Downham Market mum tells of fears for son, 6, over drug funding for rare illness

Kayla Yates and Thomas Lichterfeld (6) from Downham Market
Kayla Yates and Thomas Lichterfeld (6) from Downham Market
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A West Norfolk mum fears her son will die younger because of new guidelines on the amount that should be spent on drugs for rare illnesses.

Kayla Yates’ six-year-old son, Thomas Lichterfeld, has a condition known as Hunter’s disease, a genetic disorder that affects one in every 162,000 boys worldwide. There is no known cure.

Trials of a new treatment, which is believed to be capable of reversing some of the damage the disease does to patients’ brains, have already been carried out.

But campaigners fear new rules on the amounts that can be spent on such treatments, which were introduced at the weekend, will deny patients like Thomas, and sufferers of other rare diseases, the chance of living longer, more fulfilling lives.

Kayla, who lives in Downham, said: “What gives some guy in a suit the authority to decide whether my son lives or dies?”

However, health chiefs have insisted they had not capped the amount that can be spent on new, expensive treatments.

They also maintain they are speeding up access to new treatments, which they claim is now faster than anywhere else in Europe.

The condition means Thomas needs 24-hour care, is unable to communicate with strangers and has no spatial awareness or understanding of danger.

Kayla says new funding guidelines which came into force at the weekend mean a new therapy, which she put Thomas forward for trials of last year, would not have a chance of being funded, as it does not extend patients’ lives for long enough and costs even more than already funded treatments.

Most Hunter’s patients die between the ages of 12 and 17. While it is believed the new drug would enable patients to live well into their 20s, it is thought that would not be long enough for it to be funded.

Kayla spoke out after NHS chief executive Simon Stevens admitted on Friday that patients were likely to have to wait longer for non-emergency operations to prioritise improved care in other areas.

She said she accepted the NHS was under financial pressure, but added: “I can’t stay impartial when it’s my son. I wouldn’t wish this on my worst enemy.”

The National Institute for Health and Care Excellence (NICE), which approved the measures last month, has insisted they will maintain access to innovative treatments despite the continuing funding pressures on the NHS.

Its chief executive, Sir Andrew Dillon, yesterday insisted there was no cap on the amount that could be spent on a new drug and only a “minority” of treatments would be affected by a requirement for talks between the NHS and pharmaceutical firms if costs exceeded £20 million a year.

He added: “Other changes, some already in place, are speeding up the evaluation and funding of new treatments, particularly urgently needed cancer drugs, for which funding is now available from the day they are licensed, which is faster than elsewhere in Europe.”

But the MPS Society, a charity which supports patients with conditions such as the one that Thomas has, has warned the rules are likely to put treatments out of the reach of many patients.

Chairman Paul Moody said: “A decision by NICE and NHS England to implement this new policy confirms that children and young adults with ultra-rare diseases going forward are economic pawns in a failing NHS and cheaper dead than alive.”

Kayla, who has also lobbied her local MP Elizabeth Truss, said: “If Thomas had cancer, there would be no qualms about providing a drug, even if it was a rare cancer. But because it’s a rare disease that isn’t a cancer, it feels like it’s being swept under the carpet.”

Almost 4,000 people have already signed an online petition calling for the recommendations to be put on hold. The petition can be found by searching for MPS Society at