Scott Got­tlieb is clos­ing a big loop­hole in the or­phan drug law

FDA com­mis­sion­er Scott Got­tlieb an­nounced Tues­day that the agency will re­lease guid­ance to close a loop­hole that al­lows com­pa­nies to avoid their oblig­a­tion to study phar­ma­ceu­ti­cals in pe­di­atric pop­u­la­tions.

The sit­u­a­tion, ac­cord­ing to Got­tlieb, aris­es if spon­sors re­ceived an or­phan des­ig­na­tion for a pe­di­atric sub­type of an oth­er­wise com­mon and non-or­phaned adult dis­ease.

For ex­am­ple, with a con­di­tion like in­flam­ma­to­ry bow­el dis­ease, a drug may be ap­proved to treat the large pop­u­la­tion of adults with the con­di­tion but then the same drug may be grant­ed an or­phan des­ig­na­tion to treat a sub­set of chil­dren suf­fer­ing from IBD.

“But once a drug re­ceives an or­phan des­ig­na­tion for a pe­di­atric pop­u­la­tion of the adult dis­ease, the drug then be­comes statu­to­ri­ly ex­empt from the re­quire­ments” of the Pe­di­atric Re­search Eq­ui­ty Act (PREA), Got­tlieb ex­plains.

“It’s a loop­hole that is in di­rect op­po­si­tion to what Con­gress in­tend­ed,” he wrote. “No­body en­vi­sioned this un­in­tend­ed con­flict be­tween the orig­i­nal ODA [Or­phan Drug Act] and the pro­vi­sions out­lined in PREA. In ef­fect, by let­ting spon­sors des­ig­nate pe­di­atric sub­pop­u­la­tions of drugs in­tend­ed to treat adult dis­eases, the drug mak­ers re­ceive an un­in­tend­ed ‘free pass’ from hav­ing to study drugs in these or oth­er pe­di­atric us­es. Thus, rather than en­sur­ing more pe­di­atric re­search, as Con­gress en­vi­sioned, we can end up with few­er pe­di­atric stud­ies. FDA will soon is­sue a draft guid­ance doc­u­ment that’s aimed at clos­ing this in­ad­ver­tent loop­hole.”

Nan­cy Good­man of Kids v Can­cer, whose RACE for Chil­dren’s Act was in­clud­ed in the FDA user fee reau­tho­riza­tion law, told me that the lan­guage on clos­ing the loop­hole was ini­tial­ly go­ing to be in­clud­ed in FDARA. It’s un­clear why it didn’t make it in the fi­nal lan­guage.

A pub­lic meet­ing an­nounced by Got­tlieb will al­so ad­dress some of the statutes from FDARA and get in­put on com­plex sci­en­tif­ic and reg­u­la­to­ry is­sues, such as those raised by mol­e­c­u­lar­ly tar­get­ed drugs and bi­o­log­ics and the ap­pro­pri­ate ap­pli­ca­tion of or­phan in­cen­tives.

In ad­di­tion to clos­ing that loop­hole and the meet­ing, Got­tlieb said FDA has now re­viewed all or­phan drug des­ig­na­tion re­quests old­er than 120 days and will put in place new poli­cies to im­prove the ef­fi­cien­cy of FDA’s re­view process to en­sure that we meet our new 90-day man­date to pre­vent new back­logs.

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.

Pho­to: AP Im­ages / end­points news


Zachary Brennan

managing editor, RAPS

Hal Barron, GSK

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I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

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Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

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