Dis­may over re­jec­tion of ‘life-sav­ing’ drugs on NHS

●Cam­paign­ers’ shock as rou­tine use of cys­tic fi­bro­sis treat­ments ruled out

The Scotsman - - FRONT PAGE - By CHRIS GREEN

Peo­ple with cys­tic fi­bro­sis in Scot­land will con­tinue to be de­nied po­ten­tially life-ex­tend­ing drugs af­ter they were re­jected for use by the NHS, prompt­ing an out­pour­ing of dis­may and anger.

The Scot­tish Medicines Con­sor­tium (SMC) said it had heard “pow­er­ful tes­ti­monies” from pa­tients about the im­pact of Orkambi and Symkevi but had doubts over their cost-ef­fec­tive­ness.

Hun­dreds of Scots suf­fer­ing from the dis­ease, which is caused by a faulty gene and can be to­tally de­bil­i­tat­ing, had been hop­ing that the body would ap­prove the two drugs.

Fam­i­lies in Eng­land, where the drugs are also un­avail­able on the NHS, had said they would move to Scot­land to al­low their chil­dren to be treated if they were ap­proved.

Orkambi and Symkevi slows the de­cline of lung health in both chil­dren and adults – the main cause of death in those with cys­tic fi­bro­sis – but the drugs are said to cost £100,000 a year per pa­tient.

The Cys­tic Fi­bro­sis Trust said the SMC’S de­ci­sion would “shock” peo­ple liv­ing with the dis­ease, who now faced an­other “tragic wait” for the “life-sav­ing” medicines.

Jour­nal­ist and aca­demic James Mce­naney, whose wife Ruth has the dis­ease, said: “Big thanks to the SMC for de­cid­ing that keep­ing peo­ple like my wife alive isn’t worth the money. Feel free to come and ex­plain the de­ci­sion to my five-year-old son any time. In Scot­land we’ll fund ut­ter bulls*** like home­opa­thy on the NHS, but sav­ing the lives of peo­ple with cys­tic fi­bro­sis isn’t cost-ef­fec­tive. Got it.”

Marion Fer­gu­son, whose son Thomas has cys­tic fi­bro­sis and who runs a group cam­paign­ing for pre­ci­sion medicines to treat the dis­ease, said:

“We have waited pa­tiently over the last three-and-a-half years when Orkambi was first sub­mit­ted to and sub­se­quently not rec­om­mended by the smc.

“Along with every­thing that CF throws at us as a com­mu­nity, we have cam­paigned, given press in­ter­views, at­tended meet­ings and lob­bied Par­lia­ment, all of this while watch­ing loved ones and chil­dren de­te­ri­o­rat­ing and dy­ing.

“It’s sim­ply not ac­cept­able in a civilised so­ci­ety that chil­dren and young adults are left to suf­fer when a medicine ex­ists that would end that suf­fer­ing .”

SNP MP Marion Fel­lows, whose three-year-old grand­daugh­ter Saoirse suf­fers from cys­tic fi­bro­sis, said the dis­ease was a heavy bur­den for chil­dren and their par­ents to bear.

“It’s not a pretty thing watch­ing a three-week-old baby scream when they’re get­ting their first lot of physio and you’re told by their par­ents no, we’re not al­lowed to com­fort her be­cause her body has to get used to this,” she added.

SMC chair­man Dr Alan Mac­don­ald said he recog­nised the de­ci­sion would be “dis­ap­point­ing” but there was “sig­nif­i­cant un­cer­tainty” around the drugs’ long-term health ben­e­fits.

“We un­der­stand the pro­found im­pact that cys­tic fi­bro­sis has on pa­tients and their fam­i­lies and we are aware of the need for ef­fec­tive treat­ments that tar­get the un­der­ly­ing cause,” he added.

Ver­tex, the phar­ma­ceu­ti­cal com­pany that makes the drugs, said it was work­ing with the Scot­tish Gov­ern­ment to al­low “broad ac­cess” to the medicines for el­i­gi­ble pa­tients.

“We are all deeply com­mit­ted to reach­ing such a solution as a mat­ter of ur­gency and we are work­ing closely with the Gov­ern­ment to fi­nalise it over the com­ing weeks,” a spokes­woman said.

Health Sec­re­tary Jeane Free­man said some pa­tients in Scot­land al­ready had ac­cess to the drugs through an ex­ist­ing scheme, which has so far ap­proved around 65 peo­ple on a case-by-case ba­sis.

“I fully un­der­stand the great dis­ap­point­ment that peo­ple with cys­tic fi­bro­sis, to­gether with their par­ents, fam­i­lies and friends, will feel about [this de­ci­sion],” she added.

David Rams­den, chief ex­ec­u­tive at the Cys­tic Fi­bro­sis Trust, said the de­ci­sion would come as a shock to peo­ple with cys­tic fi­bro­sis and their fam­i­lies.

“While talk of fur­ther ne­go­ti­a­tion be­tween Ver­tex Phar­ma­ceu­ti­cals and the Scot­tish gov­ern­ment is pos­i­tive, peo­ple liv­ing in Scot­land need th­ese life-sav­ing drugs now,” he said.

“The tragic wait has to end and now is the time for the Scot­tish Gov­ern­ment to re­main com­mit­ted and Ver­tex to do all they can to en­sure that this chance does not slip away again.”

“I fully un­der­stand the great dis­ap­point­ment that peo­ple with cys­tic fi­bro­sis, to­gether with their par­ents, fam­i­lies and friends, will feel about [this de­ci­sion].”



0 Marion Fel­lows with her grand-daugh­ter Saoirse who suf­fers from CF

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