cptr overview cptr 2012 workshop october 2-4, 2012

CPTR Overview

CPTR 2012 Workshop October 2-4, 2012

The Challenge

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Accelerate the development of new, safe, and highly effective regimens for TB by enabling early testing of

drug combinations.

CPTR Mission

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http://www.gatesfoundation.org/Pages/home.aspx

CPTR Working Group Structure

[Enter Presentation Title in Insert Tab > Header & Footer 4

• Data Standards & Integration

WG

• Biomarkers & Clinical Endpoints

WG

• Preclinical & Clinical Sciences WG

• Modeling and Simulation WG

• Health Authorities Submission WG

Regulatory Science Consortium

Research Resources Group

Drug Development Coalition

• Global Regulatory Pathways WG

• Stakeholder & Community Engagement WG

• Access & Appropriate Use WG

• Clinical Trials Infrastructure WG

Integrated Sciences Team

CPTR Structure




ADVISO

RY PANEL

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CPTR Key Accomplishments:Regulatory Science Consortium

CPTR 2012 Workshop October 2-4, 2012


CPTR Regulatory Science Consortium Governance




Coordinating Committee

Government/Regulatory participants

Non-profit research membersIndustry members

Regulatory Science Consortium Members and Partners

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Research Resources

Group

Drug Development

Coalition

Our role in accelerating the process includes :• Identify tools and methods that can bring the most value• Reach scientific consensus through sharing of expertise,

information and data• Proceed to regulatory qualification when appropriate as an

outcome9

Regulatory Sciences Structure and Role

Regulatory Science

Consortium

Biomarkers and Clinical Endpoints

TB Data Standards and

Integration

Health Authority

Submissions

Pre-Clinical and Clinical

Sciences

Modeling andSimulation

Regulatory Authority

Input

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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]

TB Data Standardsand Integration

Pre-Clinical and Clinical Sciences

Key Accomplishments


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



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]

TB Data Standardsand Integration

Pre-Clinical and Clinical Sciences

Key Accomplishments

Modeling and Simulation Work Group

Continuously revise and refine with relevant data (summary and subject-level)

Hollow fiber model Physiologically-based PK models

Clinical trial simulation tools (drug-disease-trial models)

PreclinicalPKPDRight

Molecule

Vision: More efficient translation between each stage of drug development

ClinicalPharmacology

RightDose

Pharmaco-metrics

RightPatients

Pharmacology

RightTarget

NetworkBiology

RightPathway

Pathway Target Drug Disease

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Today:CPTR 2012

Workshop

CPTR 2013

Workshop

TB Data Standards

v 1.0 Published

TB Data Standards Repository Launched

Remap TB Data & Load to

RepositoryS22, 27, 28

Publish DPM-WG

papers

Publish Preclinical Sciences Papers

Submit LOIto FDA for

Hollow Fiber Qualification

Begin Briefing Package for

Hollow Fiber Qualification

Identify Next Set of

DDTs for Qualification

Begin Briefing Package for

Liquid Culture Qualification

Submit LOI to FDA for Liquid

Culture Qualification

Milestone Horizon

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Research Resources

CPTR Structure

Regulatory Science

Consortium


Drug Development

Coalition

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cptr overview cptr 2012 workshop october 2-4, 2012

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