CANAKINUMAB

The PBAC recommended listing of canakinumab for the treatment of colchicine resistant Familial Mediterranean Fever (FMF) in adults and children aged 2 years and older. FMF is rare genetic condition that causes recurrent febrile attacks (flares) and severe systemic inflammation, which leads to deterioration in kidney function. The PBAC welcomed input from individuals, health care professionals and consumer organisations. The PBAC acknowledged the debilitating nature of the disease and the benefit of flare reduction on improving patients’ quality of life and life expectancy and the high unmet clinical need for treatment options for patients with FMF.

The PBAC noted the clinical evidence provided in the submission demonstrating that canakinumab reduces the frequency of febrile attacks and reduces systemic inflammation, which is expected to reduce kidney deterioration and failure. 

The clinical evidence presented in the submission did not allow confidence about the extent to which the benefits claimed in support to the proposed price would be realised in practice. However, the PBAC also recognised the difficulty in obtaining clinical data for this condition due to its rarity. 

The PBAC considered the value for money of canakinumab at the price requested by the sponsor was uncertain due to the limited clinical data, but that in the context of this rare and life-limiting disease, canakinumab would be considered acceptably cost-effective with a price reduction. The PBAC noted this recommendation is in line with other treatments for rare diseases funded on the PBS and takes into account clinical need, available evidence, nature of the benefits, and the size of the patient population. 

The PBAC considered that a risk-sharing arrangement should be put in place to reduce the financial risk of the cost of the drug to the PBS being higher than expected.