Summary¹

The U.S. medical countermeasures (MCMs)² enterprise is interconnected, complex, and dynamic. It includes public and private entities that (1) develop and manufacture new and existing MCMs; (2) ensure procurement, storage, and distribution of MCMs; and (3) administer, monitor, and evaluate MCMs. The interagency group the Public Health Emergency Medical Countermeasures Enterprise (PHEMCE) is the nation’s sole coordinating body, based in the U.S. Department of Health and Human Services (HHS) with interagency participation and responsible for ensuring end-to-end MCM preparedness and response. PHEMCE’s authorities limit its membership to federal entities. However, it must also collaborate closely with nonfederal and private-sector partners and stakeholders, as they are the ultimate implementers of PHEMCE’s mission and develop, manufacture, distribute, and administer the MCMs over which PHEMCE has responsibility. These engagements must support the entire life cycle of MCM preparedness and response, where appropriate, including threat identification, development, manufacturing, deployment, distribution, administration, and evaluation. An effective national MCM enterprise requires a mechanism to coordinate and integrate the activities and expertise of

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¹ This Summary does not include references. Citations for the discussion presented in the Summary appear in the subsequent report chapters.

² 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.).

the diverse landscape of federal agencies involved in these activities, and PHEMCE has the legislative mandate to serve that role.

Recognizing the important role of PHEMCE in coordinating the federal MCM preparedness and response activities, HHS’s Office of the Assistant Secretary for Preparedness and Response (ASPR) charged the National Academies of Sciences, Engineering, and Medicine with convening an ad hoc committee to evaluate PHEMCE’s policy and practices and make recommendations for a re-envisioned PHEMCE. The committee heard from knowledgeable experts reflecting a range of disciplines and experiences, including developers, producers, funders, responders, and federal, state, and local officials (see Appendix A). The committee also examined government documents, including some first made public for this review.

Four priority areas of improvement emerged from committee deliberations: (1) articulating PHEMCE’s mission and role and explicating the principles guiding its operating principles and processes; (2) revising PHEMCE operations and processes; (3) collaborating more effectively with external public and private partners needed for the entire life cycle of MCMs, from research through use; and (4) navigating legal and policy issues. This report proposes recommendations for these four domains, re-envisioning PHEMCE to service its mandate to protect the nation in public health emergencies (PHEs).

HISTORICAL PERSPECTIVES ON PHEMCE: LOOKING BACK TO LOOK FORWARD

The 2019 42 U.S. Code 300hh-10a charges PHEMCE with advancing national preparedness for natural, accidental, and intentional threats by coordinating research, advanced research, development, procurement, stockpiling, deployment, distribution, and utilization with respect to MCMs within HHS and in cooperation with partners in other federal agencies. That mission has evolved in practice over the past two decades, from its initial focus of advising on MCMs for weapons of mass destruction, to an end-to-end MCM interagency body tasked with protecting the nation from all chemical, radiological, biological and nuclear (CBRN) threats and pandemics and emerging infectious diseases. PHEMCE has developed processes and procedures to help improve the coordination process, and it is now a congressionally authorized coordination body.

Chapter 1 provides a brief history on PHEMCE’s evolution through four distinct eras:

• Foundational Structures and Policy (2001–2008), a focus on WMD MCMs;

• Restructuring PHEMCE following the 2009 H1N1 experience (2009–2016), a redesign to a capabilities-based approach, with expanded authorities to deliver end-to-end MCM preparedness for unknown, novel or re-emerging, and natural or intentional threats;
• The second restructuring (2017–2019) streamlined decision making, enhanced national security by classifying its deliberations, and narrowed its scope; and
• COVID-19 and Operation Warp Speed (2020 and beyond), an additional interagency coordinating group launched to identify MCMs explicitly for COVID-19.

DELIVERING ON PHEMCE’S MISSION

Guiding Principles

These changes have left PHEMCE’s role in MCM preparedness and response unclear. Presentations to the committee expressed substantial confusion regarding its mission, scope, and responsibilities. The committee observed that the lack of clarity in PHEMCE’s authority would hinder its ability to carry out its mandate and work with its federal and external partners and the stakeholders depending on its success. If PHEMCE is to play the important roles it is intended for, it will need clear exposition of its of mission, scope, and responsibilities and an explicit affirmation to its mission, especially by both federal authorities and stakeholders that have may have undermined its function and authority in recent years. Chapter 2 proposes guiding principles for redesigning PHEMCE’s system. The committee advocates four sets of principles to guide PHEMCE decisions and policies:

• Ethics
  • Trust building: creating strong relations among PHEMCE member agencies and partners by linking plans to outcomes, promoting innovation, and respecting diverging opinions;
  • Fairness: ensuring that PHEMCE planning and response operations promote health equity and do not exacerbate existing health inequities related to race, ethnicity, income, gender, location, abilities, and age;
  • Transparency: making PHEMCE processes accessible, communicating with partners and stakeholders, and creating appropriate records, with explicit procedures for making and documenting exceptions for national security or intellectual property;

• Accountability: making PHEMCE mission-driven to solve problems innovatively and in collaboration with partners; performing regular and unbiased after-action reviews, incorporating lessons learned and improving quality of processes and outcomes; committing to processes that promote long-term public well-being, evidence-based and collaborative decisions, and protecting it from undue political or commercial influence; and
• Stewardship: demonstrating responsible management of resources, especially under conditions of scarcity of resources.
• Operations
  • Effectiveness
    • Evidence-based decisions: employing sound decision-making processes, based on the best evidence, while acknowledging uncertainty; and
    • Impact: delivering on all strategic objectives consistent with PHEMCE’s mission, from development through usage by affected populations;
  • Efficiency: carefully stewarding the resources for protecting public health;
  • Value: pursuing desired public health outcomes cost-effectively;
  • Adaptability: responding agilely to new opportunities and threats, based on long-term investments in capabilities;
  • A systems approach: coordinating activities across the life cycle of each MCM and across the portfolio, from development to delivery, including research, facilities, supply chains, and workforce;
  • Collaboration: creating effective mechanisms for coordination across federal agencies, private companies, and state or local entities; and
  • Metrics-driven: assessing performance with measures that allow external evaluation and internal learning.
• Strategic objectives
  • Aligning with PHEMCE’s mission, describing how the right products, but also people and services, will be delivered to the right population, at the right time; and
  • Articulating in the annual Strategy and Implementation Plan, as mandated by section 2811(d) of the Public Health Service Act (PHSA) and amended by section 102 of the Pandemic and All-Hazards Preparedness Reauthorization Act.

• Budget
  • Aligning across PHEMCE member agencies and the MCM life cycle through a multiyear budget plan, as mandated by section 2811(b)(7) of the PHSA, necessary to advance MCM preparedness.

ENSURING DEFENSIBLE PHEMCE RECOMMENDATIONS

Sound Business Practices in a Changing Environment

PHEMCE must develop and sustain the mutually supporting formal and informal processes required for effective business practices. Formal pro-

cedures provide stability and accountability; informal processes and interpersonal networks facilitate collaboration. The committee recognizes the challenges that PHEMCE faces in establishing formal and informal processes required for a predictable enterprise, including the need to do the following:

• Coordinate the work of multiple organizations.
• Accommodate personnel changes associated with political events or public health emergencies, including hiring subject-matter experts for any and all aspects of emergency operations.
• Shift priorities in response to emerging threats.
• Balance competing interests, including political, efficiency, and effectiveness concerns and the considerations of stakeholders.

Stabilizing an enterprise faced with these challenges will require defining PHEMCE priorities through a transparent process that avoids conflicts of interest (COIs) and then implementing those priorities with a robust quality management system, enhancing coordination, resource allocation, monitoring, and accountability across the enterprise with metrics-driven evaluations. Chapter 3 recommends a strategic approach and implementation methods.

Priority Setting, Threat Assessments, and Needs Assessments

PHEMCE priority setting must consider the probabilities and potential impact of threats and the expected return on MCM investments (in research, development, manufacturing, training, inventory, etc.). It is built around threats to national security identified by the U.S. Department of Homeland Security. In the past, PHEMCE has used a Preparedness Assessment Framework and probabilistic risk analysis for risk factors that can be quantified. PHEMCE may also look to procedures recently recommended by the National Academies’ Committee on Equitable Allocation of Vaccine for the Novel Coronavirus to set priorities, including

• Adopting widely acceptable ethical principles to maximize benefits, mitigate health inequities, and promote fairness, transparency, and evidence-based actions;
• Translating those factors into transparent operational metrics;
• Setting priorities in terms of those metrics;
• Using readily available data to estimate those metrics and monitor progress;
• Conducting sensitivity analyses to create contingency plans; and
• Establishing two-way dialogue with stakeholders to communicate and address concerns related to the priorities.

Priorities should be informed by assessments of threats, operational capacities, and MCM multifunctionality, considering moderating criteria such as population vulnerabilities, life cycle costs, and time needed to meet the stated goals/objectives and see return on investments. Clear definitions of priorities would be critical to focus on PHEMCE’s needs and give partners greater confidence in engaging with clarity of timeline and expected investment. This, in turn, would provide a stronger environment for strengthening the industrial base.

Evaluation and Accountability

Regular monitoring and evaluation is essential for measuring PHEMCE’s performance and ensuring continuous improvement. Evaluation could be conducted through periodic performance assessments with a metrics-driven approach. Performance may be assessed through audits, exercises, and after-action reviews. Simulation and tabletop exercises provide opportunities to stress test organizational relationships across MCM pathways. Whenever possible, simulations should involve realist emergency and low-probability scenarios with the organizations and individuals who will need to implement MCMs.

Metrics-driven evaluation requires establishing key performance indicators (KPIs) that are meaningful and representative of various outputs throughout the MCM life cycle. A robust data system is required to collect and analyze KPIs along with other indicators needed by the quality management system, with the flexibility to adapt to changing MCM preparedness or response requirements, while also continuously collecting and analyzing other types of data that are important for preparedness planning and response activities.

EXTERNAL PARTNERS AND THE PUBLIC HEALTH SUPPLY CHAIN SYSTEM

PHEMCE must sustain a high-performing, collaborative network involving nonfederal and private-sector partners and stakeholders. Maintaining such a responsive, multisectoral enterprise requires transparent, two-way communication within these partners; internal processes that facilitate sustainable collaborations, including cross-sectoral learning and collaboration; and end-to-end engagement through the public health supply chain system. Chapter 4 addresses these issues.

Incorporating Nonfederal and Private-Sector Partners and Stakeholders into PHEMCE Decision Making

PHEMCE must make decisions by incorporating the perspectives of diverse nonfederal and private-sector partners, stakeholders, and experts across the life cycles of MCM and PHEs. To avoid fragmentation that undermines the mission, PHEMCE needs an advisory committee with an encompassing view of its challenges, activities, and performance, for both existing and potential threats and options. PHEMCE leadership may consider, for example, a body similar to the Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices or the National Vaccine Advisory Committee (NVAC) for relevant experiences. Both bodies include scientific leaders and liaison partner and stakeholder organizations. Additional examples establish the precedent of an advisory committee. One is the Federal Emergency Management Agency’s National Advisory Committee, which focuses on decisions in the emergency preparedness and response space, similar to PHEMCE.

Transparency, Two-Way Communication, and Clarifying Expectations with Nonfederal and Private-Sector Partners, Stakeholders, and the Public

As the nation’s MCM coordinating body, PHEMCE must be a reliable partner in MCM priority setting, development, and distribution. To this end, PHEMCE must establish and maintain two-way communication across the enterprise, creating shared expectations and keeping the parties apprised of potential changes. PHEMCE must be transparent and trustworthy in fulfilling its obligations to its partners, stakeholders, and the public. Early, accurate, evidence-based communication fosters trust, preempts misinformation, and ensures appropriate expectations with the public, partners, and stakeholders. The advisory committee will have the overarching perspective needed to allow PHEMCE to speak with a clear voice even during disagreements and uncertainties.

PHEMCE must sustain two-way communication to clarify roles and expectations with nonfederal and private-sector partners and stakeholders. Clear, coordinated roles and authorities, with mutually accepted performance measures, are the foundation for effective collaboration. They may be formalized through contracting, advance commitments, pricing agreements, and incentives, designed for routine and crisis operations. Partnerships should be stress tested periodically through simulation exercises.

Public Health Supply Chains and Stockpiling Considerations

PHEMCE plays a critical governance, leadership, and coordination role in ensuring that the U.S. Strategic National Stockpile (SNS) meets the nation’s preparedness and response needs. Per the National Strategy for a Resilient Public Health Supply Chain, a re-envisioned PHEMCE must create appropriate incentives and commitments for private-sector engagement if it is to help promote a resilient and adaptable public health sup-

ply chain with strong business practices and effective coordination. These relationships should be designed to reveal vulnerabilities to the industrial base needed to scale MCM provisions in response to public health crises; the approaches and relationships may also build on best practices from emergency management.

Faithful completion of the mandated SNS Annual Review and the associated findings are necessary as a signal to PHEMCE and its member agencies of potential gaps in MCM preparedness and ways to improve stockpiling decisions for future emergencies. As noted in the 2021 U.S. Government Accountability Office (GAO) report, that annual SNS review has not been conducted since 2016, creating a major vulnerability in our nation’s ability to respond to the COVID-19 pandemic. It is urgently necessary to understand and address SNS failures through a root-cause assessment (e.g., including a lack of mandated annual review, investments without PHEMCE partner input or full transparency, and insufficient funding), understanding that this is just one of many after-action reports reports to be done regarding the COVID-19 response.

Global Considerations and Synergies

MCM preparedness and response is inherently international in nature and structure, involving a global network of resources, manufacturing, and distribution, therefore making it imperative that PHEMCE be engaged at the international level. U.S. efforts to improve MCM development and delivery must consider opportunities for global coordination, leveraging relationships with international governmental, nonprofit, and industry organizations that are recognized leaders in MCM preparedness. The global

impact of COVID-19 presents opportunities for lessons learned, challenges, opportunities, and barriers to be evaluated.

LEGAL AND POLICY ISSUES

Overview of the Federal Emergency Legal Landscape

PHEMCE is bound and empowered by federal and other laws and policies. Some of these laws are routine, standing provisions. Others respond to crises, enacted through presidential national security powers (e.g., executive orders, federal emergency declarations) or state-based emergency declarations. Federal law may override state law, as demonstrated for the COVID-19 pandemic in Public Readiness and Emergency Preparedness (PREP) Act declarations addressing local actions prohibiting MCM administration.

PHEMCE Regulatory Options

MCM development, authorization, procurement, and distribution exist within a complex regulatory landscape. The committee recognizes the regulatory challenges underlying several PHEMCE core functions:

• MCM research support and procurement: PHEMCE makes recommendations to ASPR on MCM research and procurement, while agency partners, such as the National Institutes of Health, the U.S. Food and Drug Administration (FDA), and the Biomedical Advanced Research and Development Authority, support biomedical research, regulatory science, and procurement of end products. Thus, PHEMCE must be aware of issues related to and, to the extent of its power, ensure research support; fair, documented, and efficient procurement processes; and adequate supply, while minimizing COIs and providing appropriate incentives to industry partners.
• MCM research regulation and FDA marketing authorization pathways: MCM development requires establishing and monitoring compliance with safety and effectiveness standards. During emergencies, legal flexibility and regulatory tools exist, and have been used, to accelerate research, evidence review, and authorization

processes while still helping to ensure sufficient information about safety and effectiveness.

• Legal concerns facing nongovernmental partners in MCM manufacturing and distribution: Nongovernmental partners face additional legal challenges related to MCM manufacturing and distribution, including concerns related to balancing the need to protect proprietary information against the need to collaborate on difficult scientific questions and ensuring adequate protections for intellectual property. Although HHS and PHEMCE partner agencies have some tools and programs intended to help, nongovernmental partners nevertheless may remain reticent about entering the MCM market if they do not perceive these programs as adequately addressing their concerns, particularly in light of high-profile bankruptcies in the space.

Addressing and Solving Emergency Legal Issues in Real Time

The legal and regulatory landscape underpinning PHEMCE has been evolving in response to PHEs such as COVID-19, which have presented novel legal questions and actual and perceived regulatory barriers. Substantial legal and policy changes have come about as a result, and additional legal adjustments in emergency preparedness and response are highly predictable. In response, PHEMCE agencies must triage legal principles and options to reduce uncertainty, mitigate barriers, and generate solutions to actual or perceived barriers. Regulatory readiness helps ensure awareness of legal flexibilities in PHEs among PHEMCE agencies.

CONCLUDING REMARKS

The COVID-19 pandemic has created an urgent need to re-envision PHEMCE, so that it can protect public health in the current emergency and those to come. PHEMCE plays a critical coordinating role in all-of-government MCM preparedness and response. The recommendations set forth in this report provide the high-level strategic guidance that ASPR, and all federal partners, need to ensure that PHEMCE fulfills its mission.