Drug reg­u­la­tors look to har­mo­nize how they tack­le in­no­va­tion

The In­ter­na­tion­al Coali­tion of Med­i­cines Reg­u­la­to­ry Au­thor­i­ties (ICM­RA) re­cent­ly re­leased a re­port on how var­i­ous reg­u­la­tors world­wide are work­ing to­geth­er to bet­ter iden­ti­fy and ad­dress fu­ture reg­u­la­to­ry chal­lenges posed by new cat­e­gories of ther­a­peu­tics, like cell and gene ther­a­pies, and new tools for drug de­vel­op­ment, such as ar­ti­fi­cial in­tel­li­gence (AI).

The re­port, which is part of a wider ef­fort to re­duce du­plica­tive work and in­crease har­mo­niza­tion among drug reg­u­la­tors from the US, Eu­rope, Japan and else­where, delves in­to the top­ic of hori­zon scan­ning for new in­no­va­tions, which for most reg­u­la­tors is “still in its in­fan­cy.” The re­port al­so ad­dress­es nov­el reg­u­la­to­ry path­ways, such as ex­pe­dit­ed path­ways, and oth­ers re­lat­ed to ear­ly en­gage­ment with stake­hold­ers, such as Health Tech­nol­o­gy As­sess­ment agen­cies.

“The pace of in­no­va­tion in med­ical de­vices has sur­passed med­i­cines due adapt­able ap­proach­es to the reg­u­la­tion of in­no­v­a­tive prod­ucts. While the ex­ist­ing nov­el li­cenc­ing path­ways for med­i­cines have seen pos­i­tive re­sults in pro­vid­ing time­ly pa­tient ac­cess, cur­rent and up­com­ing in­no­va­tions will con­tin­ue to chal­lenge reg­u­la­tors,” the re­port notes.

Eu­ro­pean, Japan­ese and Sin­ga­pore drug reg­u­la­tors al­so fur­ther ex­plored the top­ics of ad­di­tive man­u­fac­tur­ing, genome edit­ing and AI.

“The prod­ucts and tech­nolo­gies ex­plored in the case stud­ies show that many reg­u­la­to­ry sci­ence tools are miss­ing at present (e.g. off-tar­get ef­fects de­tec­tion meth­ods) and must be de­vel­oped in or­der for reg­u­la­tors to be able to as­sess the prod­ucts across their life-cy­cle. In ad­di­tion, new tech­nolo­gies (such as ad­di­tive man­u­fac­tur­ing) re­quire adap­ta­tion of the ex­ist­ing reg­u­la­to­ry frame­works, as they fa­cil­i­tate the pro­duc­tion of more com­plex prod­ucts at the point of care rather than in ded­i­cat­ed man­u­fac­tur­ing sites,” the re­port adds.

Reg­u­la­tors will need to train staffers on the new prod­uct types, but will al­so have to ac­cess out­side ex­perts (e.g., soft­ware en­gi­neers), who will re­quire reg­u­la­to­ry train­ing, as well. The agen­cies pro­posed de­vel­op­ing a ca­pac­i­ty frame­work for ex­per­tise as a com­mon re­source, fea­tur­ing the map­ping of the re­quired skills and ex­per­tise for var­i­ous types of prod­ucts or tech­nolo­gies to as­sist reg­u­la­tors in iden­ti­fy­ing their ca­pac­i­ty needs.

ICM­RA, which is al­so look­ing to es­tab­lish an in­for­mal in­no­va­tion net­work among ICM­RA mem­bers lat­er this year, will seek to bet­ter ar­tic­u­late the com­mon char­ac­ter­is­tics of nov­el reg­u­la­to­ry path­ways and to as­sist the analy­ses and con­sid­er­a­tions of new and ex­ist­ing reg­u­la­to­ry meth­ods.

In ad­di­tion to the strate­gic pri­or­i­ty project re­port on in­no­va­tion, ICM­RA re­cent­ly re­leased a frame­work for the in­volve­ment of health au­thor­i­ties in the man­age­ment of glob­al health crises.

In­no­va­tion Strate­gic Pri­or­i­ty Pro­ject Re­port


First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

Im­age: Shut­ter­stock

Author

Zachary Brennan

managing editor, RAPS

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

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Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

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Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Am­gen puts its foot down in shiny new South San Fran­cis­co hub as it re­or­ga­nizes R&D ops

Amgen has signed up to be AbbVie’s neighbor in South San Francisco as it moves into a nine-story R&D facility in the booming biotech hub.

The arrangement gives Amgen 240,000 square feet of space on the Gateway of Pacific Campus, just a few minutes drive from its current digs at Oyster Point. The new hub will open in 2022 and house the big biotech’s Bay Area employees working on cardiometabolic, inflammation and oncology research.

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Scripps and AbbVie go way back. Research conducted in the lab of Scripps scientist Richard Lerner led to the discovery of Humira. The antibody, approved by the FDA in 2002 and sold by AbbVie, went on to become the world’s bestselling treatment. In 2018, the drugmaker and the non-profit organization signed a pact focused on developing cancer treatments — and now, the scope of that partnership has broadened to encompass a range of diseases, including immunological and neurological conditions.

South Ko­rea jails 3 Sam­sung ex­ecs for de­stroy­ing ev­i­dence in Bi­o­Log­ics probe

Three Samsung executives in Korea are going to jail.

The convictions came in what prosecutors had billed as “biggest crime of evidence destruction in the history of South Korea”: a case of alleged corporate intrigue that was thrown open when investigators found what was hidden beneath the floor of a Samsung BioLogics plant. Eight employees in total were found guilty of evidence tampering and the three executives were each sentenced to up to two years in prison.

Nick Plugis, Avak Kahvejian, Cristina Rondinone, Milind Kamkolkar and Chad Nusbaum. (Cellarity)

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Cellarity started with a simple — but far from easy — idea that Avak Kahvejian and his team were floating around at Flagship Pioneering: to digitally encode a cell.

As he and his senior associate Nick Plugis dug deeper into the concept, they found that most of the models others have developed take a bottom-up approach, where they assemble the molecules inside cells and the connections between them from scratch. What if they opt for a top-down approach, aided by single-cell transcriptomics and machine learning, to gauge the behavior of the entire cellular network?

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The biopharma R&D food chain is paying off with a plan at Sanofi to pitch regulators on a new drug for an orphan disease called cold agglutinin disease.

The pharma giant ushered out a statement Tuesday morning — after it spelled out plans to radically restructure the company, abandoning cardio and diabetes research altogether — saying that their C1s inhibitor sutimlimab had cleared the pivotal study.