DE­CI­SION IS A BIT­TER BLOW FOR FAM­ILY

Par­ents slam or­gan­i­sa­tions ‘pri­ori­tis­ing cost over health of chil­dren’

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

A FAM­ILY has been left dev­as­tated fol­low­ing a gov­ern­ment de­ci­sion to not rec­om­mend fund­ing for a break­through drug.

Louisa Hill – who’s son Archie suf­fers from mus­cu­lar dys­tro­phy – slammed a re­cent de­ci­sion made by the Na­tional In­sti­tute for Health and Care Ex­cel­lence (NICE) over the Duchenne mus­cu­lar dys­tro­phy drug Translana (ataluren).

She said: “We are ab­so­lutely dev­as­tated.

“How do we tell Archie he is not al­lowed a drug that will keep him walk­ing and liv­ing for longer be­cause NHS England and drug com­pa­nies can­not agree on a price?

“What sort of an ex­am­ple are th­ese peo­ple set­ting for so­ci­ety if they are pri­ori­tis­ing cost over the health of our chil­dren? We will not be giv­ing up. This fight is far from over.”

The Na­tional In­sti­tute for Health and Care Ex­cel­lence (NICE) an­nounced in its draft guid­ance ear­lier this month it will not rec­om­mend fund­ing on the NHS for Translana.

But NICE have asked PTC Ther­a­peu­tics, man­u­fac­tur­ers, to pro­vide fur­ther jus­ti­fi­ca­tion for the cost of the drug and clar­i­fi­ca­tion of its ben­e­fits ahead of con­firm­ing its fi­nal po­si­tion in the com­ing months and stress this is not their fi­nal de­ci­sion.

Pro­fes­sor Ca­role Long­son, NICE Health Tech­nol­ogy Eval­u­a­tion Cen­tre di­rec­tor, said: “We are dis­ap­pointed not to be able to rec­om­mend ataluren in this draft guid­ance. DMD is one of the most com­mon and se­vere forms of mus­cu­lar dys­tro­phy. It re­duces life ex­pectancy and causes de­bil­i­tat­ing symp­toms that se­verely af­fect the qual­ity of life of peo­ple with the con­di­tion, and their fam­i­lies and car­ers.”

She added: “How­ever, the Com­mit­tee was not con­vinced that the pro­posed cost of ataluren was jus­ti­fied by the ev­i­dence pre­sented on the ad­di­tional health ben­e­fits as­so­ci­ated with ataluren over stan­dard ther­apy.

“There­fore, on the ba­sis of the cur­rent ev­i­dence, the Com­mit­tee was minded not to rec­om­mend ataluren for treat­ing non­sense mutation DMD.”

In Au­gust 2014, Translarna be­came the first drug ad­dress­ing the cause of Duchenne mus­cu­lar dys­tro­phy to be ap­proved in the EU, and since Au­gust last year has been avail­able in sev­eral EU coun­tries.

It is said to keep those who suf­fer from Duchenne mus­cu­lar dys­tro­phy walk­ing for longer.

Re­cently a lo­cal NHS Trust in Scot­land ap­proved the drug for one lo­cal boy, fol­low­ing an in­di­vid­ual fund­ing re­qust.

Robert Mead­owcroft, chief ex­ec­u­tive of Mus­cu­lar Dys­tro­phy UK, said: “The news is ex­tremely dis­ap­point­ing, and a bit­ter blow for all of us await­ing the news.

“Hav­ing spo­ken to some of the fam­i­lies af­fected, it re­ally is im­pos­si­ble to de­scribe their heart­break, par­tic­u­larly as just last week Loth­ian NHS Trust ap­proved the drug for a fam­ily in Scot­land.

“Many par­ents across the rest of the UK are watch­ing their child weaken and lose mo­bil­ity day by day, as this re­ally is a race against time. If we are not care­ful, it will be too late for many of th­ese boys to even be el­i­gi­ble to take Translarna.

“Duchenne is a life­lim­it­ing con­di­tion, and we must take ev­ery op­por­tu­nity to pro­tect chil­dren and their qual­ity of life.

“The chance to be able to walk for longer can be im­mea­sur­ably pre­cious. We may not yet be able to halt the dif­fi­cult phys­i­cal and emo­tional chal­lenges th­ese chil­dren face, but with this drug, we have the chance to de­lay them.

“PTC Ther­a­peu­tics needs to work with NICE and NHS England to find an ur­gent so­lu­tion to ap­prov­ing this break­through ther­apy. We will not give up on th­ese boys who are el­i­gi­ble for Translarna – they must have ac­cess to this life-chang­ing drug.”

The next com­mit­tee meet­ing is sched­uled for Novem­ber 17, fol­low­ing which the next draft guid­ance will be is­sued.

A fi­nal rec­om­men­da­tion is ex­pected to signed off early next year. For MP Ch­eryl Gil­lan’s thoughts about this is­sue see page 15

Deves­tated: Archie Hill with his par­ents Louisa and Gary and elder brother Ley­ton

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