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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Alice Shaw, Lung Cancer Foundation of America

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
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