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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,200+ biopharma pros reading Endpoints daily — and it's free.

Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.

Per­pet­u­al cri­sis? Phar­ma com­mu­ni­ca­tions and pub­lic re­la­tions pros just wait for the next shoe to drop

Welcome to pharma public relations where every day feels like it might be a crisis.

More than three-fourths (76%) of pharma communications leaders expect to face three or more crises this year — compared to less than half (49%) of their peers in other industries, according to new survey from risk intelligence company Crisp. The same 76% also expect that a brand new risk that is yet to be identified will crop up this year, compared to 66% of peers across other industries who worry about the same thing.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,200+ biopharma pros reading Endpoints daily — and it's free.

As new C. diff treat­ments prep for mar­ket, spe­cial­ists di­vid­ed on 'poop in a pil­l' or small mol­e­cule, study finds

What do physicians think about the emerging market for recurrent C. diff? That depends on who you talk to, especially which specialists you talk to.

Fecal microbiota transplants, or FMT, are favored by gastroenterologists familiar with them, while infectious disease doctors prefer traditional small molecule therapies, according to a recent future market evaluation by Spherix Global Insights. While current FMT involves transplanting healthy stool into a C. diff patient through a colonoscopy, FMT encapsulated microbiome pills that are swallowed – referred to by doctors as “poop in a pill” in Spherix’s interviews – are likely on the way to market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,200+ biopharma pros reading Endpoints daily — and it's free.