Julian Pushes NHS England to Provide Better Care for Sufferers of Tuberous Sclerosis
October 25, 2016
Julian is calling on NHS England to urgently publish a new prescribing policy for the drug Everolimus, used to treat brain tumours caused by Tuberous Sclerosis Complex (TSC).
TSC is a life-threatening genetic disease which can lead to the growth of benign tumours throughout the body, including in vital organs such as the heart, kidneys, lungs and brain. Tumours in the brain can lead to severe epilepsy as well as having a huge impact on intellectual development in young sufferers.
In 2011 the highly effective drug treatment, Everolimus, was approved in the EU for the treatment of TSC-related tumours of the brain. However, NHS England have not approved a prescribing policy for Everolimus for brain tumours, resulting in an estimate 20 deaths per year.
Under a new prioritization process sufferers of TSC-related brain tumours lost out as the treatment was deemed ‘unaffordable’. This is despite treatment costing less than dialysis for kidney failure.
Without this treatment all patients with inoperable brain tumours will suffer progressive neurological deterioration and die, usually within 2 years, but treatment with Everolimus results in up to 95% of patients living long and healthy lives according to the Tuberous Sclerosis Association. Sadly 20 children and young adults are dying every year in England due to TSC-related brain tumours.
At a meeting in Parliament Julian met with his constituent Mr Nicholas Dale whose son Sam suffers from TSC.
Commenting Mr Dale said, “All everyone wants is a transparent and fair system. In our case the only reason we currently have our son’s medication funded is because we paid for it ourselves to prove that it worked. Sam is very lucky that we have family and friends who wanted and are capable of supporting us financially. Most people are not so lucky and that is simply not fair. In our experience care in the NHS is brilliant, but we need decision makers and policy staff to meet the high standards set by the medical staff.
“Unfortunately, it does not appear that this will ever happen unless there is political pressure from MP’s to force it to happen. No family should have to watch their child deteriorate towards death whilst the NHS hides behind an unworkable administrative procedure. It is simply not fair to delay policy when this policy ultimately leads to children dying.”
Following the meeting Julian said, “We must ensure that sufferers of rare diseases like TSC are not left behind by drug prescription policies. It was a pleasure to meet with Mr Dale and his family, and to find out more about this issue from the Tuberous Sclerosis Association.
“I am organising a joint letter from MPs to lobby NHS England on this incredibly important issue. Where such highly effective medicines are available we must ensure that patients get access to the treatments they so desperately need.”
