Table 2 List and description of identified variables for orphan drugs authorised in Spain from 2006 to 2021
Commercial name | P&R status | Therapeutic Area | Existence of therapeutic alternatives | Rarity of disease | Outcome | Safety | Type of population | TPR conclusion | Conditional approval |
|---|---|---|---|---|---|---|---|---|---|
Nexavar® | Reimbursed | Oncologic | Yes | Rare | Survival | No | Other | Positive | No |
Soliris® | Reimbursed | Oncologic | Yes | Rare | Other | No | Other | Positive | No |
Vpriv® | Reimbursed | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Tobi Podhaler® | Rejected | Other | Yes | Rare | Other | No | Other | Negative | No |
Votubia® | Reimbursed | Oncologic | No | Rare | Other | No | Other | Positive | No |
Vyndaqel® | Reimbursed | Other | No | Ultra-rare | Other | Yes | Other | Positive | No |
Xaluprine® | Under P&R decision process | Oncologic | Yes | Rare | Other | No | Other | Not published | No |
Bronchitol® | Rejected | Other | Yes | Rare | Other | No | Other | Negative | No |
Signifor® | Reimbursed | Other | Yes | Rare | Other | No | Other | Positive | No |
Kalydeco® | Reimbursed | Other | No | Rare | Other | No | Other | Positive | No |
Revestive® | Reimbursed | Other | No | Ultra-rare | Other | Yes | Other | Positive | No |
Dacogen® | Reimbursed | Oncologic | Yes | Rare | Survival | No | Other | Positive | No |
Adcetris® | Reimbursed | Oncologic | Yes | Ultra-rare | Survival | No | Other | Positive | Yes |
NexoBrid® | Rejected | Other | No | Rare | Other | Yes | Other | Negative | No |
Iclusig® | Reimbursed | Oncologic | No | Rare | Other | Yes | Other | Positive | No |
Imnovid® | Reimbursed | Oncologic | Yes | Rare | Survival | Yes | Other | Positive | No |
Procysbi® | Rejected | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Orphacol® | Reimbursed | Other | No | Ultra-rare | Survival | No | Other | Positive | No |
Opsumit® | Reimbursed | Other | Yes | Ultra-rare | Survival | No | Other | Positive | No |
Sirturo® | Rejected | Other | Yes | Rare | Other | No | Other | Positive | Yes |
Cometriq® | Rejected | Oncologic | Yes | Ultra-rare | Survival | No | Other | Positive | Yes |
Adempas® | Reimbursed | Other | No | Ultra-rare | Other | No | Other | Positive | No |
Granupas® | Rejected | Other | No | Rare | Other | No | Other | Negative | No |
Deltyba® | Reimbursed | Other | Yes | Rare | Other | No | Other | Positive | Yes |
Vimizim® | Rejected | Other | No | Ultra-rare | Other | Yes | Other | Negative | No |
Sylvant® | Reimbursed | Oncologic | No | Rare | Other | Yes | Other | Positive | No |
Gazyvaro® | Reimbursed | Oncologic | Yes | Rare | Survival | No | Other | Positive | No |
Translarna® | Rejected | Other | No | Rare | Other | No | Other | Negative | Yes |
Scenesse® | Rejected | Other | No | Ultra-rare | Other | No | Other | Negative | No |
Cerdelga® | Reimbursed | Other | Yes | Rare | Other | Yes | Other | Positive | No |
Holoclar® | Rejected | Other | No | Rare | Other | No | Other | Positive | Yes |
Strensiq® | Rejected | Other | No | Ultra-rare | Other | No | Paediatric | Positive | No |
Farydak® | Rejected | Oncologic | Yes | Rare | Survival | No | Other | Negative | No |
Kanuma® | Reimbursed | Other | No | Ultra-rare | Survival | Yes | Other | Positive | No |
Raxone® | Rejected | Other | No | Rare | Other | No | Other | Negative | No |
Cresemba® | Reimbursed | Other | Yes | Ultra-rare | Survival | No | Other | Positive | No |
Kyprolis® | Reimbursed | Oncologic | Yes | Rare | Survival | No | Other | Positive | No |
Blincyto® | Rejected | Oncologic | Yes | Rare | Survival | Yes | Other | Negative | No |
Ravicti® | Reimbursed | Other | Yes | Rare | Other | Yes | Other | Positive | No |
Wakix® | Reimbursed | Other | Yes | Rare | Other | Yes | Other | Negative | No |
Idelvion® | Reimbursed | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Alprolix® | Reimbursed | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Darzalex® | Reimbursed | Oncologic | Yes | Rare | Survival | No | Other | Positive | No |
Galafold® | Reimbursed | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Onivyde pegylated® | Reimbursed | Oncologic | Yes | Rare | Survival | No | Other | Positive | No |
Ninlaro® | Rejected | Oncologic | Yes | Rare | Survival | No | Other | Negative | Yes |
SomaKit TOC® | Reimbursed | Other | Yes | Rare | Other | No | Other | Positive | No |
Ocaliva® | Reimbursed | Other | No | Ultra-rare | Other | No | Other | Positive | Yes |
Cystadrops® | Rejected | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Ledaga® | Rejected | Oncologic | Yes | Rare | Other | No | Other | Positive | No |
Chenodeoxycholic acid Leadiant® | Reimbursed | Other | No | Ultra-rare | Other | No | Other | Positive | No |
Natpar® | Rejected | Other | No | Ultra-rare | Other | No | Other | Positive | Yes |
Qarziba® | Under P&R decision process | Oncologic | Yes | Rare | Other | Yes | Other | Not published | No |
Spinraza® | Reimbursed | Other | No | Ultra-rare | Survival | No | Other | Positive | No |
Brineura® | Under P&R decision process | Other | No | Ultra-rare | Other | Yes | Other | Not published | No |
Besponsa® | Reimbursed | Oncologic | No | Rare | Survival | No | Other | Positive | No |
Oxervate® | Rejected | Other | No | Rare | Survival | No | Other | Negative | No |
Xermelo® | Rejected | Other | Yes | Rare | Other | No | Other | Negative | No |
Rydapt® | Reimbursed | Oncologic | Yes | Ultra-rare | Survival | No | Other | Positive | No |
Lutathera® | Reimbursed | Other | Yes | Ultra-rare | Survival | No | Other | Positive | No |
Zejula® | Reimbursed | Oncologic | Yes | Ultra-rare | Survival | No | Other | Positive | No |
Jorveza® | Rejected | Other | Yes | Rare | Other | No | Other | Positive | No |
Prevymis® | Reimbursed | Other | Yes | Rare | Other | No | Other | Negative | No |
Crysvita® | Reimbursed | Other | No | Ultra-rare | Other | Yes | Paediatric | Positive | Yes |
Alofisel® | Reimbursed | Oncologic | No | Rare | Other | No | Other | Positive | No |
Lamzede® | Rejected | Other | No | Ultra-rare | Other | No | Other | negative | No |
Mylotarg® | Reimbursed | Oncologic | No | Rare | Survival | No | Other | Positive | No |
Amglidia® | Rejected | Other | Yes | Ultra-rare | Other | No | Paediatric | Negative | No |
Tegsedi® | Reimbursed | Other | Yes | Rare | Other | No | Other | Positive | No |
Verkazia® | Rejected | Other | Yes | Rare | Other | No | Paediatric | Negative | No |
Myalepta® | Rejected | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Kymriah® | Reimbursed | Oncologic | Yes | Rare | Survival | Yes | Other | Positive | No |
Yescarta® | Reimbursed | Oncologic | Yes | Rare | Survival | Yes | Other | Positive | No |
Vyxeos® | Rejected | Oncologic | Yes | Rare | Survival | No | Other | Positive | No |
Mepsevii® | Reimbursed | Other | No | Ultra-rare | Other | No | Other | Positive | No |
Onpattro® | Reimbursed | Other | Yes | Rare | Other | No | Other | Positive | No |
Cablivi® | Reimbursed | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Symkevi® | Reimbursed | Other | Yes | Rare | Other | No | Other | Positive | No |
Takhzyro® | Reimbursed | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Luxturna® | Reimbursed | Other | No | Rare | Other | Yes | Other | Positive | No |
Poteligeo® | Reimbursed | Oncologic | Yes | Rare | Survival | No | Other | Positive | No |
Namuscla® | Rejected | Other | No | Rare | Other | No | Other | Negative | No |
Palynziq® | Rejected | Other | No | Rare | Other | Yes | Other | Negative | No |
Waylivra® | Under P&R decision process | Other | No | Ultra-rare | Other | Yes | Other | Not published | No |
Trecondi® | Under P&R decision process | Oncologic | Yes | Rare | Other | No | Other | Not published | No |
Epidyolex® | Reimbursed | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Xospata® | Rejected | Oncologic | No | Rare | Survival | No | Other | Negative | No |
Isturisa® | Under P&R decision process | Other | Yes | Ultra-rare | Other | No | Other | Not published | No |
Polivy® | Reimbursed | Oncologic | Yes | Rare | Other | Yes | Other | Positive | Yes |
Givlaari® | Reimbursed | Other | Yes | Ultra-rare | Other | No | Other | Positive | No |
Trepulmix® | Reimbursed | Other | Yes | Rare | Other | No | Other | Positive | No |
Zolgensma® | Reimbursed | Other | Yes | Ultra-rare | Other | Yes | Paediatric | Positive | Yes |
Reblozyl® | Under P&R decision process | Other | Yes | Rare | Other | No | Other | Not published | No |
Daurismo® | Under P&R decision process | Oncologic | Yes | Rare | Survival | No | Other | Not published | No |
Hepcludex® | Under P&R decision process | Other | No | Rare | Other | No | Other | Not published | No |
Kaftrio® | Reimbursed | Other | Yes | Rare | Other | Yes | Other | Positive | No |
Blenrep® | Under P&R decision process | Oncologic | Yes | Rare | Survival | No | Other | Not published | Yes |
Idefirix® | Under P&R decision process | Oncologic | No | Rare | Other | Yes | Other | Not published | Yes |
Adakveo® | Under P&R decision process | Other | No | Rare | Other | Yes | Other | Not published | Yes |
Oxlumo® | Under P&R decision process | Other | No | Rare | Other | Yes | Other | Not published | No |
Tecartus® | Under P&R decision process | Oncologic | Yes | Rare | Survival | No | Other | Not published | Yes |
Libmeldy® | Under P&R decision process | Other | No | Rare | Survival | No | Paediatric | Not published | No |
Fintepla® | Under P&R decision process | Other | Yes | Rare | Other | Yes | Other | Not published | No |
Inrebic® | Under P&R decision process | Oncologic | Yes | Rare | Survival | Yes | Other | Not published | No |
Pemazyre® | Under P&R decision process | Oncologic | No | Rare | Survival | No | Other | Not published | Yes |
Evrysdi® | Under P&R decision process | Other | Yes | Rare | Survival | No | Other | Not published | No |
Koselugo® | Under P&R decision process | Oncologic | No | Rare | Survival | Yes | Paediatric | Not published | Yes |
Enspryng® | Under P&R decision process | Oncologic | Yes | Rare | Other | No | Other | Not published | No |
Bylvay® | Under P&R decision process | Other | No | Ultra-rare | Other | No | Other | Not published | No |
Minjuvi® | Under P&R decision process | Oncologic | Yes | Rare | Survival | No | Other | Not published | Yes |
Aspaveli® | Under P&R decision process | Oncologic | No | Ultra-rare | Other | Yes | Other | Not published | No |