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Table 1 Identified variables for each orphan drug authorised in Spain and approved by the European Commission between 2003 & 2019

From: Impact of the therapeutic positioning report in the P&R process in Spain: analysis of orphan drugs approved by the European Commission and reimbursed in Spain from 2003 to 2019

Brand name P&R status in Spain Clinical variables Regulatory variables
Therapeutic Area Existence of therapeutic alternatives Rarity of disease Outcomes classification Efficacy profile Safety profile* Type of population TPR conclusion Conditional approval
Adcetris® Approved Oncologic Yes Ultra-rare Hard Similar No Adults Positive Yes
Adempas® Approved Other No Ultra-rare Medium Superior No Adults Positive No
Alofisel® Approved Oncologic Yes Rare Soft Similar No Adults Positive No
Alprolix® Rejected Other Yes Ultra-rare Medium Similar No All ages Positive No
Besponsa® Approved Oncologic No Rare Hard Superior No Adults Positive No
Blincyto® Rejected Oncologic Yes Rare Hard Similar Yes All ages Positive No
Brineura® Under P&R decision process Other No Ultra-rare Medium Similar Yes All ages Not published No
Bronchitol® Rejected Other Yes Rare Medium Similar No All ages Not published No
Cablivi® Under P&R decision process Other Yes Ultra-rare Soft Superior No Adults Not published No
Carbaglu® Approved Other Yes Ultra-rare Soft Superior Yes Adults Not published No
Cerdelga® Approved Other Yes Ultra-rare Soft Superior Yes Adults Positive No
Chenodeoxycholic acid Leadiant® Under P&R decision process Other No Ultra-rare Soft Similar No All ages Not published No
Cometriq® Rejected Oncologic Yes Ultra-rare Hard Similar No Adults Positive Yes
Cresemba® Approved Other Yes Ultra-rare Hard Similar No Adults Positive No
Crysvita® Under P&R decision process Other No Ultra-rare Medium Similar Yes Paediatric Not published Yes
Cystadrops® Rejected Other Yes Ultra-rare Soft Superior No All ages Positive No
Dacogen® Approved Oncologic Yes Rare Hard Superior No Adults Positive No
Darzalex® Approved Oncologic Yes Rare Hard Superior No Adults Positive No
Deltyba® Approved Other Yes Rare Soft Similar No Adults Positive Yes
Epidyolex® Under P&R decision process Other Yes Ultra-rare Hard Similar No All ages Not published No
Esbriet® Approved Oncologic No Rare Medium Similar No Adults Positive No
Farydak® Rejected Oncologic Yes Rare Hard Similar No Adults Negative No
Firazyr® Approved Other Yes Rare Medium Similar No All ages Not published No
Firdapse® Rejected Other No Ultra-rare Hard Superior No Adults Not published No
Galafold® Approved Other Yes Ultra-rare Soft Similar No Adults Positive No
Gazyvaro® Approved Oncologic Yes Rare Hard Superior No Adults Positive No
Granupas® Rejected Other No Rare Medium Similar No All ages Not published No
Holoclar® Rejected Other No Rare Hard Similar No All ages Negative Yes
Iclusig® Approved Oncologic No Rare Soft Similar Yes Adults Not published No
Idelvion® Rejected Other Yes Ultra-rare Hard Similar No All ages Positive No
Imbruvica® Approved Oncologic Yes Rare Hard Similar No Adults Positive No
Imnovid® Approved Oncologic Yes Rare Hard Superior Yes Adults Positive No
Jorveza® Rejected Other Yes Rare Soft Superior No Adults Negative No
Kalydeco® Approved Other No Rare Medium Similar No All ages Positive No
Kanuma® Approved Other No Ultra-rare Hard Superior Yes All ages Positive No
Kuvan® Approved Other Yes Rare Soft Superior No All ages Not published No
Kymriah® Approved Oncologic Yes Rare Hard Similar Yes All ages Positive No
Kyprolis® Approved Oncologic Yes Rare Hard Superior No Adults Positive No
Lamzede® Under P&R decision process Other No Ultra-rare Soft Superior No All ages Not published No
Ledaga® Rejected Oncologic Yes Rare Hard Superior No Adults Negative No
Lutathera® Approved Other Yes Ultra-rare Hard Superior No Adults Positive No
Luxturna® Under P&R decision process Other No Rare Medium Superior Yes All ages Not published No
Mepsevii® Under P&R decision process Other No Ultra-rare Soft Similar No All ages Not published No
Mozobil® Approved Oncologic Yes Rare Medium Superior No All ages Not published No
Myalepta® Rejected Other Yes Ultra-rare Soft Similar No All ages Not published No
Mylotarg® Approved Oncologic No Rare Hard Superior No All ages Positive No
Namuscla® Under P&R decision process Other No Rare Hard Superior No Adults Not published No
Natpar® Rejected Other Yes Ultra-rare Soft Superior No Adults Negative Yes
Nexavar® Approved Oncologic Yes Rare Hard Superior No All ages Not published No
NexoBrid® Rejected Other No Rare Medium Superior Yes Adults Not published No
Ninlaro® Rejected Oncologic Yes Rare Hard Superior No Adults Negative Yes
Ocaliva® Approved Other Yes Ultra-rare Soft Superior No Adults Positive Yes
Ofev® Approved Oncologic No Rare Medium Similar No Adults Positive No
Onivyde® Approved Oncologic Yes Rare Hard Superior No Adults Positive No
Onpattro® Under P&R decision process Other Yes Rare Hard Superior No Adults Not published No
Opsumit® Approved Other Yes Ultra-rare Hard Superior No Adults Positive No
Orphacol® Approved Other No Ultra-rare Hard Similar No All ages Not published No
Oxervate® Rejected Other No Rare Hard Superior No Adults Negative No
Palynziq® Under P&R decision process Other Yes Rare Soft Superior Yes All ages Not published No
Plenadren® Rejected Other Yes Rare Soft Similar No Adults Not published No
Poteligeo® Under P&R decision process Oncologic Yes Rare Hard Superior No Adults Not published No
Prevymis® Rejected Other Yes Rare Medium Superior No Adults Negative No
Procysbi® Rejected Other Yes Ultra-rare Soft Similar No All ages Negative No
Qarziba® Under P&R decision process Oncologic Yes Rare Soft Similar Yes Paediatric Not published No
Ravicti® Approved Other Yes Rare Soft Similar Yes All ages Not published No
Raxone® Under P&R decision process Other No Rare Medium Similar No All ages Not published No
Revestive® Approved Other No Ultra-rare Medium Superior Yes Adults Not published No
Rydapt® Approved Oncologic Yes Ultra-rare Hard Superior No Adults Positive No
Scenesse® Rejected Other No Ultra-rare Soft Superior No Adults Not published No
Signifor® Approved Other Yes Rare Soft Superior No Adults Positive No
Sirturo® Rejected Other Yes Rare Soft Superior No Adults Positive Yes
Soliris® Approved Oncologic Yes Rare Medium Superior No All ages Positive No
SomaKit TOC® Approved Other Yes Rare Soft Similar No Adults Positive No
Spinraza® Approved Other No Ultra-rare Hard Superior No All ages Positive No
Strensiq® Rejected Other No Ultra-rare Soft Similar No Paediatric Positive No
Sylvant® Approved Oncologic No Rare Medium Superior Yes Adults Positive No
Symkevi® Approved Other Yes Rare Medium Superior No All ages Positive No
Takhzyro® Under P&R decision process Other Yes Ultra-rare Medium Superior No All ages Not published No
Tegsedi® Under P&R decision process Other Yes Rare Medium Superior No Adults Not published No
Tepadina® Approved Oncologic Yes Ultra-rare Hard Similar No All ages Not published No
Tobi Podhaler® Rejected Other Yes Rare Medium Superior No All ages Not published No
Translarna® Rejected Other No Rare Medium Similar No All ages Negative Yes
Verkazia® Under P&R decision process Other Yes Rare Soft Superior No Paediatric Not published No
Vimizim® Rejected Other No Ultra-rare Medium Superior Yes All ages Negative No
Votubia® Approved Oncologic No Rare Medium Superior No Adults Not published No
Vpriv® Approved Other Yes Ultra-rare Soft Similar No All ages Not published No
Vyndaqel® Approved Other No Ultra-rare Medium Similar Yes Adults Positive No
Vyxeos® Rejected Oncologic Yes Rare Hard Superior No Adults Not published No
Wakix® Rejected Other Yes Rare Hard Similar Yes Adults Positive No
Xaluprine® Under P&R decision process Oncologic Yes Rare Soft Similar No All ages Not published No
Xermelo® Rejected Other Yes Rare Soft Superior No Adults Not published No
Xospata® Under P&R decision process Oncologic No Rare Hard Superior No Adults Not published No
Yescarta® Approved Oncologic Yes Rare Hard Similar Yes Adults Positive No
Zejula® Approved Oncologic Yes Ultra-rare Hard Superior No Adults Positive No
  1. P&R Pricing and reimbursement; TPR Therapeutic positioning report. *Obligation or not to conduct a post-authorisation safety study (PASS)