BIO, Al­ler­gan and Roche call for FDA flex­i­bil­i­ty with pa­tient ex­pe­ri­ence da­ta

In­dus­try group BIO and bio­phar­ma com­pa­nies Al­ler­gan and Roche are call­ing for the FDA to be flex­i­ble in its ap­proach for us­ing pa­tient ex­pe­ri­ence da­ta (PED) and in al­low­ing the use of such da­ta in la­bel­ing.

BIO said that it “strong­ly be­lieves” that in or­der for a broad adop­tion of PED, any up­com­ing guid­ance doc­u­ments on pa­tient-fo­cused drug de­vel­op­ment “should em­pha­size the FDA’s will­ing­ness to ex­er­cise reg­u­la­to­ry flex­i­bil­i­ty and ac­cep­tance of in­no­v­a­tive de­signs and ap­proach­es for col­lect­ing PED.”

Danielle Friend

For ex­am­ple, the draft guid­ance on gath­er­ing pa­tient in­put, ac­cord­ing to Danielle Friend, di­rec­tor of sci­ence and reg­u­la­to­ry af­fairs at BIO, is “high­ly fo­cused on sta­tis­ti­cal re­quire­ments for col­lec­tion of sub­jec­tive mea­sures and al­though rep­re­sen­ta­tion of the pop­u­la­tion is para­mount, there is al­so a need to bal­ance such rep­re­sen­ta­tive­ness with sta­tis­ti­cal va­lid­i­ty, as the re­quire­ment of many stra­ta for rep­re­sen­ta­tion could have a neg­a­tive im­pact on sta­tis­ti­cal pow­er.”

Sim­i­lar to BIO, Al­ler­gan urges the FDA to con­sid­er the need for flex­i­bil­i­ty with re­spect to the method­olog­i­cal re­quire­ments and op­por­tu­ni­ties for en­gage­ment to pro­vide spon­sors with clar­i­ty to in­form ev­i­dence gen­er­a­tion in a time­ly man­ner.

BIO al­so calls for flex­i­bil­i­ty on sam­pling meth­ods and in col­lect­ing da­ta for small­er pa­tient pop­u­la­tions, such as with rare dis­eases. The in­dus­try group ad­di­tion­al­ly re­quests that the FDA de­lin­eate be­tween col­lect­ing PED to in­form clin­i­cal stud­ies (e.g., de­vel­op­ment of a clin­i­cal out­come as­sess­ment tool or to in­form clin­i­cal study end­points) and PED col­lect­ed with­in a clin­i­cal study meant to in­form a reg­u­la­to­ry de­ci­sion.

Roche’s Genen­tech, mean­while, calls on the agency to fur­ther clar­i­fy when PED can be la­bel-en­abling.

Janet Jenk­ins-Showal­ter

“As dif­fer­ent types of pa­tient ex­pe­ri­ence da­ta serve dif­fer­ent pur­pos­es, we urge the FDA to de­scribe rea­son­able stan­dards that en­able the in­clu­sion of fit-for-pur­pose pa­tient ex­pe­ri­ence da­ta with­in dif­fer­ent sec­tions of drug la­bel,” Janet Jenk­ins-Showal­ter, head of US reg­u­la­to­ry pol­i­cy at Genen­tech, writes.

Al­ler­gan al­so calls on the FDA to sub­stan­ti­ate the dif­fer­ences in ev­i­den­tiary stan­dards for PED for reg­u­la­to­ry-de­ci­sion mak­ing vs. la­bel­ing.

Jenk­ins-Showal­ter adds: “We would al­so high­light the im­por­tance of cre­den­tial­ing and shar­ing pa­tient ex­pe­ri­ence da­ta deemed of suf­fi­cient qual­i­ty to in­form drug de­vel­op­ment pro­gram de­sign (e.g., bur­den of dis­ease, bur­den of ex­ist­ing treat­ments) that is col­lect­ed out­side the con­text of an IND pro­gram. It is im­por­tant that these reg­u­la­to­ry qual­i­ty da­ta are shared with the broad­er com­mu­ni­ty of Spon­sors, mea­sure­ment sci­en­tists, pa­tients, and reg­u­la­tors such that clin­i­cal sci­ence is able to ad­vance to­wards the de­vel­op­ment of out­comes that are mean­ing­ful to pa­tients.”

First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

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