Pathways for Medical Countermeasure Development and Use
Luciana Borio, M.D. Assistant Commissioner for Counterterrorism and Emerging Threats Director, Office of Counterterrorism and Emerging Threats (OCET)
Food and Drug Administration
BARDA Industry Day - Contracting for Countermeasures December 10, 2012
Objective
• Provide the FDA perspective on goal 2 of the 2012 PHEMCE Strategy
• Goal 2 - Establish and communicate clear pathways to facilitate medical countermeasure development and use
– Objective 2.1 - Identify scientific and regulatory issues that challenge
medical countermeasure development or use during public health emergencies and coordinate activities among PHEMCE partners to address those challenges
– Objective 2.2 - Assist medical countermeasure developers in working
interactively with FDA during product development and regulatory review
Regulatory Mechanisms for the Development and Use of MCMs
• The goal for MCM development is approval, licensure, or clearance
• In situations where MCMs are available but not yet approved, licensed, or cleared for the particular CBRN indication, FDA has a variety of regulatory mechanisms to facilitate access to MCMs:
– Investigational Use (e.g., expanded access) – Emergency Use Authorization
• All of FDA’s regulatory mechanisms for MCMs are based on risk-benefit assessments that are founded on the available scientific evidence
Scientific and Regulatory Issues that Challenge MCM Development or Use
• FDA’s regulatory review of MCMs for approval, licensure, clearance or authorization is data-driven
• There are often gaps in scientific knowledge that result in uncertainties that impede or prevent regulatory review
• Gaps in scientific knowledge arise from multiple sources including: – Intrinsic uncertainties about the biologic behavior of threat agents – Insufficient drug development tools (e.g., animal models) for establishing the
necessary data to support regulatory decision-making
• FDA is aggressively working with PHEMCE partners and product developers to identify and narrow these scientific gaps
Pillar I Pillar II Pillar III
Enhance the Medical Countermeasure
Regulatory Review Process
Advance Regulatory Science for Medical
Countermeasure Development And
Evaluation
Modernize the Legal, Regulatory, and Policy
Framework for Effective Public Health Response
OCET
CBER, CDER, CDRH
PHEMCE
FDA Medical Countermeasures Initiative
Resolving Scientific and Regulatory Uncertainties
Selected MCMi Accomplishments
8/1/2010 4/30/2013Nov 11Passage of
HR2112 (MCMi Base Funding)
Dec 11Smallpox Drugs AC
Nov 12Burkholderia Workshop
Sep 12FDA Joins
NICBR
Mar 12ASPR MOU
Nov 12Raxibacumab
AC
Sep 12Clearance of non-Variola
Orthopoxvirus Assay
Jun 11Animal Model Qualification
Program Launch
Jul 11Issuance of
Doxy EUA
May 12Issuance of BAA
Jun 121st Regulatory
Science Symposium
Apr 13Pilot Course on GLP-Equivalent
Standards in BSL 3/4
Apr 12Approval
of Levaquin for Plague
Aug 10 - Apr 11Pan Flu/Dual Use Restricted
Apr 12Plague
MCMs AC
Sep 121st Award
Under BAA
May 11Submission of Proposals for
PAHPA Reauthorization
to HHS
Oct 11Multiplexed
IVD Workshop
Sep 11Close of RFI
Sep 12Radiation
Biodosimety Workshop
Sep 12Action Plan
for Intra-Event
Surveillance
Sep 11Smallpox Vaccines Workshop
Aug 11Posting of RFI
Nov 12GSK H5N1 Vaccine AC
Aug 12Issuance of
UTMB Contract
Apr 12Collaboration with
DTRA, NCBI, LLNL on Reference
Database
Aug 10MCMi
Launch
Jul 12Completion of
Review of pre-EUA for MVA
Feb 12Pediatric MCMs
Workshop
Apr 11Passage of
HR1473
Jan 11Intramural MCMi Reg
Sci Program Launch
Feb 11DARPA MOU
Mar 11IOM
Meeting
Nov 12Clearance of
Aura Ventilator
Nov 12Licensure of
Flucelvax
May 132nd Regulatory
Science Symposium
Broad Agency Announcement
• Solicitation for research and development to support regulatory science and innovation including for MCMs (FDA BAA-12-00118) – Develop, characterize, and qualify animal
models for MCM development – Modernize tools to evaluate MCM
product safety, efficacy, and quality – Develop and qualify biomarkers of
diseases or conditions – Enhance emergency communication
• Remains open until May 23, 2013
Assisting MCM Developers During Product Development Regulatory Review
• FDA assists MCM developers via a number of methods including: – Formal and informal meetings with product sponsors or applicants seeking
technical assistance related to the development, regulatory assessment, and manufacturing of MCMs
– Enhanced inspection, including pre-approval and compliance activities to support early identification of problems that might impede MCM development /availability
– Issuance of guidance documents and regulations to guide MCM developers – Stakeholder engagements, including meetings, conferences, and workshops,
to educate the public on both FDA regulatory processes and its current thinking on regulatory issues and to garner input from interested parties on regulatory issues
– Advisory Committee meetings to obtain independent expert advice on scientific, technical, and policy matters on specific topics
