CBO finds Sen­ate's FDA user fee bill will re­duce the fed­er­al deficit by about $1.4B

As de­bate rages on Capi­tol Hill over whether to add rid­ers, and which rid­ers to add, to the must-pass FDA user fee leg­is­la­tion, the Con­gres­sion­al Bud­get Of­fice on Thurs­day made clear that the Sen­ate ver­sion will re­duce the fed­er­al deficit by about $1.4 bil­lion over the next 10 years.

Chad Land­mon

Con­gress has un­til the end of Sep­tem­ber to reau­tho­rize the user fees, which the FDA col­lects for its re­view of pre­scrip­tion drugs, gener­ics, biosim­i­lars and med­ical de­vices.

While there are still ma­jor gaps be­tween the Sen­ate and House ver­sions, and health com­mit­tee rank­ing mem­ber Richard Burr (R-NC) re­cent­ly in­tro­duced a blank ver­sion of the bill with no rid­ers, many ex­pect the user fee bills will still get done on time.

“I gen­er­al­ly still think that a user fee deal will get done be­fore the end of Sep­tem­ber giv­en that there is broad, bi­par­ti­san sup­port for user fees,” Chad Land­mon, part­ner at the law firm Ax­inn, Vel­trop & Harkrid­er, told End­points News via email, adding:

there’s still a lot of dis­tance be­tween the House and Sen­ate bills, and there is a lot of po­lit­i­cal pres­sure on the Biden Ad­min­is­tra­tion and De­moc­rats in Con­gress to ad­dress drug costs more broad­ly. So, it’s al­ways pos­si­ble that the po­lit­i­cal pres­sure and dis­putes over oth­er pol­i­cy is­sues get in the way and de­lays the pas­sage of user fee leg­is­la­tion. But, I think the gen­er­al con­sen­sus by pol­i­cy­mak­ers and in­dus­try that user fees have had a pos­i­tive im­pact on FDA’s abil­i­ty to do its job will hope­ful­ly over­come oth­er, com­pet­ing con­cerns.

In un­veil­ing its deficit-re­duc­ing score, CBO points to five key sec­tions of the bill, known as S.4348 or the FDASLA Act of 2022, that will have the biggest im­pact.

First, the non-par­ti­san or­ga­ni­za­tion point­ed to Sec­tion 509, which would au­tho­rize the FDA to ap­prove a gener­ic drug ap­pli­ca­tion (AN­DA) even if the la­bel dif­fers from that of the brand name prod­uct when the brand-name la­bel changes with­in 90 days of the date on which the gener­ic ap­pli­ca­tion would oth­er­wise have been el­i­gi­ble for ap­proval.

The spon­sor of the gener­ic would still be re­quired to up­date that drug’s la­bel with­in 60 days of ap­proval, but based on con­ver­sa­tions with stake­hold­ers, “CBO ex­pects that the pro­vi­sion would ac­cel­er­ate the avail­abil­i­ty of low­er-priced gener­ic drugs be­cause man­u­fac­tur­ers would bring them to mar­ket ear­li­er than un­der cur­rent law,” there­by re­duc­ing the av­er­age prices for those drugs that are paid for by fed­er­al health pro­grams.

In an­oth­er move to ac­cel­er­ate the avail­abil­i­ty of low­er-priced drugs, sec­tion 511 of FDASLA would al­low the FDA to de­ny a cit­i­zen pe­ti­tion if it de­ter­mines that the pe­ti­tion was sub­mit­ted with the pri­ma­ry pur­pose of de­lay­ing the ap­proval of an ap­pli­ca­tion or if it doesn’t raise valid sci­en­tif­ic or reg­u­la­to­ry is­sues. These types of pe­ti­tions are fre­quent­ly sub­mit­ted and used as a de­lay­ing tac­tic when gener­ic drugs are read­ied for launch.

The bill al­so would re­quire a pe­ti­tion to be sub­mit­ted with­in 60 days af­ter the pe­ti­tion­er knew, or rea­son­ably should have known, the in­for­ma­tion that forms the ba­sis of the pe­ti­tion, there­by push­ing com­pa­nies away from fil­ing these pe­ti­tions at the last minute.

“CBO ex­pects that the new time­ly-sub­mis­sion re­quire­ment and re­lat­ed changes to the dis­missal pro­ce­dures in­volv­ing civ­il ac­tions would fur­ther en­hance the FDA’s abil­i­ty to ex­pe­di­tious­ly de­ny pe­ti­tions that oth­er­wise would have de­layed the mar­ket­ing ap­proval of gener­ic or biosim­i­lar ap­pli­ca­tions,” the CBO said.

Sec­tion 512 of the Sen­ate bill, high­light­ed by CBO, would al­so ac­cel­er­ate the avail­abil­i­ty of low­er-priced gener­ic drugs, there­by sav­ing the gov­ern­ment mon­ey, as it would au­tho­rize the FDA to dis­close qual­i­ta­tive and quan­ti­ta­tive in­for­ma­tion on the in­ac­tive in­gre­di­ents of the brand-name drug in re­sponse to a re­quest in a con­trolled cor­re­spon­dence from a gener­ic drug man­u­fac­tur­er.

Kurt Karst

An­oth­er con­tro­ver­sial sec­tion, sec­tion 515, would al­low the FDA to ap­prove a sub­se­quent AN­DA, un­der cer­tain con­di­tions, if the first ap­pli­cant that is el­i­gi­ble for 180 days of gener­ic mar­ket ex­clu­siv­i­ty has not yet be­gun mar­ket­ing.

While the CBO ex­pects that the pro­vi­sion would ac­cel­er­ate the avail­abil­i­ty of low­er-priced gener­ic drugs be­cause man­u­fac­tur­ers would bring them to mar­ket ear­li­er than un­der cur­rent law, gener­ic drug ex­perts think this sec­tion might do the re­verse.

Kurt Karst, reg­u­la­to­ry coun­sel at Hy­man, Phelps & Mc­Na­ma­ra, pre­vi­ous­ly told End­points that this pro­vi­sion could cre­ate new lit­i­ga­tion.

“This new pro­vi­sion cre­ates an ex­cep­tion to 180-day ex­clu­siv­i­ty to al­low a sub­se­quent ap­pli­cant to, in a sense, leapfrog in­to first ap­pli­cant sta­tus al­most, and be able to get ap­proved. Notwith­stand­ing a first ap­pli­cant’s ex­clu­siv­i­ty sub­ject to a se­ries of five events. Some of them are sub­ject to quite a bit of dis­cre­tion and any­time the D word comes up, that of­ten­times means the L word, right? The law­suit,” he said dur­ing a re­cent End­points pan­el dis­cus­sion on the user fee bill.

And CBO point­ed to Sec­tion 605 of the bill, which would reau­tho­rize through 2027 a pro­vi­sion that may slight­ly in­crease the deficit over 10 years and al­lows spon­sors of drugs de­vel­oped from a par­tic­u­lar type of mol­e­cule called an enan­tiomer to gain five years of da­ta ex­clu­siv­i­ty un­der cer­tain cir­cum­stances.

Un­der cur­rent law, the CBO says, the “max­i­mum pe­ri­od of da­ta ex­clu­siv­i­ty would re­vert to 3 years in 2023. (Da­ta ex­clu­siv­i­ty be­gins when a drug is ap­proved by the FDA. Dur­ing that time, the agency does not ac­cept ap­pli­ca­tions for mar­ket­ing ap­proval for gener­ic ver­sions of the drug.) Based on his­tor­i­cal da­ta, CBO ex­pects that ex­tend­ing mar­ket ex­clu­siv­i­ty for pre­scrip­tion drugs de­vel­oped from enan­tiomers would, in some cas­es, de­lay the en­try of low­er-priced gener­ic ver­sions of those drugs.”

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

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With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Lina Khan, FTC chair (Graeme Jennings/Pool via AP)

FTC makes an ex­am­ple of GoodRx, bans dis­counter from shar­ing pri­vate health da­ta with ad­ver­tis­ers

Prescription drug discount provider GoodRx will no longer be allowed to share its users’ sensitive health data with advertisers after the Federal Trade Commission charged the online coupon provider with failing to notify consumers of such disclosures to Facebook, Google, and other companies.

GoodRx agreed to pay a $1.5 million civil penalty for violating the FTC’s Health Breach Notification Rule after the FTC said it repeatedly violated a 2017 promise to not share sensitive personal health information. The FTC alleged that the company shared users’ prescription medications and personal health conditions with third party advertisers and platforms like Facebook, Google, Criteo, Branch and Twilio.

FDA ap­proves GSK's ane­mia drug with safe­ty warn­ing — af­ter bat­ting back sim­i­lar drugs

GSK has secured the first of four US approvals it’s hoping for this year, as the FDA greenlit daprodustat as a treatment for anemia due to chronic kidney disease.

But the FDA limited the use of the drug, to be marketed as Jesduvroq, to patients who have been receiving dialysis for at least four months and stopped short of approving it for patients not dependent on dialysis — in line with the recommendations of the advisory committee it consulted.

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Photo: Julia Weeks/AP Images

FDA ax­es re­quire­ment for pos­i­tive Covid test be­fore Paxlovid use

FDA announced today that doctors and pharmacists can now prescribe Paxlovid to patients without a positive test for Covid-19.

CDER Director Patrizia Cavazzoni reissued Paxlovid’s authorization letter Wednesday, saying it has revised the authorization to “no longer require positive results of direct SARS-CoV-2 viral testing.” The EUA now requires instead that adults and kids 12 years of age and older have a “current diagnosis of mild-to-moderate COVID-19.”

David Kirn, 4D Molecular Therapeutics CEO (via website)

FDA places hold on 4D Mol­e­c­u­lar’s Fab­ry gene ther­a­py

4D Molecular Therapeutics quietly tucked an FDA clinical hold on its Fabry gene therapy into an SEC filing.

Meanwhile, the biotech issued a press release the same day after the closing bell on Thursday touting an IND for another asset, in diabetic macular edema.

The California biotech had paused enrollment of patients in its two trials of the Fabry gene therapy (4D-310) last month after three patients experienced kidney issues, all of which were resolved within four weeks. At the time, 4DMT said it would wait until the second half of this year to look at 12-month clinical data on six patients in the Phase I/II trials, one in the US and one in Taiwan and Australia.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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