An agency of the European Union

A new paradigm for TB drug development?

A regulator’s perspective

Hans-Georg Eichler

Washington, October 2012

CPTR

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

2

Research 101

• Temperature• Concentration of substrate• Concentration of inhibitor• …

3

Experiment: all factors except one are kept constant

Too many steps at once?

(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?)

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 !”

7

Learn – confirm - license

The drug development / licensing paradigm

Learn – license - confirm

Lifecycle approach to research and licensing

(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…

10

Thank you!(EMA, Canary Wharf, London)