Three years of fundraising has led to Sydney Children’s Hospital being funded by ATSS to take part in the first randomized controlled trial for the topical use of Rapamycin for angiofibromas, the skin condition that affects up to 90% of people with Tuberous Sclerosis Complex. Thank you to the hundreds of people that organized fundraising activities and donated. Over $200,000 was raised; however, the average donation was under $100.
This is an important study for several reasons. Our goals are to have topical Rapamycin to be approved by the Therapeutic Goods Administration (TGA) and subsequently included in the pharmaceutical benefits scheme (PBS); this is the way that we will ensure this treatment is made accessible and affordable for all TSC affected people that require the cream. The Treatment Trial will provide the clinical trial evidence required for this approval process. It will also help to confirm the strength of the cream and the way it should be used and give doctors and families more evidence of its safety. The principal investigator for the trial at Sydney Children’s Hospital, Dr Orli Worgan, will provide an update on the trial progress in the November edition of Reach Out.
During the trial and the lengthy approvals process that might follow, there is still a way for TSC families to access the treatment. You need to discuss this treatment option with a dermatologist who will assess each individual case. This is called using the medicine “off-label” and may also be subject to approval processes. The team at the Princess Margaret Hospital for Children in Perth have published their experience with the cream and their recipe.
You may like to refer your dermatologist to the following scientific journal article: Foster, R. S., Bint, L. J. and Halbert, A. R. (2012), Topical 0.1% rapamycin for angiofibromas in paediatric patients with Tuberous Sclerosis: A pilot study of four patients. Australasian Journal of Dermatology, 53: 52–56. This article suggests an annual cost of approximately $3000. Because there is no centralized manufacturer and the medicine is not yet on the Pharmaceutical Benefits Scheme, funding arrangements will differ between health services, so you should also discuss this with your doctor. Please contact ATSS if you need any more information.