No­var­tis' SMA gene-ther­a­py, if priced at $2M, could be more cost-ef­fec­tive than Bio­gen's Spin­raza — ICER

Bio­gen’s Spin­raza was ap­proved by the FDA amidst much fan­fare in 2016 as the first and on­ly dis­ease-mod­i­fy­ing treat­ment for SMA, a rare and of­ten fa­tal ge­net­ic mus­cu­lar dis­or­der. But the price tag of $750,000 for the first year of ther­a­py (and a low­er price there­after) prompt­ed heavy crit­i­cism, al­though many pay­ers even­tu­al­ly agreed to re­im­burse the treat­ment. How­ev­er, a re­port by the In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view (ICER) on Thurs­day has sug­gest­ed No­var­tis’ ex­per­i­men­tal SMA gene ther­a­py, Zol­gens­ma, could be more cost-ef­fec­tive in the long run ver­sus Spin­raza.

Zol­gens­ma is cur­rent­ly un­der FDA re­view and the agency is ex­pect­ed to an­nounce its de­ci­sion on the drug in May 2019. In its re­view, ICER has as­sumed Zol­gens­ma will car­ry a list price of $2 mil­lion, al­though the Swiss drug­mak­er has sug­gest­ed a price of $4 mil­lion for the cu­ra­tive ther­a­py, which it ac­quired via its $8.7 bil­lion takeover of AveX­is, may be jus­ti­fied.

De­spite the lack of long-term da­ta on ei­ther ther­a­py, the non-prof­it es­ti­mat­ed in­cre­men­tal cost-ef­fec­tive­ness of Spin­raza is $728,000 per QALY in presymp­to­matic SMA pa­tients, while Zol­gens­ma has an in­cre­men­tal cost-ef­fec­tive­ness of $247,000 per QALY in pa­tients with symp­to­matic Type I SMA (on the ba­sis of a place­hold­er price of $2 mil­lion).

QALYs, or qual­i­ty-ad­just­ed life-years, are a mea­sure of the state of health of a per­son or group in which the ben­e­fits — in terms of length of life — are ad­just­ed to re­flect the qual­i­ty of life. Es­sen­tial­ly, one QALY is equal to one year of life in per­fect health.

“No­var­tis has es­ti­mat­ed that Zol­gens­ma would yield 13.3 QALYs in SMA Type I pa­tients, and could be priced to $4-5 mil­lion based on the re­la­tion­ship of 10-year cost of ap­proved drugs for ul­tra-rare dis­eases and their cor­re­spond­ing in­cre­men­tal QALYs gained. ICER es­ti­mates Zol­gens­ma pro­vides 11.33 QALYs, and us­es a place­hold­er price of $2 mil­lion. While ICER un­der­cut both of No­var­tis’ es­ti­mates, the high cost/QALY of Spin­raza in Type I SMA pa­tients of­fers No­var­tis bar­gain­ing pow­er with pay­ers if the com­pa­ny can ar­gue Spin­raza use could be re­duced or elim­i­nat­ed to off­set to­tal costs,” Leerink an­a­lysts wrote in a note on Fri­day.

Cur­rent­ly avail­able da­ta on Spin­raza and Zol­gens­ma show pro­longed sur­vival and im­proved mo­tor func­tion com­pared with his­tor­i­cal con­trols or place­bo, but “there re­mains con­sid­er­able un­cer­tain­ty in the gen­er­al­iz­abil­i­ty of the re­sults and in the long-term dura­bil­i­ty and tol­er­a­bil­i­ty of treat­ment. In par­tic­u­lar, for both in­ter­ven­tions, the nar­row el­i­gi­bil­i­ty cri­te­ria of tri­als and the lim­it­ed sam­ple size (es­pe­cial­ly for Zol­gens­ma) rais­es con­cerns about gen­er­al­iz­abil­i­ty of re­sults to the wider pop­u­la­tion of pa­tients with SMA,” the re­port added.

Bio­gen un­der­scored this dif­fer­ence in sam­ple size in its re­sponse to the re­port. “The draft ICER re­port is an in­com­plete rep­re­sen­ta­tion of Spin­raza’s val­ue to pa­tients and health care sys­tems. Fur­ther­more, to com­pare a treat­ment that has helped near­ly 6,000 pa­tients world­wide against an in­ves­ti­ga­tion­al treat­ment on da­ta from 15 pa­tients with an ar­ti­fi­cial­ly se­lect­ed price is mis­lead­ing and wrong,” a spokesper­son told End­points News.

The ICER re­port is not fi­nal and has been opened to the pub­lic for com­ment, which will be in­cor­po­rat­ed in­to an ev­i­dence re­port in Feb­ru­ary 2019. This re­port will be sub­ject to fur­ther de­lib­er­a­tion by one of ICER’s in­de­pen­dent ev­i­dence ap­praisal com­mit­tees in ear­ly March, af­ter which a fi­nal re­port will be re­leased by the end of the month.

“If con­firmed in a fi­nal re­port…these find­ings could of­fer sup­port for No­var­tis in pric­ing and ac­cess ne­go­ti­a­tions for Zol­gens­ma ahead of 2019 ap­proval,” Leerink an­a­lysts added.

In re­sponse to the re­port, No­var­tis said that the find­ings had af­firmed the com­pa­ny’s ini­tial as­sess­ment of the val­ue of their prod­uct. “While we have not yet de­ter­mined the price of our in­ves­ti­ga­tion­al prod­uct…once we de­ter­mine a price, we are com­mit­ted to flex­i­bly part­ner­ing with health­care stake­hold­ers to en­sure ac­cess,” a spokesper­son told End­points News. 

Like NICE in the UK, ICER an­a­lyzes the ev­i­dence on the ef­fec­tive­ness and val­ue of drugs and oth­er med­ical ser­vices in the Unit­ed States, how­ev­er un­like NICE, it is not a gov­ern­ment-af­fil­i­at­ed body.

In Au­gust, NICE re­fused to rec­om­mend the use of Spin­raza in the UK — de­spite Bio­gen dis­count­ing its price — on the ba­sis that the long-term im­pact of the drug was un­clear for pa­tients, some of whom die by the age of 2. With­out that da­ta, the price of the drug couldn’t be jus­ti­fied, they said.

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