OCTOBER2018OrphanDrugsintheUnitedStatesGrowthTrendsinRareDiseaseTreatmentsIntroductionRarediseaseshaveincreasinglybeendiscussedinthemediaoverthepastseveralyearsasthenumberofnewtreatmentshassurgedandprovidednewhealthbenefitstopatients.Thereareapproximately7,000rarediseasesthataffect25to30millionpeopleintheUnitedStates–morethanhalfofwhomarechildren–buttreatmentsareavailableforjust5%ofthem.Manyofthesediseasesarelife-threateningorlife-limiting,andwhilesomeconditionscannowbetreatedandmanagedaschronicconditions,forthefirsttimenewcurativetreatmentsarebringingtheprospectofabetterlifetosomepatients.Evenwithadramaticincreaseinthenumbersoforphandrugsapprovedinthep...
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