FDA rais­es ques­tions with Eu­ro­pean da­ta law's re­stric­tions on re­view­ing clin­i­cal da­ta

In 2018, the EU signed off on a new law re­quir­ing or­ga­ni­za­tions to pro­tect how per­son­al da­ta is col­lect­ed or used from those re­sid­ing in the EU or its mem­ber states.

Known as the Gen­er­al Da­ta Pro­tec­tion Reg­u­la­tion (GDPR), the law has changed how com­pa­nies and on­line ad­ver­tis­ing work, but it has al­so been not-so-qui­et­ly af­fect­ing the FDA.

“So far, the FDA’s biore­search mon­i­tor­ing pro­gram (BI­MO), which over­sees the con­duct and re­port­ing of FDA-reg­u­lat­ed re­search, has been most im­pact­ed by the law,” Heather Mes­sick, a for­mer in­ter­na­tion­al pol­i­cy an­a­lyst in FDA’s Eu­rope Of­fice, who’s now a reg­u­la­to­ry coun­sel in the FDA’s Of­fice of Com­pound­ing Qual­i­ty and Com­pli­ance, wrote in a new blog post.

FDA in­ves­ti­ga­tors, for in­stance, have been un­able to com­plete ei­ther in-per­son BI­MO in­spec­tions or con­duct vir­tu­al da­ta re­views in some cas­es due to tech chal­lenges, re­source con­straints, or GDPR-like da­ta shar­ing poli­cies, Mes­sick writes, adding that “lack of clar­i­ty around GDPR has im­ped­ed our abil­i­ty to re­view da­ta re­mote­ly dur­ing the pan­dem­ic.”

And the agency’s con­cern with GDPR stretch­es be­yond BI­MO.

Bio­phar­ma com­pa­nies are re­quired to sub­mit par­tic­i­pant lev­el da­ta from clin­i­cal tri­als to sup­port their med­ical prod­uct ap­pli­ca­tions, and much of that da­ta comes from mul­ti-na­tion­al sites, in­clud­ing EU cit­i­zens. With­hold­ing the agency’s abil­i­ty to trans­fer such da­ta from the EU “could neg­a­tive­ly im­pact the ro­bust­ness of da­ta sub­mit­ted to the FDA and im­pact in­ves­ti­ga­tion­al prod­uct re­views and ap­provals,” Mes­sick notes.

Cer­tain de­mo­graph­ic da­ta al­so may be pro­tect­ed un­der the GDPR, she adds, and this in­for­ma­tion may im­pact the FDA’s abil­i­ty to com­plete re­views of new drugs or bi­o­log­ics.

HHS raised oth­er con­cerns when the GDPR took force in 2018, not­ing that if a re­searcher re­ceives no­tice that a da­ta sub­ject has with­drawn con­sent to da­ta pro­cess­ing, the EU guide­lines con­clude that the da­ta con­troller “should delete or anonymise the per­son­al da­ta straight away.”

But HHS says that such dele­tion, “how­ev­er, could se­ri­ous­ly im­per­il the in­tegri­ty of the re­search, there­by un­der­min­ing the in­vest­ment made by HHS in mul­ti-site, trans-na­tion­al stud­ies with sites lo­cat­ed in the EEA.  It could al­so im­per­il the abil­i­ty of U.S.-based re­search in­sti­tu­tions, in­dus­try spon­sors and re­searchers to re­spond to re­quests from FDA and/or from cog­nizant IRBs, as they would be hin­dered from us­ing for their re­spons­es the per­son­al da­ta of the in­di­vid­ual who has with­drawn con­sent.”

The NIH has been work­ing di­rect­ly with coun­ter­parts in the EU to as­sist with any GDPR im­ped­i­ments to re­search col­lab­o­ra­tions, Mes­sick notes, par­tic­u­lar­ly as the US and EU ear­li­er this year agreed “in prin­ci­ple” on a new da­ta agree­ment for trans­fers of per­son­al pri­va­cy da­ta for com­mer­cial pur­pos­es, but the sit­u­a­tion is still not re­solved.

“GDPR is an on­go­ing area of con­cern, and the sit­u­a­tion will no doubt con­tin­ue to change as the US and EU con­tin­ue ne­go­ti­a­tions on a new da­ta agree­ment, and as the over­all le­gal and pol­i­cy land­scape in the EU con­tin­ues to evolve. The Eu­rope Of­fice will be close­ly track­ing de­vel­op­ments in the months and years ahead,” the blog post said.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

No­var­tis un­veils 'US-first' strat­e­gy ahead of San­doz spin­off

Weeks after announcing the spinoff of generics arm Sandoz, Vas Narasimhan paints a picture of the new, slimmer Novartis — with a “US-first mindset,” he said at an investor event on Thursday.

The CEO unveiled ambitious plans to become a top-five player in the US by 2027 at Novartis’ “Meet the Management” event in Basel, Switzerland, which means ramping up clinical trials in the states and “building capability and talent, among other things.” The company’s also shooting for a top-three ranking in China.

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Pfiz­er sacks phar­ma com­pe­ti­tion in ear­ly NFL TV ad­ver­tis­ing sea­son

If pharma advertising had a fantasy football league, Pfizer would be crushing the competition. A dive into the National Football League’s TV commercial buys across early season games by iSpot shows a hefty lead with its Covid-19 Comirnaty vaccine ads.

More than 175 million impressions with $9.5 million in media spending put Pfizer in the top spot with a 65% share of voice across NFL pharma spending, according to the real-time TV ad tracker. In a distant second place is Bristol Myers Squibb’s Opdivo with 44 million impressions, $5.2 million in spending and a 16% share, followed by BMS’ Zeposia with 31 million impressions, $3.3 million in media buys and an 11% share.

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