Op­er­a­tion Warp Speed for rare dis­eases: CBER leader says pi­lot is com­ing soon

The next gen­er­a­tion of Op­er­a­tion Warp Speed is com­ing soon, and this time it’s go­ing to take aim at rare dis­eases, Pe­ter Marks, the di­rec­tor of the FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search, told at­ten­dees of the Bio­phar­ma Con­gress in Wash­ing­ton, DC on Mon­day.

“We’re get­ting ready to pi­lot that pro­gram,” Marks said, mak­ing clear the goal is to help ar­eas with high un­met needs.

I think the goal is to take a drug with some promise in the rare dis­ease space for dis­eases that don’t have al­ter­na­tives … and then take these prod­ucts, maybe they have break­through or ad­vanced ther­a­py des­ig­na­tion, they have promise and there is a prod­uct in de­vel­op­ment and not just a con­cept. And then give them the op­por­tu­ni­ty with not just chem­istry, man­u­fac­tur­ing and con­trols, which we have a pi­lot for, but al­low the clin­i­cal de­vel­op­ment to hap­pen in con­stant com­mu­ni­ca­tion and shar­ing of po­ten­tial re­sults be­fore a sub­mis­sion of an NDA or BLA. That’s the idea, is to move things as fast as pos­si­ble.

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