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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,100+ biopharma pros reading Endpoints daily — and it's free.

Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,100+ biopharma pros reading Endpoints daily — and it's free.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

J&J ad­comm live blog: J&J is al­so test­ing a two-dose vac­cine. What hap­pens if it's more ef­fec­tive?

J&J has spent the day touting the data behind their Covid-19 vaccine, but one advisor pointed that, in a few months, another batch of data could force them into a curious dilemma.

J&J was the only major vaccine developer to attempt to produce a single-dose vaccine, a huge asset when trying to rapidly inoculate the world against an ongoing public health threat, but they hedged their bets. Alongside their main Phase III trial, they also launched another that would test two doses, each spaced two months apart.

Genen­tech plots $53M dis­cov­ery quest aimed at spark­ing a 'Holy moly' piv­ot in neu­ro R&D

Genentech has committed $53 million to back a 10-year quest aimed at going back to the drawing board to use new technology and fresh scientific insights to generate a pipeline of drugs for neurological diseases.

Roche’s big South San Francisco hub will mix it up with the scientists drawn together for the Weill Neurohub — formed in 2019 as a joint research partnership involving UCSF, Berkeley and the University of Washington — in an exploration of the field to develop new therapies for some of the toughest diseases in drug R&D: Alzheimer’s, Parkinson’s, Huntington’s, ALS and autism.

Am­gen, As­traZeneca speed to­ward fil­ing next-gen an­ti­body for asth­ma af­ter un­cork­ing full late-stage da­ta

On the hunt for a novel competitor to Sanofi and Regeneron’s Dupixent in severe asthma, Amgen and AstraZeneca posted “exciting” results from their next-gen antibody late last year. Now, the partners are showing their hands, and the results look good enough for approval.

Amgen and AstraZeneca’s tezepelumab plus standard of care cut the rate of severe asthma attacks by 56% at the one-year mark compared with SOC alone, according to full data from the Phase III NAVIGATOR study presented Friday at the virtual American Academy of Allergy, Asthma & Immunology meeting. And those significant results were consistent regardless of patients’ baseline eosinophil counts.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,100+ biopharma pros reading Endpoints daily — and it's free.