Epi­der­mol­y­sis bul­losa: FDA seeks to help de­vel­op­ment of new treat­ments

The FDA re­cent­ly re­leased guid­ance to help com­pa­nies de­vel­op new treat­ments for epi­der­mol­y­sis bul­losa (EB), which is a de­bil­i­tat­ing and of­ten fa­tal rare dis­ease.

The six-page draft guid­ance of­fers de­tails on con­sid­er­a­tions for clin­i­cal tri­al de­sign, in­clud­ing tri­al pop­u­la­tion (es­ti­mates sug­gest 25,000-50,000 peo­ple in the US have EB), dis­cus­sion of ef­fi­ca­cy end­points (i.e. ef­fects on pa­tients’ signs or symp­toms such as itch­ing, pain, blis­ter pre­ven­tion and wound heal­ing) and spe­cial con­sid­er­a­tions to max­i­mize pa­tient com­fort (i.e. al­low­ing pho­to or video doc­u­men­ta­tion of wounds dur­ing rou­tine dress­ing changes in the home).

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