MPs call for ‘urgent review’ of the way medicines are approved for rare diseases

MPs have called for an “urgent review” of the way medicines for rare diseases are approved as patients are being “left behind” under the current system.

Labour MP Liz Twist said Nice (National Institute of Clinical Excellence) does “not properly address the medical treatment needs of people” with conditions such as muscular dystrophy and cystic fibrosis.

The member for Blaydon, who had been granted the Commons debate on the issue, said she developed an interest in the matter due to her 12-year-old constituent Archie McGovern, who suffers from the incurable PKU (phenylketonuria).

The condition, where people cannot process protein, requires sufferers to live on an extremely restrictive diet.

Ms Twist said there is a drug which can be used to treat it, called Kuvan, but despite its availability elsewhere in Europe and other countries around the world, is not on the NHS.

Tory MP James Morris (Halesowen and Rowley Regis) backed her calls to have the guidelines changed, saying Nice’s appraisal process “does not take into account of the social costs of the alternative treatments”, saying the restriction has a huge impact on families.

Ms Twist agreed, and said: “We are reducing the lives of these children and adults to a cost-effectiveness analysis.”

As well as calling on drug manufacturers to make their treatments more affordable, she called on Nice to “review and make the necessary changes so people don’t fall through the cracks”.

In response Health Minister Steve Brine said Nice has “initiated an appraisal of Kuvan” and said “a final decision will be taken promptly”.

Elsewhere in the debate, Labour MP Stephen Twigg (Liverpool West Derby) called for full-extract cannabis oil to be more widely available to help people with rare disorders.

He told the House about a 10-month-old son of a constituent called Nathaniel who had been suffering near-constant seizures.

Mr Twigg said: “Nathaniel is 10 months old.

“He has a type of epilepsy that is so rare that when he was diagnosed, his doctors told Lauren, his mum, and his dad, James, that Nathaniel is the only known case with this type of genetic mutation recorded anywhere in the world.

“For the next three months of his life, Nathaniel was in Alder Hey hospital undergoing tests and, for most of that time, he was in critical care.”

Mr Twigg said that, due to the “extremely rare type of epilepsy Nathaniel has”, anti-epileptic drugs do not work, but he was granted the use of medicinal cannabis products which he had responded positively to.

The MP said access to full-extract cannabis oil “might offer him the better quality of life that Nathaniel surely deserves”.

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