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

Table 2 Subject of included articles

	Included studies	Effectiveness	Cost-effectiveness	Budget impact
ALL	8	7	1	1
CAPS	7	7	0	0
CLL	3	2	1	0
Fabry (agalsidase α)	65	63	0	2
Fabry (agalsidase β)	66	64	1	1
MPSI	26	26	0	0
MPSII	22	21	1	1
MPSVI	24	24	0	0
PNH	23	21	1	2
Pompe	53	53	0	0
STS	41	38	3	1
Total	338	326	8	8

Six studies described both cost-effectiveness and budget impact; Six studies were included both for agalsidase α and agalsidase β; Six studies were included for multiple MPS diseases; In ten articles (one for CAPS; four for Fabry (α); one for Fabry (β); one for MPSI and three for Pompe) more than one study was described.

ISSN: 1750-1172