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

 

#ES­MO20: Trodelvy da­ta show that Gilead­'s $21B buy­out may have been worth the big pre­mi­um

Gilead CEO Dan O’Day has been on a shopping spree. And while some analysts gawked at the biotech’s recent $21 billion Immunomedics buyout, new data released at virtual ESMO 2020 suggest the acquisition may have been worth the hefty price.

The deal, announced last weekend, gives California-based Gilead $GILD Trodelvy, which was recently approved for metastatic triple-negative breast cancer (mTNBC).

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,300+ biopharma pros reading Endpoints daily — and it's free.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,300+ biopharma pros reading Endpoints daily — and it's free.

#ES­MO20: As­traZeneca bur­nish­es Tagris­so's ad­ju­vant NSCLC pro­file with 'un­prece­dent­ed' re­duc­tion in brain mets. Can they win over skep­tics?

When AstraZeneca trumpeted “momentous” and “transformative” results for Tagrisso earlier this year at ASCO, some practitioners threw cold water on the ADAURA fervor. Sure, the disease-free survival data look good, but overall survival is the endpoint that matters when it comes to choosing adjuvant therapy for non-small cell lung cancer patients, the experts said.

The OS data still aren’t here, but AstraZeneca is back at ESMO to bolster their case with a look at brain metastasis data.

Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,300+ biopharma pros reading Endpoints daily — and it's free.

Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.

#ES­MO20: Alk­er­mes of­fers their first snap­shot of a ben­e­fit for their next-gen IL-2 drug. But why did 1 pa­tient starve to death?

Everyone in the cancer R&D arena is looking to build new franchises around better drugs and combos. And one busy pocket of that space is centered entirely on creating an IL-2 drug that can be as effective as the original without the toxicity that damned it to the sidelines.

Alkermes $ALKS formally tossed its hat into the ring of contenders at virtual ESMO today, highlighting the first glimpse of efficacy for their candidate, ALKS 4230, as both a monotherapy as well as in combination with Merck’s Keytruda.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,300+ biopharma pros reading Endpoints daily — and it's free.

Albert Bourla (Photo by Steven Ferdman/Getty Images)

Pfiz­er match­es Mod­er­na with their full Covid-19 tri­al blue­print — As­traZeneca says it will un­veil its pro­to­col 'short­ly'

Yesterday, after sustained public pressure as Moderna released its Phase III Covid-19 trial blueprint, Pfizer released its own full trial design for their vaccine trials. The move was designed to boost transparency and shore up public trust in the vaccines, but it also revealed differences in how the two companies are approaching the much-watched studies while failing to satisfy the demands of the fiercest advocates for transparency.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,300+ biopharma pros reading Endpoints daily — and it's free.

Stronger to­geth­er? Boehringer and Mi­rati team to put first KRAS-KRAS com­bo in the clin­ic

Researchers are still waiting to see how much any of the vaunted KRAS drugs now in the clinic can, after decades of preclinical research and some early human studies, help patients. But while they do, two of the leading developers will look to see whether a KRAS-KRAS combo might pose a better shot than any KRAS alone.

Boehringer Ingelheim and Mirati have signed a collaboration to combine Mirati’s closely-watched lead KRAS inhibitor, MRTX849, in a clinical trial with the pan-KRAS blocker that Boehringer has quietly developed with high expectations behind their flashier contenders.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,300+ biopharma pros reading Endpoints daily — and it's free.

#ES­MO20: Re­gen­eron, Sanofi eye an­oth­er first for their PD-1 con­tender Lib­tayo with promis­ing da­ta for on­col­o­gy niche

Regeneron and Sanofi took another step forward in the long march towards a greatly expanded market for their late-bloomer PD-1 checkpoint Libtayo.

The two occasional allies posted an objective response rate of 31% for Libtayo among 84 patients suffering from advanced cases of basal cell carcinoma at virtual ESMO. That spotlights progress for 26 patients, 5 of whom had a complete response. The data also reflect a boost in the number of responses seen from the last cut of the numbers.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,300+ biopharma pros reading Endpoints daily — and it's free.