PBAC's latest decision on Betaine: Recommended with restriction (2014). Considered for Adjunct treatment for homocystinuria, a rare metabolic condition caused by inherited disorders of amino acid metabolism characterised by excessive homocysteine levels.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
1
first 2014
Submissions
1
2014 → 2014
Eligible population
Patients with homocystinuria, primarily those non-responsive to pyridoxine, managed in consultation with a metabolic physician.
Therapy area
Metabolic
Line of therapy
Not applicable
Evidence base
Single-arm
Primary endpoint
Surrogate
Pivotal trial size
35 patients
Key trials
Singh et al 2004, Walter et al 1998, Wilcken et al 1983, Wilcken et al 1985, Wilcken et al 1997, Yap et al 2001, Yap et al 2003
Comparator
standard care without betaine
Economic model
CEA
Budget impact
$10M
Eligible patients/year
10,000
ICER note
ICER not calculated in traditional health economics format; submission presented cost per unit reduction in homocysteine (<$2,000 per 1 µmol/L reduction).
Risk sharing
Risk-sharing arrangement in place — Risk share arrangement based on a cap on the number of patients, using the patient numbers presented in the submission, to manage uncertainty around utilisation and total financial cost.