In an un­usu­al move, ICER re­scinds draft re­port on JAK drugs for rheuma­toid arthri­tis to re­vise its mod­el­ing ap­proach

Two years ago, the In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view (ICER) ad­mon­ished Ab­b­Vie for over­pric­ing the best-sell­ing drug in the world — Hu­mi­ra — along with the raft of ri­val drugs ap­proved for rheuma­toid arthri­tis. On Wednes­day, the in­flu­en­tial cost-ef­fec­tive­ness watch­dog re­scind­ed its draft re­port on the new crop of JAK in­hibitors, say­ing it was re­vis­it­ing the mod­el­ing ap­proach it em­ployed in the analy­sis pub­lished in late Sep­tem­ber.

The re­view en­com­passed Eli Lil­ly’s baric­i­tinib (brand­ed as Olu­mi­ant), Pfiz­er’s to­fac­i­tinib (sold as Xel­janz) and Ab­b­Vie’s re­cent­ly ap­proved upadac­i­tinib (chris­tened Rin­voq).

In the draft re­port, pub­lished Sep­tem­ber 26, ICER sought to eval­u­ate the cost-ef­fec­tive­ness of the three JAK in­hibitors ver­sus the es­tab­lished an­ti-TNF Hu­mi­ra (known chem­i­cal­ly as adal­i­mum­ab) — but found it could on­ly make the com­par­i­son with the new Ab­b­Vie treat­ment, due to a pauci­ty of com­para­tor da­ta on the oth­er two JAK treat­ments.

“Our base-case find­ings sug­gest that upadac­i­tinib pro­vides mar­gin­al clin­i­cal ben­e­fit in com­par­i­son to adal­i­mum­ab, at high­er costs. To­geth­er, these out­comes trans­late in­to cost-ef­fec­tive­ness es­ti­mates that ex­ceed com­mon­ly cit­ed cost-util­i­ty thresh­olds,” the re­view­ers wrote. “Re­sults from the in­di­rect mod­el­ing com­par­i­son of to­fac­i­tinib to adal­i­mum­ab sug­gest that for the mar­gin­al ben­e­fit to­fac­i­tinib of­fers, prices for this drug much high­er than the price of adal­i­mum­ab may not be jus­ti­fied.”

Then, on Wednes­day, ICER is­sued a brief state­ment sug­gest­ing it was reeval­u­at­ing the mod­el­ing ap­proach used in the draft ev­i­dence re­port. A new draft re­port will be pub­lished in the com­ing days, the agency said.

The un­usu­al move comes at a time when ICER is, in any case, re­assess­ing its meth­ods. In De­cem­ber, the in­sti­tute is ex­pect­ed to up­date the frame­work that un­der­pins its ev­i­dence re­ports on new drugs and oth­er health care in­ter­ven­tions. ICER last up­dat­ed its val­ue as­sess­ment frame­work in June 2017, and al­so cre­at­ed a fresh ap­proach to eval­u­at­ing ul­tra-rare or­phan drugs in No­vem­ber 2017.

In Ju­ly, the agency in its re­view of Duchenne mus­cu­lar dy­s­tro­phy treat­ments ac­knowl­edged it had used sig­nif­i­cant­ly high­er price es­ti­mates for two ther­a­pies in its mod­els — ver­sus the es­ti­mates used in their draft re­port in May. When queried by End­points News, ICER sug­gest­ed the num­bers used in the May re­port were in­cor­rect.

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 gain­ing trac­tion with pay­ers.

Each of the drugs re­viewed in the re­scind­ed re­port be­long to a fam­i­ly of med­i­cines called Janus ki­nase (JAK) in­hibitors — which have been plagued with safe­ty con­cerns. Named af­ter the two-faced Ro­man God Janus, the fam­i­ly con­sists of four en­zymes: JAK1, JAK2, JAK3 and TYK2, which are as­so­ci­at­ed with cy­tokine re­cep­tors on the sur­face of cells and form part of a path­way in­volved in in­flam­ma­to­ry and im­mune re­spons­es.

Pfiz­er’s JAK1/JAK3 in­hibitor — Xel­janz — has been blight­ed by reg­u­la­to­ry re­stric­tions af­ter the high­er dose of the block­buster drug was found to be as­so­ci­at­ed with the risk of blood clots and death. Lil­ly’s $LLY JAK1/JAK2 Olu­mi­ant, mean­while, was ini­tial­ly re­ject­ed by the US agency due to safe­ty con­cerns — on­ly to even­tu­al­ly se­cure ap­proval for the low­er dose. Lil­ly’s part­ner, In­cyte $IN­CY, elect­ed to walk away from co-fund­ing the drug’s de­vel­op­ment as fears about the ben­e­fit-risk pro­file of the class of drugs ac­cu­mu­lat­ed. In Au­gust, Ab­b­Vie’s $AB­BV JAK1 upadac­i­tinib was cleared by the FDA for rheuma­toid arthri­tis with a black box warn­ing.

Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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Sean Parker, AP

Sean Park­er helps cre­ate a CRISPRed cell ther­a­py 2.0 play — and he’s got a high-pro­file set of lead­ers on the team

You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. It’s another appealing Bay Area group that’s attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.

Today it’s Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenberg’s new venture investment operation based in LA.
Drazan — a J&J Innovation vet with a long record of entrepreneurial endeavors — exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasn’t long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.