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.

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.