PEDIATRIC MEDICAL 

DEVICES

Provisions Support 

Development, but 

Better Data Needed 

for Required 

Reporting

Report to Congressional Committees

December 2011

GAO-12-225

United States Government Accountability Office

GAO
United States Government Accountability Office

Highlights of GAO-12-225, a report to 

congressional committees

December 2011

PEDIATRIC MEDICAL DEVICES

Provisions Support Development, but Better Data 

Needed for Required Reporting

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 FDA’s 

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 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 GAO’s 

recommendations.

What GAO Found

Certain characteristics unique to pediatric populations—including physiological 

differences from adult patients and challenges with recruiting pediatric 

participants for clinical trials—are 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 

consortia—these 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. GAO’s 

review of the indication for use statements of devices approved since FDAAA 

was enacted identified 18 devices—including 3 HDE devices and 15 premarket 

approval (PMA) devices—that 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.

PMA and HDE Devices Approved in Fiscal Years 2008 through 2011 by Indicated Population

Note: Totals do not add to 100 percent because of rounding.

View GAO-12-225. For more information, 

contact Marcia Crosse at (202) 512-7114 or 

crossem@gao.gov.
Page i GAO-12-225 Pediatric Medical Devices

Letter 1 

Background 6

Characteristics of the Population and Limited Economic Incentives 

Cited as Barriers to Pediatric Device Development 13

Pediatric Device Consortia Have Assisted over 100 Device Projects; 

Many Were in Early Development 18

FDA Lacks Reliable Information to Readily Identify All Pediatric 

PMA and HDE Devices 23

Conclusions 32

Recommendation for Executive Action 33

Agency Comments 33

Appendix I Summary of HDE Devices Granted Exemption from the HDE 

Profit Prohibition 35

Appendix II PMA and HDE Devices Explicitly Indicated for Use in Pediatric 

Patients since Fiscal Year 2006 41

Appendix III PMA and HDE Devices Explicitly Indicated for Use in Pediatric 

Patients, Fiscal Years 2008 through 2011 43

Appendix IV Comments from the Department of Health and Human Services 47

Appendix V GAO Contact and Staff Acknowledgments 49

Related GAO Products 50

Contents
Page ii GAO-12-225 Pediatric Medical Devices

Tables

Table 1: Pediatric Subpopulations for Medical Devices and Related 

Ages 11

Table 2: Summary of Pediatric Device Consortia Grant Amounts 

and Funded Activities, Fiscal Years 2009 and 2010 19

Table 3: Number of Device Projects by Development Phase That 

Were Mentored or Supported by the Pediatric Device 

Consortia 21

Table 4: Medical Device Projects Presented in Consortia Grant 

Applications and Funded in Fiscal Years 2009 and 2010 22

Table 5: Approved PMA and HDE Devices Explicitly Indicated for 

Use in Pediatric Patients since FDAAA Was Enacted, by 

Pediatric Subpopulation 29

Table 6: Characteristics of the Two Pediatric HDE Devices Granted 

Exemption from the HDE Profit Prohibition 36

Table 7: Number of Physicians Reporting Difficulty Obtaining 

Insurance Coverage for Melody TPV Device, by Insurance 

Type and Extent of Difficulty Experienced 39

Table 8: PMA and HDE Devices Explicitly Indicated for Use in 

Pediatric Patients, Fiscal Years 2006 through 2011 42

Figures

Figure 1: PMA and HDE Devices Approved in Fiscal Years 2008 

through 2011 by Indicated Patient Population 27

Figure 2: HUD Requests, HUD Designations, and Approved HDE 

Devices Explicitly Indicated for Use in Pediatric Patients, 

Fiscal Years 2001 through 2011 31
Page iii GAO-12-225 Pediatric Medical Devices

Abbreviations

ADN annual distribution number

CDRH Center for Devices and Radiological Health

CHIP Children’s Health Insurance Program

CPT current procedural terminology

CTS Center Tracking System

FDA Food and Drug Administration

FDAAA FDA Amendments Act of 2007

HDE humanitarian device exemption

HHS Department of Health and Human Services 

HUD humanitarian use device

IRB institutional review board

PMA premarket approval 

RVOT right ventricular outflow tract

This is a work of the U.S. government and is not subject to copyright protection in the 

United States. The published product may be reproduced and distributed in its entirety 

without further permission from GAO. However, because this work may contain 

copyrighted images or other material, permission from the copyright holder may be 

necessary if you wish to reproduce this material separately.
Page 1 GAO-12-225 Pediatric Medical Devices

United States Government Accountability Office

Washington, DC 20548

December 20, 2011

The Honorable Tom Harkin

Chairman

The Honorable Michael B. Enzi

Ranking Member

Committee on Health, Education, Labor, and Pensions

United States Senate

The Honorable Fred Upton

Chairman

The Honorable Henry A. Waxman

Ranking Member

Committee on Energy and Commerce

House of Representatives

Medical devices prevent premature death and significantly improve the 

quality of life for many children each year.1

FDA, an agency within the Department of Health and Human Services 

(HHS), is responsible for ensuring that medical devices, including 

pediatric medical devices, sold in the United States provide reasonable 

assurance of safety and effectiveness and do not pose a threat to public 

However, stakeholders from 

the pediatric community have raised concerns about the availability of 

devices developed and approved for pediatric use. In particular, concerns 

have been raised about challenges to developing and marketing pediatric 

medical devices. Designing and developing pediatric medical devices can 

be challenging because children’s bodies are often smaller, are structured 

differently than adults, and change throughout childhood. According to the 

Food and Drug Administration (FDA), in 2011 the development of medical 

devices for children lagged 5 to 10 years behind the development of 

devices for adults. Some medical devices are designed specifically for 

pediatric patients and some are designed for patients above a certain 

age. Frequently, providers modify devices designed for adults for use in 

their pediatric patients.

 

1

Generally, medical devices include items used for the diagnosis, cure, mitigation, 

treatment, or prevention of a disease. See 21 U.S.C. § 321(h). Throughout this report, the 

term device refers to a medical device that is not being regulated as a drug or a biological 

product.

 
 

Page 2 GAO-12-225 Pediatric Medical Devices

health. Before medical devices may be legally marketed in the United 

States, they are generally subject to FDA premarket review unless 

exempt by FDA regulations.2

To encourage the development of pediatric medical devices, the FDA 

Amendments Act of 2007 (FDAAA) removed the profit prohibition for HDE 

devices that are used to treat children and meet other requirements,

Under FDA’s premarket approval (PMA) 

process, manufacturers must supply evidence providing reasonable 

assurance of safety and effectiveness to obtain FDA approval to market a 

device in the United States; we refer to devices approved through this 

process as PMA devices. To encourage development of devices that treat 

or diagnose rare diseases or conditions, devices that receive a 

humanitarian device exemption (HDE) are exempt from the effectiveness 

requirement of the PMA process. Under FDA’s HDE process, 

manufacturers are instead required to show that the probable benefit 

outweighs the risk of using the device. In general, manufacturers of 

devices approved through the HDE process are allowed to recover 

certain development and production costs but may not make a profit on 

their device; we refer to devices approved through this process as HDE 

devices.

3 and 

established a demonstration grant program for nonprofit consortia to 

stimulate pediatric medical device development.4

 

2

Devices are generally subject to one of two types of FDA premarket review—the 510(k) 

premarket notification process and the more stringent premarket approval process—

unless exempt by FDA regulations. Under the 510(k) premarket review process, a medical 

device may be marketed in the United States after being cleared by FDA if FDA 

determines that the device is substantially equivalent to a legally marketed device that is 

not required to go through the premarket approval (PMA) process.

Pediatric device 

consortia receiving these grants, administered by FDA, are expected to 

facilitate the development, production, and distribution of pediatric 

medical devices through activities such as connecting innovators and 

physicians to existing federal resources, assessing the scientific merit of 

proposed pediatric device projects, and providing assistance and advice 

as needed on business development. In addition, FDAAA required FDA to 

annually report to the Senate Committee on Health, Education, Labor, 

and Pensions and the House Committee on Energy and Commerce on 

3

Pub. L. No. 110-85, § 303(a)(3), 121 Stat. 823, 860-62 (2007) (codified in part at 

21 U.S.C. § 360j(m)(6)).

4

Pub. L. No. 110-85, § 305, 121 Stat. 823, 863-64 (2007).
 

Page 3 GAO-12-225 Pediatric Medical Devices

pediatric medical devices, including the number of devices approved in 

the preceding year that were labeled for use in pediatric patients.5

FDAAA also required GAO to report on pediatric medical device 

development.6 In this report, we (1) describe barriers to developing 

pediatric medical devices, (2) describe how the demonstration grants for 

pediatric device consortia have contributed to the development of 

pediatric medical devices, and (3) examine FDA’s data on the number of 

pediatric PMA and HDE devices approved since FDAAA was enacted.7

To describe the barriers to developing pediatric medical devices, we 

interviewed officials and reviewed documents from key pediatric medical 

device stakeholders, including the pediatric device consortia; 

manufacturer organizations, including the Medical Device Manufacturers 

Association and the Advanced Medical Technology Association; 

professional associations, such as the American Academy of Pediatrics, 

the Pediatric Orthopaedic Society of North America, and the American 

Thoracic Society; advocacy organizations, such as the Elizabeth Glaser 

Pediatric AIDS Foundation and the National Organization for Rare 

Disorders; and the two manufacturers of HDE devices approved for use in 

pediatric patients and exempted from the HDE profit prohibition. In 

addition, we interviewed officials from FDA and the National Institutes of 

Health who conduct work related to medical devices. We also conducted 

a literature review of government and independent reports and peerreviewed publications and reviewed stakeholder testimonies and 

summaries regarding barriers to pediatric medical device development.8

 

5

Pub. L. No. 110-85, § 302, 121 Stat. 823, 859-60 (2007) (codified at 21 U.S.C. § 360e-1).

In addition, we examined policy papers written by key stakeholders, 

including FDA and professional associations.

6

Pub. L. No. 110-85, § 303(b), 121 Stat. 823, 862 (2007).

7

In addition, FDAAA required that we describe the key characteristics of pediatric HDE 

devices granted exemption from the HDE profit prohibition. We discuss these devices in 

app. I.

8

Reports we reviewed included, for example, Institute of Medicine, Rare Diseases and 

Orphan Products: Accelerating Research and Development (Washington, D.C.: The 

National Academies Press, October 2010), and American Academy of Pediatrics,

Summary of Pediatric Stakeholder Meetings (Washington, D.C.: November 2004).
 

Page 4 GAO-12-225 Pediatric Medical Devices

To describe the contributions of the pediatric device consortia, we 

reviewed pediatric device consortia quarterly and annual reports 

submitted to FDA to assess the type and amount of assistance the 

consortia provided to device innovators and manufacturers described in 

the reports.9

To examine FDA’s data on the number of pediatric PMA and HDE 

devices approved since FDAAA was enacted, we reviewed FDA 

procedures for tracking and reporting information on pediatric medical 

devices and analyzed FDA data and documents for approved PMA and 

HDE devices.

We also reviewed additional information provided by FDA 

and interviewed officials from FDA and the consortia and other relevant 

stakeholders.

10 We compared FDA’s procedures for tracking and 

reporting information on pediatric medical devices to standards described 

in Standards for Internal Control in the Federal Government—which 

specifies that information should be communicated to those who need it 

in a form and within a time frame that enables them to carry out their 

operational responsibilities.11

 

9

We reviewed quarterly and annual consortia reports available through September 2011. 

These reports included information on the device projects assisted by the consortia that 

could be developed to enter the U.S. market through one of FDA’s premarket review 

processes—PMA, HDE, or the 510(k) premarket review process. 

Specifically, we examined the agency’s 

ability to readily identify approved PMA and HDE devices labeled for use 

in pediatric patients and report on them in a timely manner. To do this and 

to determine the reliability of FDA’s data on PMA and HDE devices 

labeled for use in pediatric patients, we interviewed FDA officials; 

reviewed laws, regulations, and guidance; and reviewed FDA data 

10FDAAA required that we focus our report on medical devices that were approved for the 

U.S. market through the PMA and HDE processes. For additional information on devices 

cleared through the 510(k) premarket review process, see GAO, Medical Devices: FDA 

Should Take Steps to Ensure That High-Risk Device Types Are Approved through the 

Most Stringent Premarket Review Process, GAO-09-190 (Washington, D.C.: Jan. 15, 

2009), and the Related GAO Products section at the end of this report.

11These standards provide an overall framework for establishing and maintaining internal 

controls and for identifying and addressing major performance and management 

challenges and areas at greatest risk of fraud, waste, abuse, and mismanagement. See 

GAO, Standards for Internal Control in the Federal Government, GAO/AIMD-00-21.3.1

(Washington, D.C.: November 1999), and its supplemental guide, Internal Control 

Management and Evaluation Tool, GAO-01-1008G (Washington, D.C.: August 2001).
 

Page 5 GAO-12-225 Pediatric Medical Devices

systems.12 Based on our examination, we generally found the data that 

FDA maintains on the number of approved PMA and HDE devices 

labeled for use in pediatric patients not to be sufficiently reliable for our 

purposes and, as a result, in order to identify pediatric devices, we 

reviewed FDA documents—the approval order, approved labeling, and 

summary of safety and effectiveness data—that FDA makes publicly 

available for each approved PMA and HDE device.13 Each document 

contains the approved indication for use statement, which specifies the 

disease and population the device is intended to treat. However, the 

indication for use statement may not explicitly state if a device can be 

used in pediatric patients; in these cases, FDA may still consider the 

device to be labeled for use in pediatric patients based on other 

information in the labeling or summary documents. Given the extent of 

information and expertise needed to determine if a device is labeled for 

use in pediatric patients, for our analysis we considered pediatric devices 

to be those explicitly indicated for use in pediatric patients when the 

indication for use statement in these approval documents (1) specified a 

population that included patients age 21 years or younger, (2) stated that 

the device was for pediatric patients, or (3) stated that it was for skeletally 

immature patients.14

 

12We also examined FDA’s ability to readily identify additional information that FDAAA 

required FDA to annually report to congressional committees, including the number of 

approved devices for which there is a pediatric subpopulation that suffers from the disease 

or condition that the device is intended to treat, diagnose, or cure and the number of 

approved devices that were exempted from the fee charged by FDA to review a PMA 

application because the device was intended solely for a pediatric population.

We reviewed approval documents for PMA devices, 

13To the extent possible, we compared FDA data on pediatric devices that the agency 

generated from its Center Tracking System with data generated from our PMA and HDE 

document review to corroborate or provide perspective on the data FDA provided. 

14A device’s approved indication for use statement describes the disease and population 

the medical device is intended to treat. Although the upper age limit used to define the 

pediatric population varies among experts, FDA issued guidance in 2004 stating that 

including adolescents up to the age of 21 years in the pediatric population is consistent 

with the definition found in several sources. We counted patients age 21 years or younger 

as pediatric patients in our review of the indication for use statements because the Food, 

Drug, and Cosmetic Act (as amended by FDAAA) defines pediatric patients, for purposes 

of provisions related to HDE devices, as those who are age 21 years or younger at the 

time of diagnosis or treatment. See 21 U.S.C. § 360j(m)(6)(E)(i). FDA has also concluded 

that for purposes of a provision requiring sponsors to submit information with PMA and 

HDE device applications, the term “pediatric patients” refers to patients who are age 

21 years or younger at the time of diagnosis or treatment. See 75 Fed. Reg. 16,365, 

16,366-67 (Apr. 1, 2010). 
 

Page 6 GAO-12-225 Pediatric Medical Devices

including PMA supplements called panel-track supplements,15 approved 

in fiscal years 2006 through 2011 and HDE devices approved in fiscal 

years 2001 through 2011.16

In addition, to determine how many humanitarian use device (HUD) 

designations—a required first step for FDA approval to market an HDE 

device—FDA granted for devices used to treat diseases that occur in

pediatric populations since FDAAA was enacted, we analyzed data 

provided by FDA for all HUD requests submitted and designated in fiscal 

years 2001 through 2011. We interviewed FDA officials regarding 

methods for tracking HUD requests and designations and determined that 

the data were sufficiently reliable for our purposes. We also interviewed 

FDA officials and reviewed FDA data to identify the HDE devices 

exempted from the HDE profit prohibition and determined that these data 

were sufficiently reliable for our purposes.

We conducted this performance audit from February 2011 through 

December 2011 in accordance with generally accepted government 

auditing standards. Those standards require that we plan and perform the 

audit to obtain sufficient, appropriate evidence to provide a reasonable 

basis for our findings and conclusions based on our audit objectives. We 

believe that the evidence obtained provides a reasonable basis for our 

findings and conclusions based on our audit objectives.

Medical devices include items used for the diagnosis, cure, mitigation, 

treatment, or prevention of a disease. These devices range from simple 

tools like bandages and surgical clamps to complicated devices like 

pacemakers; unless exempt by FDA regulations, devices are generally 

 

15A PMA panel-track supplement is required if substantial clinical data are necessary to 

provide reasonable assurance of the safety and effectiveness to change the indication of 

an approved PMA device—for example, to add a pediatric population to a PMA device 

already approved for use in adults. 

16We reviewed information for PMA devices (including PMA panel-track supplements) 

approved since fiscal year 2006 in order to include information for 2 years prior to 

enactment of FDAAA on September 27, 2007. This included a total of 81 devices 

approved before FDAAA was enacted and 110 devices approved after FDAAA was 

enacted. Because a much smaller number of HDE devices are approved each year, we 

included information for HDE devices approved since fiscal year 2001 listed on FDA’s 

website as of October 2011, for a total of 23 HDE devices approved before FDAAA was 

enacted and 8 devices approved after FDAAA was enacted.

Background 
 

Page 7 GAO-12-225 Pediatric Medical Devices

subject to FDA premarket review. Pediatric patients may be among those 

a device is intended to treat and generally are defined as those age 

21 years or younger. FDAAA included provisions to support and track 

pediatric device development.

Unless exempt by regulation, medical devices are generally subject to 

FDA premarket review before they may be legally marketed in the United 

States. The extent of the premarket review is generally correlated with the 

level of risk the device poses.17 Applications to market medical devices 

are primarily submitted to and reviewed by FDA’s Center for Devices and 

Radiological Health (CDRH).18

Under the PMA premarket review process, FDA reviews and makes 

approval decisions on applications for class III devices.19

 

17FDA categorizes medical devices into three classes based on the level of risk the device 

poses and the controls necessary to provide reasonable assurance of its safety and 

effectiveness. Class I devices include those with the lowest risk (such as forceps) and 

class III devices include those with the greatest risk (such as pacemakers). See 21 U.S.C. 

§ 360c; 21 C.F.R. pts. 862-892 (2011).

Among other 

things, PMA applications must include the indication for use—including 

the disease and population the medical device is intended to treat—and 

evidence (typically clinical data) providing reasonable assurance that the 

device is safe and effective. After an initial screening of an application 

and determination that the review should proceed, FDA’s reviewers 

conduct a scientific review of the application and may refer applications to 

an external advisory committee composed of individuals with expertise in 

a particular medical specialty, such as cardiovascular or neurology—

known as a medical specialty panel. FDA charges manufacturers a fee to 

18Although FDA’s Center for Biologics Evaluation and Research also reviews medical 

device applications, including those for devices used in the testing and manufacturing of 

biological products, FDA officials noted that the majority of pediatric medical device 

applications are reviewed by CDRH and no pediatric HDE applications had, at the time of 

our review, been reviewed by the Center for Biologics Evaluation and Research.

19Some class III device types on the market before the enactment of the Medical Device 

Amendments of 1976 do not currently require PMA approval for marketing. FDA has been 

taking steps to address these device types; once this process is completed, all class III 

devices will be required to go through the PMA review process. For additional information, 

see GAO, Medical Devices: FDA’s Premarket Review and Postmarket Safety Efforts, 

GAO-11-556T (Washington, D.C.: Apr. 13, 2011).

Medical Device Premarket 

Review Process
 

Page 8 GAO-12-225 Pediatric Medical Devices

review PMA applications, but PMA applications for devices intended 

solely for pediatric use are exempt from any fees.20

For devices that have been approved through the PMA process, a 

manufacturer that wants to add a pediatric population (or otherwise 

significantly change the approved indication for use) must submit and 

receive FDA approval for a PMA panel-track supplement.21 FDA charges 

manufacturers a fee to review PMA panel-track supplements, but those 

intended solely for pediatric use are exempt.22

In 1990, Congress authorized FDA to exempt from the premarket review 

requirement to demonstrate effectiveness certain medical devices in 

order to provide an incentive for the development of devices that treat or 

diagnose rare diseases or conditions, including those in children.23

Obtaining HDE approval is a two-part process:

Manufacturers of such devices still must show that the probable benefit 

outweighs the risk of using the device and that the device will not expose 

patients to unreasonable or significant risk of illness or injury. These 

devices, called HUDs, are intended to treat or diagnose a disease or 

condition that affects fewer than 4,000 individuals in the United States. In 

order to market an HUD in the United States, an HDE application must be 

submitted to and approved by FDA.

• A manufacturer must first request and obtain HUD designation from 

FDA’s Office of Orphan Product Development. A device manufacturer 

requests HUD designation by providing a description of the rare 

disease or condition for which the device is to be used, the proposed 

indication or indications for use of the device, the reasons why such 

therapy is needed, and a description of the intended patient 

population along with data to support that there are fewer than 4,000 

of these patients within the United States. The device manufacturer 

 

20For fiscal year 2012, the standard fee for a PMA application is $220,050.

21Although FDA reviews and approves other types of PMA supplements, a panel-track 

supplement is required if the manufacturer wants to change the indication for use of an 

approved PMA device to add a pediatric population, according to FDA officials.

22For fiscal year 2012, the standard fee for a PMA panel-track supplement is $165,038.

23Pub. L. No. 101-629, § 14(a), 104 Stat. 4511, 4524-25 (1990) (codified as amended at 

21 U.S.C. § 360j(m)).
 

Page 9 GAO-12-225 Pediatric Medical Devices

must also provide a description of the device and scientific rationale 

for the use of the device for the rare disease or condition.

• Once the HUD designation is granted, the manufacturer can then 

seek device approval by submitting an HDE application. In addition to 

including the indication for use, the HDE application must include a 

detailed description of the device, along with evidence demonstrating 

device safety, and data and a rationale supporting the probable 

benefit of the device. Device labeling is also reviewed and must 

include a statement indicating that the device is a humanitarian 

device.24 According to FDA guidance, criteria for HDE approval are 

focused on safety, probable benefit, and lack of a comparable device. 

FDA does not charge manufacturers a fee to review HDE 

applications.25

Manufacturers of HDE devices are allowed to recover certain 

development and production costs but are generally prohibited from 

making a profit on their device. Unless exempt, HDE applications must 

include a cost assessment to demonstrate that the amount charged for 

the HDE device does not exceed the cost of research and development, 

fabrication, and distribution. After FDA approval, in order for an HDE 

device to be used in patient care, it must be reviewed and approved by an 

institutional review board (IRB) to assess the safety of the device.26

If FDA review results in premarket approval, FDA issues an approval 

order to the manufacturer, which includes the approved indication for use 

statement. FDA also approves all printed material accompanying the 

device—known as the device labeling. The approval order, labeling, and 

a summary of safety and effectiveness data are available on FDA’s 

 

24The HDE device label must include the following statement: “Humanitarian Device. 

Authorized by Federal law for use in the [treatment or diagnosis] of [specify disease or 

condition]. The effectiveness of this device for this use has not been demonstrated.” 

25See 21 U.S.C. § 379j(a)(2)(B)(i).

26The IRB is a committee that has been formally designated to approve, monitor, and 

review biomedical and behavioral research involving humans with the aim to protect the 

rights and welfare of the research subjects. Although IRBs generally oversee clinical 

investigations, for HDE devices IRBs are required to review and approve the use of the 

device. The IRB may review each use of an HDE device or use discretion to determine 

general rules for use of the HDE device after initial review. If an HDE device is used in an 

emergency situation, subsequent written notice to the IRB must be provided. See 

21 U.S.C. § 379j(m)(4).
 

Page 10 GAO-12-225 Pediatric Medical Devices

website; all three documents contain the approved indication for use 

statement.

Although the upper age limit experts use to define the pediatric population 

varies, for purposes of certain statutory provisions for PMA and HDE 

device applications, FDA considers pediatric patients as those who are 

age 21 years or younger at the time of diagnosis or treatment.27 FDA 

issued guidance in 2004 that specifies age ranges for four main pediatric 

subpopulations: neonate, infant, child, and adolescent (see table 1).28

According to FDA officials, the agency defined an additional 

subpopulation, transitional adolescents, for its internal application review 

purposes. These officials noted that in some cases patients in this age 

range may not require special considerations compared to adults.29 A 

pediatric medical device may be indicated for use in more than one 

pediatric subpopulation.

 

2721 U.S.C. § 360j(m)(6)(E)(i) and (ii) define pediatric patients as age 21 years or younger 

at the time of diagnosis or treatment and specifies categories of pediatric subpopulations. 

21 U.S.C. § 360e-1(a), which requires sponsors of PMA and HDE applications and 

supplements to submit information on pediatric subpopulations and pediatric patients, 

uses the same definition of “pediatric subpopulations” but does not include a definition of 

“pediatric patients.” Because no other definition of “pediatric patients” is included in statute 

and because the definitions of “pediatric patients” and “pediatric subpopulations” in section 

360j are consistent, FDA has concluded that the term “pediatric patients” for purposes of 

required information for PMA and HDE applications refers to patients who are 21 years of 

age or younger at the time of diagnosis or treatment. See 75 Fed. Reg. 16,365, 16,366-67

(Apr. 1, 2010).

28See FDA, Guidance for Industry and FDA Staff: Premarket Assessment of Pediatric 

Medical Devices. (Rockville, Md.: 2004). FDA, in its guidance, recognized that factors 

other than a patient’s numerical age may often be appropriate to consider in defining a 

pediatric population.

29FDA developed this subpopulation for determining if certain devices indicated for 

patients in the age range should be considered differently than adults. FDA officials stated 

that they will issue guidance on pediatric subpopulations that will include the transitional 

adolescent category.

Pediatric Populations for 

Medical Devices
 

Page 11 GAO-12-225 Pediatric Medical Devices

Table 1: Pediatric Subpopulations for Medical Devices and Related Ages

Pediatric subpopulation

FDA guidance on approximate age 

range for this subpopulation 

Neonate (newborn) Birth to 1 month

Infant Greater than 1 month to 2 years

Child Greater than 2 years to 12 years

Adolescent Greater than 12 years to 21 yearsa

Source: GAO analysis of FDA information.

 

Notes: This table presents FDA’s guidance on the approximate age ranges for four main pediatric 

subpopoulations as reported in FDA’s Guidance for Industry and FDA Staff: Premarket Assessment 

of Pediatric Medical Devices (2004). According to FDA officials, for internal application review 

purposes, FDA uses slightly different age ranges for these main subpopulations—for example, 

2 years to less than 12 years for children.

a

For internal application review purposes, FDA identified another subpopulation, transitional 

adolescent, to include those age 18 to 21 years.

According to FDA officials, some pediatric devices are indicated for use in 

both pediatric and adult patients, such as devices with an indication for all 

patients with a given condition above a certain age. Additionally, a 

device’s indication for use statement might not specify the age of the 

intended population, but based on additional clinical information that may 

be included in the approval documents, FDA might consider a device 

labeled for pediatric use. For example, the indication for use statement for 

an influenza diagnostic test might not specify a pediatric population, but 

FDA could consider the device labeled for patients of all ages. In addition, 

a device’s indication for use statement might specify a minimum blood 

vessel size or body weight rather than a certain patient age, and if 

pediatric patients meet the criteria, the device could be considered 

labeled for pediatric use.

To stimulate pediatric medical device development, FDAAA exempted 

HDE devices labeled for use in pediatric patients (including those for both 

pediatric and adult patients) from the HDE profit prohibition for 

applications approved on or after September 27, 2007, when FDAAA was 

signed into law.30

 

30To qualify for the exemption from the HDE profit prohibition, the sponsor must submit an 

HDE application on or before October 1, 2012.

In order to receive the exemption from the profit 

prohibition, HDE applications must include data to prove that the device 

FDAAA Provisions on 

Pediatric Medical Device 

Development
 

Page 12 GAO-12-225 Pediatric Medical Devices

can safely be used in a pediatric population and to support the number of 

individuals likely to use the device each year. Based on this information, 

FDA determines an upper limit on the number of devices that can be 

distributed or sold each year—to treat both pediatric and adult patients—

known as the annual distribution number (ADN). Manufacturers may only 

receive profits from the sale of HDE devices exempt from the profit 

prohibition and indicated for pediatric or pediatric and adult use up to the 

ADN. In addition, FDAAA requires that FDA’s Pediatric Advisory 

Committee annually review all HDE devices granted exemption from the 

profit prohibition to ensure that the exemption remains appropriate for the 

pediatric populations for which it is granted.31

FDAAA also required PMA and HDE applications to include, if readily 

available, a description of any pediatric population or subpopulation(s) 

that suffer from the disease or condition that the device is intended to 

treat, diagnose, or cure and the number of affected pediatric patients.

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.32

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.33

 

31Pub. L. No. 110-85, § 303(a)(3), 121 Stat. 823, 861-62 (2007) (codified at 21 U.S.C. 

§ 360j(m)(8)).

Pediatric device consortia receive 2-year grants, administered by FDA, to 

conduct certain activities for device projects, such as mentoring and 

managing the development process, assessing the scientific merit of a 

proposed device, and providing assistance and advice as needed on 

32Pub. L. No. 110-85, § 302, 121 Stat. 823, 859-60 (2007) (codified at 21 U.S.C. § 360e1). The third requirement applies only to PMA devices since HDE devices are already 

exempt from user fees. In addition, the report is required to include the FDA review times 

for these devices.

33FDAAA also authorized the use of contracts. Pub. L. No. 110-85, § 305, 121 Stat. 823, 

863-64 (2007).
 

Page 13 GAO-12-225 Pediatric Medical Devices

business development and navigating the regulatory process. In their 

demonstration grant applications, consortia were not only required to 

demonstrate their capability to develop pediatric devices—by having 

members with medical and scientific expertise in device development and 

relationships with manufacturers—but they were also required to identify 

two to five device projects that the consortia would directly support under 

the demonstration grant.34 Grantees must submit quarterly and annual 

reports to FDA describing how they have used grant funds to support 

innovation and the phase of development for the devices for which they 

provided assistance.35 Phases of development include, for example, 

concept, prototype (during which a model is developed), clinical (during 

which the device is tested), and marketing (during which the device goes 

through the regulatory process).36

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.

• Pediatric patients are composed of different subpopulations with 

differences in size, growth rate, metabolism, heart rate, activity level, 

and other differences. For example, younger pediatric patients—

neonates, infants, and children—have higher heart rates or respiratory 

rates than adults. This difference, as cited by some stakeholders, 

makes some devices used for adults, such as certain heart valves, not 

appropriate for use in young children; thus a device that has already 

been developed for an adult may have to be redesigned for a pediatric 

population. Another unique characteristic cited is the range of 

pediatric subpopulations and the difficulty in developing potentially 

different devices for each of these subpopulations. For example, a 

device that is developed for a toddler may not be appropriate for use 

in an adolescent, even if they have the same disease or condition. As 

 

34Directly supported device projects received a portion of the demonstration grant funding.

35Grantees are required to report on the directly supported projects that were proposed in 

the grant applications as well as on other device projects for which the consortia provided 

assistance, such as developing a prototype or assessing the scientific merit of a device 

project.

36FDA officials stated that the phases of development used in consortia reporting come 

from CDRH’s Total Product Lifecycle approach to reviewing medical devices.

Characteristics of the 

Population and 

Limited Economic 

Incentives Cited as 

Barriers to Pediatric 

Device Development
 

Page 14 GAO-12-225 Pediatric Medical Devices

a result, manufacturers may have to develop and test a different 

device for each subpopulation although the disease or condition may 

be the same.

• Recruiting pediatric patients to participate in clinical trials has also 

been cited as a challenge for manufacturers. Performing experimental 

procedures on children naturally raises concern for their safety, and it 

may be difficult to obtain parental consent on behalf of the child to 

conduct experimental procedures. This issue is compounded because 

the pediatric population is not just one population but rather many 

subpopulations. Therefore, manufacturers find it difficult to recruit 

enough children for each pediatric subpopulation over a reasonable 

time frame and within a manageable number of investigational sites to 

ensure enough test subjects to meet clinical trial requirements.

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. Stakeholders 

indicated that the cost of developing pediatric medical devices relative to 

the potential revenue generated by sales in a small market is a significant 

barrier to the development of new pediatric medical devices.

Stakeholders also noted that the cost of research and development for 

pediatric medical devices can be the same as for adult devices while the 

market size is often smaller. Given these factors, stakeholders report that 

the return on the investment to develop and test pediatric medical devices 

usually falls below the profit goals of most medical device manufacturers.

Stakeholders noted that the FDAAA provision allowing pediatric HDE 

devices to earn a profit is one helpful step to encourage pediatric medical 

device development; however, they indicated that this provision is not 

enough to address the small market for pediatric devices and the 

resources necessary to study a device in a pediatric population. An 

official from one device manufacturer that had a pediatric HDE device 

approved since FDAAA was enacted told us that the ability to earn a profit 

for the HDE device, while helpful, was not part of this manufacturer’s 

decision to develop and market it. Stakeholders noted that the maximum 

number of 4,000 individuals for HDEs limited the effectiveness of this 

incentive for development of devices for larger pediatric populations. 

Stakeholders also noted that incentives used for pediatric drug 

development would not necessarily work for pediatric medical device 

development. For example, patent protection for medical devices is not as 

meaningful an incentive as it is for the development of pediatric drugs 
 

Page 15 GAO-12-225 Pediatric Medical Devices

because the typical life cycle for medical devices (about 18 months) is 

significantly shorter than the typical life cycle for a drug.37

Other barriers identified by stakeholders included an incomplete 

understanding of unmet pediatric device needs and issues related to the 

medical device regulatory process.

• Several stakeholders commented that there is no consistent inventory 

of the devices that need to be developed for pediatric populations.

Although it is unclear who should maintain this inventory, FDAAA did 

require FDA to annually report on the number of devices approved in 

the preceding year that were labeled for use in pediatric patients or for 

which there is a pediatric subpopulation that suffers from the disease 

or condition that the device is intended to treat, diagnose, or cure. 

FDA has reported to Congress that the pediatric device data 

contained in these reports may help identify areas of pediatric device 

research and development that need to be addressed.38 However, 

FDA has issued only one of these reports to date.39

 

37Generally, it is possible to design a medical device in a number of different ways that 

could be marketed by different manufacturers. As noted by the medical device industry, 

the typical “life cycle” or the amount of time before competitors begin selling an alternative 

product of a device is 18 months. The Best Pharmaceuticals for Children Act authorizes 

FDA to provide an incentive of an additional 6 months of market exclusivity to drug 

manufacturers that conduct pediatric studies requested by FDA. See 21 U.S.C. § 355a; 

42 U.S.C. § 284m.

Furthermore, 

some of the information from which FDA would develop these reports 

may not be as useful as possible in determining unmet need. 

Specifically, manufacturers are to include the information on pediatric 

subpopulations in their premarket approval applications if readily 

available; however, one manufacturer organization stated that it was 

unclear about the specific information requirements, and thus 

38See Department of Health and Human Services, Agency for Healthcare Research and 

Quality, Food and Drug Administration, and National Institutes of Health, Report to 

Congress on the Plan for Expanding Pediatric Medical Device Research and Development

(Washington, D.C.: 2009).

39FDA issued a report for PMA and HDE devices approved in fiscal year 2008. 
 

Page 16 GAO-12-225 Pediatric Medical Devices

manufacturers may not be submitting useful information for tracking 

unmet pediatric device needs.40

• Stakeholders also cited as barriers to pediatric device development 

certain issues related to the medical device regulatory process in 

general. For instance, it was reported that the amount of time that it 

will take to get through the regulatory process cannot always be 

predicted, which limits the ability to appropriately budget for 

development. One manufacturer organization also noted that FDAAA 

authorized FDA to consider published data from adult studies and 

studies in different age groups when determining effectiveness for 

pediatric device approvals; this manufacturer organization commented 

that where appropriate, FDA should also use scientific evidence other 

than randomized controlled studies, such as well-documented case 

histories, to show probable benefit or demonstrate effectiveness.41

Recognizing the need to discuss the ways scientific research data can 

be used for pediatric medical device applications, FDA announced in 

November 2011 that it would hold a public workshop to discuss the 

use of scientific research data, including published scientific literature, 

to support and establish pediatric indications for medical devices.42

Stakeholders also noted barriers that were unique to HDE devices, 

including pediatric HDE devices. One manufacturer organization stated 

that FDA has provided no general guidance to manufacturers regarding 

the type or level of evidence that must be developed to demonstrate that 

an HDE device meets the probable benefit standard. According to the 

organization, this lack of guidance ultimately hinders the use of the HDE 

program as a pathway to market devices that treat or diagnose diseases 

and conditions that affect fewer than 4,000 patients, including pediatric 

patients. According to stakeholders, difficulties in obtaining insurance 

coverage for HDE devices are also a barrier to development because 

HDE devices are approved by demonstrating safety but not effectiveness.

 

40FDA officials stated that the agency was working on draft guidance for tracking pediatric 

device information; however, at the time of our review, FDA had not issued this guidance.

In addition, FDA is working to finalize a proposed rule regarding the requirement for 

submission of information on pediatric subpopulations that suffer from a disease or 

condition that a device is intended to treat, diagnose, or cure. See 75 Fed. Reg. 16,365 

(Apr. 1, 2010).

41Pub. L. No. 110-85, § 302, 121 Stat. 823, 860 (codified at 21 U.S.C. § 360e-1(b)). 

42See 76 Fed. Reg. 67,463 (Nov. 1, 2011).
 

Page 17 GAO-12-225 Pediatric Medical Devices

Although the two manufacturers’ representatives we spoke with reported 

experiencing frustration with the HDE approval process, they noted that 

overall, their interaction with FDA reviewers was positive in that FDA 

responded to their questions in a timely manner and provided specific 

guidance where necessary.

Finally, stakeholders stated that individuals with innovative ideas for new 

pediatric medical devices do not necessarily know what steps to take to 

bring their ideas for pediatric medical devices to the market, and it would

be helpful to connect these individuals with manufacturers and to help 

them navigate the device development and regulatory process. 

Individuals with ideas for medical devices range from engineers to 

clinicians, who often need to work together to develop and manufacture a 

pediatric medical device. In addition, it is necessary to have funds to 

sustain development and an understanding of how to navigate the 

regulatory process. A commonly cited example of the challenges faced by 

pediatric device innovators is the experience developing the titanium rib, 

which took about 14 years to develop before it was approved for the 

market in part because its inventor, a physician who saw the need for 

such a device, did not have the information or resources to navigate the 

development and regulatory process.43 FDA officials and other 

stakeholders reported that the pediatric device consortia have helped to 

address this barrier. In addition, FDA officials reported that other efforts 

by FDA to organize early meetings before device manufacturers submit 

applications to clarify required clinical information and meetings with 

groups such as the pediatric orthopedic surgeons have also helped 

device innovators navigate the device development and regulatory 

process.

 

43FDA approved the Vertical Expandable Prosthetic Titanium Rib for market through the 

HDE process in August 2004. 
 

Page 18 GAO-12-225 Pediatric Medical Devices

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).44 The consortia receiving these grants

provided assistance that included review of projects by clinical experts to 

evaluate the devices’ usefulness in a clinical environment and help 

connecting innovators with manufacturers as well as potential sources of 

additional federal grant funding.45 The consortia also conducted other 

activities to support pediatric medical device development and connect 

individuals who have ideas for medical devices with information and 

resources, such as lectures, forums, and relevant conferences.46

 

44Although the pediatric device consortia grant program was authorized as a 

demonstration grant program under FDAAA, appropriations were not designated for the 

program in committee reports until fiscal year 2009. 

45Federal grant funding included grants through the National Institutes of Health.

46In 2011, FDA awarded grants for fiscal years 2011 and 2012 totaling $5 million to three 

pediatric device consortia; the grant period for these awards had not yet started at the 

time of our review. Two of the grantees—Michigan Pediatric Device Consortium-PMDI and 

University of California at San Francisco Pediatric Device Consortium—had previously 

received pediatric device consortia grants; the third grantee—Atlanta Pediatric Device 

Consortium—had not previously received a pediatric device consortium grant. FDA also 

awarded an additional $500,000 to MISTRAL Device Consortium. 

Pediatric Device 

Consortia Have 

Assisted over 100 

Device Projects; Many 

Were in Early 

Development 
 

Page 19 GAO-12-225 Pediatric Medical Devices

Table 2: Summary of Pediatric Device Consortia Grant Amounts and Funded Activities, Fiscal Years 2009 and 2010

Pediatric device consortia grantees 

(location) 

Grant amountsa Number of device

(in thousands) projects assisted

Examples of activities to facilitate 

pediatric device development

Michigan Pediatric Device Consortium

(Ann Arbor, MI)

$2,311 63 Hired postgraduate professionals with 

medical, dental, doctorate, or masters 

degrees for a 1-year fellowship to focus on 

bringing device innovations to the market 

MISTRAL Device Consortiumb 500

(Menlo Park, CA)

17 Organized two teleconferences on issues 

surrounding neonatal intensive care units 

for the purposes of developing new devices

Pediatric Cardiovascular Device 

Consortium

(Boston, MA)

1,102 9 Held monthly forums for device innovators 

to discuss new device innovations with 

experts such as engineers and scientists

University of California at San Francisco 

Pediatric Device Consortium

(San Francisco, CA)

1,087 18 Held weekly forums to discuss new device 

innovations

Total

c

$5,000 107

Source: GAO analysis of FDA information.

Notes: The total number of device projects assisted by the consortia is not proportional to the amount 

each consortium received in grant funding. Some device projects were more complex and needed 

more consortium resources than others. In addition, some consortia received funding outside of the 

demonstration grants to support their projects. This table presents grant funding for fiscal years 2009 

and 2010 and the number of device projects assisted using those funds.

a

Grant award amounts were rounded to the nearest thousand.

b

Information presented for MISTRAL Device Consortium is for activities supported with funds from a 

grant made in fiscal year 2010.

c

This innovator forum is scheduled two times per week and is open to anyone with a device idea or 

who wants to learn about device development. A 1-hour agenda is created where ideas are heard 

and consulted on. In addition, expert speakers are also brought in who can speak on manufacturing a 

device or taking a device through the regulatory process.

For the device projects assisted by the consortia, the phase of 

development varied. Although the consortia assisted a total of 107 device 

projects, 34 were inactive or were device projects that the consortia were 

no longer assisting at the time of our review.47 Of the 73 active device 

projects, about half (36) were in the prototype phase (see table 3).48

 

47In some cases inactive device projects stopped receiving consortia support because 

they could not obtain outside funding or there were complex development challenges. 

In 

48Active projects were those that were reported in the most recent consortia reports 

available at the time of our review, supplemented by additional information provided by 

FDA. Projects that we considered inactive were those that the consortia had mentored or 

supported at some point with grant funds but that were no longer receiving consortia 

assistance at the time of our review.
 

Page 20 GAO-12-225 Pediatric Medical Devices

order to monitor the progress of the device projects the pediatric consortia 

assisted, FDA requires each consortium to report on the development 

phase of each device project—including the directly supported device 

projects included in the grantees’ applications as well as other device 

projects that received consortia assistance—on a quarterly and annual

basis. Throughout the grant period, device projects were presented to the 

consortia and consortia members determined the kind of assistance to 

provide for development of these devices. The assistance provided 

depended on the device’s phase of development. For example, for a 

device in the concept phase a consortium could assess its scientific merit 

to see if there is a need for the device or if it makes sense to develop it; 

for devices that were further along, a consortium could help produce 

prototypes. For devices in the marketing phase, the consortia could 

consult on next steps, including working with FDA, a step which the 

developer needs to take to market a device. As of the most recent 

grantee reports available in September 2011, 3 of the 107 devices that 

were assisted by the consortia were commercially available and 1 had 

received HUD designation.
 

Page 21 GAO-12-225 Pediatric Medical Devices

Table 3: Number of Device Projects by Development Phase That Were Mentored or 

Supported by the Pediatric Device Consortia 

Phase of development Total

Active

Concept

(development of an idea for a device)

8 

Prototype

(the design and development phase where a model is used to conceive 

the concept)

36

Preclinical

(testing a prototype in laboratories or animals)

20

Clinical (testing the device in humans) 4 

Manufacturing

(production of the device)

2 

Marketing

(includes the regulatory process and putting the device on the market)

0 

Commercial use

(use of the device by the general public)

3 

Active total 73 a

Inactive total 34 b

Total inactive and active 107

Source: GAO analysis of FDA information.

Notes: The table presents the total number of device projects assisted by all four consortia. Data 

presented cover device projects that were assisted with funds from grants made in fiscal years 2009 

and 2010.

a

Active projects were those that were reported as supported with funds from grants made in fiscal 

year 2010 in the most recent consortia reports available at the time of our review in 2011, 

supplemented by additional information provided by FDA.

b

Projects that we considered inactive were those that the consortia had mentored or supported at 

some point during their grant periods but that were no longer receiving consortia assistance with 

fiscal year 2010 funds. In some cases, inactive device projects stopped receiving consortia support 

because they could not obtain outside funding or there were complex development challenges.

Of the 107 device projects assisted by the consortia, 13 were directly 

supported—that is, they were identified in the grant awards to receive a 

portion of the grantee’s demonstration grant funding. Table 4 lists these 

directly supported device projects, which were identified by the grantees 

in their applications and selected by FDA to receive support through the 

consortia grants. FDA’s evaluation criteria included (1) the significance of 

the device to address pediatric needs, (2) how well the project was 

designed, (3) the likelihood for marketing success, (4) the expertise and 

training of project leaders, and (5) evidence that the institution where the 

consortium was located supported the project.
 

Page 22 GAO-12-225 Pediatric Medical Devices

Table 4: Medical Device Projects Presented in Consortia Grant Applications and Funded in Fiscal Years 2009 and 2010 

Pediatric device 

consortia grantees Device project Condition treated or use 

Michigan Pediatric Device 

Consortium 

Bowel lengthening device Treats short bowel syndrome

Nonthrombogenic and antiseptic 

catheters for infants

a

Treats clotting and infection problems with catheters in children

MISTRAL Device Consortium Pyloromyotomy surgical tool Used in making laparoscopic pyloromyotomy safer and easierb

Esophageal atresia surgical tool

 

Treats esophageal atresiac

Neurosurgical articulated tools

 

Used in pediatric brain surgery

NICU dashboard Used to monitor and synthesize multiple pediatric vital sign 

inputs

Catheter for peripheral nerve 

blocks

Used to secure catheter placement in pediatric pain 

management

Pediatric Cardiovascular Device 

Consortium

Septal cincher Used to reduce the opening of a cardiac valve

Biodegradable valve ring for 

children

Used in repair of cardiac valve

Pediatric ultrasound imaging for 

diagnostics and surgical planning

Used for cardiac imaging

PediVAS pediatric circulatory 

assist device

Used in acute and temporary life support for infants and small 

children

University of California at San 

Francisco Pediatric Device 

Consortium

MAGNETIC MINI-MOVER for 

pectus excavatum: 3rd 

generation magnimplant device

Treats pectus excavatum (sunken chest)

ROBOIMPLANT (remotely 

operated bionic ortho implant) for 

scoliosis

d

Treats congenital and acquired spine disorders, such as early 

onset scoliosis 

Source: GAO analysis of FDA information.

Notes: This table presents information on the device projects that were directly supported under the 

pediatric device consortia grants—that is, they were proposed in the grantees’ applications and 

selected by FDA to receive support under the grants. All device projects were developing devices for 

use in pediatric patients.

a

Short bowel syndrome represents a loss of sufficient length of intestine to allow for the absorption of 

nutrients.

b

Laparoscopic pyloromyotomy is a minimally invasive surgery that makes an incision in the pylorus—

the region of the stomach that connects to the small intestine.

c

Esophageal atresia is a congenital medical condition that causes the esophagus to end rather than 

connecting normally to the stomach. 

d

Pectus excavatum is also known as sunken chest and is a congenital chest wall deformity in which 

several ribs and the sternum grow abnormally, producing a concave, or caved-in, appearance in the 

anterior chest wall.
 

Page 23 GAO-12-225 Pediatric Medical 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. 

Under standards for internal control in the federal government, relevant, 

reliable, and timely information should be available for external reporting 

purposes; however, FDA has not consistently collected reliable and timely 

information needed to report on pediatric devices.49 In response to 

FDAAA, FDA developed an electronic flag within its Center Tracking 

System (CTS) in 2008, to help CDRH internally identify PMA and HDE 

devices that were labeled for use in pediatric patients.50 According to FDA 

officials, the agency’s medical device reviewers should check the 

pediatric flags during the PMA and HDE process based on all of the 

information available in the application. However, FDA officials reported 

that these electronic flags are not consistently applied, and that the 

agency does not provide formal training or guidance to its device 

reviewers regarding proper implementation of the pediatric flags. 

Therefore, information from FDA’s tracking system is not sufficiently 

reliable to identify PMA and HDE devices labeled for use in pediatric 

patients.

 

49See GAO/AIMD-00-21.3.1 and GAO-01-1008G. 

50CTS is a web-based system used by CDRH for tracking premarket documents through 

the review process. According to FDA officials, the agency started using CTS in 2004 and 

implemented the pediatric flags in 2008.

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
 

Page 24 GAO-12-225 Pediatric Medical Devices

In the absence of reliable information from its internal tracking system that 

could be retrieved in a timely manner to identify approved PMA and HDE 

devices labeled for use in pediatric patients, FDA reported that staff 

completed labor-intensive file reviews—which included examining 

voluminous application files as well as discussion with FDA reviewers—to 

complete its first annual report.51 This report, which covered devices 

approved in fiscal year 2008, did not specify the criteria FDA used to 

identify devices labeled for use in pediatric patients in its manual data 

collection process. An FDA official involved in the preparation of the fiscal 

year 2008 report told us that FDA staff considered devices to be labeled 

for pediatric patients if FDA officials determined that the devices were for 

patients younger than 18 years of age; however, this criterion was not 

explicit in the report. Instead, FDA’s report noted that a committee report 

described pediatric medical devices as those used to treat or diagnose 

diseases or conditions in patients from birth through age 21.52

In addition, FDA does not have readily available, reliable information to 

identify other pediatric information that FDAAA required the agency to 

report annually. Specifically, FDA officials said that the agency does not 

have reliable information to readily identify and report on the number of 

PMA and HDE devices approved each year for which there is a pediatric 

subpopulation that suffers from the disease or condition that the device is 

Further, in 

the absence of documented criteria used to prepare the fiscal year 2008 

report, FDA officials involved in preparing the combined fiscal years 2009-

2010 report at the time of our review were not aware of the specific 

criteria used for the fiscal year 2008 report and, as a result, created their 

own criteria and labor-intensive file review for identifying and reporting on 

approved PMA and HDE devices labeled for use in pediatric patients. As 

of October 2011, the combined fiscal years 2009-2010 report had not 

been issued; FDA officials reported that it was in the agency’s internal 

review and clearance process.

 

51See FDA, Report to Congress: Premarket Approval of Pediatric Uses of Devices—

FY2008 (March 2009), accessed October 31, 2011, 

www.fda.gov/downloads/MedicalDevices/.../UCM268801.pdf. 

52The report, H.R. Rep No. 110-225, was associated with a version of FDAAA that did not 

become law.
 

Page 25 GAO-12-225 Pediatric Medical Devices

intended to treat, diagnose, or cure.53

Without readily available information on the approved PMA and HDE 

devices labeled for use in pediatric patients or the number of approved 

devices for which a pediatric subpopulation might be indicated, FDA 

cannot easily identify and aggregate data to complete statutorily required 

reporting in a consistent and timely manner. The inconsistent information 

presented in these reports makes it more difficult for policymakers, 

industry stakeholders, and innovators to identify what opportunities and 

needs exist for devices to diagnose and treat pediatric patients.

Furthermore, FDAAA required FDA 

to annually report the number of PMA devices exempt from a user fee 

because they were solely intended for pediatric use. Although the agency 

reported that none of the devices approved in fiscal year 2008 were 

exempt from a user fee for this reason, FDA officials stated that as a 

result of our inquiry they reevaluated some of the PMA applications for 

which user fees were paid and identified certain PMA applications from 

other years that may not have properly received the user fee exemption.

FDA officials said they were in the process of providing refunds to 

applicants that should have been exempt from the user fee.

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.54

 

53In April 2010, FDA issued a proposed rule and a direct final rule implementing the 

FDAAA requirement for HDE and PMA device applications and supplements to include 

this information, if readily available. However, FDA withdrew the direct final rule after 

receiving significant adverse comments. According to FDA officials, the agency is in the 

process of reviewing the comments before issuing a final rule. See 75 Fed. Reg. 16,347 

(Apr. 1, 2010); 75 Fed. Reg. 16,365 (Apr. 1, 2010); 75 Fed. Reg. 41,986 (July 20, 2010).

Specifically, our review 

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. However, the approved 

indication for use statements for most of the 118 PMA and HDE devices 

approved since FDAAA was enacted do not specify the age of the 

intended patient population. Specifically, the indication for use statement 

54See apps. II and III for additional information on PMA and HDE devices approved before 

and after FDAAA was enacted that were explicitly indicated for use in pediatric patients.

Fifteen PMA and 3 HDE 

Devices Have Been 

Explicitly Indicated for 

Use in Pediatric Patients 

since FDAAA Was Enacted
 

Page 26 GAO-12-225 Pediatric Medical Devices

was silent on the age of the intended patient population for 75 percent of 

PMA devices and 38 percent of HDE devices approved during that time 

(see fig. 1). Given that a device’s indication for use statement may not 

explicitly state the age of the intended patient population or specify if it is 

for pediatric patients, additional pediatric devices could exist because 

other labeling and clinical information can be used to determine if the 

device can be used in pediatric patients.55

 

55Based on our review of additional labeling information, at least 1 HDE device and 8 

original PMA devices approved since fiscal year 2008 with indication for use statements 

that were silent on the age of the intended patient population were approved based on 

clinical studies that included one or more subjects age 21 or younger and did not include 

contraindications stating that the device should not be used in pediatric patients. At least 

12 additional devices with indication for use statements that were silent on the age of the 

intended patient population included contraindication statements stating that the device 

should not be used in or was not tested in patients under age 18—that is, the device could 

potentially be used in patients age 18 to 21 years and older. 
 

Page 27 GAO-12-225 Pediatric Medical Devices

Figure 1: PMA and HDE Devices Approved in Fiscal Years 2008 through 2011 by Indicated Patient Population

Notes: Totals do not add to 100 percent because of rounding.

In this figure, devices approved through FDA’s premarket approval (PMA) and humanitarian device 

exemption (HDE) processes were considered to be for pediatric patients if they were explicitly 

indicated for use in pediatric patients—that is, if the approved indication for use statement 

(1) specified a population that included patients age 21 years or younger, (2) specifically stated it was 

for pediatric use, or (3) stated it was for skeletally immature patients. Devices explicitly indicated for 

use in pediatric patients include those explicitly indicated for use in pediatric patients or those for both 

pediatric and adult patients. 

Adult only devices are those explicitly indicated for use in adult patients—that is, (1) if the indication 

for use statement specified a population that only included patients age 22 or older or (2) if the 

indication for use statement did not specify an age, but specifically stated it was for adult use.

Silent means devices for which the indication for use statement did not specify the age of the 

intended patient population.

a

PMA devices include original and panel-track supplement devices.

Of the 18 PMA and HDE devices explicitly indicated for use in pediatric 

patients since FDAAA was enacted, most were explicitly indicated for 

adolescents. The majority of those indicated for adolescents were 

indicated for patients age 18 and older. Nine devices (50 percent) were 

indicated solely for patients age 18 or older—the age group FDA 
 

Page 28 GAO-12-225 Pediatric Medical Devices

considers to be transitional adolescents for internal application review 

purposes—and 2 of those were indicated solely for patients age 21 or 

older.56 Two devices were explicitly indicated for children and none were 

explicitly indicated for neonates or infants (see table 5). In addition, the 

most common medical specialties of devices explicitly indicated for use in 

pediatric patients were microbiology (39 percent); cardiovascular 

(22 percent); anesthesiology and ear, nose, and throat (11 percent each); 

and clinical chemistry, ophthalmology, and gastroenterology (6 percent 

each).57

 

56FDA officials told us that during their premarket review they consider some devices 

indicated for patients age 18 to 21 years—transitional adolescents—the same as adult 

devices. However, because patients age 21 years or younger are considered pediatric 

under the definition in 21 U.S.C. § 360j(m)(6)(E)(i), devices labeled for patients age 19, 

20, or 21 are considered pediatric devices for certain purposes.

57We identified each device’s medical specialty based on data from FDA’s product code 

classification database—which contains device names and product codes assigned based 

upon the medical device product classification designated under 21 C.F.R. Parts 862-892. 

The database did not include the medical specialty for some device product codes; for 

these we used information on the medical specialty provided by FDA.
 

Page 29 GAO-12-225 Pediatric Medical Devices

Table 5: Approved PMA and HDE Devices Explicitly Indicated for Use in Pediatric Patients since FDAAA Was Enacted, by 

Pediatric Subpopulation

Pediatric subpopulationa

Adolescent

Device type Neonate Infant Child

Twelve years to 

less than 18 years

Transitional 

adolescent 

(18 to 21 years)

Pediatric 

subpopulation not 

specified b

PMA (n=15)

c

0 0 2 4 12 3 

HDE (n=3) 0 0 0 1 2 1 

Total (n=18) 0 0 2 5 14 4 

Source: GAO analysis of FDA documents.

Notes: This table presents PMA and HDE devices approved in fiscal years 2008 through 2011. 

FDAAA was enacted September 27, 2007; no devices approved through FDA’s premarket approval 

(PMA) and humanitarian device exemption (HDE) processes were approved in fiscal year 2007 after 

FDAAA was enacted. We considered PMA and HDE devices to be for pediatric patients if they were 

explicitly indicated for use in pediatric patients—that is, if the approved indication for use statement 

(1) specified a population that included patients age 21 years or younger, (2) specifically stated it was 

for pediatric use, or (3) stated it was for skeletally immature patients. Devices explicitly indicated for 

use in pediatric patients include those explicitly indicated for use in pediatric patients or those for both 

pediatric and adult patients.

a

A device can be indicated for more than one pediatric subpopulation. For purposes of this table, 

pediatric subpopulation ages are those that FDA uses for internal tracking purposes: neonate (birth to 

28 days), infant (29 days to <2 years), child (2 years to <12 years), adolescent (12 years to 

<18 years), and transitional adolescent (18 years to <=21 years).

b

Nine of the devices were explicitly indicated solely for transitional adolescents and adults, seven of 

which were indicated for patients age 18 or older and two of which were indicated for patients age 21 

or older.

c

The indication for use statement for four devices did not specify the pediatric subpopulation, but one 

was indicated solely for pediatric patients and three were indicated for both pediatric and adult 

patients according to the approved indication for use statement.

Although three HDE devices approved since FDAAA was enacted were 

explicitly indicated for use in pediatric patients, the number per year may 

increase in the future, as the number of HUD requests and designations 

per year for devices intended to treat diseases that occur in pediatric 

patients increased after FDAAA was enacted.58

 

58These three HDE devices explicitly indicated for use in pediatric patients—those age 21 

or younger—or both pediatric and adult patients accounted for 38 percent of HDE devices 

approved after FDAAA was enacted. In the 7 fiscal years before FDAAA was enacted, six 

HDE devices (26 percent of approved HDE devices) were explicitly indicated for use in 

pediatric patients or both pediatric and adult patients.

Because HUD 

designation is the first step to obtaining HDE approval, increasing HUD 
 

Page 30 GAO-12-225 Pediatric Medical Devices

designations indicate potential for future pediatric HDE devices.59

According to FDA, the agency received about one HUD request per year 

on average for devices intended to treat a disease that occurs in pediatric 

patients before FDAAA was enacted and about five requests per year on 

average in the years after the law was enacted. Likewise, FDA granted 

about one HUD designation for devices intended to treat a disease that 

occurs in pediatric patients per year prior to FDAAA and almost five such 

HUD designations per year in the years after (see fig. 2).

 

59The length of time between HUD designation and HDE device approval for HDE devices 

approved in the past decade ranged from 4 months to 7.5 years, with an average across 

the decade of about 2 years. However, to qualify for exemption from the HDE profit

prohibition, the sponsor must submit an HDE application on or before October 1, 2012.
 

Page 31 GAO-12-225 Pediatric Medical Devices

Figure 2: HUD Requests, HUD Designations, and Approved HDE Devices Explicitly 

Indicated for Use in Pediatric Patients, Fiscal Years 2001 through 2011

Notes: Some humanitarian use device (HUD) designations during a particular fiscal year may be from 

HUD requests submitted in an earlier fiscal year. FDA identified HUD requests and designations for 

pediatrics based on each device’s indication for use statement, which includes the disease the device 

is intended to treat, diagnose, or cure.

We considered devices approved through FDA’s humanitarian device exemption (HDE) process to be 

for pediatric patients if they were explicitly indicated for use in pediatric patients—that is, if the 

approved indication for use statement (1) specified a population that included patients age 

21 years or younger, (2) specifically stated it was for pediatric use, or (3) stated it was for skeletally 

immature patients. Devices explicitly indicated for pediatric use include those explicitly indicated for 

use in pediatric patients or those for both pediatric and adult patients.

Since FDAAA was enacted, two of the three HDE devices explicitly 

indicated for use in pediatric patients (patients age 21 years or younger) 

were granted the exemption from the HDE profit prohibition.60

 

60An additional HDE device was approved for use in patients age 18 years and older (and 

therefore includes pediatric patients age 18 through 21 years), but the manufacturer did 

not request and receive exemption from the HDE profit prohibition for a pediatric device. 

FDA officials reported that the agency internally tracks HDE devices that have been 

approved for use in pediatric patients and granted exemption from the profit prohibition. 

One of 
 

Page 32 GAO-12-225 Pediatric Medical Devices

these two exempted devices, the Medtronic Melody® Transcatheter 

Pulmonary Valve and Ensemble® Transcatheter Valve Delivery System 

(Melody® TPV) was approved in January 2010 for use in treating 

pediatric and adult patients with a blocked or leaky pulmonary heart valve 

that has previously been replaced to correct congenital (birth) heart 

defects and with a vessel size above a certain threshold.61 In September 

2011, FDA’s Pediatric Advisory Committee reviewed the Melody TPV and 

determined that the HDE profit prohibition exemption remained 

appropriate.62 The ELANA (Excimer Laser Assisted Non-Occlusive 

Anastamosis) Surgical KitHUD was approved in March 2011 for use in 

neurosurgery during intracranial vascular bypass procedures in patients 

13 years of age or older who meet certain criteria. (See app. I for more 

information on the two devices.) 

It is too early to determine if the FDAAA provisions authorizing 

demonstration grants for pediatric device consortia or allowing 

manufacturers to earn a profit on pediatric HDE devices have had a 

substantial impact on the number of approved pediatric devices; however, 

the provisions show potential for more pediatric devices in future years.

Although stakeholders identified barriers to developing and receiving FDA 

approval to market pediatric medical devices, programs such as the 

pediatric device consortia can foster an environment for device innovators 

to share ideas and advance the development of pediatric medical 

devices. The number of devices that the pediatric device consortia have 

supported through the early phases of development and the positive 

feedback from stakeholders indicate that pediatric devices are being 

developed. Further, the increase in the number of HUD requests and 

designations for devices that treat pediatric populations since FDAAA was 

enacted indicate potential for development of additional pediatric HDE 

devices in the near future. Over the past 10 years, it has taken a few 

years for a manufacturer to submit an HDE application and receive FDA 

market approval after receiving HUD designation; therefore the profit 

 

61The Melody TPV includes two parts—the Melody Transcatheter Pulmonary Valve (the 

valve) and Ensemble Transcatheter Valve Delivery System (the delivery system). The 

manufacturer refers to both parts together as Melody TPV.

62FDAAA requires that FDA’s Pediatric Advisory Committee annually review all HDE 

devices granted exemption from the profit prohibition to ensure that the exemption 

remains appropriate for the pediatric populations for which it is granted. See Pub. L. 

No. 110-85, § 303(a)(3), 121 Stat. 823, 861-62 (2007) (codified at 21 U.S.C. § 360j(m)(8)).

Conclusions 
 

Page 33 GAO-12-225 Pediatric Medical Devices

eligibility incentive for pediatric HDE devices may have induced an 

increase in the number of pediatric HUD requests.

Although the future for pediatric device development may be promising, 

FDA lacks reliable and timely information on pediatric devices approved 

for the U.S. market. To meet statutory reporting requirements, FDA must 

have data systems in place to produce timely, reliable information to 

readily identify and track devices the agency has approved for use in 

pediatric patients. Without a systematic database to identify medical 

devices approved for use in pediatric patients, FDA cannot provide data 

so that policymakers, innovators, and physicians may understand the 

extent of medical device availability and needs for further development.

We recommend that the Commissioner of FDA collect reliable information 

to report data related to pediatric medical devices. 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 received comments on a draft of this report from HHS, which are 

reproduced in appendix IV. The department agreed with our 

recommendation that FDA collect reliable information related to pediatric 

medical devices and commented that FDA is in the process of modifying 

and updating its current tracking system, related data quality controls, and 

training program requirements for pediatric medical device premarket 

submissions. The department further stated that when this process is 

complete, FDA’s pediatric information collection efforts will satisfy the 

department’s pediatric device operations and oversight needs, including 

the development of the annual pediatric report.

We are sending copies of this report to the Secretary of Health and 

Human Services, the Commissioner of FDA, and other interested parties.

In addition, the report will be available at no charge on the GAO website 

at http://www.gao.gov. 

Recommendation for 

Executive Action

Agency Comments
 

Page 34 GAO-12-225 Pediatric Medical Devices

If you or your staff have any questions about this report, please contact 

me at (202) 512-7114 or crossem@gao.gov. Contact points for our 

Offices of Congressional Relations and Public Affairs may be found on 

the last page of this report. GAO staff who made major contributions to 

this report are listed in appendix V.

Marcia Crosse

Director, Health Care
Appendix I: Summary of HDE Devices Granted 

Exemption from the HDE Profit Prohibition

Page 35 GAO-12-225 Pediatric Medical Devices

The Food and Drug Administration Amendments Act of 2007 (FDAAA) 

also required that we describe the key characteristics of devices 

approved through the humanitarian device exemption (HDE) process that 

were granted exemptions from the HDE profit prohibition because they 

were labeled for use in pediatric patients. This appendix describes the 

conditions and the indicated patient populations of the two pediatric 

medical devices that were granted exemption from the HDE profit 

prohibition—Medtronic Melody® Transcatheter Pulmonary Valve and 

Ensemble® Transcatheter Valve Delivery System (Melody® TPV) and 

ELANA (Excimer Laser Assisted Non-Occlusive Anastamosis) Surgical 

KitHUD.1 We also describe when each device was approved for the U.S. 

market and what is known about profits made by the manufacturers of 

each device. In addition, we describe what is known about the costs and 

insurance coverage and off-label use of the Melody TPV device—the one 

HDE device exempt from the HDE profit prohibition that was sold and in 

use in the United States at the time of our review.

To describe the key characteristics of each device, we interviewed 

officials from Medtronic, Inc., and Elana, Inc.—manufacturers of the 

Melody TPV and ELANA devices, respectively—and examined 

manufacturer-provided documentation, such as billing code information, 

related to the Melody TPV device. We also interviewed officials from the 

Food and Drug Administration (FDA) who manage the HDE process and 

examined FDA documents for each device, including approval documents 

and an annual report for the Melody TPV—which manufacturers granted 

exemption from the HDE profit prohibition are required to submit annually 

to FDA. (Table 6 provides key characteristics of the two pediatric HDE 

devices granted exemption from the HDE profit prohibition.)

 

1

The Melody TPV includes two parts—the Melody Transcatheter Pulmonary Valve (the 

valve) and Ensemble Transcatheter Valve Delivery System (the delivery system). The 

manufacturer refers to both parts together as Melody TPV.

Appendix I: Summary of HDE Devices 

Granted Exemption from the HDE Profit 

Prohibition 

Summary of Pediatric 

HDE Devices Exempt 

from the HDE Profit 

Prohibition 
Appendix I: Summary of HDE Devices Granted 

Exemption from the HDE Profit Prohibition

Page 36 GAO-12-225 Pediatric Medical Devices

Table 6: Characteristics of the Two Pediatric HDE Devices Granted Exemption from the HDE Profit Prohibition

Medtronic Melody® Transcatheter Pulmonary 

Valve and Ensemble® Transcatheter Valve 

Delivery System (Melody TPV)

ELANA (Excimer Laser Assisted Non-Occlusive 

Anastamosis) Surgical KitHUD

Manufacturer Medtronic, Inc., United States Elana, Inc., Netherlands

Summary of indication for 

use

The Melody device is used to repair a blocked or 

leaky pulmonary heart valve that has previously 

been replaced to correct congenital (birth) heart 

defects. The Melody device is put in place 

without using open heart surgery and while the 

heart is beating, and can delay the need for 

more invasive open heart surgery.

The ELANA device is intended to allow 

neurosurgeons to reroute blood flow around an 

aneurysm or a tumor in the brains of patients at 

greater risk of stroke during standard bypass 

surgery. (Standard bypass surgery in the brain 

requires clipping the artery to halt blood flow during 

the procedure, and this standard surgery is not 

considered safe for about 1,000 patients annually for 

whom temporarily shutting off their blood flow would 

put them at high risk of stroke.)

Approved indication for use 

– including population

Indicated for use as an adjunct to surgery in the 

management of pediatric and adult patients

with the following clinical conditions: Existence of 

a full (circumferential) Right Ventricular Outflow 

Tract (RVOT) conduit that was equal to or 

greater than 16 mm in diameter when originally 

implanted and Dysfunctional RVOT conduits with 

a clinical indication for intervention, and either 

Regurgitation: ≥ moderate regurgitation, or 

Stenosis: mean RVOT gradient ≥ 35 mmHg.

Indicated for creating arteriotomies during an 

intracranial vascular bypass procedure in patients 

13 years of age or older with an aneurysm or a 

skull base tumor affecting a large (> 2.5 mm), 

intracranial artery that failed balloon test occlusion, 

cannot be sacrificed, or cannot be treated with 

conventional means due to local anatomy or 

complexity.

Medical specialty Cardiovascular Cardiovascular

HUD designation date

a

7/10/2007 9/26/2003

HDE application: date filed 

and date approved

HDE application filed: 8/29/2008

HDE approved: 1/25/2010

HDE application filed: 12/5/2008

HDE approved: 3/10/2011

Annual distribution number 2,996 b 1,000

Number of devices sold in 

the United States since HDE 

approval

695 4 c

Marketing in other countries

d

European Union: September 2006

Canada: December 2006

European Union: January 2006

Canada: November 2007

U.S. list price $30,500 (combined)

$24,000 (valve)

$6,500 (delivery system)

Not available

Profit from device Not available Not available

Source: GAO analysis of FDA and manufacturer information.

Notes: Humanitarian use devices (HUD) are approved for the U.S. market through FDA’s 

humanitarian device exemption (HDE) process.
Appendix I: Summary of HDE Devices Granted 

Exemption from the HDE Profit Prohibition

Page 37 GAO-12-225 Pediatric Medical Devices

a

We identified each device’s medical specialty based on data from FDA’s product code classification 

database—which contains device names and product codes assigned based upon the medical device 

product classification designated under 21 C.F.R. Parts 862-892. Although the database indicated a 

medical specialty of cardiovascular for the ELANA Surgical KitHUD, FDA officials reported that upon 

further review they determined that the product code assigned for the device is technically inaccurate. 

To correct this technical inconsistency, FDA will issue a corrected product code for this product in the 

near future. The corrected medical specialty for the device should be neurology, according to FDA 

officials. 

b

FDA determines an upper limit on the number of devices that can be distributed each year—to treat 

both pediatric and adult patients—known as the annual distribution number (ADN). Manufacturers 

may only receive profits on devices exempt from the profit prohibition up to the ADN.

c

Number of Melody TPV devices sold from January 26, 2010, through August 31, 2011. According to 

the manufacturer, 386 devices (56 percent) were sold for use in patients age 21 or younger.

d

According to the manufacturer, the ELANA Surgical KitHUD was first sold in the U.S. market in 

November 2011.

To obtain information regarding costs of the Melody TPV device as well 

as insurance coverage for and off-label use of the device, we surveyed 36 

physicians at the 25 children’s hospitals and pediatric units of medical 

centers (which we collectively refer to as children’s hospitals) that 

according to the device’s manufacturer, had used the Melody TPV at 

least 10 times.2 We received complete responses from 27 physicians at 

20 children’s hospitals for an overall response rate of 75 percent of 

physicians and 80 percent of hospitals in our sample.3

Almost all (93 percent) of the 27 respondents reported that their hospital 

purchased the Melody TPV at the manufacturer’s list price of $30,500.4

 

2

We also interviewed officials from the Centers for Medicare & Medicaid Services and 

America’s Health Insurance Plans regarding public and private health insurance coverage 

of HDE devices, respectively.

All but 2 physicians reported that their hospital purchased the two 

components—the valve and the delivery system—of the Melody TPV 

device separately. A physician that we spoke with said his hospital kept 

several valves and several of each size delivery system in stock and 

3

We fielded our survey from August 19, 2011, through October 3, 2011. At this time, the 

Melody TPV device was the only HDE device exempt from the HDE profit prohibition that 

was on the U.S. market. For questions regarding cost and insurance coverage for the 

device, someone other than the physician—such as a purchasing department 

representative—may have provided information on behalf of the physician.

4

Thirty-three percent of respondents stated that they were not sure if the price they 

reported included all rebates or discounts their institution received for their most recent 

Melody TPV order. We did not further examine whether the reported prices included all 

rebates or discounts, and therefore additional rebates or discounts for these devices may 

have existed. 

Survey of Physicians 

and Hospitals That 

Have Used the Melody 

TPV
Appendix I: Summary of HDE Devices Granted 

Exemption from the HDE Profit Prohibition

Page 38 GAO-12-225 Pediatric Medical Devices

purchased more as needed. Twenty-four of the 25 physicians at 

children’s hospitals that purchased the components separately reported 

paying the list price for each component—$24,000 for each Melody 

Transcatheter Pulmonary Valve and $6,500 for each Ensemble 

Transcatheter Valve Delivery System.

Many physicians we surveyed reported that they experienced at least 

some difficulty obtaining health insurance coverage for the Melody TPV 

device regardless of the type of insurance held by the patient (see 

table 7).5 Twenty-five of the 27 responding physicians reported 

experiencing difficulty obtaining coverage for patients with private health 

insurance at least some of the time, with 3 of those physicians reporting 

that it was always difficult.6 For Medicaid and Children’s Health Insurance 

Program (CHIP) coverage, 4 physicians reported that the device was not 

covered at all by this type of insurance. Additionally, 18 of the 22 

responding physicians who reported receiving reimbursement for the 

device through Medicaid or CHIP also reported difficulty obtaining that 

coverage at least some of the time, with 4 of those reporting that it was 

always difficult. Most responding physicians (19 of 27) reported not 

treating any patients covered by Medicare, but 4 of those who did treat 

patients with Medicare coverage said that obtaining Medicare coverage 

for the device was always difficult.7

 

5

We did not determine if issues in obtaining coverage were specifically related to off-label 

use. 

Further, 7 of the 27 responding 

physicians reported that health insurance coverage influenced their 

clinical decision to use the device. For example, 3 physicians reported 

that delay or denial of insurance coverage for the Melody TPV device has 

led them to conduct open heart surgery in patients for whom use of the 

device would otherwise have allowed them to delay this more invasive 

surgery.

6

According to officials from America’s Health Insurance Plans, private health insurers 

often determine coverage for HDE devices on a case-by-case basis.

7

According to officials at the Centers for Medicare & Medicaid Services, the agency has 

not issued any national guidance to state Medicaid programs regarding coverage of HDE 

devices. In addition, officials reported that in most cases Medicare coverage for HDE 

devices is determined on a case-by-case basis.
Appendix I: Summary of HDE Devices Granted 

Exemption from the HDE Profit Prohibition

Page 39 GAO-12-225 Pediatric Medical Devices

Table 7: Number of Physicians Reporting Difficulty Obtaining Insurance Coverage for Melody TPV Device, by Insurance Type 

and Extent of Difficulty Experienced 

Number of physicians reporting difficulty obtaining insurance coverage for Melody TPV

Type of health 

insurance

Melody TPV 

not covered 

by this type 

of insurance

No patients 

with this type 

of insurance

Never 

difficult

Sometimes 

difficult

About half the 

time difficult

Most of the 

time difficult

Always 

difficult

At least 

sometimes 

difficult

Private health 

insurance 

0 0 2 11 9 2 3 25

Medicaid/ 

Children’s Health 

Insurance 

Program 

4 1 4 10 2 2 4 18

Medicare 0 a 19 2 0 0 0 4 4 

Source: GAO.

Notes: This table presents the responses from 27 physicians who responded to GAO’s 2011 Survey 

of Physicians Who Use the Melody TPV device.

a

Two physicians did not respond to the question regarding Medicare insurance.

Consistent with what stakeholders reported about challenges to obtaining 

insurance coverage for HDE devices in general, physicians we surveyed 

reported challenges obtaining health insurance coverage for the Melody 

TPV device. These included comments about the time required to discuss 

coverage with the insurers to obtain reimbursement, insurers’ lack of 

familiarity with the medical device, challenges because insurers consider 

the medical device experimental, and problems because the Melody TPV 

procedure did not have a current procedural terminology (CPT) code at 

the time of our survey.8

About half (52 percent) of physician respondents reported that they have 

implanted the Melody TPV device in a pediatric or adult patient for an 

indication other than the indication on the label of the device—an off-label 

indication—since it was approved for the U.S. market in February 2010.

However, all but 1 of those physicians said they used it off-label 

20 percent of the time or less across instances when they implanted the 

device in pediatric patients. Further, 29 percent of responding physicians 

 

8

CPT is a uniform coding system maintained by the American Medical Association used to 

identify and bill for medical procedures and services under public and private health 

insurance programs. Officials from the device manufacturer said they implemented new 

CPT codes for outpatient and inpatient use of the Melody TPV device in July and October 

2011.
Appendix I: Summary of HDE Devices Granted 

Exemption from the HDE Profit Prohibition

Page 40 GAO-12-225 Pediatric Medical Devices

stated that they never used the device for an off-label indication in 

pediatric patients. In addition, each instance of off-label use should have 

been for an indication approved by the hospital’s institutional review 

board (IRB). For example, 1 physician reported that he routinely 

implanted the Melody TPV for one of three IRB-approved protocols, two 

of which were for an off-label indication but all were for pediatric patients.9

 

9

The three protocols approved by the hospital’s IRB were (1) in accordance with the 

approved indication for use, (2) transcatheter use outside the parameters of the approved 

indication for use, and (3) for surgical use instead of a conduit. The physician reported that 

the hospital used the second two off-label protocols in a small percentage of cases. 
Appendix II: PMA and HDE Devices Explicitly 

Indicated for Use in Pediatric Patients since 

Fiscal Year 2006

Page 41 GAO-12-225 Pediatric Medical Devices

Of the 118 devices approved through FDA’s premarket approval (PMA) 

and HDE processes in the 4 years since FDAAA was enacted, 18 

(15 percent) were explicitly indicated for use in only pediatric patients—

those age 21 or younger—or both pediatric and adult patients. Of the 

86 PMA and HDE devices approved in the 2 years before FDAAA was 

enacted, 21 were explicitly indicated for use in only pediatric patients or 

both pediatric and adult patients (see table 8). Of these, few devices were 

explicitly indicated for use in patients under age 18—that is, in each fiscal 

year, most devices that explicitly indicated a pediatric population were 

indicated for use in patients age 18 years and older.1

 

1

Five of the PMA devices approved in the 2 years before FDAAA was enacted were 

indicated solely for use in patients age 18 or older. An additional six PMA devices 

approved before FDAAA was enacted were indicated solely for use in patients age 21 or 

older. Six of the PMA devices and one of the HDE devices approved in the 4 years after 

FDAAA was enacted were indicated solely for use in patients age 18 or older. An 

additional two PMA devices approved after FDAAA was enacted were indicated solely for 

use in patients age 21 or older. We consider these to be explicitly indicated for use in 

pediatric patients because the minimum age includes patients age 21 years or younger.

Appendix II: PMA and HDE Devices 

Explicitly Indicated for Use in Pediatric 

Patients since Fiscal Year 2006
Appendix II: PMA and HDE Devices Explicitly 

Indicated for Use in Pediatric Patients since 

Fiscal Year 2006

Page 42 GAO-12-225 Pediatric Medical Devices

Table 8: PMA and HDE Devices Explicitly Indicated for Use in Pediatric Patients, Fiscal Years 2006 through 2011

Pre-FDAAA Post-FDAAAa

Device type FY2006 FY2007 FY2008 FY2009 FY2010 FY2011 

Total 

pre- FDAAA

Total 

post- FDAAA

PMAb

Pediatric only 1 1 0 1 0 0 2 1 

Pediatric and adult 13 5 2 2 3 7 18 14 c

All pediatric PMA

d

14 6 2 3 3 7 20 15

HDE

Pediatric only 1e 0 0 0 0 0 1 0 

Pediatric and adult 0 0 1 0 1 1 0 3

All pediatric HDE

f

1 0 1 0 1 1 1 3 

Total pediatric 15 6 3 3 4 8 21 18

Source: GAO analysis of FDA documents.

Notes: We considered pediatric devices approved through FDA’s premarket approval (PMA) and 

humanitarian device exemption (HDE) processes to be those explicitly indicated for use in pediatric 

patients when the approved indication for use statement (1) specified a population that included 

patients age 21 years or younger, (2) specifically stated it was for pediatric use, or (3) stated it was for 

skeletally immature patients. Devices explicitly indicated for use in pediatric patients include those 

explicitly indicated for use in pediatric patients or those for both pediatric and adult patients. 

Additional medical devices for which the indication for use was silent on the age of the patient 

population may have been approved for pediatric use during this time period.

a

The FDA Amendments Act of 2007 (FDAAA) was enacted September 27, 2007, but no PMA or HDE 

devices were approved after FDAAA was enacted in fiscal year 2007.

b

PMA devices include original and panel-track supplement devices.

c

Five of the PMA devices approved in the 2 years before FDAAA was enacted were indicated solely 

for use in patients age 18 or older. An additional six were indicated solely for use in patients age 21 or 

older. We consider these to be explicitly indicated for use in pediatric patients because the minimum 

age includes patients age 21 years or younger.

d

Six of the PMA devices approved in the 4 years after FDAAA was enacted were indicated solely for 

use in patients age 18 or older. An additional two PMA devices were indicated solely for use in 

patients age 21 or older. We consider these to be explicitly indicated for use in pediatric patients 

because the minimum age includes patients age 21 years or younger.

e

The HDE device approved in fiscal year 2006 was indicated for fetuses whose gestational age is 

between 16 and 26 weeks. We considered this device to be explicitly indicated for use in pediatric 

patients.

f

One of the HDE devices approved in the 4 years after FDAAA was enacted was indicated solely for 

use in patients age 18 or older.
Appendix III: PMA and HDE Devices Explicitly 

Indicated for Use in Pediatric Patients, Fiscal 

Years 2008 through 2011

Page 43 GAO-12-225 Pediatric Medical Devices

Device name, 

number

Date 

approved a Summary of indication for use

Pediatric 

subpopulation 

(if available) Medical specialty b

Devices approved through the Food and Drug Administration’s (FDA) premarket approval (PMA) process 

Propel sinus implant, 

P100044

8/11/2011 Indicated for use in patients 18 years of age 

or older following ethmoid sinus surgery to 

reduce the need for postoperative 

intervention.

Transitional 

adolescent, adult

Ear, nose, and throat

Vitros 

Immunodiagnostic 

Products Anti-HBe 

Reagent Pack, 

Calibrator and 

Controls, P100001

7/20/2011 Indicated for the in vitro qualitative detection 

of antibodies to hepatitis B e antigen (antiHBe) in human adult and pediatric (2 to 21 

years old) serum from individuals who have 

symptoms of chronic hepatitis and those 

who have recovered from hepatitis B 

infection.

Child, adolescent, 

transitional 

adolescent, adult

Microbiology

Solseta Injectable Gel, 

P100014

5/27/2011 Indicated for the treatment of fecal 

incontinence in patients 18 years and 

older who have failed conservative therapy 

(e.g., diet, fiber therapy, and antimotility 

medications).

Transitional 

adolescent, adult

Gastroenterology/Urology

Vitros 

Immunodiagnostic 

Products HBeAg 

Reagent Pack, 

Calibrator and 

Controls, P090028 

5/11/2011 Indicated for the in vitro qualitative detection 

of hepatitis B e antigen (HBeAg) in human 

adult and pediatric (2 to 21 years old) 

serum from individuals who have symptoms 

of hepatitis or who may be at risk for 

hepatitis B virus infection. 

Child, adolescent, 

transitional 

adolescent, adult

Microbiology

Cobas HPV Test, 

P100020

4/19/2011 Indicated to screen patients 21 years and 

older with ASC-US (atypical squamous cells 

of undetermined significance) cervical 

cytology test results to determine the need 

for referral to colposcopy or to assess the 

presence or absence of high-risk Human 

Papillomavirus genotypes 16 and 18, and 

for other uses in women 30 years and older.

Transitional 

adolescent,c Microbiology

adult

Oraquick HCV Rapid 

Antibody Test, 

P080027

6/25/2010

d

Indicated for the qualitative detection of 

antibodies to hepatitis C virus in 

venipuncture whole blood specimens from 

individuals 15 years or older.

Adolescent, 

transitional 

adolescent, adult

Microbiology

Appendix III: PMA and HDE Devices 

Explicitly Indicated for Use in Pediatric 

Patients, Fiscal Years 2008 through 2011
Appendix III: PMA and HDE Devices Explicitly 

Indicated for Use in Pediatric Patients, Fiscal 

Years 2008 through 2011

Page 44 GAO-12-225 Pediatric Medical Devices

Device name, 

number

Date 

approved a Summary of indication for use

Pediatric 

subpopulation 

(if available) Medical specialty b

Alair Bronchial 

Thermoplasty System, 

P080032

4/27/2010 Indicated for the treatment of severe 

persistent asthma in patient 18 years and 

older whose asthma is not well controlled 

with inhaled corticosteroids and long-acting 

beta agonists.

Transitional 

adolescent, adult

Anesthesiology

Esteem Totally 

Implantable Hearing 

System, P090018

3/17/2010 Indicated for patients with hearing loss who 

meet the following criteria: (1) 18 years of 

age or older, (2) stable bilateral 

sensorineural hearing loss, (3) moderate to 

severe sensorineural hearing loss defined 

by Pure Tone Average, (4) unaided speech 

discrimination test score greater than or 

equal to 40 percent, (5) normally functioning 

eustachian tube, (6) normal middle ear 

anatomy, (7) normal tympanic membrane, 

(8) adequate space for Esteem implant 

determined via a high resolution 

computerized tomography (CT) scan, and 

(9) minimum 30 days of experience with 

appropriately fit hearing aids.

Transitional 

adolescent, adult

Ear, nose, and throat

Architect Core 

Reagent Kit, Architect 

Core Calibrator and 

Architect Core 

Controls, P080023

4/10/2009 Indicated for the qualitative detection of 

antibodies to hepatitis B core antigen in 

human adult and pediatric serum and 

plasma and neonatal serum. It is intended 

as an aid in the diagnosis of acute, chronic, 

or resolved hepatitis B virus infection in 

conjunction with other laboratory results 

and clinical information. 

(No age provided, 

indicated for 

pediatric and adult 

patients)

Microbiology

REPEL-CV 

Bioreabsorbable 

Adhesion Barrier, 

P070005 

3/6/2009 Indicated for reducing the severity of 

postoperative cardiac adhesions in pediatric 

patients who are likely to require 

reoperation via sternotomy.

(No age provided, 

indicated for 

pediatric patients)

Cardiovascular

Freestyle Navigator 

Continuous Glucose 

Monitor, P050020

3/12/2008 Indicated for continually recording interstitial 

fluid glucose levels in people ages 18 and 

older with diabetes mellitus for the purpose 

of improving diabetes management.

Transitional 

adolescent, adult

Clinical chemistry

Architect Core-M 

Reagent Kit, Architect 

Core Calibrator and 

Architect Core 

Controls, P060035

11/6/2007 Indicated for the qualitative detection of IgM 

antibodies to hepatitis B core antigen in 

human adult and pediatric serum and 

plasma and neonatal serum. It is intended 

as an aid in the diagnosis of acute, chronic, 

or resolved hepatitis B virus infection in 

conjunction with other laboratory results 

and clinical information.

(No age provided, 

indicated for 

pediatric and adult 

patients)

Microbiology
Appendix III: PMA and HDE Devices Explicitly 

Indicated for Use in Pediatric Patients, Fiscal 

Years 2008 through 2011

Page 45 GAO-12-225 Pediatric Medical Devices

Device name, 

number

Date 

approved a Summary of indication for use

Pediatric 

subpopulation 

(if available) Medical specialty b

PMA panel-track supplement devices

Meditec MEL 80 

Excimer Laser, 

P060004-S001 

3/28/2011 Indicated for primary lasik treatments for 

(1) the reduction or elimination of naturally 

occurring hyperopia, (2) in patients who are 

21 years of age or older, and (3) with

documentation of stable manifest refraction 

over the past year.

Transitional 

adolescent,c Ophthalmic

adult

Oraquick HCV Rapid 

Antibody Test, 

P080027-S001

2/18/2011

d

A single-use immunoassay for the 

qualitative detection of antibodies to 

hepatitis C virus in fingerstick whole blood 

specimens and venipuncture whole blood 

specimens from individuals 15 years or 

older.

Adolescent, 

transitional 

adolescent, adult

Microbiology

Navistar & Celsius 

Thermacool Catheters, 

P030031-S011

2/6/2009 Indicated for catheter-based cardiac 

electrophysiological mapping (stimulating 

and recording), for the treatment of (1) type 

I atrial flutter in patients age 18 or older, 

(2) recurrent drug/device refractory 

sustained monomorphic ventricular 

tachycardia due to prior myocardial 

infarction in adults, and (3) drug refractory 

recurrent symptomatic paroxysmal atrial 

fibrillation.

Transitional 

adolescent, adult

Cardiovascular

Devices approved through FDA’s humanitarian device exemption process (HDE)

ELANA Surgical 

KitHUD, H080005

3/10/2011 Indicated for creating arteriotomies during 

an intracranial vascular bypass procedure 

in patients 13 years of age or older with an 

aneurysm or a skull base tumor affecting a 

large (> 2.5 mm), intracranial artery that 

failed balloon test occlusion, cannot be 

sacrificed, or cannot be treated with 

conventional means due to local anatomy 

or complexity.

Adolescent, 

transitional 

adolescent, adult

Cardiovascular

Medtronic Melody 

Transcatheter 

Pulmonary Valve and 

Ensemble 

Transcatheter Valve 

Delivery System, 

H080002 

e

1/25/2010 Indicated for use as an adjunct to surgery in 

the management of pediatric and adult 

patients with the following clinical 

conditions: Existence of a full 

(circumferential) Right Ventricular Outflow 

Tract (RVOT) conduit that was equal to or 

greater than 16 mm in diameter when 

originally implanted and Dysfunctional 

RVOT conduits with a clinical indication for 

intervention, and either Regurgitation: ≥

moderate regurgitation, or Stenosis: mean 

RVOT gradient ≥ 35 mmHg.

(No age provided, 

indicated for 

pediatric and adult 

patients)

Cardiovascular
Appendix III: PMA and HDE Devices Explicitly 

Indicated for Use in Pediatric Patients, Fiscal 

Years 2008 through 2011

Page 46 GAO-12-225 Pediatric Medical Devices

Device name, 

number

Date 

approved a Summary of indication for use

Pediatric 

subpopulation 

(if available) Medical specialty b

NeuRx RA/4, H070003 6/17/2008 Indicated for use in patients with stable, 

high spinal cord injuries with stimulatable 

diaphragms, but who lack control of their 

diaphragms. The device is indicated to 

allow the patients to breathe without the 

assistance of a mechanical ventilator for at 

least 4 continuous hours a day. For use 

only in patients 18 years of age or older.

Transitional 

adolescent, adult

Anesthesiology

Source: GAO analysis of FDA data and documents.

Notes: We considered pediatric devices to be those explicitly indicated for use in pediatric patients 

when the approved indication for use statement (1) specified a population that included patients age 

21 years or younger, (2) specifically stated it was for pediatric use, or (3) stated it was for skeletally 

immature patients. Devices explicitly indicated for use in pediatric patients include those explicitly 

indicated for use in pediatric patients or those for both pediatric and adult patients.

a

FDA assigns a number to each device application that denotes the type of device (P=PMA, H=HDE), 

year the application was received, and number assigned chronologically in the order it was received. 

FDA also assigns PMA supplements, including panel-track supplements, a supplement number (S).

b

For purposes of this table, pediatric subpopulation ages are those that FDA uses for internal tracking 

purposes: neonate (birth to 28 days), infant (29 days to <2 years), child (2 years to <12 years), 

adolescent (12 years to <18 years), and transitional adolescent (18 years to <=21 years).

c

Two PMA devices are indicated solely for use in patients age 21 or older—we consider these 

devices to be indicated for use in pediatric patients because the minimum age includes pediatric 

patients.

d

The original application and a panel-track supplement for the Oraquick HCV Rapid Antibody Test 

were both approved for use in pediatric patients after the FDA Amendments Act of 2007 was enacted.

e

We identified each device’s medical specialty based on data from FDA’s product code classification 

database—which contains device names and product codes assigned based upon the medical device 

product classification designated under 21 C.F.R. Parts 862-892. Although the database indicated a 

medical specialty of cardiovascular for the ELANA Surgical KitHUD, FDA officials reported that upon 

further review they determined that the product code assigned for the device is technically inaccurate. 

To correct this technical inconsistency, FDA will issue a corrected product code for this product in the 

near future. The corrected medical specialty for the device should be neurology, according to FDA 

officials.
Appendix IV: Comments from the Department 

of Health and Human Services

Page 47 GAO-12-225 Pediatric Medical Devices

Appendix IV: Comments from the 

Department of Health and Human Services 
Appendix IV: Comments from the Department 

of Health and Human Services

Page 48 GAO-12-225 Pediatric Medical Devices
Appendix V: GAO Contact and Staff 

Acknowledgments

Page 49 GAO-12-225 Pediatric Medical Devices

Marcia Crosse, (202) 512-7114 or crossem@gao.gov

In addition to the contact named above, Kim Yamane, Assistant Director; 

Rebecca Abela; Carolyn Fitzgerald; Cathleen Hamann; Shirin Hormozi; 

Lisa Motley; and Dan Ries made key contributions to this report.

Appendix V: GAO Contact and Staff 

Acknowledgments 

GAO Contact

Staff 

Acknowledgments 
Related GAO Products

Page 50 GAO-12-225 Pediatric Medical Devices

Medical Devices: FDA Should Enhance Its Oversight of Recalls. 

GAO-11-468. Washington, D.C.: June 14, 2011.

Medical Devices: FDA’s Premarket Review and Postmarket Safety 

Efforts. GAO-11-556T. Washington, D.C.: April 13, 2011.

High-Risk Series: An Update. GAO-11-278. Washington, D.C.: February 

2011.

Food and Drug Administration: Overseas Offices Have Taken Steps to 

Help Ensure Import Safety, but More Long-Term Planning Is Needed. 

GAO-10-960. Washington, D.C.: September 30, 2010.

Food and Drug Administration: Opportunities Exist to Better Address 

Management Challenges. GAO-10-279. Washington, D.C.: February 19, 

2010.

Food and Drug Administration: FDA Faces Challenges Meeting Its 

Growing Medical Product Responsibilities and Should Develop Complete 

Estimates of Its Resource Needs. GAO-09-581. Washington, D.C.: 

June 19, 2009.

Medical Devices: Shortcomings in FDA’s Premarket Review, Postmarket 

Surveillance, and Inspections of Device Manufacturing Establishments. 

GAO-09-370T. Washington, D.C.: June 18, 2009.

High-Risk Series: An Update. GAO-09-271. Washington, D.C.: January 

2009.

Medical Devices: FDA Should Take Steps to Ensure That High-Risk 

Device Types Are Approved through the Most Stringent Premarket 

Review Process. GAO-09-190. Washington, D.C.: January 15, 2009.

Health-Care-Associated Infections in Hospitals: Number Associated with 

Medical Devices Unknown, but Experts Report Provider Practices as a 

Significant Factor. GAO-08-1091R. Washington, D.C.: September 26, 

2008.

Medical Devices: FDA Faces Challenges in Conducting Inspections of 

Foreign Manufacturing Establishments. GAO-08-780T. Washington, D.C.: 

May 14, 2008.

Related GAO Products
Related GAO Products

Page 51 GAO-12-225 Pediatric Medical Devices

Reprocessed Single-Use Medical Devices: FDA Oversight Has 

Increased, and Available Information Does Not Indicate That Use 

Presents an Elevated Health Risk. GAO-08-147. Washington, D.C.: 

January 31, 2008.

Medical Devices: Challenges for FDA in Conducting Manufacturer 

Inspections. GAO-08-428T. Washington, D.C.: January 29, 2008.

Medical Devices: FDA’s Approval of Four Temporomandibular Joint 

Implants. GAO-07-996. Washington, D.C.: September 17, 2007.

Food and Drug Administration: Methodologies for Identifying and 

Allocating Costs of Reviewing Medical Device Applications Are 

Consistent with Federal Cost Accounting Standards, and Staffing Levels 

for Reviews Have Generally Increased in Recent Years. GAO-07-882R. 

Washington, D.C.: June 25, 2007.

Medical Devices: Status of FDA’s Program for Inspections by Accredited 

Organizations. GAO-07-157. Washington, D.C.: January 5, 2007.

(290925)
The Government Accountability Office, the audit, evaluation, and 

investigative arm of Congress, exists to support Congress in meeting its 

constitutional responsibilities and to help improve the performance and 

accountability of the federal government for the American people. GAO 

examines the use of public funds; evaluates federal programs and 

policies; and provides analyses, recommendations, and other assistance 

to help Congress make informed oversight, policy, and funding decisions. 

GAO’s commitment to good government is reflected in its core values of 

accountability, integrity, and reliability.

The fastest and easiest way to obtain copies of GAO documents at no 

cost is through GAO’s website (www.gao.gov). Each weekday afternoon, 

GAO posts on its website newly released reports, testimony, and 

correspondence. To have GAO e-mail you a list of newly posted products, 

go to www.gao.gov and select “E-mail Updates.”

The price of each GAO publication reflects GAO’s actual cost of 

production and distribution and depends on the number of pages in the 

publication and whether the publication is printed in color or black and 

white. Pricing and ordering information is posted on GAO’s website, 

http://www.gao.gov/ordering.htm. 

Place orders by calling (202) 512-6000, toll free (866) 801-7077, or 

TDD (202) 512-2537. 

Orders may be paid for using American Express, Discover Card, 

MasterCard, Visa, check, or money order. Call for additional information.

Connect with GAO on Facebook, Flickr, Twitter, and YouTube. 

Subscribe to our RSS Feeds or E-mail Updates. Listen to our Podcasts. 

Visit GAO on the web at www.gao.gov. 

Contact:

Website: www.gao.gov/fraudnet/fraudnet.htm

E-mail: fraudnet@gao.gov

Automated answering system: (800) 424-5454 or (202) 512-7470

Ralph Dawn, Managing Director, dawnr@gao.gov, (202) 512-4400

U.S. Government Accountability Office, 441 G Street NW, Room 7125

Washington, DC 20548

Chuck Young, Managing Director, youngc1@gao.gov, (202) 512-4800

U.S. Government Accountability Office, 441 G Street NW, Room 7149 

Washington, DC 20548

GAO’s Mission

Obtaining Copies of 

GAO Reports and 

Testimony 

Order by Phone

Connect with GAO

To Report Fraud, 

Waste, and Abuse in 

Federal Programs

Congressional 

Relations 

Public Affairs

Please Print on Recycled Paper.