An­timi­cro­bial re­sis­tance: FDA dis­cuss­es re­im­burse­ment re­forms

Re­im­burse­ment re­forms for an­timi­cro­bial treat­ments could in­clude “a mix of mile­stone pay­ments and sub­scrip­tion fees for de­vel­op­ers of FDA-ap­proved prod­ucts with high eco­nom­ic and clin­i­cal val­ue, tar­get­ed at mul­ti-drug re­sis­tant or­gan­isms and linked to proven clin­i­cal out­comes,” FDA Com­mis­sion­er Scott Got­tlieb said in a speech at Pew Char­i­ta­ble Trusts on Fri­day.

He al­so of­fered the idea of us­ing a “sub­scrip­tion-based mod­el” that could see hos­pi­tals pay­ing a flat rate for ac­cess to a cer­tain num­ber of dos­es of a new an­timi­cro­bial.

Cur­rent­ly, the Cen­ters for Dis­ease Con­trol and Pre­ven­tion (CDC) es­ti­mates that the di­rect cost of an­timi­cro­bial re­sis­tance on the US econ­o­my is $20 bil­lion an­nu­al­ly.

“These sub­scrip­tion fees could be priced at a lev­el to cre­ate a suf­fi­cient re­turn on the in­vest­ment to de­vel­op drugs with a cer­tain pro­file,” Got­tlieb said. “This should have the ef­fect of cre­at­ing a nat­ur­al mar­ket for drugs that meet cer­tain im­por­tant spec­i­fi­ca­tions.”

The FDA is al­so dis­cussing pay­ment pi­lots with oth­er fed­er­al agen­cies, in­clud­ing the Cen­ters for Medicare and Med­ic­aid Ser­vices (CMS), not­ing in­ter­est in do­ing such pi­lots in con­cert with the pri­vate sec­tor as well.

New ap­proach­es to re­im­burse­ment that could be ex­plored might “al­so in­clude new tech­nol­o­gy add-on pay­ments for cer­tain new an­tibac­te­r­i­al drugs that meet crit­i­cal pa­tient and pub­lic health needs,” he said.

Mean­while, ear­li­er this week, the FDA re­leased a new re­quest for in­for­ma­tion (RFI) to ob­tain ex­ter­nal in­put in­to de­vel­op­ing an an­nu­al list of reg­u­la­to­ry sci­ence ini­tia­tives specif­i­cal­ly for an­timi­cro­bial prod­ucts. Part of such a list will be a fo­cus on the “de­lay in avail­abil­i­ty of in­for­ma­tion re­gard­ing the iden­ti­fi­ca­tion and an­tibac­te­r­i­al sus­cep­ti­bil­i­ty of the causal pathogen, when iso­lat­ed.”

Re­spons­es to the RFI will be ac­cept­ed un­til 31 Oc­to­ber 2018.


Im­age: Scott Got­tlieb. SHUT­TER­STOCK

First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a potential EUA application, Moderna announced they have completed enrollment in their 30,000-person Phase III Covid-19 vaccine trial, with over a third of volunteers non-white and a quarter over the age of 65.

The announcement caps what has been the most closely-watched recruitment race in the history of drug development, as Pfizer and Moderna rushed to get enough volunteers to prove whether or not experimental vaccines could actually protect people from contracting Covid-19. Pfizer reached that mark on Sept. 15. Moderna said around the same time that they would slow down enrollment to ensure they enrolled enough participants from minority and at-risk groups.

Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

With lumasir­an on the FDA's doorstep, Al­ny­lam reads out new PhI­II da­ta in PH1

Just over a month away from its December PDUFA date, Alnylam flaunted new data from two Phase III studies to back lumasiran in primary hyperoxaluria type 1 (PH1), a rare liver condition.

The Cambridge, MA-based biotech snagged a priority review for the candidate back in June, and got positive feedback from the EMA’s Committee for Medicinal Products for Human Use just last week. Lumasiran uses RNA interference (RNAi) to silence the gene for glycolate oxidase, an enzyme used in the production of oxalate.

News brief­ing: UK biotech 4D phar­ma heads for Nas­daq via SPAC; Dr. Red­dy's shuts down man­u­fac­tur­ing af­ter cy­ber­at­tack

Another pharma company is intending to use a SPAC to join the Nasdaq.

4D pharma, a UK-based biotech, is reverse-merging with a blank check company in a deal worth up to $37.6 million. The move will give 4D pharma a new Nasdaq ticker, which will be $LBPS, using the American Depositary Share program.

As a result of the move, 4D pharma will gain $14.6 million in cash held by the blank check company, dubbed Longevity. The merger is expected to be completed in early 2021, after which shares will be immediately tradeable.

Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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HHS secretary Alex Azar (at the podium) and FDA commissioner Stephen Hahn (Pete Marovich/Getty Images)

Covid-19 roundup: Azar open­ly plan­ning Hahn ouster — re­port; Vul­ner­a­ble pop­u­la­tions like­ly to get vac­cines by Jan­u­ary

The relationship between HHS secretary Alex Azar and FDA commissioner Stephen Hahn has deteriorated to the point where Azar has suggested replacing Hahn, according to a Politico report.

Azar was angered by the FDA’s pushback of the Trump administration’s proposals for authorizing Covid-19 vaccines, so much so that he began openly floating potential replacements for Hahn. The report cited six unnamed sources that said Azar discussed bringing up Hahn’s removal to the White House.

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