FDA fi­nal­izes guid­ance clos­ing pe­di­atric study loop­hole

The FDA on Thurs­day fi­nal­ized guid­ance stat­ing that it does not in­tend to grant or­phan drug des­ig­na­tion for pe­di­atric sub­pop­u­la­tions of com­mon dis­eases in an ef­fort to close a loop­hole in the Pe­di­atric Re­search Eq­ui­ty Act (PREA).

While PREA was en­act­ed to pro­mote pe­di­atric re­search, the act in­clud­ed an ex­emp­tion from pe­di­atric study re­quire­ments for or­phan des­ig­nat­ed drugs.

This ex­emp­tion cre­at­ed a loop­hole that al­lowed spon­sors to get a pe­di­atric sub­pop­u­la­tion des­ig­na­tion for a non-or­phan dis­ease in adult pop­u­la­tions and be ex­empt­ed from con­duct­ing pe­di­atric stud­ies nor­mal­ly re­quired un­der PREA when ap­ply­ing for an adult in­di­ca­tion for that dis­ease.

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