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.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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Ab­b­Vie sur­veys emo­tion­al im­pact of chron­ic leukemia con­di­tion, finds 'roller coast­er' of emo­tions

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.

FDA's vac­cine ad­comm to re­view first fe­cal trans­plant to treat C. dif­fi­cile in­fec­tions

Back in 2018, Swiss drugmaker Ferring Pharmaceuticals made a big bet on Minnesota-based Rebiotix, buying up the company for its experimental poop-based drug implant to treat an infection caused by C. difficile, a potentially dangerous bacteria, in a new way.

Four years later, Ferring’s fecal microbiota transplant, dubbed RBX2660 or Rebyota, will face the FDA’s adcomm of outside vaccine experts on Sept. 22, debating whether the agency should license the transplant as a treatment for adults following antibiotic treatment for recurrent C. difficile infection.