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

Badia, Xavier; Vico, Tania; Shepherd, John; Gil, Alicia; Poveda-Andrés, José Luis; Hernández, César

doi:10.1186/s13023-020-01507-4

Orphanet Journal of Rare Diseases

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
Brand name	P&R status in Spain	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

P&R Pricing and reimbursement; TPR Therapeutic positioning report. *Obligation or not to conduct a post-authorisation safety study (PASS)

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ISSN: 1750-1172

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