PBAC's latest decision on Agalsidase alfa: Noted (2009). Considered for treatment of Fabry disease, a rare X-linked genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A.
PBAC outcome
Noted
2009
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
1
first 2009
Submissions
1
2009 → 2009
Eligible population
patients with Fabry disease
Therapy area
Rare disease
Evidence base
RCT, Registry
Primary endpoint
OS, PFS, DFS, ORR, QoL, Surrogate
Key trials
Vedder et al. (2007), Schiffmann et al. (2001), Hajioff et al. (2003)
Comparator
agalsidase beta
ICER note
This is a literature review document for regulatory/policy comparison of two already-listed drugs under the Life Saving Drugs Program. No economic evaluation or ICER was conducted.