Critical Path to TB Drug Regimens (CPTR)

Transcription

1 Critical Path to TB Drug Regimens (CPTR)

2 Why do we need a CPTR initiative? We finally have a pipeline of TB drug candidates Today, we test drugs same way we did 20 years ago Need better tests for drug safety Need more efficient and accurate measures of effectiveness Need to monitor disease response instead of symptom response

3 Critical Path to TB Drug Regimens (CPTR) CPTR is a broad collaboration of pharmaceutical companies; government, regulatory, and multilateral agencies; donors; academia; advocates; and NGOs that aims to accelerate the development of new, safe, and highly effective tuberculosis (TB) treatment regimens with shorter therapy durations. CPTR will form innovative partnerships to significantly accelerate delivery of new TB medicines an urgent public health need with the goal of saving millions of lives.

4 CPTR Mission and Vision VISION To reduce the global burden of TB by developing TB regimens that are of short duration, more rapidly effective and safer than currently available treatments. MISSION To bring innovation to TB drug development by creating consensus and qualifying new regulatory science tools To develop new TB drug combinations To ensure optimal infrastructure for the CPTR Initiative

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6 CPTR Initiative BMGF, C-Path, TB Alliance and its partners will work to accelerate the development of new TB drug regimens CPTR RegSci Consortium Regulatory Science Data standards/integration Qualified biomarkers Disease progression models Pharma companies TB Alliance TB experts Regulators Patient representatives Others? CPTR Drug Coalition Drug Development Focus New clinical trial designs Drug combination testing and development Participants Pharma companies TB Alliance TB experts Others CPTR Infrastructure Key Success Factors Clinical trial capacity Regulatory harmonization Funding Pharma companies TB Alliance Regulators Patient representatives Reagan-Udall Foundation NIH CDC BMGF Other funders

7 CPTR Initiative Infrastructure Clinical trial capacity, regulatory harmonization & funding Drug regimen development New TB Drug Combinations Regulatory Science Consortium Greater Efficiency & Safety 7

8 CPTR: How will success look? Qualified tools that maximize learning and minimize delay in the testing of new TB drugs in combination and individually Novel regimens for treatment of DS and MDR TB approved by regulatory agencies and endorsed by the WHO The CPTR approach will become the gold standard for rapid, safe, and efficient testing and development of new TB drug combinations 8

9 CPTR: A Critical Path Project 9

10 C-Path s Consortia Model FDA EMEA Other Reg. Agencies Multiple Companies A B Patients Formal Legal Agreement C D E C-Path Research Academia Precompetitive Neutral ground 10

11 C-Path s Consortia Predictive Safety Testing Consortium (PSTC) SAFETY Patient-Reported Outcomes (PRO) EFFICACY Coalition Against Major Diseases (CAMD) SHARING DISEASE KNOWLEDGE 11

12 Participants in C-Path s Consortia 30 Major Pharmaceutical Companies FDA, EMEA, NIA, NINDS, NCI, NHLBI Six Patient Advocacy Organizations 12

13 Qualification of New Regulatory Science Tools A new pathway.. FDA Advice FDA Advice Planning Phase Execution Phase FDA Review Phase Legal Agreement Coordinating Work Scope Working Methods & FDA FDA Results Submission Review Qualified Methods Committee Document Groups Sharing Planning & 1. BQRT Writing Groups Scientific Consensus Greater Efficiency & Safety

14 CPTR Tools Consortium: Objective Develop a scientific consensus on which tools are qualified for use in drug regimen development Find consensus among 1) those who will use the methods (industry), 2) those who will accept the methods (FDA, EMEA, others) 14

15 CPTR RegSci Consortium: Proposed Working Groups Data Standards and Integration Biomarkers and Endpoints Disease Progression Models Mycobacterial Assays Pharmacokinetics and pharmacodynamics including drug interactions Others 15