More ef­fi­cient rare pe­di­atric drug de­vel­op­ment: FDA drafts guid­ance

The FDA on Wednes­day re­leased a draft guid­ance that could help speed the de­vel­op­ment of treat­ments for rare pe­di­atric dis­ease drugs.

Janet Wood­cock, di­rec­tor of FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search, said the draft guid­ance “could elim­i­nate the need for cer­tain clin­i­cal stud­ies and, when pe­di­atric clin­i­cal stud­ies are need­ed, could re­duce the to­tal num­ber of pa­tients who would re­ceive a place­bo in­stead of a po­ten­tial­ly help­ful drug.”

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