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

 

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

Federal Trade Commission commissioner Rohit Chopra testifies on Capitol Hill (AP Photo/Susan Walsh)

FTC clears Bris­tol-My­ers’ $74B deal to buy Cel­gene — but Dems sig­nal a po­ten­tial hard shift against Big Phar­ma M&A

Bristol-Myers Squibb’s record $74 billion takeover of Celgene is a done deal. And it will all be over — except for the lingering complaints from die-hard Celgene investors — on Wednesday.

Like much else that’s going on in Washington these days, the vote among the 5 FTC commissioners split along party lines, with the 3 Republicans voting to clear the way and the 2 Democrats steamed over what they see as a major M&A move that will lessen competition and innovation. And that split has big implications for the M&A side of the business if the Dems take the White House in 2020.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

What does $62B buy you these days? A lot, says Take­da ex­ecs as the phar­ma play­er promis­es a block­buster R&D fu­ture

First comes the $62 billion buyout. Then comes the asset auction and reorganization to pay down debt. Now comes the detailed pledge of a bigger, brighter future in drug development.

That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.