Goals and Strategies

BARDA will pursue its strategic goals through an approach comprised of the following core principles.

Goal 1 | Goal 2 | Goal 3 | Goal 4 | Goal 5

Goal 1

An advanced development pipeline replete with medical countermeasures and platforms to address unmet public health needs, emphasizing innovation, flexibility, multi-purpose and broad spectrum application, and long-term sustainability

Since its inception, BARDA has prioritized the development and procurement of medical countermeasures for the highest risk threats, including pandemic influenza and terrorist use of chemical, biological, radiological, or nuclear materials. The Pandemic and All-Hazards Preparedness Act additionally called for the public health and medical needs of at-risk individuals to be taken into account in developing a national stockpile of medical countermeasures. Consistent with statutory requirements, BARDA supports the advanced development of medical countermeasures for all populations, with the goal of making them available for use as needed during public health emergencies.

BARDA's CBRN programs have focused on developing medical countermeasures to meet the requirements determined to be necessary to address the 13 Material Threats identified by the DHS in accordance with Project BioShield. BARDA's influenza program has contributed to pandemic preparedness by supporting the development of improved and/or next-generation influenza vaccines, antiviral medications, ventilators, and other non-pharmaceutical countermeasures; stockpiles of pre-pandemic vaccines and antiviral medications; and increased domestic influenza vaccine manufacturing capacity. More recently, BARDA has begun to address the threat of antimicrobial resistance, emerging infectious diseases, and other novel threats by initiating programs supporting the development of broad-spectrum antimicrobials and technologies and platforms with multi-use potential. BARDA will maintain this three-pronged focus on CBRN threats, pandemic influenza, and emerging infectious diseases, modifying its current portfolio as needed to (1) ensure that the medical countermeasure needs of all populations are addressed and (2) enhance long-term sustainability of our preparedness posture.

Goal 1 Strategies

• Promote development and acquisition of needed medical countermeasures by
• Maintaining and where appropriate expanding the portfolio of medical countermeasures against CBRN threats and pandemic influenza
• Establishing a program to support the development of platforms and countermeasures to address the threats of emerging infectious diseases and antimicrobial resistance
• Supporting the development of medical countermeasures suitable for use in special populations such as children, pregnant women, the elderly, and persons with compromised immune systems, prioritizing and supporting projects that provide benefits to all populations where possible and exploring focused development projects or studies where necessary.
• Improving processes governing the solicitation, review, and award of medical countermeasure contracts
• Promote the development of flexible, multiproduct platforms and countermeasures with broad-spectrum activity and application by
  • Expanding BARDA's Broad Spectrum Antimicrobials and Innovations programs to develop a robust portfolio of countermeasures to address an array of known and unknown threats
  • Investing in the development of host-directed therapeutics, such as small molecule immune modulators, anti-inflammatory agents, and regulators of innate immunity
• Invest more strategically by
  • Supporting the development of robust diagnostic capabilities in order to improve surveillance, facilitate earlier treatment, and minimize medical countermeasure wastage
  • Engaging in more proactive outreach, identifying candidate technologies and innovations that promise greater sustainability or that BARDA wishes to see advance because of portfolio requirements
  • Establishing a medical countermeasures Strategic Investor that will pursue the strategic objectives of the PHEMCE while acting as, and providing all the services and benefits of, a venture capital firm

Goal 2

A capability base to provide enabling core services to medical countermeasure innovators

Considerable expertise and capabilities are required to overcome the technical, regulatory, manufacturing, commercialization, and business challenges inherent in the development of innovative medical countermeasure candidates. Over the past ten years, innovative companies developing CBRN medical countermeasures have encountered many challenges moving products along the process development, manufacturing, and regulatory pathways. BARDA already provides an array of services to manufacturers and innovators of medical countermeasures but has observed that many of these companies would benefit from additional support in moving from laboratory to commercial scale production and clinical evaluation through the regulatory approval process.

Goal 2 Strategies

• Provide core product development services by
• Establishing the Centers for Innovation in Advanced Development and Manufacturing (CIADM), which will have the capability to support analytical testing, scale-up manufacturing, manufacturing process optimization; provide regulatory and quality expertise and analytical and process validation support; and produce clinical investigational lots, engineering lots, process validation lots, and commercial-scale lots for stockpiling
• Developing and qualifying animal models through the new Animal Models Development Program
• Performing GLP efficacy evaluation and testing of CBRN countermeasures in qualified animal models
• Provide biotechnology firms working on medical countermeasures access to financial and business services by establishing a medical countermeasures Strategic Investor that will serve as a non-profit venture capital firm
• Reduce regulatory barriers and clarify regulatory pathways by
  • Assisting private sector partners in the development of regulatory strategies and design and execution of clinical studies for needed medical countermeasures
  • Involving FDA experts in providing technical assistance on projects supported by CIADM
  • Contributing to the development and implementation of a PHEMCE regulatory science agenda
• Promote the development of a highly trained biopharmaceutical workforce by:
  • Incorporating training in biopharmaceutical science and other workforce development opportunities into the day-to-day functions of the CIADM

Goal 3

Agile, robust and sustainable U.S. manufacturing infrastructure capable of rapidly producing vaccines and other biologics against pandemic influenza and other emerging threats

The 2005 National Strategy for Pandemic Influenza established a commitment to making vaccine available for all Americans within six months of the emergence of a virus with pandemic potential. BARDA has made significant progress towards increasing and securing domestic manufacturing capacity for pandemic influenza vaccine development. However, the recent response to 2009 H1N1 shows that we are still hampered by the limits of current technology. The first domestic cases of 2009 H1N1 influenza were identified in April and the pandemic peaked about six months later, in late October. Despite an intensive effort to develop a pandemic vaccine, the 2009 H1N1 vaccine arrived too late to have a significant effect on the dynamics of the fall disease wave. Influenza vaccines licensed in the U.S. use egg-based technology that is more than 50 years old and a substantial portion of the manufacturing capacity is based overseas.

Consistent with recommendations from the President's Council of Advisors on Science and Technology in its August 2010 Report to the President on Reengineering the Influenza Vaccine Production Enterprise to Meet the Challenges of Pandemic Influenza BARDA is working with several partners to develop more modern platforms for manufacturing influenza vaccines and this investment is beginning to pay off. In 2011, two manufacturers plan to file Biologic License Applications (BLAs) for cell-based influenza vaccines, with new vaccines based on recombinant technologies to come in following years. Products based on these newer, faster, more scalable technologies will move the field forward and significantly increase the flexibility, surge capacity, and reliability of influenza vaccine production increasing and securing domestic manufacturing capacity for pandemic influenza vaccine development.

Goal 3 Strategies

• Speed the delivery of pandemic influenza vaccines following the emergence of an influenza virus with pandemic potential by
• Developing improved vaccine seed strains, sterility tests, and potency reagents and testing
• Expanding the number of FDA-licensed influenza vaccines, to include cell-based products
• Supporting the development of faster, more scalable next-generation recombinant influenza vaccines
• Completing the development and evaluation of adjuvanted pandemic influenza vaccines
• Optimizing the BARDA emergency manufacturing response
• Increase domestic manufacturing surge capacity for pandemic influenza by
  • Supporting the construction or retrofitting of influenza vaccine manufacturing facilities
  • Supporting the development of next-generation manufacturing processes
  • Securing the availability of essential vaccine supplies and services by enhancing domestic fill finish networks and ensuring a year-round supply of eggs for pandemic vaccine surge production
  • Requiring CIADM awardees to maintain a capability to manufacture pandemic influenza vaccine
• As in GoaL 2, promote the development of a highly trained biopharmaceutical workforce to ensure commercial-scale manufacturing capacity by
  • Incorporating training in biopharmaceutical science and other workforce development opportunities into the day-to-day functions of the CIADM
  • Sponsoring academic training programs in bio-manufacturing techniques as part of a broader initiative to build influenza vaccine manufacturing capacity in developing countries

Goal 4

Responsive and nimble programs and capabilities to address novel and emerging threats

The Pandemic and All-Hazards Preparedness Act charges BARDA with the advanced development of medical countermeasures for emerging infectious disease threats, which come in many forms. New and lethal infectious diseases, such as MRSA, Dengue, Ebola, SARS, and Nipah virus, continue to emerge in nature. The extensive use of antiviral and antibacterial agents has resulted in the propagation of drug resistant strains of bacteria and viruses and promoted the evolution of extensively drug-resistant strains of many common pathogens. As has been the case with past influenza pandemics where secondary infections with resistant organisms accounted for a significant proportion of deaths, drug-resistant strains are likely to complicate the treatment of CBRN-caused illnesses. Where deliberate threats are concerned, advances in genetic engineering and synthetic biology may provide a means for bioterrorists to generate more dangerous pathogens through the insertion of virulence factors or drug-resistance genes.

Preparing for the threat of previously unknown emerging diseases, whatever the source, requires a different approach than preparing for known threats or pathogens. To address these evolving threats to public health, acquisition of broad spectrum antibiotic and antiviral medications and new classes of antimicrobials, including host-directed therapeutics and small molecule immune modulators, will be a major focus 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 emerging 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 development of broad spectrum antimicrobials. In addition, BARDA's next-generation influenza antiviral drug advanced development program is pursuing new classes of drugs and innovative candidate antiviral therapies that will be less susceptible to viral resistance.

Goal 4 Strategies

• Develop a formal methodology to identify medical countermeasure projects that are appropriate for inclusion in the BARDA Emerging Infectious Diseases portfolio
• Implement a long-term strategy to address previously undefined threats by
• Expanding BARDA's Broad Spectrum Antimicrobials and Innovations programs
• Emphasizing the development of multipurpose and/or broad-spectrum products
• Investing in capabilities (platforms and facilities) for the rapid development and production of countermeasures
• Investing in the development of host- directed therapeutics, such as small molecule immune modulators, anti-inflammatory agents, and regulators of innate immunity
• As in GoaL 1, establishing a medical countermeasures Strategic Investor that will pursue the strategic objectives of the PHEMCE while acting as, and providing all the services and benefits of, a venture capital firm
• Combat the growing problem of antimicrobial resistance through development of new antimicrobials and innovative therapeutics by pursuing new classes of drugs and innovative antimicrobial therapies that will be less susceptible to drug resistance

Goal 5

A ready capability to develop, manufacture and facilitate distribution of medical countermeasures during public health emergencies

Ensuring the availability of safe and effective medical countermeasures during public health emergencies is BARDA's core mission. Supporting the scientific development of vaccines, therapeutics, and diagnostics is only one aspect of BARDA's role in promoting pandemic influenza and public health preparedness. The 2009 H1N1 pandemic underscored the critical importance of advanced development, manufacturing, and distribution planning. During the pandemic, BARDA worked in coordination with the NIH, FDA and CDC to procure, test, license and deliver the vaccines and antiviral drugs deployed as part of the U.S. Government response.

Ultimately, 138 million doses of licensed 2009 H1N1 vaccine were produced and distributed for use by the U.S. civilian population, 2.7 million doses for DoD, and 16.9 million doses for international assistance. As part of a contingency-based public health response, BARDA also purchased supplies of adjuvants to be used, if necessary, as part of a pandemic vaccine and additional supplies of antiviral medications to replenish treatment courses that were distributed from the Strategic National Stockpile, focusing on pediatric formulations to address critical needs for this population. 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 countermeasures within regional, state, and tribal jurisdictions were critical enablers of the overall response to the pandemic.

Goal 5 Strategies

• Refine BARDA planning and readiness capabilities by drawing on the many lessons learned during the pandemic
• In concert with the ASPR Office of Preparedness and Emergency Operations and in close coordination with CDC, FDA, and other U.S. Government departments and agencies, develop comprehensive plans that will facilitate the response to future pandemics, CBRN events, and emerging epidemics by
• Establishing mechanisms for interfacing with medical countermeasure manufacturers and distributors during a public health emergency
• Establishing mechanisms for positioning BARDA medical countermeasure production subject matter experts in manufacturing plants as needed
• Providing a framework for developing and validating emergency funding requests
• Facilitating the prompt evaluation of investigational products for emergency use authorization as warranted.
• Exercise these readiness capabilities plans to ensure that they are fully consistent with existing ASPR and HHS response plans as well as the overarching National Response Framework