Re­searchers find 15% of tri­als could be repli­cat­ed us­ing re­al world da­ta

A study pub­lished this week in JA­MA Net­work Open finds that cur­rent­ly avail­able re­al-world da­ta (RWD) sources can on­ly be used to fea­si­bly repli­cate 15% of clin­i­cal tri­als.

The aim of the study was to de­ter­mine whether RWD could be used to pow­er ob­ser­va­tion­al stud­ies that an­swer the same clin­i­cal ques­tions as tra­di­tion­al clin­i­cal tri­als.

Ran­dom­ized con­trolled tri­als (RCTs) are con­sid­ered the gold stan­dard for clin­i­cal ev­i­dence to sup­port the safe­ty and ef­fi­ca­cy of med­ical prod­ucts due to high lev­els of in­ter­nal con­sis­ten­cy and re­duced bias.

How­ev­er, as the au­thors of the study write, “Com­pared with RCTs, RWE [re­al-world ev­i­dence] bet­ter re­flects the ac­tu­al clin­i­cal en­vi­ron­ments in which med­ical in­ter­ven­tions are used, in­clud­ing pa­tient de­mo­graph­ics, co­mor­bidi­ties, ad­her­ence, and con­cur­rent treat­ments,” not­ing that RCTs are cost­ly and time in­ten­sive com­pared to ob­ser­va­tion­al stud­ies.

To con­duct the study, the au­thors re­viewed 220 clin­i­cal tri­als con­duct­ed in the US that were pub­lished in the top sev­en med­ical jour­nals in 2017 and de­ter­mined whether RWD ob­tained from in­sur­ance claims and elec­tron­ic health records (EHRs) con­tained the in­for­ma­tion nec­es­sary to repli­cate the stud­ies.

Of those tri­als, the au­thors were on­ly able to iden­ti­fy 86 (39%) that “had an in­ter­ven­tion that could be as­cer­tained from in­sur­ance claims and/or EHR da­ta.” From there, the au­thors nar­rowed the tri­als fur­ther to iden­ti­fy ones with an in­di­ca­tion and in­clu­sion/ex­clu­sion cri­te­ria that could be ex­tract­ed from RWD.

From there the au­thors were able to iden­ti­fy just 33 (15%) tri­als with one or more pri­ma­ry end­points that could be as­cer­tained from avail­able RWD sources.

“This find­ing sug­gests the po­ten­tial for re­al-world ev­i­dence to com­ple­ment clin­i­cal tri­als, both by ex­am­in­ing the con­cor­dance be­tween ran­dom­ized ex­per­i­ments and ob­ser­va­tion­al stud­ies and by com­par­ing the gen­er­al­iz­abil­i­ty of the tri­al pop­u­la­tion with the re­al-world pop­u­la­tion of in­ter­est,” the au­thors write.

How­ev­er, the au­thors cau­tion that for new prod­ucts, RWE is un­like­ly to serve as a re­place­ment for RCTs and point out that many of the tri­als they looked at could not be repli­cat­ed be­cause the da­ta nec­es­sary to do so “are un­like­ly to ap­pear in an EHR in struc­tured form if at all.”

But the au­thors stress that RWE could be used to pro­vide “crit­i­cal in­sights” in­to prod­uct safe­ty and ef­fi­ca­cy in the postap­proval set­ting and could al­low the FDA to iden­ti­fy and act on safe­ty is­sues more quick­ly.


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.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Paul Hudson, Sanofi CEO (ROMUALD MEIGNEUX/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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