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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER