Only a small fraction of rare pediatric designations have led to vouchers, new FDA analysis shows

Out of hun­dreds of po­ten­tial drugs with rare pe­di­atric dis­ease des­ig­na­tions, on­ly 7% of them have won pri­or­i­ty re­view vouch­ers from the FDA, ac­cord­ing to an agency re­view of a decade’s worth of da­ta that are be­ing made pub­lic for the first time.

The analy­sis, pub­lished Mon­day in the Or­phanet Jour­nal of Rare Dis­eases, looked at 569 rare pe­di­atric dis­ease des­ig­na­tions from the FDA be­tween 2013 and the end of 2022. On­ly 38 des­ig­nat­ed prod­ucts have won pri­or­i­ty re­view vouch­ers, which are award­ed when these prod­ucts win ap­proval and the vouch­ers can be sold or used to speed up an­oth­er drug’s re­view time­line.

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