BetaineCystadane
Adjunct treatment for homocystinuria, a rare metabolic condition caused by inherited disorders of amino acid metabolism characterised by excessive homocysteine levels.
Decision on record
- Meeting Mar 2014 Recommended Cystadane® Emerge Health Pty Ltd Homocystinuria To request an Authority Required General Schedule listing for the treatment of patients with homocystinuria.
Access path
- TGA registered · Cystadane
TGA label narrower than the PBS population
- Mar 2014Recommended · restricted
vs standard care without betaine
- PBS listing · Authority Required
From the public summary
The PBAC considered that it was appropriate that a risk share arrangement is implemented to manage the uncertainty around the utilisation and total financial cost. This risk share arrangement should include a cap on patient numbers, using the estimates provided in the submission.PSD · Mar 2014
The PBAC noted that its recommendation was in the context of a small patient population with a high unmet clinical need, and a modest overall likely financial impact to the PBS.PSD · Mar 2014
Cost-effectiveness
ICER not calculated in traditional health economics format; submission presented cost per unit reduction in homocysteine (<$2,000 per 1 µmol/L reduction).
The submission presented a cost effectiveness analysis based on the annual cost of betaine and the pooled reduction in homocysteine levels from the included studies. PSD · 2014
Decision context
PopulationPatients with homocystinuria, primarily those non-responsive to pyridoxine, managed in consultation with a metabolic physician.
Risk sharingRisk 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.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2014 | Recommended · restricted | standard care without betaine | — | Single-arm · Surrogate |
Consumer voice
The Australasian Society for Inborn Errors of Metabolism (ASIEM) provided input highlighting the effectiveness of betaine in treating homocysteinurias and the difficulty patients face in obtaining betaine at a reasonable price without PBS listing.
ASIEM's input highlighted the effectiveness of betaine in treating homocysteinurias, and the difficulty faced by patients in obtaining betaine at a reasonable price in the absence of PBS listing. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients non-responsive to pyridoxine and requiring metabolic physician consultation; TGA label has no such restrictions.