Their Staying Power Lies in their Patient-Centricity

Map­ping the Fu­ture of De­cen­tral­ized Clin­i­cal Tri­als

De­cen­tral­ized clin­i­cal tri­als (DCTs) were tra­di­tion­al­ly uti­lized in an iso­lat­ed fash­ion pri­or to the COVID-19 pan­dem­ic. To con­tin­ue their re­search with­in the con­straints of the pan­dem­ic, spon­sors and clin­i­cal in­ves­ti­ga­tors piv­ot­ed to a de­cen­tral­ized mod­el out of ne­ces­si­ty. At the on­set, reg­u­la­to­ry agen­cies of­fered some guid­ance on the dig­i­tal ap­proach­es that are ac­cept­able to en­sure DCT ap­proach­es are ap­plied in a way that main­tains pa­tient safe­ty, as well as da­ta qual­i­ty and in­tegri­ty.

De­cen­tral­ized ap­proach­es re­duce the bur­den of a clin­i­cal tri­al on the pa­tient, ac­cel­er­ate en­roll­ment, im­prove re­ten­tion, broad­en the di­ver­si­ty of the pa­tient pop­u­la­tion and po­ten­tial­ly short­en time­lines. As with any new process, the chal­lenge lies in the con­tin­u­al­ly evolv­ing land­scape. As a re­sult, the lack of es­tab­lished reg­u­la­to­ry guide­lines can be tricky to nav­i­gate.

The par­a­digm of de­cen­tral­ized ap­proach­es is de­vel­op­ing and we should ex­pect that the reg­u­la­to­ry guide­lines will evolve as well. From our broad and var­ied ex­pe­ri­ence in con­duct­ing de­cen­tral­ized stud­ies, we have con­sid­er­a­tions that are help­ful when lay­ing the course for a suc­cess­ful clin­i­cal tri­al.

De­sign Ear­ly En­gage­ment in­to Your Tri­al

In the ab­sence of con­crete, strin­gent guide­lines to guide your tri­al de­sign, en­gage with stake­hold­ers ear­ly in the process. This mit­i­gates any un­pleas­ant sur­pris­es amid the tri­al, as well as the risk of wast­ing valu­able time and re­sources. In­clud­ing reg­u­la­to­ry bod­ies in the ear­ly plan­ning stages through con­sis­tent com­mu­ni­ca­tion is the sin­gle best thing you can do to de­sign a pro­to­col that has the con­text that reg­u­la­to­ry bod­ies need.

How to Keep the Reg­u­la­to­ry Bod­ies En­gaged

While it might seem log­i­cal to re­ly on the guid­ance for de­cen­tral­ized ap­proach­es that were uti­lized dur­ing the height of the COVID-19 pan­dem­ic, we would cau­tion against this. We ex­pect the guid­ance to evolve and vary by re­gion, so di­rect en­gage­ment on sev­er­al points at the out­set is im­per­a­tive. Those af­fect­ed by the Eu­ro­pean Med­i­cine Agency (EMA) will be man­dat­ed to use a cen­tral­ized data­base and por­tal for clin­i­cal tri­als in Jan­u­ary 2022. We’ve out­lined the key points be­low:

Knowl­edge Man­age­ment:

We rec­om­mend hav­ing a clear sys­tem for knowl­edge man­age­ment. This should in­clude reg­u­la­to­ry in­for­ma­tion and ex­pe­ri­en­tial da­ta, so you can ref­er­ence how cer­tain as­pects were ac­cept­ed and con­duct­ed in pre­vi­ous stud­ies, rec­og­niz­ing that the con­text of the study pro­to­col is piv­otal in the in­ter­pre­ta­tion.

Trans­par­ent Da­ta:

Build da­ta sys­tems trans­par­ent­ly so that da­ta can clean­ly flow from one sys­tem to an­oth­er. Reg­u­la­tors will want to see these da­ta maps, and the da­ta maps demon­strate how we will en­sure da­ta in­tegri­ty even when da­ta are col­lect­ed from mul­ti­ple sources. It’s crit­i­cal to have clear mech­a­nisms to en­sure the prin­ci­pal in­ves­ti­ga­tor can achieve the nec­es­sary over­sight.

Mon­i­tor­ing Should be Un­am­bigu­ous:

Cre­at­ing a mon­i­tor­ing plan that fo­cus­es on the crit­i­cal da­ta and process­es and pro­vides clar­i­ty on who is mon­i­tor­ing what da­ta and how, is cru­cial. We rec­om­mend con­sid­er­ing how the au­dit tri­al func­tion­al­i­ty with­in the var­i­ous sys­tems can be used to sup­port mon­i­tor­ing in­tegri­ty.

Pre­pare for Am­bi­gu­i­ty:

It is like­ly that the reg­u­la­tions re­leased won’t be pre­scrip­tive giv­en that the con­text of the pro­to­col is vi­tal in mak­ing an as­sess­ment. Be ex­treme­ly clear on how you’re mea­sur­ing the da­ta, and that it can be re­viewed in a way that’s mean­ing­ful.

In­volve Pa­tients from the Be­gin­ning

Tak­ing a de­cen­tral­ized ap­proach of­ten re­lieves much of the bur­den on pa­tients par­tic­i­pat­ing in the tri­al. As with reg­u­la­to­ry bod­ies, en­gag­ing with pa­tients ear­ly in the process will help you bet­ter em­bed them in the de­sign of a tru­ly pa­tient-cen­tric tri­al. The aim of gain­ing their per­spec­tive is to al­low pa­tients the flex­i­bil­i­ty on how they ac­cess their care, whether through mo­bile re­search nurs­ing, or on site.

En­sur­ing a spec­trum of pa­tients across rep­re­sen­ta­tive ages, di­ver­si­ty and so­cioe­co­nom­ic fac­tors, is ul­ti­mate­ly bet­ter for all pa­tients. Ac­cess­ing them via pa­tient ad­vo­ca­cy groups or through mul­ti­ple spe­cial­ist com­pa­nies that have built pa­tient com­mu­ni­ties, helps you con­nect to a much more var­ied pa­tient pop­u­la­tion.

Part­ner­ing With Tech­nol­o­gy Providers

Tech­nol­o­gy is the linch­pin to DCTs to al­low for re­mote da­ta col­lec­tion and height­ened da­ta in­tegri­ty. But we of­ten cau­tion against let­ting tech­nol­o­gy lead the study. When you are en­gag­ing a tech­nol­o­gy part­ner, it’s op­ti­mal to have a holis­tic view of their of­fer­ing and ex­pe­ri­ence.

While a tech­nol­o­gy part­ner’s ex­pe­ri­ence is valu­able, it doesn’t al­ways guar­an­tee ro­bust in­tel­li­gence. You need to un­der­stand the con­text in which the in­tel­li­gence was gath­ered, and whether it was ap­plied cor­rect­ly to en­sure that the study pa­ra­me­ters and the in­tend­ed use of the da­ta were com­pa­ra­ble.

An­tic­i­pate In­ter­play

Con­duct­ing a glob­al DCT will in­evitably lead to ad­di­tion­al com­plex­i­ties from re­gion to re­gion. Spon­sors should be flex­i­ble and adapt­able de­pend­ing on the lo­cal reg­u­la­tions. There is al­so in­ter­op­er­abil­i­ty be­tween poli­cies and reg­u­la­tions, from clin­i­cal tri­al reg­u­la­tions to da­ta pri­va­cy reg­u­la­tions, as well as stan­dard med­ical man­age­ment guide­lines. It is vi­tal to have an aware­ness of the reg­u­la­tions that can af­fect any giv­en sec­tion and that the most strin­gent guide­lines are ap­plied.

Be­come a Part of the Evo­lu­tion

The reg­u­la­to­ry en­vi­ron­ment for DCTs is go­ing to con­tin­u­al­ly evolve as more and more clin­i­cal tri­als adopt this method­ol­o­gy. One of the most im­pact­ful ways to ef­fi­cient­ly work with­in this land­scape is through ear­ly stake­hold­er en­gage­ment. In­cor­po­rat­ing stake­hold­er feed­back in the ear­ly stages and cre­at­ing a flow of in­for­ma­tion will help en­sure that you re­al­ize the ben­e­fits DCTs of­fer.

We view hav­ing the abil­i­ty to have a tru­ly pa­tient-cen­tric ap­proach as one of the most im­por­tant ben­e­fits a DCT can of­fer. High­er ef­fi­cien­cy, a re­duc­tion in study time­lines, greater ac­cess to a wider and more di­verse pa­tient pop­u­la­tion, as well as high-qual­i­ty da­ta, are the ben­e­fits of DCTs.

To gain full ad­van­tage of these ben­e­fits, think­ing through all as­pects of the DCT and en­gag­ing with rel­e­vant stake­hold­ers ear­ly on will help you em­bed this knowl­edge in­to the tri­al. Trans­paren­cy with reg­u­la­tors, along with the learn­ings from pre­vi­ous tri­als, will help in­form fu­ture guide­lines as DCTs be­come a more prac­ticed method.

Learn more about De­cen­tral­ized So­lu­tions from Sy­neos Health here.


Noolie Gre­go­ry
Vice Pres­i­dent,
De­cen­tral­ized Clin­i­cal
Tri­al Op­er­a­tions,
Sy­neos Health
He­len Howitt
Ex­ec­u­tive Di­rec­tor,
Process Qual­i­ty
Sy­neos Health