Ex­perts talk IRA im­pacts as CMS an­nounces it will pub­lish first 10 drugs up for ne­go­ti­a­tion in Sep­tem­ber

Ex­perts went back and forth on the im­pacts of the In­fla­tion Re­duc­tion Act, or IRA, on the bio­phar­ma in­dus­try in a vir­tu­al pan­el host­ed by End­points News at JP Mor­gan Wednes­day.

The ma­jor drug pric­ing re­form passed last year and al­lowed Medicare drug price ne­go­ti­a­tions for the first time ever. It al­so capped se­nior drug ex­pens­es at $2,000 a year and in­sulin costs at $35 a month.

Chiq­ui­ta Brooks-La­Sure

Wednes­day morn­ing, the Cen­ters for Medicare & Med­ic­aid Ser­vices (CMS) an­nounced its time­line for ne­go­ti­a­tions. In a me­dia call, CMS ad­min­is­tra­tor Chiq­ui­ta Brooks-La­Sure said, “Our ac­tions this year will cul­mi­nate in the an­nounce­ment of a list of 10 part D, high-spend brand name drugs to par­tic­i­pate in the ne­go­ti­a­tion pro­gram.”

CMS plans to pub­lish that list in Sep­tem­ber. Af­ter that, it will ne­go­ti­ate with drug man­u­fac­tur­ers, and in Sep­tem­ber 2024, CMS ex­pects to pub­lish its max­i­mum fair prices for said drugs, which will be ef­fec­tive in 2026.

Ex­perts on End­points’ IRA pan­el not­ed that they were watch­ing what max­i­mum fair prices will look like. Man­att Health se­nior ad­vi­sor Ian Spatz said, “Are we cut­ting off the en­tire tail of rev­enue af­ter nine years for a small mol­e­cule drug and 13 for a bi­o­log­ic? We don’t know that to­day be­cause we don’t know what a max­i­mum fair price is go­ing to be.” Un­der the IRA, new small mol­e­cule drugs will have nine years be­fore they can be sold at a CMS-ne­go­ti­at­ed price, while bi­o­log­ics have 13 years.

Sta­cie Duset­z­i­na

No­tably, drug launch prices are un­touched by the act. Van­der­bilt Uni­ver­si­ty Med­ical Cen­ter health pol­i­cy pro­fes­sor Sta­cie Duset­z­i­na said, “This is a re­al­ly im­por­tant area that I think we’re all go­ing to be watch­ing, but com­pa­nies may be think­ing about if price in­creas­es lim­it­ed to in­fla­tion. Launch price is where I think we’ll see a lot of re­sponse from in­dus­try be­cause they think [they] may be se­lect­ed lat­er on.”

Bio­phar­ma com­pa­nies are al­ready cit­ing the IRA as a rea­son for stop­ping de­vel­op­ment on drugs. In Oc­to­ber, Al­ny­lam point­ed to the act when it dropped plans for a Phase III tri­al for its re­cent­ly ap­proved drug, vutrisir­an, in Star­gardt dis­ease, a rare ge­net­ic eye con­di­tion. Vutrisir­an, mar­ket­ed as Amvut­tra, is cur­rent­ly in­di­cat­ed for AT­TR with polyneu­ropa­thy. In No­vem­ber, Eli Lil­ly said it scrapped an ear­ly-stage can­cer can­di­date in re­ac­tion to the act as well.

Dur­ing the pan­el, Tufts Med­ical Cen­ter pro­fes­sor Dan Ol­len­dorf com­ment­ed that Al­ny­lam’s use of the IRA in this case “seems to be a bit of sub­terfuge.”

Chris Garabe­di­an

Xon­toge­ny CEO Chris Garabe­di­an com­ment­ed that biotech R&D teams would work “on how to dri­ve drug de­vel­op­ment to work around be­ing on that list that ul­ti­mate­ly gets picked for price ne­go­ti­a­tion.” Spatz not­ed a num­ber of strate­gies com­pa­nies may use, in­clud­ing start­ing with larg­er in­di­ca­tions in­stead of small­er ones, mak­ing one prod­uct com­pa­nies, and hav­ing mul­ti­ple prod­ucts that are on­ly slight­ly dif­fer­ent, which he dubbed “the break­fast ce­re­al strat­e­gy.”

ATI Ad­vi­so­ry’s An­na Kaltenboeck said what ex­act­ly de­fines a drug in ne­go­ti­a­tions will be im­por­tant. “How are they go­ing to ag­gre­gate dif­fer­ent for­mu­la­tions, dif­fer­ent ex­ten­sions of the same prod­uct or re­lat­ed prod­ucts in­to one in or­der to as­sess whether or not it meets the thresh­old for ne­go­ti­a­tion or qual­i­fies for it?” she said. “I think that’s an­oth­er one that’s go­ing to be very, very im­por­tant in terms of the po­ten­tial in­dus­try re­spons­es and de­ci­sions re­lat­ed to R&D in­vest­ment.”

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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