Four years af­ter EU ap­proval, NICE fi­nal­ly backs Revlim­id use in cer­tain first-line mul­ti­ple myelo­ma pa­tients

Four years af­ter the Eu­ro­pean Com­mis­sion sanc­tioned the use of Cel­gene’s flag­ship Revlim­id to treat adults with pre­vi­ous­ly-un­treat­ed mul­ti­ple myelo­ma who are not el­i­gi­ble for trans­plant, Eng­land’s Na­tion­al Health Ser­vice (NHS) will adopt its use as the first or sec­ond line of de­fense — in cer­tain pa­tients. Al­though the block­buster drug is rou­tine­ly used as a back­bone treat­ment in much of the de­vel­oped world, un­til now, the drug was on­ly cleared for third-line use by NICE.

Revlim­id, which gen­er­at­ed rough­ly $9.7 bil­lion in glob­al 2018 sales, has long been Cel­gene’s key­stone treat­ment, but its use in Eng­land had been re­strained due to pric­ing dis­agree­ments. The drug is al­so cru­cial to Bris­tol-My­er’s $BMY $74 bil­lion bet on the big biotech (al­though the loss of patent pro­tec­tion is loom­ing).

Mul­ti­ple myelo­ma is a life-threat­en­ing form of blood can­cer, which orig­i­nates in the bone mar­row, and af­fects rough­ly 17,500 peo­ple in the UK at any giv­en time, ac­cord­ing to char­i­ty Myelo­ma UK, which es­ti­mates each year that about 5,700 new pa­tients are di­ag­nosed with the dis­ease.

NICE pub­lished two pieces of fi­nal draft guid­ance on Fri­day. In the first re­port, NICE rec­om­mend­ed the use of Revlim­id — known chem­i­cal­ly as lenalido­mide — in com­bi­na­tion with the cor­ti­cos­teroid dex­am­etha­sone as an op­tion for treat­ment-naive adults with mul­ti­ple myelo­ma who are not el­i­gi­ble for a stem cell trans­plant and can­not take thalido­mide. This new op­tion could ben­e­fit about 2,100 pa­tients, NICE said.

Thalido­mide (or borte­zomib, or Take­da’s $TAK Vel­cade, for those who can’t take thalido­mide) re­mains the first line of de­fense, al­though NICE ac­knowl­edged that se­vere side-ef­fects as­so­ci­at­ed with its use can rule out its adop­tion. How­ev­er, “(l)enalido­mide plus dex­am­etha­sone can­not be rec­om­mend­ed for un­treat­ed mul­ti­ple myelo­ma in peo­ple who could take thalido­mide be­cause this would not be cost ef­fec­tive,” NICE un­der­scored.

Lenalido­mide is avail­able as a 21‑cap­sule pack, and its cost varies per dose, up to £4,368 for the high­est 25 mg dose/pack. But, Cel­gene $CELG has now agreed to a (con­fi­den­tial) dis­count that has sat­is­fied NICE. 

In the sec­ond re­port, NICE backed the use of lenalido­mide as an op­tion for treat­ing mul­ti­ple myelo­ma in adults if they have had on­ly one pre­vi­ous ther­a­py, in­clud­ing borte­zomib. Par­tic­u­lar­ly for those that have been first treat­ed with borte­zomib, the on­ly next treat­ment in the doc­tor’s tool­box is chemother­a­py — how­ev­er, ev­i­dence in­di­cates that the lenalido­mide+dex­am­etha­sone is more ef­fec­tive than cy­to­tox­ic chemother­a­py. In one clin­i­cal study, pa­tients tak­ing lenalido­mide lived on av­er­age 7 months longer, NICE said.

About 320 pa­tients are ex­pect­ed to ben­e­fit, it added.

The most ‘plau­si­ble’ cost-ef­fec­tive­ness es­ti­mate for lenalido­mide plus dex­am­etha­sone may be above the range that NICE nor­mal­ly con­sid­ers be­ing a cost-ef­fec­tive use of NHS re­sources, re­searchers high­light­ed in the re­port, but con­ced­ed that since lenalido­mide has been rec­om­mend­ed for use as a first treat­ment (for which it is cost-ef­fec­tive) — the need for lenalido­mide as a sec­ond treat­ment will like­ly de­crease be­cause peo­ple are more like­ly to have it as a first treat­ment in the fu­ture.

Last month, NICE al­so backed the use of J&J’s $JNJ Darza­lex in com­bi­na­tion with Vel­cade as a sec­ond-line treat­ment for mul­ti­ple myelo­ma via the can­cer drugs fund.

Cel­gene, mean­while, is ad­vanc­ing the use of triplet mul­ti­ple myelo­ma ther­a­pies. On Thurs­day, the EU ap­proved 1) Revlim­id in com­bi­na­tion with Vel­cade and dex­am­etha­sone for adult pa­tients with pre­vi­ous­ly un­treat­ed mul­ti­ple myelo­ma who are not el­i­gi­ble for trans­plant; 2) Im­novid in com­bi­na­tion with Vel­cade and dex­am­etha­sone in adult pa­tients with mul­ti­ple myelo­ma, who have re­ceived at least one pri­or treat­ment reg­i­men in­clud­ing Revlim­id.


Im­age Source: As­so­ci­at­ed Press

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