New study of FDA cites the flaws be­hind rapid-fire can­cer drug OKs

Richard Padzur

Over the past few years the FDA’s on­col­o­gy group un­der Richard Paz­dur has tru­ly rev­o­lu­tion­ized the way can­cer drugs are de­vel­oped and re­viewed for mar­ket­ing ap­proval. In or­der to ramp up the ar­rival of new drugs, reg­u­la­tors waived old rules on over­all sur­vival, flag­ging their will­ing­ness to carve years out of the R&D process in or­der to field new ther­a­pies.

It was a straight up trade off. The FDA was will­ing to ac­cept pre­lim­i­nary ev­i­dence of ef­fi­ca­cy and safe­ty and de­lay con­fir­ma­to­ry stud­ies un­til af­ter a drug was on the mar­ket. And pa­tient groups have cheered the FDA on at every turn.

But there is an­oth­er per­spec­tive on how this is all play­ing out, now that we’re a few years in­to the brand new game of can­cer R&D.

A group of re­searchers, in­clud­ing some fa­mil­iar FDA crit­ics, took a look at 22 drugs that were giv­en an ac­cel­er­at­ed ap­proval. Three years af­ter ap­proval, on­ly half of the 38 con­fir­ma­to­ry stud­ies re­quired by the FDA had been com­plet­ed, and most still used end­points that had strayed far from the old gold stan­dard on ran­dom­ized sur­vival re­sults.

Huseyin Naci

The study, pub­lished by the Jour­nal of the Amer­i­can Med­ical As­so­ci­a­tion, was done by Huseyin Naci, Kate­lyn Smal­l­ey and Aaron Kessel­heim, a pol­i­cy ex­pert at Har­vard who’s been re­peat­ed­ly crit­i­cal of the FDA’s ap­proval process.

“Drugs grant­ed Ac­cel­er­at­ed Ap­proval should be rig­or­ous­ly eval­u­at­ed us­ing con­vinc­ing pa­tient-cen­tered clin­i­cal out­comes in rig­or­ous stud­ies,” Naci told Reuters. But, he added, “we have found nu­mer­ous sit­u­a­tions in which re­quired con­fir­ma­to­ry stud­ies with rig­or­ous de­signs and out­comes are not pur­sued or are not com­plet­ed in a time­ly fash­ion, and in these cas­es, we are con­cerned that reg­u­la­tors ap­pear to ac­cept da­ta that would not oth­er­wise meet FDA stan­dards.”

Don’t look for any sud­den tough­en­ing of can­cer R&D stan­dards at the FDA. As Scott Got­tlieb was un­der re­view for the top job at the agency, he her­ald­ed the can­cer group’s work in find­ing new ways to ap­prove on­col­o­gy drugs faster than ever be­fore, a game-chang­er in the field made pos­si­ble for drugs that are typ­i­cal­ly ini­tial­ly used as a last re­sort among drug-re­sis­tant, dy­ing pa­tients.

The em­pha­sis at the FDA is to make the rest of the agency more like the on­col­o­gy or­ga­ni­za­tion.

Michel Younatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Alex­ion cre­ates new post for chief di­ver­si­ty of­fi­cer; Bar­ry Greene step­ping down at Al­ny­lam, Yvonne Green­street named as suc­ces­sor

Alexion has carved out a new position for chief diversity officer and filled it with an inside promotion.

Uzair Qadeer will now be responsible for their “diversity, inclusion and belonging” strategy, looking to reshape the biotech’s corporate culture. A veteran of Deloitte and Bristol Myers Squibb, Qadeer was working on executive coaching and helping create the diversity program he now leads.

President Trump (AP Images)

FDA takes the lead on defin­ing es­sen­tial un­der Trump's 'Buy Amer­i­can' ex­ec­u­tive or­der — as in­dus­try warns of sup­ply chain dis­rup­tion

President Donald Trump has signed an executive order detailing how the federal government should help on-shore drug manufacturing — and the FDA will play a central role.

The agency now has three months to draw up the list of “essential medicines, medical countermeasures, and their critical inputs” that the US must have available at all times. Various departments and agencies are then directed to buy these drugs and their ingredients from American manufacturers.

Covid-19 roundup: Pfiz­er teams with Gilead on remde­si­ivr; Japan, Brazil, Switzer­land, In­dia get vac­cines

Pfizer has joined the list companies helping Gilead manufacture remdesivir. The pharma giant announced today they signed a multi-year agreement to provide Gilead with contract manufacturing services at their McPherson, Kansas plant. The deal is part of a broad effort by Gilead to scale up the drug, the only currently authorized therapy for Covid-19, to 2 million doses this year.

That effort now includes 40 different companies on 3 continents, according to a press release the biotech put out yesterday, not including the generic drugmakers the company has allowed to produce the anti-viral for low and middle-income countries. Dozens of state governments, though, have said those efforts have not been extensive enough to keep up with demand and have called upon the federal government to sidestep Gielad’s patents and begin scaling the drug itself.  – Jason Mast

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In sur­pris­ing set­back, com­bo of Roche’s Tecen­triq and chemo fails to help pa­tients with triple-neg­a­tive breast can­cer

Roche broke ground last year when they secured the first FDA approval for a checkpoint therapy in triple-negative breast cancer, a notoriously difficult-to-treat indication that has been passed over by the wave of targeted therapies.

Now, though, doctors are puzzling over why a combination of drugs meant to make that therapy more potent instead appeared to make it less effective.

Roche said Thursday that in a Phase III trial, combining their PD-1/L1 checkpoint therapy Tecentriq with the chemotherapy paclitaxel, did not significantly improve progression-free survival for patients with locally advanced or metastatic triple-negative breast cancer over giving those patients chemotherapy alone. In fact, patients on the Tecentriq-chemo arm had lower overall survival than patients on chemo, although the drugmaker cautioned that the trial was not powered for that endpoint and the data were immature.

President Trump speaks with members of the media before boarding Marine One (AP Images)

'Oc­to­ber is com­ing,' and every­one still wants to know if a Covid-19 vac­cine will be whisked through the FDA ahead of the elec­tion

Right on the heels of a lengthy assurance from FDA commissioner Stephen Hahn that the agency will not rush through a quick approval for a Covid-19 vaccine, the President of the United States has some thoughts on timing he’d like to share.

In an exchange with Fox News’ Geraldo Rivera on Thursday, President Trump allowed that a vaccine could be ready to roll “sooner than the end of the year, could be much sooner.”

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Donald Trump, AP

Trump reach­es for his pen as long-await­ed ex­ec­u­tive or­der on drug man­u­fac­tur­ing on-shoring fi­nal­ly ar­rives — re­ports

President Trump will sign a long-rumored executive order later today that is designed to spur more made-in-the-USA therapies, according to a slate of new reports out Thursday morning.

Drug manufacturing circles have been buzzing about this EO for months now, wondering how Trump plans to require the on-shoring of certain therapies.

According to the Bloomberg report, White House trade adviser Peter Navarro told reporters that the administration will come up with a list of essential medicines Trump wants to have made inside the US. The order will direct government purchasers to buy these essential meds from US suppliers. The reports add that the order relaxes drug safety and environmental regulations to make it happen.

Yvonne Greenstreet, incoming Alnylam president (Alnylam)

Al­ny­lam pres­i­dent Bar­ry Greene leaves af­ter 17 years, hand­ing po­si­tion over to Yvonne Green­street as biotech looks to­ward prof­itabil­i­ty

After 17 years helping Alnylam steer control of buzzy but unproven science they promised could change medicine, president Barry Greene is leaving the RNAi biotech just as that technology is beginning to hit prime time.

Leaving to “pursue outside interests in the biopharmaceutical industry,” the longtime executive will hand over the reins on October 1 to current COO Yvonne Greenstreet. Greenstreet, a former Pfizer and GlaxoSmithKline executive, inherits the high-profile spot at a company that’s proven its tech can work in rare diseases but now faces the daunting task of turning a couple successes and a new mountain of cash into drugs that are broadly applicable and, crucially, profitable.

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