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Access to mTOR Inhibitors for Epilepsy

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Thank you to all the members of the TSC community who made a contribution to our draft submission to the pharmaceutical benefits advisory committee (PBAC). The submission to include Afinitor (Everolimus, an mTOR inhibitor medicine) on the pharmaceutical benefits scheme (PBS) is being considered at the December 2017 meeting of the committee. We will share the outcomes of this decision as soon as they are available via our website, email newsletter and Facebook.

TSA’s submission on behalf of the Australian TSC community included the perspectives of over 60 people who contributed to our survey and interviews. We are not publishing the full submission to protect the privacy of these people. Below is the first part of the submission, before personal information is included.

Ms Jo Watson
Consumer Representative and Co-Chair
Pharmaceutical Benefits Advisory Committee

13 November 2017


Dear Jo,

Re: Listing of Everolimus for the treatment of patients with refractory seizures associated with tuberous sclerosis complex in combination with other anti-epileptic medications

Thank you for sharing this submission with the other members of the Pharmaceutical Benefits Advisory Committee (PBAC). I write this submission on behalf of our membership of hundreds of families living with tuberous sclerosis complex (TSC). Tuberous Sclerosis Australia (TSA), established in 1981, is the only patient organisation for TSC in Australia and we are a founding member of Tuberous Sclerosis Complex International.

TSA encourages PBAC to make a positive recommendation for this submission for the following reasons:

  1. TSC has a major impact on quality of life and refractory epilepsy is one of the most disabling symptoms of TSC.
  2. Even modest reductions in seizure number or severity can lead to significant improvements in quality of life for people with TSC and their families.
  3. When patients and their specialists weigh up the risk and benefits of this treatment, they will also consider the ‘positive side affects’ that many people with TSC experience.
  4. Everolimus offers patients and their neurologists an additional, and much needed, tool to tackle epilepsy in TSC.

This submission describes these four reasons in more detail.

If you determine that a consumer hearing would be valuable for this important decision we will gladly work with you to invite the appropriate members of the Australian TSC community to participate.

Declaration of interest

A number of members of our organisation are prescribed Everolimus (under brand name Afinitor) for the management of other TSC symptoms including kidney angiomyolipomas (AML) and brain subpendymal giant cell astrocytomas (SEGA).

TSA has received funding from Novartis, the manufacturers of Everolimus, for specific TSA projects including education events and information resources. The percentage of TSA revenue has varied from between 0% (2016/17) and 30% ($45,000 in 2014/15). This specific application by Novartis to the PBAC has been discussed with TSA staff. Discussions have occurred between myself (Clare Stuart) and the medical manager Eleanor Sum and the health economist Trish Palmer at Novartis.

The TSA management committee and staff have full control over TSA’s strategy and the delivery of all projects. The TSA management committee is elected by the members of TSA and makes independent decisions on advocacy positions and priorities in the best interests of families with TSC. To read TSA’s policy about when and how TSA works with pharmaceutical companies, please visit  www.tsa.org.au/about-us/policies/

Our methods

This submission is informed by a survey we conducted in 2017. This survey included 60 people and families living with TSC as well as interviews with four parents caring for children with TSC. The survey participants included three adults living independently with TSC, 42 families caring for a child (<18 years old) and 25 families caring for an adult child with TSC. This sample is not representative, but it helps us to describe the experiences of a group of people with TSC and epilepsy who may benefit from the modified listing of Everolimus.

As a part of this survey, we asked how respondents obtained information on Everolimus. Three main sources were reported: their medical specialists (53%), Tuberous Sclerosis Australia (40%) and the internet or social media (29%). Thirteen percent reported not knowing anything about Everolimus.

About tuberous sclerosis complex (TSC)

TSC is a rare genetic condition in which benign tumours grow in organs of the body variously disrupting organ functions. The organs most commonly affected are the brain, skin, heart, lungs, kidneys, liver, pancreas and eyes. TSC can cause epilepsy, developmental delay and autism. The impact on the life of the person with TSC and their family can be profound, with some people requiring 24 hour, life-long care for this incurable condition. Treatments, including surgery, to remove or limit the growth of the tumours, are used to try and restrict tumour development, slow the progress of the disease and limit the resulting damage to organs.

(submission continues)

TSA will continue to work alongside our researchers, health professionals, medicines companies and regulators to ensure Australians with TSC have access to the best healthcare throughout their lives. Thank you to our donors and fundraisers who ensure we have the ability to do this.

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