Re­pub­li­can sen­a­tors slam pro­posed ac­cel­er­at­ed ap­proval pay­ment re­forms

Re­pub­li­can sen­a­tors are speak­ing out against a pro­posed test mod­el in which drug­mak­ers would ef­fec­tive­ly make less for drugs ap­proved via the FDA’s ac­cel­er­at­ed ap­proval.

The test pro­gram, dubbed the Ac­cel­er­at­ing Clin­i­cal Ev­i­dence Mod­el, was an­nounced last month as a way to in­cen­tivize quick­er con­fir­ma­to­ry tri­als and “re­duce Medicare spend­ing on drugs that have no con­firmed clin­i­cal ben­e­fit.” HHS Sec­re­tary Xavier Be­cer­ra di­rect­ed CMS to be­gin con­sul­ta­tion with the FDA on the mod­el this year.

How­ev­er, 18 Re­pub­li­can sen­a­tors urged Be­cer­ra and CMS Ad­min­is­tra­tor Chiq­ui­ta Brooks-La­Sure not to pur­sue the mod­el in a let­ter on Wednes­day.

“By bring­ing ground­break­ing ther­a­pies to pa­tients years be­fore these prod­ucts could oth­er­wise reach the mar­ket, the FDA’s Ac­cel­er­at­ed Ap­proval Pro­gram (AAP) has saved count­less lives while up­hold­ing strong safe­guards and stan­dards,” they wrote. “The Ac­cel­er­at­ing Clin­i­cal Ev­i­dence Mod­el re­cent­ly an­nounced by the Biden Ad­min­is­tra­tion, un­for­tu­nate­ly, risks un­der­min­ing or even re­vers­ing this trend, trig­ger­ing ac­cess gaps for se­niors, as well as im­pos­ing cuts on front­line providers.”

In an Oc­to­ber re­port, HHS’ in­spec­tor gen­er­al said that Medicare and Med­ic­aid spent more than $18 bil­lion from 2018 to 2021 for ther­a­pies with in­com­plete con­fir­ma­to­ry tri­als past their orig­i­nal planned com­ple­tion dates.

“Giv­en the in­creas­ing num­ber of AAP ap­provals, some ex­perts are con­cerned the num­ber of past-due tri­als may con­tin­ue to in­crease,” the re­port states. Con­gress and Pres­i­dent Joe Biden re­cent­ly signed off on new au­thor­i­ty for the FDA to be­gin re­quir­ing con­fir­ma­to­ry tri­als pri­or to an ap­proval.

Last month, a CMS spokesper­son told End­points News that the agency 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.”

While HHS’ re­port claimed that any ad­just­ments would be made “in a man­ner that at­tempts to avoid pe­nal­iz­ing physi­cians or ben­e­fi­cia­ries for choos­ing (or avoid­ing) an ac­cel­er­at­ed ap­proval treat­ment,” the sen­a­tors ar­gued on Wednes­day that cer­tain con­fir­ma­to­ry tri­als may still take years to com­plete in dif­fi­cult dis­eases such as Alzheimer’s or some can­cers.

“Puni­tive treat­ment of prod­ucts that opt for ac­cel­er­at­ed ap­proval sends a pre­car­i­ous sig­nal to pa­tients, providers, pay­ers, and the pub­lic that CMS lacks con­fi­dence in this life-sav­ing path­way,” they wrote.

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

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

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.

Hugo Peris, Spiral Therapeutics CEO

Hear­ing-fo­cused biotech grabs trio of pro­grams from Oton­o­my's fire sale

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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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.”

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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Sally Susman, Pfizer EVP and chief corporate affairs officer

Q&A: Pfiz­er cor­po­rate com­mu­ni­ca­tions chief Sal­ly Sus­man dis­cuss­es book craft­ed in pan­dem­ic and per­son­al lessons

From the political arena to the finance and beauty industries to pharmaceuticals, Pfizer’s Sally Susman has broken barriers, stereotypes and conventions. And now the chief communicator is “Breaking Through,” the title of her first book about effective and innovative communications launching today. The full official title is “Breaking Through: Communicating to Open Minds, Move Hearts, and Change the World.”

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