Eltrombopag
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.
Decisions on record
- Meeting Nov 2023 Recommended Severe aplastic anaemia
- Meeting Nov 2014 Not recommended tablets, 25 mg, 50 mg Revolade® GlaxoSmithKline Australia Pty Ltd Change to listing (Minor submission) Decreased platelet count Amend continuation restriction to allow continuation of treatment with eltrombopag in patients whose disease is stable and responding to treatment with romiplostim or vice
- Meeting Mar 2011 Recommended Idiopathic thrombocytopenia purpura (ITP) – a bleeding disorder
- Meeting Nov 2010 Not recommended Idiopathic thrombocytopenia purpura – a bleeding disorder
Access path
- Mar 2011Recommended · restricted
vs romiplostim
- Jul 2013Not recommended
Comparator changed: romiplostim → No treatment (placebo) for enabling phase; mix of no…
- Nov 2014Not recommended
Clinical impact for patients switching for reasons other than treatment failure is unclear; cost-effectiveness and…
- ↻ resubmittedNov 2023Recommended
Comparator changed: romiplostim → hATG + ciclosporin A (first-line); best supportive care…
- PBS listing · Authority Required
From the public summary
The PBAC recommended the listing of eltrombopag for the treatment of severe aplastic anaemia (sAA), as first-line treatment in combination with horse anti- thymocyte globulin (hATG) and ciclosporin A (CsA), and as a second-line treatment in patients with an inadequate response to immunosuppressive therapy (IST). The PBAC considered there is a clinical need for effective treatment options for this rare condition.PSD · Nov 2023
The PBAC noted the consumer comments highlighted the significant impact of sAA on a patient’s quality of life and the clinical need for effective treatment options for this rare condition. The PBAC noted the range of patient relevant benefits of treatment with eltrombopag, including fewer blood transfusions, reduced complications (from other treatments and from transfusions) and fewer hospital visits.PSD · Nov 2023
The submission did not present an economic evaluation. The potential cost savings associated with achieving transfusion independence were outlined in the Pre-Sub- Committee Response (PSCR) (see below). The ESC estimated the cost per responder using the drug costs, transfusion costs and response rates.PSD · Nov 2023
The ESC noted the majority of use of eltrombopag will be in the first line treatment setting and the cost per patient is estimated to be $42,886 (Table 11).PSD · Nov 2023
In patients with an insufficient response to IST, eltrombopag provides a significant clinical benefit in terms of haematologic response and transfusion independence, with an acceptable safety profile.PSD · Nov 2023
Source: Constructed during the evaluation. The submission did not define a clinical claim, and the evaluation has used the “clinical conclusion” on p21 of the submission for this purpose. hATG = horse anti-thymocyte globulin; IST = immunosuppressive therapyPSD · Nov 2023
The PBAC noted and welcomed the input from health care professionals (11) and organisations (1) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with eltrombopag including the increased overall and complete response rates, reduced need for platelet transfusions, and resulting decrease in complications.PSD · Nov 2023
The National Paediatric Medicines Forum outlined the experience of Children’s Hospitals in using eltrombopag in paediatric patients and stated the availability of eltrombopag for sAA would improve equity of access and clinical outcomes for paediatric patients with sAA.PSD · Nov 2023
Cost-effectiveness
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.
The submission did not present an economic evaluation. PSD · 2023
Decision context
PopulationAdults 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.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2023 | Recommended | hATG + ciclosporin A (first-line); best supportive care including infection treatment and transfusion support (second-li | — | RCT · Haematological response, transfusion independence |
| Nov 2014 | Not recommended | romiplostim | — | Single-arm · Other |
| Jul 2013 | Not recommended | No treatment (placebo) for enabling phase; mix of no treatment and antiviral therapy with Australian dose-modification f | — | RCT · SVR |
| Mar 2011 | Recommended · restricted | romiplostim | — | RCT · other |
Consumer voice
Healthcare professionals and organisations reported that eltrombopag provides significant benefits for severe aplastic anaemia (sAA) including improved response rates, reduced transfusion needs, and improved quality of life, with manageable side effects and potential for improved equity of access, particularly for paediatric patients.
The comments described a range of benefits of treatment with eltrombopag including the increased overall and complete response rates, reduced need for platelet transfusions, and resulting decrease in complications. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients ineligible for haemopoietic stem cell transplant; TGA label lacks this transplant-eligibility criterion.