MP hits out at NHS trust for ‘misleading’ family

HENLEY MP Freddie van Mierlo has called on a hospital trust to apologise for “misleading” the family of a boy with a muscle-wasting condition.

Ben Clarke, 11, a pupil at Gillotts School in Henley, has Duchenne muscular dystrophy, a condition which causes significant muscle weakness.

His father Alex Clarke was assured in March by Mr van Mierlo that the Oxford University Hospitals NHS Trust was ready to roll out an early access programme to allow patients to access new medicine Givinostat.

Despite this, the trust later admitted to Mr Clarke that it was not in fact ready to do so and could not commit to a timescale.

Now Mr van Mierlo has written to the trust to formally request an apology.

The trust has said that it was exploring how to deliver the drug, but funding challenges meant the process was “not straightforward”.

Ben, who lives in Sonning Common, was first diagnosed with Duchenne aged four after his family noticed him falling over a lot.

The condition, which primarily affects boys, means that he will slowly lose the ability to move independently.

The condition occurs in one in 3,500 to 5,000 newborns and is caused when very little or no dystrophin, a protein, is made by the body due to a genetic mutation.

Ben’s family had been hopeful that the new drug Givinostat would improve his quality of life. It works by reducing inflammation and fibrosis in muscle tissues, promoting muscle regeneration, and potentially slowing disease progression.

The drug has shown positive results in trials and was given conditional approval in the UK in December.

It has been made available to the NHS for free through an early access programme by its manufacturer, ITF Pharma. Other NHS trusts across the country have already begun providing Givinostat under the programme and Ben is eligible for the treatment. Despite this, the Oxford trust is yet to apply for Ben’s inclusion in the programme, citing a lack of medical and pharmacy resources to support its provision.

Mr van Mierlo said the trust had shown a “lack of respect” towards his constituents.

“I took the trust at its word and shared the information with my constituent,” he said.

“To later find out the trust was not ready and could not provide Ben with the treatment he needs was devastating for the family.”

Mr van Mierlo leads an all-party parliamentary group for access to medicines and medical devices. He demanded urgent action to deliver the new treatment for Duchenne.

Mr van Mierlo said: “It beggars belief that a transformative new medicine is being denied to young boys with Duchenne due to red tape and bureaucracy.

“The company is providing the medicine for free, meaning the trust only has to find the staff time needed to carry out the consultations needed.

“Other trusts in the UK are now providing this medicine and yet boys in Oxfordshire are being let down.”

Mr Clarke described the situation as “absolutely gut-wrenching”. He said: “As a family, we are doing everything we can to ensure Ben has the best chance of living a fulfilling life.

“This drug could slow down the progression of his condition but we need action now. Every day that passes without access to Givinostat feels like we are running out of time. I urge the trust to follow through on their earlier assurances and get Ben the treatment he urgently needs.” Ben is currently mobile on his feet but uses a wheelchair to cover longer distances.

Mr Clarke said that the delay in getting the drug could prolong his son’s ability to walk for an extended amount of time.

He added: “This could be the difference between him walking for the next 12 months or being in a wheelchair.”

Professor Andrew Brent, chief medical officer at the trust, said: “We are currently in the process of reviewing how we might provide Givinostat, including consulting colleagues across the country.

“Unfortunately, although the drug is being offered for free by the company, there are many other costs to delivery of the treatment as the medication needs close monitoring which will require additional staffing resource and expertise.

“We are currently investigating how we might do this but it is not straightforward without NHS commissioning funding to support the service and at a time when we, like all of the NHS, are being asked to reduce our costs.

“We want to do all we can to support patients with Duchenne and are exploring whether there is any way we can deliver Givinostat without stopping other essential care.

“We understand Alex’s and other key stakeholders’ frustration that this can’t be done more easily or quickly and we will continue to keep them updated. We are also hoping to meet Alex in the near future to discuss this in person.”