking system or otherwise developing internal controls. HHS concurred with GAOs recommendations.