In­dus­try calls for changes in two REMS guid­ances

In­dus­try groups rep­re­sent­ing the phar­ma­ceu­ti­cal, biotech­nol­o­gy and gener­ic drug in­dus­tries are call­ing for changes to two re­cent FDA draft guid­ances on REMS.

The draft guid­ances, re­leased for com­ment in Jan­u­ary, de­tail a frame­work for com­pa­nies to de­vel­op an as­sess­ment plan for their REMS pro­grams and pro­vide rec­om­men­da­tions for sur­vey­ing pa­tient and health care provider knowl­edge of REMS in­for­ma­tion.

While PhRMA says it com­mends the FDA for pro­vid­ing rec­om­men­da­tions for de­vel­op­ing REMS as­sess­ments, the group ar­gues that the guid­ance should be prospec­tive and not ap­ply to ex­ist­ing REMS that al­ready have as­sess­ments in place to min­i­mize dis­rup­tion to those pro­grams.

PhRMA says it is con­cerned that new met­rics for as­sess­ing a REMS in­tro­duced in the draft guid­ance may pose chal­lenges to spon­sors with ex­ist­ing REMS. “Such changes may sig­nif­i­cant­ly mod­i­fy the REMS pro­gram and in­crease stake­hold­er bur­den through changes to da­ta col­lec­tion re­quire­ments,” PhRMA writes.

In­stead, PhRMA asks the FDA to weigh the util­i­ty of new met­rics against how ef­fi­cient­ly they can mea­sure the ef­fec­tive­ness of the REMS.

PhRMA al­so asks the FDA for ad­di­tion­al de­tails on how met­rics on pa­tient ac­cess and bur­den to the health­care sys­tem should be in­cor­po­rat­ed in­to as­sess­ment plans. “PhRMA be­lieves that if a pre-spec­i­fied thresh­old is not met, there should be an op­por­tu­ni­ty to re­vise and im­prove the REMS goals,” PhRMA writes.

Sim­i­lar­ly, BIO calls for the FDA to clar­i­fy how the guid­ance will ap­ply to ex­ist­ing and well-es­tab­lished REMS and en­cour­ages the FDA to ex­empt ex­ist­ing REMS from eval­u­at­ing pa­tient ac­cess and bur­den on the health­care sys­tem.

BIO points out that there are dif­fer­ent chal­lenges to as­sess­ing a REMS re­quired as part of a prod­uct’s ap­proval and one re­quired af­ter ap­proval due to a post-mar­ket safe­ty is­sue. “In the case of REMS that are re­quired as part of an ini­tial ap­proval, there is no com­para­tor group of pa­tients who had ac­cess to the prod­uct with­out REMS. With­out that com­para­tor group, it is very dif­fi­cult to mea­sure the ef­fect of the REMS pro­gram on pa­tient ac­cess,” BIO writes.

Rep­re­sent­ing the gener­ic drug in­dus­try, AAM rais­es con­cerns about how the guid­ance will ap­ply to shared REMS sys­tems. “The rec­om­men­da­tions in the guid­ance are eas­i­er to im­ple­ment in a brand REMS con­text and will be much more dif­fi­cult to em­ploy for sin­gle, shared sys­tem REMS or sep­a­rate shared REMS that have mul­ti­ple AN­DA par­tic­i­pants,” AAM writes.

In its com­ments on the sur­vey method­olo­gies guid­ance, PhRMA re­quests that the FDA ex­pand the guid­ance to in­clude rec­om­men­da­tions for sur­vey­ing pa­tient and health­care prac­ti­tion­er at­ti­tudes in ad­di­tion to sur­vey­ing their knowl­edge.

The draft guid­ance spec­i­fies that pa­tients should ei­ther be cur­rent­ly tak­ing the drug or have tak­en it with­in a “rea­son­able time,” which PhRMA says should be flex­i­ble de­pend­ing on the char­ac­ter­is­tics of the drug and its dos­ing sched­ule.

The draft guid­ance al­so says that sur­vey par­tic­i­pants should not have pre­vi­ous­ly par­tic­i­pat­ed in a REMS sur­vey for the same prod­uct. “PhM­RA be­lieves this will be chal­leng­ing for spon­sors and oth­er stake­hold­ers … where the drug has been on the mar­ket for a long time, or for a rare dis­ease prod­uct used in a lim­it­ed pa­tient pop­u­la­tion and pre­scribed by a small­er num­ber of spe­cial­ized pre­scribers,” PhRMA writes.

Ad­di­tion­al­ly, PhRMA asks for more de­tails from the FDA on the util­i­ty of open-end­ed ques­tions in the sur­veys. While the guid­ance rec­om­mends sur­veys in­clude a va­ri­ety of ques­tion types, PhRMA says it be­lieves the util­i­ty of re­spons­es to open-end­ed ques­tions in this con­text would be lim­it­ed.


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.

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