National Preparedness
Improvements Needed for Acquiring Medical Countermeasures to Threats from Terrorism and Other Sources Gao ID: GAO-12-121 October 26, 2011The United States remains vulnerable to terrorist and other threats posed by chemical, biological, radiological, and nuclear (CBRN) agents. Medical countermeasures--drugs, vaccines, and diagnostic devices--can prevent or treat the effects of exposure, but few are currently available. The Department of Health and Human Services (HHS) leads federal efforts to develop and acquire countermeasures, primarily through the Public Health Emergency Medical Countermeasures Enterprise (PHEMCE), an interagency body. This report examines the extent to which HHS (1) based its priorities for developing and acquiring countermeasures on CBRN risk assessments; (2) addressed its own recommendations to improve acquisition and development; and (3) coordinated internally for these efforts. GAO reviewed relevant laws, agency documents, CBRN risk assessments, and reports from outside experts; interviewed HHS and industry officials; and analyzed HHS funding for CBRN countermeasures from fiscal years 2007 through 2010.
Through PHEMCE, HHS laid out its CBRN medical countermeasure development and acquisition priorities in 2007 in a publicly available plan based primarily on two types of CBRN risk assessments--one from the Department of Homeland Security (DHS) and one from HHS--but HHS has not updated the plan as intended. The 2007 plan outlined spending for these priorities through 2013, when special federal funding for countermeasure acquisition will expire. HHS invested about $1.9 billion in development and $2.4 billion for acquisition of countermeasures to fulfill these priorities from fiscal year 2007 to fiscal year 2010. Since 2007, DHS and HHS have continued to assess the risks that CBRN agents pose to national security and public health, and HHS has reassessed decisions on the quantities and types of medical countermeasures needed. However, HHS has not updated its plan, as it had intended to do biennially, to indicate whether any priorities have changed. Further, HHS has not provided specific information on anticipated budget priorities for countermeasure acquisition--information desired by companies to help them decide whether to invest in product development. HHS has begun to address most recommendations from its August 2010 review of PHEMCE and of HHS's countermeasure activities, but HHS has not developed an adequate strategy to monitor implementation. HHS's initiatives to address the recommendations are intended to improve product development and acquisition and PHEMCE's structure and management. These initiatives are led by different agencies and offices--for example, the Food and Drug Administration has begun efforts to improve its regulatory framework, while the National Institutes of Health has begun to implement a program to increase the number of potential products in the pipeline. HHS officials said they have a monitoring strategy that includes quarterly updates of a planning document and quarterly and annual reviews of progress. However, the planning document contains incomplete information and does not allow for measuring progress across all initiatives. Thus, HHS's monitoring strategy is not consistent with federal internal control standards and program management best practices. Given the initiatives' complexity and dispersed HHS leadership responsibilities, an adequate monitoring strategy would help HHS assess overall progress and provide information about whether HHS is meeting its countermeasure development and acquisition objectives. HHS's establishment of PHEMCE in 2006 and its subsequent written agreements have facilitated intradepartmental coordination on the development and acquisition of CBRN medical countermeasures, but some coordination challenges remain. PHEMCE established an intradepartmental coordination process and documented the roles and responsibilities of its partners through written agreements. However, some industry and outside experts have reported that HHS's agencies and offices do not coordinate well to advance products through development to acquisition, which hampers industry's efforts to supply countermeasures. HHS officials are renewing the PHEMCE intradepartmental memorandum of understanding and charter for the governing body. These written agreements, once finalized, should continue to enhance and sustain intradepartmental coordination on countermeasure development and acquisition activities. In addition, effectively implementing some of the initiatives from HHS's August 2010 review may help mitigate these coordination challenges. GAO recommends that HHS update its development and acquisition plan; provide budget priorities for acquisitions; and develop a strategy to monitor implementation of its initiatives. HHS agreed with the first two recommendations. For the third one, HHS said that it had a strategy to track implementation, but in GAO's assessment, the strategy does not meet standards and practices to allow for adequate monitoring.
RecommendationsOur recommendations from this work are listed below with a Contact for more information. Status will change from "In process" to "Open," "Closed - implemented," or "Closed - not implemented" based on our follow up work.
Director: Marcia G. Crosse Team: Government Accountability Office: Health Care Phone: (202) 512-3407GAO-12-121, National Preparedness: Improvements Needed for Acquiring Medical Countermeasures to Threats from Terrorism and Other Sources
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United States Government Accountability Office:
GAO:
Report to the Committee on Homeland Security and Governmental Affairs,
U.S. Senate:
October 2011:
National Preparedness:
Improvements Needed for Acquiring Medical Countermeasures to Threats
from Terrorism and Other Sources:
GAO-12-121:
GAO Highlights:
Highlights of GAO-12-121, a report to the Committee on Homeland
Security and Governmental Affairs, U.S. Senate.
Why GAO Did This Study:
The United States remains vulnerable to terrorist and other threats
posed by chemical, biological, radiological, and nuclear (CBRN) agents.
Medical countermeasures”drugs, vaccines, and diagnostic devices”can
prevent or treat the effects of exposure, but few are currently
available. The Department of Health and Human Services (HHS) leads
federal efforts to develop and acquire countermeasures, primarily
through the Public Health Emergency Medical Countermeasures Enterprise
(PHEMCE), an interagency body. This report examines the extent to which
HHS (1) based its priorities for developing and acquiring
countermeasures on CBRN risk assessments; (2) addressed its own
recommendations to improve acquisition and development; and (3)
coordinated internally for these efforts. GAO reviewed relevant laws,
agency documents, CBRN risk assessments, and reports from outside
experts; interviewed HHS and industry officials; and analyzed HHS
funding for CBRN countermeasures from fiscal years 2007 through 2010.
What GAO Found:
Through PHEMCE, HHS laid out its CBRN medical countermeasure
development and acquisition priorities in 2007 in a publicly available
plan based primarily on two types of CBRN risk assessments”one from the
Department of Homeland Security (DHS) and one from HHS”but HHS has not
updated the plan as intended. The 2007 plan outlined spending for these
priorities through 2013, when special federal funding for
countermeasure acquisition will expire. HHS invested about $1.9 billion
in development and $2.4 billion for acquisition of countermeasures to
fulfill these priorities from fiscal year 2007 to fiscal year 2010.
Since 2007, DHS and HHS have continued to assess the risks that CBRN
agents pose to national security and public health, and HHS has
reassessed decisions on the quantities and types of medical
countermeasures needed. However, HHS has not updated its plan, as it
had intended to do biennially, to indicate whether any priorities have
changed. Further, HHS has not provided specific information on
anticipated budget priorities for countermeasure acquisition”
information desired by companies to help them decide whether to invest
in product development.
HHS has begun to address most recommendations from its August 2010
review of PHEMCE and of HHS‘s countermeasure activities, but HHS has
not developed an adequate strategy to monitor implementation. HHS‘s
initiatives to address the recommendations are intended to improve
product development and acquisition and PHEMCE‘s structure and
management. These initiatives are led by different agencies and offices”
for example, the Food and Drug Administration has begun efforts to
improve its regulatory framework, while the National Institutes of
Health has begun to implement a program to increase the number of
potential products in the pipeline. HHS officials said they have a
monitoring strategy that includes quarterly updates of a planning
document and quarterly and annual reviews of progress. However, the
planning document contains incomplete information and does not allow
for measuring progress across all initiatives. Thus, HHS‘s monitoring
strategy is not consistent with federal internal control standards and
program management best practices. Given the initiatives‘ complexity
and dispersed HHS leadership responsibilities, an adequate monitoring
strategy would help HHS assess overall progress and provide information
about whether HHS is meeting its countermeasure development and
acquisition objectives.
HHS‘s establishment of PHEMCE in 2006 and its subsequent written
agreements have facilitated intradepartmental coordination on the
development and acquisition of CBRN medical countermeasures, but some
coordination challenges remain. PHEMCE established an intradepartmental
coordination process and documented the roles and responsibilities of
its partners through written agreements. However, some industry and
outside experts have reported that HHS‘s agencies and offices do not
coordinate well to advance products through development to acquisition,
which hampers industry‘s efforts to supply countermeasures. HHS
officials are renewing the PHEMCE intradepartmental memorandum of
understanding and charter for the governing body. These written
agreements, once finalized, should continue to enhance and sustain
intradepartmental coordination on countermeasure development and
acquisition activities. In addition, effectively implementing some of
the initiatives from HHS‘s August 2010 review may help mitigate these
coordination challenges.
What GAO Recommends:
GAO recommends that HHS update its development and acquisition plan;
provide budget priorities for acquisitions; and develop a strategy to
monitor implementation of its initiatives. HHS agreed with the first
two recommendations. For the third one, HHS said that it had a strategy
to track implementation, but in GAO‘s assessment, the strategy does not
meet standards and practices to allow for adequate monitoring.
View [hyperlink, http://www.gao.gov/products/GAO-12-121] or key
components. For more information, contact Marcia Crosse at (202) 512-
7114 or crossem@gao.gov.
[End of section]
Contents:
Letter:
Background:
HHS Uses CBRN Risk Assessments in Determining Investment Priorities for
Developing and Acquiring Countermeasures but Has Not Updated These
Priorities since 2007:
HHS Has Begun to Address Most of Its Own Recommendations with
Initiatives Intended to Improve Medical Countermeasure Activities, but
Does Not Have an Adequate Strategy to Monitor Implementation:
HHS's PHEMCE and Related Written Agreements Have Facilitated
Intradepartmental Coordination, but Challenges Remain That May Be
Addressed through HHS's New Initiatives:
Conclusions:
Recommendations for Executive Action:
Agency Comments and Our Evaluation:
Appendix I: HHS Medical Countermeasure Development and Acquisition
Priorities and Investments:
Appendix II: HHS Initiatives to Address Its Recommendations to Improve
Medical Countermeasure Activities:
Appendix III: Comments from the Department of Health and Human
Services:
Appendix IV: GAO Contact and Staff Acknowledgments:
Related GAO Products:
Tables:
Table 1: Recommendations from HHS's August 2010 Review of PHEMCE and
Its Medical Countermeasure Activities:
Table 2: HHS's Medical Countermeasure Priorities and Time Frames by
Fiscal Year, as of April 2007:
Table 3: NIH Investments in CBRN Medical Countermeasure Early Research
and Development, Fiscal Year 2007 through Fiscal Year 2010:
Table 4: BARDA Investments in CBRN Medical Countermeasure Advanced
Research and Development, Fiscal Year 2007 through Fiscal Year 2010:
Table 5: HHS Investments in CBRN Medical Countermeasures Using the
Project BioShield Special Reserve Fund, Fiscal Year 2007 through Fiscal
Year 2010:
Table 6: Recommendations from HHS's August 2010 Review of PHEMCE and
Its Medical Countermeasure Activities and HHS's Initiatives in
Response:
Figure:
Figure 1: Processes for Medical Countermeasure Development and
Acquisition:
Abbreviations:
ASPR: Office of the Assistant Secretary for Preparedness and Response:
BARDA: Biomedical Advanced Research and Development Authority:
CBRN: chemical, biological, radiological, and nuclear:
CDC: Centers for Disease Control and Prevention:
DHS: Department of Homeland Security:
DOD: Department of Defense:
ESC: Enterprise Senior Council:
FDA: Food and Drug Administration:
HHS: Department of Health and Human Services:
MOU: memorandum of understanding:
MTA: material threat assessment:
NBSB: National Biodefense Science Board:
NIH: National Institutes of Health:
PHEMCE: Public Health Emergency Medical Countermeasures
Enterprise:
SNS: Strategic National Stockpile:
TRA: terrorism risk assessment:
[End of section]
October 26, 2011:
The Honorable Joseph I. Lieberman:
Chairman:
The Honorable Susan M. Collins:
Ranking Member:
Committee on Homeland Security and Governmental Affairs:
United States Senate:
The anthrax attacks of 2001 raised concerns about the United States'
vulnerability to intentional terrorist threats from chemical,
biological, radiological, and nuclear (CBRN) agents, and the 2007
National Strategy for Homeland Security stated that terrorists have
declared their intention to acquire and use CBRN agents as weapons to
inflict catastrophic attacks against the United States.[Footnote 1]
More recently, the May 2010 National Security Strategy noted that the
American people face no greater or more urgent danger than a terrorist
attack with a nuclear weapon, and the effective dissemination of a
lethal biological agent within a U.S. city would endanger the lives of
hundreds of thousands of people and have unprecedented economic,
societal, and political consequences.[Footnote 2] In addition, the
recent earthquake and resulting tsunami in Japan that caused a nuclear
reactor to release radioactive material highlighted a population's
vulnerability to unintentional CBRN exposure.
Rapid diagnosis, treatment, and prevention may minimize the public
health impact of a release of a CBRN agent. Congress appropriated a
total of about $5.6 billion to be available for obligation from fiscal
year 2004 through fiscal year 2013 for the Project BioShield Special
Reserve Fund to acquire certain CBRN medical countermeasures, such as
drugs, vaccines, and devices to diagnose, treat, prevent, or mitigate
potential effects of exposure to these agents.[Footnote 3] However,
there are currently few available medical countermeasures. Research and
development to create useable countermeasures is a lengthy, complex,
and expensive process that involves public and private investment.
However, the general lack of a commercial market for medical
countermeasures against these agents may reduce incentives for
industry--pharmaceutical and medical device manufacturers--to invest
millions of dollars to develop countermeasures instead of other
products that may be more lucrative.
The Department of Health and Human Services (HHS) is the federal agency
primarily responsible for identifying needed medical countermeasures to
prevent or mitigate potential health effects from exposure to CBRN
agents and engaging with industry to develop them. In addition, because
CBRN agents differ in their potential to cause widespread illness and
death, the Department of Homeland Security (DHS) and HHS assess the
risks and potential public health consequences of attacks with CBRN
agents to identify those agents that represent the highest risk and to
help guide response planning and countermeasure development. In 2006,
HHS established the Public Health Emergency Medical Countermeasures
Enterprise (PHEMCE), a federal interagency body that includes various
HHS agencies and offices, DHS, the Department of Defense (DOD), and
others and is responsible for providing recommendations to the
Secretary of HHS on medical countermeasure priorities and development
and acquisition activities.[Footnote 4] However, members of Congress
and several expert organizations have raised concerns about whether HHS
and its agencies and offices are successfully developing and acquiring
medical countermeasures to respond to CBRN incidents.[Footnote 5] In
December 2009, the HHS Secretary called for a comprehensive review of
HHS's medical countermeasure development and acquisition activities. As
a result, HHS issued an August 2010 review with several recommendations
intended to improve its CBRN medical countermeasure development and
acquisition efforts.[Footnote 6]
You asked us to examine the extent to which HHS has developed risk-
informed investment priorities and strategies for developing CBRN
medical countermeasures. Our current review addresses (1) the extent to
which HHS has based its priorities for and the resulting investments in
medical countermeasure development and acquisition on CBRN risk
assessments, (2) the extent to which HHS has addressed its own
recommendations to improve its CBRN medical countermeasure development
and acquisition activities, and (3) the extent to which HHS's agencies
and offices have coordinated with each other to develop and acquire
CBRN medical countermeasures.
To determine the extent to which HHS has based its medical
countermeasure development and acquisition priorities and investments
on CBRN risk assessments, we analyzed relevant laws and presidential
directives to determine requirements for HHS to use CBRN risk
assessments for medical countermeasure decision making.[Footnote 7] We
analyzed DHS and HHS CBRN risk assessments and other HHS documents on
stated countermeasure development and acquisition priorities developed
since 2004 to determine whether the stated priorities align with the
highest-risk agents identified in the risk assessments. We also
interviewed officials from HHS agencies and offices--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), the Centers for Disease Control
and Prevention (CDC), and the Food and Drug Administration (FDA)--to
obtain information on how HHS officials determined priorities for
countermeasures, and whether and how HHS officials used CBRN risk
assessments to do so. In addition, to determine how much funding HHS
has invested in CBRN medical countermeasure development and acquisition
from fiscal year 2007 through fiscal year 2010, we analyzed agency data
and reports on BARDA, CDC, and NIH investments, compared these
investments with HHS's stated countermeasure priorities and with its
acquisitions, and interviewed HHS officials.[Footnote 8] We chose this
period because it coincided with the beginning of the period covered by
HHS's plan for developing and acquiring CBRN medical countermeasures.
The overall estimates of annual NIH investments for CBRN medical
countermeasure research and development that we are reporting are based
on information provided by NIH. NIH investments may have multiple
components, only some of which may be related to CBRN medical
countermeasure development. NIH provided us with estimates of the
amounts related to specific types of CBRN research, based on
percentages of research awards. We cross-checked publicly available
information on funding for medical countermeasure development and
acquisition with the funding information provided by HHS and
interviewed agency officials about how they ensure the accuracy of the
funding information provided, but did not independently verify the
funding information provided by HHS. Through these steps, we determined
that the data we received from HHS were sufficiently reliable for our
purposes.
To determine the extent to which HHS has addressed its own
recommendations to improve its CBRN medical countermeasure development
and acquisition activities, we analyzed HHS's August 2010 review of
these activities to identify recommendations the department made that
were specific to CBRN countermeasure research, development, and
acquisition.[Footnote 9] We reviewed other HHS documentation, such as
the department's fiscal year 2012 budget requests, and interviewed HHS
officials to determine how HHS planned to implement initiatives to
address the recommendations, including which agencies and offices are
responsible for specific initiatives, time frames for and status of
implementation, and any barriers to implementation. In order to assess
HHS's overall plans for implementing initiatives to address the
recommendations, we compared HHS's plans to federal standards for
internal control and the Project Management Institute's The Standard
for Program Management for program management best practices.[Footnote
10]
To determine the extent to which HHS's agencies and offices have
coordinated to develop and acquire CBRN medical countermeasures, we
reviewed HHS policies and procedures, including memorandums of
understanding, charters, and other documents, and expert assessments of
HHS's CBRN medical countermeasure development and acquisition
activities and interviewed HHS officials. We also conducted interviews
with six industry officials from five companies who were knowledgeable
about HHS's medical countermeasure activities. We selected these
officials based on their experience working with HHS on advanced
countermeasure development or acquisition, from companies that varied
in size and products offered. Through these steps we identified
guidance for intradepartmental coordination, used them to assess the
extent to which HHS agencies coordinate to develop and acquire CBRN
medical countermeasures and to understand any barriers to this
coordination, and compared HHS's coordination practices with our best
practices for enhancing and sustaining agency collaboration.[Footnote
11]
We conducted this performance audit from February 2011 through October
2011 in accordance with generally accepted government auditing
standards. Those standards require that we plan and perform the audit
to obtain sufficient, appropriate evidence to provide a reasonable
basis for our findings and conclusions based on our audit objectives.
We believe that the evidence obtained provides a reasonable basis for
our findings and conclusions based on our audit objectives.
Background:
HHS is responsible for identifying needed medical countermeasures to
prevent or mitigate the potential health effects from exposure to CBRN
agents and researching, developing, and acquiring these
countermeasures. The Project BioShield Act of 2004 authorized the
appropriation of a total of about $5.6 billion from fiscal years 2004
through 2013.[Footnote 12] The act facilitated the creation of a
government market by authorizing the government to commit to make the
Special Reserve Fund available to purchase certain medical
countermeasures,[Footnote 13] including those countermeasures that may
not yet be FDA-approved or licensed.[Footnote 14] The act also allowed
the HHS Secretary to authorize, under specified conditions, the
temporary emergency use of products that have not yet received FDA
approval.[Footnote 15]
In 2006, HHS established PHEMCE, a federal interagency body responsible
for providing recommendations to the Secretary of HHS on medical
countermeasure priorities, development and acquisition activities, and
strategies for distributing and using medical countermeasures held in
the U.S. Strategic National Stockpile (SNS), the national repository of
medications, medical supplies, and equipment for use in a public health
emergency.[Footnote 16] The PHEMCE working groups and senior council
serve as the primary means of communication between HHS and
participating federal departments on CBRN medical countermeasure
issues. As required by the Pandemic and All-Hazards Preparedness Act,
PHEMCE also conducts annual reviews of the contents of the
SNS.[Footnote 17]
Roles and Responsibilities of HHS's Agencies and Offices:
Within HHS, several agencies and offices have specific responsibilities
for CBRN medical countermeasure development and acquisition. ASPR leads
PHEMCE and the medical and public health response to potential CBRN
incidents, including strategic planning, medical countermeasure
prioritization, and support for developing and acquiring medical
countermeasures. Within ASPR, BARDA--established by the Pandemic and
All-Hazards Preparedness Act of 2006--oversees advanced development and
acquisition of some CBRN medical countermeasures into the SNS.[Footnote
18] The National Biodefense Science Board (NBSB), established by the
Pandemic and All-Hazards Preparedness Act, is an advisory committee
composed of 13 voting members with expertise in science, medicine, and
public health that provides the HHS Secretary with expert advice and
guidance on scientific and technical matters related to current and
future CBRN agents, including those that occur naturally.[Footnote 19]
NIH conducts and funds basic and applied research to develop new or
enhanced medical countermeasures and related medical tools to protect
the nation against threats posed by CBRN agents. CDC maintains the SNS
and supports state and local public health departments' efforts to
detect and respond to public health emergencies, including providing
guidance and recommendations for the mass distribution and use of
medical countermeasures. FDA assesses the safety and effectiveness of
CBRN medical countermeasures and regulates their development, approval
or licensure, and postmarket surveillance. FDA also provides technical
support for the creation of tools to support medical countermeasure
development and may authorize the emergency use of medical products
that have not yet been approved or licensed or were approved or
licensed only for other uses.
DHS and HHS CBRN Risk Assessments:
As part of its preparedness role to plan and coordinate the federal
interagency response to catastrophic CBRN incidents, DHS develops two
types of CBRN risk assessments--terrorism risk assessments (TRA) and
material threat assessments (MTA)--with some input from HHS. The
requirements for these departments to develop such risk assessments are
in provisions in the Project BioShield Act of 2004 and Homeland
Security Presidential Directives 10 (Biodefense for the 21st Century),
18 (Medical Countermeasures Against Weapons of Mass Destruction), and
22 (Domestic Chemical Defense). TRAs assess the relative risks posed by
multiple CBRN agents based on variable threats, vulnerabilities, and
consequences, and DHS updates TRAs regularly.[Footnote 20] MTAs assess
the threat posed by given CBRN agents or classes of agents and the
potential number of human exposures in plausible, high-consequence
scenarios. DHS uses the MTAs to determine which CBRN agents pose a
material threat sufficient to affect national security.[Footnote 21]
The Project BioShield Act of 2004 calls for HHS to assess the public
health consequences of exposure to those CBRN agents that DHS
determines are material threats to the nation and to determine for
which of these agents medical countermeasures are necessary to protect
the public's health.[Footnote 22] HHS's public health consequence
modeling reports use the exposure information from DHS's MTAs to
calculate the number of individuals who may become ill, be
hospitalized, or die based on the MTA scenario. These modeling reports
represent an interim step in determining needed countermeasures. HHS
uses the modeling reports as part of an assessment process to establish
requirements for medical countermeasures that need to be developed and
acquired to respond to a CBRN incident.
Medical Countermeasure Research, Development, Acquisition, and Support:
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 high-risk 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.[Footnote 23] NIH typically provides federal funding for
basic and applied research and early development.[Footnote 24] BARDA
typically funds advanced development of medical
countermeasures.[Footnote 25] If a countermeasure is not FDA-approved
or licensed, its acquisition into the SNS is typically funded by the
Project BioShield Special Reserve Fund.[Footnote 26] If a
countermeasure is FDA-approved or licensed, CDC purchases the
countermeasure for the SNS.
Figure 1: Processes for Medical Countermeasure Development and
Acquisition:
[Refer to PDF for image]
Processes for countermeasure priority-setting: DHS: Identify threats;
Processes for countermeasure priority-setting: PHEMCE: Assess medical
consequences;
Processes for countermeasure priority-setting: PHEMCE: Establish
counter-measure requirements;
Processes for countermeasure priority-setting: PHEMCE: Prioritize
development and acquisition.
Processes for countermeasure development and acquisition: PHEMCE:
Determine if existing products can serve as countermeasures;
Processes for countermeasure development and acquisition: NIH: Support
basic research;
Processes for countermeasure development and acquisition: NIH: Support
applied research;
Processes for countermeasure development and acquisition: NIH: Support
early development;
Processes for countermeasure development and acquisition: BARDA:
Support Advances development.
(Acquisition & licensure PHEMCE, BARDA, FDA[A])
Source: GAO analysis of HHS information.
[A] In addition to approving or licensing medical countermeasures, FDA
works with researchers throughout the development stages to review
safety and effectiveness test results and provide technical assistance
to help ensure that research meets FDA's regulatory requirements.
[End of figure]
HHS Uses CBRN Risk Assessments in Determining Investment Priorities for
Developing and Acquiring Countermeasures but Has Not Updated These
Priorities since 2007:
HHS based its medical countermeasure development and acquisition
priorities and investments primarily on two types of CBRN risk
assessments, but the department has not updated its plan outlining
these priorities since 2007. Through PHEMCE, HHS laid out its
countermeasure priorities in a publicly available plan in 2007. The
priorities included countermeasures for every agent that DHS considered
a material threat to national security. From fiscal year 2007 through
fiscal year 2010, HHS invested about $4.3 billion in countermeasure
development and acquisition--$1.9 billion in research and development
and $2.4 billion for acquisition of countermeasures to fulfill these
priorities. Since 2007, DHS and HHS have continued to assess the risks
that CBRN agents pose to national security and public health, and HHS
has reassessed the quantities and types of medical countermeasures
needed. However, HHS has not used this information to update the
countermeasure priorities established in the 2007 PHEMCE plan and
communicate them to pharmaceutical and medical device manufacturers and
private partners.
HHS Uses Two Types of CBRN Risk Assessments as a Basis for Its Medical
Countermeasure Priorities and Has Invested $4.3 Billion in Their
Development and Acquisition:
HHS based its priorities for CBRN medical countermeasure development
and acquisition investments primarily on two types of risk assessments,
DHS's MTAs and HHS's public health consequence modeling reports. DHS
used the MTAs to determine which agents pose material threats to
national security. Using information from DHS's MTAs, HHS assessed the
public health consequences of attacks with those agents by modeling the
health effects from exposure to the agents according to the attack
scenario in the MTAs. Based on the provisions of the Project BioShield
Act of 2004, HHS determined whether medical countermeasures were needed
for the agents DHS determined to pose material threats. HHS officials
told us they also consulted with experts and reviewed public health
literature to determine needed quantities, types, and desired
characteristics of countermeasures. HHS has generally not used DHS's
TRAs to help determine which countermeasures to develop and acquire.
HHS officials told us that the TRAs do not provide enough specific
information, such as estimates of the number of people potentially
exposed to particular agents, to be useful in assessing public health
consequences and determining requirements for the needed types and
quantities of medical countermeasures.[Footnote 27]
In 2007, HHS released the PHEMCE implementation plan, a public document
containing its CBRN medical countermeasure development and acquisition
priorities. In the 2007 plan, HHS assigned priority to developing and
acquiring particular countermeasures for all CBRN agents that DHS
deemed material threats up to that time and for an additional chemical
agent, based on DHS's MTAs and HHS's public health consequence modeling
reports. (See app. I, table 2, for HHS's countermeasure priorities and
time frames from the 2007 plan.) For example, HHS priorities include
the need to develop and acquire diagnostic tests for several agents,
such as anthrax, as a midterm priority.
From fiscal year 2007 through fiscal year 2010, HHS invested about $4.3
billion in CBRN medical countermeasure research, development, and
acquisition for those countermeasures that HHS determined to be
priorities based on the MTAs and the modeling reports.[Footnote 28] Of
this $4.3 billion, HHS invested $1.9 billion in countermeasure research
and development for those countermeasures that were not immediately
available for acquisition. Of this $1.9 billion, NIH invested $1.2
billion in early research and early development,[Footnote 29] and BARDA
invested $681 million in advanced development for HHS priorities. (See
app. I, tables 3 and 4, for NIH and BARDA investments, respectively.)
For example, from fiscal year 2007 through fiscal year 2010, NIH
invested $295 million for diagnostics and countermeasures for smallpox,
$187 million for diagnostics and countermeasures for exposure to
radiological and nuclear agents, $197 million for countermeasures for
chemical agents,[Footnote 30] and almost $10 million for early research
and development of diagnostics for glanders and melioidosis
(Burkholderia).[Footnote 31] From fiscal years 2007 through 2010, BARDA
invested $244 million for advanced development of anthrax
vaccine[Footnote 32] and $146 million for advanced development of
countermeasures for radiological and nuclear agents, such as
antioxidants and chelating agents to remove radioactive material from
the body. BARDA also invested $53 million from fiscal years 2007
through 2009 in advanced development for smallpox countermeasures, of
which $16 million was for advanced development of smallpox antivirals,
which HHS subsequently acquired for the SNS in fiscal year
2011.[Footnote 33]
From fiscal year 2007 through fiscal year 2010, HHS invested the rest
of the $4.3 billion--approximately $2.4 billion--to acquire available
countermeasures, all of which it had identified as priorities based on
the MTAs and the modeling reports.[Footnote 34] For example, HHS
invested $1.1 billion of the Project BioShield Special Reserve Fund
from fiscal years 2007 through 2010 to acquire anthrax vaccine, anthrax
antitoxin, and smallpox vaccine for the SNS.[Footnote 35] (See app. I,
table 5, for HHS Project BioShield Special Reserve Fund investments.)
CDC also spent $1.3 billion from fiscal year 2007 through fiscal year
2010 to maintain the quantities of CBRN countermeasures held in the
SNS. However, many of the countermeasures that HHS and PHEMCE
determined to be priorities are not available. For example, no FDA-
approved, rapid, point-of-care diagnostics exist for any of the
biological agents deemed material threats.[Footnote 36]
HHS Has Not Updated Its CBRN Medical Countermeasure Priorities in a
Publicly Available Plan since 2007 to Make Its Current Priorities Clear
to Industry:
HHS has not updated the PHEMCE implementation plan since 2007 to
publicly present its current priorities for CBRN medical countermeasure
research, development, and acquisition. HHS stated in its 2007 PHEMCE
plan that it would update the plan in 2009 and biennially thereafter to
reflect any changes in threats posed by specific CBRN agents and the
availability of new countermeasures. However, HHS officials told us the
department's efforts to review and update the PHEMCE plan in 2009 were
suspended until HHS finished an overall examination of PHEMCE in the
summer of 2010. This review was completed and published in August 2010,
but HHS officials told us in June 2011 that PHEMCE had not yet made key
decisions on what would be included in an updated plan. In August 2011,
HHS officials told us the department was in the process of establishing
a steering committee, composed of PHEMCE interagency participants,
which is tasked with updating the PHEMCE strategy and implementation
plan. According to HHS officials, the department plans to finalize and
publicly release the updated PHEMCE plan in spring 2012. In addition,
HHS officials said that the next version of the plan would likely be an
all-hazards plan that includes influenza and emerging infectious
diseases.
The 2007 plan called for biennial review and revision of PHEMCE's
countermeasure priorities to incorporate updated information from DHS's
risk assessments. For example, HHS's countermeasure priorities in the
anticipated 2009 plan were to be further informed by DHS's 2008
integrated CBRN TRA, which provided assessments of which CBRN agents
presented the highest risk, based on DHS's individual 2006 biological
and chemical TRAs. The 2007 plan also stated that future versions would
incorporate more detailed assessments of needed countermeasures for
enhanced, emerging, or advanced biological agents to improve
preparedness against changing CBRN threats.[Footnote 37] When HHS
publicly released the PHEMCE implementation plan containing its medical
countermeasure development and acquisition priorities in 2007, the
department focused on acquisition of CBRN countermeasures using the
remainder of the Project BioShield Special Reserve Fund. The plan
established near-term (fiscal year 2007 through fiscal year 2008),
midterm (fiscal year 2009 through fiscal year 2013), and long-term
(beyond fiscal year 2013) acquisition periods to correspond with the
appropriation of the funds.[Footnote 38] Planned acquisition was based
on the status of countermeasure research and development at the time.
For example, the plan states that HHS's planned acquisitions for the
near term were based on the availability of candidate countermeasures
already in advanced development and nearing readiness for acquisition
and on the ability of antibiotics already in the SNS to be used to
treat the health effects of more than one biological agent.
HHS indicated in the 2007 PHEMCE plan that its updated plan would
reflect its considerations of how to fund medical countermeasure
development and acquisition after the funding in the Special Reserve
Fund is no longer available for obligation after fiscal year 2013. The
Special Reserve Fund has also been used to provide much of BARDA's
advanced research and development funding, rather than annual
appropriations.[Footnote 39] HHS has indicated that it will begin a 5-
year budget planning process, which could help the department consider
options for funding medical countermeasure development and acquisition
going forward. However, HHS has not begun to identify future funding
needs for countermeasure advanced research, development, and
acquisition or considered specific funding levels needed for acquiring
particular countermeasures. HHS officials said that while Congress has
discussed viable ways to fund countermeasure advanced research,
development, and acquisition, it was premature to anticipate
congressional action on the Special Reserve Fund.[Footnote 40]
Although HHS has not updated the 2007 plan, DHS and HHS have continued
to update their assessments of the risks that CBRN agents pose to
national security and public health since the plan was released, and
HHS has periodically evaluated its progress in acquiring
countermeasures. DHS has conducted biennial TRAs on classes of CBRN
agents and more MTAs to determine whether additional CBRN agents pose
material threats.[Footnote 41] HHS has modeled the public health and
medical consequences of some of these additional agents and established
requirements for desired countermeasures for these agents, as well as
more recent requirements for priority countermeasures. In addition,
PHEMCE's annual reviews of the contents of the SNS have evaluated HHS's
progress in acquiring countermeasures. PHEMCE conducts these annual SNS
reviews to determine HHS's progress in acquiring needed countermeasures
and remaining gaps, such as how many more doses of anthrax vaccine HHS
may need to acquire to reach its desired goal.[Footnote 42] HHS has
also conducted reviews of its medical countermeasure portfolios to
identify gaps and the need for designation of additional countermeasure
investment priorities. The results of the risk assessments, the PHEMCE
SNS annual reviews, and the portfolio reviews could lead HHS to revise
its priorities, but these reviews are not publicly available because
they are sensitive. Further, these reviews do not serve the same
purpose that a review and update of the PHEMCE plan would serve because
they do not fully outline HHS's intentions to develop and acquire
countermeasures for additional or different CBRN agents. Unlike an
updated PHEMCE plan, researchers and industry partners cannot use them
to determine whether they have a viable concept or candidate product
that might help fulfill HHS's countermeasure needs.
Industry officials and expert groups expressed concern about the lack
of specificity in the 2007 PHEMCE plan. Specifically, their concerns
centered on the lack of specific information on requirements for HHS's
priority countermeasures and anticipated spending to support
countermeasure development. The Institute of Medicine, in its 2010
workshop summary on countermeasure development, reported that
pharmaceutical and medical device manufacturing companies need
additional information on how much of a countermeasure HHS wants to
acquire in order to develop companies' business plans.[Footnote 43]
Industry officials also told us that they would like earlier and more
specific information on requirements for desired countermeasures to
guide their decisions about what products to develop. For example, one
industry representative told us that pharmaceutical and medical device
manufacturers do not know how many doses of a product HHS will buy, how
much the company should spend on product development, and how to scale
production. HHS makes information available on the desired
countermeasure quantity, type, and storage requirements in requests for
proposals, but some industry officials told us that it would be useful
to have that information earlier.[Footnote 44] The Institute of
Medicine report also stated that the acquisition spending levels in the
2007 plan arbitrarily define the market, with the result that some
companies will not invest in countermeasures predicted to gross less
than $100 million. Some experts have estimated the average cost of
developing one countermeasure to be from $800 million to over $1
billion. Large companies are not likely to invest in developing a
countermeasure if the perceived market is small. In addition, the
Institute of Medicine report states that HHS must address long-term
financing for medical countermeasures, including budgeting costs to
hold or replenish these countermeasures in the SNS, to make a more
rational business case for industry to invest.
Industry officials and expert groups also expressed concern about the
lack of ranking within HHS's priority CBRN countermeasures. Some
industry officials told us that without any further ranking, they lack
assurance that their products would fulfill HHS's needs or that HHS
would be committed to buying such products if their companies invested
in development. Expert groups and an HHS advisory board have also
reported that the lack of ranking is problematic and have made
suggestions to improve the 2007 PHEMCE plan by ranking the
countermeasure priorities. For example, in its 2010 report on HHS's
countermeasure activities, the NBSB pointed out that HHS's
countermeasure needs require further prioritization because the CBRN
threats and their countermeasures are numerous.[Footnote 45] As such,
the NBSB recommended that HHS identify at least three high-priority,
new CBRN countermeasures to develop and targeted timelines for
development.[Footnote 46] Similarly, the Institute of Medicine, in its
2010 report, stated that the PHEMCE plan provides limited guidance to
industry and is simply a list of countermeasures that HHS hopes to
acquire.[Footnote 47] The report suggested revising the plan to provide
more specific guidance.
HHS officials told us that the department has signaled to industry its
focus on specific countermeasures for certain agents from among the
larger group of priorities outlined in the 2007 plan and continues to
discuss these more specific priorities with industry. As a result, HHS
officials did not agree that the department needed to implement the
NBSB recommendation. For example, HHS officials said that when the
department began pursuing development and acquisition of
countermeasures to fulfill the 2007 priorities, it focused first on
acquiring anthrax vaccine. Officials told us that once HHS acquired
that vaccine for the SNS, they then focused on acquiring smallpox
vaccine, and once that had been acquired, smallpox antivirals. HHS
officials said they discuss HHS's countermeasure priorities during the
department's annual meetings with PHEMCE stakeholders in Washington,
D.C., and the department has begun holding similar meetings more
recently with stakeholders in other regions of the United States.
Further, HHS officials told us that the department's countermeasure
priorities are very clearly articulated by the requests for proposals
and other notices the department issues.
The lack of an updated plan leaves HHS without assurance that its most
current needs for countermeasures to address the greatest national
security and public health risks have been clearly and transparently
communicated to researchers and industry partners so that the most
needed countermeasures are being developed. In addition, not
communicating the department's countermeasure needs in an updated plan
would prolong industry's concerns about transparency and the need to
have information earlier in order to develop business plans. Not
providing estimates of anticipated budget priorities for developing and
acquiring specific countermeasures could preclude HHS and industry from
suitably targeting long-term research and development to fulfill
specific countermeasure priorities, especially in tighter budget
climates. The lack of specificity on spending levels could also provide
further disincentives to industry to develop CBRN countermeasures in
the face of developing other drugs with commercial markets that would
provide a return on investment.
HHS Has Begun to Address Most of Its Own Recommendations with
Initiatives Intended to Improve Medical Countermeasure Activities, but
Does Not Have an Adequate Strategy to Monitor Implementation:
HHS has begun to address most recommendations from its August 2010
review of its medical countermeasure activities with specific
initiatives intended to improve the product development and acquisition
process and PHEMCE's structure. ASPR has gathered information from HHS
agencies and offices on the early stages of implementation of the
initiatives that they are leading. However, the department has not
developed an adequate strategy to monitor its overall implementation of
the initiatives, many of which are complex in nature.
HHS Agencies and Offices Have Begun to Address Most of HHS's
Recommendations with Initiatives Intended to Improve PHEMCE and Its
Medical Countermeasure Activities:
In HHS's August 2010 review of its medical countermeasure activities,
HHS made 13 recommendations designed to improve its ability to develop
and acquire medical countermeasures for novel or unknown threats, as
well as countermeasures for known threats.[Footnote 48] HHS's review
noted that the department has continued to face challenges associated
with the slow rate of progress and cost of medical countermeasure
research and development. As shown in table 1, four of HHS's 13
recommendations focused on efforts to improve the infrastructure to
support product development and acquisition, such as advanced
development activities for, and regulatory oversight of, products,
including those that can address multiple threats. The remaining nine
recommendations focused on enhancing PHEMCE's structure and management,
such as its decision-making processes, in order to provide
pharmaceutical and medical device manufacturers with more clarity and
predictability. (See app. II, table 6, for a more detailed description
of the recommendations from HHS's August 2010 review and information on
how HHS agencies and offices are addressing them.)
Table 1: Recommendations from HHS's August 2010 Review of PHEMCE and
Its Medical Countermeasure Activities:
Infrastructure to support product development and acquisition;
1: Enable innovative regulatory science and oversight.
Infrastructure to support product development and acquisition;
2: Expand pipeline of potential products.
Infrastructure to support product development and acquisition;
3: Foster partnerships to support flexible manufacturing and advanced
development activities.
Infrastructure to support product development and acquisition;
4: Infrastructure to support product development and acquisition.
Enhancements to PHEMCE‘s structure and management;
5: Establish a medical countermeasure development leader.
Enhancements to PHEMCE‘s structure and management;
6: Establish better coordination within HHS.
Enhancements to PHEMCE‘s structure and management;
7: Coordinate and collaborate with federal government partners.
Enhancements to PHEMCE‘s structure and management;
8: Use a systematic approach to decision making.
Enhancements to PHEMCE‘s structure and management;
9: Improve contracting and communication.
Enhancements to PHEMCE‘s structure and management;
10: Improve management of product development.
Enhancements to PHEMCE‘s structure and management;
11: Reexamine the statutory framework for how liability protection is
provided.
Enhancements to PHEMCE‘s structure and management;
12: Update the requirements for current and future products.
Enhancements to PHEMCE‘s structure and management;
13: Develop multiyear budget planning process.
Source: GAO summary of HHS's Public Health Emergency Medical
Countermeasures Enterprise Review.
[End of table]
HHS agencies and offices--primarily FDA, NIH, and BARDA--have begun to
address the four recommendations to improve the infrastructure to
support product development and acquisition. Specifically, HHS agencies
and offices have developed initiatives intended to collectively address
regulatory, technical, and business aspects of the development and
acquisition process that can pose obstacles for industry and affect its
ability to develop CBRN countermeasures for acquisition into the SNS.
To reduce regulatory obstacles that can prevent the successful
development and approval of these countermeasures, FDA has begun
efforts to improve aspects of its regulatory review process; the state
of regulatory science; and the legal, regulatory, and policy framework
for public health response. According to some industry officials, these
obstacles have included insufficient interaction with FDA officials and
a lack of clarity in the regulatory process, especially when
researchers are working to prove a countermeasure's effectiveness using
animals as proxies for humans, as humans generally cannot ethically be
used in studies involving CBRN agents.[Footnote 49] Among other things,
FDA's Medical Countermeasure Initiative is intended to provide industry
with greater clarity on aspects of the regulatory framework and more
productive interactions with FDA regulators. For example, FDA is in the
process of establishing action teams to identify resources and subject-
matter experts to help identify and address scientific, regulatory, and
policy issues earlier in the development process and thereby facilitate
an improved regulatory review process. As of August 2011, FDA officials
told us they have established three action teams, including one focused
on laboratory diagnostic tests.
To reduce technical obstacles affecting the number of potential
products in the research and development pipeline, NIH has begun to
implement the Concept Acceleration Program. This program would
proactively seek out and provide additional resources to investigators
who are conducting promising basic research with the potential to
fulfill a particular CBRN medical countermeasure need. NIH officials
told us they have begun hiring staff for this program and hope to
complete hiring by the end of 2011.
To reduce technical and business obstacles that can affect the
probability of successfully developing technologies and
countermeasures, BARDA has begun to address two of the recommendations
intended to provide expertise and resources to pharmaceutical and
medical device manufacturers with limited experience or knowledge of
the technical and business aspects of product development. First, to
assist small companies that have limited technical experience with
advanced development and flexible manufacturing services, BARDA has
begun to implement its plan to open Centers for Innovation in Advanced
Development and Manufacturing. The centers are intended to provide
industry with technical expertise and services in scale-up
manufacturing, regulatory affairs, and other aspects of advanced
development and manufacturing. BARDA issued a request for proposals for
these centers in March 2011 with the expectation that the first centers
would open in 2014. Second, to assist small companies with limited
experience in a range of business and financing issues, BARDA has
developed plans for a Medical Countermeasure Strategic Investor. This
independent investment firm would identify companies with technologies
that are both likely to result in a commercially attractive product and
capable of producing medical countermeasures that satisfy government-
specific needs. The investment firm would assist these companies with
capital and business expertise as they navigate the countermeasure
development process and become eligible for advanced research and
development funding. HHS has sought statutory authority to create the
investment firm and has developed various options for how to structure
it.
HHS agencies and offices--especially ASPR--have begun steps intended to
address many of the nine recommendations to enhance PHEMCE's structure
and management but have not taken steps to address all of them. (See
app. II, table 6, for more information on how HHS agencies and offices
are addressing each of the recommendations from HHS's August 2010
review of PHEMCE and its medical countermeasure activities.) For
example, HHS has taken the following actions:
* In response to two recommendations in the August 2010 review--to
improve coordination within HHS and with federal government partners--
ASPR created the Enterprise Senior Council (ESC) as the decision forum
for PHEMCE. According to HHS officials, the ESC, unlike its
predecessor, includes voting members representing each federal
department that participates in PHEMCE.[Footnote 50] HHS officials also
said that some officials who have participated in the ESC have been at
more senior levels within their departments than the officials who
served on the ESC's predecessor.
* To improve management of countermeasure development--as recommended
in HHS's August 2010 review--HHS agencies and offices, through ASPR,
have jointly conducted portfolio reviews of all of HHS's investments in
countermeasures to address a given agent (including anthrax, smallpox,
and radiological and nuclear agents). HHS officials told us that during
these reviews, each agency presented its perspective on the critical
issues in developing countermeasures for that agent. Officials said
that these reviews have allowed senior leaders from across HHS to
better understand the range of available countermeasures for each
threat and highlight any gaps or issues in countermeasure development
or response capabilities. HHS developed a list of key challenges within
and across the portfolios and assigned responsibility for addressing
those challenges to the appropriate agencies and offices.
* HHS has begun to develop a 5-year budget planning process, as
recommended in HHS's August 2010 review. This planning process, which
would not replace the annual budget and appropriations process, would
help the department determine how to fund medical countermeasures as
they move through development, acquisition, and stockpile replenishment
and as responsibility for the countermeasures moves from one HHS agency
or office to another. Some industry officials we spoke with told us
that such a process would increase their confidence in HHS by
demonstrating, for example, that the department has thought through the
funding for the whole lifecycle of a product, including replenishment
of the product once it is in the SNS.
HHS has not taken steps to address one of the recommendations from its
August 2010 review. As of August 2011, HHS officials said that they
have not appointed a medical countermeasure development leader with the
sole job of coordinating and integrating the multiple HHS medical
countermeasure development activities, as recommended in the review.
Officials said that the ESC has instead acted in this capacity.
HHS Has Gathered Information on Agencies' and Offices' Implementation
of the Initiatives but Has Not Developed an Adequate Strategy to
Monitor Overall Progress:
HHS has gathered information on agencies' and offices' implementation
of its initiatives from the August 2010 review intended to improve
PHEMCE and the department's medical countermeasure activities. With
ASPR as the lead agency responsible for monitoring implementation, ASPR
officials have gathered information from the responsible HHS agencies
and offices on the individual initiatives. This information, which ASPR
officials have compiled into an overall planning document, includes
planned time frames and milestones, status of any funding necessary to
implement initiatives, potential barriers to implementation, and
possible options for mitigating these barriers. ASPR officials said
that they intend to request quarterly updates to revise the planning
document.
Although HHS has compiled a planning document and conducted periodic
reviews of progress, HHS has not developed an adequate strategy that
meets federal internal control standards and best practices for program
management for monitoring the overall progress of the initiatives.
Federal internal control standards and best practices for program
management indicate the importance of a strategy to monitor
implementation. Federal internal control standards call for agencies to
review and evaluate actual performance against planned or expected
results on an ongoing basis and determine proper actions to address
identified differences.[Footnote 51] Best practices for program
management call for a centralized and coordinated strategy to align
efforts between the multiple projects that make up a program and to
monitor the overall progress of the program.[Footnote 52] According to
HHS officials, their monitoring strategy calls for quarterly updates of
ASPR's planning document, a high-level annual review of progress by
senior HHS officials, and more focused quarterly reviews by the ESC.
However, it is not clear that these activities will provide ASPR or the
ESC with the ongoing information needed to centrally and actively
monitor the agencies' and offices' overall implementation of the
initiatives, completion of any associated tasks or activities, and
resolution of any deficiencies. For example:
* As of June 2011, ASPR's planning document did not contain complete
information for each initiative that could be used by ASPR officials
for monitoring purposes. For example, the document does not have
information on planned time frames and milestones for four initiatives,
including the initiatives to improve management of product development
and develop a multiyear budget planning process.
* As of June 2011, the planning document did not portray information in
a way that makes it easy for ASPR officials to clearly identify
progress or gaps in implementation across all of the initiatives.
* Various HHS and PHEMCE entities have responsibility for monitoring
and oversight of these initiatives, including conducting the quarterly
and annual reviews, but it is not clear how monitoring and
implementation activities are centrally coordinated throughout the
department. In August 2011, HHS officials told us ASPR is the lead
agency for monitoring overall implementation of the initiatives and, as
such, designated a senior official to maintain the planning document.
However, the various agencies and offices implementing the initiatives
are responsible for any coordination and partnerships that are needed
to implement their respective initiatives.
* Even if HHS had clear and coordinated leadership, HHS does not
actively monitor the agencies' and offices' progress in implementing
the initiatives or their resolution of any deficiencies. ASPR sends
requests for status updates to the responsible office or agency.
Therefore, the information in the planning document depends upon the
completeness of the information that ASPR receives. In addition, ASPR
officials told us that the various agencies and offices implementing
the initiatives--and not ASPR--are responsible for bringing any issues
or deficiencies in implementation to the attention of the ESC during
the quarterly reviews.
Because HHS does not have an adequate strategy to monitor HHS agencies'
and offices' ongoing implementation of their initiatives that meets
federal internal control standards and best practices for program
management, the department lacks assurance that implementation of the
initiatives is coordinated across HHS and PHEMCE in order to improve
HHS's ability to develop and acquire medical countermeasures.
HHS's PHEMCE and Related Written Agreements Have Facilitated
Intradepartmental Coordination, but Challenges Remain That May Be
Addressed through HHS's New Initiatives:
HHS's establishment of PHEMCE in 2006 and its subsequent written
agreements have facilitated intradepartmental coordination on the
development and acquisition of medical countermeasures, but some
coordination challenges remain that may be addressed by HHS's new
initiatives. Features that have facilitated intradepartmental
coordination include establishing an agreed-upon collaboration process
through PHEMCE and documenting the agreements on the roles and
responsibilities of PHEMCE's intradepartmental partners. However,
industry and outside experts have reported that HHS's agencies and
offices lack internal coordination in the development and acquisition
of medical countermeasures, which hampers industry's ability to develop
needed CBRN countermeasures. Implementing the initiatives from HHS's
August 2010 review may mitigate some of these coordination challenges.
HHS's PHEMCE Structure and Related Written Agreements Have Facilitated
Intradepartmental Coordination:
Since 2006, PHEMCE has provided a structure for improved coordination
among HHS's agencies and offices, including ASPR, BARDA, CDC, FDA, and
NIH. HHS officials told us that before PHEMCE was established, CDC had
limited information about what medical countermeasures were being
developed and faced challenges in working with BARDA on setting
requirements for medical countermeasures for the SNS. CDC officials
indicated that since PHEMCE was established, coordination with BARDA
has improved. Further, officials from NIH told us that through PHEMCE,
NIH has coordinated with other HHS agencies to move medical
countermeasures from the early development phase into the advanced
development phase at BARDA. For example, NIH helped move a smallpox
antiviral from a support and guidance phase at NIH to BARDA for
advanced development and acquisition. In addition, NIH officials said
that for some CBRN agents, FDA has been proactive in supporting medical
countermeasure development. For example, FDA has helped to identify
suitable animal models or other tools necessary for its regulatory
review process.
Further, the establishment of related written agreements--a PHEMCE
memorandum of understanding (MOU) and charters for PHEMCE's working
groups--reinforced intradepartmental coordination and collaboration on
efforts to support medical countermeasure development and acquisition
by establishing areas of responsibility. The PHEMCE MOU and the working
group charters described the roles and responsibilities for each HHS
agency and office with respect to the development and acquisition of
medical countermeasures, and stipulated the minimum meeting frequency
of the PHEMCE governing body and working groups. Consistent with our
best practices for enhancing and sustaining coordination,[Footnote 53]
these written agreements served as guidance for HHS's agencies and
offices, clarifying the roles of the various agencies, whose missions
often have conflicting objectives that reflect different aspects of
complex public problems. The written agreements also facilitated clear
lines of responsibility and accountability for crosscutting program
efforts.[Footnote 54]
These agreements have lapsed or become outdated, but HHS is working to
renew them. The PHEMCE intradepartmental MOU expired in July 2010. In
August 2011, HHS officials told us that a new MOU had been drafted and
was being routed through the various HHS agencies and offices for
approval and signature. Similarly, the original PHEMCE governing body's
charter has become outdated due to changes in PHEMCE membership and the
establishment of the ESC. HHS officials also told us that a new charter
for the ESC would enhance and sustain intradepartmental coordination on
countermeasure development and acquisition activities. HHS drafted the
ESC charter to incorporate modifications made to the PHEMCE governing
body. As of August 2011, the charter had been routed through the
department for approval and signature and had been sent to the other
federal department partners for approval and signature.
Industry and Outside Experts Indicate That New Initiatives May Mitigate
Some Coordination Challenges:
Although PHEMCE and the related written agreements have facilitated
intradepartmental coordination, industry officials and outside experts
that we spoke with said that certain challenges in coordination have
continued. They indicated that the challenges were due primarily to
inconsistent procedures for coordination and unclear roles and
responsibilities of each of HHS's agencies in advancing products along
HHS's product development and acquisition pipeline. According to our
best practices, agencies can enhance and sustain their coordination by
adopting key practices--including defining and articulating a common
outcome, agreeing on roles and responsibilities, and establishing
compatible policies and procedures for operating across agency
boundaries.[Footnote 55] Comprehensive written agreements, such as the
PHEMCE MOU and related charters, can help clarify roles,
responsibilities, policies, and procedures. Industry officials stated
that the roles and responsibilities of each of HHS's agencies and
offices in coordinating to advance products along HHS's product
development and acquisition pipeline are not transparent, thus
hampering industry's ability to develop CBRN countermeasures.
Inconsistent procedures and a lack of clarity on the roles and
responsibilities of each HHS agency or office in moving CBRN
countermeasures through HHS's development pipeline can slow the already
lengthy and complex development process for acquiring products. In its
2010 report on HHS's countermeasure activities, the NBSB reported that
there has been insufficient coordination among HHS's agencies and
offices to successfully develop and acquire medical
countermeasures.[Footnote 56] For example, the NBSB reported that NIH
and BARDA needed to coordinate the transition of products between the
two agencies so that the evaluation of promising candidates can proceed
effectively. Similarly, in its 2010 countermeasure development workshop
summary, the Institute of Medicine reported that the process to acquire
countermeasures for the SNS is perceived by industry to be lengthy,
opaque, and unpredictable.[Footnote 57] In particular, the process to
transition countermeasures from advanced development to acquisition is
unclear.
Industry officials told us that FDA, NIH, and BARDA have not always
coordinated effectively to communicate regulatory requirements
throughout the development process for CBRN medical countermeasures.
Some industry officials told us that companies must work with both NIH
and FDA during the early development stage to ensure that they are
adhering to good manufacturing practices and to anticipate and help
prevent any problems in obtaining future FDA approval or licensure
after countermeasures are delivered to the SNS. In addition, BARDA is
to work with companies during the advanced development stage to prepare
them for the FDA approval process. However, there is no established
mechanism for ensuring that these interactions occur between the
companies, FDA, NIH, and BARDA. In addition, some industry officials
told us that BARDA and FDA have not coordinated effectively to reduce
uncertainties associated with the FDA regulatory process in the
advanced development stage. The officials said that, for example, one
company had worked closely with BARDA throughout the development
process to determine information needed for countermeasure development
but that when the company applied for FDA approval for its
countermeasure, FDA asked for information that BARDA had not discussed
with the company, and therefore, the company did not anticipate having
to provide. When the company could not supply the information, FDA
withheld approval. According to HHS, it is common practice for FDA to
request additional information from pharmaceutical companies that had
not been disclosed in previous discussions regarding safety and
efficacy testing of their products. HHS officials told us that BARDA
has expanded its outreach to FDA to address specific requirements,
resolve problems, and help move products toward approval.
Furthermore, industry officials from several companies also indicated
that roles and responsibilities for BARDA and CDC to move
countermeasures into the SNS have not always been clear. CDC is
responsible for maintaining the SNS, and CDC officials told us that
they develop and update a 5-year project plan for each countermeasure
in the SNS for shelf life, storage and space requirements, and other
specific needs. Despite this role, industry officials from several
companies indicated that CDC was not brought into the process early
enough in product development. For example, industry officials from one
company told us that CDC had not been involved in the development
process when their company was making decisions regarding product
characteristics, such as refrigeration or multi-dose or single-dose
vials. According to these officials, if CDC had been involved earlier,
the company might have developed the product with different
characteristics that CDC might consider improvements for dispensing.
Industry officials from another company said that CDC officials were
not familiar with the product characteristics of a new countermeasure
that they had developed for delivery to the SNS--even though BARDA
officials were familiar with their product. These industry officials
indicated that more interaction with CDC earlier in the process could
improve countermeasure development. CDC officials stated that they were
involved in the development of product requirements.
HHS officials and some industry officials we spoke with expect that
some of the new initiatives may help reinforce coordination among HHS's
agencies for developing and acquiring medical countermeasures. However,
it is too early to tell whether the initiatives will improve these
challenges. Examples of initiatives that may reinforce coordination
include the following:
* The creation of the ESC as PHEMCE's decision forum represents HHS's
effort to build cohesiveness within the PHEMCE structure by providing a
stronger governing role and by ensuring that the agencies' senior
leaders participate in the medical countermeasure decision-making
process.
* The medical countermeasure development leader, if created, could help
solve challenges related to coordinating HHS's multiple countermeasure
development activities and working with HHS's agencies that have their
own missions and infrastructures.
* ASPR's portfolio reviews, intended to provide overviews and updates
on the range of medical countermeasure activities for specific CBRN
agents, could further facilitate intradepartmental coordination. During
these reviews, senior leaders from each HHS agency come together to
discuss critical issues in developing countermeasures for these agents
(including anthrax, smallpox, and radiological and nuclear agents). HHS
officials told us that during these reviews, agency officials
identified issues that needed to be resolved to move forward with
development of countermeasures and worked together to determine
specific actions HHS needed to take to address these issues.
Conclusions:
Given the complexity and importance of developing and acquiring medical
countermeasures, HHS would benefit from updating its countermeasure
plan to indicate whether there have been any changes in priorities and
monitoring implementation of its new initiatives to address its own
recommendations. Establishing a new or revised PHEMCE implementation
plan for countermeasure development and acquisition that includes
planned funding for these activities in the coming years could set the
stage for a more transparent, integrated, and focused dialogue between
HHS and its federal and industry partners. Including more specific
information on anticipated budget priorities for countermeasure
acquisition would also be helpful to encourage industry to invest in
countermeasure development. Further, as the HHS agencies and offices
begin to implement initiatives to improve PHEMCE and HHS's medical
countermeasure activities, the department could benefit by developing a
strategy to monitor the overall progress of the initiatives that meets
federal internal control standards and best practices for program
management. Although various HHS agencies and offices have the lead in
implementing the different initiatives, virtually all of HHS's
initiatives require the concerted and coordinated efforts of its
intradepartmental agencies. Without an adequate monitoring strategy,
HHS will be unable to track its overall progress in implementing the
initiatives and hold its agencies and offices accountable for improving
its emergency medical countermeasure activities. One key aspect of
HHS's PHEMCE review was to improve coordination within HHS. By
implementing the initiatives to improve coordination and renewing its
written agreements, HHS is working to ensure that its agencies and
offices reach concurrence on their expected levels of involvement and
responsibility at each stage of the countermeasure development and
acquisition process.
Recommendations for Executive Action:
We are making three recommendations to improve HHS's countermeasure
enterprise and activities.
To ensure HHS's stated medical countermeasure development and
acquisition priorities are based on current risk assessments for CBRN
agents and the status of recent countermeasure research and development
and are clearly and transparently communicated, we recommend that the
Secretary of HHS take the following two actions:
* update the PHEMCE implementation plan for CBRN medical countermeasure
development and acquisition, and:
* incorporate into the updated plan more specific information on
anticipated budget priorities for countermeasure acquisition.
To provide reasonable assurance that HHS's initiatives will achieve the
intended results of improving PHEMCE and its medical countermeasure
activities, we recommend that the Secretary of HHS develop a strategy
to monitor the overall progress and results of implementing the
initiatives by obtaining complete information on all initiatives.
Agency Comments and Our Evaluation:
We provided a draft of this report to HHS, and its comments are
reprinted in appendix III. In its comments, HHS agreed with two of our
recommendations and provided information related to the third one. HHS
concurred with the first recommendation to update the PHEMCE
implementation plan. Consistent with our second recommendation to
incorporate more specific information on anticipated budget priorities
for countermeasure acquisition into the updated plan, HHS agreed that
one critical issue for the updated PHEMCE plan was how best to
communicate information on anticipated spending levels for the highest-
priority medical countermeasures. For the third recommendation on
having a strategy to monitor the overall progress and results of
implementing the initiatives to address its own recommendations, HHS
stated in its September 2011 comments that one of the overarching
PHEMCE accomplishments was having developed such a strategy and
provided us with an outline of the strategy. However, as of August
2011, HHS had not provided us with any evidence of such a strategy. As
we state in the report, the strategy for monitoring implementation of
its initiatives that HHS outlined in its comments does not meet federal
internal control standards and best practices for program management.
HHS's planning document did not contain complete information for each
initiative and did not portray information in a way that would
facilitate understanding of progress or gaps in implementation across
the initiatives as of June 2011, the latest quarter for which
documentation was available. As late as June 2011, HHS officials told
us that no individual or office was designated as responsible for
actively monitoring implementation across the initiatives. Further, the
HHS agencies and offices responsible for implementing the initiatives
are also responsible for bringing any issues or deficiencies in
implementation to the attention of the ESC. Therefore, we maintain that
HHS needs to develop a monitoring strategy that involves obtaining
complete information on all the initiatives, including planned time
frames and milestones, and to actively assess progress across all the
initiatives to ensure that management's directives are achieved.
HHS's comments also provided information on PHEMCE and its actions and
agency contributions to PHEMCE efforts. For example, HHS provided
information on activities of PHEMCE, BARDA, FDA, CDC, and NIH,
information that it characterized as high-level snapshots of activities
under way that reflect the significant and ongoing investment of energy
and resources for medical countermeasure research, development,
acquisition, procurement, and distribution. In addition, HHS provided
technical comments, which we incorporated as appropriate.
We are sending copies of this report to the Secretary of HHS and to
interested congressional committees. The report is also available at no
charge on the GAO Web site at [hyperlink, http://www.gao.gov]. If you
or your staffs 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 IV.
Signed by:
Marcia Crosse:
Director, Health Care:
[End of section]
Appendix I: HHS Medical Countermeasure Development and Acquisition
Priorities and Investments:
Since 2007, the Department of Health and Human Services (HHS) has
invested approximately $4.3 billion in medical countermeasure research,
development, and acquisition for chemical, biological, radiological,
and nuclear (CBRN) agents.[Footnote 58] HHS has sought to develop and
acquire particular medical countermeasures for the CBRN agents that the
Department of Homeland Security (DHS) deemed material threats to the
nation and for an additional chemical agent that DHS has not declared a
material threat, based on DHS's material threat assessments (MTA) and
HHS's public health consequence modeling reports.[Footnote 59] HHS
published its CBRN medical countermeasure priorities in the 2007 Public
Health Emergency Medical Countermeasures Enterprise (PHEMCE)
implementation plan, which included three time frames for planned
acquisitions based on the timing of the availability of funds from the
Project BioShield Special Reserve Fund and the status of countermeasure
research and development at the time. (See table 2 for HHS's
countermeasure priorities and time frames from the 2007 plan.)
Table 2: HHS's Medical Countermeasure Priorities and Time Frames by
Fiscal Year, as of April 2007:
HHS CBRN medical countermeasure priorities: Anthrax antitoxin;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Anthrax vaccine;
Near term (FY 2007 -FY 2008): x;
Midterm (FY 2009 -FY 2013): [Empty];
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Anthrax broad spectrum
antibiotics;
Near term (FY 2007 -FY 2008): x;
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Anthrax diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Botulism diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Ebola/Marburg broad
spectrum antivirals;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): [Empty];
Long term (beyond FY 2013): x.
HHS CBRN medical countermeasure priorities: Ebola/Marburg
countermeasures;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Ebola/Marburg diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Glanders/melioidosis broad
spectrum antibiotics;
Near term (FY 2007 -FY 2008): x;
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Glanders/melioidosis
diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Junin broad spectrum
antivirals;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): [Empty];
Long term (beyond FY 2013): x.
HHS CBRN medical countermeasure priorities: Junin diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Plague broad spectrum
antibiotics;
Near term (FY 2007 -FY 2008): x;
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Plague diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Radiological and nuclear,
acute radiation syndrome/delayed effects of acute radiation exposure
countermeasures;
Near term (FY 2007 -FY 2008): x;
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Radiological and nuclear
biodosimetry/bioassay (diagnostics);
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Radionuclide-specific
agents;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Smallpox broad spectrum
antivirals;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): [Empty];
Long term (beyond FY 2013): x.
HHS CBRN medical countermeasure priorities: Smallpox diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Smallpox antiviral;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Smallpox vaccine;
Near term (FY 2007 -FY 2008): x;
Midterm (FY 2009 -FY 2013): [Empty];
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Tularemia broad spectrum
antibiotics;
Near term (FY 2007 -FY 2008): x;
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Tularemia diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Typhus broad spectrum
antibiotics;
Near term (FY 2007 -FY 2008): x;
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Typhus diagnostics;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
HHS CBRN medical countermeasure priorities: Chemical/volatile nerve
agent single antidote;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): [Empty];
Long term (beyond FY 2013): x.
HHS CBRN medical countermeasure priorities: Chemical/Enterprise
CHEMPACKs;
Near term (FY 2007 -FY 2008): [Empty];
Midterm (FY 2009 -FY 2013): x;
Long term (beyond FY 2013): [Empty].
Source: GAO analysis of HHS Public Health Emergency Medical
Countermeasure Enterprise Implementation Plan for Chemical, Biological,
Radiological and Nuclear Threats, April 2007.
[End of table]
Because HHS has not been able to immediately acquire some
countermeasures listed as priorities in the 2007 PHEMCE plan, HHS has
invested approximately $1.9 billion in research and development from
fiscal year 2007 through fiscal year 2010. Of this $1.9 billion, the
National Institutes of Health (NIH) invested $1.2 billion in early
research and early development, and the Biomedical Advanced Research
and Development Authority (BARDA) invested $681 million in advanced
research and development. (See tables 3 and 4 for NIH and BARDA
investments, respectively.) From fiscal year 2007 through fiscal year
2010, HHS spent $2.4 billion to acquire available countermeasures for
the Strategic National Stockpile (SNS). Of this $2.4 billion, HHS
invested $1.1 billion of the Special Reserve Fund to acquire
countermeasures for the SNS, and the Centers for Disease Control and
Prevention (CDC) invested $1.3 billion to maintain quantities of
countermeasures held in the SNS. (See table 5 for HHS Project BioShield
Special Reserve Fund investments.)
Table 3: NIH Investments in CBRN Medical Countermeasure Early Research
and Development, Fiscal Year 2007 through Fiscal Year 2010:
Disease/agent: Anthrax (Bacillus anthracis)[B];
Medical countermeasure: Diagnostics;
FY 2007 investments: $4,227,137;
FY 2008 investments: $1,496,177;
FY 2009 investments[A]: $3,669,816;
FY 2010 investments[A]: $2,683,865;
Totals: $12,076,995.
Disease/agent: Anthrax (Bacillus anthracis)[B];
Medical countermeasure: Therapeutics (anthrax antitoxin)[C];
FY 2007 investments: 39,747,858;
FY 2008 investments: 28,154,770;
FY 2009 investments[A]: 33,385,361;
FY 2010 investments[A]: 27,076,406;
Totals: 128,364,395.
Disease/agent: Anthrax (Bacillus anthracis)[B];
Medical countermeasure: Vaccine;
FY 2007 investments: 26,014,877;
FY 2008 investments: 32,346,017;
FY 2009 investments[A]: 16,457,633;
FY 2010 investments[A]: 44,778,727;
Totals: 119,597,254.
Disease/agent: Total, anthrax;
Medical countermeasure: [Empty];
FY 2007 investments: 69,989,872;
FY 2008 investments: 61,996,964;
FY 2009 investments[A]: 53,512,810;
FY 2010 investments[A]: 74,538,998;
Totals: 260,038,644.
Disease/agent: Botulism toxin (Clostridium botulinum);
Medical countermeasure: Diagnostics;
FY 2007 investments: 1,894,757;
FY 2008 investments: 380,915;
FY 2009 investments[A]: 2,131,347;
FY 2010 investments[A]: 2,175,551;
Totals: 6,582,570.
Disease/agent: Total, botulism toxin;
Medical countermeasure: [Empty];
FY 2007 investments: 1,894,757;
FY 2008 investments: 380,915;
FY 2009 investments[A]: 2,131,347;
FY 2010 investments[A]: 2,175,551;
Totals: 6,582,570.
Disease/agent: Ebola and Marburg (viral hemorrhagic fevers);
Medical countermeasure: Antivirals (Ebola);
FY 2007 investments: 3,066,405;
FY 2008 investments: 11,602,269;
FY 2009 investments[A]: 8,130,700;
FY 2010 investments[A]: 8,853,362;
Totals: 31,652,736.
Disease/agent: Ebola and Marburg (viral hemorrhagic fevers);
Medical countermeasure: Antivirals (Marburg);
FY 2007 investments: 797,157;
FY 2008 investments: 532,309;
FY 2009 investments[A]: 2,847,050;
FY 2010 investments[A]: 2,240,903;
Totals: 6,417,419.
Disease/agent: Ebola and Marburg (viral hemorrhagic fevers);
Medical countermeasure: Diagnostics (Ebola);
FY 2007 investments: --;
FY 2008 investments: 145,194;
FY 2009 investments[A]: 196,963;
FY 2010 investments[A]: 1,281,618;
Totals: 1,623,775.
Disease/agent: Ebola and Marburg (viral hemorrhagic fevers);
Medical countermeasure: Diagnostics (Marburg);
FY 2007 investments: --;
FY 2008 investments: 145,194;
FY 2009 investments[A]: 121,320;
FY 2010 investments[A]: 176,057;
Totals: 442,571.
Disease/agent: Ebola and Marburg (viral hemorrhagic fevers);
Medical countermeasure: Vaccine (Ebola);
FY 2007 investments: 31,180,016;
FY 2008 investments: 30,586,592;
FY 2009 investments[A]: 21,653,115;
FY 2010 investments[A]: 37,163,860;
Totals: 120,583,583.
Disease/agent: Ebola and Marburg (viral hemorrhagic fevers);
Medical countermeasure: Vaccine (Marburg);
FY 2007 investments: 10,962,249;
FY 2008 investments: 18,580,539;
FY 2009 investments[A]: 15,137,164;
FY 2010 investments[A]: 26,822,113;
Totals: 71,502,065.
Disease/agent: Total, Ebola and Marburg;
Medical countermeasure: [Empty];
FY 2007 investments: 46,005,827;
FY 2008 investments: 61,592,097;
FY 2009 investments[A]: 48,086,312;
FY 2010 investments[A]: 76,537,913;
Totals: 232,222,149.
Disease/agent: Glanders/melioidosis (Burkholderia);
Medical countermeasure: Diagnostics;
FY 2007 investments: 1,044,626;
FY 2008 investments: 1,675,818;
FY 2009 investments[A]: 3,383,319;
FY 2010 investments[A]: 3,437,768;
Totals: 9,541,531.
Disease/agent: Total, glanders and melioidosis;
Medical countermeasure: [Empty];
FY 2007 investments: 1,044,626;
FY 2008 investments: 1,675,818;
FY 2009 investments[A]: 3,383,319;
FY 2010 investments[A]: 3,437,768;
Totals: 9,541,531.
Disease/agent: Junin (viral hemorrhagic fever);
Medical countermeasure: Diagnostics;
FY 2007 investments: 55,477;
FY 2008 investments: 44,176;
FY 2009 investments[A]: 32,601;
FY 2010 investments[A]: 67,080;
Totals: 199,334.
Disease/agent: Total, Junin;
Medical countermeasure: [Empty];
FY 2007 investments: 55,477;
FY 2008 investments: 44,176;
FY 2009 investments[A]: 32,601;
FY 2010 investments[A]: 67,080;
Totals: 199,334.
Disease/agent: Plague (Yersinia pestis);
Medical countermeasure: Diagnostics;
FY 2007 investments: 754,835;
FY 2008 investments: 738,778;
FY 2009 investments[A]: 1,607,449;
FY 2010 investments[A]: 1,392,686;
Totals: 4,493,748.
Disease/agent: Total, plague;
Medical countermeasure: [Empty];
FY 2007 investments: 754,835;
FY 2008 investments: 738,778;
FY 2009 investments[A]: 1,607,449;
FY 2010 investments[A]: 1,392,686;
Totals: 4,493,748.
Disease/agent: Smallpox (Variola major)[D];
Medical countermeasure: Diagnostics;
FY 2007 investments: 1,068,331;
FY 2008 investments: 2,145,659;
FY 2009 investments[A]: 2,179,066;
FY 2010 investments[A]: 3,798,182;
Totals: 9,191,238.
Disease/agent: Smallpox (Variola major)[D];
Medical countermeasure: Therapeutics (smallpox antiviral);
FY 2007 investments: 32,264,000;
FY 2008 investments: 21,983,318;
FY 2009 investments[A]: 32,532,188;
FY 2010 investments[A]: 24,041,364;
Totals: 110,820,870.
Disease/agent: Smallpox (Variola major)[D];
Medical countermeasure: Vaccine;
FY 2007 investments: 55,650,473;
FY 2008 investments: 58,558,339;
FY 2009 investments[A]: 26,568,676;
FY 2010 investments[A]: 34,189,768;
Totals: 174,967,256.
Disease/agent: Total, smallpox;
Medical countermeasure: [Empty];
FY 2007 investments: 88,982,804;
FY 2008 investments: 82,687,316;
FY 2009 investments[A]: 61,279,930;
FY 2010 investments[A]: 62,029,314;
Totals: 294,979,364.
Disease/agent: Tularemia (Francisella tularensis);
Medical countermeasure: Diagnostics;
FY 2007 investments: 2,788,482;
FY 2008 investments: 33,939;
FY 2009 investments[A]: 1,361,838;
FY 2010 investments[A]: 1,360,410;
Totals: 5,544,669.
Disease/agent: Total, tularemia;
Medical countermeasure: [Empty];
FY 2007 investments: 2,788,482;
FY 2008 investments: 33,939;
FY 2009 investments[A]: 1,361,838;
FY 2010 investments[A]: 1,360,410;
Totals: 5,544,669.
Disease/agent: Typhus (Rickettsia prowazekii);
Medical countermeasure: Diagnostics;
FY 2007 investments: --;
FY 2008 investments: 222,387;
FY 2009 investments[A]: 169,731;
FY 2010 investments[A]: 181,923;
Totals: 574,041.
Disease/agent: Total, typhus;
Medical countermeasure: [Empty];
FY 2007 investments: --;
FY 2008 investments: 222,387;
FY 2009 investments[A]: 169,731;
FY 2010 investments[A]: 181,923;
Totals: 574,041.
Disease/agent: Chemical;
Medical countermeasure: [Empty];
FY 2007 investments: 49,542,368;
FY 2008 investments: 48,651,701;
FY 2009 investments[A]: 49,138,476;
FY 2010 investments[A]: 49,847,755;
Totals: 197,180,300.
Disease/agent: Total, chemical;
Medical countermeasure: [Empty];
FY 2007 investments: 49,542,368;
FY 2008 investments: 48,651,701;
FY 2009 investments[A]: 49,138,476;
FY 2010 investments[A]: 49,847,755;
Totals: 197,180,300.
Disease/agent: Radiological and nuclear;
Medical countermeasure: [Empty];
FY 2007 investments: 46,487,561;
FY 2008 investments: 45,700,000;
FY 2009 investments[A]: 47,571,244;
FY 2010 investments[A]: 46,858,859;
Totals: 186,617,664.
Disease/agent: Total, radiological and nuclear;
Medical countermeasure: [Empty];
FY 2007 investments: 46,487,561;
FY 2008 investments: 45,700,000;
FY 2009 investments[A]: 47,571,244;
FY 2010 investments[A]: 46,858,859;
Totals: 186,617,664.
Disease/agent: Totals;
Medical countermeasure: [Empty];
FY 2007 investments: 307,546,609;
FY 2008 investments: 303,724,091;
FY 2009 investments[A]: 268,275,057;
FY 2010 investments[A]: 318,428,257;
Totals: 1,197,974,014.
Source: GAO analysis of NIH data.
Note: NIH does not break out its investments in early research and
development of radiological, nuclear, and chemical agents by type of
countermeasure. Also, in addition to NIH investments in the specific
countermeasures HHS identified as priorities, NIH has invested in
research related to other countermeasures for the 14 agents in the 2007
PHEMCE plan. For example, HHS's only priority countermeasure identified
to address botulinum toxin was for diagnostics, but NIH also invested
in research on countermeasures to treat the effects of the toxin on
affected individuals. NIH's investments also include research on other
potential public health threats, including foodborne diseases such as
salmonella, emerging infectious diseases such as severe acute
respiratory syndrome, and other agents such as ricin toxin.
[A] NIH CBRN medical countermeasure investments for fiscal years 2009
and 2010 include the use of funds from the American Recovery and
Reinvestment Act of 2009. Pub. L. No. 111-5, 123 Stat. 115 (2009).
[B] NIH investments in countermeasures for anthrax include multidrug-
resistant anthrax.
[C] NIH investments in anthrax therapeutics may also include
antibiotics for preventing infection after exposure, in addition to
treating the health effects of infection with anthrax.
[D] NIH investments in countermeasures for smallpox include other pox
viruses, such as monkeypox.
[End of table]
Table 4: BARDA Investments in CBRN Medical Countermeasure Advanced
Research and Development, Fiscal Year 2007 through Fiscal Year 2010:
Medical countermeasure: Anthrax therapeutics;
FY 2007 investments: $27,080,000;
FY 2008 investments: $12,750,000;
FY 2009 investments: $31,430,000;
FY 2010 investments: $43,400,000;
Totals: $114,660,000.
Medical countermeasure: Anthrax vaccines;
FY 2007 investments: 43,420,000;
FY 2008 investments: 13,070,000;
FY 2009 investments: 81,370,000;
FY 2010 investments: 105,660,000;
Totals: 243,520,000.
Medical countermeasure: Broad spectrum antibiotics;
FY 2007 investments: 9,000,000;
FY 2008 investments: 1,050,000;
FY 2009 investments: 4,900,000;
FY 2010 investments: 27,560,000;
Totals: 42,510,000.
Medical countermeasure: Smallpox medical countermeasures;
FY 2007 investments: 2,980,000;
FY 2008 investments: 18,070,000;
FY 2009 investments: 21,000,000;
FY 2010 investments: 11,300,000;
Totals: 53,350,000.
Medical countermeasure: Radiological and nuclear medical
countermeasures;
FY 2007 investments: 10,000,000;
FY 2008 investments: 29,860,000;
FY 2009 investments: 67,190,000;
FY 2010 investments: 38,950,000;
Totals: 146,000,000.
Medical countermeasure: Biodosimetry (radiological and nuclear)[A];
FY 2007 investments: --;
FY 2008 investments: --;
FY 2009 investments: 36,050,000;
FY 2010 investments: 1,930,000;
Totals: 37,980,000.
Medical countermeasure: Chemical medical countermeasures;
FY 2007 investments: 6,320,000;
FY 2008 investments: 700,000;
FY 2009 investments: 2,000,000;
FY 2010 investments: 2,100,000;
Totals: 11,120,000.
Medical countermeasure: Innovation[B];
FY 2007 investments: --;
FY 2008 investments: --;
FY 2009 investments: 7,000,000;
FY 2010 investments: 24,680,000;
Totals: 31,680,000.
Medical countermeasure: Totals;
FY 2007 investments: 98,800,000;
FY 2008 investments: 75,500,000;
FY 2009 investments: 250,940,000;
FY 2010 investments: 255,580,000;
Totals: 680,820,000.
Source: GAO analysis of BARDA data.
[A] Biodosimetry, including radiation bioassays, are diagnostic tools
to determine the level of radiation and the type of radioactive isotope
to which an individual is exposed.
[B] Innovation includes investments in tools to enhance individuals'
immune response to vaccines, stabilize vaccines, and other tools.
[End of table]
Table 5: HHS Investments in CBRN Medical Countermeasures Using the
Project BioShield Special Reserve Fund, Fiscal Year 2007 through Fiscal
Year 2010:
Medical countermeasure: Anthrax antitoxin;
Project BioShield investment FY 2007: --;
Project BioShield investment FY 2008: --;
Project BioShield investment FY 2009: $152,000,000;
Medical countermeasure: Anthrax vaccine;
Project BioShield investment FY 2007: 448,000,000;
Project BioShield investment FY 2008: --;
Project BioShield investment FY 2009: --;
Project BioShield investment FY 2010: --;
Totals: 448,000,000.
Medical countermeasure: Smallpox vaccine;
Project BioShield investment FY 2007: 505,000,000;
Project BioShield investment FY 2008: --;
Project BioShield investment FY 2009: --;
Project BioShield investment FY 2010: --;
Totals: 505,000,000.
Medical countermeasure: Totals;
Project BioShield investment FY 2007: 953,000,000;
Project BioShield investment FY 2008: --;
Project BioShield investment FY 2009: 152,000,000;
Project BioShield investment FY 2010: --;
Totals: 1,105,000,000.
Source: GAO analysis of HHS information.
Note: From 2004 through 2006, HHS also invested about $1 billion of the
Project BioShield Special Reserve Fund to acquire certain medical
countermeasures. Specifically, HHS invested $318 million to acquire
anthrax antitoxin, $245 million to acquire anthrax vaccine, $415
million to acquire botulinum antitoxin, and $40 million to acquire a
pediatric formulation of potassium iodide and calcium and zinc
diethylene triamine penta-acetic acid--countermeasures that block the
absorption of radioactive iodine or remove radiation from the body.
[End of table]
[End of section]
Appendix II: HHS Initiatives to Address Its Recommendations to Improve
Medical Countermeasure Activities:
In 2006, the Department of Health and Human Services (HHS) established
the Public Health Emergency Medical Countermeasures Enterprise
(PHEMCE), a federal interagency body responsible for providing
recommendations to the Secretary of HHS on the development and
acquisition of chemical, biological, radiological, and nuclear (CBRN)
medical countermeasures, among other responsibilities.[Footnote 60] In
December 2009, the HHS Secretary called for a comprehensive review of
PHEMCE and the department's medical countermeasure development and
acquisition activities. As a result, HHS issued an August 2010 review
with 13 recommendations to improve its infrastructure to support
product development and acquisition and to enhance PHEMCE's structure
and management.[Footnote 61] Table 6 shows the 13 recommendations,
HHS's initiatives to address the recommendations, the HHS lead
organization that is responsible for implementation, and HHS's reported
actions to implement the initiatives.
Table 6: Recommendations from HHS's August 2010 Review of PHEMCE and
Its Medical Countermeasure Activities and HHS's Initiatives in
Response:
Recommendations and Initiatives on Infrastructure to Support Product
Development and Acquisition:
#: 1;
Recommendation from HHS's August 2010 Review: Enable innovative
regulatory science and oversight;
HHS's Initiative to Address Recommendation (lead organization): Medical
Countermeasure Initiative to provide developers of medical
countermeasures with greater clarity on aspects of the regulatory
framework and more interaction with FDA regulators by (1) enhancing the
review process by establishing action teams; (2) advancing regulatory
science; and (3) modernizing the legal, regulatory, and policy
framework for public health response (FDA);
Reported Actions to Implement Initiatives: FDA officials reported
taking several actions to address the recommendations, including
establishing action teams to identify resources and subject-matter
expertise to facilitate an improved regulatory review process for
different products and developing statutory proposals that could
improve public health response.
# 2;
Recommendation from HHS's August 2010 Review: Expand pipeline of
potential products;
HHS's Initiative to Address Recommendation (lead organization): Concept
Acceleration Program to increase the number of potential products in
the pipeline by proactively seeking out and providing additional
resources to investigators who are conducting promising basic research
with the potential to fulfill a particular CBRN medical countermeasure
need (NIH);
Reported Actions to Implement Initiatives: NIH officials said they have
begun hiring staff for this program and hope to complete hiring by the
end of 2011.
#: 3;
Recommendation from HHS's August 2010 Review: Foster partnerships to
support flexible manufacturing and advanced development activities;
HHS's Initiative to Address Recommendation (lead organization): Centers
for Innovation in Advanced Development and Manufacturing to provide
companies with technical expertise and services in scale-up
manufacturing, regulatory affairs, and other aspects of advanced
development and manufacturing (BARDA);
Reported Actions to Implement Initiatives: BARDA issued a request for
proposals in March 2011, with a June 2011 deadline for responses. BARDA
officials anticipate that the first centers will open in 2014.
#: 4;
Recommendation from HHS's August 2010 Review: Create an independent
strategic investment firm for innovation in medical countermeasures;
HHS's Initiative to Address Recommendation (lead organization):
Strategic Investor Initiative to identify and provide capital and
business expertise to companies with technologies that are both likely
to result in a commercially attractive product and capable of producing
medical countermeasures that satisfy government-specific needs (BARDA);
Reported Actions to Implement Initiatives: BARDA has sought statutory
authority from Congress to create the investment firm and has developed
various options for how to structure it.
Recommendations and Initiatives on Enhancements to PHEMCE's Structure
and Management:
#: 5;
Recommendation from HHS's August 2010 Review: Establish a medical
countermeasure development leader;
HHS's Initiative to Address Recommendation (lead organization): Medical
countermeasure development leader, independent of the ASPR, to
coordinate and integrate the multiple efforts and programs within HHS
to assist in successful development of medical countermeasures (HHS
Office of the Secretary);
Reported Actions to Implement Initiatives: HHS officials said that they
have not appointed a medical countermeasure development leader and that
the Enterprise Senior Council (ESC) is acting in this capacity.
#: 6;
Recommendation from HHS's August 2010 Review: Establish better
coordination within HHS;
HHS's Initiative to Address Recommendation (lead organization):
Establishment of ESC as the decision forum for PHEMCE with expanded
representation by non-HHS partners (ASPR); Reported Actions to
Implement Initiatives: HHS officials said the HHS agencies and offices
have signed the charter to create the ESC, which first met in February
2011.
#: 7;
Recommendation from HHS's August 2010 Review: Coordinate and
collaborate with federal government partners;
HHS's Initiative to Address Recommendation (lead organization):
Establishment of ESC as the decision forum for PHEMCE with expanded
representation by non-HHS partners (ASPR); Reported Actions to
Implement Initiatives: HHS officials said the HHS agencies and offices
have signed the charter to create the ESC, which first met in February
2011.
#: 8;
Recommendation from HHS's August 2010 Review: Use a systematic approach
to decision making;
HHS's Initiative to Address Recommendation (lead organization):
Development of a systematic approach for analyzing the considerations
and trade-offs in decisions throughout the process of developing and
acquiring medical countermeasures (ASPR);
Reported Actions to Implement Initiatives: ASPR has approved the use of
a decision management system that can be used to assess competing
investments in medical countermeasures based on economic and other
decision analysis tools.
#: 9;
Recommendation from HHS's August 2010 Review: Improve contracting and
communication;
HHS's Initiative to Address Recommendation (lead organization): Methods
to improve speed of contracting and decision-making processes and
development of HHS-level policy on use of Other Transaction
Authorities, which are mechanisms other than contracts, grants, and
cooperative agreements that can provide more flexible, faster ways of
procuring goods and services (ASPR, BARDA, NIH);
Reported Actions to Implement Initiatives: A team of ASPR and BARDA
contracting officials and scientists reviewed the contracting process
and recommended potential actions and new procurement methods to
improve the speed of the process. In addition, HHS officials reported
that they have provided ASPR employees with additional training on
communicating with companies during the contracting process, and HHS
officials have begun holding meetings with industry stakeholders in
different regions of the United States.
#: 10;
Recommendation from HHS's August 2010 Review: Improve management of
product development; HHS's Initiative to Address Recommendation (lead
organization): Portfolio reviews across all of HHS's investments for
countermeasures for addressing a given agent to better understand the
range of countermeasures for each threat and highlight any gaps or
issues (ASPR);
Reported Actions to Implement Initiatives: According to ASPR officials,
senior leaders from across HHS--including ASPR, CDC, FDA, and NIH--
conducted portfolio reviews of major investments in the efforts for
anthrax, smallpox, radiological/nuclear, and pandemic influenza
threats. After the reviews concluded in March 2011, the leaders
developed a list of key challenges within and across the portfolios and
assigned responsibility for addressing those challenges to the
appropriate agencies and offices.
#: 11;
Recommendation from HHS's August 2010 Review: Reexamine the statutory
framework for how liability protection is provided;
HHS's Initiative to Address Recommendation (lead organization): Review
of the Public Readiness and Emergency Preparedness Act and its
framework for providing appropriate liability protection for the
development, testing, manufacture, and administration of medical
countermeasures without commercial uses (ASPR);
Reported Actions to Implement Initiatives: According to HHS officials,
they have begun to identify challenges with and possible improvements
to the Public Readiness and Emergency Preparedness Act.
#: 12;
Recommendation from HHS's August 2010 Review: Update the requirements
for current and future products;
HHS's Initiative to Address Recommendation (lead organization): Review
of underlying planning assumptions and requirements in order to develop
a more flexible and capabilities-based strategy (ASPR);
Reported Actions to Implement Initiatives: According to HHS officials,
they plan to develop planning scenarios for high-priority threats and
create a prioritized list of requirements. They also plan to determine--
and gain PHEMCE approval on--the type of information that should be
included in the requirements for certain scenarios and for certain
products.
#: 13;
Recommendation from HHS's August 2010 Review: Develop multiyear budget
planning process;
HHS's Initiative to Address Recommendation (lead organization):
Coordinated multiyear process to plan for lifecycle costs across PHEMCE
(HHS Assistant Secretary for Financial Resources);
Reported Actions to Implement Initiatives: HHS officials said that they
have begun to develop a 5-year budget planning process to help the
department determine how to fund medical countermeasures as they move
through the stages of development, acquisition, and stockpile
replenishment and as responsibility for the countermeasures moves from
one HHS agency or office to another.
Source: GAO analysis of HHS information.
[End of table]
[End of section]
Appendix III: Comments from the Department of Health and Human
Services:
Department Of Health & Human Services:
Office Of The Secretary:
Assistant Secretary for Legislation:
Washington, DC 20201:
September 1, 2001:
Marcia Crosse, Director:
Health Care:
U.S. Government Accountability Office:
441 G Street NW:
Washington, DC 20548:
Dear Ms. Crosse:
Attached are comments on the U.S. Government Accountability Office's
(GAO) draft report entitled: "National Preparedness: Improvements
Needed for Acquiring Medical Countermeasures Against Threats from
Terrorism and Other Sources" (GAO-11-840).
The Department appreciates the opportunity to review this report before
its publication.
Sincerely,
Signed by:
Jim R. Esquea:
Assistant Secretary for Legislation:
Attachment:
General Comments of the Department of Health and Human Services (HHS)
on the Government Accountability Office's (GA0) Draft Report Entitled:
"National Preparedness: Improvements Needed for Acquiring Medical
Countermeasures Against Threats from Terrorism and Other Sources (GAO-
11-840)"
The Department of Health and Human Services (HHS) appreciates the
opportunity to provide comments to the Government Accountability
Office's (GAO) report National Preparedness: Improvements Needed for
Acquiring Medical Countermeasures Against Threats from Terrorism and
Other Sources.
Tragic events such as the anthrax attacks of 2001 and the HI NI
influenza pandemic of 2009 underscore the reality that our nation
remains vulnerable to deliberate chemical, biological, radiological,
and nuclear (CBRN) threats as well as pandemic and other emerging
infectious diseases. HHS agrees with the Government Accountability
Office (GAO) that efficient interagency coordination is critical to
effective development and acquisition of medical countermeasures to
counter CBRN threats as well as pandemic and other emerging infectious
diseases. In August 2010, the HHS Secretary issued the Public Health
Emergency Medical Countermeasures Enterprise Review (2010 MCM Review)
that assessed all aspects of medical countermeasure development, from
concept to approval. Using these recommendations, HHS has implemented a
strategic approach toward strengthening all phases of the medical
countermeasure pipeline. HHS launched numerous efforts to strengthen
coordination among relevant agencies with the goal of supporting the
efficient development and approval of medical countermeasures. Other
processes implemented under this strategy ensure HHS is able to more
effectively oversee the full life-cycle management of medical products
and has a robust operational component to support the nation's
capability to respond. A key focus of many agency activities within
this strategy has been to reduce uncertainties for developers of
promising medical countermeasures and to expand communications among
relevant agencies and between the agencies and prospective developers.
The Public Health Emergency Medical Countermeasures Enterprise (PHEMCE)
is the overarching interagency convening body that coordinates the
multiple efforts and programs that enable the nation to respond to CBRN
threats as well as to pandemic and emerging diseases. The Assistant
Secretary for Preparedness and Response (ASPR) leads the PHEMCE, which
brings together three primary HHS internal agencies: the National
Institutes of Health, National Institute of Allergy and Infectious
Diseases (NIH/NIAID), the Centers for Disease Control and Prevention
(CDC), and the Food and Drug Administration (FDA), along with four key
interagency partners: Department of Homeland Security (DHS), Department
of Defense (DoD), Department of Veterans Affairs (VA) and Department of
Agriculture (USDA). Together, these Enterprise agencies and
organizations work to optimize preparedness for public health
emergencies in connection with the creation, stockpiling and use of
medical countermeasures. The agencies engage directly by regular
participation in Enterprise-led workgroups and meetings[Footnote 62] as
well as on numerous scientific and technical teams.[Footnote 63] The
PHEMCE's efforts are already helping to foster information-sharing and
education among agencies, medical countermeasure developers and other
relevant external stakeholders. The Enterprise is specifically
responsible for the following:
* Defining and prioritizing requirements for public health emergency
medical countermeasures;
* Focusing research, development and procurement activities on the
identified requirements; and:
* Establishing deployment and use strategies for medical
countermeasures in the Strategic National Stockpile.
Overarching accomplishments by the PHEMCE over the recent past include:
* Developed a strategy to track progress in implementing the major
initiatives called for in the 2010 MCM Review.
* In May 2010 and over the course of the last year, instituted the
first ever full portfolio reviews for priority threats, including
anthrax, smallpox, radiological /nuclear threats and pandemic
influenza. These reviews identified gaps across the life cycle of
products” from setting requirements through product development and
procurement, into stockpile and use. Important gaps were captured as
Priority Action Items and are being tracked via a biweekly process to
assure they are addressed.
* Instituted a "Case Management" approach for specific products or
product areas that require the concerted effort of
NINIH/NIAID,ASPR/BARDACDC, and FDA to solve issues related to the
setting of product specific requirements up to advanced development,
approval and procurement. Case Management is defined as a new process
to increase attention on a specific product or program for the purpose
of accelerating solutions for particular advanced development
requirements and engaging a dedicated team from across the agencies to
effect the needed changes.
* Established a governance structure for the PHEMCE which has
effectively overseen all programmatic and policy efforts and includes
teams from the program level up to senior leadership. Teams include
Integrated Product Teams, a Portfolio Advisory Committee, the
Enterprise Executive Committee and the Enterprise Senior Council.
* Quarterly convened the Enterprise Senior Council (ESC) to provide
strategic oversight and decision making on key issues, such as a
systematic process for identifying the major scenarios against which
PHEMCE requirements must be developed. The ESC has also been
instrumental in developing consensus advice around major procurement
actions under consideration by the ASPR and BARDA.
* Improved outreach and communication with stakeholders and industry
partners on current medical countermeasure investment and
prioritization including:
- The ASPR-maintained website [hyperlink,
www.medicalcountermeasures.gov], which provides up-to-date information
on medical countermeasure advanced research and development and
procurement contracts, as well as broad policy documents issued by the
White House and/or Congress that may impact medical countermeasure
programs.
- Biomedical Advanced Research and Development Authority's (BARDA)
Stakeholders Workshop and Industry Day during which participants
receive information on BARDA's strategy, goals, and areas of interest;
its roles and responsibilities, the ASPR Office of Acquisitions
Management, Contracts, and Grants (AMCG), and Contractors in contract
administration and performance; BARDA's core capabilities to support
product development; new initiatives designed to address the challenges
associated with developing medical countermeasures; and opportunities
to develop medical countermeasures for both government and commercial
markets. In addition, at the request of participants, BARDA program
staff and/or AMCG Contracting Officers are available to meet one-on-
one.
Agency Contributions to PHEMCE Efforts:
BARDA is coordinating an integrated, systematic approach to the
development and purchase of necessary vaccines, drugs, therapies, and
diagnostic tools for use during a public health emergency. It manages
Project BioShield, which pursues the procurement and advanced
development of medical countermeasures for CBRN threats. BARDA also
works to develop and procure medical countermeasures for pandemic
influenza and other emerging infectious diseases that fall outside the
auspices of Project BioShield. In addition, over the past year, BARDA
has implemented routine in-process reviews (IPRs) to evaluate progress,
obstacles, challenges, and solutions for medical countermeasure
development and manufacturing projects facing pivotal milestones. IPRs,
which are conducted in consultation with MAID, CDC, DoD, and FDA,
provide useful technical and regulatory evaluation and advice to
contractors outside of the usual framework between FDA and product
sponsors. Private sector partners have expressed their appreciation for
this new forum for discussion of product development and regulatory
issues with all members of the Enterprise. The IPRs have provided FDA
with an opportunity to interface with product sponsors much earlier in
the development process for very specific indications against CBRN,
pandemic influenza and other emerging infectious disease threats. BARDA
has furthermore instituted a Case Management approach to assist
contractors needing support in technical, clinical, and regulatory
areas. BARDA partners with CDC, NIAID and FDA staff and senior leaders
on a regular basis to address specific challenges and issues in order
to move a candidate product toward approval and/or acquisition.
The FDA regulates all medical products within the PHEMCE and is
integral to strategic success in approving products for procurement or
assessing emergency use approvals for those awaiting final approval. In
response to the 2020I0CM Review, FDA launched an MCM initiative to
build upon ongoing medical countermeasure activities and expand
communication and collaboration with federal partners and prospective
developers. The initiative aims to overcome regulatory hurdles, reduce
regulatory uncertainties, and anticipate and communicate to developers
the new regulatory strategies needed to develop novel concepts and
obtain approval of candidate products. FDA is working closely with
BARDA, CDC, DoD and NIH in the earliest stages of requirements setting
and product development to make sure that appropriate regulatory
requirements are clear and that product developers are able to gather
the necessary data for determining safety and efficacy. FDA is
establishing internal collaborations and Action Teams to identify
regulatory holdups and recommend changes to improve product review
processes. Regulatory science research is already under way to develop
innovative tools to support novel medical countermeasure development.
The CDC develops and exercises a concept of operations to deliver and
dispense medical countermeasures where and when needed. CDC is the
critical link to state, local, territorial and tribal end-users, thus
representing within the PHEMCE the voice of the public health and
medical community needs in product development and deployment. CDC also
manages the Strategic National Stockpile and addresses the logistical
requirements and allocation of resources for maintaining and replacing
its multiple products.
NIAID oversees a robust and forward-looking research program that
informs advanced development efforts through exploring scientific
knowledge of threats and producing a pipeline of candidate medical
countermeasures. NIAID has established the Concept Acceleration Program
to accelerate this transition as described in the 2010 MCM Review. This
effort will actively identify promising technologies and concepts and
will provide added funding and access to services to more effectively
incubate and transition good concepts into advanced development
potential.
PHEMCE efforts are already underway to ensure the continued success of
these important programs. Component organizations have developed five-
year spending plans and have identified strategic goals that reflect
the priorities of the enterprise. HHS concurs with GAO that the 2007
PHEMCE Strategy and Implementation Plans for Chemical, Biological,
Radiological and Nuclear Threats should be updated and, as noted in the
report, efforts have already begun to enable the release of such
updated Plans in Spring 2020I2. Steering Committee to develop these
Plans for ultimate review and approval by the Enterprise Senior Council
has been established and will be led by ASPR staff. The Steering
Committee has begun discussions concerning, among other critical
issues, how best to frame and communicate anticipated spending levels
for the highest priority medical countermeasure programs. Any such
estimates must consider the future status of the Special Reserve Fund
which funds Project BioShield contracts, as well as the annual
appropriations process that funds the vast majority of PHEMCE
activities. At this time, the information and subsequent prioritization
included in the 2007 PHEMCE is still relevant. Priorities identified in
the 2007 PHEMCE Implementation plan were based on DHS MTDs and other
available information. Since 2007, necessary countermeasures for CBRN
threats have not significantly changed.
The high-level snapshots of activities underway reflect the significant
and ongoing investment of energy and resources for medical
countermeasure research, development, acquisition, procurement, and
distribution to better prepare the nation against CBRN, pandemic and
other emerging infectious disease threats. These investments also
demonstrate the federal public health community's long-term commitment
to this critical national effort. HHS is committed to ensuring the
nation is prepared for a potential CBRN event and pandemic or emerging
infectious disease threat and will continue to refine its medical
countermeasure priorities and investments.
[End of section]
Appendix IV: GAO Contact and Staff Acknowledgments:
GAO Contact:
Marcia Crosse, (202) 512-7114 or crossem@gao.gov:
Acknowledgments:
In addition to the contacts named above, Sheila K. Avruch, Assistant
Director; Shana R. Deitch; Tracey King; Corissa Kiyan; Carolina Morgan;
Roseanne Price; and Jessica C. Smith made significant contributions to
this report.
[End of section]
Related GAO Products:
National Preparedness: DHS and HHS Can Further Strengthen Coordination
for Chemical, Biological, Radiological, and Nuclear Risk Assessments.
[hyperlink, http://www.gao.gov/products/GAO-11-606]. Washington, D.C.:
June 21, 2011.
Public Health Preparedness: Developing and Acquiring Medical
Countermeasures Against Chemical, Biological, Radiological, and Nuclear
Agents. [hyperlink, http://www.gao.gov/products/GAO-11-567T].
Washington, D.C.: April 13, 2011.
Measuring Disaster Preparedness: FEMA Has Made Limited Progress in
Assessing National Capabilities. [hyperlink,
http://www.gao.gov/products/GAO-11-260T]. Washington, D.C.: March 17,
2011.
Biosurveillance: Efforts to Develop a National BioSurveillance
Capability Need a National Strategy and a Designated Leader.
[hyperlink, http://www.gao.gov/products/GAO-10-645]. Washington, D.C.:
June 30, 2010.
National Security: Key Challenges and Solutions to Strengthen
Interagency Collaboration. [hyperlink, http://www.gao.gov/products/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.
[hyperlink, http://www.gao.gov/products/GAO-10-204]. Washington, D.C.:
January 29, 2010.
Homeland Defense: Planning, Resourcing, and Training Issues Challenge
DOD's Response to Domestic Chemical, Biological, Radiological, Nuclear,
and High-Yield Explosive Incidents. [hyperlink,
http://www.gao.gov/products/GAO-10-123]. Washington, D.C.: October 7,
2009.
Interagency Collaboration: Key Issues for Congressional Oversight of
National Security Strategies, Organizations, Workforce, and Information
Sharing. [hyperlink, http://www.gao.gov/products/GAO-09-904SP].
Washington, D.C.: September 25, 2009.
Project BioShield Act: HHS Has Supported Development, Procurement, and
Emergency Use of Medical Countermeasures to Address Health Threats.
[hyperlink, http://www.gao.gov/products/GAO-09-878R]. Washington, D.C.:
July 24, 2009.
Project BioShield: HHS Can Improve Agency Internal Controls for Its New
Contracting Authorities. [hyperlink, http://www.gao.gov/products/GAO-09-
820]. Washington, D.C.: July 21, 2009.
Emergency Management: Observations on DHS's Preparedness for
Catastrophic Disasters. [hyperlink, http://www.gao.gov/products/GAO-08-
868T]. Washington, D.C.: June 11, 2008.
Project BioShield: Actions Needed to Avoid Repeating Past Problems with
Procuring New Anthrax Vaccine and Managing the Stockpile of Licensed
Vaccine. [hyperlink, http://www.gao.gov/products/GAO-08-88].
Washington, D.C.: October 23, 2007.
Results-Oriented Government: Practices That Can Help Enhance and
Sustain Collaboration among Federal Agencies. [hyperlink,
http://www.gao.gov/products/GAO-06-15]. Washington, D.C.: October 21,
2005.
[End of section]
Footnotes:
[1] White House, National Strategy for Homeland Security (Washington,
D.C.: October 2007).
[2] White House, National Security Strategy (Washington, D.C.: May
2010).
[3] The Project BioShield Act of 2004 authorized the appropriation of
funds for the Special Reserve Fund for the acquisition of certain CBRN
medical countermeasures using the authorities granted under that law. 6
U.S.C. § 321j. Prior to enactment of this law, the Department of
Homeland Security appropriations act for fiscal year 2004 had
appropriated the amount of the Special Reserve Fund. Pub. L. No. 108-
90, 117 Stat. 1137, 1148 (2003).
[4] From 2004 to 2006, the Executive Office of the President led
interagency coordination efforts to establish CBRN medical
countermeasure requirements.
[5] See House Committee on Homeland Security, Subcommittee on Emergency
Preparedness, Response, and Communication, Taking Measure of
Countermeasures (Part 1): A Review of Government and Industry Efforts
to Protect the Homeland through Accelerated Research, Development, and
Acquisition of Chemical, Biological, Radiological and Nuclear Medical
Countermeasures, 112TH Cong., 1ST sess., 2011; Senate Committee on
Appropriations, Subcommittee on Labor, Health and Human Services, and
Education, and Related Agencies, Defending Against Public Health
Threats, 111TH Cong., 2ND sess., 2010; Senate Committee on Homeland
Security and Governmental Affairs, Six Years after Anthrax: Are We
Better Prepared to Respond to Bioterrorism? 110TH Cong., 1ST sess.,
2007; and House Committee on Homeland Security, Subcommittee on
Emerging Threats, Cybersecurity, and Science and Technology, Can
BioShield Effectively Procure Medical Countermeasures That Safeguard
the Nation? 110TH Cong., 1ST sess., 2007.
[6] See Department of Health and Human Services, Assistant Secretary
for Preparedness and Response, The Public Health Emergency Medical
Countermeasures Enterprise Review: Transforming the Enterprise to Meet
Long-Range National Needs (Washington, D.C.: August 2010).
[7] For the purposes of this report, we consider CBRN risk assessments
to include DHS's terrorism risk assessments (TRA) and material threat
assessments (MTA) and HHS's public health and medical consequence
modeling reports. TRAs assess the risks posed by CBRN agents based on
variable threats, vulnerabilities, and consequences. MTAs assess the
threat posed by given CBRN agents and the potential number of human
exposures in plausible high-consequence scenarios. Modeling reports
assess the public health and medical consequences of attacks with CBRN
agents for given scenarios.
[8] For the purposes of this report, we used the term "invested" to
mean obligated.
[9] See HHS, The Public Health Emergency Medical Countermeasures
Enterprise Review. We did not report on recommendations from this
report that related solely to pandemic influenza. We have previously
examined how HHS intends to address some of its recommendations in the
review related to pandemic influenza vaccine. See GAO, Influenza
Pandemic: Lessons Learned from the H1N1 Pandemic Should be Incorporated
into Future Planning, [hyperlink, http://www.gao.gov/products/GAO-11-
632] (Washington, D.C.: June 27, 2011); and Influenza Vaccine: Federal
Investments in Alternative Technologies and Challenges to Development
and Licensure, [hyperlink, http://www.gao.gov/products/GAO-11-435]
(Washington, D.C.: June 27, 2011).
[10] See GAO, Standards for Internal Control in the Federal Government,
[hyperlink, http://www.gao.gov/products/GAO/AIMD-00-21.3.1]
(Washington, D.C.: November 1999); and Project Management Institute,
The Standard for Program Management, 2nd ed. (Newton Square, Pa.:
2008).
[11] These practices require that components of an organization's
management provide reasonable assurance that certain objectives,
including effectiveness and efficiency of operations, are being
achieved. See GAO, Results-Oriented Government: Practices That Can Help
Enhance and Sustain Collaboration among Federal Agencies, [hyperlink,
hyperlink, http://www.gao.gov/products/GAO-06-15] (Washington, D.C.:
October 21, 2005), and [hyperlink, http://www.gao.gov/products/GAO/AIMD-
00-21.3.1].
[12] 6 U.S.C. § 321j. Prior to enactment of this law, the Department of
Homeland Security appropriations act for fiscal year 2004 had
appropriated the amount of the Special Reserve Fund. Pub. L. No. 108-
90, 117 Stat. 1137, 1148 (2003). In 2009, the Consolidated
Appropriations Act for fiscal year 2010 transferred the remaining
balance of the fund to HHS. Pub. L. No. 111-117, 123 Stat. 3034, 3255
(2009).
[13] 42 U.S.C. §§ 247d-6b(c)(1), (4). The Project BioShield Act also
authorizes the federal government to use specific contracting
authorities to procure certain medical countermeasures for CBRN agents
and requires HHS to report on its use of these contracting authorities
and procurements using the Special Reserve Fund. 42 U.S.C. §§ 247d-
6b(c)(7), 247d-6c.
[14] The Special Reserve Fund may be used to acquire medical
countermeasures that are reasonably expected to qualify for FDA
approval or licensure within 8 years. 42 U.S.C. § 247d-
6b(c)(1)(B)(i)(III). Under federal law and FDA regulations, vaccines
and other biologics are "licensed," drugs are "approved," and devices
may either be "approved" or "cleared." See 42 U.S.C. § 262, 21 U.S.C. §
355, 21 U.S.C. §§ 360e, 360(k). For this report, we use the term
"approve" to refer to both approval and clearance.
[15] 21 U.S.C. § 360bbb-3.
[16] In addition to these responsibilities, PHEMCE is also responsible
for providing recommendations on countermeasures for pandemic influenza
and other emerging infectious diseases. PHEMCE is composed primarily of
officials from HHS's ASPR, CDC, FDA, and NIH, which have specific
responsibilities for countermeasure development and acquisition. PHEMCE
also includes officials from other federal departments and offices,
such as DHS, DOD, the Department of Veterans Affairs, the Department of
Agriculture, and the Executive Office of the President.
[17] 42 U.S.C. § 247d-6b(a)(1).
[18] 42 U.S.C. § 247d-7e. The act also gave BARDA the authority to make
advance and milestone-based payments to vendors prior to product
delivery to the SNS. 42 U.S.C. § 247d-7e(c)(5)(C),(D).
[19] 42 U.S.C. § 247d-7f. Additionally, the National Biodefense Science
Board, includes nonvoting members, as deemed appropriate by the
Secretary.
[20] According to the DHS Risk Lexicon, threats are entities, actions,
or occurrences, whether natural or man-made, that have or indicate the
potential to harm life, information, operations and/or property;
vulnerabilities are physical features or operational attributes that
render an entity, asset, system, network, or geographic area
susceptible or exposed to hazards; and consequences are potential or
actual effects of an event, incident, or occurrence. Department of
Homeland Security (DHS), DHS Risk Lexicon: 2010 Edition (Washington,
D.C.: September 2010).
[21] See GAO, Public Health Preparedness: Developing and Acquiring
Medical Countermeasures Against Chemical, Biological, Radiological, and
Nuclear Agents. [hyperlink, http://www.gao.gov/products/GAO-11-567T]
(Washington, D.C.: April 13, 2011).
[22] 42 U.S.C. § 247d-6b(c)(2)(B).
[23] In addition to approving or licensing medical countermeasures, FDA
works with researchers throughout the development stages to review
safety and effectiveness test results and provide technical assistance
to help ensure that research meets FDA's regulatory requirements.
[24] Early, or basic, research seeks 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
physiologic 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.
[25] In the advanced development stage, potential medical
countermeasures are further evaluated to demonstrate safety and
effectiveness 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.
[26] The Project BioShield Act of 2004 provides that the Special
Reserve Fund may be used to acquire countermeasures for which the HHS
Secretary determines the scientific research supports a reasonable
conclusion that the product will qualify for FDA approval or licensing
within 8 years. 42 U.S.C. § 247d-6b(c)(1)(B)(i)(III)(bb).
[27] HHS officials told us that the two departments are working
together to discuss how DHS may be able to make the TRAs more useful
for HHS's purposes. For example, HHS officials said they were working
to understand the data inputs and outputs of the integrated CBRN TRA
and the biological TRA to understand how DHS determines relative risk
among the agents in the TRAs and use that information to help determine
needed quantities and types of medical countermeasures for specific
CBRN agents.
[28] For the purposes of this report, we used the term "invested" to
mean obligated.
[29] NIH investment amounts may over-or underestimate NIH's actual
investments in CBRN countermeasure research and development. NIH
officials told us that some research projects may have broader
applications than for CBRN countermeasures. For example, vaccine
research may be applicable to CBRN agents and to other diseases. In
addition, other NIH research not included in the agency's CBRN-specific
investments may be applicable to countermeasure development, such as
cancer research that may help inform research on countermeasures for
exposure to radiological and nuclear agents.
[30] NIH does not break out its investments in research and development
of radiological, nuclear, and chemical agents by type of
countermeasure.
[31] In addition to NIH investments in the specific countermeasures HHS
identified as priorities based on the MTAs and the modeling reports,
NIH has also invested in research related to other countermeasures for
the 14 agents in the 2007 PHEMCE plan. For example, HHS's only priority
countermeasure identified in the plan to address botulinum toxin was
for diagnostics, but NIH also invested in research on countermeasures
to treat the effects of the toxin on affected individuals. NIH's
investments also include research on other potential public health
threats, including foodborne diseases such as salmonella, emerging
infectious diseases such as severe acute respiratory syndrome, and
other agents such as ricin toxin.
[32] BARDA investments in anthrax vaccine for fiscal years 2008 through
2010 included investments for two types of anthrax vaccine and portable
ventilators to treat inhalation anthrax.
[33] In May 2011, BARDA invested $433 million of the Project BioShield
Special Reserve Fund to acquire 1.7 million doses of smallpox
antivirals for delivery to the SNS within 5 years.
[34] From fiscal year 2004 through fiscal year 2006, HHS spent
approximately $1 billion of the Special Reserve Fund to acquire
countermeasures such as botulism antitoxin, countermeasures to combat
effects of radiation exposure, and other anthrax countermeasures.
[35] We previously reported on the status of HHS's medical
countermeasure acquisitions with the Project BioShield Special Reserve
Fund. See GAO, Project BioShield Act: HHS Has Supported Development,
Procurement, and Emergency Use of Medical Countermeasures to Address
Health Threats, [hyperlink, http://www.gao.gov/products/GAO-09-878R]
(Washington, D.C.: July 24, 2009).
[36] According to FDA, rapid, point-of-care diagnostics would help
guide the public health response to a CBRN incident and ensure that
patients receive the most appropriate treatment.
[37] According to Homeland Security Presidential Directive 18, Medical
Countermeasures Against Weapons of Mass Destruction, enhanced agents
are traditional biological agents that have been modified or selected
to enhance their ability to harm human populations or circumvent
current countermeasures, such as bacteria that has been modified to
resist antibiotic treatment. Emerging agents are previously
unrecognized pathogens that might be naturally occurring and present a
serious risk to human populations, such as the virus responsible for
severe acute respiratory syndrome. Advanced agents are novel pathogens
or other materials of biological nature that have been artificially
engineered in the laboratory to bypass traditional countermeasures or
produce a more severe or otherwise enhanced spectrum of disease.
[38] The Department of Homeland Security appropriations act for fiscal
year 2004 provided that no more than $3.4 billion of the total
appropriation of approximately $5.6 billion for the Special Reserve
Fund could be obligated through fiscal year 2008, with the remainder of
the funds available for obligation from fiscal year 2009 through fiscal
year 2013. Pub. L. No. 108-90, 117 Stat. 1137, 1148 (2003).
[39] Since BARDA was established in 2006, appropriations acts have
transferred $1.3 billion from the Special Reserve Fund for CBRN
countermeasure basic research and advanced development, $995 million of
which has been used to fund BARDA and its advanced development
contracts.
[40] Congress is currently in the process of determining the future of
the Special Reserve Fund. H.R. 2405, introduced June 28, 2011, would
authorize the appropriation of $2.8 billion for the Special Reserve
Fund and BARDA advanced research and development investments for fiscal
year 2014 through fiscal year 2018. No more than 30 percent of the
amounts authorized to be appropriated could be used for BARDA advanced
research and development. As of September 2011, the Senate has not
introduced any legislation pertaining to the fund. In May 2011, the
Senate Committee on Health, Employment, Labor, and Pensions held a
hearing on medical and public health preparedness, in which
reauthorizing the Special Reserve Fund was discussed.
[41] Since 2007, DHS has issued six terrorism risk assessments (TRA).
DHS issued biological TRAs in 2008 and 2010, chemical TRAs in 2008 and
2010, and integrated CBRN TRAs in 2008 and 2011. While DHS has issued
TRAs biennially in the past, the department plans to issue them
quadrennially in the future. For example, DHS plans to issue the next
biological and chemical TRAs in 2014 and the next integrated CBRN TRA
in 2015.
[42] Under the requirements of the Pandemic and All-Hazards
Preparedness Act, HHS is to conduct annual reviews of the SNS. HHS's
first review of the SNS encompassed the years 2007 and 2008. HHS's
subsequent reviews for 2009 and 2010 were finalized in 2011.
[43] See Institute of Medicine, The Public Health Emergency Medical
Countermeasures Enterprise: Innovative Strategies to Enhance Products
from Discovery Through Approval (Washington, D.C.: 2010).
[44] Requests for proposals announce that HHS would like to award
contracts to meet specific needs, such as for the development of
certain quantities and formulations of a particular CBRN medical
countermeasure or a particular development or manufacturing capability.
[45] See National Biodefense Science Board, Where Are the
Countermeasures? Protecting America's Health from CBRN Threats
(Washington, D.C.: March 2010).
[46] The National Biodefense Science Board, recommendation further
stated that at least one of these three countermeasures should address
radiation exposure.
[47] See Institute of Medicine, The Public Health Emergency Medical
Countermeasures Enterprise: Innovative Strategies to Enhance Products
from Discovery Through Approval.
[48] We examined those recommendations that were specific to CBRN
countermeasure development and acquisition, and excluded those related
only to pandemic influenza.
[49] We have previously reported on challenges with regulatory
processes for evaluating promising medical countermeasures. In addition
to challenges in proving a countermeasure's effectiveness using animals
as proxies for humans, other challenges include (1) determining
appropriate doses of countermeasures for children, who may be more
vulnerable to exposure to CBRN agents, and (2) evaluating the safety
and effectiveness of medical countermeasures for use in a public health
emergency if they have not yet been FDA-approved or licensed. See
[hyperlink, http://www.gao.gov/products/GAO-11-567T].
[50] These departments include DHS, DOD, the Department of Agriculture,
and the Department of Veterans Affairs. The ESC's predecessor included
only HHS agencies among its voting members.
[51] [hyperlink, http://www.gao.gov/products/GAO/AIMD-00-21.3.1].
[52] Project Management Institute, The Standard for Program Management.
[53] [hyperlink, http://www.gao.gov/products/GAO-06-15] and [hyperlink,
http://www.gao.gov/products/GAO/AIMD-00-21.3.1].
[54] See GAO, National Preparedness: DHS and HHS Can Further Strengthen
Coordination for Chemical, Biological, Radiological, and Nuclear Risk
Assessments, [hyperlink, http://www.gao.gov/products/GAO-11-606]
(Washington, D.C.: June 21, 2011).
[55] [hyperlink, http://www.gao.gov/products/GAO-06-15] and [hyperlink,
http://www.gao.gov/products/GAO/AIMD-00-21.3.1].
[56] See National Biodefense Science Board, Where Are the
Countermeasures? Protecting America's Health from CBRN Threats.
[57] See Institute of Medicine, The Public Health Emergency Medical
Countermeasures Enterprise: Innovative Strategies to Enhance Products
from Discovery Through Approval.
[58] For the purposes of this report, we used the term "invested" to
mean obligated.
[59] See Department of Health and Human Services, Assistant Secretary
for Preparedness and Response, HHS Public Health Emergency Medical
Countermeasure Enterprise Implementation Plan for Chemical, Biological,
Radiological and Nuclear Threats (Washington, D.C.: April 2007).
[60] PHEMCE is composed primarily of officials from HHS's Office of the
Assistant Secretary for Preparedness and Response (ASPR), the
Biomedical Advanced Research and Development Authority (BARDA), the
Centers for Disease Control and Prevention (CDC), the Food and Drug
Administration (FDA), and the National Institutes of Health (NIH). In
addition, PHEMCE includes officials from the Department of Homeland
Security, the Department of Defense, the Department of Veterans
Affairs, the Department of Agriculture, and the Executive Office of the
President.
[61] See Department of Health and Human Services, Assistant Secretary
for Preparedness and Response, The Public Health Emergency Medical
Countermeasures Enterprise Review: Transforming the Enterprise to Meet
Long-Range National Needs (Washington, D.C.: August 2010). We examined
only those recommendations that were specific to CBRN countermeasures
and not those for pandemic influenza.
[62] Examples include the Enterprise Senior Council, the Enterprise
Executive Committee, the Integrated Program Teams, and the Project
Coordination Teams, among others.
[63] Examples include the Filovirus Animal Non-Clinical Group and the
Inter-agency Diagnostics Working Group.
[End of section]
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