The mum of a five-year-old boy with a rare form of dementia has to pay almost £3m for his life saving treatment.
Tate McDaid, from Swansea, was diagnosed with Sanfilippo Syndrome – a form of dementia that just over 100 UK children have – when he was four.
In September, the first gene therapy to treat the condition in children was approved in the US.
His mum Tammy said she spent “months praying” for its approval but discovered the treatment is to set cost £2.95 million per child, with it unclear how families outside the US can fund this.
Health officials from Wales are currently talking to those in the US to see what potential access pathways there are for Tate.
“I was excited when the treatment initially got approved but then reality hit hard the next day,” Tammy said.
This was when she read the US price for the one-time treatment, external had been set at $3.95 million.
Tammy contacted Ultragenyx, who developed the treatment, in the hope of understanding how families outside the US, who do not have health insurance there, will cover the costs.
“We understand what they are charging for American citizens, but they’ve got health insurance,” she added.
“What is it for us in the UK?
“Not even just the UK, but everyone else in the world looking for this treatment.”
Explaining how the US approved drug, FAYUVI, would work for families outside the US, Ultragenyx said: “There is not a single estimate we can provide for the total cost of care for an international patient, as those costs would depend on the patient’s individual medical needs and the treating institution.”
