Pa­tient reg­istries may prove valu­able in reg­u­la­to­ry de­ci­sions, EMA study finds

Al­though pa­tient reg­istries can be het­ero­ge­neous in their qual­i­ty and de­sign, a new study in Drug Safe­ty co-au­thored by an EMA of­fi­cial ex­plains how pa­tient reg­istries could pro­vide valu­able da­ta for reg­u­la­to­ry de­ci­sions.

The au­thors ex­plain how such reg­istries can be used when ran­dom­ized con­trolled tri­als are not fea­si­ble, such as with some rare dis­eases, or to meet post­mar­ket­ing com­mit­ments.

“For prod­ucts grant­ed con­di­tion­al mar­ket­ing ap­proval, reg­istry stud­ies may pro­vide post-au­tho­ri­sa­tion da­ta to ful­fil reg­u­la­tor-im­posed spe­cif­ic oblig­a­tions to con­firm safe­ty and/or ef­fec­tive­ness, as is the case with the re­cent­ly au­tho­rised chimeric anti­gen re­cep­tor (CAR) T-cell prod­ucts, tia­gen­le­cleu­cel and axi­cab­ta­gene ciloleu­cel,” the study says.

The eight au­thors, from the EMA, Queens Mary Uni­ver­si­ty of Lon­don, the Uni­ver­si­ty of Gronin­gen in the Nether­lands and the Dutch Med­i­cines Eval­u­a­tion Board, al­so ex­plain how reg­istries are cur­rent­ly un­der­used.

Of 335 prod­ucts rec­om­mend­ed for ap­proval by the EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use be­tween 2005 and 2013, 31 reg­istries were re­quest­ed to ful­fill a con­di­tion of the mar­ket­ing au­tho­riza­tion, but by De­cem­ber 2017, just ten had been com­plet­ed.

To try to fig­ure out what fac­tors fa­cil­i­tate reg­istry use, the au­thors syn­the­sized ob­ser­va­tions and rec­om­men­da­tions pub­lished in five re­ports aris­ing from a mul­ti-stake­hold­er EMA con­sul­ta­tion and four dis­ease-spe­cif­ic work­shops con­duct­ed as part of the EMA’s Pa­tient Reg­istries Ini­tia­tive.

The study ex­plains how the ab­sence or in­com­plete­ness of 10 dif­fer­ent fac­tors “great­ly im­ped­ed reg­istry use.”
Such fac­tors in­clude the use of com­mon core da­ta sets, com­mon da­ta cod­ing ter­mi­nolo­gies, da­ta ac­cess and shar­ing and da­ta link­age ca­pac­i­ty, among oth­ers.

“From a reg­u­la­to­ry per­spec­tive, the ul­ti­mate re­quire­ment of pa­tient reg­istries is that they per­mit the con­duct of high-qual­i­ty stud­ies that eval­u­ate the safe­ty and ef­fec­tive­ness of med­i­cines. Recog­nis­ing the ben­e­fits and chal­lenges in­her­ent in us­ing ob­ser­va­tion­al da­ta for med­i­cines as­sess­ments, in 2019, EMA will pub­lish method­olog­i­cal and op­er­a­tional ad­vice on han­dling reg­istry da­ta in post-au­tho­ri­sa­tion stud­ies, tak­ing in­to ac­count re­spons­es to its open con­sul­ta­tion on a pre­lim­i­nary dis­cus­sion pa­per,” the study adds.

Pa­tient Reg­istries: An Un­der­used Re­source for Med­i­cines Eval­u­a­tion

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: Shut­ter­stock


Zachary Brennan

managing editor, RAPS

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