FDA to roll out mod­ern­iza­tion plan in push for da­ta in­ter­op­er­abil­i­ty

Amy Aber­nethy FDA

An ac­tion plan will be rolled out in the com­ing months to de­fine the FDA’s ap­proach to mod­ern­iz­ing its tech­nol­o­gy in­fra­struc­ture and frame­work, FDA Prin­ci­pal Deputy Com­mis­sion­er Amy Aber­nethy said dur­ing a Thurs­day keynote at the Of­fice of the Na­tion­al Co­or­di­na­tor for Health In­for­ma­tion Tech­nol­o­gy’s (ONC) 3rd in­ter­op­er­abil­i­ty fo­rum.

FDA mod­ern­iza­tion ef­forts have been un­der­way in re­cent years, with the re­or­ga­ni­za­tions of the Cen­ter for De­vices and Ra­di­o­log­i­cal Health and the Cen­ter for Drug Eval­u­a­tion and Re­search. The plan that the FDA in­tends to roll out soon is a cross-cen­ter ap­proach.

Aber­nethy said that the FDA’s plan con­tains an agency-wide ob­jec­tive—ad­vanc­ing in­ter­op­er­abil­i­ty to in­form reg­u­la­to­ry de­ci­sion-mak­ing in re­al-time. This, in turn, is in­tend­ed to ex­pe­dite de­ter­mi­na­tions on sub­mis­sions and im­prove reg­u­la­to­ry over­sight to in­crease pa­tient ac­cess to med­ical prod­ucts deemed to be safe and ef­fec­tive at a faster pace, as well as to en­able ear­li­er prod­uct in­ter­ven­tions in the post-mar­ket set­ting.

Crit­i­cal­ly eval­u­at­ing in­ter­op­er­a­ble tech­nolo­gies, match­ing the best so­lu­tions to the char­ac­ter­is­tics of a pa­tient at the point of care and en­sur­ing that da­ta are “avail­able, re­li­able and well-char­ac­ter­ized” are the next steps in the jour­ney to­ward in­ter­op­er­abil­i­ty, said Aber­nethy. As such, the fo­cus of the plan is cen­tered on mod­ern­iz­ing the FDA’s tech in­fra­struc­ture and frame­work and pro­mot­ing in­ter­op­er­abil­i­ty use cas­es.

Aber­nethy not­ed the agency’s vi­sion for in­ter­op­er­abil­i­ty with­in the con­text of re­al-world ev­i­dence. This seeks to make use of ap­pli­ca­tion pro­gram­ming in­ter­faces to cre­ate ef­fi­cien­cies around reg­u­la­to­ry re­view process­es and track med­ical prod­ucts through­out their life­cy­cle by lever­ag­ing da­ta sources like elec­tron­ic health records (EHRs) to cre­ate a to­tal­i­ty of ev­i­dence that bet­ter in­forms ben­e­fit-risk pro­files.

The agency is “tak­ing a thought­ful and proac­tive ap­proach to help ad­vance” the in­ter­op­er­abil­i­ty sto­ry part­ly via “ap­pro­pri­ate and fit to scale use of tech­nol­o­gy with­in a mod­ern­ized re­view in­fra­struc­ture,” as well as “build­ing and en­sur­ing that tech­nol­o­gy is in place at a mod­ern FDA,” said Aber­nethy. “We are build­ing a dy­nam­ic reg­u­la­to­ry en­vi­ron­ment across the en­tire cy­cle of prod­uct de­vel­op­ment to help us meet the pace of med­ical in­no­va­tion.”

As part of the work to up­date how the agency in­ter­faces with the stake­hold­er com­mu­ni­ty, the plan will look to bet­ter en­gage with the tech com­mu­ni­ty. “We need to de­fine the in­ter­faces so that there is a core un­der­stand­ing” of how in­for­ma­tion can be passed in­to FDA “as ef­fi­cient­ly as pos­si­ble,” she added.

Aber­nethy al­so point­ed to “a world where FDA and ONC are work­ing to­geth­er as sis­ter agen­cies to pro­vide an ac­tu­al roadmap” of how da­ta sets can be gen­er­at­ed as well as pres­sure test­ing “what hap­pens when a da­ta el­e­ment tra­vers­es from the EHR all the way through the process in­to clin­i­cal re­search.” She not­ed the po­ten­tial of an en­vi­sioned project she calls “the life of a da­ta el­e­ment,” to chart a path that helps the FDA iden­ti­fy ar­eas where it should pro­vide guid­ance to spon­sors and EHR ven­dors.


RAPS: 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.

So­cial im­age: Amy Aber­nethy, World Med­ical In­no­va­tion Fo­rum via Youtube

 

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

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Kathy High (file photo)

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Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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Tim Mayleben (file photo)

Es­pe­ri­on's goldilocks cho­les­terol fight­er wins FDA ap­proval — will its 'tra­di­tion­al' pric­ing ap­proach spur adop­tion?

It’s more effective than decades-old statins but not as good as the injectable PCSK9 — the goldilocks treatment for cholesterol-lowering, bempedoic acid, has secured FDA approval.

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The sky-high expectations for the pair of PCSK9 drugs that were first approved in 2015 quickly simmered — and despite a 60% price cut, coupled with data showing the therapies also significantly cut cardiovascular risk, sales have not really perked up.

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“We’re kind of like the old comfortable shoe,” Esperion’s chief commercial officer Mark Glickman remarked in an interview with Endpoints News ahead of the decision date. “It’s an oral product, once-daily and nontitratable — these are things that just resonate so true with patients and physicians and I think we’ve kind of forgotten about that.”

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James Collins, Broad Institute via Youtube

UP­DAT­ED: A space odyssey for new an­tibi­otics: MIT's ma­chine learn­ing ap­proach

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Gilead los­es two more patent chal­lenges on HIV pill, set­ting up court­room fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

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Christos Kyratsous (via LinkedIn)

He built a MERS treat­ment in 6 months and then the best Ebo­la drug. Now Chris­tos Kyrat­sous turns his sights on Covid-19

TARRYTOWN, NY — In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascal’s roommates sat her down on their couch and staged an intervention.

“Are you sure this is what you want to be doing with your life?” she recalls them asking her.

Special report

Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didn’t see her roommate for a week. For months, that was life in Christos Kyratsous’ lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

“I’m ok, I don’t have Ebola,” Pascal told them. “I see that death toll rising and I can’t not do something about it.”

Last August, Regeneron learned they had succeeded: In a large trial across West Africa, their drug, REGN-EB3, was vastly more effective than the standard treatments. It was surprise news for the company, coming just 10 months into a trial they thought would take several years and a major victory in the global fight against a deadly virus that killed over 2,000 in 2019 and can carry a mortality rate of up to 90%.

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“Christos likes things immediately,” Matt Frieman, Regeneron’s coronavirus collaborator at the University of Maryland, told Endpoints. “That’s what makes us good collaborators: We push each other to develop things faster and faster.”

Kristen Pascal (Regeneron)

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Jim Scholefield via PR Newswire

Mer­ck los­es its chief dig­i­tal of­fi­cer, spot­light­ing tal­ent hunt for the hottest ti­tle in Big Phar­ma

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Don't let Ab­b­Vie fool FTC with an easy di­vesti­ture, plead crit­ics in lat­est at­tack on $63B Al­ler­gan buy­out

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