Ex­perts urge trans­paren­cy, ad­vi­so­ry com­mit­tee re­view for Covid-19 vac­cines

At a vir­tu­al brief­ing on Tues­day, sev­er­al ex­perts told mem­bers of the House Com­mit­tee on Over­sight and Re­form Sub­com­mit­tee on Eco­nom­ic and Con­sumer Pol­i­cy that a trans­par­ent process and ad­vi­so­ry com­mit­tee re­view will be key to the ef­fec­tive roll­out of a vac­cine to pre­vent coro­n­avirus dis­ease.

The brief­ing comes just two weeks af­ter the FDA is­sued guid­ance on the de­vel­op­ment and li­cen­sure of vac­cines against SARS-CoV-2, the virus that caus­es Covid-19, and at a time when some of the most ad­vanced vac­cine can­di­dates are mov­ing in­to Phase 3 tri­als.

Bruce Gellin

Dur­ing the brief­ing, Bruce Gellin, pres­i­dent, glob­al im­mu­niza­tion at the Sabin Vac­cine In­sti­tute, said that FDA’s re­cent guid­ance helps pro­vide trans­paren­cy about its de­ci­sion-mak­ing process for an even­tu­al Covid-19 vac­cine.

“Guid­ances like these, while di­rect­ed to in­dus­try, are re­al­ly for all to see, so all know the con­sid­er­a­tions that go in­to the process,” Gellin said, warn­ing that cut­ting cor­ners in the race to ap­prove a vac­cine would be a “mis­step” with “se­ri­ous im­pli­ca­tions,” such as fu­el­ing vac­cine skep­ti­cism and erod­ing the pub­lic’s trust in FDA’s ap­proval process.

“I strong­ly rec­om­mend that any vac­cine that’s be­ing con­sid­ered for any type of ap­proval by the FDA be re­viewed in an open pub­lic meet­ing by FDA’s Vac­cine and Re­lat­ed Bi­o­log­i­cal Prod­ucts Ad­vi­so­ry Com­mit­tee [VRB­PAC],” Gellin said, not­ing his past par­tic­i­pa­tion on the com­mit­tee.

“With­out such a pub­lic re­view, I have no doubt that sto­ries will ap­pear about vac­cine ap­provals made be­hind closed doors be­cause the gov­ern­ment didn’t want the pub­lic to see what or who may have in­flu­enced those de­ci­sions,” he said.

Ruth Kar­ron

Two oth­er ex­perts, Ruth Kar­ron, di­rec­tor of the Cen­ter for Im­mu­niza­tion Re­search at Johns Hop­kins Bloomberg School of Pub­lic Health, and Ja­son Schwartz, as­sis­tant pro­fes­sor of health pol­i­cy at the Yale School of Pub­lic Health, con­curred.

Kar­ron, who pre­vi­ous­ly chaired the com­mit­tee, said that VRB­PAC’s “ad­vice will be crit­i­cal as the FDA re­views vac­cine tri­al da­ta and makes de­ci­sions about emer­gency use au­tho­riza­tions (EUAs) and li­cen­sure.”

Schwartz added that both VRB­PAC and the Cen­ters for Dis­ease Con­trol and Pre­ven­tion’s Ad­vi­so­ry Com­mit­tee on Im­mu­niza­tion Prac­tices (ACIP) have helped to sup­port the gov­ern­ment’s reg­u­la­to­ry and pol­i­cy­mak­ing work for vac­cines for decades.

“The clos­er that our gov­ern­ment health agen­cies ad­here to their well-es­tab­lished, time test­ed process­es for eval­u­at­ing the safe­ty and ef­fec­tive­ness of Covid-19 vac­cines and de­vel­op­ing ev­i­dence-based ap­proach­es for their de­ploy­ment, the more con­fi­dence the pub­lic can have in the in­tegri­ty and qual­i­ty of those de­ci­sions and those vac­cines,” Schwartz said.

Ap­provals and EUAs

Jesse Good­man

Jesse Good­man, di­rec­tor of the Cen­ter on Med­ical Prod­uct Ac­cess, Safe­ty and Stew­ard­ship at George­town Uni­ver­si­ty and for­mer chief sci­en­tist at FDA, said he thinks the 50% ef­fec­tive­ness thresh­old set in FDA’s guid­ance is “a rea­son­able start­ing point.”

Good­man al­so said that with ad­e­quate cau­tion, an EUA or ex­pand­ed ac­cess pro­to­col could pro­vide ear­ly ac­cess to a vac­cine.

“We need to be very thought­ful about any vac­cine ac­cess pri­or to ap­proval. There may be cir­cum­stances where use of an un­ap­proved vac­cine could be ap­pro­pri­ate in a se­vere out­break. For ex­am­ple, if a vac­cine with doc­u­ment­ed safe­ty and promis­ing ef­fec­tive­ness were avail­able, but all the da­ta need­ed [was] not yet sub­mit­ted, FDA could pro­vide ac­cess through an ex­pand­ed ac­cess pro­gram or through an emer­gency use au­tho­riza­tion,” Good­man said.

Ja­son Schwartz

Both Good­man and Schwartz raised con­cerns that the pub­lic might not un­der­stand the dif­fer­ence be­tween an EUA and full ap­proval.

“I would be con­cerned … that an emer­gency use au­tho­riza­tion would not be un­der­stood among the pub­lic to be dif­fer­ent from the full ap­proval with all the de­tails from the FDA,” Schwartz said, be­fore adding that is­su­ing an EUA pri­or to hav­ing Phase 3 da­ta in hand would be a “very se­ri­ous gam­ble.”

Good­man sug­gest­ed that FDA re­quire in­formed con­sent as part of the process for ad­min­is­ter­ing a vac­cine un­der an EUA and said that such use should be “tar­get­ed to those at high­est risk of in­fec­tion and com­pli­ca­tions.”

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.


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.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,800+ biopharma pros reading Endpoints daily — and it's free.

Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,800+ biopharma pros reading Endpoints daily — and it's free.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,800+ biopharma pros reading Endpoints daily — and it's free.

Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,800+ biopharma pros reading Endpoints daily — and it's free.