FDA com­mis­sion­er Scott Got­tlieb just broke a pub­lic promise on pub­lish­ing CRLs — and yes, it mat­ters

Scott Got­tlieb

In his tes­ti­mo­ny to the US Sen­ate dur­ing his con­fir­ma­tion hear­ings to lead the FDA, Scott Got­tlieb clear­ly enun­ci­at­ed his po­si­tion on pub­lish­ing the com­plete re­sponse let­ters the FDA sends to bio­phar­mas when reg­u­la­tors re­ject their new drug ap­pli­ca­tions.

Vow­ing to achieve greater trans­paren­cy, Got­tlieb promised to open up more. He wrote: “This in­cludes the com­plete re­sponse let­ters, af­ter prop­er redac­tion of com­mer­cial con­fi­den­tial in­for­ma­tion.”

To­day, af­ter hint­ing at it in a few in­ter­views, Got­tlieb sub­stan­tial­ly re­neged on that promise. In­stead of pub­lish­ing redact­ed CRLs, the com­mis­sion­er now says that he may be will­ing to pub­lish pieces of some of the re­jec­tion no­tices — pro­vid­ed it serves what the agency deems is a need to pro­vide in­for­ma­tion rel­a­tive to pub­lic health con­cerns.

In an ad­dress this morn­ing iron­i­cal­ly ti­tled “Fos­ter­ing Trans­paren­cy to Im­prove Pub­lic Health,” Got­tlieb now has this to say on CRLs:

Re­leas­ing all the CRLs would be ad­min­is­tra­tive­ly bur­den­some, giv­en the like­li­hood we would con­tin­ue to redact cer­tain pro­pri­etary in­for­ma­tion from these let­ters. And not all the let­ters have in­for­ma­tion that would di­rect­ly in­form clin­i­cal prac­tice. For ex­am­ple, many let­ters pri­mar­i­ly re­late to man­u­fac­tur­ing short­com­ings with new drug ap­pli­ca­tions that are even­tu­al­ly re­solved.

But some of the let­ters do con­tain in­for­ma­tion that could be di­rect­ly rel­e­vant to pa­tients. We’re eval­u­at­ing whether there is a sub­set of the com­plete re­sponse let­ters where there are es­pe­cial­ly im­por­tant pub­lic health rea­sons to redact and re­lease these let­ters. For ex­am­ple, let­ters that have safe­ty-re­lat­ed find­ings or rec­om­men­da­tions that could help in­form pa­tients and providers about the pro­file of al­ready-mar­ket­ed prod­ucts. Re­leas­ing this in­for­ma­tion could en­hance pa­tient safe­ty, by re­duc­ing the num­ber of po­ten­tial­ly fu­tile tri­als, and spare pa­tients ex­po­sure to po­ten­tial risks with­out the prospect of a like­ly ben­e­fit. It can al­so help bet­ter in­form clin­i­cal prac­tice.

So the FDA, at a time when there are grow­ing con­cerns that po­lit­i­cal in­flu­ence could be shap­ing the agency’s de­ci­sions, now wants to be left in charge of de­cid­ing what is an im­por­tant pub­lic health rea­son and what isn’t.

And why did Got­tlieb re­treat now? What changed his mind?

Trans­paren­cy at the FDA is a pre­cious com­mod­i­ty, rarely found and of­ten bad­ly ne­glect­ed. Why don’t we let the peo­ple de­cide for them­selves what is im­por­tant and what is not in a CRL? If it’s a rou­tine mat­ter that can be eas­i­ly re­solved, then it should be a boon to the com­pa­ny in­volved to have that re­leased. If the com­pa­ny screwed up their da­ta, can’t prove ef­fi­ca­cy or raise unan­swered safe­ty is­sues, re­gard­less of what­ev­er class of drugs — on or off the mar­ket — this could re­late to, the pub­lic has a right to know.

Aside from in­form­ing the pub­lic about this process, re­searchers at the NIH, in acad­e­mia and com­pa­nies work­ing in drug de­vel­op­ment all have their own need to see be­hind the veil.

Every­one has a clear right to what Got­tlieb promised to win Sen­ate sup­port — which on­ly re­peat­ed his ear­li­er state­ments sup­port­ing the pub­li­ca­tion of CRLs. It’s all a pub­lic health is­sue, and com­pa­nies will be­have bet­ter if they know their own worst mis­steps will be a mat­ter of pub­lic record.

Too bur­den­some? Then pub­lish with­out a redac­tion. Prob­lem solved. Bur­den lift­ed. Or just try and match the greater trans­paren­cy achieved in Eu­rope, where no great hur­dles had to be over­come.

As of now, the FDA has re­versed it­self on three CRLs since Got­tlieb took the head of­fice at the FDA. We don’t know for cer­tain what prompt­ed the CRLs, we don’t know why the FDA changed its mind — though we do know that at least one of the com­pa­nies was will­ing to lob­by se­nior of­fi­cials at the agency with a case that it nev­er went pub­lic with.

That’s the op­po­site of trans­paren­cy.

As of now, this is the first promise Got­tlieb has clear­ly bro­ken.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

[via AP Images]

Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.