The Pharmaceutical Benefits Advisory Committee (PBAC) has announced on Friday that it will recommend Gilenya (fingolimod) and Tysabri (natalizumab) to be reimbursed for young children with relapsing-remitting MS (RRMS) under the Pharmaceutical Benefits Scheme (PBS).
MS is most commonly diagnosed during young adulthood, but up to 5% of those diagnosed with MS are children. There has been a lack of information on the safety and side effects of MS medications in children because clinical trials in children are challenging. However, it is common for children to be treated “off-label” with adult medication. PBS-supported treatments for MS is vital to maximising the availability of evidence-based treatments for people with MS and provide affordable access for all those who need it, regardless of their age.
Gilenya is an oral medication that selectively targets white blood cells and redirects them away from the brain and spinal cord, and is normally taken daily at a dose of 0.5mg. Clinical trials involving children aged 10 to 17 years were given Gilenya at a dose of 0.5mg or 0.25mg a day for people weighing less than 40kg, and found a lower rate of relapse and less accumulation of lesions over a two-year period. Other studies suggest that Gilenya also reduces the annualised relapse rate compared to placebo and interferon beta-1a in young adults. The PBAC has recommended that Gilenya be provided as 0.25mg capsules, making it available for people who are less than 40kg, including children.
Tysabri is provided as a monthly infusion and acts by binding to white blood cells to prevent them from moving into the brain and spinal cord where they cause inflammation. There have been a number of observational studies that have shown that treatment with Tysabri is safe, well-tolerated and highly effective in children. The PBAC has recommended that the age restriction on Tysabri be removed.
The PBAC needs to consider a number of factors when making recommendations, including the effectiveness and cost of a treatment relative to other available medicines, and the cost of a new listing for the government. This decision is the first step in a process which will now see the decision go before the Federal Government for final approval. If approved, it will then allow young people with MS across Australia access these treatment options.
MS Research Australia supports affordable access to all proven treatment options allowing people of all ages with MS and their doctors to find effective therapies suited to their individual circumstances, and looks forward to the addition of Gilenya and Tysabri on to the PBS, making them affordable treatment options for younger people with MS.