Systematic review of available evidence on 11 high-priced inpatient orphan drugs

Table 1 Orphan drugs and indications on the Dutch policy rule

Indication	Treatment	Prevalence (100,000 live births)*	Estimated annual costs / patient in € **	Dutch budget impact in € **	Year costs were assessed	Exceptional circumstances authorization
Acute Lymphoid Leukemia (ALL) in children	Clofarabine (Evoltra)	8.1	54 K	0.7 M	2007	Yes
Cryopyrin Associated Periodic Syndromes (CAPS)	Canakinumab (Ilaris)	0.10 ***	66 K	2.6 M	2010	Yes
Chronic Lymphoid Leukemia (CLL) in patients refractory to fludarabine and alemzumab	Ofatumumab (Arzerra)	30	39 K	1.0 M	2011	No
Fabry disease	Agalsidase α (Replagal)	30	199 K	3.2 M	2009	Yes
Fabry disease	Agalsidase β (Fabrazyme)	30	194 K	5.7 M	2009	No
Mucopolysaccharidosis I (MPSI)	Laronidase (Aldurazyme)	1.3	300 K	7.5 M	2003	Yes
Mucopolysaccharidosis II (MPSII)	Idursulfase (Elaprase)	0.6	600 K	9.6 M	2007	Yes
Mucopolysaccharidosis VI (MPSVI)	Galsulfase (Naglazyme)	0.16	600 K	4.2 M	2007	Yes
Paroxysmal Nocturnal Hemoglobinuria (PNH)	Eculizumab (Soliris)	0.8	358 K	10.6 M *****	2008-2010	No
Pompe disease	Alglucosidase alfa (Myozyme)	1.5	38 K; 382 K ****	31.0 M	2007	No
Soft Tissue Sarcoma (STS)	Trabectedin (Yondelis)	23.7	22 K	5.3 M	2010	Yes

* European estimates obtained from Orphanet [8]; ** Obtained from CFH (accessible through http://www.cvz.nl, in Dutch); *** U.S. estimate [9]; **** Costs for infantile patients are estimated at €38 K, and costs for adults are estimated at €382 K ***** Average estimated yearly budget impact for the period 2008-2010.

ISSN: 1750-1172