The first ever treatment specifically for Tuberous Sclerosis Complex (TSC) has recently been recommended for inclusion on the pharmaceutical benefits scheme (PBS), making it accessible to those living with the impacts of this devastating disease.
One of the difficult to manage symptoms of TSC is a type of brain tumour called a subpendymal giant cell astrocytoma (SEGA). Up to 15% of individuals with TSC will develop a SEGA. SEGAs mostly grow from late childhood and the chance for growth greatly decreases after the mid-20s. Even though they are not cancerous, SEGAs can be problematic because they may grow sufficiently large to block the flow of fluid within the brain, causing an increase in the pressure within the head and enlargement of the fluid-filled ventricles (a process known as hydrocephalus). This build up of pressure can result in symptoms such as vomiting, nausea, headache and changes in appetite, behavior and mood. If left untreated, hydrocephalus can lead to death. You can read more about SEGAs here.
Up until the approval of Everolimus by the Therapeutic Goods Administration (TGA) in early 2012, brain surgery was the only treatment option for large SEGAs. Listing of Everolimus on the PBS will make this much needed second option affordable for those patients and their families that require this treatment.
ATSS welcomes the decision by the PBAC. We look forward to the Health Minister authorising the listing in the coming months. We also thank the many families who contributed to this success by participating in clinical trials of this medicine, by making submissions to the PBAC and by supporting ATSS in our advocacy work on this issue.
You can see the outcomes of the PBAC meeting here.
Further information about some of the terms in this article
Afinitor is the brand name given to Everolimus, an mTOR inhibiting medicine. Afinitor has only been approved for the treatment of one particular type of TS tumour: SEGAs. Other uses of Afinitor that are currently being researched include for the treatment of kidney angiomyolipomas (AMLs), for the treatment of epilepsy and for improvements in cognition. The manufacturer of Afinitor, Novartis, may seek approval from the TGA for other uses of Afinitor in the future.
SEGAs are a brain tumour that affects approximately 15% of people with Tuberous Sclerosis. For more information on SEGAs, visit https://tsa.org.au/i-need-to-know-more/signs/brain/
The Pharmaceutical Benefits Scheme (PBS) is an Australian Government program that subsidises medicines to make them more affordable for Australians. For more information about the PBS, visit: https://www.pbs.gov.au/info/general/faq
The Pharmaceutical Benefits Advisory Committee (PBAC) is the group of people that make a recommendation on whether a medicine should be included on the PBS. Most are medical doctors or researchers.
About the relationship between ATSS and Novartis Pharmaceuticals
The ATSS has received funding from Novartis, the manufacturers of Afinitor, for certain ATSS projects including annual conferences and the ATSS website. ATSS committee and staff have full control over ATSS strategy and the delivery of all projects. The ATSS Committee makes its own decisions about what to advocate on, in the best interests of families with TSC.
To read ATSS policy about when and how ATSS works with Pharmaceutical companies, please visit: https://tsa.org.au/about-us/policies/
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