ATSS_ReachOut_April17_FINAL

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TSA News Advocating for access to mTOR inhibitor medicines Background on mTOR inhibitors The term mTOR inhibitor refers to a group of medicines. The generic (official) names for the two main medicines are Sirolimus and Everolimus. mTOR inhibitor medicines seem to do a similar job in the body as the TSC1 and TSC2 proteins, controlling how many cells grow and how large they grow. Scientists think this is the reason the medicines are effective in reducing the size of the tumours in some people with tuberous sclerosis complex (TSC). In Australia, both medicines are used to treat TSC and lymphangioleiomyomatosis (LAM, a lung condition associated with TSC). The manufacturers of Everolimus have sought approval from the Therapeutic Goods Administration (TGA) and listing on the Pharmaceutical Benefits Scheme (PBS) after funding large clinical trials into the use of this medicine in TSC. The manufacturers of Sirolimus have not pursued these approvals but there is still research on their use. People with TSC should discuss treatment options with their health professional, to decide if either of these mTOR medicines, or another medicine, is most appropriate for them. The rest of this article is about the approvals for Everolimus only, which is marketed in Australia with the brand name Afinitor. Recent review of Everolimus on the PBS In March this year, the Pharmaceutical Benefits Advisory Committee (PBAC) reviewed the use of Everolimus by Australian patients receiving the medicine through the PBS. This review involves comparing the actual use of the medicine to the estimates provided by the manufacturer of the medicine when it was approved for listing in 2014. Tuberous Sclerosis Australia made a submission to the PBAC to ensure their review of the medicine reflected the impact it was having on Australians with TSC. This submission included stories from six families living with TSC. These stories highlighted both the positive and negative aspects of the medicine. They also illustrated that there is significant unmet need in TSC, particularly for epilepsy and TSC Associated Neuropsychiatric Disorders (TAND). Thank you to the families who so willingly gave up their time and stories for this submission. We are also grateful for the support of the Centre for Community Driven Research (CCDR) and Ms Jo Watson, the consumer representative on the PBAC. Submission to TGA to list Everolimus for the treatment of epilepsy in TSC We expect the TGA to make a decision in November 2017 on whether Everolimus should be listed in Australia as a treatment for refractory epilepsy in TSC. This submission has been made after the results of the EXIST-3 clinical trial have been published, showing that for some people with TSC this medicine, used in combination with their existing anti-seizure medicines, led to a reduction in the number of seizures. You can help with making this medicine accessible for the treatment of epilepsy in TSC After the TGA listing, the next step is expansion of the existing PBS listing so that the medicine can be funded by the PBS for the treatment of seizures. This is where you can help. TSA will make a submission on behalf of our members that will help the PBAC to understand the impact of uncontrolled seizures and the benefits of having this additional treatment option. The PBAC will likely call for comments during May 2017. If you are able to assist with TSA’s submission or would like to know more about the process, send an email to clare@tsa.org.au. Australia leading the world in access to these medicines for TSC In working on these advocacy projects, we think it is important to appreciate and celebrate our achievements. The existing PBS listings for SEGA brain tumours and kidney angiomyolipomas (AMLs) and the funding at a hospital level for other uses of this medicine, is above what most people with TSC around the world have access to. This includes the USA where private insurance cover varies and the UK where our sister organisation, the Tuberous Sclerosis Association, continues to battle the NHS to improve access. TSA will continue to work alongside our researchers, health professionals, pharmaceutical companies and regulators to ensure Australians with TSC have access to the best healthcare for TSC throughout their lives. Thank you to our donors and fundraisers who ensure we have the ability to do this. We look forward to announcing these and other advocacy successes in the future. 6 Reach Out APRIL 2017 ISSUE 105
Events Melbourne TSC Information Day On Sunday 8 October 2017 TSA will hold an information day in Melbourne for people with TSC and their families. The event will include TSC expert speakers, a chance to meet others living with TSC and the presentation of the 2016 and 2017 Elizabeth Pinkerton Memorial Awards. Visit our website for more information and to register www.tsa.org.au/melbourne TSC online education events As announced in the previous issue of Reach Out, TSA is running a series of webinars this year which will bring our educational events directly to you at work or at home. In response to the topic suggestions from our 2016 survey, we’ve invited some of our Australian TSC experts to present in our upcoming webinars. We hope you’ll join us online for these unique opportunities to hear the latest information and ask your questions directly to these experts. We’re still confirming details for our third webinar. Visit our website for more information and to register www.tsa.org.au/webinar. Date Topic Presenter Tuesday 2 May 2017 at 7:30pm AEST Emerging treatments for epilepsy in TSC: Medicinal Cannabis and mTOR inhibitor medicines • Learn about the medicinal cannabis clinical trials being run in Australia and about the results of research mTOR inhibitors in epilepsy (Everolimus/Afinitor/Sirolimus/ Rapamycin). • Have the opportunity to ask your questions about access to these new treatment options for epilepsy. Dr John Lawson, Paediatric Neurologist from Sydney Children’s Hospital Friday 16 June 2017 at 12:30pm AEST Understanding the genetics of TSC and the importance of lifelong surveillance • Hear about the genetics of TSC, including what we know and what questions researchers are still exploring. • Learn about genetic testing and how to access genetic services in your area. • Take advantage of this unique opportunity to ask questions. Dr Chirag Patel, Clinical Geneticist, Queensland Clinical Genetics 7
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Page 3 and 4: TSA News Editorial Teresa Llewellyn
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Page 23 and 24: Fundraising Daphna’s 40 for FORTY

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