MacitentanOPSUMIT
Idiopathic pulmonary arterial hypertension (IPAH), PAH secondary to connective tissue disease (PAH-CTD), and PAH associated with congenital heart disease (PAH-CHD) in patients with WHO Functional Class III and IV severity.
Decision on record
- Meeting Mar 2014 Recommended Opsumit® Actelion Pharmaceuticals Australia Pty Ltd Pulmonary Arterial Hypertension (PAH) To request a Section 100 Highly Specialised Drugs (HSD) Program Authority Required listing for the treatment of idiopathic pulmonary arterial hypertension (PAH) and PAH associated with connective tissue disease
Access path
- TGA registered · OPSUMIT
TGA label narrower than the PBS population
- Mar 2014Recommended · restricted
vs bosentan
- PBS listing · Authority Required
Cost-effectiveness
Cost-minimisation analysis was conducted; no ICER calculated as macitentan was demonstrated to be non-inferior to bosentan with equivalent drug costs.
The PBAC considered that a cost-minimisation economic analysis was the appropriate approach based on the evidence presented for non-inferiority. PBAC · 2014
Decision context
PopulationPatients with WHO Functional Class III or IV idiopathic PAH (IPAH), PAH secondary to connective tissue disease (PAH-CTD), or PAH associated with congenital systemic-to-pulmonary shunt (including Eisenmenger's physiology) (PAH-CHD) who have not received prior PBS-subsidised treatment with a PAH agent, or who wish to recommence or switch to macitentan.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2014 | Recommended · restricted | bosentan | — | RCT · Morbidity/mortality |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| SERAPHIN | Ph 3 | 742 | Time to First Confirmed Morbidity or Mortality Event up to the End of Treatment (Kaplan-Me… | completed |
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to WHO Class III–IV only, excluding Class II patients eligible under the TGA label.