Biomedical Advanced Research and Development Authority (BARDA) Strategic Plan 2011-2016

2 BARDA Strategic Plan 2011-2016 Message from the BARDA Director Dear Colleagues, The nation has endeavored over the past ten years to become better prepared for the medical consequences of catastrophic events such as bioterrorist events and pandemic influenza. During that time period Project BioShield, The National Strategy for Pandemic Influenza, and the Biomedical Advanced Research and Development Authority have emerged to support the development and acquisition of medical countermeasures (MCMs) and the domestic manufacturing capacity to prepare for and respond to an emergency. Where very limited medical countermeasures were available in 2001, there are now vaccines, therapeutic drugs, and diagnostic devices for anthrax, smallpox, botulism, and pandemic influenza and a robust pipeline of products in development for these and other threats. While only one manufacturing site existed in the U.S. during the influenza vaccine shortage of 2004, now there are three with more on the way. Yet there is much more work to be done, and a clear path is needed to guide the way. Thus I am pleased to provide an update of the BARDA Strategic Plan for 2011-2016. The Plan describes our strategic goals and vision to accomplish BARDA’s mission over the next five years and beyond to develop and provide medical countermeasures for Chemical, Biological, Radiological, and Nuclear threats, pandemic influ­ enza, and emerging infectious diseases. The implementation measures to achieve these strategic goals include support of product advanced development, stockpile acquisition, manufacturing surge capacity infrastructure building, and product innovation. These medical countermeasures include vaccines, biological and small mol­ ecule therapeutics, antiviral drugs and antibiotics, diagnostics, respiratory devices, and other medical supplies. As BARDA completes its fifth year of existence, the results of our advanced development support are becoming evident with a robust product development pipeline for nearly all threats. Many of these products in the de­ velopment pipeline will become mature enough for acquisition and regulatory review over the next five years. Initiatives recommended by the 2010 Public Health Emergency Medical Countermeasure Enterprise Review are expected to enable more of the MCM products to become available as FDA-licensed approved or cleared products. BARDA will lead several of these initiatives including the establishment of Centers for Innovation in Advanced Development and Manufacturing and the Medical Countermeasures Strategic Investor. Greater emphasis on multi-purpose MCMs and more flexible and innovative manufacturing technologies will make im­ proved and greater quantities of MCMs available sooner. MCMs and manufacturing capabilities that afford the nation the ability to respond immediately to known and unknown threats will be high priorities. The BARDA Strategic Plan is a dynamic process with the steadfast aim to enhance national MCM preparedness and the flexibility to adopt better technologies, meet unforeseen challenges, and avoid unnecessary risks. MCM preparedness and response and this Plan are the results of the dedicated professionals at BARDA, ASPR, and across federal, State, local, and industry sectors. I wish to thank them for their tireless efforts to ensure that ours is “a nation prepared.” Respectfully, Robin A. Robinson, Ph.D., Director BARDA Strategic Plan 2011-2016 3 Introduction WITHIN THE LAST TEN YEARS, THE UNITED STATES HAS BARDA is a core component of the Office of the Assis­ experienced an intentional attack with a biological tant Secretary for Preparedness and Response (ASPR) agent in the form of the anthrax letters as well as natu­ and as such contributes to the broader ASPR mission to rally emerging outbreaks of high-consequence infec­ “Lead the country in preparing for, responding to, and tious diseases, including pandemic influenza and SARS. recovering from the adverse health effects of emer­ These events represented national security threats and gencies and disasters by supporting our communities’ public health emergencies. The past decade has also ability to withstand adversity, strengthening our health witnessed the dramatic spread of antimicrobial resis­ and response systems, and enhancing national health tance and community outbreaks of multidrug-resistant security.”1 bacteria that had previously been confined to hospitals. The first BARDA Strategic Plan for Medical Counter­ Concerns persist about the potential use of chemical, measure Research, Development, and Procurement biological, radiological and nuclear agents by terrorists. (Draft) was published in July 2007. The specific goals Effective medical countermeasures such as vaccines, of the 2007 Plan included aligning and coordinating antimicrobials, therapeutics, and diagnostics have the BARDA goals and programs with key pandemic influ­ potential to reduce the impact of such threats and are enza and CBRN strategy documents; developing an a bulwark of our national defense and public health HHS Public Health Emergency Medical Countermea­ emergency preparedness. sure Enterprise CBRN implementation plan; and es­ tablishing programs that promote innovation in MCM The mission of the Biomedical Advanced Research development. and Development Authority (BARDA) is to develop and procure medical countermeasures that address the The 2011 BARDA Strategic Plan builds on BARDA’s public health and medical consequences of chemical, successes in achieving those goals, defines BARDA’s vision, and lays out a series of capability goals over biological, radiological, and nuclear (CBRN) accidents, the next five years (2011-2016). This Plan reflects the incidents and attacks, pandemic influenza, and emerg­ dynamic evolution of BARDA’s mission and respon­ ing infectious diseases. sibilities, since its establishment in early 2007 and is Specifically, BARDA supports the advanced develop­ informed by the National Health Security Strategy ment and procurement of drugs, vaccines and other (NHSS; December 2009)2, The Public Health Emer­ gency Medical Countermeasures Enterprise Review: products that are considered priorities for national Transforming the Enterprise to Meet Long-Range Na­ health security. BARDA funding bridges the “valley of tional Needs (PHEMCE Review; August 2010)3, and the death” characterizing the late stages of product devel­ many lessons learned from the 2009 H1N1 pandemic opment. BARDA’s support ensures continuity of fund­ influenza response. ing at a critical point for medical countermeasures 1 HHS Assistant Secretary for Preparedness and Response Strategic developed by industry or emerging from the basic Plan 2011-2015. http://www.phe.gov/about/aspr/strategic-plan/ Pages/default.aspx research and preclinical development activities spon­ 2 National Health Security Strategy of the United States of America. sored by the National Institutes of Health (NIH). In pro­ U.S. Department of Health and Human Services. 2009. http:// www.phe.gov/Preparedness/planning/authority/nhss/strategy/ curing medical countermeasures for the Strategic Na­ Documents/nhss-final.pdf tional Stockpile, BARDA enhances the capabilities of 3 The Public Health Emergency Medical Countermeasures Enterprise Review: Transforming the Enterprise to Meet Long-Range National the Centers for Disease Control and Prevention (CDC) Needs. U.S. Department of Health and Human Services. 2010. to organize an effective response. h t t p s : / / w w w. m e d i c a l c o u n t e r m e a s u r e s . g ov / d o c u m e n t s / MCMReviewFinalcover-508.pdf

4 BARDA Strategic Plan 2011-2016 Who We Are And WhAt We do laboration with manufacturers, the NIH, the CDC, the FDA, and the Departments of Defense (DoD) and The Biomedical Advanced Research and Develop­ Homeland Security (DHS); supporting technology in­ ment Authority is the component of the Public Health novation through strategic initiatives; and overseeing Emergency Medical Countermeasures Enterprise that Project BioShield acquisitions. focuses on the advanced development, manufacturing and acquisition of medical countermeasures against BARDA is comprised currently of the Office of the Di­ chemical, biological, radiological and nuclear (CBRN) rector and seven functional divisions that oversee BAR­ threats, pandemic influenza, and emerging infectious DA’s programmatic activities. These divisions include diseases. BARDA’s staff includes experts in the fields of  Division of Chemical, Biological, Radiological physical, chemical and biological sciences, engineer­ and Nuclear (CBRN) Countermeasures ing, clinical medicine, public health, product devel­  Division of Influenza opment, regulatory affairs, and program management. The Pandemic and All-Hazards Preparedness Act  Division of Strategic Science and Technology (PAHPA) established BARDA in December 2006.  Division of Manufacturing, Facilities and BARDA oversees the Project BioShield program and Engineering Special Reserve Fund and is charged by statute with (1) promoting collaboration and communication be­  Division of Regulatory and Quality Affairs tween the U.S. Government and interested parties in  Division of Clinical Studies the advanced development and licensure of needed  Division of Modeling medical countermeasures; (2) directing and coordi­ nating the countermeasure and product advanced Additionally, to address issues relating to countermea­ research and development activities of the U.S. De­ sure development that cut across divisional boundar­ partment of Health and Human Services (HHS); (3) ies, BARDA operates collaboratively in “matrix teams” facilitating medical countermeasure development by in the areas of vaccines, biological therapeutics, small providing advice or directing interested parties to the molecule therapeutics, and diagnostics. These matrix relevant Centers of the U.S. Food and Drug Adminis­ teams help knit the BARDA organization together and tration (FDA); and (4) supporting innovation through facilitate sharing of information and best practices. strategic initiatives and investment in technologies BArdA’s Future and research tools that facilitate countermeasure de­ velopment. BARDA is committed to improving its workforce, in­ creasing the expertise and skills of BARDA personnel, PAHPA established a dual mandate for BARDA. BAR­ and helping to prepare the pharmaceutical industry DA is charged with coordinating the development of, to meet the challenges of the 21st Century. The in­ and bridging the “valley of death” for, MCMs deter­ vestment BARDA makes now in developing needed mined to be critical to the Nation’s health security. infrastructure, capabilities, processes, and human It is important to note that many such MCMs lack resources will pay off in the long run by enhancing meaningful commercial markets and without U.S. national preparedness to meet the public health threats Government support would be unlikely candidates of the future. for development. In supporting their development, BARDA fills the gap between NIH, which supports Continually looking forward, scanning the horizon for basic and preclinical research, and CDC, which de­ new technologies, and striving to develop a fast, flexible velops utilization plans and deploys MCMs during and efficient armamentarium of medical countermea­ public health emergencies. BARDA fulfills its mission sure capabilities and products will be key contribut­ by supporting advanced research and development ing factors to BARDA’s success in addressing medical of needed medical countermeasures; working in col­ countermeasure requirements for the nation. BARDA BARDA Strategic Plan 2011-2016 5 will address these current and evolving requirements, A number of themes cut across one or more of the aggressively seek out and support promising technol­ strategic goals. Cultivating public-private partnerships, ogy and platforms, and facilitate collaboration across for example, is a critical element in almost every as­ HHS and other Federal Departments and agencies pect of BARDA’s work. Developing tools and pro­ participating in medical countermeasure research cesses to facilitate systematic portfolio management and development while striving continually for great­ and program and project evaluation is an important er overall transparency. priority for BARDA, given BARDA’s broad mandate and foreseeable budgetary constraints. In pursuing its Context oF the BArdA mandate to develop medical countermeasures to pro­ strAtegiC PlAn tect civilian populations, BARDA must also address The BARDA strategic plan is guided by the priori­ the needs of vulnerable populations such as chil­ ties of the Administration and of ASPR and BARDA dren, pregnant women, persons with compromised leadership, and by other guidance including the U.S. immune systems, and the elderly. These and other Department of Health and Human Services Strategic cross-cutting issues have been considered as each Plan, governing legislation, Presidential Policy Direc­ one of the five strategic goals was developed and are tives, the 2009 NHSS, the 2010 PHEMCE Review, and reflected in the guiding principles. the 2011 HHS Assistant Secretary for Preparedness and Response Strategic Plan 2011-2015 (ASPR Strate­ iMPleMentAtion gic Plan). The NHSS establishes a framework for na­ BARDA will implement this strategic plan with full in­ tional efforts, to promote health security and encom­ volvement of its leadership and a firm commitment passes BARDA’s mission. National health security is a to turning strategy into action, tracking progress, and state in which the nation and its people are prepared correcting course when needed. The strategic plan for, protected from and resilient in the face of health maps out a broad five-year strategic direction. More threats or incidents with potentially negative health detailed actions for accomplishing the goals and strat­ consequences. All elements of this strategic plan align egies as well performance measures and milestones with and support the BARDA role in contributing to will be developed and annually reviewed by BAR­ national health security under the NHSS and, as spec­ DA staff and leadership. All BARDA employees are ified in PAHPA, the BARDA Strategic Plan is consid­ critical to the organization’s success, and should see ered to be incorporated as part of the NHSS. themselves and their work directly in one or more of the goals and strategies. BARDA divisions and pro­ orgAnizAtion oF this PlAn gram teams may develop even more granular action This strategic plan sets the direction for BARDA over plans, and budgets will be formulated and executed the next five years. Five strategic goals define the in line with strategic priorities. highest level aims of BARDA, each one is supported Like the HHS and ASPR strategic plans, the BARDA by strategies for accomplishing that goal. These goals strategic plan will be published online and updated are interrelated and derive from the mission outlined periodically to reflect evolving activities and progress in the PHEMCE Review that “Our nation must have toward goals. BARDA’s partners and the public will the nimble, flexible capacity to produce medical be able to see the latest information on priorities and countermeasures rapidly in the face of any attack or accomplishments, reinforcing the plan’s purpose as threat, known or unknown, including a novel, pre­ an evolving, living tool for guiding decision making viously unrecognized, naturally occurring emerging and action. infectious disease.”

6 BARDA Strategic Plan 2011-2016 BARDA’S Vision, Mission, and Values BARDA’S OVERARCHING VISION IS OF A NATION WITH AsPr VAlues the capability to respond quickly and effectively to deliberate, natural, and emerging threats so as to Diversity minimize their impact and recover promptly. A criti­ We are dedicated to creating and maintaining an cal enabling factor for the realization of this vision open, inclusive environment that nurtures differing is the existence of a robust domestic pharmaceutical world views. and biotechnology sector that actively collaborates Excellence with the U.S. Government to address unmet medical countermeasure and public health requirements. We demonstrate quality in what we do, set high ex­ pectations for ourselves and others, and innovate and BARDA’s vision for the next five years is to enhance improve through the introduction of new concepts the capability of the U.S. Government to respond and methods. quickly to both known and emerging threats by sup­ porting the development of a comprehensive portfo­ Integrity lio of medical countermeasures, needed manufactur­ We are honest and reliable, we hold ourselves ac­ ing infrastructure, and countermeasure production countable, and our actions and decisions are guided platforms while establishing an affordable and sus­ by fairness and transparency. tainable foundation for the maintenance and future Leadership operations of the PHEMCE. We motivate and inspire our colleagues to achieve BARDA’s mission is to develop and procure needed success through shared vision and unity of effort. medical countermeasures, including vaccines, thera­ peutics, diagnostics, and non-pharmaceutical coun­ Public Service termeasures, against a broad array of public health We commit to serve our country, our community, and threats, whether natural or intentional in origin. BARDA our fellow citizens, and are accountable to the public was created to address gaps in the U.S. Government for our actions medical countermeasure development and procure­ Respect ment process and to bridge the “valley of death” that separates candidates identified in early research from We treat people with dignity and consideration and potential FDA licensure/approval by providing fund­ listen openly to what others have to say, to benefit ing, technical support and services necessary to ad­ from varied perspectives and create environments vance candidate products through the developmental that foster trust. pipeline. Teamwork BARDA supports the ASPR Values Statement and en­ We support and motivate each other, achieving more deavors to embody these values in its efforts to fulfill together than we can alone. its mission and realize its vision. BARDA Strategic Plan 2011-2016 7 BARDA Guiding Principles BARDA WILL PURSUE ITS STRATEGIC GOALS THROUGH cal countermeasures during public health emergen­ an approach comprised of the following core cies, BARDA will establish a network of providers to principles. formulate, fill, and finish bulk vaccine product pro­ vided by primary manufacturers in the event that the CollABorAting in PuBliC- primary manufacturers lack sufficient surge capacity PriVAte PArtnershiPs of their own. The PHEMCE Review highlighted the critical role of public-private partnerships in addressing the chal­ ProMoting PlAtForM lenges that arise throughout the product development teChnologies process and in rapidly adapting to new technologies BARDA is supporting the development of novel plat­ and changing needs. BARDA’s commitment to public- form technologies that provide flexible systems for private partnerships is deep-rooted and extends across the production of medical countermeasures against its entire portfolio. a variety of threats. BARDA will also leverage estab­ lished programs to maximize the value of current in­ Where vaccine and biological therapeutics manufac­ vestments in technological innovations. Strategic sup­ turing capacity is concerned, BARDA will continue to port for platform technologies will greatly enhance enter into public-private partnerships with manufac­ the value provided by BARDA investments and posi­ turers to build and/or retrofit medical countermeasure tion the U.S. Government to respond more effectively production facilities within the U.S., increasing do­ to the unknown threats of the future. mestic access to medical countermeasures. Interna­ tionally, BARDA will maintain its financial and techni­ As an illustration, adjuvants, which can be used to cal support of the WHO Global Action Plan, working increase the supply of pandemic influenza vaccine, in concert with the Developing Countries Vaccine may have the potential to enhance the efficacy of vac­ Manufacturers Network and other partners to expand cines against biological threats or emerging diseases influenza vaccine manufacturing capacity in develop­ and may be essential components of next-generation ing countries. recombinant protein influenza vaccines. By support­ ing the development and use of adjuvant “platforms” BARDA will establish its Centers of Innovation for to enhance the efficacy of currently licensed vaccines, Advanced Development and Manufacturing as part­ BARDA is also supporting the establishment of a regu­ nerships in which the U.S. Government and private latory pathway for other vaccines that exploit adjuvant sector partners share facility construction costs, col­ technology – a critical step in speeding the availability laboratively facilitate medical countermeasure prod­ of new vaccines against a broad array of threats. uct development, and jointly ensure domestic vac­ cine manufacturing surge capacity. These Centers will provide advanced development and manufacturing Prioritizing MultiPurPose capabilities for medical countermeasures to address ProduCts both national security and public health needs on a BARDA is committed, where feasible, to developing cost-effective, reliable, and sustainable basis. In order products that demonstrate efficacy against a wide to minimize delays in the availability of critical medi­ range of specified threats and will seek out products

8 BARDA Strategic Plan 2011-2016 with such potential through its Strategic Science and using CAse MAnAgeMent And Technology program. Investing in multi-use products A MAtrixed orgAnizAtionAl such as broad-spectrum therapeutics and diagnos­ struCture tic platforms that can find durable and well-defined The PHEMCE Review requested by the Secretary of commercial markets and leverage market forces will HHS included recommendations to adopt a new, greatly facilitate the development of medical counter­ more intensive approach (“case management”) to pro­ measures for use in public health emergencies and gram and project management. Under case manage­ enhance long-term sustainability for the PHEMCE. ment, BARDA leads the effort to ensure that intra- and BARDA’s Broad Spectrum Antimicrobial Program was inter-agency stakeholders are kept abreast of product established in January 2010 and is focused on devel­ development progress and issues in the identified oping novel antibacterial and antiviral drugs for the programs. BARDA is adopting a rigorous approach to treatment or prevention of disease caused by current­ project, program, and portfolio management that will ly defined and future biological threats. Recognizing establish cost and schedule metrics for each phase the immediate value of new antimicrobials in address­ of product development, and allow the identification ing the increasingly prevalent public health threat of of best practices and earlier interventions in troubled antibiotic resistance, as well as the likelihood that an­ programs. In Process Review of program contracts timicrobial resistance will complicate primary treat­ occur on a regular basis by a team of experts from ment of a wide array of threats, BARDA is supporting HHS and PHEMCE partners who provide critical in­ the development of public-private partnerships for the put on key decisions in program development and development of novel antimicrobials. Through these guidance on issues that may arise during product de­ partnerships, BARDA will support the development of velopment. candidate antimicrobials for their commercial, clini­ When challenges with product development arise, cally prevalent infectious disease indications, as long BARDA employs a case management approach of as our private sector partners concomitantly support intensive support and guidance to private sector part­ the development of these products for biodefense ners, which also entails appropriate input and exper­ threat agent indications. By adopting this approach, tise from relevant federal agencies (BARDA, CDC, BARDA will support the development of new classes DoD, FDA, and NIH). Case management of BARDA of antimicrobials, increase the number of antimicro­ programs is essential to the proper stewardship of the bial drugs in the developmental pipeline, address the funding provided for these endeavors and will ensure important and immediate threat of antimicrobial re­ that all U.S. Government stakeholders are aware of sistance, and prepare the nation for the predictable products being developed and the long-term budget­ threat of biological terrorism and the unpredictable ary impact those products may have on other agen­ threat of emerging infectious diseases. cies through the course of their life cycle. Many other BARDA projects demonstrate the variety BARDA has adopted a matrix-like organizational of ways in which “dual utility” and multi-use value structure that makes the specialized capabilities of can be achieved. For example, next-generation, low- its different components available to project manag­ cost, easy-to-use ventilators and improved respiratory ers throughout the organization. This matrix-like or­ protective devices all have applications against a wide ganizational structure will maximize efficiency and array of threats. BARDA will continue to seek out mul­ collaboration across BARDA, promote a culture of tipurpose products and support their development in continual process improvement, and facilitate the order to enhance our Nation’s ability to respond effec­ ultimate application of case management to all sup­ tively to the broad array of threats we face. ported programs. BARDA Strategic Plan 2011-2016 9 ensuring An integrAted with greater efficiency and effectiveness. Although PortFolio With dod DoD and HHS each have certain unique requirements due to their different missions and focus, there are In addition to the management of specific programs many areas of convergent requirements for products under the effort highlighted above, BARDA, in col­ or capabilities where resources and information will laboration with its HHS and DoD partners, has ad­ be appropriately shared to optimize investments and opted an integrated approach to co-managing the na­ preclude unnecessary redundancies. This integrated tion’s aggregate portfolio of medical countermeasure approach will ensure balance and completeness development programs for CBRN threats. The overall across all agencies funding the research, develop­ goal is to optimize the use of resources to address ment, and procurement of medical countermeasures the broad range of common threats and requirements for CBRN threats.

10 BARDA Strategic Plan 2011-2016 Goals and Strategies goAl 1 needed to (1) ensure that the medical countermea­ sure needs of all populations are addressed and (2) An advanced development pipeline replete with enhance long-term sustainability of our preparedness medical countermeasures and platforms to address posture. unmet public health needs, emphasizing innova­ tion, flexibility, multi-purpose and broad spectrum goAl 1 strAtegies application, and long-term sustainability  Promote development and acquisition of needed Since its inception, BARDA has prioritized the devel­ medical countermeasures by opment and procurement of medical countermeasures for the highest risk threats, including pandemic influ­  Maintaining and where appropriate expand­ enza and terrorist use of chemical, biological, radio­ ing the portfolio of medical countermea­ logical, or nuclear materials. The Pandemic and All- sures against CBRN threats and pandemic Hazards Preparedness Act additionally called for the influenza public health and medical needs of at-risk individu­  Establishing a program to support the devel­ als to be taken into account in developing a national opment of platforms and countermeasures stockpile of medical countermeasures. Consistent with to address the threats of emerging infectious statutory requirements, BARDA supports the advanced diseases and antimicrobial resistance development of medical countermeasures for all popu­  Supporting the development of medical lations, with the goal of making them available for use countermeasures suitable for use in spe­ as needed during public health emergencies. cial populations such as children, pregnant BARDA’s CBRN programs have focused on develop­ women, the elderly, and persons with com­ ing medical countermeasures to meet the require­ promised immune systems, prioritizing and ments determined to be necessary to address the 13 supporting projects that provide benefits to Material Threats identified by the DHS in accordance all populations where possible and explor­ with Project BioShield. BARDA’s influenza program ing focused development projects or studies has contributed to pandemic preparedness by sup­ where necessary. porting the development of improved and/or next-  Improving processes governing the solicita­ generation influenza vaccines, antiviral medications, tion, review, and award of medical counter­ ventilators, and other non-pharmaceutical counter­ measure contracts measures; stockpiles of pre-pandemic vaccines and antiviral medications; and increased domestic influ­  Promote the development of flexible, multiprod­ enza vaccine manufacturing capacity. More recently, uct platforms and countermeasures with broad- BARDA has begun to address the threat of antimi­ spectrum activity and application by crobial resistance, emerging infectious diseases, and  Expanding BARDA’s Broad Spectrum other novel threats by initiating programs supporting Antimicrobials and Innovations programs the development of broad-spectrum antimicrobials to develop a robust portfolio of counter­ and technologies and platforms with multi-use po­ measures to address an array of known and tential. BARDA will maintain this three-pronged focus unknown threats on CBRN threats, pandemic influenza, and emerging infectious diseases, modifying its current portfolio as BARDA Strategic Plan 2011-2016 11  Investing in the development of host- goAl 2 strAtegies directed therapeutics, such as small mole­  Provide core product development services by cule immune modulators, anti-inflammatory agents, and regulators of innate immunity  Establishing the Centers for Innovation in Advanced Development and Manufacturing  Invest more strategically by (CIADM), which will have the capability to  Supporting the development of robust diag­ support analytical testing, scale-up manufac­ nostic capabilities in order to improve sur­ turing, manufacturing process optimization; veillance, facilitate earlier treatment, and provide regulatory and quality expertise and minimize medical countermeasure wastage analytical and process validation support;  Engaging in more proactive outreach, iden­ and produce clinical investigational lots, tifying candidate technologies and innova­ engineering lots, process validation lots, and tions that promise greater sustainability or commercial-scale lots for stockpiling that BARDA wishes to see advance because  Developing and qualifying animal mod­ of portfolio requirements els through the new Animal Models  Establishing a medical countermeasures Development Program Strategic Investor that will pursue the strate­  Performing GLP efficacy evaluation and test­ gic objectives of the PHEMCE while acting ing of CBRN countermeasures in qualified as, and providing all the services and ben­ animal models efits of, a venture capital firm  Provide biotechnology firms working on medical countermeasures access to financial and busi­ goAl 2 ness services by establishing a medical counter­ measures Strategic Investor that will serve as a A capability base to provide enabling core services non-profit venture capital firm to medical countermeasure innovators  Reduce regulatory barriers and clarify regulatory Considerable expertise and capabilities are required pathways by to overcome the technical, regulatory, manufacturing, commercialization, and business challenges inherent  Assisting private sector partners in the devel­ in the development of innovative medical counter­ opment of regulatory strategies and design measure candidates. Over the past ten years, innova­ and execution of clinical studies for needed tive companies developing CBRN medical counter­ medical countermeasures measures have encountered many challenges moving  Involving FDA experts in providing technical products along the process development, manufac­ assistance on projects supported by CIADM turing, and regulatory pathways. BARDA already provides an array of services to manufacturers and  Contributing to the development and imple­ innovators of medical countermeasures but has ob­ mentation of a PHEMCE regulatory science served that many of these companies would benefit agenda from additional support in moving from laboratory to  Promote the development of a highly trained commercial scale production and clinical evaluation biopharmaceutical workforce by: through the regulatory approval process.  Incorporating training in biopharmaceutical science and other workforce development opportunities into the day-to-day functions of the CIADM

12 BARDA Strategic Plan 2011-2016 goAl 3 goAl 3 strAtegies Agile, robust and sustainable U.S. manufacturing in­  Speed the delivery of pandemic influenza vac­ frastructure capable of rapidly producing vaccines cines following the emergence of an influenza and other biologics against pandemic influenza and virus with pandemic potential by other emerging threats  Developing improved vaccine seed strains, sterility tests, and potency reagents and The 2005 National Strategy for Pandemic Influenza es­ testing tablished a commitment to making vaccine available for all Americans within six months of the emergence  Expanding the number of FDA-licensed of a virus with pandemic potential. BARDA has made influenza vaccines, to include cell-based significant progress towards increasing and securing products domestic manufacturing capacity for pandemic influ­  Supporting the development of faster, more enza vaccine development. However, the recent re­ scalable next-generation recombinant influ­ sponse to 2009 H1N1 shows that we are still hampered enza vaccines by the limits of current technology. The first domestic  Completing the development and evaluation cases of 2009 H1N1 influenza were identified in April of adjuvanted pandemic influenza vaccines and the pandemic peaked about six months later, in  Optimizing the BARDA emergency manu­ late October. Despite an intensive effort to develop a facturing response pandemic vaccine, the 2009 H1N1 vaccine arrived too  Increase domestic manufacturing surge capacity late to have a significant effect on the dynamics of the for pandemic influenza by fall disease wave. Influenza vaccines licensed in the U.S. use egg-based technology that is more than 50  Supporting the construction or retrofitting of years old and a substantial portion of the manufactur­ influenza vaccine manufacturing facilities ing capacity is based overseas.  Supporting the development of next-genera­ tion manufacturing processes Consistent with recommendations from the President’s Council of Advisors on Science and Technology in its  Securing the availability of essential vaccine August 2010 Report to the President on Reengineering supplies and services by enhancing domes­ the Influenza Vaccine Production Enterprise to Meet tic fill finish networks and ensuring a year- the Challenges of Pandemic Influenza BARDA is work­ round supply of eggs for pandemic vaccine ing with several partners to develop more modern plat­ surge production forms for manufacturing influenza vaccines and this  Requiring CIADM awardees to maintain a investment is beginning to pay off. In 2011, two man­ capability to manufacture pandemic influ­ ufacturers plan to file Biologic License Applications enza vaccine (BLAs) for cell-based influenza vaccines, with new vac­  As in GOAL 2, promote the development of a cines based on recombinant technologies to come in highly trained biopharmaceutical workforce to following years. Products based on these newer, faster, ensure commercial-scale manufacturing capacity more scalable technologies will move the field forward by and significantly increase the flexibility, surge capacity,  Incorporating training in biopharmaceutical and reliability of influenza vaccine production increas­ science and other workforce development ing and securing domestic manufacturing capacity for opportunities into the day-to-day functions pandemic influenza vaccine development. of the CIADM  Sponsoring academic training programs in bio-manufacturing techniques as part of a broader initiative to build influenza vac­ cine manufacturing capacity in developing countries BARDA Strategic Plan 2011-2016 13 goAl 4 goAl 4 strAtegies Responsive and nimble programs and capabilities to  Develop a formal methodology to identify medi­ address novel and emerging threats cal countermeasure projects that are appropriate for inclusion in the BARDA Emerging Infectious The Pandemic and All-Hazards Preparedness Act Diseases portfolio charges BARDA with the advanced development of medical countermeasures for emerging infectious dis­  Implement a long-term strategy to address previ­ ease threats, which come in many forms. New and ously undefined threats by lethal infectious diseases, such as MRSA, Dengue,  Expanding BARDA’s Broad Spectrum Anti­ Ebola, SARS, and Nipah virus, continue to emerge in microbials and Innovations programs nature. The extensive use of antiviral and antibacte­ rial agents has resulted in the propagation of drug re­  Emphasizing the development of multi­ sistant strains of bacteria and viruses and promoted purpose and/or broad-spectrum products the evolution of extensively drug-resistant strains of  Investing in capabilities (platforms and facil­ many common pathogens. As has been the case with ities) for the rapid development and produc­ past influenza pandemics where secondary infections tion of countermeasures with resistant organisms accounted for a significant proportion of deaths, drug-resistant strains are likely  Investing in the development of host- directed to complicate the treatment of CBRN-caused illness­ therapeutics, such as small molecule immune es. Where deliberate threats are concerned, advances modulators, anti-inflammatory agents, and in genetic engineering and synthetic biology may pro­ regulators of innate immunity vide a means for bioterrorists to generate more dan­  As in GOAL 1, establishing a medical coun­ gerous pathogens through the insertion of virulence termeasures Strategic Investor that will pur­ factors or drug-resistance genes. sue the strategic objectives of the PHEMCE Preparing for the threat of previously unknown emerg­ while acting as, and providing all the ser­ ing diseases, whatever the source, requires a different vices and benefits of, a venture capital firm approach than preparing for known threats or patho­  Combat the growing problem of antimicrobial gens. To address these evolving threats to public resistance through development of new antimi­ health, acquisition of broad spectrum antibiotic and crobials and innovative therapeutics by pursuing antiviral medications and new classes of antimicro­ new classes of drugs and innovative antimicro­ bials, including host-directed therapeutics and small bial therapies that will be less susceptible to drug molecule immune modulators, will be a major focus resistance of BARDA’s efforts in coming years. Over the next five years, building on the experiences of the CBRN, Influenza, and Strategic Science and Technology Divisions, BARDA will enhance its efforts to provide solutions to the growing problem of emerg­ ing pathogens and antimicrobial resistance. In the near term, BARDA is developing programs to address the threat posed by multidrug-resistant pathogens and future unknown biological threats. A key element of this effort is identifying and supporting the develop­ ment of broad spectrum antimicrobials. In addition, BARDA’s next-generation influenza antiviral drug ad­ vanced development program is pursuing new classes of drugs and innovative candidate antiviral therapies that will be less susceptible to viral resistance.

14 BARDA Strategic Plan 2011-2016 goAl 5 goAl 5 strAtegies A ready capability to develop, manufacture and  Refine BARDA planning and readiness capabili­ facilitate distribution of medical countermeasures ties by drawing on the many lessons learned dur­ during public health emergencies ing the pandemic Ensuring the availability of safe and effective medical  In concert with the ASPR Office of Preparedness countermeasures during public health emergencies and Emergency Operations and in close coor­ is BARDA’s core mission. Supporting the scientific dination with CDC, FDA, and other U.S. development of vaccines, therapeutics, and diagnos­ Government departments and agencies, develop tics is only one aspect of BARDA’s role in promoting comprehensive plans that will facilitate the pandemic influenza and public health preparedness. response to future pandemics, CBRN events, and The 2009 H1N1 pandemic underscored the critical emerging epidemics by importance of advanced development, manufactur­  Establishing mechanisms for interfacing ing, and distribution planning. During the pandemic, with medical countermeasure manufactur­ BARDA worked in coordination with the NIH, FDA ers and distributors during a public health and CDC to procure, test, license and deliver the vac­ emergency cines and antiviral drugs deployed as part of the U.S.  Establishing mechanisms for positioning Government response. BARDA medical countermeasure produc­ tion subject matter experts in manufacturing Ultimately, 138 million doses of licensed 2009 H1N1 plants as needed vaccine were produced and distributed for use by the U.S. civilian population, 2.7 million doses for DoD,  Providing a framework for developing and validating emergency funding requests and 16.9 million doses for international assistance. As part of a contingency-based public health response,  Facilitating the prompt evaluation of investi­ BARDA also purchased supplies of adjuvants to be gational products for emergency use autho­ used, if necessary, as part of a pandemic vaccine and rization as warranted. additional supplies of antiviral medications to replen­  Exercise these readiness capabilities plans to ish treatment courses that were distributed from the ensure that they are fully consistent with exist­ Strategic National Stockpile, focusing on pediatric ing ASPR and HHS response plans as well as the formulations to address critical needs for this popula­ overarching National Response Framework tion. BARDA’s collaborations with domestic vaccine manufacturers and coordination with the response activities of CDC and industry partners to ensure effective and timely distribution of pandemic coun­ termeasures within regional, state, and tribal jurisdic­ tions were critical enablers of the overall response to the pandemic. BARDA Strategic Plan 2011-2016 15 Blank Inside back cover Blank U.S. Department of Health and Human Services Assistant Secretary for Preparedness and Response

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