CMS to work close­ly with FDA on ac­cel­er­at­ed ap­proval pay­ment re­forms

The FDA has his­tor­i­cal­ly not dealt with drug pric­ing, un­less it’s re­lat­ed to sav­ings from gener­ic drug com­pe­ti­tion.

But that’s slow­ly chang­ing now, with CMS say­ing it will work close­ly with the US drug reg­u­la­tor on both a new pi­lot project to try to save the fed­er­al gov­ern­ment on ac­cel­er­at­ed ap­proval drugs, and on im­ple­ment­ing the In­fla­tion Re­duc­tion Act, which will es­tab­lish new gov­ern­ment price ne­go­ti­a­tions.

“CMS is still ex­plor­ing the spe­cif­ic ap­proach­es to pay­ment ad­just­ments in the Ac­cel­er­at­ing Clin­i­cal Ev­i­dence Mod­el, and any ap­proach will be de­vel­oped in close co­or­di­na­tion with the Food and Drug Ad­min­is­tra­tion,” a CMS spokesper­son told End­points News via email.

That close co­or­di­na­tion be­tween FDA and CMS on the ac­cel­er­at­ed ap­proval pi­lot will be key, as in­dus­try in­sid­ers pre­dict drug­mak­ers will pull out all the stops to dodge what may be a ma­jor im­ped­i­ment to a big source of in­dus­try rev­enue, es­pe­cial­ly for small­er com­pa­nies with on­ly a few as­sets.

The move to en­gage the pi­lot rep­re­sents a big shift in CMS and the fed­er­al gov­ern­ment’s think­ing.

Phar­ma com­pa­nies some­times drag their feet on these con­fir­ma­to­ry tri­als, re­quired as part of the ac­cel­er­at­ed ap­provals, know­ing that pa­tients and pay­ers are still pay­ing full price even with­out the com­plete set of clin­i­cal ev­i­dence the agency typ­i­cal­ly re­quires for a full ap­proval. But Con­gress and Pres­i­dent Joe Biden re­cent­ly signed off on new au­thor­i­ty for the FDA to be­gin re­quir­ing these con­fir­ma­to­ry tri­als pri­or to the ap­proval.

The ques­tion that CMS’ In­no­va­tion Cen­ter now needs to an­swer with its pi­lot is: Do re­duced pay­ments for ac­cel­er­at­ed ap­provals ac­tu­al­ly speed up con­fir­ma­to­ry tri­als, fa­cil­i­tate ear­li­er with­drawals of drugs that don’t prove to work, or lead to few­er pay­ments from CMS for drugs that end up not work­ing?

HHS’ in­spec­tor gen­er­al said in a Sep­tem­ber re­port that Medicare and Med­ic­aid spent more than $18 bil­lion from 2018 to 2021 for 18 drugs (or 35 drug ap­pli­ca­tions grant­ed ac­cel­er­at­ed ap­proval) with in­com­plete con­fir­ma­to­ry tri­als that are past their orig­i­nal planned com­ple­tion dates.

And what’s to stop phar­ma com­pa­nies from just fac­tor­ing in this re­duced pay­ment in­to the ini­tial launch price? Not much, ex­perts say.

An­na Kaltenboeck, who leads the pre­scrip­tion drug re­im­burse­ment group at health re­search firm ATI Ad­vi­so­ry, ex­plained via email that how phar­ma tries to game the sys­tem “will de­pend on the price bench­mark that de­ter­mines pay­ment un­der the de­mo,” adding:

If the mod­el ap­plies a dis­count to that amount, then man­u­fac­tur­ers could cer­tain­ly fac­tor that in­to launch prices for prod­ucts that haven’t come to the mar­ket yet. (Drugs that are al­ready on the mar­ket and re­im­bursed un­der Part B may find it hard­er to raise list prices dra­mat­i­cal­ly on short no­tice. It takes 6 months for high­er prices to be re­flect­ed in Medicare’s pay­ment rate to physi­cians, who of­ten still have to pay the high­er price at ac­qui­si­tion in the mean­time.)

An­na Kaltenboeck

But there are oth­er ways CMS could po­si­tion the ac­cel­er­at­ed ap­proval pi­lot so high­er launch prices won’t help.

For in­stance, if CMS us­es a bench­mark for pay­ment that man­u­fac­tur­ers don’t con­trol, Kaltenboeck notes, then “a high launch price strat­e­gy won’t work. There are sev­er­al ways to do that. For ex­am­ple, CMS could use an av­er­age of pay­ments for oth­er drugs in a giv­en ther­a­peu­tic class, known as ‘ther­a­peu­tic ref­er­ence pric­ing.’ An­oth­er ex­am­ple is what the Trump ad­min­is­tra­tion pro­posed a much broad­er de­mo in Part B, which was to make use of in­ter­na­tion­al prices.”

What­ev­er the so­lu­tion, it may be more com­pli­cat­ed than it seems, par­tic­u­lar­ly as CMS has al­ready raised con­cerns about chang­ing dif­fer­ing prices for drugs that have won ac­cel­er­at­ed ap­provals for cer­tain in­di­ca­tions but full ap­provals for oth­ers.

Rachel Sachs

Rachel Sachs, a law pro­fes­sor at Wash­ing­ton Uni­ver­si­ty in St. Louis, point­ed out to End­points that CMS won’t be “forc­ing com­pa­nies to charge less — this is a change in re­im­burse­ment to providers. Com­pa­nies can charge what they want. Providers may not be will­ing to pre­scribe.”

But as to con­cerns that prices for some of these ac­cel­er­at­ed ap­provals would po­ten­tial­ly fall so low that some physi­cians will stop us­ing these Part B drugs, Kaltenboeck said she thinks “that is avoid­able,” adding:

The re­port stat­ed clear­ly that any mod­el would seek to avoid pe­nal­iz­ing pa­tients and providers. Past demos have ex­per­i­ment­ed with ways to make providers whole with­out re­ly­ing on markups; Med­Pac has point­ed to ways to do this as well.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”