Aca­d­e­mics and re­searchers raise con­cerns with FDA’s plan for ‘in­te­grat­ed re­views’

More than 50 aca­d­e­mics and re­searchers from Har­vard, Yale, Johns Hop­kins and oth­er uni­ver­si­ties around the world are call­ing on the FDA to not re­place its orig­i­nal re­views of med­ical prod­ucts with an “in­te­grat­ed re­view” be­cause of the valu­able in­for­ma­tion that would be lost.

The re­searchers claimed that such a shift would de­prive them of in­for­ma­tion and da­ta on the clin­i­cal stud­ies and tri­als sub­mit­ted to the FDA, in­for­ma­tion on the post­mar­ket­ing re­quire­ments, and re­view­er con­cerns with an ap­pli­ca­tion that might not be in­clud­ed in a sum­ma­ry doc­u­ment like the in­te­grat­ed re­view, among oth­er im­por­tant de­tails.

“The FDA should not re­place in­di­vid­ual re­views with an in­te­grat­ed re­view. Rather, an ad­di­tion­al doc­u­ment that sum­ma­rizes the in­di­vid­ual re­views can be pub­lished, as FDA has al­ready been do­ing in pub­lish­ing ‘Sum­ma­ry Re­view’ doc­u­ments,” the re­searchers wrote.

Sim­i­lar­ly, non­prof­it Pub­lic Cit­i­zen said it “strong­ly ob­jects” to the FDA’s pro­pos­al, which was first ex­plained in a June Fed­er­al Reg­is­ter no­tice. “This ill-con­ceived pro­pos­al would, at the least, be a ma­jor step back­wards in agency trans­paren­cy with re­spect to the da­ta that the agency re­lies on when ap­prov­ing new drugs or bi­o­log­ics and the agency’s as­sess­ment of these da­ta,” Pub­lic Cit­i­zen said.

Health tech­nol­o­gy com­pa­ny Flat­iron Health al­so not­ed that some of the sec­tions in the orig­i­nal doc­u­ment ap­pear to be miss­ing in the new in­te­grat­ed sum­ma­ry. “We al­so note that the new doc­u­ment is con­sid­er­ably short­er than the orig­i­nal, which sug­gests that some po­ten­tial­ly use­ful con­tent may be lost in the new for­mat. We urge FDA to en­sure that high­ly valu­able in­sight in­to re­view­ers’ think­ing is re­tained in the new in­te­grat­ed re­view for­mat. We are par­tic­u­lar­ly in­ter­est­ed in en­sur­ing that any ‘con­sul­ta­tive’ re­views from ex­perts out­side the core re­view team are in­clud­ed in the in­te­grat­ed sum­ma­ry re­view or oth­er­wise ac­ces­si­ble,” the com­pa­ny said.

In­dus­try group BIO, how­ev­er, said the new in­te­grat­ed tem­plate is an im­prove­ment over what the agency of­fers now, but al­so cau­tioned the FDA “to strict­ly lim­it dele­tions to the in­for­ma­tion re­peat­ed across re­view doc­u­ments rather than in­for­ma­tion that FDA deems to not be im­por­tant to ex­ter­nal en­ti­ties.”

BIO of­fered the ex­am­ple of the FDA re­cent­ly trim­ming the con­tent of pub­licly post­ed ac­tion pack­ages, which led to the elim­i­na­tion of “a sig­nif­i­cant num­ber” of meet­ing min­utes. In­dus­try group PhRMA al­so said it is im­por­tant for the FDA to en­sure that the com­bi­na­tion of the in­te­grat­ed re­view doc­u­ment and its ap­pen­dices “is no less com­pre­hen­sive than the ex­ist­ing doc­u­men­ta­tion that is post­ed on Drugs@FDA.”

BIO added: “These doc­u­ments for non-mile­stone de­vel­op­ment phase meet­ings, mid­cy­cle com­mu­ni­ca­tions, and late-cy­cle meet­ings are im­por­tant for stake­hold­ers’ un­der­stand­ing of the evo­lu­tion of the FDA’s think­ing dur­ing the de­vel­op­ment pro­gram and through­out the Agency’s re­view of an ap­pli­ca­tion. Like­wise, ad­min­is­tra­tive and cor­re­spon­dence doc­u­ments should con­tin­ue to be made avail­able.”

On the top­ic of the FDA con­tin­u­ing to re­lease spon­sors’ clin­i­cal study re­ports (CSRs), which the agency is con­sid­er­ing end­ing, the aca­d­e­mics and re­searchers al­so dis­agreed with BIO on what to do.

The aca­d­e­mics and re­searchers of­fered their sup­port for the FDA to con­tin­ue its ef­forts to re­lease CSRs, even though on­ly one has been re­leased so far. But BIO and PhRMA said that the re­lease of CSRs should not be pur­sued by the agency.

BIO al­so called on the FDA to work with the Eu­ro­pean Med­i­cines Agency and Health Cana­da to bet­ter align its trans­paren­cy ef­forts.

Com­ments


RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

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?’”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

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.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

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.”