DeferiproneFERRIPROX
Recommended Rare diseaseRestrictedNot applicable line 💬 consumer voice
Treatment of transfusional iron overload associated with thalassaemia major in patients unable to take or in whom desferrioxamine therapy has proven ineffective.
1
Submissions
2017–17
On the record
—
ICER range
Cost-min
Cost basis
Decisions on record
- Meeting Nov 2017 Recommended Iron overload
- Meeting Nov 2014 Recommended Ferriprox® Orphan Australia Pty Ltd, no PSD
- Meeting Jul 2010 Recommended Iron overload in patients with thalassaemia no PSD
- Meeting Jul 2008 Recommended Thalassaemia major no PSD
Access path
- TGA registered · FERRIPROX
TGA label equal than the PBS population
- Nov 2017Recommended
vs deferiprone 500 mg tablets
- PBS listing · Restricted
RecommendedDeferredNot recommended
From the public summary
PBAC outcome
7.1 The PBAC recommended the listing of deferiprone 1000 mg as a Section 100 Highly Specialised Drugs, Private and Public Hospitals, Authority Required (STREAMLINED) for the treatment of transfusional iron overload associated with thalassaemia major.PSD · Nov 2017
7.2 The PBAC noted that the 1000 mg tablets would reduce the patient’s tablet burden.PSD · Nov 2017
Cost-effectiveness
Cost-minimisation analysis; submission was cost-neutral against the current 500 mg tablet listing.
The PBAC noted that the sponsor estimated that the proposed listing of the 1000 mg tablets would be cost-neutral against the current listing of the 500 mg tablets. PBAC · 2017
Cost-effectiveness accepted
Decision context
PopulationPatients with thalassaemia major who are unable to take or in whom desferrioxamine therapy has proven ineffective.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2017 | Recommended | deferiprone 500 mg tablets | — | RCT · Surrogate |
Consumer voice
No consumer comments were received for this item.
The PBAC noted that no consumer comments were received for this item. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both specify thalassaemia major patients unable to take or in whom desferrioxamine has proven ineffective; PBAC explicitly states transfusional iron overload, which is implicit in TGA label.