FDA to al­low on­line sub­mis­sions of or­phan des­ig­na­tion re­quests

Amy Aber­nethy

As part of work build­ing on the FDA’s or­phan drug mod­ern­iza­tion plan from 2017, the agency said Fri­day that lat­er this year it will move from a pa­per-based process to a new cloud-based on­line sub­mis­sion por­tal for or­phan drug des­ig­na­tion re­quests.

The shift to on­line sub­mis­sions will ease the process of mak­ing or­phan drug des­ig­na­tion re­quests, which are cur­rent­ly mailed in­to the Of­fice of Or­phan Prod­ucts De­vel­op­ment (OOPD) on a CD.

“The Or­phan Drug Tech­nol­o­gy Mod­ern­iza­tion ef­fort will al­low for a more con­nect­ed in­for­ma­tion tech­nol­o­gy sys­tem, ad­vanced an­a­lyt­ics, and im­prove­ments in fa­cil­i­tat­ing knowl­edge man­age­ment. In ad­di­tion, it will pro­vide ex­ter­nal spon­sors with more ef­fi­cient sub­mis­sion of doc­u­ments and en­hanced di­rect com­mu­ni­ca­tion with the FDA,” Amy Aber­nethy, prin­ci­pal deputy com­mis­sion­er and act­ing CIO and Janet May­nard, di­rec­tor of the FDA’s OOPD, wrote in a blog post.

Aber­nethy and May­nard said this ef­fort is al­so part of the FDA’s tech­nol­o­gy mod­ern­iza­tion plan.

Janet May­nard FDA

The shift on or­phan drug des­ig­na­tion re­quest sub­mis­sions fol­lows the Cen­ter for Drug Eval­u­a­tion and Re­search’s (CDER) ap­proval of more than twice as many or­phan drugs in the past eight years as in the pre­vi­ous eight years.

In ad­di­tion, the FDA will hold a meet­ing on rare dis­ease drug de­vel­op­ment next month to ob­tain stake­hold­ers’ per­spec­tives on chal­lenges and so­lu­tions in rare dis­ease prod­uct de­vel­op­ment. A pan­el dis­cus­sion with FDA cen­ter di­rec­tors and oth­er di­a­logue with FDA se­nior staff will take place.

The FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search (CBER) is al­so or­ga­niz­ing a pub­lic work­shop, to be held in March, ti­tled, “Fa­cil­i­tat­ing End-to-End De­vel­op­ment of In­di­vid­u­al­ized Ther­a­peu­tics.”

Janet Wood­cock FDA

In an ed­i­to­r­i­al pub­lished in the New Eng­land Jour­nal of Med­i­cine in Oc­to­ber, Janet Wood­cock, di­rec­tor of CDER, and Pe­ter Marks, di­rec­tor of CBER, ex­plained how these new per­son­al­ized treat­ments can “per­mit the de­lin­eation of path­ways for tru­ly in­di­vid­u­al­ized drug de­vel­op­ment,” but cur­rent­ly this type of “drug dis­cov­ery and de­vel­op­ment is most ad­vanced for ASOs [an­ti­sense oligonu­cleotides], oth­er types of treat­ments, in­clud­ing in­di­vid­u­al­ized cell and gene ther­a­pies, are fol­low­ing close­ly be­hind.”

RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

So­cial im­age: FDA, AP Im­ages


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