Stake­hold­ers brace for bumps in rare pe­di­atric dis­ease PRV reau­tho­riza­tion

A vouch­er pro­gram de­signed to stim­u­late drug de­vel­op­ment for rare pe­di­atric dis­eases will sun­set lat­er this year if law­mak­ers don’t reau­tho­rize it — and rare dis­ease ad­vo­cates and ex­perts are brac­ing for bumps in the road to re­new­al.

The Rare Pe­di­atric Dis­ease Pri­or­i­ty Re­view Vouch­er Pro­gram al­lows the FDA to award vouch­ers to rare dis­ease drug de­vel­op­ers who then can use the vouch­er to re­ceive pri­or­i­ty re­view on a sub­se­quent ap­pli­ca­tion or sell it to an­oth­er de­vel­op­er. The pro­gram was first es­tab­lished by Con­gress in 2012 and has since been reau­tho­rized in four-year in­cre­ments.

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