United States Department of
Health & Human Services
Office of the Assistant Secretary for Preparedness and Response
BARDA 2013-2015:
Goals, Priorities,
Successes & Challenges
North Carolina Biodefense Summit
August 26, 2014
Dr. Robin Robinson
BARDA Director
ASPR Deputy Assistant Secretary
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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Man-Made & Mother Nature’s Threats
Have Become Reality Since 9/11
MERS-CoV
H5N1
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
1
Ebola 2014:
Worst Outbreak in History
MERS-CoV
H5N1
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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Drug Development is
Expensive, Lengthy, & Risky
IND
PHASES
Discovery
Preclinical
Development
NDA/BLA
Phase I
Phase II
Phase III
NIH
NIH ($11.8B)
Licensure
Production
& Delivery
Project BioShield ($5.6B)
BARDA
ARD
BARDA
($540M)
PRODUCT PIPELINE
Valley
of
Death
Licensed
Product
1-3%
5-17%
10-25%
18-35%
45-70%
90%
PROBABILITY OF SUCCESS TO LICENSURE
TIME
PIPELINE
PHASE COST
3-7 yr
$100M -130M
0.5-2 yr
$60-70M
1-2 yr
$70M-100M
2-3.5 yr
$130M-160M
2.5 -4 yr
1-2 yrs
$190M-220M
$18M-20M
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA’s Commitment to
Medical Countermeasures
BARDA Mission: Support
development of and make available
countermeasures for CBRN threats,
pandemic influenza, and emerging
infectious diseases through product
development , innovation, and
acquisition, stockpiling, building
manufacturing infrastructure, & core
service assistance
O’Neil Building
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
BARDA 2013: BARDA Built a Formidable
& Robust MCM Development Pipeline
•
150+ MCM candidates in development through 2013
─ Using better business practices (e.g. OTA, IPR)
─ 12 new CBRN MCM candidates and 5 new H7N9 vaccine candidates in FY2013
•
•
•
12 MCMs provided under Project BioShield since 2004 with 12 more by 2019
National stockpiles of H5N1 & H7N9 pre-pandemic vaccines & adjuvants
7 FDA-approved MCMs in 2012-13 with more expected by the end of 2015
─ Simplexa® influenza & RSV diagnostic POC device (Jun. 2012) – 3M/Focus [510K
clearance]
─ Flucelvax cell-based influenza vaccine (Nov. 2012) – Novartis [licensure]
─ Raxibacumab anthrax antitoxin (Dec. 2012) – GlaxoSmithKline/Human Genome
Sciences [approval]
─ Aura portable ventilator (Dec. 2012) –Covidien [510k cleared for adults]
─ FluBlǾk recombinant-based influenza vaccine (Jan. 2013) – Protein Sciences
─ HBAT botulinum antitoxin (Mar. 2013) – Cangene [licensure]
─ Q-PAN H5N1 pandemic influenza vaccine (Nov. 2013) – GlaxoSmithKline [licensure]
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Has Established Robust
CBRN MCM Development Pipeline
• BARDA CBRN MCM development pipeline has supported
85+ candidates since 2004 ($2.2 B)
• Biothreats
•
•
•
•
•
Anthrax vaccines (7) and antitoxins (7)
Smallpox vaccine (3) and antiviral drugs (2)
Botulinum antitoxin (1)
Other biothreat antimicrobial drugs (9)
Viral hemorrhagic fever (1)
• Rad/Nuc threats
•
•
•
•
Acute Radiation Syndrome drugs (36)
Decorporation agents (6)
Thermal burn therapies (9)
Biodosimetry devices (11)
• Chem threats – antidotes & decon (4)
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA MCMs Developed & Purchased
under Project BioShield
Smallpox
Radiation
Anthrax
Chemical
Botulism
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Influenza Vaccine Development Strategy
Has Delivered More & Better Vaccine Sooner
“Universal”
Vaccines
Recombinant
Vaccines
FluBlØk
Licensed 01/16/13
Cell-based
Vaccines
Egg-based
Vaccines
Flucelvax
Licensed 11/20/12
H5N1 Vaccine
Licensed 04/17/07
FOUO- Procurement Sensitive
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Advanced
Development
Start FY15
FDA-Approved BARDA Influenza MCMs
Cell-based
Influenza Vaccine
Q-PAN H5N1 Vaccine
GlaxoSmithKline
Novartis
Next-Gen Portable
Ventilators
Covidien
Recombinant-based
Influenza Vaccine
Flu/RSV
POC Diagnostic
Protein
Sciences
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
3M/Focus
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BARDA MCM Partnerships
• BARDA has established multiple solid public-private partnerships
with industry/academia to expand domestic & international manufacturing
response capabilities
• Retrofitted manufacturing facilities
─
─
─
─
Sanofi – PA
MedImmune – CA and PA
CIADM TAMUS NTSB – TX
CIADM Emergent - MD
• New manufacturing facilities (Novartis – NC)
• WHO and PATH for flu vaccine manufacturing facilities in developing
countries
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Uses Public-Private Partnerships to
Build Domestic MCM Manufacturing Capacity
• Expanding Existing Capacity
by Retrofitting Vaccine
Manufacturing Infrastructure
sanofi pasteur – Swiftwater, PA
• Changing Flu Vaccine Industry
Novartis – Holly Springs, NC
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Expanded Domestic Flu Vaccine
Manufacturing Surge Capacity Multi-fold
USG pandemic influenza vaccine policy is two doses for everyone
(~ 600 M doses) within 4 months of pandemic onset.
800
700
Rec 3
600
Rec 2
Cell 2
500
Red 1
Cell 1
400
E 5gg
Egg 4
300
Egg 3
Egg 2
200
Egg 1
100
0
2005
2009
2014
2018
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Expands Flu Vaccine Manufacturing
Capacity in Developing Countries
Romania
Cantacuzino
Institute
Kazakhstan
RIBSP
Utah
State
N.C.
State
Serbia
Torlak
Institute
Mexico
Birmex
Egypt
VASERA
BARDA Training Sites
Vietnam
IVAC
VABIOTECH
PATH
India
Serum
Institute
Thailand
GPO
Brazil
Instituto
Butantan
South Korea
Green Cross
Indonesia
Bio Farma
South
Africa
Biovac
Licensed/Active Influenza Vaccine Producers
BARDA/WHO Cooperative Agreement Grantees
BARDA/WHO Licensed Pandemic Vaccine for Human Use as of 2013
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA 2014: CBRN & Influenza
MCM Programs Stay the Course
• BARDA CBRN MCM program will continue support for existing
projects as warranted and funds are available and begin support for
new projects on following:
─ Viral hemorrhagic fever therapeutics - NEW
─ Chemical antidotes
─ Broad spectrum antimicrobial drugs
•
BARDA will support the purchase of the following MCMs under Project
BioShield:
─ Smallpox vaccines
─ Anthrax antitoxins
─ Anti-neutropenia cytokines (Vendor-managed inventory)
•
BARDA Influenza MCM program will continue support for existing/new
projects in the following areas:
─ Vaccine
• cell-based, recombinant-based, antigen-sparing
• egg supply
• stockpiling
─ Antivirals (host-targeted)
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
─ Diagnostics
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BARDA Provides Critical Core Service
Assistance to MCM Developers
Centers for
Innovation in
Advanced
Development &
Manufacturing
2012
Regulatory &
Technical
Expertise
Fill Finish Mfg.
Network
2006
15
2013
Modeling
Hub
Animal
Studies
Network
2009
2010
Strategic
Investor
Clinical
Studies
Network
TBD
2014
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
BARDA Assists MCM Developers
thru our Animal Models Network
Battelle Pacific
Northwest
Division
IIT Research Institute
University of Illinois, Chicago
Battelle Memorial Institute
SRI
International
BioQUAL, Inc.
Southern Research
Lovelace
Respiratory
Research
Institute
MRIGlobal
University of Texas
Medical Branch
Defence Science and Technology
Laboratory, Porton Down
Health Protection Agency, Porton Down
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Assists MCM Developers Directly with
Product Development & Manufacturing
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Assists MCM Developers thru our
New Fill Finish Manufacturing Network
Cook
Pharmica
JHP/Par
Pharmaceuticals
DSM/JLL
Pharmaceuticals
Nanotherapeutics/Baxter
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Assists MCM Developers thru our
New Clinical Studies Network
CRM
EMMES
TRI
PPD
Rho
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA 2015: Harvesting ARD Bounty for PBS
& Moving the Frontiers of Influenza MCMs
• CBRN MCM Program:
─ Continue existing projects
─ Expand BSA Program to combat antimicrobial drug resistance
─ Launch New Viral Hemorrhagic Fever Vaccine Program
• Project BioShield: New MCMs from BARDA ARD
─ Artificial skin for thermal and radiation burns New
─ Biodosimetry device for measuring radiation exposure in persons within
hours using biomarker assays New
─ Chemical antidotes for cyanide poisoning New
─ Replenishing anthrax antitoxins, smallpox vaccines, & anti-neutropenia
cytokines
• Influenza MCM Program:
─ Continue existing projects
─ Launch New Universal Influenza Vaccine Development Program
─ Launch New Influenza Immunotherapy Program for Critically Ill
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Investments in the
North Carolina MCM Enterprise
• CBRN MCM Programs ($109 M)
─ Biothreats
•
•
Chimerix (2011 – current) smallpox antiviral drug
Cempra (2013 – current) 4th generation macrolide
─ Radiation Threat
• Parion Sciences (2008) – Ling Trauma
•
•
•
Duke University (2009 – current) – gene expression biodosimetry device
Keranetics (2012 – current) thermal burn gel
Novan (2013 –current) thermal burn ancillary
─ Chemical Threat Agents
•
Countervail (2011 – current) galantamine
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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BARDA Investments in the
North Carolina MCM Enterprise
• Influenza MCM Programs ($1.3 B)
─ Vaccines
•
•
Novartis (2009 – current)
─ Cell-based flu vaccine development & mfg. for federal stockpile
─ MF59 adjuvant development & mfg. for federal stockpile
─ New intl. award-winning cell-based vaccine & biologics R&D /mfg. facility
North Carolina State University (2010 – current) vaccine mfg. training
─ Antivirals
•
GlaxoSmithKline (2006- current) Relenza
─ Antiviral drug development
─ Antiviral drug manufacturing for federal & state stockpiles
• Core Service Programs ($61 M)
─ Centers for Innovation In Advanced Development & Manufacturing
•
Novartis (2012 – current)
─ Fill Finish Manufacturing Network
•
DSM (2013 – current)
─ Clinical Studies Network
•
•
Pharmaceutical Product Development (PPD) (2014-current)
Rho Federal Systems (2014-current)
ASPR: Resilient People. Healthy Communities. A Nation Prepared.
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Contact Us
BARDA:
URL: http://www.phe.gov
BARDA e-mail: [email protected]
• Upcoming Events
• PHEMCE Strategy and Implementation Plan
• CBRN and Pan Flu Programs
• Business Toolkit
─ www.phe.gov/amcg
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Tech Watch program
Federally-sponsored conferences
Funding opportunities
Resources 7 core service programs
Regulatory guidance
Federal strategies and reports
ASPR: Resilient People. Healthy Communities. A Nation Prepared.