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Spinraza: Bristol teenager 'ineligible' for SMA drug

BBC Jake OgborneBBC
Jake Ogborne's condition is deteriorating and he is unable to walk

A teenager with a rare muscle-wasting disease has learned he is not eligible for a life-changing drug months after he held a party to celebrate it becoming available on the NHS.

Jake Ogborne, 18, from Bristol, has spinal muscular atrophy (SMA).

But he does not meet criteria from medicines regulator, NICE, for access to the drug Spinraza.

NICE said it would review guidelines early if new evidence on benefits for patients like Jake became available.

SMA is a genetic condition which affects the nerves in the spinal cord, weakening muscles and causing problems with movement, breathing and swallowing.

While not a cure, trials have shown that Spinraza, the trade name of drug nusinersen, can slow the effects of SMA in some cases.

Family photo Family holding champagne glassesFamily photo
Jake celebrated with family and friends when he thought he would have access to the drug

In May this year, when NICE (the National Institute for Health and Care Excellence) announced a deal had been negotiated to access the drug on the NHS, Jake celebrated with family and friends.

But a month later he discovered a list of criteria ruled him out and may not be reviewed for five years.

"I felt it was so unfair and discriminatory," he said.

"It made no sense to think that they thought that it wouldn't help me enough even though it clearly would."

His mother Kate Ogborne said: "We don't know where Jake's going to be within five weeks, five months. Five years is too long.

"He's going to be so much weaker. It's absolutely not good enough."

Jake's case has been raised in Parliament by Bristol East MP Kerry McCarthy.

"There was no mention of eligibility criteria and then suddenly he was told that he wouldn't be eligible," the Labour MP said.

"It's immensely cruel for an 18-year-old to be faced with a decision like that."

Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said it was "not closing the door" to people such as Jake.

"Uniquely for this type of arrangement we have made a commitment that, during the five-year course of the managed access agreement, should evidence become available on the potential benefits for patients that are currently not included, we will review that evidence to see whether it would support a change in the inclusion criteria," she said.