AvatrombopagDoptelet
Treatment of severe thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenia purpura (ITP) who have had an inadequate response or are intolerant to corticosteroids and intravenous immunoglobulin (IVIg).
Decision on record
- Meeting Nov 2022 Recommended Chronic immune (idiopathic) thrombocytopenia purpura
Access path
- TGA registered · Doptelet
TGA label narrower than the PBS population
- Nov 2022Recommended · restricted
vs eltrombopag
- PBS listing · Authority Required
From the public summary
The PBAC recommended the Section 100 (Highly Specialised Drugs Program – Public and Private Hospital), Authority Required listing of avatrombopag for the treatment of severe thrombocytopenia in patients with chronic idiopathic thrombocytopenic purpura (ITP). The PBAC’s recommendation for listing was based on, among other matters, its assessment that the cost-effectiveness of avatrombopag would be acceptable if it were cost minimised to eltrombopag.PSD · Nov 2022
The submission presented a CMA for the economic analysis. The approach and assumptions are shown in Table 10. The prices were based on the published AEMP 20PSD · Nov 2022
Table 10: Key components and assumptions of the cost-minimisation approach Component Claim or assumption Therapeutic claim: effectiveness Based on evidence presented, effectiveness is assumed to be non-inferior Therapeutic claim: safety Based on evidence presented, safety is assumed to be non-inferior Evidence base Indirect comparison of proposed medicine and main comparator Equi-effective doses Avatrombopag 22.34 mg /day = eltrombopag 55.2 mg dose/day …PSD · Nov 2022
The submission described avatrombopag as non-inferior in terms of effectiveness and safety compared to eltrombopag. The evaluation considered the point estimates for some important efficacy outcomes are meaningfully different for eltrombopag and avatrombopag, and although these differences were not statistically significant, the very small sample sizes of the clinical trials used resulted in such wide confidence intervals around the point estimates that …PSD · Nov 2022
• Romiplostim, eltrombopag and avatrombopag are all are effective versus placebo in ITP, but their relative efficacy has not been evaluated in head-to-head RCTs.PSD · Nov 2022
The PBAC noted and welcomed the input from individuals (8) and healthcare professionals (1) via the Consumer Comments facility on the PBS website. The comments from individuals described the impact of living with ITP and treatment regimens, including risk of bleeding, side effects and administration challenges of currently available TPO-RA therapies and the impact of splenectomy.PSD · Nov 2022
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated by design.
The PBAC advised that the cost minimisation should be performed against the least costly comparator, eltrombopag, so that it is not more costly than any of the alternative therapies. PBAC · 2022
Decision context
PopulationAdult patients (aged 18 years or older) with severe chronic ITP who have had an inadequate response to or are intolerant to both corticosteroid therapy and immunoglobulin therapy.
Risk sharingSpecial Pricing Arrangement (SPA) with confidential rebate on published AEMP to realise the cost-minimisation claim once confidential eltrombopag price in chronic ITP is incorporated.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2022 | Recommended · restricted | eltrombopag | — | RCT · Durable platelet response |
Consumer voice
Eight individuals and one healthcare professional provided input describing the burden of living with ITP and managing current TPO-RA treatments, including bleeding risk, side effects, and administration challenges. The health professional advocated for early access to TPO-RAs and highlighted avatrombopag's tolerability advantages over romiplostim and eltrombopag.
The comments from individuals described the impact of living with ITP and treatment regimens, including risk of bleeding, side effects and administration challenges of currently available TPO-RA therapies and the impact of splenectomy. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to severe ITP with inadequate response/intolerance to both corticosteroids and IVIg; TGA label only requires insufficient response to previous treatment.