This is a statement from Tuberous Sclerosis Australia released today (22 August 2018):


We are pleased to share news that Everolimus (Afinitor) for epilepsy has been given a positive recommendation by the pharmaceutical benefits advisory committee (PBAC). This is a recommendation to the Federal Health Minister to add this medicine to the pharmaceutical benefits scheme, which we expect to occur before the end of the year. Listing on the PBS will secure funding for this new treatment option for seizures for people with TSC.

Tuberous Sclerosis Australia’s submission to the pharmaceutical benefits advisory committee (PBAC) was informed by 60 members of the Australian TSC community who generously shared their stories. Thank you also to Dr Anna Williamson for her help with this submission.

The goal of our submission was to ensure the PBAC understood the significant impact that TSC and epilepsy have on Australians and the need to have this additional option for people who have seizures that cannot be controlled with other treatments. The feedback we have received for our submission has been very positive, specifically that “the PBAC noted the high clinical need for a treatment for this condition”. Although this decision took a long time to be confirmed, we thank the PBAC and the Novartis team for their persistence in reaching this recommendation.

Australia continues to be a world leader in affordable access to everolimus and other mTOR inhibitor medicines. We stand with our sister organisation, Tuberous Sclerosis Association in the United Kingdom, as they campaign against the recent decision of NHS England to not fund this medicine.

Everolimus (Afinitor) has been approved by the Therapeutic Goods Administration (TSA) as a safe and effective treatment for several signs and symptoms of TSC: subepyndymal giant cell astrocytomas (SEGAs), kidney angiomyolipomas (AMLs) and seizures. The implementation of this positive recommendation will see funding under the PBS for all of these signs and symptoms.

Funding is one of the final steps in a long process of making this medicine available to people with TSC. Thank you to all the researchers, clinical trial participants and funders who contributed. Our particular thanks to Dr John Lawson and the Australian families who participated in the Exist-3 trial at Sydney Children’s Hospital.

To read the statement and supporting documents in full please CLICK HERE.

And to support our #everolimusforepilepsy #nhsenglandwrongdecision #wewontgiveup campaign please CLICK HERE.


Maxine Smeaton, Interim CEO of the TSA, said: ‘We are delighted to hear the news that the Australia government listened to evidence from more than 60 patients before making its decision to fund everolimus for TSC-related refractory epilepsy. Huge congratulations to Clare Stuart and her team in Sydney. They have done a fantastic job of driving this outcome for their community. TSA Australia are also pro-actively involved in supporting our campaign here in England, which we very much appreciate.

‘NHS Scotland’s recent decision to fund everolimus for TSC-related epilepsy, and now Australia’s decision to do the same, proves that community involvement really can and does have an impact. By getting in touch with your MP and asking them to lobby NHS England on our behalf you really could make a difference. So please take action today. Thank you.’