PBAC's latest decision on Deutetrabenazine: Not recommended (2021). Considered for Treatment of chorea associated with Huntington's disease in patients who have failed prior tetrabenazine treatment due to intolerance or inadequate response.
PBAC outcome
Not recommended
Authority Required
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
1
first 2021
Submissions
1
2021 → 2021
Eligible population
Adults with chorea associated with Huntington's disease who have failed prior tetrabenazine treatment due to intolerance or inadequate response
Therapy area
Neurology
Line of therapy
Second-line or Third-line (as requested); First-line (as clinically appropriate per ESC/PBAC)
Evidence base
RCT
Primary endpoint
ORR
Pivotal trial size
90 patients
Key trials
FIRST-HD, ARC-HD-ROLLOVER, ARC-HD-SWITCH
Comparator
placebo (as submitted); tetrabenazine (as advised by ESC/PBAC)
Economic model
CEA
ICER note
No ICER was presented or calculated. The submission was based on a cost-effectiveness analysis versus placebo, but PBAC considered the comparator inappropriate and the clinical positioning untenable, precluding economic evaluation.
Why PBAC said no
Reasons cited in the latest PSD: Inappropriate clinical positioning (requested as second/third-line when evidence supports first-line use); clinical and economic comparisons not informative for decision-making; no evidence in the proposed subsequent-line treatment setting; mismatch between requested PBS restriction and available clinical data; risk of high leakage to first-line use; pricing not viable for first-line listing as an alternative to established tetrabenazine; proposed PBS restriction criteria highly subjective with no well-accepted definitions