When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US bio­phar­ma world, ICER is team­ing up with a high-pro­file com­pa­ny to in­te­grate re­al-world ev­i­dence in their as­sess­ment of treat­ment val­ue.

The drug pric­ing watch­dog — for­mal­ly the In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view — said it will uti­lize Ae­tion’s ev­i­dence plat­form in “se­lect up­com­ing as­sess­ments” and their new 24-month re-eval­u­a­tions of drugs grant­ed ac­cel­er­at­ed ap­proval by the FDA.

Even though ICER doesn’t play an of­fi­cial role in set­ting drug prices, its ver­dicts on whether treat­ments are cost-ef­fec­tive are in­creas­ing­ly shap­ing pay­er at­ti­tude and con­sumer per­cep­tion, as well as, gen­er­al dis­course as the heat­ed de­bate around US drug prices rages on.

Car­olyn Mag­ill

Led by Unit­ed­Health vet Car­olyn Mag­ill, Ae­tion de­rives much of its da­ta from in­sur­ance claims, but it al­so works with elec­tron­ic health records, reg­istries and clin­i­cal tri­als. The FDA and Brigham and Women’s Hos­pi­tal have a pact in place with the com­pa­ny to try pre­dict­ing the re­sults of ran­dom­ized clin­i­cal tri­als in a pi­lot project in­volv­ing sev­en stud­ies.

Scott Got­tlieb, who pushed the in­dus­try to try new things with RWE dur­ing his tenure as FDA com­mis­sion­er, joined Ae­tion’s board a few months af­ter leav­ing the agency.

David Whitrap

For its part, ICER has com­mit­ted to ex­pand­ing its RWE use, not on­ly us­ing ex­ist­ing sources such as man­u­fac­tur­ers and pa­tient or­ga­ni­za­tions but ex­plor­ing ways to gen­er­ate da­ta that can com­ple­ment pub­lished sources. The deal with Ae­tion al­lows them to do it at greater scale, David Whitrap, ICER’s VP of com­mu­ni­ca­tions and out­reach, told End­points News.

Where­as RWE can come in handy for com­par­isons to ex­ist­ing treat­ments and the nat­ur­al trend of cer­tain con­di­tions (such as sick­le cell dis­ease, where ICER is con­duct­ing a re­view of ther­a­pies with Ae­tion’s help), their re­la­tion­ship will be most use­ful in the new process they are pi­lot­ing: re-eval­u­at­ing treat­ments af­ter they’ve been on the mar­ket for sev­er­al years.

“Ran­dom­ized clin­i­cal tri­als re­main the gold stan­dard for de­vel­op­ing un­bi­ased ev­i­dence, but as the FDA is in­creas­ing­ly ap­prov­ing treat­ments based on thin­ner clin­i­cal tri­al da­ta, the en­tire health in­dus­try will need to fill larg­er knowl­edge gaps about how well a new drug ac­tu­al­ly works,” he said. “And RWE is one way to do so.”

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

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In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Ramy Farid, Schrödinger CEO (Schrödinger)

Bris­tol My­ers fronts new Schrödinger al­liance with $55M up­front, ex­pand­ing pre­ci­sion on­col­o­gy pro­file

Bristol Myers Squibb has a new R&D partner, one to which they’re paying a pretty penny to use their discovery platform.

The pharma company is doling out $55 million upfront to Schrödinger $SDGR to work on up to five small molecules, with the potential for $2.7 billion in milestone payments. Schrödinger’s initial targets include HIF-2 alpha and SOS1/KRAS for a type of kidney cancer and KRAS-driven cancers, respectively.