Download Presentation - Critical Path to TB Drug Regimens

CPTR 2012 Workshop October 2-4, 2012
CPTR Overview
The Challenge
2
CPTR Mission
Accelerate the development of new, safe, and highly
effective regimens for TB by enabling early testing of
drug combinations.
3
CPTR Working Group Structure
• Data
Standards
& Integration
WG
Regulatory
Science Consortium
• Biomarkers &
Clinical Endpoints
WG
Research
Resources Group
Integrated
Sciences Team
• Preclinical & Clinical
Sciences WG
• Modeling and
Simulation WG
• Clinical
Trials
Infrastructure
WG
• Global
Regulatory
Pathways WG
• Stakeholder & Community
Engagement WG
Drug Development
Coalition
• Access & Appropriate Use
WG
• Health Authorities
Submission WG
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CPTR Structure
Research
Resources Group
Regulatory
Science Consortium
ADVISORY PANEL
Drug Development
Coalition
5
CPTR 2012 Workshop October 2-4, 2012
CPTR Key Accomplishments:
Regulatory Science
Consortium
CPTR Regulatory Science Consortium Governance
Coordinating Committee
Regulatory
Science Consortium
Research
Resources Group
Drug Development
Coalition
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7
Regulatory Science Consortium
Members and Partners
Government/Regulatory
participants
Industry members
Non-profit research
members
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Regulatory Science Consortium
Regulatory
Science Consortium
Research
Resources
Group
Our role in accelerating the process includes :
• Identify tools and methods that can bring the most value
• Reach scientific consensus through
sharing of expertise,
Drug
Development
information and data
Coalition
• Proceed to regulatory qualification
when appropriate as an
outcome
9
Regulatory Sciences Structure and Role
Biomarkers
and Clinical
Endpoints
Modeling and
Simulation
Pre-Clinical
and Clinical
Sciences
Regulatory
Science
Consortium
TB Data
Standards and
Integration
Health
Authority
Submissions
Regulatory
Authority
Input
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Key Accomplishments
•
TB Data Standards
and Integration
Biomarkers and
Clinical
Endpoints
Pre-Clinical and
Clinical Sciences
Modeling and
Simulation
•
•
•
•
•
•
•
TB Data Standard Version 1.0 launched and available for
use on CDISC site (3Q2012)
TB data repository developed and launched (3Q2012)
for remapping of key data sets to the new standard
Liquid Culture to be proposed as a predictive or
prognostic biomarker for clinical outcome (2Q2012)
Letter of Intent submitted to FDA (3Q2012)
Evaluation of key pre-clinical efficacy models for early
selection of combination partners complete (2Q2012)
In vitro hollow fiber model selected and context of use
for this tool is under development (3Q2012)
Existing TB progression models evaluated and review
manuscript submitted (2Q 2012)
Develop a PBPK module to evaluate drug distribution in
the infected lung [2Q2013 and ongoing]
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TB Data Standard
• Clinical data standard used/preferred by FDA review divisions
• Enable data sharing between organizations
• Enable aggregation and querying of data
• When implemented from the start, can lower costs
acquiring and analyzing data
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12
Key Accomplishments
•
TB Data Standards
and Integration
Biomarkers and
Clinical
Endpoints
Pre-Clinical and
Clinical Sciences
Modeling and
Simulation
•
•
•
•
•
•
•
TB Data Standard Version 1.0 launched and available for
use on CDISC site (3Q2012)
TB data repository developed and launched (3Q2012)
for remapping of key data sets to the new standard
Liquid Culture to be proposed as a predictive or
prognostic biomarker for clinical outcome (2Q2012)
Letter of Intent submitted to FDA (3Q2012)
Evaluation of key pre-clinical efficacy models for early
selection of combination partners complete (2Q2012)
In vitro hollow fiber model selected and context of use
for this tool is under development (3Q2012)
Existing TB progression models evaluated and review
manuscript submitted (2Q 2012)
Develop a PBPK module to evaluate drug distribution in
the infected lung [2Q2013 and ongoing]
[Enter Presentation Title in Insert Tab > Header & Footer
13
Modeling and Simulation Work Group
Vision: More efficient translation between each stage of drug development
Continuously revise and refine with relevant
data (summary and subject-level)
Network
Biology
Right
Pathway
Pharmacology
Right
Target
Pathway
Preclinical
PKPD
Right
Molecule
Target
Hollow fiber model
Clinical
Pharmacology
Right
Dose
Drug
Pharmacometrics
Right
Patients
Disease
Clinical trial simulation
Physiologicallytools (drug-disease-trial
based PK models
models)
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Milestone Horizon
Today:
CPTR
2012
Workshop
Submit LOI
to FDA for
Hollow Fiber
Qualification
TB Data
Standards
Repository
Launched
TB Data
Standards
v 1.0
Published
CPTR
2013
Workshop
Submit LOI
to FDA for
Liquid
Culture
Qualification
Begin Briefing
Package for
Liquid Culture
Qualification
Begin
Briefing
Package for
Hollow Fiber
Qualification
Publish
Preclinical
Sciences
Papers
Publish
DPM-WG
papers
Identify
Next Set of
DDTs for
Qualification
Remap TB
Data &
Load to
Repository
S22, 27, 28
15
Research Resources
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CPTR Structure
Research
Resources Group
Regulatory
Science
Consortium
Drug
Development
Coalition
17