Cerliponase AlfaBRINEURA
Treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare neurodegenerative genetic condition caused by deficiency of the enzyme TPP1.
Decision on record
- Meeting Jul 2018 Not recommended Neuronal ceroid lipofuscinosis type 2 (CLN2) disease (also known as tripeptidyl peptidase 1 deficiency)
Access path
- TGA registered · BRINEURA
TGA label narrower than the PBS population
- Jul 2018Not recommended
vs standard care
From the public summary
7.1 The PBAC did not recommend the listing of cerliponase alfa for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease on the basis of unacceptable high cost-effectiveness at the proposed price and uncertainty that the treatment effect observed in the trial would equate to a survival benefit. The PBAC considered that the primary outcome of motor language (ML) score alone was not adequate to derive an informative model of survival benefit.PSD · Jul 2018
7.2 The PBAC acknowledged the many consumer comments received from health professionals, families and carers on behalf of patients with CLN2 disease and the correspondence from Batten Disease Support and Research Association (BDSRA) Australia and BDSRA North America. The PBAC acknowledged the high and urgent clinical need for treatments for CLN2 disease, particularly given the severity of this rare condition.PSD · Jul 2018
6.30 The submission presented a stepped economic evaluation, with a modelled cost effectiveness (life years gained) and cost utility analysis. A summary of the economic evaluation is presented in Table 7 and Figure 3. 14PSD · Jul 2018
Outcomes LYG and QALY gained Methods used to generate Markov model results A total of 8 health states: 7 individual health states for each ML score (6 to 0) and death as Health states an absorbing health state.PSD · Jul 2018
The comments described the rapid progression, debilitating symptoms and significant burden of CLN2 disease on the patient and family and emphasised that there are currently no other treatment options for these patients. The comments also described the benefits of treatment in children currently receiving cerliponase alfa including reduction of seizures and maintenance of mobility and speech functions.PSD · Jul 2018
6.6 Batten Disease Support and Research Association (BDSRA) Australia and BDSRA North America expressed their strong support for the subsidisation of cerliponase alfa and indicated the importance of access to this treatment for families of patients diagnosed with CLN2 disease regardless of whether the treatment is curative.PSD · Jul 2018
Cost-effectiveness
ICER values are redacted (marked as '''''' in the document).
Decision context
PopulationPatients with confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 disease, initially aged 3-15 years with early to moderate symptoms (ML score 3-6) and stable seizures; continuing treatment for patients who have not had an unreversed motor-language score of 0 for 6 months.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2018 | Not recommended | standard care | — | Single-arm · ML score response |
Consumer voice
Consumers reported that CLN2 disease causes rapid progression and debilitating symptoms with significant burden on patients and families, with no current alternative treatments. They described benefits of cerliponase alfa in reducing seizures and maintaining mobility and speech, and emphasized the importance of subsidized access regardless of curative potential.
The comments described the rapid progression, debilitating symptoms and significant burden of CLN2 disease on the patient and family and emphasised that there are currently no other treatment options for these patients. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to ages 3-15 years with ML score 3-6 and stable seizures; TGA label has no age or severity limits.