Questions over cost of muscular dystrophy drug

Questions are being asked over why Belfast Trust is quoting ten times the amount of money to treat young muscular dystrophy sufferers with a new drug as a trust in England.

The issue was raised in the House of Commons by Chris McDonald, MP for Stockton North, who is concerned patients across the UK are facing a “postcode lottery”.

The drug, Givinostat, has been approved as safe for use, and the National Institute for Health and Care Excellence (NICE) is due to make a decision on whether to recommend it for use on the NHS later this year.

Despite drug manufacturers offering it for free, Belfast Health Trust, NI’s specialist centre for the condition, has so far declined to begin the treatment.

The Belfast Trust has cited the need for “additional staffing resources” as the reason for not offering the drug.

Some trusts in England, Scotland and Wales have already started offering treatment under an early access programme (EAP).

Under a Freedom of Information request, Belfast Trust revealed it had estimated it would cost £309,000 annually to treat 13 patients with the new drug. That’s more than £23,000 per patient.

In a separate FOI request, the Oxford University Hospitals said they estimated the cost at £66,000 for 35 to 40 patients: just under £2,000 a head.

12 year old Alfie Pentony from Newry has Duchenne Muscular Dystrophy.

It is a life-limiting muscle wasting disease which affects boys.

He will eventually need a wheelchair and there is currently no cure but this new drug Givinostat has been shown to keep patients on their feet for longer.

His mother Colleen Pentony said she cannot understand how the Belfast Trust came to their cost estimate.

“It’s ridiculous. To me, all they need is a nurse and a consultant.

“They take the medicine at home, I would administer it to Alfie in the morning and night at home.

“It’s a stable medicine, you don’t even have to keep it refrigerated. It’s like a bottle of Calpol.”

Mrs Pentony said the only additional resources needed would be to facilitate the blood tests the boys need, at first every two weeks, then every three months.

She said Belfast Trust had included a physiotherapist, play therapist and psychologist in the costings.

“These job roles should not be included in this costing. We are supposed to have a physiotherapist already, why would these boys need a play therapist, the boys are all over 6?”

“And any time in the past I asked for psychiatric help for Alfie, they refer me to CAHMS (Children and Adolescent Mental Health Services)”, she added.

Speaking in the House of Commons on Thursday, Chris McDonald MP, said there should be consistency in approach across the UK.

“Currently the Belfast Trust is not offering this drug to lads in Northern Ireland because they’re claiming it will cost £309,000,” he said.

“That’s an order of magnitude out with the rest of the UK so I’d be grateful if the Minister would look very carefully at the situation in NI.”

A spokesperson for Belfast Trust apologised to patients, saying said it was “not in a position” to offer the treatment.

The spokesperson said a multi-disciplinary team approach is adopted for patients with Duchenne Muscular Dystrophy which includes physiotherapists, neurology, cardiology and respiratory specialists as well as endocrine specialists.

When asked why their costing was higher than Oxford University Hospitals Trust, a spokesperson said: “It is not possible for the Belfast Trust to comment on costs associated with service delivery within another organisation as individual providers will have different infrastructure and staffing levels in place.”

A statement from the charity Duchenne UK said patients in Northern Ireland are losing out to a “cruel postcode lottery”.

“The estimated cost that Belfast Trust has produced seems high and does not align with any other figures we have seen from other places.”