FDA dis­cuss­es RWD, RWE with in­dus­try, acad­e­mia

In­dus­try, acad­e­mia and FDA of­fi­cials gath­ered Fri­day in Bethes­da, MD to dis­cuss the use of re­al-world da­ta (RWD) and re­al-world ev­i­dence (RWE) in the pre­mar­ket and post­mar­ket set­tings.

Jacque­line Cor­ri­g­an-Cu­ray, di­rec­tor of CDER’s Of­fice of Med­ical Pol­i­cy, kicked off the joint FDA-Amer­i­can As­so­ci­a­tion for Can­cer Re­search meet­ing to dis­cuss the FDA’s frame­work for us­ing RWE, which was un­veiled in De­cem­ber 2018. The frame­work is meant to guide the FDA on eval­u­at­ing the po­ten­tial use of RWE to sup­port changes to la­bel­ing, in­clud­ing adding or mod­i­fy­ing an in­di­ca­tion or adding com­par­a­tive ef­fec­tive­ness or safe­ty in­for­ma­tion.

“Trans­paren­cy on study de­sign and analy­sis be­fore ex­e­cu­tion is crit­i­cal for en­sur­ing con­fi­dence in the re­sult,” Cor­ri­g­an-Cu­ray said, not­ing that RWE may be able to de­tect long-term but in­fre­quent out­comes.

More re­cent­ly, the FDA is­sued draft guid­ance on the types of RWD and RWE in­for­ma­tion that might be sub­mit­ted. Cor­ri­g­an-Cu­ray al­so of­fered sev­er­al ex­am­ples of when RWE has in­formed ef­fec­tive­ness de­ter­mi­na­tions.

She fur­ther dis­cussed how com­pa­nies should de­cide if da­ta can be con­sid­ered fit for use and how the qual­i­ty of the da­ta can be as­sured by the FDA.

Work on RWE du­pli­ca­tion demon­stra­tion projects is al­so un­der­way, with in­ves­ti­ga­tors from Brigham and Women’s Hos­pi­tal build­ing an ev­i­dence base for re­al world da­ta through large-scale repli­ca­tion of ran­dom­ized con­trolled tri­als. The in­ves­ti­ga­tors said their goal is to un­der­stand for what types of clin­i­cal ques­tions RWD analy­ses can be con­duct­ed with con­fi­dence and how to im­ple­ment such stud­ies.

In ad­di­tion, the FDA is work­ing on a demon­stra­tion project for the FDA’s MyS­tud­ies mo­bile app, de­signed to fa­cil­i­tate the in­put of re­al-world da­ta di­rect­ly by pa­tients which can be linked to elec­tron­ic health da­ta sup­port­ing tra­di­tion­al clin­i­cal tri­als, prag­mat­ic tri­als, ob­ser­va­tion­al stud­ies and reg­istries.

Look­ing for­ward, Cor­ri­g­an-Cu­ray point­ed to ef­forts to in­crease in­ter­nal FDA ed­u­ca­tion on RWE and RWD, con­tin­u­ing to hold pub­lic meet­ings and ob­tain ex­perts’ in­put, and to con­sid­er mech­a­nisms and stan­dards to as­sess RWD qual­i­ty. “We wel­come in­put on ideas re­lat­ed to the reg­u­la­to­ry sci­ence of us­ing this da­ta,” she said, es­pe­cial­ly as the FDA iden­ti­fies ad­di­tion­al guid­ance and eval­u­ates what sit­u­a­tions can re­ly on ob­ser­va­tion­al stud­ies.

Oth­er ses­sions through­out the day dis­cussed pre­mar­ket and post­mar­ket use cas­es, as well as large ge­nom­ic data­bas­es and fu­ture di­rec­tions for RWE.

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.

So­cial im­age: FDA, AP Im­ages


Zachary Brennan

managing editor, RAPS

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