As ne­go­ti­a­tions with Eng­land la­bor on, Scot­land re­jects rou­tine use of Ver­tex's cys­tic fi­bro­sis drugs

As the bat­tle be­tween Ver­tex and Eng­land’s Na­tion­al Health Ser­vice (NHS) con­tin­ues — the Scot­tish NHS has re­ject­ed two of the drug­mak­er’s cys­tic fi­bro­sis med­i­cines.

The US drug­mak­er has been locked in ne­go­ti­a­tion with NICE, which has re­fused to al­low the drug in to Eng­land’s NHS un­til Ver­tex $VRTX of­fers it a dis­count on the treat­ment’s price tag that would com­pel the agency to look fa­vor­ably up­on its cost-ef­fec­tive­ness. On Mon­day, the Scot­tish NHS spurned two of the com­pa­ny’s med­i­cines — Orkam­bi and Symke­vi — for the same rea­son, to the dis­may of thou­sands of pa­tients.

Each drug car­ries a list price of more than £100,000 per pa­tient per year. The UK has more than 10,400 cys­tic fi­bro­sis pa­tients – the largest CF pop­u­la­tion out­side the US, ac­cord­ing to the Cys­tic Fi­bro­sis Foun­da­tion.

Cam­paign­ers in the Unit­ed King­dom have been coax­ing the NHS to cov­er Ver­tex’s CF drugs but to no avail. To the hor­ror of UK cys­tic fi­bro­sis pa­tients, a Ver­tex ex­ec­u­tive dis­closed in March, that last year close to 8,000 packs (each con­tain­ing a 28-day sup­ply) of the com­pa­ny’s treat­ment, Orkam­bi, were de­stroyed af­ter cross­ing their ex­piry date. In a stand­off with UK par­lia­ment in March, Ver­tex chief Jeff Lei­den stood his ground, de­spite be­ing chas­tised by a pletho­ra of MPs for Ver­tex’s pric­ing strat­e­gy, busi­ness mod­el and ethics.

Jeff Lei­den Ver­tex

The cys­tic fi­bro­sis drugs made by Ver­tex are the first treat­ments that ad­dress the un­der­ly­ing ge­net­ic caus­es of cys­tic fi­bro­sis, which is char­ac­ter­ized by a thick sticky mu­cus in the lungs, di­ges­tive sys­tem and oth­er or­gans that re­duces life ex­pectan­cy. In its sec­ond-quar­ter re­sults — pub­lished in late Ju­ly — Ver­tex’s tri­fec­ta of CF med­i­cines com­bined gen­er­at­ed near­ly $1 bil­lion in sales, up 25% from the pre­ced­ing quar­ter. The FDA is al­so re­view­ing the Boston biotech’s three-drug cock­tail for CF, which is ex­pect­ed to treat 90% of CF pa­tients.

The com­pa­ny is dis­ap­point­ed that Orkam­bi and Symke­vi have not been rec­om­mend­ed for rou­tine use in Scot­land, a Ver­tex spokesper­son told End­points News, adding that since learn­ing about the de­ci­sion, Ver­tex is work­ing with the Scot­ting gov­ern­ment to al­low “broad ac­cess” for all el­i­gi­ble pa­tients — which the drug­mak­er hopes to set­tle in the com­ing weeks.

“Ver­tex will con­tin­ue to pro­vide ac­cess to our med­i­cines via the PACS Tier 2 process — which al­lows clin­i­cians to ap­ply for ac­cess for in­di­vid­ual pa­tients based on clin­i­cal need — and was put in­to place pri­or to Ver­tex’s sub­mis­sion in Scot­land. So far, about 65 pa­tients have been ap­proved to re­ceive these med­i­cines un­der this scheme,” the spokesper­son added.

Apart from its rul­ing on Ver­tex’s med­i­cines, Scot­land’s cost-ef­fec­tive­ness watch­dog — the Scot­tish Med­i­cines Con­sor­tium — has made the de­ci­sion to back Akcea Ther­a­peu­tics’ drug for hered­i­tary transthyretin-me­di­at­ed amy­loi­do­sis, Tegse­di; Ab­b­Vie’s blood can­cer drug, Ven­clyx­to; Ca­mu­rus’ treat­ment for opi­oid de­pen­dence, Bu­vi­dal, in a re­strict­ed ca­pac­i­ty; and Almi­rall’s ther­a­py for plaque pso­ri­a­sis, Ilumetri, for lim­it­ed use.

So­cial im­age: Ver­tex, via com­pa­ny site

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Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

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