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Medical treatment for SEGAs considered for listing on the Australian pharmaceutical benefits scheme

Everolimus is an mTOR inhibitor medicine and may be an effective treatment for some people with Subpendymal Giant Cell Astrocytomas (SEGAs) caused by Tuberous Sclerosis (TSC). Everolimus is marketed by Novartis under the brand name Afinitor. In November 2012 the Pharmaceutical Benefits Advisory Committee (PBAC) considered listing Everolimus for PBS subsidy.

As a part of this process, many families living with TSC around Australia made personal submissions to the PBAC. The Australasian Tuberous Sclerosis Society also made a submission on behalf of all families living with TSC.

The outcome of the discussion by the PBAC is:

The PBAC deferred making a recommendation as a cost-effectiveness ratio could not be determined due to insufficient trial data from small patient numbers. The PBAC considered discussion with the sponsor about listing through a managed entry scheme to be a potential way forward, in the context of a high clinical need and uncertain clinical efficacy.

This means that the committee deferred their decision on whether to make Everolimus available to TSC-SEGA patients through the PBS. Whilst not ideal, this is still a positive sign that the PBAC acknowledge the value of Everolimus for some TSC-SEGA patients. The PBAC have requested further information on the use of Everolimus in TSC. Novartis and the PBAC are exploring a way to allow patient access to Everolimus at the same time as the company collects more information about its use, effectiveness and cost in Australian patients. We will update you as more information becomes available in mid-2013.

ATSS welcomes the PBAC’s decision to look at how this medicine can be made accessible and affordable for children and adults with SEGAs caused by TSC.

Any families who encounter difficulties in accessing this medicine or any others are advised to contact ATSS for further information. More information about SEGAs is available here.

Link to the PBAC outcome

 

 

 

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