EMA joins reg­u­la­to­ry coali­tion in call­ing for RWE to be in­te­grat­ed in­to reg­u­la­to­ry de­ci­sions

As more and more biotechs are look­ing in­to the po­ten­tial ap­pli­ca­tions of us­ing re­al-world ev­i­dence in reg­u­la­to­ry sub­mis­sions, the Eu­ro­pean Med­i­cines Agency is call­ing for more wide­spread adop­tion.

EMA en­dorsed a state­ment pub­lished Fri­day by ICM­RA, the In­ter­na­tion­al Coali­tion of Med­i­cines Reg­u­la­to­ry Au­thor­i­ties, high­light­ing the use of both RWE and re­al-world da­ta in de­vel­op­ment and reg­u­la­to­ry au­tho­riza­tions, not­ing their role

in sup­port­ing the de­vel­op­ment of med­i­cines across their dif­fer­ent stages of de­vel­op­ment and use is evolv­ing rapid­ly. How­ev­er, chal­lenges ex­ist, due for ex­am­ple to het­ero­ge­neous da­ta sources, dif­fer­ent lev­els of da­ta qual­i­ty, and var­i­ous gov­er­nance mod­els for da­ta shar­ing and ac­cess. Close col­lab­o­ra­tion be­tween reg­u­la­tors across the world can help ad­dress these chal­lenges. ICM­RA can play an im­por­tant role by catalysing in­creased co­op­er­a­tion on the use of RWE for reg­u­la­to­ry de­ci­sion-mak­ing.

Shirley Wang

RWE has been a par­tic­u­lar­ly nit­picky are­na — an FDA pi­lot pro­gram said two months ago that on­ly about half of a se­lect group of clin­i­cal tri­als could be well-em­u­lat­ed with avail­able re­al-world ev­i­dence. RWE was al­so a fo­cal point in the 21st Cen­tu­ry Cures Act, which pushed the FDA in new di­rec­tions on the ev­i­dence.

Ac­cord­ing to Har­vard Med­ical School as­so­ciate pro­fes­sor Shirley Wang at the time, chal­lenges were seen in re­searchers’ at­tempts to repli­cate stud­ies in os­teo­poro­sis, chron­ic kid­ney dis­ease, heart fail­ure, asth­ma and COPD.

It was not­ed, ac­cord­ing to the pi­lot pro­gram, that oth­er tri­als could be more close­ly em­u­lat­ed, such as atri­al fib­ril­la­tion, VTE and hy­per­ten­sion.

In their state­ment, the 24 mem­ber agen­cies of ICM­RA, in­clud­ing the FDA, NM­PA and EMA pledged “to fos­ter glob­al ef­forts and fur­ther en­able the in­te­gra­tion of re­al-world ev­i­dence in­to reg­u­la­to­ry de­ci­sion-mak­ing,” EMA said. As part of that, the agen­cies not­ed four fo­cus ar­eas.

First, is the “har­mon­i­sa­tion of ter­mi­nolo­gies for re­al-world da­ta and re­al-world ev­i­dence.” Ac­cord­ing to ICM­RA, one of the fo­cus­es is on gen­er­at­ing “com­mon op­er­a­tional de­f­i­n­i­tions of RWD and RWE, with clear scope and lev­el of gran­u­lar­i­ty.” Next, the coali­tion point­ed to a reg­u­la­to­ry con­ver­gence of “re­al-world da­ta and re­al-world ev­i­dence guid­ance and best prac­tice.” The guid­ance and best prac­tices would in­clude tem­plates, suit­able sce­nar­ios where re­al-world ev­i­dence can con­tribute to reg­u­la­to­ry de­ci­sion-mak­ing, and meta­da­ta.

The third fo­cus area is on readi­ness — specif­i­cal­ly fo­cused on ad­dress­ing pub­lic health chal­lenges and what ICM­RA called “emerg­ing health threats.”

That would work by al­low­ing for the quick cre­ation of ex­pert groups on spe­cif­ic top­ics and col­lab­o­rat­ing to make stud­ies more ef­fi­cient. The fi­nal fo­cus area is on trans­paren­cy — defin­ing prin­ci­ples and prac­tices to reg­is­ter pre-spec­i­fied study pro­to­cols and study re­sults in pub­licly avail­able reg­istries. And on top of that, pro­mot­ing the pub­li­ca­tion of re­sults in peer-re­viewed and open-source jour­nals.

“These po­ten­tial ar­eas for reg­u­la­to­ry col­lab­o­ra­tion on RWD and RWE could be tak­en for­ward through a va­ri­ety of ex­ist­ing fo­ra in­clud­ing ICH, in­ter­na­tion­al stan­dard­i­s­a­tion bod­ies, and clus­ters of in­ter­est­ed reg­u­la­tors. ICM­RA re­mains com­mit­ted to steer­ing this work in the in­ter­ests of pa­tient health and in­no­va­tion,” ICM­RA added in a state­ment.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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Stanley Erck, Novavax CEO (Andrew Harnik/AP Images)

No­vavax pulls out of Covid-19 vac­cine al­liance with Gavi

Novavax is pulling out of its Covid-19 vaccine deal with Gavi, the Vaccine Alliance, a global partnership tasked with ensuring vaccine access in lower-income countries, following an alleged contract violation.

The Maryland-based company claimed on Friday that Gavi failed to purchase at least 350 million doses of its protein-based vaccine Nuvaxovid by the end of the year, per an advanced purchase agreement. Gavi, the World Health Organization and the Coalition for Epidemic Preparedness Innovations (CEPI) are co-leaders of COVAX, an effort to ensure that all participating countries, regardless of income levels, have access to vaccines.

Fu­ji­film to build $188M man­u­fac­tur­ing plant in North Car­oli­na’s re­search tri­an­gle

As the Japanese conglomerate Fujifilm continues to invest heavily in its CDMO arm, one of its manufacturing divisions is teeing up a major investment.

Fujifilm Irvine Scientific announced on Tuesday that parent Fujifilm is making a $188 million investment to build a cell culture media manufacturing site in the Research Triangle Park in North Carolina. The new site will mark Fujifilm Irvine’s fifth manufacturing site globally and its second in the US.