The FDA on Wednes­day kicked off a two-day meet­ing fo­cused on what's next for can­cer drug reg­istries, the of­ten messy de­pos­i­to­ries of raw pa­tient da­ta that are in­creas­ing­ly be­com­ing more use­ful in sup­port­ing ap­proval de­ci­sions.

FDA of­fi­cials opened the dis­cus­sion with some of the lim­i­ta­tions of reg­istries, de­pend­ing on their scope and de­sign, and oth­er ques­tions re­lat­ed to bias, da­ta het­ero­gene­ity, their rep­re­sen­ta­tion of the gen­er­al pa­tient pop­u­la­tion, and vary­ing da­ta qual­i­ty.

Even so, reg­istries have been used to sup­port new can­cer drug ap­provals. With the pro­lif­er­a­tion of more EHR and oth­er re­al-world hos­pi­tal and treat­ment da­ta, the FDA made clear that their use­ful­ness will like­ly progress.

Over­all sur­vival da­ta should be in­clud­ed in all ran­dom­ized can­cer drug clin­i­cal tri­als, the FDA said in new draft guid­ance.

Call­ing over­all sur­vival (OS) "a gold stan­dard end­point," the 15-page draft guid­ance re­leased last week says the mea­sure is nec­es­sary to "ad­e­quate­ly eval­u­ate the po­ten­tial for harm." OS is gen­er­al­ly de­fined as the time from tri­al ran­dom­iza­tion to death from any cause.

The push for more OS da­ta comes as the FDA's On­col­o­gy Cen­ter of Ex­cel­lence has al­lowed dif­fer­ent types of pri­ma­ry end­points to be used in piv­otal tri­als, es­pe­cial­ly when there are few or no oth­er treat­ment op­tions. This is the agen­cy's first draft guid­ance on the as­sess­ment of OS in can­cer drug tri­als, but agency of­fi­cials have made clear since at least 2023 that, "ven when ear­li­er end­points have been used to sup­port ap­proval, the FDA al­ways eval­u­ates OS if ap­proval is based on a ran­dom­ized tri­al."

Vinay Prasad, who briefly left his role as head of the FDA's Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search, has come back to the agency with­out his for­mer ad­di­tion­al ti­tles of chief med­ical and sci­en­tif­ic of­fi­cer.

FDA Com­mis­sion­er Mar­ty Makary orig­i­nal­ly gave him the ex­pan­sive roles to help lead on “cross-cut­ting and emerg­ing med­ical and sci­en­tif­ic is­sues im­pact­ing reg­u­la­to­ry sci­ence and pub­lic health.”

HHS did­n't re­spond to a ques­tion on why Prasad no longer holds those ti­tles, but it con­firmed that his up­dat­ed FDA pro­file is ac­cu­rate. Prasad did­n't re­spond to a re­quest for com­ment.

Prasad's sur­prise ex­it last month fol­lowed a string of con­tro­ver­sies in­volv­ing a con­ser­v­a­tive ac­tivist cam­paign against him and the high-pro­file pull from the mar­ket (and then re­turn) of Sarep­ta Ther­a­peu­tics’ Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py Ele­v­idys.

The FDA is speed­ing up its pub­lish­ing of ad­verse event da­ta po­ten­tial­ly linked to drugs and vac­cines, mov­ing from quar­ter­ly re­ports to dai­ly up­dates, the agency an­nounced Fri­day.

Known as the FDA Ad­verse Events Re­port­ing Sys­tem, or FAERS, the sys­tem col­lects da­ta in un­ver­i­fied, self-sub­mit­ted re­ports from health­care work­ers, pa­tients or oth­ers — which can some­times lead to in­for­ma­tion gaps or mis­in­ter­pre­ta­tions.

FDA Com­mis­sion­er Mar­ty Makary said that the in­creased pub­lish­ing of the re­ports is in­tend­ed to help iden­ti­fy safe­ty sig­nals faster and in­crease trans­paren­cy — two ma­jor pri­or­i­ties for this FDA.

“Peo­ple who nav­i­gate the gov­ern­ment’s clunky ad­verse event re­port­ing web­sites should not have to wait months for that in­for­ma­tion to be­come pub­lic. We’re clos­ing that wait­ing pe­ri­od and will con­tin­ue to stream­line the process from start to fin­ish,” Makary said in a state­ment with the an­nounce­ment.

One of the main ques­tions fac­ing the Trump ad­min­is­tra­tion as it seeks to low­er pre­scrip­tion drug prices through ex­ec­u­tive or­der is: How?

Tues­day’s cab­i­net meet­ing seemed to pro­vide an an­swer.

For the first time, top mem­bers of the ad­min­is­tra­tion pub­licly linked the Com­merce De­part­ment’s 232 in­ves­ti­ga­tion in­to phar­ma­ceu­ti­cal prod­ucts — and tar­iffs that are ex­pect­ed to fol­low — with Pres­i­dent Don­ald Trump's "most fa­vored na­tion" plan to get drug­mak­ers to re­duce prices in the US.

Com­merce Sec­re­tary Howard Lut­nick told the pres­i­dent that the com­bi­na­tion of the 232 in­ves­ti­ga­tion and the most fa­vored na­tion pro­gram, which is be­ing led in part by HHS Sec­re­tary Robert F. Kennedy Jr., "re­al­ly gives Bob­by the tools to go ex­e­cute your plan."