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.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

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Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

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Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

UCB adds on more pos­i­tive PhI­II da­ta for IL-17A/17F in­hibitor bimek­izum­ab, clear­ing a path to the FDA

A month after posting positive top-line data from their first Phase III trial of the IL-17A/17F inhibitor bimekizumab, Belgium’s UCB says they’ve added more upbeat results from their second late-stage test in moderate-to-severe plaque psoriasis.

That leaves the company on track for regulatory submissions in the middle of next year, says CMO Iris Loew-Friedrich.
Their drug beat out a placebo on the co-primaries — a 90% improvement in PASI 90 (the Psoriasis Area and Severity Index) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1) at week 16, compared to placebo. Investigators also boasted of hitting some key secondaries.
UCB is angling to enter an increasingly crowded market space.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.

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