The FDA is prep­ping to rene­go­ti­ate its multi­bil­lion-dol­lar, quin­quen­ni­al user fee agree­ments with the drug in­dus­try lat­er this sum­mer, and a new deputy com­mis­sion­er on Fri­day sug­gest­ed the struc­ture of the deal may need to be re­worked.

“As we look to­wards the next reau­tho­riza­tion, it is time to take a step back and think about if the fee struc­tures and amounts have any un­in­tend­ed con­se­quences,” Grace Gra­ham, the FDA's deputy com­mis­sion­er for pol­i­cy, leg­is­la­tion and in­ter­na­tion­al af­fairs, told the Food and Drug Law In­sti­tute's an­nu­al con­fer­ence.

Gra­ham not­ed that user fees are cru­cial for the FDA and make up a sig­nif­i­cant share of agency fund­ing. About 70% of the FDA's drug cen­ter bud­get is made up of in­dus­try funds.

The gov­ern­ment “did not act con­trary to the law” by re­quir­ing drug­mak­ers to seek ap­proval be­fore im­ple­ment­ing pro­posed new 340B re­bate mod­els, a fed­er­al judge ruled.

The de­ci­sion marks a key win for HHS and its sub­agency the Health Re­sources and Ser­vices Ad­min­is­tra­tion, which have ar­gued that drug­mak­ers’ pro­posed changes for dis­trib­ut­ing 340B drug dis­counts “would have up­end­ed” the way the fed­er­al pro­gram has op­er­at­ed for more than 30 years.

The plain­tiffs, Eli Lil­ly, No­var­tis, Bris­tol My­ers Squibb and Sanofi, have ar­gued that the 340B pro­gram is abused and in­tro­duced plans that would re­quire hos­pi­tals to pur­chase 340B-el­i­gi­ble drugs at mar­ket price and re­coup the dis­counts via re­bates, as op­posed to up­front. They claimed that HRSA erred by bar­ring im­ple­men­ta­tion of those plans with­out pri­or agency ap­proval.

Sarep­ta Ther­a­peu­tics is meet­ing with the FDA in June to dis­cuss ex­pand­ing the use of its Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py to pa­tients younger than 4 years old.

The out­come could serve as a lit­mus test of how the US reg­u­la­to­ry agency will run un­der its new top cell and gene ther­a­py of­fi­cial, Vinay Prasad, who has re­peat­ed­ly crit­i­cized the ap­proval of Ele­v­idys and is wide­ly ex­pect­ed to be a more con­ser­v­a­tive reg­u­la­tor.

Prasad's pre­de­ces­sor, Pe­ter Marks, sin­gle-hand­ed­ly ap­proved Sarep­ta’s gene ther­a­py for most pa­tients in 2024, and sup­port­ed ac­cel­er­at­ing rare dis­ease pro­grams. Marks was forced out of the FDA in March over dis­agree­ments with HHS Sec­re­tary Robert F. Kennedy Jr.'s vac­cines stance.

NEW OR­LEANS — The an­nu­al Amer­i­can So­ci­ety of Gene & Cell Ther­a­py meet­ing felt qui­eter this year, amid fed­er­al fund­ing cuts and a tough time for the sec­tor as a whole.

While con­fer­ence or­ga­niz­ers said they did not have a fi­nal count as of Thurs­day af­ter­noon, they es­ti­mat­ed guest num­bers were down by about 10% com­pared to last year, when there were around 7,800 reg­is­tered at­ten­dees.

Some promi­nent gene edit­ing re­searchers — David Liu and Patrick Hsu among them — skipped the meet­ing. And ma­jor com­pa­nies in the field, in­clud­ing CRISPR Ther­a­peu­tics, were al­so ab­sent from the sci­en­tif­ic ses­sions.

FDA of­fi­cials, typ­i­cal­ly a ma­jor pres­ence at this meet­ing, were al­so ab­sent. And while some sci­en­tists from the NIH at­tend­ed, con­fer­ence or­ga­niz­ers said that the show­ing from fed­er­al em­ploy­ees was “marked­ly low­er than re­cent years.”