NATIONAL 

PREPAREDNESS

Efforts to Address the 

Medical Needs of 

Children in a 

Chemical, Biological, 

Radiological, or 

Nuclear Incident

Report to the Committee on Energy and 

Commerce, House of Representatives

April 2013

GAO-13-438

United States Government Accountability Office

GAO
United States Government Accountability Office

Highlights of GAO-13-438, a report to the 

Committee on Energy and Commerce, House 

of Representatives

April 2013

NATIONAL PREPAREDNESS 

Efforts to Address the Medical Needs of Children in 

a Chemical, Biological, Radiological, or Nuclear 

Incident

Why GAO Did This Study

The nation remains vulnerable to 

terrorist and other threats posed by 

CBRN agents. Medical 

countermeasures—drugs, vaccines, 

and medical devices—can prevent or 

treat the effects of exposure to CBRN 

agents, and countermeasures are 

available in the SNS for some of these 

agents. Children, who make up 

25 percent of the population in the 

United States, are especially 

vulnerable because many of the 

countermeasures in the SNS have only 

been approved for use in adults. HHS 

leads the federal efforts to develop and 

acquire countermeasures. 

GAO was asked about efforts to 

address the needs of children in the 

event of a CBRN incident. This report 

examines (1) the percentage of CBRN 

medical countermeasures in the SNS 

that are approved for pediatric use; 

(2) the challenges HHS faces in 

developing and acquiring CBRN 

medical countermeasures for the 

pediatric population, and the steps it is 

taking to address them; and (3) the 

ways that HHS has addressed the 

dispensing of pediatric medical 

countermeasures in its emergency 

response plans and guidance, and 

ways that state and local governments 

have addressed this issue. To address 

these objectives, GAO reviewed 

relevant laws, agency documents, and 

reports, and interviewed HHS officials, 

industry representatives, and subjectmatter experts. GAO also reviewed a 

stratified sample of emergency 

response plans from seven state and 

seven local governments, based on 

geographic location and population 

size, to assess how these 

governments address pediatric 

dispensing.

What GAO Found

According to the Department of Health and Human Services (HHS), about 60 

percent of the chemical, biological, radiological, and nuclear (CBRN) medical 

countermeasures in the Strategic National Stockpile (SNS) have been approved 

for children, but in many instances approval is limited to specific age groups. In 

addition, about 40 percent of the CBRN countermeasures have not been 

approved for any pediatric use. Furthermore, some of the countermeasures have 

not been approved to treat individuals for the specific indications for which they 

have been stockpiled. For example, ciprofloxacin is stockpiled in the SNS for the 

treatment of anthrax, plague, and tularemia, but is not approved for these 

indications. Countermeasures may be used to treat unapproved age groups or 

indications under an emergency use authorization (EUA) or an Investigational 

New Drug (IND) application submitted to the Food and Drug Administration 

(FDA).

HHS faces a variety of economic, regulatory, scientific, and ethical challenges in 

developing and acquiring pediatric CBRN medical countermeasures. High costs 

and the high risk of failure associated with testing and research of 

pharmaceutical products on children, difficulties in meeting regulatory 

requirements for approving CBRN countermeasures, and scientific and ethical 

obstacles to safely evaluating countermeasures for children all pose challenges 

to developing pediatric countermeasures. Despite these challenges, HHS has 

taken steps to focus agency efforts on the pediatric population, adapt pediatric 

formulations from existing medical countermeasures, and prepare and review 

materials for EUAs and INDs in advance of public health emergencies.

HHS addresses dispensing of pediatric medical countermeasures in more than 

half of its 12 response plans and in its guidance, and seven state and seven local 

government plans that GAO reviewed included details about pediatric 

dispensing. Seven of the 12 HHS plans include information about pediatric 

medical countermeasures; however, HHS officials stated that these plans are 

intended to provide guidance for emergency response at the federal level, and 

not at the state or local levels, which is where dispensing would occur. CDC and 

FDA also provide guidance on pediatric dispensing that state and local 

governments can use in their planning. For example, CDC developed guidance 

about receiving, distributing, and dispensing contents from the SNS to help state 

and local emergency management and public health personnel plan for the use 

of countermeasures from the SNS. Response plans for all 14 of the state and 

local governments that GAO reviewed also included details about dispensing to 

the pediatric population during an emergency. For example, these seven states 

and seven local governments all adopted some version of a “family member pickup” policy—sometimes referred to as a “head of household” policy—which would 

allow adults to pick up medicines for other family members, including children, 

during an event.

In commenting on a draft of this report, HHS concurred with our findings. HHS 

emphasized that the needs of the pediatric population have been a priority for 

View GAO-13-438. For more information, HHS and that the department is continuously progressing in this area. 

contact Marcia Crosse at (202) 512-7114 or 

crossem@gao.gov.
Page i GAO-13-438 Pediatric Medical Countermeasures

Letter 1 

Background 5

More than Half of CBRN Medical Countermeasures Have Been 

Approved for at Least Some Children 13

HHS Faces Challenges in Developing and Acquiring Pediatric 

Medical Countermeasures but Is Taking Steps to Address the 

Needs of the Pediatric Population 16

HHS and State and Local Governments Address Dispensing of 

Pediatric Medical Countermeasures in Their Response Plans 23

Agency Comments 28

Appendix I Pediatric CBRN Medical Countermeasures in the Strategic 

National Stockpile 30

Appendix II Comments from the Department of Health and Human 

Services 34

Appendix III GAO Contact and Staff Acknowledgments 38

Related GAO Products 39

Table

Table 1: Overview of Certain Types of Chemical, Biological, 

Radiological, and Nuclear (CBRN) Medical 

Countermeasures Available in the Strategic National 

Stockpile (SNS) and Their Regulatory Status for the 

Pediatric Population 30

Figure

Figure 1: Processes for Medical Countermeasure Development and 

Acquisition 12

Contents 
Page ii GAO-13-438 Pediatric Medical Countermeasures

Abbreviations

ASPR Office of the Assistant Secretary for Preparedness 

 and Response

AVA anthrax vaccine adsorbed

BARDA Biomedical Advanced Research and Development

 Authority

CBRN chemical, biological, radiological, and nuclear

CDC Centers for Disease Control and Prevention

CHILD Children’s HHS Interagency Leadership on Disasters

DHS Department of Homeland Security

EUA Emergency Use Authorization

FDA Food and Drug Administration

HHS Department of Health and Human Services

IND Investigational New Drug

NIH National Institutes of Health

PHEMCE Public Health Emergency Medical Countermeasures

 Enterprise

POD point of dispensing

SNS Strategic National Stockpile

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 

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Page 1 GAO-13-438 Pediatric Medical Countermeasures

United States Government Accountability Office

Washington, DC 20548

April 30, 2013

The Honorable Fred Upton

Chairman

The Honorable Henry Waxman

Ranking Member

Committee on Energy and Commerce

House of Representatives

The 2001 anthrax attacks and the 2011 earthquake and tsunami in Japan 

raised concerns that the United States is vulnerable to intentional and 

unintentional threats from chemical, biological, radiological, and nuclear 

(CBRN) agents.1

 Rapid diagnosis, treatment, and prevention could 

mitigate the public health impact of a release of CBRN agents, and there 

are medical countermeasures available for some of these agents.2

Members of Congress, federal commissions, and other experts have 

noted the need for the United States to develop and acquire medical 

countermeasures to protect the public, and children in particular, from 

public health emergencies involving CBRN agents. In 2010, the National 

Commission on Children and Disasters reported that children, who make 

up nearly 25 percent of the U.S. population, are more susceptible to 

CBRN threats and require different countermeasures, dosages, and 

delivery systems than adults.3

The Department of Health and Human Services (HHS) is responsible for 

leading federal efforts to prepare for and respond to public health and 

medical CBRN incidents, including identifying needed medical 

countermeasures to diagnose, treat, prevent, or mitigate potential health 

effects from exposure, as well as engaging with industry to develop and 

 

1

CBRN agents have the potential to cause widespread illness and death.

2

Medical countermeasures for CBRN agents include drugs, biologics—such as 

vaccines—and devices to diagnose, treat, prevent, or mitigate potential effects of 

exposure. This report focuses on drugs and biologics.

3

National Commission on Children and Disasters, 2010 Report to the President and 

Congress (Rockville, Md.: October 2010). For the purpose of this report, “adults” are 

individuals 18 years of age and older, and the terms “children” and “pediatric population” 

are equivalent and refer to individuals younger than 18 years of age. The Food and Drug 

Administration (FDA) generally defines the pediatric population as individuals younger 

than 16 years of age, but relevant research has included children as old as 18.

 
 

Page 2 GAO-13-438 Pediatric Medical Countermeasures

acquire the countermeasures.4

You raised questions concerning pediatric medical countermeasures that 

are available in HHS’s SNS, as well as medical and public health 

preparedness and response activities that are in place at the federal, 

state, and local levels to address the needs of the pediatric population in 

the event of a CBRN incident. This report addresses (1) the percentage of 

CBRN medical countermeasures in the SNS that are approved for 

pediatric use; (2) the challenges HHS faces in developing and acquiring 

CBRN medical countermeasures for the pediatric population, and the 

steps it is taking to address them; and (3) the ways that HHS has 

addressed the dispensing of pediatric medical countermeasures in its 

emergency response plans and guidance, and ways that state and local 

governments have addressed this issue.

Because desired CBRN medical 

countermeasures may not be developed to a point where they are 

available for acquisition, HHS’s responsibility extends to overseeing and 

supporting research, as well as the development of these 

countermeasures by manufacturers. In 2006, HHS established the Public 

Health Emergency Medical Countermeasures Enterprise (PHEMCE), a 

federal interagency body that includes various HHS agencies and offices, 

as well as other federal departments, to provide recommendations to the 

Secretary of HHS on medical countermeasure priorities and development 

and acquisition activities. HHS’s Centers for Disease Control and 

Prevention (CDC) manages the U.S. Strategic National Stockpile (SNS)—

the national repository of medications, medical supplies, and equipment 

for HHS’s use in mitigating the potential effects of exposure to CBRN 

agents and other public health emergencies. PHEMCE annually reviews 

the contents of the SNS to help HHS prioritize the maintenance and 

acquisition of medical countermeasures. CDC is responsible for 

distributing medical countermeasures from the SNS to states. In most 

instances, states are in turn responsible for distributing the 

countermeasures to local governments to dispense to affected 

populations. In addition, HHS’s Food and Drug Administration (FDA) 

reviews safety, efficacy, and product quality data on medical 

countermeasures and approves, licenses, or clears them for specific 

indications and for use by various populations, including children.

 

4

For the purposes of this report, we are using the term “industry” to include small research 

companies and manufacturing companies, both of which support HHS in efforts to 

research and develop CBRN medical countermeasures.
 

Page 3 GAO-13-438 Pediatric Medical Countermeasures

To determine the percentage of CBRN medical countermeasures in the 

SNS that are approved for pediatric use, we reviewed and analyzed HHS 

documentation, such as the PHEMCE annual reviews of the contents in 

the SNS, and determined that the information was sufficiently reliable.5

We interviewed officials from the HHS agencies and offices that are 

responsible for CBRN emergency preparedness—including the Office of 

the Assistant Secretary for Preparedness and Response (ASPR), the 

Biomedical Advanced Research and Development Authority (BARDA), 

the National Institutes of Health (NIH), CDC, FDA, the Children’s HHS 

Interagency Leadership on Disasters (CHILD) Working Group, as well as 

officials from PHEMCE. We also analyzed BARDA and CDC reports on 

their investments and interviewed HHS officials.6

To identify challenges that HHS faces in developing and acquiring CBRN 

medical countermeasures for the pediatric population and steps it is 

taking to address those challenges, we reviewed relevant laws, our earlier 

reports,7

To determine the ways that HHS has addressed the dispensing of 

pediatric medical countermeasures in its emergency response plans and 

guidance, and to assess how state and local governments have 

HHS documents, and other reports and information from 

subject-matter experts on emergency preparedness and pediatric health. 

We also interviewed officials from ASPR, BARDA, CDC, FDA, NIH, and 

PHEMCE, as well as industry officials and other expert groups, such as 

the American Academy of Pediatrics, Trust for America’s Health, the 

Center for Infectious Disease Research and Policy, and state and local 

organizations, such as the Association of State and Territorial Health 

Officials and the National Association of County and City Health Officials.

 

5

We reviewed supporting documentation provided by PHEMCE that included the list of 

CBRN medical countermeasures in the SNS as well as its analysis of whether each 

countermeasure was approved for all children, some children, or no children.

6

For the purposes of this report, we used the term “investments” to mean obligations.

7

GAO, National Preparedness: Improvements Needed for Acquiring Medical 

Countermeasures to Threats from Terrorism and Other Sources, GAO-12-121

(Washington, D.C.: Oct. 26, 2011), National Preparedness: DHS and HHS Can Further 

Strengthen Coordination for Chemical, Biological, Radiological, and Nuclear Risk 

Assessments, GAO-11-606 (Washington, D.C.: June 21, 2011), Public Health 

Preparedness: Developing and Acquiring Medical Countermeasures Against Chemical, 

Biological, Radiological, and Nuclear Agents, GAO-11-567T (Washington, D.C.: Apr. 13, 

2011).
 

Page 4 GAO-13-438 Pediatric Medical Countermeasures

addressed pediatric dispensing, we reviewed HHS documents, such as 

the department’s 12 emergency response plans for specific CBRN threats 

and CDC’s emergency operations plan and guidance on dispensing 

medical countermeasures. In addition, we reviewed a sample of 

emergency response plans8 from seven states and seven local 

governments to assess how state and local governments address 

pediatric dispensing.9

We conducted this performance audit from July 2012 through March 2013 

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.

Our findings related to the documentation and 

responses from the state and local governments are for illustrative 

purposes only and are not generalizable to other state and local 

governments’ emergency response plans. We interviewed HHS and CDC 

officials responsible for addressing information in emergency response 

plans and providing guidance to state and local governments on 

dispensing pediatric medical countermeasures. We also interviewed state 

and local health officials and interest groups, such as the Association of 

State and Territorial Health Officials and the National Association of 

County and City Health Officials, to confirm our understanding about how 

dispensing CBRN medical countermeasures to children is handled during 

emergencies.

 

8

For the purposes of this report, we are using the term “emergency response plan” to 

include any document a state or local government provided to us that describes an 

approach for responding to, or is used in the event of, an emergency.

9

For the states, we received and reviewed emergency response plans from Arkansas, 

Michigan, Missouri, Oklahoma, Washington, and West Virginia, selected by a stratified 

random sample of the states, based on population size. We also received and reviewed 

documents from Virginia, although it was not part of the original sample. For local 

governments, we received and reviewed emergency response plans from Chicago 

(Illinois), Fresno (California), Milwaukee (Wisconsin), Nashville and Memphis 

(Tennessee), New York City (New York), and Virginia Beach (Virginia), selected by a 

stratified random sample of local governments that participate in CDC’s Cities Readiness 

Initiative, based on the Census regions.
 

Page 5 GAO-13-438 Pediatric Medical Countermeasures

Since the terrorist attacks of 2001, HHS has worked to prepare for and 

mitigate potential consequences resulting from the intentional or 

unintentional release of CBRN agents. This work has involved the efforts 

of various agencies, industry, and subject-matter experts to develop, 

acquire, and store medical countermeasures.

The development of medical countermeasures generally begins with 

research on able-bodied adults, according to the National Commission on 

Children and Disasters.10 However, children have unique anatomical, 

physiological, and psychological differences that can predispose them in 

some circumstances to more serious or different adverse effects during 

public health emergencies compared to adults. HHS classifies children as 

part of the “at-risk population” and evaluates CBRN medical 

countermeasures for this group after developing them for the general 

adult population.11 The at-risk population generally has unique 

characteristics that may interfere with an individual’s ability to access or 

receive medical countermeasures. For example, individuals who have a 

limited ability to receive or respond to information because of hearing, 

vision, speech, or cognitive limitations would need to have information 

provided in such a way that they could understand it. In addition, before, 

during, and after an emergency, individuals may lose the support of 

caregivers, family, or friends. If separated from their caregivers, young 

children may be unable to identify themselves, and may lack the cognitive 

ability to assess situations and react appropriately. The National 

Commission on Children and Disasters and other experts in the field of 

pediatrics and emergency preparedness have reported that a disparity 

exists in the quality of adult and pediatric emergency care, especially in 

HHS’s efforts to acquire FDA-approved pediatric medical 

countermeasures.12

 

10National Commission on Children and Disasters, 2010 Report to the President and 

Congress.

11In addition to those individuals specifically recognized as “at-risk individuals” in the 

Pandemic and All-Hazards Preparedness Act of 2006—children, senior citizens, and 

pregnant women—individuals who may need additional response assistance include 

those who have disabilities, live in institutionalized settings, are from diverse cultures, 

have limited English proficiency or are non-English speaking, are transportation 

disadvantaged, have chronic medical disorders, or have pharmacological dependency.

12Institute of Medicine, Safe and Effective Medicines for Children: Pediatric Studies 

Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research 

Equity Act (Washington, D.C.: The National Academies Press, 2012).

Background 
 

Page 6 GAO-13-438 Pediatric Medical Countermeasures

HHS leads the federal public health and medical response to potential 

CBRN incidents, including identifying needed medical countermeasures 

to prevent or mitigate potential health effects from exposure and engaging 

with industry to develop and acquire the countermeasures. The following 

agencies and offices within HHS have responsibilities related to medical 

countermeasures.

• ASPR is responsible for leading federal government efforts to 

research, develop, evaluate, and acquire medical countermeasures to 

diagnose, prevent, treat, or mitigate the potential health effects from 

exposure to CBRN agents. Within ASPR, BARDA, which was 

established by the Pandemic and All-Hazards Preparedness Act of 

2006, is responsible for overseeing and funding advanced 

development and acquisition of CBRN medical countermeasures.

• NIH is responsible for conducting and coordinating basic and applied 

research to develop new or enhanced medical countermeasures for 

CBRN agents.

• FDA is responsible for regulating the development and approval of 

drugs, biologics, diagnostics, and devices, which includes assessing 

the safety, efficacy, and quality of CBRN medical countermeasures 

before approval and postmarket.

• CDC is responsible for maintaining the SNS and supporting state and 

local public health departments in their efforts to respond to public 

health emergencies, including providing guidance and 

recommendations for the mass distribution and use of medical 

countermeasures.

Since 2004, the Department of Homeland Security (DHS), in consultation 

with the Secretary of HHS, has determined that certain CBRN agents 

pose a threat to the nation that could affect national security.13

 

13Pub. L. No. 108-276. § 3(a)(2), 118 Stat. 835, 844 (2004). The Project BioShield Act of 

2004 calls for HHS to assess the public health consequences of exposure to those CBRN 

agents that the Department of Homeland Security, in consultation with the Secretary of 

HHS, determines are material threats to the nation and to determine for which of these 

agents medical countermeasures are necessary to protect the public health. 42 U.S.C. 

§ 247d-6b(c)(2). 

HHS has 

used these material threat determinations to assess the potential public 

Roles and Responsibilities 

of HHS’s Agencies and 

Offices

CBRN Threats and Medical 

Countermeasures 
 

Page 7 GAO-13-438 Pediatric Medical Countermeasures

health and medical consequences of the CBRN agents, and to establish 

specific medical requirements for developing countermeasures. 

Assessing the medical consequences and establishing medical 

requirements are both interim steps in determining the types and 

quantities of medical countermeasures required to respond to the agents.

CBRN agents differ from one another in their potential to cause 

widespread illness and death. In the event of a release of a CBRN agent, 

medical countermeasures may be needed for rapid diagnosis, treatment, 

and prevention of infection, illness, and injury, but the dose and 

formulation of the countermeasures needed for an individual may vary 

according to traits such as the individual’s age and weight, especially 

when considering the needs of children.14 For example, in some cases, it 

is desirable to have multiple formulations of countermeasures available—

such as oral liquid suspensions and tablets—to facilitate patient 

compliance. There are an increasing number of available medical 

countermeasures to protect the nation against CBRN agents. Supplies of 

countermeasures that are available are generally held in the SNS for use 

in a public health emergency. The SNS is designed to supplement and 

resupply state and local public health departments in the event of a 

national public health emergency such as a CBRN incident.15

To acquire medical countermeasures for use during a CBRN incident 

where there is a lack of a significant commercial market, Congress 

authorized the appropriation of approximately $5.6 billion and the use of a 

Special Reserve Fund for the procurement of certain countermeasures.16

 

14For example, the palatability of a mixture made by crushing solid oral doses of a drug 

into a food or liquid may need to be addressed for pediatric formulations. Palatability 

refers to the taste, texture, and smell of a medication, and is considered in the 

development of pediatric formulations.

The Special Reserve Fund has been used to acquire CBRN medical 

15Many state and local jurisdictions have purchased their own supplies of CBRN medical 

countermeasures, such as ciprofloxacin and doxycycline, to be better prepared to respond 

to public health emergencies. These stockpiles are typically created to ensure early 

access to medicines to protect first responders, among others, so that these essential 

employees can quickly respond to the emergency.

16The 2004 DHS Appropriations Act appropriated approximately $5.6 billion for biodefense 

countermeasures to remain available until 2013. Pub. L. No. 108-90, 117 Stat. 1137, 1148 

(2003). The Project Bioshield Act defined the Special Reserve Fund for purposes of the 

countermeasures program to include the Biodefense Countermeasures appropriations 

account. Pub. L. No. 108-276, 118 Stat. 852.
 

Page 8 GAO-13-438 Pediatric Medical Countermeasures

countermeasures for the SNS. The Pandemic and All-Hazards 

Preparedness Reauthorization Act of 2013 reauthorizes the Special 

Reserve Fund and authorizes $2.8 billion over 5 years, from fiscal year 

2014 through fiscal year 2018.17

In general, drugs, devices, diagnostics, and biologics such as vaccines, 

including CBRN countermeasures, cannot be marketed legally in the 

United States without FDA approval. To approve a countermeasure, FDA 

requires manufacturers to sufficiently demonstrate the safety, efficacy, 

and product quality of the countermeasure for the intended indication and 

population specified in the application.18 When products are approved, 

clinicians might prescribe them for unapproved indications or to 

unapproved populations, also referred to as “off-label use.” Generally, a 

doctor-patient relationship should exist for off-label dispensing of a 

medical countermeasure. In a CBRN incident, dispensing of 

countermeasures for approved or off-label indications may have to occur 

outside of the doctor-patient relationship.19

 

17Pub. L. No. 113-5, title IV, 127 Stat. 161, 193-195 (2013). An authorization of 

appropriations does not constitute an appropriation of public funds. Normally, an 

authorization of appropriations legislation is a prerequisite for making appropriations for 

the given programs or agencies.

According to HHS, many 

medicines and vaccines used in normal, standard medical care for the 

pediatric population are prescribed using this off-label practice. However, 

one possible consequence of off-label use of countermeasures by 

children is that some children, depending on weight or growth rates, may 

not receive the most appropriate dose of a medical countermeasure. In 

addition, some children may be at risk for side effects that are unique to 

18FDA requires drug companies, also known as drug manufacturers, to specify in their

applications for government approval of a product the indication intended for their 

products’ use. The indication generally describes the disease, condition, or symptoms that 

the drug is intended to treat, prevent, mitigate, cure, or diagnose, as well as the 

populations for which the manufacturer has conducted testing and for which it is seeking 

approval.

19Medical countermeasure dispensing provides medical countermeasures in support of 

treatment or prophylaxis (oral or vaccination) to the identified population in accordance 

with public health guidelines and recommendations.

FDA Approval and 

Emergency Authorization 

of Medical 

Countermeasures 
 

Page 9 GAO-13-438 Pediatric Medical Countermeasures

children, including adverse effects on their growth and development.20 To 

encourage the study of more drugs for pediatric use, Congress passed 

the Best Pharmaceuticals for Children Act in 2002, which provides 

financial incentives to product sponsors, and the Pediatric Research 

Equity Act of 2003. These laws encourage or require product sponsors to 

conduct pediatric studies, and also include labeling requirements, 

including labeling updates that result from pediatric drug studies.21

Although FDA regulates the use of medical countermeasures by 

approving their use by specific populations and for specific indications, 

during a public health emergency—such as a CBRN incident—FDA can 

authorize the use of those countermeasures by populations or for 

indications for which they have not been approved. This can occur in one 

of two ways. First, FDA can authorize the use of countermeasures that 

are unapproved, or approved but for a different indication, in a declared 

emergency to diagnose, treat, or prevent serious or life-threatening 

diseases or conditions caused by CBRN agents, when there are no 

adequate, approved, and available alternatives, and other criteria are 

met.22

 

20The Public Readiness and Emergency Preparedness Act provides compensation to 

individuals for serious physical injuries or deaths from countermeasures identified in a 

declaration of a public health emergency issued by the Secretary of HHS. The act 

authorizes HHS to issue a declaration that provides immunity from tort liability—except for 

willful misconduct—to certain individuals and organizations involved in the development, 

manufacture, distribution, administration, and use of these countermeasures. Pub. L. No. 

109-148, division C, 119 Stat. 2680, 2818-2832 (2005) (codified at 42 U.S.C. § 247d-6d).

This is referred to as an Emergency Use Authorization (EUA). In 

order for FDA to authorize the emergency use of a countermeasure, it 

must be reasonable to believe that, based on the evidence available, the 

product may be effective in diagnosing, treating, or preventing such 

21The Best Pharmaceuticals for Children Act offers drug sponsors—typically 

manufacturers—incentives to conduct pediatric studies, such as by extending the 

company’s market exclusivity for that product for 6 months. Protection from competition 

under market exclusivity can include the marketing of a generic copy. Pub. L. No. 107-

109, 115 Stat. 1408 (2002). The Pediatric Research Equity Act allows FDA to require 

manufacturers of drugs and biological products to submit pediatric testing data at the time 

of new drug applications. Pub. L. No. 108-155, 117 Stat. 1936 (2003). Both acts were 

extended in 2007 and are now permanent in the Food and Drug Administration Safety and 

Innovation Act of 2012. Pub. L. No. 110-85, §§ 401-404, 501-503, 121 Stat. 823, 866-890 

(2007); Pub. L. No. 112-144, § 501, 126 Stat. 993, 1039-1040 (2012) (pertinent provisions 

codified as amended at 21 U.S.C. §§ 355a, 355c).

22See 21 U.S.C. § 360bbb-3. The Secretary of HHS has delegated authority to make such 

determinations to the Commissioner of FDA.
 

Page 10 GAO-13-438 Pediatric Medical Countermeasures

disease or condition. In addition, the known and potential benefits of the 

product must outweigh the known and potential risks.23

Second, if a countermeasure’s safety and efficacy are not sufficiently 

documented, FDA may allow its use under an Investigational New Drug 

(IND) application, which allows for expanded access to medical 

countermeasures during an emergency24 while the safety and efficacy are

being investigated. In order for children to receive investigational medical 

countermeasures under an IND, written, informed consent is required. 

Documentation of informed consent is not required when children receive 

countermeasures through an EUA; however, to the extent practicable 

given the circumstances of the emergency, patients are to be informed 

about the known and potential benefits and risks of the countermeasure 

and the right to refuse the countermeasure.

HHS’s and PHEMCE’s medical countermeasure acquisition strategy is 

based on a multistep process. This process includes assessing the threat 

and public health consequences of CBRN agents, determining the type 

and quantity of needed medical countermeasures, evaluating the public 

health response capability, and developing and acquiring 

countermeasures against CBRN agents for the SNS. Because desired 

CBRN medical countermeasures may not be developed to a point where 

they are available for acquisition, HHS oversees and supports research 

and development of these countermeasures. (See fig. 1.) NIH and 

BARDA oversee and support CBRN medical countermeasure research 

and development, which is conducted in several stages: (1) basic 

research, (2) applied research, (3) early development, and (4) advanced 

development.25

 

23If state or local governments dispense countermeasures in a manner that is inconsistent 

with the terms of an EUA, their liability protections afforded by the Public Readiness and 

Emergency Preparedness Act may be affected.

NIH typically provides federal funding to academic 

2421 U.S.C. § 360bbb. The Secretary of HHS has delegated authority to make such 

determinations to the Commissioner of FDA. While INDs may be submitted for a variety of 

purposes, in this report we use the term IND to refer to only those submitted for expanded 

access to medical countermeasures during an emergency.

25In addition to approving or licensing medical countermeasures, FDA works with 

researchers throughout the development stages to review safety and efficacy test results 

and provide technical assistance to help ensure that research meets FDA’s regulatory 

requirements.

Medical Countermeasure 

Research, Development, 

Acquisition, and Support
 

Page 11 GAO-13-438 Pediatric Medical Countermeasures

centers and small biotechnology companies for basic and applied 

research and early development.26 Once an organization’s research is at 

the advanced development stage and moving toward the development of 

a product that will meet HHS’s specific requirements, it may partner with 

an established pharmaceutical or manufacturing company to continue the 

advanced development. Within HHS, the product transitions from NIH to 

BARDA to support its advanced development.27 If a countermeasure is 

not FDA-approved or licensed, its acquisition into the SNS is typically 

funded by the Project BioShield Special Reserve Fund.28

 

26Early, or basic, research is intended to better understand CBRN agents and the 

response of the host organism to the agents through the study of the cellular and 

molecular biology of agents and hosts, their physiological processes, and their genome 

sequences and structures. Applied, or translational, research builds on basic research by 

validating and testing concepts in practical settings to identify potential products. 

Successful concepts move from the applied research stage into the early development 

stage, in order to demonstrate basic safety, reproducibility, and ability to be used in 

humans.

If a 

countermeasure is FDA-approved or licensed, CDC generally purchases 

the countermeasure for the SNS.

27In the advanced development stage, potential medical countermeasures are further 

evaluated to demonstrate safety and efficacy for preventing, diagnosing, or treating 

disease. Successful products are then available for development and acquisition. In 

addition, BARDA determines that manufacturing, scale-up production, and licensing of 

countermeasures can be achieved in a timely and reliable manner.

28The Project BioShield Act of 2004 provides that the Special Reserve Fund may be used 

to acquire countermeasures for which the HHS Secretary determines that sufficient and 

satisfactory clinical experience or research data support a reasonable conclusion that the 

product will qualify for FDA approval or licensing within 8 years. In 2013, the Pandemic 

and All-Hazards Preparedness and Reauthorization Act extended the timeline for 

acquisitions, allowing for the use of the Special Reserve Fund to acquire countermeasures 

for which the HHS Secretary determines that sufficient and satisfactory clinical experience 

or research data support a reasonable conclusion that the product will qualify for FDA 

approval or licensing within 10 years. 42 U.S.C. § 247d-6b(c)(1)(B)(i)(III)(bb). 
 

Page 12 GAO-13-438 Pediatric Medical Countermeasures

Figure 1: Processes for Medical Countermeasure Development and Acquisition

a

In addition to approving, licensing, or clearing medical countermeasures, FDA works with 

researchers throughout the development stages to review safety and efficacy test results and provide 

technical assistance to help ensure that research meets FDA’s regulatory requirements.

Public health emergency response planning for CBRN incidents requires 

efforts at the federal, state, and local levels. Governments at each level 

have developed emergency response plans that outline their respective 

responsibilities during public health emergencies, which include 

responsibilities for distributing and dispensing medical countermeasures 

to the public. The federal government is responsible for planning the 

federal response to CBRN incidents. HHS has developed response plans 

for specific CBRN agents to help coordinate the federal response to such 

emergencies.29

 

29HHS has developed 12 response plans to address CBRN incidents as of December 

2012: 8 are For Official Use Only, and 4 are publicly available. Officials told us there are 

fewer publicly available plans than For Official Use Only plans because some plans are 

currently in draft form and will be available in public versions once they have been cleared 

by HHS for release. The For Official Use Only response plans address some chemical 

(nerve agent, blister agent, and chlorine tank explosion), biological (pneumonic plague, 

botulism food contamination, and aerosolized anthrax), radiological (radiological dispersal 

device), and nuclear (improvised nuclear device) incidents. The publicly available 

response plans address biological (botulinum food contamination and aerosolized 

anthrax), radiological (radiological dispersal device), and nuclear (nuclear detonation) 

incidents.

Federal assistance to state and local governments would 

Planning for Medical 

Countermeasure 

Distribution and 

Dispensing 
 

Page 13 GAO-13-438 Pediatric Medical Countermeasures

be provided if resources were unavailable or if state and local 

governments were overwhelmed and requested public health or medical 

assistance from the federal government. HHS would then direct CDC to 

distribute medical countermeasures from the SNS to the states.

Once CDC distributes medical countermeasures from the SNS to a state, 

in most instances the state then distributes the countermeasures to local 

governments, based on established plans. State and local governments 

are responsible for developing plans for receiving, distributing, and 

dispensing medical countermeasures from the SNS. These plans are 

intended to describe all functions that are required to accomplish these 

tasks, in order to get medical countermeasures to the affected population 

as quickly and efficiently as possible. Some states plan to receive 

countermeasures and immediately turn them over to a local jurisdiction 

for staging, distributing, and dispensing during an emergency. Other 

states plan to receive medical countermeasures at a state warehouse 

facility and then deliver them directly to points of dispensing (POD) or 

hospitals. Each state is responsible for determining the best method for 

its circumstances and resources. Local governments, in turn, are 

responsible for receiving and dispensing medical countermeasures in a 

timely and efficient manner.

About 60 percent of CBRN medical countermeasures in the SNS have 

been approved for children, but in many instances approval is limited to 

specific age groups. Specifically, PHEMCE officials stated that about 

38 percent of the CBRN medical countermeasures in the SNS have been 

approved for children of all ages for treatment of certain CBRN threats. 

For example, ciprofloxacin and doxycycline as antimicrobials indicated for 

postexposure prophylaxis of inhalational anthrax, atropine as a treatment 

for exposure to nerve gas, and raxibacumab as an anthrax antitoxin have 

all been approved for use in the pediatric population for these indications. 

In addition, 22 percent of the CBRN medical countermeasures in the SNS 

have been approved for use by some, but not all, pediatric age groups for 

treatment of certain CBRN threats. For example, Prussian blue—a 

medical countermeasure that removes certain internalized radiological 

particles from the body—is approved for children ages 2 and above for 

that indication, but has not been approved for those less than 2 years of 

age. PHEMCE officials stated that the remaining 40 percent of the CBRN 

More than Half of 

CBRN Medical 

Countermeasures 

Have Been Approved 

for at Least Some 

Children 
 

Page 14 GAO-13-438 Pediatric Medical Countermeasures

countermeasures have not been approved for any pediatric use.30 For 

example, anthrax vaccine adsorbed (AVA) has not been approved for use 

by any children.

31

According to HHS officials, almost all medical countermeasures in the 

SNS can be used by children in an emergency if already approved for 

children or if FDA authorizes their use through an EUA or if an IND 

protocol is in effect. Even products that are not approved for a CBRN 

indication may be used in a public health emergency under an EUA—

after an emergency has been declared—or under an IND protocol. HHS 

officials told us that almost all medical countermeasures that are not 

approved for use by the pediatric population can be used in an 

emergency under an EUA. However, some countermeasures, such as 

AVA vaccine, lack sufficient data to support their use by the pediatric 

population under an EUA. This vaccine would need to be administered to 

the pediatric population under an IND protocol. HHS officials explained 

that two different IND protocols for AVA have been developed in 

preparation for a potential anthrax-related public health emergency. The 

first protocol is designed to provide children access to the vaccine. It 

would require parents or guardians to sign consent forms to allow their 

Furthermore, some of the CBRN medical 

countermeasures in the SNS have not been approved to treat individuals 

of any age for the specific indications for which they have been 

stockpiled. For example, ciprofloxacin is stockpiled in the SNS for the 

treatment of anthrax, plague, and tularemia, but is not approved for these 

indications. It is, however, approved for postexposure prophylaxis of 

inhalational anthrax in all age groups. In addition, for certain CBRN 

threats, there are no countermeasures available in the SNS because 

treatments for the conditions do not exist beyond supportive care. 

 

30According to members of PHEMCE, these percentages were derived from an analysis of 

the regulatory status of the contents of the SNS performed in June 2012. However, these 

percentages may not entirely reflect the availability of countermeasures to be used by 

children because three items that were counted as not approved for use by children are 

therapeutic ointments that are not labeled by age. An official from PHEMCE stated that 

therapeutic ointments are assumed to be safe for use by children unless there is a 

warning against their use.

31In March 2013, the Presidential Commission for the Study of Bioethical Issues reported 

that the federal government would have to take multiple steps, including minimal-risk 

research with adult volunteers, before considering pediatric clinical trials to test the safety 

and efficacy of AVA in children. The Presidential Commission for the Study of Bioethical 

Issues, Safeguarding Children: Pediatric Medical Countermeasure Research 

(Washington, D.C.: March 2013).
 

Page 15 GAO-13-438 Pediatric Medical Countermeasures

children to receive anthrax vaccine during an emergency. The second 

IND protocol would require parents or guardians to sign consent forms to 

allow their children to participate in a research study after receiving the 

vaccine.32

Although HHS can provide information on the proportion of SNS 

countermeasures that can be used by children, it cannot provide 

information on the funds invested in procuring these countermeasures. 

HHS does not separately track the funds that it has invested in the 

acquisition and development of pediatric medical countermeasures 

because it does not account separately for investments related to the 

different populations. HHS procures CBRN medical countermeasures for 

the SNS based on, among other things, the list of material threats to the 

nation, public health response and medical consequence assessments, 

and available resources. Since 2004, BARDA has invested over $4 billion 

of Project BioShield’s Special Reserve Fund, which supports advanced 

development and manufacturing of potential CBRN medical 

countermeasures, in contracts to support medical countermeasure 

development in healthy and at-risk populations, including the pediatric 

population. In addition, between 2009 and 2012, CDC’s budget to 

maintain or acquire licensed CBRN medical countermeasures for the 

SNS was over $1.5 billion; however, no funds were specifically 

designated exclusively for products to be used only by the pediatric 

population.

(See app. I for additional information on the regulatory status of 

types of CBRN medical countermeasures in the SNS for the pediatric 

population.)

 

32HHS officials stated that under this second IND protocol, parents would consent to 

having blood drawn from their children when the vaccine was administered during an 

emergency, as well as submitting other health information about their children after they 

were vaccinated in order to follow up on the safety of the vaccine in children. Officials 

stated that these actions could provide FDA with some safety and immunogenicity data to 

inform use of the vaccine in children in future emergencies. Officials emphasized that if 

there are no data available in advance of public health emergencies, the gaps in the data 

could be addressed only during an actual event.
 

Page 16 GAO-13-438 Pediatric Medical Countermeasures

HHS faces economic, regulatory, scientific, and ethical challenges in its 

efforts to develop and acquire CBRN medical countermeasures for 

children in an emergency. Despite these challenges, HHS is taking steps 

to focus on the pediatric population and develop pediatric formulations of

existing medical countermeasures.

HHS faces a variety of interrelated challenges in developing and 

acquiring pediatric CBRN medical countermeasures, including economic, 

regulatory, scientific, and ethical issues that impede the ability of HHS to 

address the needs of children during a CBRN incident.33

 

33These challenges have been described in past GAO reports, various HHS reports, and 

reports by subject-matter experts. See 

Economic 

challenges facing HHS include the high failure rate of research, 

development, approval, and licensure of most drugs, vaccines, and 

diagnostic devices. Agency and industry officials told us that the risk of 

failure in developing countermeasures for children is even higher than the 

risk in developing them for the adult population. This risk, as well as a 

lack of a commercial market for most CBRN medical countermeasures, 

has made it difficult for HHS to attract companies willing to invest in such 

development and has therefore impeded HHS’s ability to acquire the 

needed countermeasures. In addition, various reports have stated that 

insufficient economic incentives are available to encourage the private 

sector to invest millions of dollars to develop potential new pediatric 

GAO-12-121, GAO-11-606, and GAO-11-567T. 

See also Department of Health and Human Services, The Children’s HHS Interagency 

Leadership on Disasters (CHILD) Working Group: Summary of Recommendations and 

Implementation Efforts (April 2012); National Commission on Children and Disasters, 

2010 Report to the President and Congress. 

HHS Faces 

Challenges in 

Developing and 

Acquiring Pediatric 

Medical 

Countermeasures but 

Is Taking Steps to 

Address the Needs of 

the Pediatric 

Population 

HHS Faces Economic, 

Regulatory, Scientific, and 

Ethical Challenges in 

Developing and Acquiring 

Pediatric Medical 

Countermeasures 
 

Page 17 GAO-13-438 Pediatric Medical Countermeasures

medical countermeasures.34

The FDA regulatory pathway for developing pediatric CBRN medical 

countermeasures also poses unique challenges making it more difficult 

than for adult countermeasures. The Institute of Medicine, the National 

Biodefense Science Board, HHS, and FDA have stated that it is difficult to 

meet FDA’s requirements for data from adequate and well-controlled 

clinical investigations to support the approval of pediatric medical 

countermeasures because large, complex clinical trials are needed to 

prove safety and efficacy. As a result of this regulatory challenge, FDA 

has increasingly relied on alternative sources of data in order to approve, 

license, or authorize the use of medical countermeasures by children. For 

example, FDA allows researchers to submit evidence of efficacy obtained 

from historical inferences and appropriate studies in animals in 

accordance with FDA’s Animal Rule.

According to CDC officials, while it is 

desirable to have oral liquid formulations available for young children who 

cannot swallow pills, it is not always practical to develop and acquire 

them for the SNS because such countermeasures for children can be 

more costly to procure and maintain, have shorter shelf lives, and may 

exceed manufacturer capability, as opposed to alternatives such as pill 

crushing and mixing that can be explored for all but the youngest children. 

CDC estimated that it would cost approximately $3 billion to purchase 

sufficient quantities of oral liquid formulations of countermeasures for the 

SNS to support the needs of all children—an amount well over CDC’s 

approximately $600 million annual budget for CBRN medical 

countermeasures over the past 5 years. Further, because the shelf life of 

liquids is shorter than that of tablets, oral liquid formulations would require 

more frequent investments. Finally, even if such funds were available, the 

manufacturing capacity to meet such acquisition requests may not be 

available.

35

 

34Institute of Medicine, Advancing Regulatory Science for Medical Countermeasure 

Development (National Academy of Sciences: 2010); National Biodefense Science Board, 

Where Are the Countermeasures? Protecting America’s Health from CBRN Threats

(2010); Department of Health and Human Services, The Children’s HHS Interagency 

Leadership on Disasters (CHILD) Working Group: Summary of Recommendations and 

Implementation Efforts. 

Another challenge to developing 

3521 C.F.R. §§ 314.600-650; 601.90-.95. The Animal Rule states that when it is neither 

ethical nor feasible to conduct human efficacy studies, FDA may grant marketing approval 

of CBRN countermeasures based on adequate and well-controlled animal studies when 

the results of those studies establish that the drug or biological product is reasonably likely 

to produce clinical benefit in humans.
 

Page 18 GAO-13-438 Pediatric Medical Countermeasures

countermeasures for children is that manufacturers are permitted, under 

certain circumstances, to avoid testing drugs for use by children. For 

example, manufacturers of CBRN countermeasures often seek an orphan 

drug designation for a new countermeasure,36 which, if granted, exempts 

the manufacturer from the requirement to conduct pediatric studies under 

the Pediatric Research Equity Act.37

There are scientific challenges in obtaining sufficient pediatric safety and 

efficacy information to appropriately inform the use of CBRN medical 

countermeasures for children. For example, FDA officials told us that 

extrapolating data to support efficacy information on a medical 

countermeasure from animal studies presents not only regulatory, but 

also complex scientific challenges to understanding how children would 

react to exposure to CBRN agents. NIH and industry officials told us that 

exposure in juvenile animal models is also not well understood. For 

example, extrapolating data from animal studies presents other scientific 

challenges in understanding the response to the medical 

countermeasures used to prevent or treat the disease or condition. 

Appropriate animal models have not yet been developed for many CBRN 

agents. In addition, the presentation of the disease or condition that 

humans manifest following exposure to a CBRN agent may not be the 

same as that for animals following exposure to the same CBRN agent, 

thus complicating the task of researchers that are relying on animal 

models. Further, there is an initial hurdle of extrapolating data from animal 

models for adults first; only after those data have been extrapolated and 

applied to the adult population can researchers extrapolate to children. 

For example, in order to develop or indicate a countermeasure for use in 

children, scientists generally take an existing countermeasure that has 

 

36A drug may receive an orphan designation if it is intended for conditions or diseases that 

affect fewer than 200,000 persons in the United States. CBRN and non-CBRN drugs that 

qualify and receive orphan designation are exempt from pediatric research requirements 

under the Pediatric Research Equity Act. Orphan designation qualifies the sponsor of the 

product for the tax credit and marketing incentives of the Orphan Drug Act. See Pub. L. 

No. 97-414, 96 Stat. 2049 (1983) (codified at 21 U.S.C. §§ 360aa-360cc; 26 U.S.C. 

§ 45C).

37Until 1997, most drugs used to treat children were tested for safety and efficacy only in 

adults. The Pediatric Research Equity Act of 2003 requires assessments of safety and 

efficacy of drugs and biological products for pediatric use for the indication for which they 

are approved in the adult population. HHS may grant deferrals and full or partial waivers 

from these requirements. Products with an orphan drug designation for a rare disease or 

condition are exempt from the requirements of the Pediatric Research Equity Act.
 

Page 19 GAO-13-438 Pediatric Medical Countermeasures

already been developed for adults and then adjust certain variables such 

as weight-based dosing and delivery mechanisms to test the safety and 

efficacy of the countermeasure in children. This is not straightforward 

because children may have special susceptibilities to the CBRN agent 

and special age- and weight-dependent responses to the medical 

countermeasures. 

Finally, HHS faces ethical challenges in its efforts to develop and acquire 

pediatric medical countermeasures because, absent a CBRN event, it is 

generally not ethical or feasible to obtain dosing, safety, and efficacy data 

for children when there is no potential direct benefit to them in the context 

of a clinical trial. FDA-regulated clinical trials that include children as 

subjects must consider both the risks to which a child may be exposed in 

a clinical investigation and whether the proposed intervention offers a 

prospect of direct benefit to the child.38 Because children can be enrolled 

as subjects in research only when directly necessary and when the 

research is ethically sound, industry officials we met with stated that 

researching CBRN medical countermeasures’ effect on children is nearly 

impossible. Industry officials told us that knowing that CBRN medical 

countermeasure research has a clear, direct benefit to a child 

participating in a study would always be unlikely because diseases 

caused by CBRN agents do not generally occur naturally.

Although challenges persist in developing and acquiring pediatric medical 

countermeasures, HHS is beginning to address gaps in the SNS for 

pediatric medical countermeasures by focusing agency efforts on 

children, developing pediatric formulations of medical countermeasures in 

the SNS, and preparing and reviewing EUA and IND application materials 

in advance of emergencies.

HHS is taking steps to focus department-wide efforts on children’s CBRN 

medical countermeasure needs. In 2010, FDA announced its Medical 

Countermeasures Initiative, which is intended to foster the development 

and availability of medical countermeasures, including those intended to 

 

3821 C.F.R. part 50, subpart D (Additional Safeguards for Children in Clinical 

Investigations).

HHS Is Taking Steps to 

Focus on the Pediatric 

Population and Develop 

Pediatric Formulations of 

Existing Medical 

Countermeasures 

Focusing Department-wide 

Efforts
 

Page 20 GAO-13-438 Pediatric Medical Countermeasures

be used by children.39 FDA officials said the initiative has improved the 

regulatory pathway for advancing the development and acquisition of 

medical countermeasures, for example, by clarifying and streamlining its 

review process and countermeasure requirements, which may entice 

manufacturers to develop new, novel medical countermeasures, including 

countermeasures with pediatric applicability. In addition, in 2010, HHS 

increased its focus on children’s needs with the establishment of the 

CHILD Working Group, which was formed to identify and integrate 

activities related to the needs of children across all HHS inter- and 

intragovernmental disaster planning activities and operations. The CHILD 

Working Group has developed recommendations for how HHS can 

improve the delivery of care to children who are affected by disasters. In 

2011, in the area of medical countermeasures, the CHILD Working Group 

recommended that HHS provide clarity in the regulatory pathway for 

pediatric medical countermeasures; obtain the appropriate data, when 

available, to provide clinical pediatric dosing and use guidance for 

existing medical countermeasures; and gather safety and efficacy data 

from nontraditional sources to support the use of pediatric medical 

countermeasures under EUAs and for eventual FDA approval.40

 

39The Medical Countermeasures Initiative is composed of three pillars that (1) enhance 

the medical countermeasure review process; (2) advance regulatory science for medical 

countermeasure development; and (3) optimize the legal and policy framework for 

approving medical countermeasures.

According to HHS officials, many of the recommendations from the 

CHILD Working Group are being adopted by HHS. For example, in 2011, 

HHS developed the Pediatric Obstetric Integrated Program Team, which 

includes pediatric and obstetric subject-matter experts who advise 

PHEMCE on pediatric and obstetric medical countermeasure issues. This 

integrated program team is intended to recommend that pediatric medical 

countermeasure needs are consistently considered throughout the entire 

medical countermeasure development process and that pediatric subjectmatter experts help consider complex ethical, scientific, and legal issues 

associated with studies that are necessary for the licensure and approval 

of medical countermeasures for children. In 2012 the integrated program 

team conducted a review of the contents of the SNS to determine the 

suitability of the contents for use by children, and it subsequently used the 

review to make recommendations to PHEMCE, and to petition for new 

40Department of Health and Human Services, 2011 Report of the Children’s HHS 

Interagency Leadership on Disasters (CHILD) Working Group: Progress and Future 

Directions (January 2012). 
 

Page 21 GAO-13-438 Pediatric Medical Countermeasures

medical countermeasure development. The content of the review is a 

work product of the integrated program team, and HHS has no plans to 

formally issue it. According to HHS, the findings and recommendations 

were considered during the 2012 SNS Annual Review. Further, in 2012, 

BARDA announced that where feasible and appropriate, it would be 

including development of medical countermeasures for the pediatric 

population as part of all base contracts moving forward.

HHS has taken steps to support the development of CBRN medical 

countermeasure formulations for children, and has begun to base the 

pediatric dosing information on other evidence, such as by extrapolating 

from relevant and historical data of the countermeasure. 

Countermeasures are not approved for an indication unless data on the 

safety and efficacy of the countermeasure are available for a particular 

population, such as children. According to FDA officials, the agency has 

determined that in a smallpox emergency, the investigational smallpox 

vaccine under development may be authorized for use under an EUA in 

populations with compromised immune systems, including children.

Specifically, in 2007 a second-generation smallpox vaccine, developed 

for persons determined to be at high risk for infection, was licensed based 

on data from clinical trials and the routine vaccination of infants in the 

United States through 1972. The vaccine was not studied in pediatric 

populations; however, this second-generation vaccine was similar to the 

vaccine that was routinely used to vaccinate infants in the United States

through 1972 and had been demonstrated to be safe and effective in 

children. Therefore, FDA has determined that this vaccine could be used 

in pediatric populations under its license in a smallpox emergency. 

Similarly, pralidoxime chloride, prescribed as an antidote to treat nerve 

agent poisoning, has also been approved for use by children based on 

the extrapolation of efficacy data from both the adult and pediatric 

populations. In addition, FDA has used historical data from other 

countries to support EUAs or product approvals for pediatric indications 

when analogous U.S. data were neither available nor obtainable. For 

example, according to FDA officials, in 1987, a radiological incident in 

Brazil provided the majority of the pediatric data that FDA reviewed to 

assess the safety and efficacy of a radiological countermeasure. The data 

included the use of the countermeasure by both adults and children. The 

review allowed FDA to approve the countermeasure for children ages 2 

and older in 2003.

PHEMCE has also encouraged adapting and manufacturing of oral liquid 

formulations of existing medical countermeasures in order to ease 

dispensing of countermeasures in the SNS for children. For example, 

Adapting Pediatric 

Formulations of Existing 

Medical Countermeasures 
 

Page 22 GAO-13-438 Pediatric Medical Countermeasures

BARDA is contracting with industry partners to manufacture a liquid form 

of a countermeasure that removes certain radioactive particles from the 

body through the intestinal tract.41 Additionally, PHEMCE is considering 

lowering the recommended age at which children should be administered 

oral liquid suspensions of doxycycline in lieu of crushed tablets. CDC 

officials told us that they would like to have more oral liquid suspensions 

considered for the SNS; however, PHEMCE officials have reported that 

crushing tablets would be an acceptable alternative and would have 

benefits for storage, dispensing, and dosing. As a result, FDA and CDC 

have developed instructions for crushing certain approved medical 

countermeasures for use by people who cannot swallow tablets or 

capsules. The crushing instructions include instructions for mixing the 

countermeasures with food or drink to make them more palatable to 

children. Crushing instructions generally include weight-based dosing 

instructions, the number of doses required per day, and instructions for 

how to crush pills and mix them.42

Relevant component agencies within HHS collaborate to prepare and 

review materials for EUAs and INDs in advance of public health 

emergencies to ensure that sufficient data are available to support 

authorizations for the use of certain medical countermeasures by 

children. According to CDC officials, the agency assesses, on a regular 

basis, the contents in the SNS and updates and assembles data and 

information for those countermeasures that are not approved for use by 

children so that in the event of a public health emergency, the contents 

can be disseminated to states quickly. Specifically, to prepare for such an 

emergency, CDC collaborates with BARDA, NIH, FDA, and 

manufacturers to develop the EUA and IND submissions in advance of an 

actual incident, so that all medical countermeasures in the SNS can be 

used by children. In addition, they consult with the American Academy of 

Pediatrics on pediatric issues.

 

41That countermeasure, Prussian blue, is currently licensed for adult and pediatric patients 

over 2 years of age. 

42Department of Health and Human Services, Public Health Emergency Medical 

Countermeasures Enterprise, Pediatric Medical Countermeasure Roundtable for National 

Health Security: Meeting Report (Oct. 13-14, 2010). Some of the medical 

countermeasures being considered for crushing include Prussian blue, doxycycline, and 

ciprofloxacin.

Preparing and Reviewing EUA 

and IND Materials in Advance 

of Emergencies 
 

Page 23 GAO-13-438 Pediatric Medical Countermeasures

More than half of HHS’s emergency response plans that we examined 

included information about pediatric medical countermeasures. CDC and 

FDA developed guidance on pediatric dispensing for state and local 

government use. The state and local plans we examined also provided 

details about dispensing to the pediatric population during an emergency.

Of the 12 HHS CBRN response plans we reviewed, more than half 

included information about dispensing pediatric medical 

countermeasures. Specifically, 7 of these threat-specific plans contained 

information about medical countermeasures that could be dispensed for 

use by children in the event of a CBRN incident.43 The type of information 

included varied by plan. For example, two of the plans for responding to 

biological threats identified preferred and alternative countermeasures, 

and the appropriate dosages, that should be dispensed to children during

an event. For a chemical incident, one of the plans indicated a 

premedication that should be used in pediatric patients before intubating 

them.44

 

43Not all of the plans would necessarily include information about which specific medical 

countermeasure is to be dispensed to children during a CBRN incident, according to HHS 

officials. For example, treatment for exposure to certain CBRN agents sometimes only 

requires supportive care, rather than a specific medical countermeasure.

The response plans for nuclear or radiological incidents also 

included information about medical countermeasures that could be used 

by children, although one plan noted that an EUA would be required 

before some of the countermeasures could be dispensed to the pediatric 

population. Although more than half of HHS’s response plans included 

information about dispensing specific countermeasures to children, HHS 

officials told us that the purpose of these plans is to provide guidance for 

emergency responses at the federal level, and not instructions for use at 

the state and local level, which is where dispensing to children would 

44Intubation is a medical procedure in which a tube is placed into the windpipe (trachea), 

through the mouth or the nose. In most emergency situations, it is placed through the 

mouth.

HHS and State and 

Local Governments 

Address Dispensing of 

Pediatric Medical 

Countermeasures in 

Their Response Plans

More than Half of HHS’s 

Response Plans We 

Reviewed Include 

Information about 

Dispensing Pediatric 

Medical Countermeasures
 

Page 24 GAO-13-438 Pediatric Medical Countermeasures

occur.45 While these plans are intended primarily to support the federal 

response, they could also be used by state and local governments to 

inform their activities as part of planning their own response to a public 

health emergency.46

HHS officials told us that they were moving away from the use of 

separate, threat-specific response plans and were developing a single 

“all-hazards” response plan.47 This all-hazards plan would include 

sections for responding to specific CBRN events. According to HHS 

officials, these sections would address pediatric dispensing, including 

EUA and IND requirements. Additionally, a briefing paper about the 

pediatric population in disasters would be included in the all-hazards plan.

Both CDC and FDA developed guidance for the dispensing of CBRN 

medical countermeasures from the SNS to the public, including children. 

For example, CDC developed guidance about receiving, distributing, and 

dispensing contents from the SNS to help state and local emergency 

management and public health personnel plan for the use of 

countermeasures from the SNS.48

 

45The federal government emergency responses are intended to provide support, 

resources, program implementation, and services that are most likely needed to save 

lives, protect property and the environment, restore essential services and critical 

infrastructure, and help victims and communities return to normal following domestic 

incidents. HHS is the primary agency responsible for efforts related to public health and 

medical services in order to provide the mechanism for coordinated federal assistance to 

supplement state, tribal, and local resources in response to an emergency.

The guidance, which could be used as 

a reference document or as a checklist by local and state planners, 

referred to the pediatric population in multiple sections, including an 

appendix about pediatric dispensing considerations. For example, the 

guidance described how the color “pink” is used to identify pediatric 

supplies included in the SNS. Additionally, the appendix noted that the 

46HHS also developed two web-based resources that can also be used to inform CBRN 

planning at other levels of government—the Chemical Hazards Emergency Medical 

Management and Radiation Emergency Medical Management guidance documents. See 

http://www.chemm.nlm.nih.gov/ and http://www.remm.nlm.gov/, respectively, accessed 

January 25, 2013.

47HHS officials said that they anticipate releasing this plan by August 2013.

48Centers for Disease Control and Prevention, Receiving, Distributing, and Dispensing 

Strategic National Stockpile Assets: A Guide for Preparedness, ver.10.02 (August 2006).

CDC and FDA Guidance 

for State and Local 

Governments Includes 

Information on Pediatric 

Dispensing 
 

Page 25 GAO-13-438 Pediatric Medical Countermeasures

SNS has limited amounts of oral suspensions of certain countermeasures 

for use by children and offered potential solutions for state and local 

governments to consider when addressing this shortage. These solutions 

included assigning someone the responsibility of mixing suspensions at a 

POD site or compounding tablets of medical countermeasures into oral 

suspensions.49

In addition, CDC and FDA developed other guidance on dispensing 

medical countermeasures, including to the pediatric population, which 

could be shared with and used by state and local governments. For 

example, CDC developed a website and training opportunities for state 

and local governments to use when planning for dispensing medical 

countermeasures. The information shared through the website and 

training addresses dispensing in general, for the most part, although a 

small portion of the training touches on dispensing to the pediatric 

population. FDA also developed guidance on dispensing pediatric 

medical countermeasures. For example, because it can be difficult for 

children to swallow pills, FDA developed instructions for how to crush and 

mix doxycycline with water and then add to food to mask the taste of 

doxycycline. Additionally, FDA, with CDC, developed an information sheet 

about using doxycycline for the prevention of anthrax that state and local 

governments could share with their populations during an event. This 

sheet included dosing instructions for administering doxycycline to 

children.

CDC and FDA also collaborated to prepare, in advance of an actual 

CBRN incident, information about medical countermeasures that require 

an EUA or IND to have ready to share with state and local governments 

should an emergency occur.50

 

49CDC has developed a new version of this guidance, which CDC has tentatively 

scheduled for release during calendar year 2013.

This included information about how a 

countermeasure would be dispensed to children under an EUA or IND

protocol, should there be a need. State and local governments could 

50This information is not shared with state and local governments in advance of a CBRN 

incident because medical countermeasures that are dispensed under an EUA or IND 

protocol have not been FDA-approved for use for these indications, according to HHS 

officials. Information about using medical countermeasures for these indications can 

change as new data are made available, and FDA and CDC wait to release such 

information until an emergency occurs to ensure that the information reflects the most 

recent data available.
 

Page 26 GAO-13-438 Pediatric Medical Countermeasures

then, in turn, share this information with their populations in the event of 

an emergency. For example, CDC and FDA developed an EUA fact sheet 

with instructions for administering doxycycline to children at home. CDC 

officials told us that they have information available for all medical 

countermeasures that could be used by children under an EUA. 

Information about pediatric dispensing under an EUA or IND protocol 

would be sent electronically—rather than in hard copy—to state and local 

governments because dispensing information can change and hard 

copies could become outdated.

State and local governments have provided details about pediatric 

dispensing in their emergency response plans. All seven of the state 

response plans we reviewed addressed the dispensing of 

countermeasures to the pediatric population during a CBRN incident.51

Although the states’ plans varied in format, they were consistent with one 

another in terms of the type of information they included about dispensing 

to children. For example, more than half of the states included information 

about pediatric dosing or formulations. Additionally, all seven states 

adopted some version of a “family member pick-up” policy—sometimes 

referred to as a “head of household” policy—which would allow adults to 

pick up medicines for other family members, including children, during an 

event. This policy is intended to eliminate disruptions to the dispensing 

process while simultaneously reducing patient numbers and increasing 

the number of persons treated during an incident. In addition, each state’s

plan provided other information about how medical countermeasures 

would be dispensed to children, either under a family member pick-up 

policy or through a POD.52

• One state’s medical countermeasure dispensing guidance noted that 

a POD should have specialized items for dispensing to children, such 

as scales for weighing children (if they are present and their parents 

do not know their weights) and mixing equipment to make pediatric 

portions. Additionally, the state’s Point of Dispensing Field Operations 

For example,

 

51Officials from one additional state also provided us with written information about its 

plans, but did not provide us with supporting documents to review.

52Despite having a family member pick-up policy in place, there are still reasons why a 

child might go to a POD. For example, parents might not want to be separated from their 

children during a CBRN incident, or unaccompanied minors may go to the POD looking for 

medication.

State and Local 

Government Plans Provide 

Details about Pediatric 

Dispensing 
 

Page 27 GAO-13-438 Pediatric Medical Countermeasures

Guide suggested that the POD include a staff member who can be 

responsible for ensuring drugs are properly packaged and instructions 

for children are given.

• One state’s plan discussed how oral antibiotic suspensions and 

syrups would be provided for the treatment of children who have 

trouble swallowing tablets, and that converting ciprofloxacin and 

doxycycline tablets into oral suspensions was recommended as an 

alternative for providing additional quantities of pediatric prophylactic 

regimens, due to the limited quantities of oral suspensions in the SNS. 

The plan included instructions for reconstituting these medical 

countermeasures.

• One state’s plan provided information about dispensing 

countermeasures to unaccompanied children who request treatment 

at PODs. The issue of consent for emergency care to a child in a 

disaster was discussed.53 Officials from this state told us that it works 

closely with the state’s health care coalitions to ensure that the 

regional guidelines include pediatric-focused strategies.54

As we found with the states, all seven of the local governments that 

provided us with plans addressed dispensing countermeasures for the 

pediatric population during a CBRN incident.

Additionally, 

because pediatric doses are not stored in the SNS in large quantities, 

the state and local jurisdictions rely on partnerships with community 

and chain pharmacies to compound and reconstitute medications for 

children in an emergency.

55

 

53In general, parental consent is required for the medical treatment of children.

Like the states’ plans, the 

local governments’ plans varied in their format but were consistent with 

one another in terms of the type of information they included about 

dispensing to children. For example, more than half of the plans included 

information about screening children at a POD. Additionally, all seven 

local governments plan to implement versions of family member pick-up 

policies in the event of an emergency. The local governments’ plans also 

54These health care coalitions, which support the state’s health care preparedness 

program, work with local partners from different regions of the state to prepare hospitals, 

emergency medical service, and supporting health care organizations to deliver 

coordinated and effective care to individuals affected by public health emergencies.

55An official from one additional local government also spoke with us about its emergency 

response plans, but could not provide us with supporting documents to review.
 

Page 28 GAO-13-438 Pediatric Medical Countermeasures

included other ways to address the needs of the pediatric population 

when dispensing medical countermeasures. For example,

• One local government’s POD plan described different types of 

dispensing, one of which is called “slow dispensing.” Slow dispensing 

requires children under the age of 9 to pass through medical 

screenings, if they go to the POD.

• Another local government’s plan described the setup of a special 

assistance station at a POD, where individuals could obtain 

medication for children under 9 years of age. Special assistance 

personnel could determine appropriate pediatric dosing by referring to 

available weight charts.

• Finally, one local government’s plan discussed allowing “fast-tracking 

protocols,” which would allow individuals with children to be diverted 

from the main lines and directed to a Help Desk to receive assistance.

CDC officials noted that state and local governments handle the 

dispensing of countermeasures to children in the same way as for adults; 

that is, the dispensing of countermeasures for both children and adults 

occurs at POD sites. However, CDC officials also told us that special 

considerations can still be made for children—for example, by 

establishing separate family lines in the PODs. We found examples of this 

consideration in some of the plans we reviewed.

We provided a draft of this report to HHS for comment. In its written 

comments, reproduced in appendix II, HHS concurred with our findings. 

HHS reiterated information provided in our report, including that the 

development, procurement, and dispensing of medical countermeasures 

for the pediatric population is integrated into the PHEMCE’s framework 

for public health preparedness across multiple component agencies, that 

state and local jurisdictions play an important role in responding to public 

health emergencies, and that the pace of progress in drug development is 

limited by the complex issues that surround the testing of 

countermeasures in children. In addition, HHS emphasized that the needs 

of the pediatric population have been a priority for HHS since the origins 

of Project Bioshield, and that the department is continuously progressing 

in this area. HHS also provided technical comments that we incorporated 

as appropriate.

Agency Comments
 

Page 29 GAO-13-438 Pediatric Medical Countermeasures

We will send copies of this report to the Secretary of Health and Human 

Services and interested congressional committees. We will also make 

copies available at no charge on GAO’s website at http://www.gao.gov. 

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. Key contributors to this report are listed in 

appendix III. 

Marcia Crosse

Director, Health Care
Appendix I: Pediatric CBRN Medical 

Countermeasures in the Strategic National 

Stockpile

Page 30 GAO-13-438 Pediatric Medical Countermeasures

Table 1 presents additional information about certain types of chemical, 

biological, radiological, and nuclear (CBRN) medical countermeasures 

available in the Strategic National Stockpile (SNS) by CBRN threat, as 

well as their regulatory status, for the pediatric population. The 

information presented is a general overview of countermeasures that are 

available for the pediatric population in the event of a CBRN incident, and 

does not include a complete list of all of the variations of each 

countermeasure.

Table 1: Overview of Certain Types of Chemical, Biological, Radiological, and Nuclear (CBRN) Medical Countermeasures 

Available in the Strategic National Stockpile (SNS) and Their Regulatory Status for the Pediatric Population

CBRN threat

Types of medical 

countermeasures 

available in the SNS Regulatory status for the pediatric population

Anthrax

(Bacillus anthracis) 

a

Oral solid antimicrobials Three oral solid antimicrobials are available in the SNS to prevent 

or treat anthrax. One is approved for postexposure prophylaxis and 

treatment of anthrax in all children. Another is approved for 

postexposure prophylaxis for all children, but it is not approved for 

treatment of anthrax in any population. The third is not approved for 

postexposure prophylaxis or treatment of anthrax in any population. 

The antimicrobials that are not approved for postexposure 

prophylaxis or treatment would need to be used under an 

Emergency Use Authorization (EUA) for unapproved age groups, 

including adults, for these indications.

Oral liquid antimicrobials Three oral liquid antimicrobials are available in the SNS to prevent 

or treat anthrax. One is approved for postexposure prophylaxis and 

treatment of anthrax in all children. Another is approved for 

postexposure prophylaxis for all children, but it is not approved for 

treatment of anthrax in any population. The third is not approved for 

postexposure prophylaxis or treatment of anthrax in any population. 

The antimicrobials that are not approved for postexposure 

prophylaxis or treatment would need to be used under an EUA for 

unapproved age groups, including adults, for these indications.

IV antimicrobials Seven IV antimicrobials are available in the SNS to treat anthrax. 

Two have been approved for use by all children for the treatment of

anthrax, and five have not been approved for the treatment of 

anthrax in any population. The antimicrobials that are not approved 

for the treatment of anthrax would need to be used under an EUA 

for all populations for this indication.

Vaccine One anthrax vaccine is available in the SNS, but it has not been 

approved for administration to children. It would need to be 

administered under an Investigational New Drug (IND) protocol.

Antitoxin or immunoglobulin Two antitoxins/immunoglobulins are available in the SNS for the 

treatment of anthrax. One is approved for use by children to treat 

anthrax, while the other is not. The unapproved product would 

need to be used under an EUA in both adults and children.

b

Appendix I: Pediatric CBRN Medical 

Countermeasures in the Strategic National 

Stockpile 
Appendix I: Pediatric CBRN Medical 

Countermeasures in the Strategic National 

Stockpile

Page 31 GAO-13-438 Pediatric Medical Countermeasures

CBRN threat

Types of medical 

countermeasures 

available in the SNS Regulatory status for the pediatric population

Plague

(Yersinia pestis) 

a

Oral solid antimicrobials Two oral solid antimicrobials are available in the SNS for 

postexposure prophylaxis of plague. One is approved for use by 

some children, while the other is not approved for postexposure 

prophylaxis of plague in any population. The first antimicrobial 

would need to be used under an EUA for use by unapproved age 

groups for postexposure prophylaxis of plague, and the second 

antimicrobial would need to be used under an EUA for this 

indication for all populations.

Oral liquid antimicrobials Two oral liquid antimicrobials are available in the SNS for 

postexposure prophylaxis of plague. One is approved for use by 

some children, while the other is not approved for postexposure 

prophylaxis of plague in any population. The first antimicrobial 

would need to be used under an EUA for use by unapproved age 

groups for postexposure prophylaxis of plague, and the second 

antimicrobial would need to be used under an EUA for this 

indication for all populations.

IV antimicrobials Four IV antimicrobials are available in the SNS to treat plague. Two 

are approved for use by some children, and two are not approved 

for the treatment of plague in any population. The antimicrobials 

that are not approved for all children would need to be used under 

EUA for unapproved age groups for this indication, and the 

antimicrobials that are not approved for the treatment of plague 

would need to be used under an EUA for all populations.

Tularemia

(Francisella tularensis) 

Oral solid antimicrobials Two oral solid antimicrobials are available in the SNS for 

postexposure prophylaxis of tularemia. One is approved for use by 

some children for this indication, while the other is not approved for 

postexposure prophylaxis of tularemia in any age population. The

first antimicrobial would need to be used under an EUA for use by

unapproved age groups for postexposure prophylaxis of tularemia, 

and the second antimicrobial would need to be used under an EUA 

for this indication for all populations.

Oral liquid antimicrobials Two oral liquid antimicrobials are available in the SNS for 

postexposure prophylaxis of tularemia. One is approved for use by 

some children for this indication, while the other is not approved for 

postexposure prophylaxis of tularemia in any age population. The 

first antimicrobial would need to be used under an EUA for use by

unapproved age groups for postexposure prophylaxis of tularemia, 

and the second antimicrobial would need to be used under an EUA 

for this indication for all populations.

IV antimicrobials Four IV antimicrobials are available in the SNS to treat tularemia. 

One is approved for use by some children for the treatment of 

tularemia, and the other three are not approved for this indication in 

any age populations. The antimicrobial that is approved for use in 

some children for the treatment of tularemia would need to be used 

under an EUA by unapproved age groups. The other three 

antimicrobials that are not approved would need to be used under 

an EUA for this indication for all populations.
Appendix I: Pediatric CBRN Medical 

Countermeasures in the Strategic National 

Stockpile

Page 32 GAO-13-438 Pediatric Medical Countermeasures

CBRN threat

Types of medical 

countermeasures 

available in the SNS Regulatory status for the pediatric population

Typhus

(Rickettsia prowazekii) 

a

Oral solid antimicrobials One oral solid antimicrobial is available in the SNS to treat typhus, 

and it is approved for use by some children for this indication. This 

antimicrobial would need to be used under an EUA to treat typhus 

in unapproved populations.

Oral liquid antimicrobials One oral liquid antimicrobial is available in the SNS to treat typhus, 

and it is approved for use by some children for this indication. This 

antimicrobial would need to be used under an EUA to treat typhus 

in unapproved populations.

IV antimicrobials One IV antimicrobial is available in the SNS to treat typhus, and it 

is approved for use by some children for this indication. This 

antimicrobial would need to be used under an EUA to treat typhus 

in unapproved populations.

Botulism

(Botulinum toxins)

Antitoxin or immunoglobulin Two antitoxins are available in the SNS to treat botulism, and one 

is approved. The unapproved antitoxin would need to be used 

under an EUA to treat botulism. The approved product can be used 

in all populations, including children, under its license.

Smallpox

(Variola virus)

Vaccine Three smallpox vaccines are available in the SNS. One vaccine is 

approved for use by all children, and the other two are not 

approved for any age populations. These vaccines would need to 

be used under an EUA or an IND protocol. 

Antitoxin or immunoglobulin One immunoglobulin is available in the SNS for treatment of 

adverse reactions to the smallpox vaccine, and it is approved for 

use by all populations.

b

IV antivirals One IV antiviral is available in the SNS to treat adverse reactions to 

the smallpox vaccine, but it has not been approved for this 

indication for any age population. This antiviral would need to be 

used under an IND protocol for this indication.

Radiation

(Radiological/nuclear agents)

Oral chelator One oral chelator is available in the SNS for treatment of internal 

radiation contamination, and it is approved for use by some 

children. This product would need to be used under an EUA for 

unapproved age groups for this indication.

c

IV chelator Two IV chelators are available in the SNS for treatment of internal 

radiation contamination, and both have been approved for use by 

all children.

c

Hematopoietic growth factors One hematopoietic growth factor is available in the SNS, but it has 

not been approved for use by any age population for the treatment 

of acute radiation syndrome. This product would need to be used 

under an EUA or an IND protocol for this indication.

d

Chemical

(Nerve agents)

Nerve agent antidotes Three nerve agent antidotes are available in the SNS for the 

treatment of poisoning by chemical nerve agents. Two of the 

antidotes have been approved for use by all children for this 

indication. The third antidote has an approved indication for use in 

seizures, but it has not been approved for use by all children and 

would need to be used under an EUA or an IND protocol for 

unapproved age groups.

Source: GAO summary of Centers for Disease Control and Prevention (CDC) and Food and Drug Administration (FDA) information.
Appendix I: Pediatric CBRN Medical 

Countermeasures in the Strategic National 

Stockpile

Page 33 GAO-13-438 Pediatric Medical Countermeasures

Notes: According to CDC officials, in general, the same medical countermeasures are used for adults 

and children. The difference may be in weight-based dosing, or the need to use the medical 

countermeasures under a regulatory mechanism such as an EUA or IND. CDC officials stated that 

Burkholderia mallei (glanders), Burkholderia pseudomallei (melioidosis), and multi-drug resistant 

Bacillus anthracis are currently undergoing discussion for further planning. There are no medical 

countermeasures available for viral hemorrhagic fevers—Ebola, Marburg, and Junin viruses. In 

addition to the CBRN medical countermeasures listed, CDC officials told us that the SNS contains 

ventilators in pediatric sizes for all age groups down to 5 kilograms, as well as thermal burn supplies 

such as bandages, burn creams, and anti-nausea medications.

a

FDA determined whether an EUA or IND protocol would be needed; we did not evaluate these 

determinations.

b

Immunoglobulins are derived from blood plasma obtained from donors who have been immunized 

with a vaccine, and they contain antibodies specific to the disease they are being used to treat.

c

Chelators help remove certain radioactive particles from the body.

d

Following radiation exposure, bone marrow can be suppressed. This reduces circulating white blood 

cells and increases the chance of infection. Infection can be prevented or mitigated by certain 

hematopoietic growth factors that activate bone marrow to protect from infection.
Appendix II: Comments from the Department 

of Health and Human Services

Page 34 GAO-13-438 Pediatric Medical Countermeasures

Appendix II: Comments from the Department 

of Health and Human Services
Appendix II: Comments from the Department 

of Health and Human Services

Page 35 GAO-13-438 Pediatric Medical Countermeasures
Appendix II: Comments from the Department 

of Health and Human Services

Page 36 GAO-13-438 Pediatric Medical Countermeasures
Appendix II: Comments from the Department 

of Health and Human Services

Page 37 GAO-13-438 Pediatric Medical Countermeasures
Appendix III: GAO Contact and Staff 

Acknowledgments

Page 38 GAO-13-438 Pediatric Medical Countermeasures

Marcia Crosse, (202) 512-7114 or crossem@gao.gov

In addition to the contact named above, Tom Conahan, 

Assistant Director; Kaitlin Coffey; Kelly DeMots; Carolina Morgan; 

Monica Perez-Nelson; and Roseanne Price made key 

contributions to this report.

Appendix III: GAO Contact and Staff 

Acknowledgments 

GAO Contact

Staff 

Acknowledgments
Related GAO Products

Page 39 GAO-13-438 Pediatric Medical Countermeasures

National Preparedness: Improvements Needed for Measuring Awardee

Performance in Meeting Medical and Public Health Preparedness Goals. 

GAO-13-278. Washington, D.C.: March 22, 2013.

National Preparedness: Countermeasures for Thermal Burns. 

GAO-12-304R. Washington, D.C.: February 22, 2012.

Chemical, Biological, Radiological, and Nuclear Risk Assessments: DHS 

Should Establish More Specific Guidance for Their Use. GAO-12-272. 

Washington, D.C.: January 25, 2012.

National Preparedness: Improvements Needed for Acquiring Medical 

Countermeasures to Threats from Terrorism and Other Sources. 

GAO-12-121. Washington, D.C.: October 26, 2011.

National Preparedness: DHS and HHS Can Further Strengthen 

Coordination for Chemical, Biological, Radiological, and Nuclear Risk 

Assessments. GAO-11-606. Washington, D.C.: June 21, 2011.

Public Health Preparedness: Developing and Acquiring Medical 

Countermeasures Against Chemical, Biological, Radiological, and 

Nuclear Agents. GAO-11-567T. Washington, D.C.: April 13, 2011.

National Security: Key Challenges and Solutions to Strengthen 

Interagency Collaboration. GAO-10-822T. Washington, D.C.: 

June 9, 2010.

Combating Nuclear Terrorism: Actions Needed to Better Prepare to 

Recover from Possible Attacks Using Radiological or Nuclear Materials. 

GAO-10-204. Washington, D.C.: January 29, 2010.

Project BioShield Act: HHS Has Supported Development, Procurement, 

and Emergency Use of Medical Countermeasures to Address Health 

Threats. GAO-09-878R. Washington, D.C.: July 24, 2009.

Project BioShield: HHS Can Improve Agency Internal Controls for Its New 

Contracting Authorities. GAO-09-820. Washington, D.C.: July 21, 2009.

Project BioShield: Actions Needed to Avoid Repeating Past Problems 

with Procuring New Anthrax Vaccine and Managing the Stockpile of 

Licensed Vaccine. GAO-08-88. Washington, D.C.: October 23, 2007.

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