To be cost-ef­fec­tive, Bio­gen should slash Spin­raza price, and No­var­tis can­not jus­ti­fy a $4M-$5M price tag for Zol­gens­ma — ICER

As treat­ments for rare dis­eases and gene ther­a­pies make strides on the US mar­ket, the is­sue of pric­ing is once again cen­ter stage. Bio­gen should cut the price of its spinal mus­cu­lar at­ro­phy (SMA) treat­ment Spin­raza and the price for No­var­tis’ one-shot Zol­gens­ma — which is ex­pect­ed to win FDA ap­proval by May — is jus­ti­fi­able up to $1.5 mil­lion, Boston-based ICER con­clud­ed on Wednes­day.

Akin to NICE in the UK, ICER is an in­de­pen­dent body that an­a­lyzes the cost-ef­fec­tive­ness of drugs and oth­er med­ical ser­vices in the Unit­ed States. Un­like NICE, though, ICER is not gov­ern­ment-af­fil­i­at­ed, but its de­ter­mi­na­tions are in­creas­ing­ly be­com­ing in­flu­en­tial with pay­ers.

Spin­raza was ap­proved by the FDA amidst much fan­fare in 2016 as the first and on­ly dis­ease-mod­i­fy­ing treat­ment for SMA, a rare and but lead­ing ge­net­ic cause of in­fant deaths. But the price tag of $750,000 for the first year of ther­a­py (and a $375,000 there­after) sparked heavy crit­i­cism, even though many pay­ers even­tu­al­ly agreed to re­im­burse the treat­ment. It gen­er­at­ed $1.7 bil­lion in 2018 sales for Bio­gen.

No­var­tis’ Zol­gens­ma is cur­rent­ly un­der FDA re­view and the agency is ex­pect­ed to an­nounce its de­ci­sion in the com­ing months. The Swiss drug­mak­er has sug­gest­ed a price of $4 mil­lion for the gene re­place­ment ther­a­py, which it ac­quired via its $8.7 bil­lion takeover of AveX­is, may be jus­ti­fied.

David Rind

“(T)he cur­rent price of Spin­raza far ex­ceeds com­mon thresh­olds for cost-ef­fec­tive­ness. The price of Zol­gens­ma is not yet known, but there has been pub­lic dis­cus­sion of prices above com­mon­ly ac­cept­ed cost-ef­fec­tive­ness thresh­olds as well. These treat­ments will be cov­ered by US in­sur­ers re­gard­less of the pric­ing, but the rip­ple ef­fect of pric­ing de­ci­sions like these threat­ens the over­all af­ford­abil­i­ty and sus­tain­abil­i­ty of the US health sys­tem,” ICER’s chief med­ical of­fi­cer David Rind said in a state­ment.

In the ini­tial draft ICER rec­om­men­da­tions is­sued late last year, ICER had sug­gest­ed Zol­gens­ma — priced at $2 mil­lion — could be more cost-ef­fec­tive in the long run ver­sus Spin­raza.

On Wednes­day, ICER’s fi­nal re­port sug­gest­ed al­though Spin­raza’s price “should be far low­er than it is, and the price for Zol­gens­ma should be low­er than the hy­po­thet­i­cal $4-5 mil­lion price the man­u­fac­tur­er has sug­gest­ed could be jus­ti­fied.”

ICER con­duct­ed its analy­ses us­ing two mea­sures: 1) 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. 2) Life years gained (LYG), which ex­press­es the ad­di­tion­al num­ber of years of life that a per­son lives as a re­sult of re­ceiv­ing a treat­ment.

His­tor­i­cal­ly, life ex­pectan­cy in the most com­mon and se­vere form of SMA (type I) is less than two years. Sur­vival de­pends on res­pi­ra­to­ry func­tion, and many in­fants and chil­dren even­tu­al­ly re­quire per­ma­nent ven­ti­la­tion.

Us­ing the QALY mea­sure for cost-ef­fec­tive­ness, Spin­raza’s val­ue was as­sessed in the presymp­to­matic pop­u­la­tion, as there are da­ta sup­port­ing its ef­fec­tive­ness in these types of pa­tients. The drug’s price should be be­tween $72,000-$130,000 for the first year of treat­ment and be­tween $36,000-$65,000 for each suc­ces­sive year. Un­der the LYG bench­mark, Spin­raza would need to be priced be­tween $83,000-$145,000 dur­ing the ini­tial year and $41,000-$72,000 for each suc­ces­sive year, ICER said.

“As the re­port notes, there is a sig­nif­i­cant dif­fer­ence in ro­bust­ness and qual­i­ty of ev­i­dence for Spin­raza as com­pared to Zol­gens­ma. The analy­sis, how­ev­er, fails to ac­count for those dif­fer­ences. Spin­raza is the stan­dard of care in SMA and has ben­e­fit­ted the lives of more than 6,600 peo­ple. In con­trast, Zol­gens­ma is an ex­per­i­men­tal ther­a­py which has re­port­ed re­sults to date for on­ly 15 pa­tients fol­lowed for up to 2.5 years, sev­en of whom are re­port­ed to have sub­se­quent­ly ini­ti­at­ed treat­ment with Spin­raza,” a Bio­gen spokesper­son said in an emailed state­ment.

Mean­while, ICER did not vote on the long-term val­ue of Zol­gens­ma, as its fi­nal price is not yet known. It con­duct­ed its as­sess­ment for the SMA type I pop­u­la­tion as this is the on­ly pop­u­la­tion in which it has been clin­i­cal­ly eval­u­at­ed. Us­ing QALY, the ther­a­py’s price for type I SMA would need to be be­tween $310,000-$900,000 per shot, while un­der the LYG bench­mark it would need to be be­tween $710,000-$1.5 mil­lion per treat­ment, ICER con­clud­ed.

“The val­ue mea­sures and thresh­olds em­ployed by ICER in this re­port are de­signed around the sta­tus quo of chron­ic care man­age­ment and can­not pos­si­bly cap­ture the full ben­e­fits of dis­ease-mod­i­fy­ing treat­ments de­liv­ered as a one-time ad­min­is­tra­tion,” a No­var­tis spokesper­son said in an emailed state­ment.

ICER al­so rec­om­mend­ed pay­ers en­dorsed the con­cept of val­ue-based con­tracts, a sys­tem in which a re­im­burse­ment for a sub­stan­tial por­tion of the treat­ment is not passed on to the drug­mak­er, should pa­tients not re­ceive ad­e­quate clin­i­cal ben­e­fit.


Im­age: Shut­ter­stock

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Fed­er­al claims court rules in fa­vor of Gilead in CDC Tru­va­da patent case

Gilead pulled a win last week in its ongoing patent battle with the CDC over the HIV drug Truvada for pre-exposure prophylaxis (PrEP).

The US Court of Federal Claims ruled on Nov. 21 that the government breached certain agreements with Gilead by failing to promptly notify the company of its patent applications for Truvada for PrEP, according to documents unsealed on Wednesday.

The issue traces back to around 2004 when Truvada won an accelerated approval to treat HIV. Because HIV is known to develop resistance to therapy, patients at the time were often required to take more than one drug at a time. Truvada combines Gilead’s prior HIV drugs, Emtriva and Viread, making life easier for patients who were taking separate pills daily. Shortly after, the CDC and Gilead struck up a partnership to research Truvada’s use as a preventative measure prior to exposure.