Some wor­thy ini­tia­tives out of the FDA fall far short of an ef­fec­tive fed­er­al plan to rein in drug prices

Over the last five months I’ve spent more time watch­ing video streams out of the na­tion’s cap­i­tal than I did in all of the past five years (thank you C-Span). Law­mak­ers, over­all, have been very oblig­ing to the bio­phar­ma in­dus­try on a bi­par­ti­san ba­sis. And when there was ir­reg­u­lar news to re­port from DC, it gen­er­al­ly dealt with is­sues like an ex­tend­ed pe­ri­od of ex­clu­siv­i­ty for bi­o­log­ics or fresh de­mands for swifter drug re­views cou­pled to new in­cen­tives.

Iron­i­cal­ly, the drug in­dus­try proved one of the very few ar­eas where Re­pub­li­cans and De­moc­rats gen­er­al­ly found it easy to co­op­er­ate on a bi­par­ti­san ba­sis.

That all changed the day Don­ald Trump was sworn in as pres­i­dent.

Trump made pub­licly whip­ping bio­phar­ma a reg­u­lar spec­ta­cle. The in­dus­try had too many over­seas jobs, he de­clared with pop­ulist fer­vor, didn’t in­vest in Amer­i­ca and more than any­thing, en­gaged in rip­ping off the coun­try with out­ra­geous­ly high drug prices.

Phar­ma ex­ec­u­tives are a sen­si­tive sort when it comes to pric­ing dis­cus­sions in gen­er­al, and you’d be hard pressed to find any ma­jor play­er who isn’t painful­ly aware of the new pric­ing en­vi­ron­ment.

The big dis­count price on sar­ilum­ab from Sanofi and Re­gen­eron re­flects in part the im­pact Trump has had. Most of the biggest com­pa­nies have al­so come up with some new method for cal­cu­lat­ing ac­cept­able drug price in­creas­es. That didn’t hap­pen in a vac­u­um.

Ge­of­frey Porges, Leerink

So some in­dus­try in­sid­ers may have found it odd that Leerink’s Ge­of­frey Porges thinks that the whole Big Phar­ma as­sault from DC may well just pe­ter out, leav­ing a few scuff marks be­hind af­ter limbs had been threat­ened.

I was think­ing that when I watched FDA com­mis­sion­er Scott Got­tlieb tes­ti­fy in front of a Con­gres­sion­al sub­com­mit­tee on Thurs­day. Got­tlieb has a few good ideas on drug pric­ing. It’s sim­ple com­mon sense that clear­ing up a back­log of gener­ics ap­pli­ca­tions and specif­i­cal­ly tar­get­ing the ap­proval path­way for com­plex gener­ics can have a re­al ef­fect on what this coun­try pays for drugs. Gener­ic com­pe­ti­tion can shred a brand­ed price in months. And we’ll like­ly see biosim­i­lars start to have an im­pact af­ter we see a num­ber of ri­vals ap­pear for every ag­ing block­buster.

If that’s it — with no Medicare ne­go­ti­a­tions or tough fed­er­al for­mu­la­ries that can kick off the most ex­pen­sive meds — then the in­dus­try chiefs will have dodged a can­non ball. Bio­phar­ma can deal with the mar­ket re­al­i­ties of gener­ics. And aban­don­ing the end-game of pro­tect­ing fran­chise drugs with var­i­ous de­lay­ing tac­tics is in the pub­lic in­ter­est.

But that won’t ad­dress the un­der­ly­ing is­sue of brand­ed drug prices in the US.

FDA com­mis­sion­er Scott Got­tlieb

Ul­ti­mate­ly, mar­ket forces may be left as the on­ly vi­able tool for re­strict­ing new drug prices. That’s not nec­es­sar­i­ly a bad thing. But the way it works now cre­ates huge is­sues for pa­tients.

Left to their own de­vices, pay­ers have come up with their own way to de­ny the peo­ple they cov­er ac­cess to ex­pen­sive drugs. Step ther­a­py has be­come a 100 mile jour­ney for some pa­tients. Rules sup­pos­ed­ly aimed at re­strict­ing cov­er­age to those who can ben­e­fit from a drug are of­ten just drawn up to bar cov­er­age. Out-of-pock­et ex­pens­es keep go­ing high­er to wave off prop­er use.

US con­sumers should have ac­cess to new drugs at a fair price.

There has to be a bet­ter way than this to man­age the pric­ing is­sue. But we may not see it any­time soon. And that means the pric­ing con­tro­ver­sy will con­tin­ue to fes­ter in­def­i­nite­ly.

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.

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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As­traZeneca pub­lish­es Covid-19 vac­cine PhI­II pro­to­cols in lock­step with Mod­er­na and Pfiz­er. How are they dif­fer­ent?

Following in the steps of Moderna and Pfizer, the other two Western drugmakers currently in Phase III trials for their Covid-19 vaccines, AstraZeneca posted its own study protocols over the weekend in an effort to ensure transparency.

The move is the latest in a series of rare peeks behind the curtain, as such blueprints are typically shared once such trials are completed.

“Given the unprecedented global impact of the Coronavirus pandemic and the need for public information, AstraZeneca has published the detailed protocol and design of our AZD1222 clinical trial. As with most clinical development, protocols are not typically shared publicly due to the importance of maintaining confidentiality and integrity of trials. AstraZeneca continues to work with industry peers to ensure a consistent approach to sharing timely clinical trial information,” the company said in a statement.

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.

Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.