Applicability and added value of novel methods to improve drug development in rare diseases

Table 2 EPARs included in the evaluation

Cluster	Drug	Date opinion	Rare or ultra-rare*	Repurposed/ new drug?
Acute: single episodes
Antracycline extravasation	Savene	2006	Ultra-rare	New drug
Patent ductus arteriosus	Pedea	2009	Rare	Repurposed
Hepatic venooclusive disease	Defitelio	2013	Rare	New drug
Tuberculosis	Sirturo	2014	Rare	New drug
Acute: recurrent episodes
Cryopirine periodic syndromes	Ilaris	2009	Ultra-rare	New drug
Gram negative lung infection in cystic fibrosis	Cayston	2009	Rare	Repurposed
Narcolepsy	Xyrem	2007	Rare	New drug
Dravet syndrome	Diacomit	2009	Rare	New drug
Sickle cell disease	Sicklos	2007	Rare	New drug
Systemic sclerosis	Tracleer	2009	Rare	New drug
Chronic: stable/slow progression
Short bowel syndrome	Revestive	2012	Rare	New drug
Adrenal insufficiency	Plenadren	2011	Rare	Repurposed
Thrombocytemia	Xagrid	2009	Rare	New drug
Deficit of lipoprotein lipase	Glybera	2012	Ultra-rare	New drug
Chronic: progressive, one system/organ
Nocturnal Paroxysmal haemoglobinuria	Soliris	2009	Rare	New drug
Wilson’s disease	Wilzin	2006	Rare	New drug
Congenital errors of bile synthesis	Orphacol	2013	Ultra-rare	Repurposed
Gastrointestinal stromal tumours	Glivec	2009	Ultra-rare	Repurposed
Chronic: progressive, multiple systems/organs
Fabry disease	Fabrazyme	2008	Ultra-rare	New drug
Cystic fibrosis	Kalydeco	2013	Rare	New drug
Familial amyloid polyneuropathy	Vyndaqel	2011	Rare	New drug
Gaucher disease	Zavesca	2009	Rare	New drug
Chronic: staged condition
Renal carcinoma	Afinitor	2009	Rare	Repurposed
Pulmonary hypertension	Opsumit	2014	Rare	New drug
Indolent non-Hodgkin lymphoma	Litak	2006	Rare	New drug
Myelodysplastic syndrome	Revlimid	2008	Rare	New drug

EPAR European Public Assessment Report
* Rare if prevalence = or > 5/10.000 and > 0.1/10.000 inhabitants; Ultrarare if prevalence = or < 0.1/10.000 inhabitants

ISSN: 1750-1172