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.

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