Scott Got­tlieb has some ideas about block­ing any more would-be Mar­tin Shkrelis from price goug­ing

FDA com­mis­sion­er Scott Got­tlieb wants to stop the next Mar­tin Shkre­li be­fore he, or she, gets start­ed on the next drug price goug­ing scan­dal. And shin­ing a bright light on the drugs most vul­ner­a­ble to pric­ing abuse will be one way to get start­ed.

In tes­ti­mo­ny be­fore the House ap­pro­pri­a­tions sub­com­mit­tee Thurs­day, the new com­mis­sion­er high­light­ed sev­er­al meth­ods that could pre­vent bio­phar­ma com­pa­nies from gam­ing the sys­tem, fo­cus­ing specif­i­cal­ly on “sit­u­a­tions where off-patent drugs lack an ap­proved gener­ic com­peti­tor.” Got­tlieb will get the FDA to pub­lish a list of drugs that are off patent “for which FDA has not ap­proved a sin­gle gener­ic ap­pli­cant.”

Scott Got­tlieb

Got­tlieb wants to flag the po­ten­tial for a re­peat of Shkre­li’s de­ci­sion to buy an old gener­ic, Dara­prim, and then rais­ing the price of the drug overnight by more than 5000% at his com­pa­ny, Tur­ing. Dara­prim had no gener­ic com­pe­ti­tion on the mar­ket, giv­ing Shkre­li a clear and le­gal path to jack­ing up the price.

There are var­i­ous ways to im­prove the gener­ic drug ap­proval process to in­crease com­pe­ti­tion and pre­vent play­ers from gam­ing prices, he says. But it’s clear­ly an is­sue he wants to tack­le ear­ly on.

“We do have sit­u­a­tions where spec­u­la­tors, for lack of a bet­ter word, can come in and buy a low vol­ume gener­ic, jack up the price know­ing that it will take po­ten­tial­ly years for gener­ic com­pe­ti­tion to come on to the mar­ket so they have that sort of an ex­clu­siv­i­ty pe­ri­od,” said the com­mis­sion­er.

Got­tlieb out­lined a three-prong plan to speed up gener­ic drug ap­provals and com­plete­ly elim­i­nate the back­log of ap­pli­ca­tions for knock­offs, hur­ry­ing along cheap­er al­ter­na­tives to brand ther­a­pies that could go a long way to re­duc­ing the coun­try’s spend­ing on phar­ma­ceu­ti­cals.

He told law­mak­ers that he plans to:

— “Cur­tail gam­ing by in­dus­try of our reg­u­la­tions which can ex­tend mo­nop­oly pe­ri­ods be­yond the time frame Con­gress in­tend­ed, hin­der­ing com­pe­ti­tion.”

— “Im­prove the process­es that en­able gener­ic ver­sions of com­plex drugs to be ap­proved for mar­ket­ing.”

— “In­crease the over­all ef­fi­cien­cy of the gener­ic drug re­view process while com­plete­ly elim­i­nat­ing the back­log of gener­ic ap­pli­ca­tions.”

The REMS pro­gram (risk eval­u­a­tion and mit­i­ga­tion strate­gies), he not­ed, has been used to slow gener­ic com­pe­ti­tion, and stream­lin­ing the process by waiv­ing the re­quire­ment that a gener­ic and brand man­u­fac­tur­er share a sin­gle sys­tem for as­sur­ing safe use could be used to elim­i­nate one tool used to slow gener­ics.

In the past, gener­ic man­u­fac­tur­ers have had to sue brand op­er­a­tions which re­fused to ne­go­ti­ate a shared safe­ty sys­tem as a way to block cheap­er com­pe­ti­tion.

Rep. Robert Ader­holt

Got­tlieb al­so sin­gled out the on­col­o­gy di­vi­sion at the FDA for em­brac­ing the break­through ther­a­py pro­gram, speed­ing along new ap­provals. Un­for­tu­nate­ly, he added, not every part of the agency does as well as the on­col­o­gy group, and he in­tends to bring the lag­gards up to speed. In ad­di­tion, he added, the FDA could do more by tak­ing a more pro­gres­sive view of some new tech­nolo­gies, like bio­mark­ers, in as­sess­ing drugs, look­ing at tri­al de­signs to find more ef­fi­cient ways to de­vel­op drugs and adding new tools for ther­a­peu­tic re­views.

It was im­me­di­ate­ly clear that the law­mak­ers of all stripes didn’t care for the Trump ad­min­is­tra­tion’s plans for the 2018 FDA bud­get.

De­moc­rats and Re­pub­li­cans alike wast­ed lit­tle time in crit­i­ciz­ing the Trump ad­min­is­tra­tion’s plan to dou­ble up on in­dus­try user fees while slash­ing the fed­er­al gov­ern­ment’s con­tri­bu­tion to the FDA’s bud­get.

“The agency’s chances of off­set­ting bud­get au­thor­i­ty with user fees face a very stiff up­hill bat­tle in the fu­ture,” not­ed Al­aba­ma Re­pub­li­can Robert Ader­holt. He al­so cit­ed the FDA’s rapid progress in hus­tling along new ap­provals of Keytru­da, which he should help, “uti­liz­ing the lat­est tech­nol­o­gy, make drugs ac­ces­si­ble be­fore a lengthy drug tri­al.”

One plus for the FDA: Got­tlieb not­ed that the FDA is no longer in the grip of a fed­er­al hir­ing freeze im­posed by Pres­i­dent Trump, ac­cord­ing to RAPS. The freeze, he told law­mak­ers, has been lift­ed.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

Robert Spurr, President Salix Pharmaceuticals

Bausch Health’s Sal­ix pi­lots study to shine light on chron­ic liv­er dis­ease and push back on stereo­types

October is both breast cancer awareness and liver disease awareness month. While there’s no doubt which condition draws more attention during the month, Salix wants to change that.

Salix, Bausch Health’s gastroenterology arm, piloted its first chronic liver disease report and physician survey with results out this week aimed at raising awareness and dispelling stereotypes.

While 4.5 million people have chronic liver disease or cirrhosis – which is even more than 3.8 million women diagnosed with breast cancer – the research found chronic liver disease “has not received the attention or level of effort needed for adequate prevention, diagnosis, and standardization of its management.”

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Katie Fanning, Mozart Therapeutics CEO

Mozart Ther­a­peu­tics makes its of­fi­cial de­but, jump­ing in­to the hot Treg R&D field with some big-name in­vestors back­ing it

Treg cells have been getting more and more attention recently among autoimmune specialists. There’s been Jeff Bluestone’s Sonoma, the $157 million launch of GentiBio this summer and Egle Therapeutics — which launched just last week — to name a few.

Now, there’s a new Treg player jumping in that wants to distinguish itself in the market: Mozart Therapeutics. Today, the biotech is emerging from stealth in its official debut with a $55 million Series A — with a bunch of A-list Big Pharma names on board a syndicate led by ARCH.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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