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).

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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FDA warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

Andy Plump, Takeda R&D chief (Jeff Rumans for Endpoints News)

What kind of PhI­Ib da­ta is worth $4B cash? Take­da’s Andy Plump has some thoughts on that

A few months back, when Takeda caused jaws to drop with its eye-watering $4 billion cash upfront for a mid-stage TYK2 drug from Nimbus, it had already taken a deep dive on the solid Phase IIb data Nimbus had assembled from its dose-ranging study in psoriasis.

Now, it’s rolling that data out, eager to demonstrate what inspired the global biopharma to go long in a neighboring, but new, disease arena for the pipeline. And the most avid students of the numbers will likely be at Bristol Myers Squibb, who will have a multi-year head start on pioneering the TYK2 space with Sotyktu (deucravacitinib) as Takeda makes its lunge for best-in-class status.

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No­vo Nordisk re­mains un­der UK scruti­ny as MHRA con­ducts its own re­view in 'in­cred­i­bly rare' case

The UK’s Medicines and Healthcare products Regulatory Agency is now reviewing Novo Nordisk’s marketing violation that resulted in its loss of UK trade group membership last week. Novo Nordisk was suspended on Thursday from the Association of the British Pharmaceutical Industry (ABPI) for two years after an investigation by its regulatory arm found the pharma broke its conduct rules.

MHRA said on Tuesday that its review of the Prescription Medicines Code of Practice Authority (PMCPA) investigation is standard practice. An MHRA spokesperson emphasized in an email to Endpoints News that the situation with Novo Nordisk is “incredibly rare” while also noting ABPI took “swift and proportionate action.”

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FTC says patent bat­tle over Parkin­son's drug could have 'sig­nif­i­cant im­pli­ca­tion­s' for pa­tients

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

Mar­ket­ingRx roundup: What could a US Tik­Tok ban mean for phar­ma? Pfiz­er, Lil­ly lead phar­ma March Mad­ness ad­ver­tis­ers

Just as pharma marketers finally make moves into TikTok, the threat of a US ban on the social media channel is now looming. Already banned on federal employee phones by an initial Congressional act, more bills and maybe bans are on the way. With rare bipartisan agreement, lawmakers have introduced legislation that would give the US president the power to ban TikTok (although not mentioned by name) and other foreign-owned technology platforms that represent a security threat to the US.

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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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