2013_Multi-BAA_Preproposal_Conference_Slides.pdf

United States Department of

Health & Human Services Office of the Assistant Secretary for Preparedness and Response

Hubert H. Humphrey Building - The Great Hall

September 4, 2013

The BARDA Multi-Broad Agency

Announcement Pre-Proposal Conference

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BARDA Mission

Develop and provide countermeasures for CBRN threats, pandemic influenza, and emerging infectious diseases by product development, stockpile acquisition, building manufacturing infrastructure, and product innovation.

Tip O’Neill Building (FOB8)

ASPR: Resilient People. Healthy Communities. A Nation Prepared.

Man-made and Natural Threats

• CBRN Threats

• Chemical nerve agents & cyanide

• Radiological and Nuclear agents

• Biothreats (anthrax, smallpox, plaque, tularemia, VHF, and others)

• Pandemic influenza

• Emerging infectious diseases

Medical Countermeasures

Antimicrobials Diagnostics Vaccines Medical Devices Therapeutics

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Discovery Phase I Phase II LicensurePhase IIIPreclinical Development

Production & Delivery

PROBABILITY OF SUCCESS TO LICENSURE

PR

O

D U

C T

PI

PE

LI

N

E

PR

O D

U C

T

PI

PE

LI

N E

Licensed Product

1-3% 5-17% 10-25% 18-35% 45-70% 90%

IND NDA/BLA

PHASESPHASES

NIH ($11.8B)

BARDA ($540M)

Project BioShield ($5.6B)

Discovery Phase I Phase II LicensurePhase IIIPreclinical Development

Production & Delivery

PROBABILITY OF SUCCESS TO LICENSURE

PR

O

D U

C T

PI

PE

LI

N

E

PR

O D

U C

T

PI

PE

LI

N E

Licensed Product

1-3% 5-17% 10-25% 18-35% 45-70% 90%

IND NDA/BLA

PHASESPHASES

NIH ($11.8B)

BARDA ($540M)

Project BioShield ($5.6B)

BARDA ($2.0 B)

NIH ($15.6 B)

3-7 yr 0.5-2 yr 1-2 yr 2-3.5 yr 2.5 -4 yr 1-2 yrs TIME

$100M -130M $60-70M $70M-100M $130M-160M $190M-220M $18M-20M PIPELINE

PHASE COST

Valley of Death

CBRN MCM Development is Expensive, Lengthy, & Risky

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• xxx

2012 PHEMCE Strategy and Implementation Plan

• PHEMCE Strategy and Implementation Plan will set the course for federal medical countermeasure (MCM) activities for next five years ─ Builds upon 2007 SIP, which established civilian

MCM priorities with focus on advanced research and development

• Strategy [June 2012] – Mission, scope, goals, and objectives

• Implementation Plan [December 2012] – Prioritized programs to accomplish goals and objectives identified in Strategy

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Pandemic Influenza Strategic Plans & Reviews

• The requirements addressed by the BARDA Influenza Portfolio are derived from a number of documents that guide the US Government efforts to prepare for pandemics

• National Strategy for Pandemic Influenza (Nov 2005)

• HHS Pandemic Influenza Plan (Nov 2005)

• Nation Strategy for Pandemic Influenza Implementation Plan (May 2006)

• Public Health Emergency Medical Countermeasures Review (Aug 2010)

• PCAST report on Reengineering the Influenza Vaccine Production

Enterprise (Aug 2010)

Future: BARDA Strategic Plan 2011-2016

• GOALS

─ Abundently supply advanced development pipeline for all known threats

─ Core service capability ─ Robust domestic MCM manufacturing infrastructure ─ Address MCM needs for emerging infectious diseases ─ Nimble response capability to known & unknown threats

• Cascades from NHSS, ASPR Strategic

Plan, MCM Review, and other national plans https://www.medicalcountermeasures.gov/Barda/documents/BARDAStrategicPlan9-28--508.pdf

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Centers for Innovation in

Advanced Development & Manufacturing

Fill Finish Mfg.

Network

Animal Studies Network

Clinical Studies Network

Strategic Investor

Modeling Hub

Regulatory & Technical Expertise

BARDA Core Services

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• BARDA is committed to developing and providing MCMs for civilian preparedness & response to CBRN, pandemic influenza, and emerging infectious threats

• BARDA utilizes public-private partnerships with industry to achieve these goals by providing funding support and critical core services to its partners

• BARDA maintains a balanced portfolio for specific and multiple threats and places a high priority on sustainable life cycle management

BARDA 2013

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• Preparedness against Unknown Threats

• Economic Woes & Budget Austerity

• Global Political Unrest Feeding Bioterrorism

• Erosion of State and Local Public Health Infrastructure

• Needs of Special Populations

• Identity Crisis in U.S. Pharmaceutical & Biotech Industry

• Sustainability of Programs and Products

• Spiraling Life Cycle Maintenance Costs of MCM Stockpiles

Broader Challenges

Contact Us

BARDA:

URL: http://www.phe.gov BARDA e-mail: BARDA@hhs.gov

• Upcoming Events

• PHEMCE Strategy and Implementation Plan

• CBRN and Pan Flu Programs

• Business Toolkit

─ www.phe.gov/amcg

Tech Watch program Federally-sponsored conferences Funding opportunities Resources 7 core service programs Regulatory guidance Federal strategies and reports mailto:BARDA@hhs.gov

CBRN Broad Agency Announcement Areas of Interest and Priorities for Focus

Gerald R. Kovacs Director, CBRN Programs

BARDA/ASPR/HHS

4 September 2013

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• BARDA CBRN MCM Broad Agency Announcement (BAA) announces annually to the public BARDA’s intentions and priorities in funding development of CBRN MCMs since 2009

• The BAA has served as an excellent tool to build a robust pipeline of 80+ CBRN MCM candidates ─ Nearly all of the BARDA CBRN MCM development projects emanate from the BAA ─ Special Instructions afford highlighted areas of emphasis ─ Fast and flexible

• BAA stats ─ Total number of White Papers received: 424 ─ Total number of Full Proposals: 106 ─ Total number of awards: 28

CBRN BAA Overview

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Advanced R&D Investments by Year

FY07 FY08 FY09 FY10 FY11 FY12 FY13

N ew

P ro gr am s Investm ents in M illions

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• In-Process Reviews

• Source Selection and Milestone Decision Authorities

• Solicitations Open Year Round

• Five Year Budgets

• Earned Value Management

Business Practices Supporting Investments

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• Products ─ Raxibacumab – monoclonal antibody (FDA approved 2012) ─ Botulinum antitoxin – equine polyclonal (FDA approved 2013) ─ Anthrax Immune globulin – human polyclonal (approval in 2014/15) ─ Anthrax vaccine adsorbed – sterile filtrate (approval in 2014/15) ─ Modified Vaccinia Ankara – live attenuated vaccine (approval in 2016, EU approved in 2013) ─ ST-246 – smallpox antiviral (approval in 2017)

• Total R&D investment ~$2.2 B

• ~ $373 M per approved product

• 7- 8yr to FDA approval from Phase I/II

BARDA Track Record

• De-emphasizing mature programs ─ Smallpox – vaccines and antiviral drugs ─ Anthrax antitoxins ─ Botulinum antitoxins ─ Field use MCMs to treat Acute Radiation Syndrome ─ Anticonvulsants ─ Biodosimetry tools

• Emphasizing programs to fill unmet gaps ─ Therapeutics for hemorrhagic fever viruses ─ Broad-spectrum antimicrobials for Burkholderia ─ Thermal burn products ─ Biothreat diagnostics

Priorities for CBRN MCMs

• Vaccines ─ Smallpox vaccines

• Enhancements to current vaccines in SNS ─ Anthrax vaccines

• 12 months of stability

• Non-inferiority to AVA in non-clinical study

• Antivirals & Antitoxins ─ Accepting proposals for therapeutics for viral hemorrhagic fever viruses ─ Enhancements to current antitoxins in SNS

• Broad Spectrum Antimicrobials ─ TRL raised to indicate lead compound must be identified ─ In vitro resistance data must be submitted ─ Favorable review for proposals that address pediatric subjects, propose cost sharing and have supportive responses from the FDA concerning development plan for candidate

CBRN MCM BAA

Refinements for 2013

• Rad/Nuc/Chem ─ No longer accepting proposals for “field use” MCMs (cytokines) for anti-neutropenics ─ Chem MCM candidates must be TRL-5 or higher (i.e., submitted pre-IND) ─ No longer accepting proposals for products to replace diazepam

(anticonvulsant)

• Biodosimetry/Diagnostics ─ Biodosimetry products must have shown biomarker feasibility ─ Encouraging submissions for biothreat diagnostics (material threats only)

CBRN MCM BAA

Refinements for 2013

BARDA:

URL: http://www.phe.gov BARDA e-mail: BARDA@hhs.gov

• Upcoming Events

• PHEMCE Strategy and Implementation Plan

• CBRN and Pan Flu Programs

Funding opportunities Resources 7 core service programs Regulatory guidance

BARDA Influenza Division

BAA 2013-2014

Robert C. Huebner, Ph.D.

Director, Influenza Division

September 4, 2013

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BARDA Mission

Develop and provide countermeasures for CBRN threats, pandemic influenza, and emerging infectious diseases by product development, stockpile acquisition/building, manufacturing infrastructure building, and product innovation.

O’Neill Building or Federal Office Building 8

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Preparing for Influenza Pandemics which are Unpredictable Events

A(H1N1) A(H2N2) A(H3N2) 1918: “Spanish Flu” 1957: “Asian Flu” 1968: “Hong Kong Flu”

20-100 m deaths ~500,000 in US

1-4 m deaths 60-80,000 in US

1-4 m deaths ~30,000 in US

Credit: US National Museum of Health and Medicine

“2009 H1N1 Pandemic”

2012 H3N2v, 2013 H7N9?

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• Vaccines

– Goal #1: Establish and maintain a dynamic pre-pandemic influenza vaccine stockpile available for 20 M persons (2 doses/person) or more depending on vaccine mfg. capacity, results of dose-sparing adjuvant studies and prime-boost immunization studies: H5N1 vaccine stockpiles - Achieved

– Goal #2: Provide pandemic vaccine to all U.S. citizens within 6 (or less) months of a pandemic declaration: pandemic vaccine (600 M doses)

• Antivirals

– Goal #1: Provide influenza antiviral drug stockpiles for pandemic treatment of

25% of U.S. population (75 M treatment courses) and federal share of antivirals for outbreak prophylactic usage as a community mitigation measure as shared responsibility - Achieved

– Goal #2: Provide influenza antiviral drug stockpiles for strategic limited containment at onset of pandemic (6 M treatment courses) - Achieved

• Diagnostics

– Goal #1: Develop new high-throughput laboratory, point-of-care (POC), and home detection influenza diagnostics for pandemic influenza virus detection

• Other Countermeasures

– Goal #1:Develop and acquire other MCMs including syringes/needles, masks/respirators, ventilators & other supplies

National Strategy for Pandemic Influenza (Nov 2005) and HHS Pandemic Influenza Plan (Nov 2005) www.pandemicflu.gov

U.S. Pan Flu MCM Strategic Goals http://www.pandemicflu.gov/

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Seasonal & Pandemic Influenza

Preparedness

All Hazards Preparedness

CBRN

Preparedness

Emerging Emerging Diseases Diseases

PreparednessPreparedness

All-Hazards Principle

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H5N1 Pre-Pandemic Vaccine

H1N1 Vaccines

BARDA MCM

Integrated Portfolio Matrix Approach

Retrofit Existing Mfg Facilities Build New Mfg Facilities

(Cell/CIADM) Egg-based Supply

Infrastructure Building

Masks & Respirators

Federal & State Stockpiles

IV Antivirals: EUA

Stockpiling & Acquisitions

Diagnostics Point of Care Clinical Lab

Next Generation Ventilators

Peramivir Laninamivir

Fludase Nitazoxinide

Cell-based Antigen-sparing Recombinant

& Molecular

Advanced

Development

Diagnostics/ Respiratory Devices Antivirals Vaccines

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National Pandemic Influenza Vaccine Development Strategy Is Multi-Step & Integrated Approach

Cell-based Vaccines

Egg-based Vaccines

Recombinant-based Vaccines

Universal Vaccines

“More and better vaccines sooner”

FluBlOk Licensed 01/16/13

Flucelvax Licensed 11/20/12

BARDA Influenza Division BAA 2013-2014

Filling in the medical countermeasure gaps

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Area of Interest #1 & #2

• Area of Interest #1: Personal Protective Equipment (Mask & Respirators) for Influenza Infection and All-Hazards ─ Offerors for Area of Interest #1 should describe the maturity level of their proposed technology. Proposed activities should offer clinical and public health benefits.

─ 1.1 Development and characterization of improved respiratory protective devices (RPD).

• Area of Interest #2: Full-Featured Continuous Ventilators for Influenza Infection and All-Hazards ─ At this time BARDA is not accepting any submissions for

Area of Interest #2.

─ Watch this space

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Area of Interest #3

• Area of Interest #3: Clinical Influenza Test Systems and Diagnostic Tools ─ Proposed activities should offer beneficial clinical and public health impact.

─ 3.1 Development of improved respiratory specimen collection materials and methods.

─ 3.2 Development of advanced sequence detection methods for identifying novel influenza strains.

─ 3.3 Development of expanded respiratory pathogen tests on existing test platforms.

─ 3.4 Rapid identification of antiviral resistant influenza strains.

ASPR: Resilient People. Healthy Communities. A Nation Prepared. 32

Area of Interest #4

• Area of Interest #4: Influenza Therapeutics

─ 4.1 Antiviral Therapeutics for Treatment of Influenza A & B

Infection

• Therapeutics proven to reduce influenza virus replication in cell culture and animal models are examples of therapeutics that would be considered. Therapeutics with a mechanism of action that precludes the rapid emergence of drug resistance and with proven broad influenza strain susceptibility are preferred. Combination therapeutics that include novel investigational compounds and licensed therapeutics would also be applicable.

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Area of Interest #5

• Area of Interest #5: Influenza Vaccines ─ Under this Area of Interest, BARDA is seeking technologies achieving TLR 6 or greater that will improve preparedness against influenza subtypes with pandemic potential.

• 5.1 Advanced development of novel influenza vaccine candidates. Support for advanced development of novel influenza vaccine candidates with the potential to stimulate a broader and more efficacious effective immune response than currently available products. Proposed activities should enable improvements to key vaccine attributes, including dose schedule, time to onset of protection, induction of improved immunogenicity, broader cross-protection across influenza A virus subtypes, and duration of protection. Use of approved or novel adjuvants to achieve enhanced or broadened immunogenicity may also be a component of the advanced development program.

Thank You for Your Attention

Robert Huebner, Ph.D.

Director of Influenza Division Biomedical Advanced Research and Development Authority (BARDA) US Department of Health and Human Services

330 Independence Avenue, SW Room G644 Washington, DC 20201

Robert.Huebner@hhs.gov

United States Department of

Health & Human Services Office of the Assistant Secretary for Preparedness and Response

Adam M. Clark, PhD.

SST Project Officer

BARDA

Strategic Science and Technology (SST) Innovations BAA: Science and Technology

Platforms Applied to Medical Countermeasure Development

ASPR: Resilient People. Healthy Communities. A Nation Prepared. 36

• History ─ Originally issued July 9, 2009 and re-issued January 1, 2011 and June 20, 2012 ─ BAA-13-100-SOL-00014 was issued July 31, 2013

• Success

─ Ten (10) contracts have been awarded across all of the previous four (4) areas of interest

─ ~$105M obligated to-date

• New Area of Interest for 2013

SST BAA Overview

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• Same overall intent as previous BAAs ─ Looking for new ideas that will improve our capabilities to prepare for and respond to public health emergencies ─ SST BAA focuses more on technologies and capabilities than developing products for specific threats (CBRN and flu focus on specific threats)

─ Support innovation through development of platform technologies that enhance capabilities for development and manufacturing of MCMs

• Narrowed focus for the upcoming year

SST BAA Intent

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• Specific interest exists in the promotion of science and technology advancements focused on technologies that facilitate administration of medical countermeasures during a public health emergency.

• The specific application of the platform technologies will be focused on biodefense, pandemic influenza, or other emerging infectious diseases (EID).

• Offerors should propose platforms having a maturity level equal to or greater than a Technology Readiness Level (TRL) of 3

• Submission Deadlines:

October 30, 2013 January 30, 2013 April 30, 2014 July 30, 2014

SST BAA

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• SST Area of Interest #1: Technologies that facilitate administration of medical countermeasures during a public health emergency ─ 1.1 Technologies, such as jet injectors or autoinjectors, that may allow vaccines or drugs to be delivered by parenteral administration with limited access to trained medical personnel resources

─ 1.2 Technologies, such as oral or transdermal delivery, that allow non-parenteral administration by the user and may deliver the countermeasure over a longer time interval

Area of Interest

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• SST-BAA@hhs.gov is the central mailing address for all communication

• Contract team ─ Elizabeth Steiner (Contracting Specialist)

• Program Team ─ Jonathan Seals (Director) ─ Alan Goldberg (CMI contractor) ─ Adam Clark ─ Stephanie Sincock ─ Mark Craven

SST BAA Team mailto:sst-baa@hhs.gov

Broad Agency Announcements (BAA)

AMCG Contracting Officer

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Process Overview

White Papers vs. Full Proposals White Papers strongly encouraged Following a White Paper submission, a decision letter is sent notifying the

Offeror whether a full proposal is requested

Timing White Paper Decision letters – 90 calendar days after submission deadline (or receipt) Cyclical review dates

Communication Contracting Officer will be the only point of communication “Blackout period” for communication with technical team

ASPR: Resilient People. Healthy Communities. A Nation Prepared. 43

Additional Information Available

Business Toolkit.

If you have not yet had a Government contract/award (or just new to BARDA), a “Toolkit” including samples and additional Earned Value Management information is available. The Business Toolkit can be found on PHE.gov , under the “About ASPR” tab, then “Office of Acquisition Management, Contracts & Grants” link.

Program and Contracting Office Availability.

In addition to TechWatch meetings and online resources, please feel free to contact the Contracting Professionals and Program Officers listed in the BAA, as appropriate.

ASPR: Resilient People. Healthy Communities. A Nation Prepared. 44

Helpful Information

New additions available this year:

Frequently Asked Questions (FAQ):

http://www.phe.gov/about/amcg/Documents/baa-faq.pdf

Information is available on Other Transaction Agreements, including sample agreements:

http://www.phe.gov/about/amcg/Pages/default.aspx http://www.phe.gov/about/amcg/Documents/baa-faq.pdf http://www.phe.gov/about/amcg/Pages/default.aspx

ASPR: Resilient People. Healthy Communities. A Nation Prepared. 45

Conclusion

Thank you for your interest in BARDA and the BAA process.

PHEMCE:

URL: http://www.medicalcountermeasures.gov BARDA e-mail: BARDA@hhs.gov

• Upcoming Events

• PHEMCE Information and Reports

• CBRN and Pan Flu Programs

Funding opportunities Resources and core service programs Regulatory information

• Please exit through the rear of the auditorium into the Great Hall

• For those who scheduled one-on-one meetings with BARDA, there will be four sessions following the presentations. Meetings will be kept to 15 minutes.

Please be on time.

• AMCG staff will be available for contracting questions

• Reminder: All guests of the Humphrey building need to be escorted by ASPR Staff when leaving this area (i.e. bathroom/anywhere)

Session Closing Notes

ASPR: Resilient People. Healthy Communities. A Nation Prepared. 48

BARDA Industry Day

November 12-14, 2013

Washington, DC metro area

Save the Date

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