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Introduction

Over the past 18 months, federal, state, and local governments; private developers, manufacturers, and distributors; and responders in both the public and private health care sectors together with multisector essential workers forged new pathways of coordination under fire while mounting a nationwide pandemic response to COVID-19. Even with these heroic personnel and coordinated organizational efforts, the pandemic revealed significant gaps in our national public health emergency (PHE) preparedness—lack of overall coordination and willingness to leverage all resources hindered efforts. The devastating effects on population health, the economy, and, by extension, national security, create compelling motivations to re-examine the nation’s approach to future PHE preparedness and response.

Effective national PHE preparedness and response requires the capability to produce, procure, and use medical countermeasures (MCMs)¹ and, given the inevitable complexity, a highly effective coordinating mechanism among the core federal agencies and an array of nonfederal partners. The Public Health Emergency Medical Countermeasures Enterprise (PHEMCE) was created to meet that coordination need and protect the nation from health security threats with a focus on MCM.

Over its nearly two-decade history, PHEMCE has changed substantially, in both structure and function. COVID-19 experiences suggest the

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¹ MCMs are “biologics, drugs, or devices that can be used to diagnose, prevent, protect from, or treat conditions associated with chemical, biological, radiological, or nuclear (CBRN) threats, or emerging infectious diseases and, MCMs may be used in the event of a potential public health emergency stemming from a terrorist attack with a biological, chemical, or radiological/nuclear material, or a naturally occurring emerging disease” (FDA, n.d.).

time is right to revisit its structure, operating principles, and practices and assess how PHEMCE is serving the mission to prepare the nation. This report was commissioned to provide guidance about that re-envisioning.

RATIONALE AND STUDY CHARGE

In light of the catastrophic experience of the COVID-19 pandemic, the committee looks to a future in which PHEMCE is a critical coordination node for all-of-government MCM preparedness and response infrastructure in a malicious or natural PHE. The purpose of this report is to help ensure that the United States has an effective and efficient coordinating mechanism to anticipate health security threats and, to the extent possible, be prepared to limit their impact with the appropriate MCMs.

In recognition of the important role of PHEMCE in coordinating the federal MCM preparedness and response and the platform for action that historical PHEMCE activities provided for the COVID-19 MCM response, the U.S. Department of Health and Human Services’ (HHS’s) Office of the Assistant Secretary for Preparedness and Response (ASPR) charged this ad hoc committee of the National Academies of Sciences, Engineering, and Medicine (the National Academies) with conducting a study to evaluate existing policy and practices of PHEMCE in order to enhance its ability to deliver on its critical mission (see Box 1-1). As requested, this report provides high-level strategic guidance to the ASPR on PHEMCE programs and activities.

The committee comprised 15 members with academic backgrounds and professional expertise in fields including medical and PHE preparedness and response; MCM research and development (R&D); chemical, biological, radiological and nuclear (CBRN) threats; biosecurity and bio-defense; legal and regulatory issues; supply chains; and decision science and organizational management. Appendix B provides the biographies of the committee members.

LOOKING BACK TO LOOK FORWARD: HISTORICAL PERSPECTIVES OF PHEMCE

PHEMCE’s functions are fully summarized in 42 U.S. Code 300hh-10a. Its responsibilities include making recommendations to the HHS secretary regarding advanced research, development, procurement, stockpiling, deployment, distribution, and use of MCM; identifying national health security needs, including gaps in public health preparedness and response related to MCMs and challenges to addressing such needs; and assisting the secretary in developing strategies related to logistics, deployment, distribution, dispensing, and use of MCMs that may be applicable to the activities of the U.S. Strategic National Stockpile (SNS).

PHEMCE is administratively managed by the PHEMCE Branch within ASPR. The PHEMCE Branch is located within the Division of Requirements, within the Office of Strategy, Policy, Planning, and Requirements, which is led by a deputy assistant secretary. According to the current ASPR, the goal of this structure is to ensure that PHEMCE meetings are held regularly, statutory mandates are met, and ASPR deliverables, such as the PHEMCE Strategy and Implementation Plan (SIP) and SNS Annual Review, are completed. The PHEMCE Branch is deliberately managed outside of ASPR program authorities to ensure a separation of policy and program determinations and reduce potential inappropriate influence by program staff on policy decisions. PHEMCE was managed by the Division of Requirements from its inception in 2005–2006 until 2017, when it was reassigned to the Biomedical Advanced Research and Development Authority (BARDA) and the ASPR Immediate Office. In 2020, the PHEMCE Branch was reestablished within the Division of Requirements

to ensure that an organizational unit was dedicated to implementing its statutory requirements.

PHEMCE as it exists today developed over time. Four eras of change that shaped PHEMCE are reviewed in the next section. The first era (2001–2008) is represented in early foundations, before it was named and detailed in legislation. The second era (2009–2016) was shaped by a restructuring following the experience of H1N1. The third era follows the restructuring in 2017. The fourth era outlines the role of PHEMCE both during and moving forward from the COVID-19 pandemic. This report is intended to help refine the role and functioning of PHEMCE going forward.

Foundational Structures and Policy (2001–2008)

Prior to the formal establishment of PHEMCE, the first era in this overview, the government entity tasked with providing advice for planning, coordination, and acquisition of MCM was the interagency Weapons of Mass Destruction Medical Countermeasures (WMD MCM) Subcommittee. The WMD MCM was convened by the White House in response to the terror attacks of September 11, 2001. A subcommittee of the Office of Science and Technology Policy’s National Science and Technology Council, it was led by HHS, in consultation with senior leadership from the U.S. Department of Homeland Security (DHS) and the U.S. Department of Defense (DoD). WMD MCM focused on questions of which MCMs should be prioritized in response to the threats related to WMD, submitting its recommendations through the Office of Management and Budget (OMB). Once approved by OMB, HHS would then manage the MCM procurement process by issuing Requests for Proposals (Russell, 2007).

The Project BioShield Act in 2004 connected threat assessment, led by DHS, with acquisitions, led by HHS. It established a 10-year, $5.6 billion reserve fund for government purchase of MCMs.²

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² From a 2005 congressional committee hearing, “To date, the Secretary of DHS has issued material threat determinations for four agents: anthrax, smallpox, botulinum antitoxin and radiological/nuclear devices. In addition, threat and risk assessments are currently under way and will be completed this year for plague, tularemia, radiological devices, and chemical nerve agents; and a threat assessment for viral hemorrhagic fevers will be issued next month in August. Once a material threat determination has been issued, HHS assesses the potential public health consequences of the identified agent, determines the needs for countermeasures, evaluates the availability of current countermeasures and the possibility of development of new countermeasures. They are assisted by the interagency Weapons of Mass Destruction Countermeasures Subcommittee. Any recommendations issued for the acquisition of a specific countermeasure are evaluated through the interagency process that forms the basis of U.S. government requirements. After approval of these requirements by the Office of Management and Budget, HHS issues a request for proposals and implements and manages the subsequent acquisition process through the delivery of countermeasures through the strategic national stockpile. Throughout this process DHS works very closely with HHS. HHS subject-matter experts participate in threat assessments and risk assessments. HHS, DHS, and Department of Defense co-chair the WMD Medical Countermeasures Subcommittee; and HHS keeps DHS informed about the subsequent acquisition processes. These interactions occur at multiple levels, from formal interagency committees through bilateral management interactions to informal but important contact and collaborations amongst the working scientists” (U.S. Congress, 2005).

The 2006 Pandemic and All-Hazards Preparedness Act (PAHPA) established the ASPR in HHS and provided authority for HHS to create BARDA and an early version of the SNS (Biocom, n.d.). The 2007 Homeland Security Presidential Directive/HSPD-18 further specified the PAHPA goals, including an integrated approach to what the administration referred to as “WMD medical countermeasure development” and seamless integration of public and private partners throughout the product development life cycle (Bush, 2007).

Restructuring PHEMCE Following the 2009 H1N1 Experience (2009–2016)

The second era was structured by the 2009 H1N1 experience, which prompted re-envisioning the MCM enterprise. This effort, driven by the president himself, was completed in 2010 (ASPR, 2010a). A capabilities approach governed the new PHEMCE design, stating the vision of a “nimble, flexible capability to produce MCMs rapidly in the face of any attack or threat, known or unknown, including a novel, previously unrecognized naturally occurring emerging infection disease” (ASPR, 2010a, p. 6). The mission included threats beyond WMD, with a focus on infectious disease. Specific goals in the 2010 report included supporting partnerships, removing constraints, modernizing regulatory oversight, and encouraging transformative technologies (ASPR, 2010b). As a result, PHEMCE operated according to the structures briefly described next and depicted in Figure 1-1 (ASPR, 2015).

As shown in Figure 1-1, the Enterprise Senior Council (ESC), an interagency group of top agency leaders, such as the ASPR, the Centers for Disease Control and Prevention (CDC) director, and the U.S. Food and Drug Administration (FDA) commissioner, provided coordinated, strategic direction and policy oversight for HHS’s “end-to-end” MCM preparedness activities. The Enterprise Executive Committee (EEC) reported to the ESC, providing operational-level decision making and coordination for all components of PHEMCE. Components reporting to the EEC included topic-specific Integrated Program Teams (IPTs) and the Flu Risk Management Meeting; Project Coordination Teams focused on acquisition and established as needed by the BARDA director; the Emerging Infection Diseases Working Group; the Public Readiness and Emergency Preparedness (PREP) Act Working Group, which was established by the ESC in 2012; and the

Requirements Working Group that addressed a core mission component of PHEMCE that is outlined in the next sections.

Stakeholder Engagement Structure

Figure 1-2 is a graphical representation of PHEMCE stakeholder engagement structure from this era, depicting mission components in yellow, federal agencies in blue (within HHS) and green (outside of HHS), and nonfederal stakeholders in green.

The eight mission components were (1) requirements setting, led by ASPR in consultation with DHS, described in more detail later; (2) early stage research, led by the National Institutes of Health (NIH) in consultation with DoD and the U.S. Department of Agriculture (USDA); (3) advanced development/manufacturing, led by ASPR/BARDA in consultation with DoD and USDA; (4) regulatory science management, led by FDA; (5) procurement/inventory/management/stockpiling, led by CDC for procuring and maintaining commercially available MCM for the SNS and BARDA for procuring certain MCM using the Special Reserve Fund, while DoD and the U.S. Department of Veterans Affairs (VA) support planning or executing stockpiling; (6) response planning, policy, guidance, and communication, led by CDC and ASPR in consultation with DoD, DHS, and the VA; (7) deployment/distribution/dispensing/administration, led by CDC

and ASPR in consultation with the VA; and (8) monitoring/evaluation/assessment, led by CDC and FDA (ASPR, 2007, 2008, 2011).

The specific engagement paths for nonfederal agencies are not specified in Figure 1-2, but the external stakeholders identified as PHEMCE partners include industry, academia, professional societies, state, local, tribal, and territorial entities, and the American public.

Requirements Setting: One Example of a Critical Mission Component in Greater Depth

The requirements setting process was a major focus of PHEMCE’s work during this era and emerged multiple times in the committee’s discussions. Based on a DHS intelligence assessments, PHEMCE IPTs tabulated the need-based quantities of MCMs, identified solutions if that quantity was likely to be unavailable, and identified the acquisitions target based on available funds (ASPR, 2012, 2015). BARDA and partners, including CDC, used the results to prioritize HHS funding to deploy acquisition plans for the MCM. Figure 1-3 outlines this process (Korch, 2016). The SNS Review is a major part; it is the mechanism by which PHEMCE recommends a portfolio of holdings for the SNS, identifying threats and estimating the costs of meeting them. The review, like the PHEMCE SIP, is mandated by the 2013 Pandemic and All-Hazards Preparedness Reauthorization Act (PAHPRA) (ASPR, 2014).

The Second Restructuring of PHEMCE (2017–2019)

The third era is defined by another restructuring of PHEMCE, initiated in 2017 (ASPR, 2020). According to then-current documents, these changes

were undertaken based on goals such as expediting decision-making processes through a top-down approach and narrowing the scope of PHEMCE to MCM development and procurement.¹⁵ However, during this period, the required SNS annual reviews for 2017, 2018, and 2019 were not conducted, and the statutorily mandated SIP was not published after December 2017 (GAO, 2021a). Thus, the role of PHEMCE in federal preparedness and response MCM activities during this era is not clear.

• According to the ASPR Strategic Plan for 2020–2023, two aims drove the 2019 restructuring: (1) streamline the deliberative process to enhance speed and more robustly engage all relevant agencies, including DoD and (2) better protect national security–sensitive and proprietary information in PHEMCE deliberations (ASPR, 2020). Ten strategies were proposed, ranging from new efforts to “solicit priorities from nonfederal stakeholders regarding activities and priorities of the PHEMCE” and “develop improved business models for MCM development and acquisition to address the strategic risks posed by supply chain disruptions” (ASPR, 2020). The ASPR Strategic Plan for 2020–2023 also determined that all PHEMCE meetings “involving discussion of aggregated information that may present security risks would be held in secret or at a higher classified level” (ASPR, 2020).

COVID-19 and Operation Warp Speed (2020 and Beyond)

In the fourth era, beginning May 2020, the White House launched a new interagency coordinating body jointly led by HHS and DoD called Operation Warp Speed (OWS). The goal was to develop therapeutics and vaccines for COVID-19, with a specific target of 300 million doses of a safe and effective vaccine available by January 2021. A GAO report on OWS describes the strategies adopted by OWS leadership in partnership with vaccine companies as accelerating development and mitigating financial risk to producers (GAO, 2021b). During meetings with the committee, government leaders involved in OWS did not refer to PHEMCE. As the committee understands it, OWS became the de facto all-of-government MCM preparedness and response effort for COVID-19. The COVID-19 pandemic required unprecedented, urgent action; surge in federal contracting; and operational leadership with the authority to make urgent decisions. Over the years, PHEMCE developed valuable and deliberative processes, but COVID-19 demanded urgent and new capacity leadership paradigms, provided by OWS without involving the PHEMCE mechanism. The committee also heard testimony and commentary indicating OWS was built on two decades of PHEMCE activities and used its personnel, staff,

and many of its leaders. At the time of this writing, OWS is transitioning to a program known as the Countermeasures Acceleration Group (CAG). Integrating and/or coordinating CAG and PHEMCE will be key. These two entities must be complementary and neither competing nor redundant. An important question is whether ad hoc bodies, such as OWS/CAG, should be encouraged as separate entities or a plan to expand PHEMCE during emergencies would be more appropriate.

ABOUT THIS REPORT

Study Approach and Scope

This report offers a new vision for how PHEMCE can optimize the development and use of MCMs for future PHEs, serving as the consistent, resilient, and steadfast coordinating entity for MCMs that is critical to effective PHE preparedness and response. The following principles guided the committee’s deliberations and resulting recommendations.

• Meeting the Mandate. PHEMCE is the only coordinating body tasked with all-of-government MCM preparedness and response. It must be empowered with clear and powerful leadership. The metrics for meeting the mandate are to reduce mortality, morbidity, inequities, and costs in PHEs.
• Transparency. PHEMCE must make clear decisions that it can defend in compelling recommendations, conveyed to the White House through ASPR and HHS. The metrics for transparency are clear, ethical business practices, reinforcing its public legitimacy.
• Adaptability. PHEMCE must evolve as threats, vulnerabilities, and MCM options change. The metrics for adaptability are continuous two-way communication with all PHEMCE partners and with experts in threats, vulnerabilities, and response options.

The committee reviewed an array of earlier reports on PHEMCE and heard testimony from many individuals with varying experiences with the government’s efforts to organize and coordinate MCM preparedness and response. The committee’s analysis of this input clearly indicates a significant lack of clarity about PHEMCE’s mission, role, and authorities because, in practice, they have shifted substantially with circumstances such as PHEs and budgets and political leadership. It could be assumed from the establishing legislation (42 U.S. Code 300hh-10A) that PHEMCE’s mandate is to serve as the major coordinating body for all aspects of U.S. MCM preparedness and response. However, little evidence indicates that it has

functioned in this way for the last several years or more, in part because the relevant federal landscape has evolved substantially. Other entities, such as OWS, have at least partly supplanted PHEMCE’s coordinating function. This report aims to be forward looking and presents a high-level blueprint of how a group with the roles and responsibilities articulated in 42 U.S. Code 300hh-10A should function.

Report Audiences and Uses

This report is intended for the immediate use, by request, of ASPR and federal PHEMCE partners engaged in strengthening PHEMCE. The committee hopes that the report will find audiences among all PHEMCE stakeholders: the White House; congressional members; federal partners; state, tribal, local, and territorial governments; private-sector developers, manufacturers, and distributors; public health; and health care. The committee sees these partnerships as an enterprise working together to ensure MCM preparedness and response.

Organization of the Report

This first chapter reviewed PHEMCE’s intended role, finding that it is not currently fulfilling its mission, despite dedicated efforts by many participants. The next four chapters offer recommendations for addressing issues identified in different sectors, drawing on scientific research, members’ experience, and interactions with PHEMCE partners and participants. The committee believes that their adoption will strengthen PHEMCE’s ability to meet its mission and protect America from PHEs. Chapter 2 recommends general principles across four areas (ethics, operations, strategy, and budget) to guide PHEMCE decisions and policies. Chapter 3 recommends ways to enhance PHEMCE operations to produce defensible decisions and recommendations. Chapter 4 recommends ways to enhance communications and transparency needed to achieve public trust, emphasizing formal and informal collaboration with nongovernment partners and stakeholders. Chapter 5 recommends ways to work within and revise the legal framework for PHEMCE, drawing lessons from COVID-19.

The ongoing COVID-19 pandemic creates an urgent need to reinvent PHEMCE, in ways that will position it for future challenges. It echoes and elaborates on themes found in the concurrently (and independently) developed White House American Pandemic Preparedness: Transforming Our Capabilities. That White House plan strengthens the committee’s belief that an effective, consistent, and reliable lead coordinating body is critical to develop, produce, and deliver MCM and related support (e.g., supplies,

staffing) for PHEs. PHEMCE was designed to fill that role. The following chapters review steps to adapt PHEMCE functions to do so and better protect the nation.

CONCLUDING REMARKS

The stakes could not be higher. The risks from emerging infectious diseases pose a growing threat to population health. Intentional acts of bioterrorism remain. The White House American Pandemic Preparedness: Transforming Our Capabilities highlights the need for a federal, central “mission control” operation to lead PHE preparedness and response. PHEMCE was designed to serve and support that role. This report recommends ways for it to do so. The cost of improving the nation’s ability to respond to PHEs in the twenty-first century will be substantial, in both material resources and changes to current systems and practices. However, the cost of not making needed changes is incalculable, in terms of lives lost or irrevocably altered and economies and communities ravaged by the loss of jobs, homes, and educational opportunity.

REFERENCES

FDA (U.S. Food and Drug Administration). n.d. What are medical countermeasures? https://www.fda.gov/emergency-preparedness-and-response/about-mcmi/what-are-medical-countermeasures (accessed September 16, 2021).

GAO (U.S. Government Accountability Office). 2021a. Continued attention needed to enhance federal preparedness, response, service delivery, and program integrity. https://www.gao.gov/assets/gao-21-551.pdf (accessed September 1, 2021).

GAO. 2021b. Operation Warp Speed: Accelerated COVID-19 vaccine development status and efforts to address manufacturing challenges. https://www.gao.gov/assets/gao-21-319.pdf (accessed September 1, 2021).

Korch, G. W. 2016. Product life cycle management and the PHEMCE. https://int.nyt.com/data/documenttools/korch-presentation-mta/a30b4451c27577ba/full.pdf (accessed September 1, 2021).

Russell, P. K. 2007. Project Bioshield: What it is, why it is needed, and its accomplishments so far. Clinical Infectious Diseases 45(Suppl 1):S68–S72.

U.S. Congress, Committee on Homeland Security House of Representatives, Subcommittee on Emergency Preparedness, Science, and Technology. 2005. Project Bioshield: Linking bioterrorism threats and countermeasure procurement to enhance terrorism preparedness. https://www.govinfo.gov/content/pkg/CHRG-109hhrg27217/html/CHRG109hhrg27217.htm (accessed December 16, 2021).

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