A new paradigm for TB drug development? A regulator’s perspective
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Transcript of A new paradigm for TB drug development? A regulator’s perspective
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An agency of the European Union
A new paradigm for TB drug development?
A regulator’s perspective
Hans-Georg Eichler
Washington, October 2012
CPTR
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What’s in this talk
• Research 101
• The message from the gatekeeper-regulator: It can’t be done
• The message from the enabler-regulator: It can be done - maybe
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Research 101
• Temperature• Concentration of substrate• Concentration of inhibitor• …
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Experiment: all factors except one are kept constant
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Too many steps at once?
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(Too) many unknown variables?
Endpoint: surrogate (predictive?)
Unit of development = regimen (new-new combo)
Treatment shortening potential?
Optimal dosage/ combination?
Post-licensing utilisation?5
Populations (DS, MDR, XDR?)
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Too many unknown variables
Please solve this equation and,by the way, all variables are unknown…
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“It can’t be done !”
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Learn – confirm - license
The drug development / licensing paradigm
Learn – license - confirm
Lifecycle approach to research and licensing
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(Too) many unknown variables?
Develop BM qualification program
Endpoint: surrogate (predictive?)
Plan to define B/R of components(?)
Unit of development = regimen (new-new combo)
Long term follow upTreatment shortening potential?
Parallel /Subsequent RCTs; Registries
Optimal dosage/ combination? Post-lice. observation
Post-licensing utilisation? Restricted distribution8
Populations (DS, MDR, XDR?)
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but show me: • a complete development plan including the (post
licensing) ‘confirming’ phase• a BM (endpoint) qualification plan• infrastructure is in place to monitor post-
licensing treatment experience• infrastructure is in place to monitor post-
licensing drug utilisation• infrastructure is in place to manage post-
licensing drug utilisation
Maybe it can be done…
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Thank you!(EMA, Canary Wharf, London)