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

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.