WhitePaperFromOrphantoOpportunity:MasteringRareDiseaseLaunchExcellenceSTEFANLUTZMAYER,SeniorConsultant,EMEAThoughtLeadershipKIRSTIESCOTT,SeniorConsultant,EMEAThoughtLeadershipSARAHRICKWOOD,VicePresident,EMEAThoughtLeadershipIntroductionMedicinesforrarediseasesarealeadingcontributortoallnoveldrugapprovalsandlaunches.Duringthepastfiveyears,orphanmedicineshavetypicallyrepresentedmorethanhalfofnewactivesubstanceapprovalsintheU.S.,andonaverage45%ofapprovalsinEurope.1Infact,143drugslaunchedwithorphandrugdesignationintheU.S.inthepastfiveyears,representing53%ofthe268launches.2Clearly,rarediseasesrepresentasignificantfocusofinnovationwith44%oftheclinicaltrialactivitygloballyfocusedonrarediseases.3Thehighlevelofunmetneedremains,withanestimated95%oftheover7,000-10,000rarediseasesstillnothavingtreatmentsavailable.4IQVIA’sLaunchExcellenceseriesshowstoday’slaunchthreatentomaketheenvironmentmorechallengingenvironmentistougherforinnovativelaunchestofororphanmedicines,assomeofthekeyenablersforfulfiltheirtruepotentialingeneral,5andthisisalsotheorphandrugsarechangedoratrisk.caseinraredisease.Whilstlegislationthatintroducedincentivesfordevelopmentinrarediseases(suchasWithbothbiotechandlargepharmaheavilyinvestedmarketexclusivities,financialwaivers,andregulatoryinrarediseasesintheircurrentportfolioandsupport)hasbeenverysuccessfu...
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