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.

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Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

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Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

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Sim­mer­ing feud be­tween blue­bird and a ri­val con­tin­ues with al­le­ga­tions of patent fraud

Bluebird bio was hit with a patent infringement lawsuit last week from a Chicago-based biotech it has had an ongoing beef with calling for $2 billion to help cure the “irreparable harm” caused by alleged willful infringement.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

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