This work has involved the efforts  of various agencies, industry, and subject-matter experts to develop,  acquire, and store medical countermeasures. HHS has  used these material threat determinations to assess the potential public  health and medical consequences of the CBRN agents, and to establish  specific medical requirements for developing countermeasures. More than Half of CBRN Medical Countermeasures Have Been Approved for at Least Some Children

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. PHEMCE officials stated that the remaining 40 percent of the CBRN  countermeasures have not been approved for any pediatric use. 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. 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 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 its  efforts to develop and acquire CBRN medical countermeasures for  children in an emergency. 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. HHS Is Taking Steps to Focus on the Pediatric Population and Develop Pediatric Formulations of Existing Medical Countermeasures

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 and State and Local Governments Address Dispensing of Pediatric Medical Countermeasures in Their Response Plans

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. 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   The response plans for nuclear or radiological incidents also  occur. CDC and FDA Guidance for State and Local Governments Includes Information on Pediatric Dispensing

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. State and Local Government Plans Provide Details about Pediatric Dispensing

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. 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. Agency Comments

We provided a draft of this report to HHS for comment. In its written  comments, reproduced in appendix II, HHS concurred with our findings. 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. Appendix I: Pediatric CBRN Medical Countermeasures in the Strategic National Stockpile

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. GAO-12-121.
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 subject-matter 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 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.
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 HHS and that the department is continuously progressing in this area.