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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

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.

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

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.

FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

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A new study finds that many patient influencers are sharing prescription drug experiences along with health information.

So­cial me­dia pa­tient in­flu­encers ‘danc­ing in the gray’ of phar­ma mar­ket­ing, more clar­i­ty need­ed, re­searcher says

It’s no surprise that patient influencers are talking about their health conditions on social media. However, what’s less clear is what role pharma companies are playing, how big the patient influencer industry is, and just how is information about prescription drugs from influencers relayed — and received — on social media.

While University of Colorado associate professor Erin Willis can’t answer all those questions, she’s been researching the issue for several years and recently published new research digging into the communication styles, strategies and thinking of patient influencers, many of whom partner with pharma companies.

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Drug short­age so­lu­tions brought be­fore Sen­ate Home­land Se­cu­ri­ty com­mit­tee

With more than 300 active drug shortages, the Senate Committee on Homeland Security and Governmental Affairs had its hands full on Wednesday with multiple experts testifying on drug shortages and possible solutions.

A picture of the shortage situation. presented by Erin Fox, an adjunct professor at the College of Pharmacy at the University of Utah, explained how some patients have died due to drug shortages, including with medication errors when substitutes were dosed incorrectly or when an emergency product was not available.

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