When or­phan drugs are block­busters: Re­searchers call on Con­gress to make com­pa­nies pay back tax cred­its

A new re­search let­ter pub­lished to­day in JA­MA ques­tions whether there needs to be con­tin­ued or­phan drug in­cen­tives con­sid­er­ing the spike in the num­ber of ap­provals in rare dis­ease ar­eas, and as many lat­er be­come block­busters.

Among 315 drugs re­viewed from 2008 to 2016, 83 (26%) were ini­tial­ly in­di­cat­ed for or­phan-des­ig­nat­ed con­di­tions. Me­di­an, five-year net sales were $719 mil­lion for or­phan-des­ig­nat­ed drugs and $812 mil­lion for non-or­phan drugs. The West Vir­ginia and Har­vard re­searchers said their da­ta showed how “drugs ini­tial­ly ap­proved for an or­phan des­ig­nat­ed con­di­tion were just as lu­cra­tive for their man­u­fac­tur­ers as drugs de­vel­oped for more com­mon con­di­tions.”

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