FDA rushed to ap­prove new drugs in 2019, some­times way ahead of sched­ule. Could that be dan­ger­ous?

While the ros­ter of 48 drugs (plus 4 no­table bi­o­log­ics) the FDA ap­proved in 2019 didn’t break any records with the sheer num­ber it was re­mark­able in one as­pect: The agency reached deep in­to a group of ther­a­pies they didn’t need to make a de­ci­sion on un­til lat­er this year, in some cas­es speed­ing drugs up by months ahead of even pri­or­i­ty re­view dead­lines.

On the sur­face, that’s great news for drug­mak­ers and the pa­tients they hope to help. But ac­cord­ing to a re­cent study on rushed ap­provals, it could al­so spell safe­ty trou­bles down the road.

Lau­ren Co­hen

Back in Ju­ly, a trio of econ­o­mists at NBER crunched the da­ta on drugs OK’d be­fore sup­posed dead­lines such as the end of months, years or be­fore hol­i­days and found that treat­ments ap­proved un­der these ap­par­ent “desk-clear­ing” ef­forts — in which reg­u­la­tors felt the pres­sure of in­for­mal per­for­mance bench­marks — are more like­ly to be as­so­ci­at­ed with post­mar­ket safe­ty is­sues.

“We see about twice as many ad­verse ef­fects,” Lau­ren Co­hen, a pro­fes­sor of fi­nance and en­tre­pre­neur­ial man­age­ment at Har­vard Busi­ness School and one of the au­thors, told the Wall Street Jour­nal.

Co­hen and his col­leagues, Umit Gu­run of the Uni­ver­si­ty of Texas at Dal­las and Danielle Li of MIT, con­sid­ered on­ly the surge of ap­provals in De­cem­ber, at the end of each month and be­fore hol­i­days such as Thanks­giv­ing.

In 2019 sev­en out of 48 nov­el chem­i­cal en­ti­ties were ap­proved in De­cem­ber, large­ly in line with the 15% pro­por­tion that the re­searchers not­ed in pre­vi­ous years. But no­tably, 21 OKs came through in Q4, ac­count­ing for 43% of the whole crop.

FDA spokesper­son Nathan Arnold ac­knowl­edged the De­cem­ber jump pat­tern but told the Jour­nal that its share has ac­tu­al­ly di­min­ished from the 1980s.

“While we can­not speak di­rect­ly to the re­sults on in­for­mal dead­lines high­light­ed in this study, FDA has—on mul­ti­ple oc­ca­sions—in­ves­ti­gat­ed a close­ly re­lat­ed is­sue, which is the re­la­tion­ship be­tween for­mal PDU­FA dead­lines and post­mar­ket safe­ty of drugs,” Arnold added. “We have not found ev­i­dence of such a re­la­tion­ship.”

Drugs that ap­peared to have been ap­proved with lit­tle heed of the PDU­FA dead­lines might be an­oth­er is­sue. Ver­tex’s Trikaf­ta (for cys­tic fi­bro­sis), BeiGene’s Brukin­sa (for man­tle cell lym­phoma), No­var­tis’ Adakveo and Glob­al Blood Ther­a­peu­tics’ Oxbry­ta (for sick­le cell dis­ease), as well as Al­ny­lam’s Givlaari (for acute he­pat­ic por­phyr­ia ) and As­traZeneca/Dai­ichi Sankyo’s En­her­tu (for HER+ breast can­cer) all be­longed to that group.

Co­hen, Gu­run and Li pre­scribed a so­lu­tion for the safe­ty con­cerns they spot­ted:

A po­ten­tial pol­i­cy re­sponse to the pat­terns we doc­u­ment is to more care­ful­ly scru­ti­nize drugs that are ap­proved dur­ing such out­put surges. Reg­u­la­tors could, for in­stance, re­view end-of-year de­ci­sions in the fol­low­ing year be­fore pro­vid­ing an ul­ti­mate ap­proval (or lack there­of).

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.