Joy for fam­ily as life-chang­ing treat­ment gets ap­proval

Buckinghamshire Advertiser - - FRONT PAGE - by Tom Her­bert tom.her­bert@trin­i­tymir­ Twit­ter: @TRHer­bert

A YOUNG boy from Chal­font St Peter has won the fight to be given ac­cess to a life-chang­ing mus­cu­lar dys­tro­phy drug.

10-year-old Archie Hill, of Nortoft Road, has cam­paigned tire­lessly for over a year to hear whether he will be granted ac­cess to break­through drug Translarna, which is said to keep boys suf­fer­ing from Duchenne walk­ing for longer.

On Fri­day morn­ing, the Na­tional In­sti­tute for Health and Care Ex­cel­lence (NICE) an­nounced it had se­cured an agree­ment to rec­om­mend the drug.

It is in­tended to cover ac­cess to Translarna for el­i­gi­ble chil­dren in Eng­land for five years. Dur­ing this time, 50 chil­dren will be closely mon­i­tored and their progress com­pared to that of other boys with the con­di­tion. The data gath­ered will in­form NICE’s ul­ti­mate de­ci­sion on fund­ing the drug, when the agree­ment con­cludes.

His mum Louisa Hill said: “You can imag­ine we’ve been ab­so­lutely buzzing. I can’t quite be­lieve it. It’s given us the in­cen­tive to fight.”

But there are fears that be­cause of the al­most 18 month long wait, some chil­dren may have lost the abil­ity to walk, mean­ing they will no longer meet el­i­gi­bil­ity cri­te­ria.

Archie’s cam­paign­ing has seen him meet Ar­se­nal foot­ball player Jack Wil­shere, hand de­liver let­ters to Prime Min­is­ter David Cameron and be in­ter­viewed on na­tional tele­vi­sion.

Mrs Hill said she wanted to thank every­body for rais­ing the pro­file of the cam­paign.

“It’s go­ing to make a huge dif­fer­ence to so many boys. It gives them those years,” she said.

But Mrs Hill warned that while it was a fan­tas­tic step for­ward, the fight is far from over.

“We need to find some­thing that halts it com­pletely. This is just the be­gin­ning, so one day fam­i­lies like ours don’t have to go through this,” she said.

She was full of praise for Archie and his older brother Ley­ton, who have been ‘in­cred­i­ble’ to have ‘shown such ma­tu­rity’ dur­ing the cam­paign.

“Never give up on some­thing you be­lieve in,” she added. “I’m proud of my fam­ily, that we have helped make a dif­fer­ence to chil­dren’s lives.”

All Archie wants to do, said Mrs Hill, is be able to carry on play­ing foot­ball with his friends, and she ad­mit­ted the hard times are yet to come: “I’m proud but I look at Archie and still think we have got a tough road ahead. This is the easy bit for him. This is go­ing to help him for the next few years.”

Mrs Hill said she will keep on fight­ing to find a drug which will stop the con­di­tion com­pletely, and will keep on go­ing for Archie.

“It’s kept me go­ing,” she said. “It’s given me a mas­sive fo­cus. It’s driv­ing me for­ward.”

Ch­eryl Gil­lan, MP for Che­sham and Amer­sham, said: “This is won­der­ful news for Archie and all the boys who can ben­e­fit from Translarna. The long wait for NICE’s ver­dict is fi­nally over and it is the out­come we have all been hop­ing for. I would like to pay trib­ute to the Hill fam­ily and to all the fam­i­lies in­volved in the cam­paign for their tena­cious and tire­less ef­forts to reach this point.”

Robert Mead­owcroft, chief ex­ec­u­tive of Mus­cu­lar Dys­tro­phy UK, said: “Par­ents of chil­dren el­i­gi­ble for Translarna have fought coura­geously for this out­come, and to give their chil­dren the chance to keep walk­ing for longer. This an­nounce­ment comes as won­der­ful news and a true vic­tory for the fam­i­lies.

“A Man­aged Ac­cess Agree­ment means that within a mat­ter of months, NHS Eng­land will be sup­ply­ing el­i­gi­ble chil­dren with the first ge­netic ther­apy avail­able to treat the con­di­tion. It is a chance to trans­form child­hoods.

“How­ever, we are con­cerned now that it could takes months for NHS Eng­land to im­ple­ment the agree­ment and get the drug to clinic. Hav­ing waited 18 months for a de­ci­sion, this is a de­lay boys and their fam­i­lies can ill af­ford.

“We call on NHS Eng­land to act with the ur­gency and re­solve that th­ese chil­dren and their fam­i­lies de­serve.”

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