FDA pro­pos­es adding 'race and eth­nic­i­ty' cat­e­go­ry to ex­pand­ed ac­cess re­quests

It’s well un­der­stood that racial and eth­nic mi­nori­ties of­ten lack ac­cess to clin­i­cal tri­als in the US, ei­ther be­cause of re­strict­ed in­clu­sion/ex­clu­sion cri­te­ria or be­cause they don’t have the time, in­sur­ance, in­ter­est or un­der­stand­ing of what clin­i­cal tri­als are avail­able.

But what about eth­nic and racial mi­nori­ties ac­cess­ing the FDA’s ex­pand­ed ac­cess pro­gram?

That’s what the FDA wants to know, as the agency is now propos­ing to add an op­tion­al item re­gard­ing race and eth­nic­i­ty un­der the “Clin­i­cal In­for­ma­tion/Brief Clin­i­cal His­to­ry” field in its form that physi­cians have to fill out for ex­pand­ed ac­cess re­quests for in­di­vid­ual pa­tient INDs.

The FDA’s ex­pand­ed ac­cess or “com­pas­sion­ate use” pro­gram, known as EAP, of­fers peo­ple with se­ri­ous or im­me­di­ate­ly life-threat­en­ing ill­ness­es or con­di­tions ac­cess to po­ten­tial new drugs, even if they don’t qual­i­fy for a clin­i­cal tri­al. The on­ly catch is that the drug­mak­er de­vel­op­ing the po­ten­tial drug has to OK its use out­side of a tri­al, which can be a lim­i­ta­tion to ac­cess, al­though the FDA signs off on more than 99% of all EAP re­quests.

Al­i­son Bate­man-House, an as­sis­tant pro­fes­sor at the NYU Gross­man School of Med­i­cine who stud­ies ex­pand­ed ac­cess, told End­points News that she’s en­cour­aged by this pro­pos­al from FDA to col­lect more race and eth­nic­i­ty da­ta around its EAPs as it will “goad spon­sors in­to col­lect­ing this da­ta” more fre­quent­ly.

“The ques­tion we want an­swered is are some of these mi­nori­ties who are shut out of tri­als able to be ac­com­mo­dat­ed by ex­pand­ed ac­cess, or is it the priv­i­leged few who are get­ting ac­cess to tri­als” and EAPs, she said. “My hunch is the lat­ter.”

The push from FDA comes as it has re­leased a new frame­work around how it plans to bet­ter ad­dress these racial and eth­nic dis­par­i­ties. Part of that frame­work in­cludes set­ting goals for en­roll­ment and spec­i­fy­ing re­ten­tion strate­gies, such as pro­vid­ing lan­guage as­sis­tance and re­duc­ing the bur­dens of par­tic­i­pa­tion with tools like tele­health.

“Go­ing for­ward, achiev­ing greater di­ver­si­ty will be a key fo­cus through­out the FDA to fa­cil­i­tate the de­vel­op­ment of bet­ter treat­ments and bet­ter ways to fight dis­eases that of­ten dis­pro­por­tion­ate­ly im­pact di­verse com­mu­ni­ties,” FDA com­mis­sion­er Rob Califf said in a state­ment.

More peo­ple are al­so in­ter­est­ed in ex­pand­ed ac­cess now be­cause of the pan­dem­ic, Bate­man-House said in ex­plain­ing the re­cent rise in EAPs, par­tic­u­lar­ly as for­mer Pres­i­dent Don­ald Trump used FDA pro­grams to gain ac­cess to Re­gen­eron’s mon­o­clon­al an­ti­body com­bo treat­ment.

For in­stance, CBER re­port­ed more than 6,000 sin­gle pa­tient emer­gency IND re­quests in FY 2020, com­pared with 216 in FY 2021. CDER al­so re­port­ed al­most twice as many re­quests in 2020 when com­pared to oth­er re­cent years.

“The thing we’re think­ing about is how much the struc­tur­al in­equal­i­ties in clin­i­cal tri­als are repli­cat­ed or en­hanced in ex­pand­ed ac­cess,” Bate­man-House added.

And the FDA is ex­pect­ing those high­er num­bers to con­tin­ue, not­ing in the Fed­er­al Reg­is­ter on Wednes­day that its in­for­ma­tion col­lec­tion around EAP “re­flects an in­crease in 254,750 bur­den hours and 11,568 re­spons­es an­nu­al­ly since the last OMB re­view and ap­proval of the in­for­ma­tion col­lec­tion. We at­tribute this to an in­crease in the num­ber of sub­mis­sions.”

The es­ti­mate of 254,750 hours cal­cu­lates out to about 122 new em­ploy­ees work­ing 40 hours per week for one year.

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

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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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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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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.

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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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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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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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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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.”