Cures Act im­ple­men­ta­tion: Got­tlieb says faster can­cer drug ap­provals com­ing for sec­ondary in­di­ca­tions

FDA Com­mis­sion­er Scott Got­tlieb and NIH Di­rec­tor Fran­cis Collins tes­ti­fied on Thurs­day be­fore the House En­er­gy & Com­merce Com­mit­tee on how their agen­cies have worked to im­ple­ment pro­vi­sions from the 21st Cen­tu­ry Cures Act passed and signed by Pres­i­dent Barack Oba­ma a month be­fore he left of­fice.

The hear­ing cen­tered on the mam­moth new law that in­cludes ma­jor tweaks to how FDA reg­u­lates drugs, de­vices and bi­o­log­ics, and pro­vides the agency with $500 mil­lion over 10 years to im­ple­ment it.

In open­ing com­ments, Got­tlieb told the com­mit­tee that the agency is work­ing on a pro­pos­al where­by can­cer treat­ments al­ready ap­proved for one in­di­ca­tion might be able to win ap­proval for a sec­ond in­di­ca­tion based on a sup­ple­men­tal ap­pli­ca­tion that re­lies on “a more tar­get­ed da­ta set, like a sin­gle-arm study. We in­tend to is­sue guid­ance fur­ther clar­i­fy­ing the cir­cum­stances in which this is ap­pro­pri­ate.

“This may be suit­able, for ex­am­ple, when there’s a clear and out­sized treat­ment ef­fect, and the sec­ond in­di­ca­tion con­cerns the same dis­ease as the first one, but for a new set­ting. For ex­am­ple, a tar­get­ed drug ap­proved for a third-line use that shows ben­e­fit in a sec­ond-line in­di­ca­tion,” Got­tlieb added.

In the ques­tion and an­swer por­tion of the hear­ing, Got­tlieb not­ed an­oth­er new guid­ance to be re­leased “very soon” on tar­get­ed ther­a­pies, out­lin­ing how to win ap­proval for drugs tar­get­ed at bio­mark­ers, “such as a can­cer that might ap­pear in mul­ti­ple or­gans.”

He al­so not­ed that since the pas­sage of the Food and Drug Ad­min­is­tra­tion Safe­ty and In­no­va­tion Act (FDA­SIA), the agency has been able to grant ac­cel­er­at­ed ap­proval based on in­ter­me­di­ate clin­i­cal end­points.

“We want to bet­ter de­fine what’s meant by in­ter­me­di­ate end­points to en­sure that prod­uct de­vel­op­ers with promis­ing drugs take full ad­van­tage of this pro­vi­sion and can con­sid­er it in a broad­er range of such set­tings,” he said.

In praise of FDA’s On­col­o­gy Cen­ter of Ex­cel­lence, Got­tlieb added that it was in­stru­men­tal in the re­cent ap­provals of two new CAR-T cell ther­a­pies, which he said could be trans­for­ma­tive: No­var­tis’ Kym­ri­ah (ti­s­agen­le­cleu­cel) and Kite Phar­ma’s Yescar­ta (axi­cab­ta­gene ciloleu­cel).


First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

Author

Zachary Brennan

managing editor, RAPS

Daniel O'Day

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