NHS England has today deferred a decision on Vimizim - the drug required by six-year-old Morquio syndrome patient Sam Brown from Otley - by a further 12-16 weeks.
Adam Fowler reports:
After a wait of more than six months, NHS England it will make its final decision on funding after after NICE, the National Institute for Health and Care Excellence has concluded its report into the drug.
NICE has said that doubts exist about the benefits and cost of this treatment in its recent draft guidance, and is therefore “minded not to recommend” the treatment for patients with Morquio A Syndrome. The manufacturer now has the opportunity to present additional evidence to NICE to address these concerns. If NICE ultimately does recommend the NHS funds this treatment then NHS England will do so. NHS England believes the manufacturer has an ethical duty to continue to support those patients it enrolled in clinical trials of elosulfase alfa whilst the NICE process is still ongoing.
Sam Brown received his last free round of treatment last week.
BioMarin, the company which provides Sam's treatment said it was "disappointed" by NHS England's decision.
BioMarin is disappointed by NHS England’s decision not to provide interim funding for Vimizim. Vimizim is the only enzyme replacement therapy for people with Morquio A syndrome, a rare genetic condition that is progressively degenerative and life limiting. BioMarin continues to work closely with all stakeholders to ensure that a long term funding solution for Vimizim is found for all patients with Morquio A syndrome, so that they are able to access this treatment in England.
Cyclist Lizzie Armitstead has tweeted to allay fears she had broken her leg following a horror smash just seconds after crossing the finish line of the Women's Tour in Suffolk.
The 26-year-old took to Twitter to confirm that she had not broken any bones and to thank hospital staff.
She will, however, not be taking any further part in the race, as she recuperates ahead of the National Cycling Championships later in the month:
I remember winning with the help of my team mates and then not much else, but I am ok, nothing broken just very sore, thank you for all your
kind messages and to the NHS staff who took care of me so well. I won't start tomorrow in the hope of recovering properly for the Nationals.
Cyclist Laura Trott says she hopes Lizzie Armitstead's injury which saw here airlifted to hospital following a crash on the first day of the Women's Tour is not too serious.
26-year-old Armitstead, from Otley, was raising her arms to celebrate winning the first stage of the race when she collided with someone inside the barriers:
Commonwealth champion Lizzie Armitstead has been airlifted to hospital after crashing in the Women's Tour. The 26-year-old, who's from Otley, was raising her arms to celebrate winning the first stage of the race when she collided with someone inside the barriers. It is thought she has broken her leg.
A representative from her team Boels Dolmas collected her winner's jersey on the podium on her behalf.
A campaign song for Sam - a six year old from Otley who suffers from a rare condition called Morquio Syndrome - has been released online and as a single.
It's called 'There is a boy' and you can watch the full version here:
It was made to raise awareness of Sam's ongoing campaign to get the drug Vimizin funded by the NHS. Due to delays in NHS England's decision making process Sam currently receives the drug for free as an "act of good will" from the drug company Biomarin, but that supply will cease if the decision is taken not to fund the drug.
Sam and his family are expecting a decision in the next fortnight.
Click here to see ITV Calendar's behind-the-scenes report - at the recording of the single.
The family of six-year-old Sam Brown say they've been dealt with a "blow of epic proportions" after he government's clinical watchdog said it is unlikely to recommend NHS funding of the drug he needs.
Sam, from Otley, has the rare genetic disease Morquio Syndrome.
The National Institute for Clinical Excellence (NICE) has now asked the company which makes the medicine to reveal just why it's charging almost £400,000 a year for the treatment.
Adam Fowler reports:
Tim Farron will officially announce his candidacy to replace Sheffield Hallam MP Nick Clegg as Leader of the Liberal Democrats when he visits Otley today.
The local Leeds North West MP Greg Mullholland will be the Westmorland and Lonsdale MP's campaign co-ordinator.
Farron served four years as President of the Liberal Democrats, until December 2014, and is hugely popular among party grassroots.
“That over 10,000 people have joined the party since polling day shows we still have huge appeal among voters.
"I have every confidence that we as a party can rebuild after a difficult general election - I want to be at the forefront of that, and that’s why I am running to be Leader of the Liberal Democrats.
“I am greatly looking forward to visiting Otley to announce my candidacy. It’s a great example of an area where people see us as the party with the right priorities and are ready to back us.
"That was clear to see in local residents re-electing Greg Mulholland for a third straight term, and overwhelmingly voting Liberal Democrats to run the local town council. Yes, we had a difficult general election result, but we are determined to rebuild and places like Otley show how we can do that.”
“I am delighted to be backing Tim Farron to be the next leader of the Liberal Democrats. We need to re-establish a strong identity for the party and reconnect and re-engage with the public.
"Tim has a clear vision for how the party can be energised to do just that and I want to be part of that vision and making it happen.
“I am very pleased that Tim agreed to come to Otley to launch his leadership campaign. Otley is a proud liberal town, with strong civic pride and a wonderful community spirit.
"It is great that after the remarkable Liberal Democrat victory in the Otley Town Council elections, Tim is launching the Liberal Democrat fightback – and we want Otley to be an example of how a strong liberal team can deliver for people.
“Tim has the ability to connect with people from all different backgrounds, in all different parts of this country and he is the right person to lead the liberal revival this country needs. "
The town of Otley has made history by becoming the first place in the country to get a successful mass listing of its pubs.
Residents must now be consulted before any of the 19 pubs can be converted into supermarkets or office space. Previously changes could be made without planning permission.
A teenage sailor from West Yorkshire is celebrating today after being picked for a voyage of a lifetime.
18-year-old Heather Thomas from Otley was down to the final two in a competition to get a crew place on the Clipper round the world yacht race.
Heather who has been sailing since she was seven will now be one of the team taking part in the sixth leg of the race when the famous yacht crosses the Pacific Ocean.
People with ultra rare genetic diseases have protested at Westminster in their continuing campaign to try and persuade the health service to keep funding a drug which could change their lives.
They include six-year-old Sam Brown, from Otley, who has Morquio Syndrome.
It is a rare condition that means his skeleton does not grow properly, but his internal organs do.
Sam has been receiving a drug called Vimizin that helps him live a normal life.
It is expensive but it had been provided free by the drug company which produces it.
However, that deal is coming to an end.
Jon Hill reports: