Step­ping in­to the pric­ing de­bate, FDA chief Scott Got­tlieb pro­pos­es new re­im­burse­ment idea for an­tibi­otics

Scott Got­tlieb

In an at­tempt to make good on its promise to tack­le the su­per­bug prob­lem, the FDA trot­ted out a new idea to­day that it hopes will ad­dress the re­im­burse­ment catch-22 in­her­ent in the in­dus­try. Their idea? Treat drugs like soft­ware, hav­ing hos­pi­tals buy li­cens­es to an­tibi­otics in­stead of re­im­burs­ing on a per-use ba­sis.

Let’s set aside, for the mo­ment, that pric­ing is typ­i­cal­ly not a top­ic the FDA likes to weigh in on. Af­ter all, drug pric­ing is not un­der FDA’s di­rect purview and would re­quire some in­ter-agency col­lab­o­ra­tion with the likes of CMS. But wor­ries around the pace of in­no­va­tion in an­tibi­otics and oth­er an­timi­cro­bial drugs ap­pear to be dri­ving the FDA to ac­tion.

Brent Ahrens

In the agency’s state­ment Tues­day, FDA com­mis­sion­er Scott Got­tlieb ar­gued that the cur­rent drug re­im­burse­ment mod­el isn’t con­ducive to an­tibi­otics R&D. The more an­tibi­otics a physi­cian pre­scribes, the more mon­ey the drug­mak­er earns. But that’s a prob­lem con­sid­er­ing a grow­ing ef­fort to scale back the use of an­timi­cro­bials.

“When such drugs be­come avail­able, we try to use them spar­ing­ly, lest pathogens be­come over-ex­posed to a new mech­a­nism of at­tack and de­vel­op re­sis­tance to it,” Got­tlieb wrote. “So, providers have im­posed un­der­stand­able re­stric­tions on the use of such drugs. While this rep­re­sents re­spon­si­ble stew­ard­ship, it al­so means that a nov­el an­tibi­ot­ic may have a very lim­it­ed mar­ket. If prod­uct de­vel­op­ers know that they will not be able to re­coup their in­vest­ments, there may be re­duced in­cen­tive to in­vest the sig­nif­i­cant mon­ey need­ed to dis­cov­er and de­vel­op such a drug.”

Brent Ahrens, a Canaan part­ner with a long his­to­ry back­ing an­tibi­otics mak­ers like Iterum and Du­ra­ta, said so-called “ef­fec­tive stew­ard­ship” of these drugs is se­ri­ous­ly af­fect­ing ROI in this field.

“If one were to look at the launch­es of the last sev­er­al an­tibi­otics — or some of the ac­qui­si­tions done — very few have done well over the past 15 years,” Ahrens said. “All of that is re­lat­ed to stew­ard­ship and the price point. The in­cen­tive for us to do this just falls apart.”

Jeff Stein

Un­der the FDA’s new idea for re­im­burse­ment, hos­pi­tals and oth­er care fa­cil­i­ties could be re­im­bursed for li­cens­es to an­timi­cro­bials rather than a per-use ba­sis. With­in this mod­el, the hos­pi­tal sys­tems would pay a fixed li­cens­ing fee for ac­cess to the drug, which would of­fer them the right to use a cer­tain num­ber of an­nu­al dos­es.

“This is sim­i­lar to the way that soft­ware of­ten gets re­im­bursed, where in­sti­tu­tions pay a li­cens­ing fee for a fixed num­ber of in­stal­la­tions,” Got­tlieb’s state­ment reads. “We have been speak­ing with our coun­ter­parts at CMS as to whether such an ap­proach is fea­si­ble, whether it can be for­mu­lat­ed as a demon­stra­tion, and as a demon­stra­tion, whether it would have the in­tend­ed pub­lic health ben­e­fits.”

The FDA hopes that such a mod­el would pro­vide a pre­dictable re­turn on in­vest­ment and rev­enue stream for drug­mak­ers. It would al­so put the in­sti­tu­tions ful­ly in charge of stew­ard­ship of the meds.

Jeff Stein, the CEO and pres­i­dent of clin­i­cal-stage an­tibi­otics mak­er Cidara, tells me it’s en­cour­ag­ing to see the FDA tack­le the prob­lem, but he does won­der if the idea would work for hos­pi­tals.

“The ques­tion is — would hos­pi­tals em­brace it?” Stein said. “Will they be will­ing to pay for a drug up­front for a lim­it­ed num­ber of pa­tients?”

Ahrens said he’s al­so skep­ti­cal about how that would play out.

“Hos­pi­tals are re­luc­tant to pay for any­thing — un­der­stand­ably so — and it’s chal­leng­ing get­ting new prod­ucts in a hos­pi­tal,” Ahrens said. Pay­ing a larg­er sum up­front — when there are low-cost op­tions al­ready avail­able — may be a tough sell to acute care fa­cil­i­ties.

Still, both Ahrens and Stein are hap­py to see the FDA take on the re­im­burse­ment chal­lenge, as both con­sid­er it the biggest is­sue fac­ing the space. In­cen­tive pro­grams like QIDP and oth­ers stem­ming from the GAIN act have helped to fu­el in­no­va­tion in the in­dus­try over the past few years, but if com­pa­nies — and in­vestors — don’t get paid for their ef­forts, the space could slow its ef­forts, they said.

“I’d hate to see a cycli­cal ef­fect,” Ahrens said. “But with­out bet­ter re­im­burse­ment, there just won’t be a lot of new de­vel­op­ment.”

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Image: Shutterstock

Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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As­traZeneca-Alex­ion merg­er slides through FTC re­view af­ter sup­posed M&A crack­down pos­es no bar­ri­ers

The AstraZeneca-Alexion megamerger received a good sign Friday, despite warning signs of the tides turning against large M&A pharma deals.

US regulators at the FTC have cleared the acquisition for approval, AstraZeneca announced, all but signing off on the deal to go through once it officially closes in the third quarter. AstraZeneca originally said it was planning to buy out Alexion back in December for $39 billion.

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J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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David Stack, Pacira Biosciences CEO

In high­ly un­usu­al move, Paci­ra sues med­ical jour­nal for li­bel over its non-opi­oid painkiller

A New Jersey biotech whose only approved drug is used as a painkiller after surgeries is suing a scientific journal, its editors and a handful of authors for libel after the publication printed numerous papers and editorials that the company says discredited the drug.

Pacira Biosciences filed the complaint against the American Society of Anesthesiologists in the US District Court for New Jersey on Wednesday afternoon. A February issue of the group’s journal Anesthesiology printed three articles and other content full of “bias” that “seriously disparaged” the drug Exparel, Pacira claimed.

Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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