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.”

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Blueprint CEO Jeff Albers (file photo)

Blue­print plots re­turn to FDA with new Ay­vak­it da­ta in rare con­di­tion — and the an­a­lysts cheer

Over a decade after launch, Blueprint Medicines nabbed the first approval for their first drug earlier this year. Now, as they move forward with a Roche-partnered global launch, they’re touting data that could push them into more patients.

The Jeff Albers-led Cambridge biotech released their full pivotal data for Ayvakit in patients with advanced systemic mastocytosis. In one 53-person study, they showed that 76% of patients responded to the drug, 36% had complete responses and that on average their responses lasted for just over 3 years. A smaller, 32-patient study had a 75% response rate and most were still responding after 10.4 months, the last follow-up.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Sebastian Nijman (file photo)

Roche looks to ge­net­ic mod­i­fiers for new drug tar­gets, team­ing up with Dutch biotech in $375M deal

Roche is gambling on a new way of discovering drug targets and, ultimately, promising to infuse more than $375 million into a small biotech if all goes well.

A spinout of the Netherlands Cancer Institute and Oxford University, Scenic Biotech set out to pioneer a field that’s gaining some traction among top VCs in the US: to harness the natural protecting powers of genetic modifiers — specific genes that suppress a disease phenotype.