PBAC's latest decision on Eltrombopag: Recommended with restriction (2023). Considered for Severe aplastic anaemia (sAA) as first-line treatment in combination with immunosuppressive therapy (hATG and ciclosporin A), and as second-line treatment in patients with inadequate response or intolerance to IST.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
4
first 2011
Submissions
4
2011 → 2023
Eligible population
Adults and children with severe aplastic anaemia (first-line in combination with IST, and second-line with inadequate response or intolerance to prior IST); patients must be ineligible for haemopoietic stem cell transplant.
Therapy area
Haematology
Line of therapy
First-line | Second-line
Evidence base
RCT
Primary endpoint
Haematological response, transfusion independence
Comparator
hATG + ciclosporin A (first-line); best supportive care including infection treatment and transfusion support (second-line)
Economic model
BIA only
ICER note
No economic evaluation was presented by the submission. The ESC estimated cost per responder using drug costs, transfusion costs, and response rates rather than calculating a formal ICER.
Submission history
Mar 2011: Recommended with restriction · Authority Required
Jul 2013: Recommended with restriction · Authority Required
Nov 2014: Not recommended · Restricted
Nov 2023: Recommended with restriction · Authority Required