Spon­sors should flag de­cen­tral­ized as­pects of can­cer tri­als to prep for the post-Covid era, FDA says

As more and more clin­i­cal tri­als dur­ing the pan­dem­ic have har­nessed the pow­er of re­mote as­sess­ments and mod­i­fi­ca­tions, the FDA On­col­o­gy Cen­ter of Ex­cel­lence and the Of­fice of On­co­log­ic Dis­eases are now re­quest­ing that ap­pli­cants vol­un­tar­i­ly add flags to datasets to dis­crim­i­nate be­tween re­mote as­sess­ments and tri­al site as­sess­ments.

The push from the FDA to flag these dif­fer­ences is part of an ef­fort to ame­lio­rate the fact that there is cur­rent­ly no stan­dard way to iden­ti­fy Covid-re­lat­ed mod­i­fi­ca­tions in clin­i­cal tri­al datasets sub­mit­ted to the FDA. And many of these re­mote mod­i­fi­ca­tions were de­ployed in the mid­dle of large on­go­ing can­cer tri­als as a way to min­i­mize Covid risks, the agency said.

An Or­a­cle sur­vey from last No­vem­ber found that 76% of bio­phar­ma com­pa­nies and CROs sur­veyed said the pan­dem­ic sped their adop­tion of de­cen­tral­ized tri­als. But in­dus­try re­spon­dents were al­so di­vid­ed on whether there has been enough reg­u­la­to­ry guid­ance around this push to­ward de­cen­tral­ized tri­als.

“FDA hopes to learn more about the op­por­tu­ni­ties and chal­lenges of these RE­MOTE mod­i­fi­ca­tions to fos­ter use of ‘de­cen­tral­ized’ as­pects of clin­i­cal tri­als prospec­tive­ly in the post-COVID era,” the agency said.

Still, the use of full or hy­brid de­cen­tral­ized de­signs by tri­al spon­sors has been rare in on­col­o­gy, the agency notes, part­ly be­cause of the un­cer­tain­ty sur­round­ing the ef­fect of re­mote as­sess­ments on da­ta qual­i­ty and out­comes.

But the FDA in­sists that bet­ter un­der­stand­ing these mod­i­fi­ca­tions can re­duce un­cer­tain­ty for spon­sors and reg­u­la­tors, and iden­ti­fy mit­i­ga­tion strate­gies for fu­ture, prospec­tive de­cen­tral­ized de­signs.

More specif­i­cal­ly, the agency is call­ing for com­pa­nies to flag any in­stances of re­mote­ly con­duct­ed clin­ic vis­its, lab or imag­ing as­sess­ments, or de­liv­ery of in­ves­ti­ga­tion­al prod­ucts.

“While tri­al datasets may al­ready be iden­ti­fy­ing RE­MOTE mod­i­fi­ca­tions as pro­to­col de­vi­a­tions, we would like all RE­MOTE as­sess­ments to be flagged, rather than on­ly those as­sess­ments that re­sult in pro­to­col de­vi­a­tions, giv­en that once a tri­al pro­to­col is amend­ed to al­low re­mote as­sess­ments they will no longer be con­sid­ered Pro­to­col De­vi­a­tions,” the agency added.

In terms of po­ten­tial re­search ques­tions to ex­plore fur­ther, the agency points to study­ing the ef­fects of re­mote lab and imag­ing as­sess­ments, as well as re­mote in­ves­ti­ga­tion­al prod­uct ad­min­is­tra­tion to com­pare dose mod­i­fi­ca­tion rates, ad­verse event rates, im­age scan qual­i­ty, and the ef­fect of re­plac­ing in-per­son fol­low-up meet­ings with telemed­i­cine.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.

Thomas Schall, ChemoCentryx CEO (file photo)

Chemo­Cen­tryx plunges as FDA rais­es ques­tions about rare dis­ease drug ahead of ad­comm

ChemoCentryx’s stock price on Wednesday was cut in half by the release of FDA briefing documents ahead of a Thursday adcomm, raising questions on the company’s clinical data to support avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

ANCA-associated vasculitides (AAV) affect small to medium-size blood vessels that can be fatal in less than a year if left untreated, according to FDA. Only Roche’s Rituxan is currently FDA-approved for the treatment of AAV, while glucocorticoids are approved for the broader indication of vasculitis.

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Stéphane Bancel at the Endpoints #JPM20 breakfast panel in San Francisco, January 2020 (Photo: Jeff Rumans, Endpoints News)

Mod­er­na says Covid-19 vac­cine boost­er in­creased im­mune re­sponse against vari­ants of con­cern

About a month after announcing their variant-specific Covid-19 vaccine boosters showed promising results in mice, Moderna says it now has some human data to back it up.

Volunteers given a booster shot about six to eight months after receiving their second dose saw increased antibody levels against SARS-CoV-2 and two variants of concern: B.1.351, which was first identified in South Africa, and P.1, first identified in Brazil, the company said on Wednesday.

Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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