Da­ta sug­gest US, UK uni­ver­si­ties fall woe­ful­ly short on re­port­ing clin­i­cal tri­al re­sults

Clin­i­cal tri­al da­ta are used by pa­tients, doc­tors and pol­i­cy­mak­ers to make in­formed choic­es about the ben­e­fits and safe­ty of in­ter­ven­tions — while the meth­ods and re­sults of all tri­als are cru­cial to the pace and di­rec­tion of sci­en­tif­ic progress. How­ev­er, there is a large body of ev­i­dence that sug­gests that com­plet­ed clin­i­cal tri­als are com­mon­ly left un­re­port­ed, and ed­u­ca­tion­al in­sti­tu­tions in the Unit­ed States and the Unit­ed King­dom — ar­guably the two biggest re­gions that breed the bulk of med­ical in­no­va­tion — have emerged as one of the key cul­prits guilty of these vi­o­la­tions.

In the Unit­ed States, Con­gress passed a law in 2007 re­quir­ing tri­al spon­sors — in­clud­ing uni­ver­si­ties — to post the re­sults of cer­tain clin­i­cal tri­als on clin­i­cal­tri­als.gov with­in a year of tri­al com­ple­tion, and a decade lat­er in Jan­u­ary 2017 the rule was fi­nal­ized. Since 2017, 40 lead­ing US uni­ver­si­ties should have post­ed the re­sults of 450 clin­i­cal tri­als — but over a third (31%) of those re­sults are miss­ing, ac­cord­ing to an analy­sis by Uni­ver­si­ties Al­lied for Es­sen­tial Med­i­cines (UAEM) in part­ner­ship with non-prof­it re­search ad­vo­ca­cy group TranspariMED.

The vi­o­la­tors in­clude some of the most ac­tive tri­al spon­sors: For ex­am­ple the MD An­der­son Can­cer Cen­ter, which has on­ly re­port­ed 77% of due tri­als, Mayo Clin­ic (42%), UC San Fran­cis­co (37%), New York Uni­ver­si­ty (21%), and Co­lum­bia Uni­ver­si­ty (17%).

A sum­ma­ry of re­sults by per­cent­age of each uni­ver­si­ty eval­u­at­ed can be seen be­low:

Source: UAEM, TranspariMED

Click on the im­age to see the full-sized ver­sion

Over­all, 140 clin­i­cal tri­als are still miss­ing re­sults and five uni­ver­si­ties are re­spon­si­ble for half of the un­re­port­ed tri­als: Uni­ver­si­ty of Cal­i­for­nia San Fran­cis­co (17 tri­als with­out re­sults), Co­lum­bia (15 tri­als), Mayo Clin­ic (13), MD An­der­son Can­cer Cen­ter (12) and Chica­go (8), ac­cord­ing to the re­port.

These trans­paren­cy vi­o­la­tions are con­cern­ing con­sid­er­ing at least half of the valu­able med­i­cines that ex­ist to­day were orig­i­nal­ly de­vel­oped in uni­ver­si­ty labs with tax­pay­er fund­ing, in­clud­ing al­most all vac­cines, many HIV and tu­ber­cu­lo­sis drugs, and even in­sulin, the re­port not­ed. Be­tween 2010 and 2016, every sin­gle one of the 210 FDA-ap­proved med­i­cines can be traced back to fund­ing from the NIH, ac­cord­ing to a study pub­lished in the of­fi­cial jour­nal of the Na­tion­al Acad­e­my of Sci­ences.

Mean­while, these trans­paren­cy trans­gres­sions are echoed in the UK. Reg­u­la­tions in Eu­rope are sim­i­lar. Any tri­al of of any med­i­c­i­nal prod­uct con­duct­ed since 2004 in an EU coun­try has al­ready been re­quired to reg­is­ter on the Eu­ro­pean Union Clin­i­cal Tri­als Reg­is­ter (EU­C­TR) and since 2012, spon­sors must en­sure that all reg­is­tered tri­als since 2004 dis­close their re­sults to the EMA with­in 12 months of tri­al com­ple­tion. But the de­lays to the EMA’s soft­ware plat­form pushed the fi­nal date for re­sults post­ing by spon­sors to late De­cem­ber 2016.

Ben Goldacre

In a BMJ study pub­lished in 2018 — led by Ben Goldacre, a best-sell­ing au­thor, med­ical doc­tor and re­searcher who fo­cus­es on un­pack­ing the mis­use of sci­ence and sta­tis­tics in his books Bad Sci­ence and Bad Phar­ma — it was found that in Eu­rope, of the 7274 tri­als where re­sults were due, 49.5% re­port­ed re­sults. Tri­als with a com­mer­cial spon­sor (such as a drug de­vel­op­er) were sub­stan­tial­ly more like­ly to post re­sults than those with a non-com­mer­cial spon­sor (68.1% v 11.0%), the analy­sis sug­gest­ed.

Out of his labs at the Uni­ver­si­ty of Ox­ford, Goldacre set up an EU Tri­al­sTrack­er to con­tin­u­ous­ly mon­i­tor the re­port­ing of tri­als. As of 10 Jan­u­ary 2019, Goldacre and his team have iden­ti­fied 8,062 reg­is­tered tri­als that are ‘un­am­bigu­ous­ly’ due to re­port re­sults — but re­sults on just over half  (53.6%) have been post­ed to the reg­istry. The da­ta, which sug­gest­ed that UK uni­ver­si­ties were less re­li­able than drug de­vel­op­ers, sparked the in­ter­est of House of Com­mons Sci­ence and Tech­nol­o­gy Com­mit­tee. UK uni­ver­si­ties could be brought in front of the com­mit­tee if they fail to im­prove their track record, and the com­mit­tee will ask them to ex­plain them­selves in a fol­low-up ev­i­dence ses­sion if im­prove­ments are not made.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

'We kept at it': Jef­frey Blue­stone plots late-stage come­back af­ter teplizum­ab shown to de­lay type 1 di­a­betes

Late-stage da­ta pre­sent­ed at the Amer­i­can Di­a­betes As­so­ci­a­tion an­nu­al meet­ing in 2010 pushed Eli Lil­ly to put a crimp on teplizum­ab as the phar­ma gi­ant found it un­able to re­set the clock on new­ly di­ag­nosed type 1 di­a­betes. At the same con­fer­ence but in dif­fer­ent hands nine years lat­er, the drug is mak­ing a crit­i­cal come­back by scor­ing suc­cess in an­oth­er niche: de­lay­ing the on­set of the dis­ease.

In a Phase II tri­al with 76 high-risk in­di­vid­u­als — rel­a­tives of pa­tients with type 1 di­a­betes who have di­a­betes-re­lat­ed au­toan­ti­bod­ies in their bod­ies — teplizum­ab al­most dou­bled the me­di­an time of di­ag­no­sis com­pared to place­bo (48.4 months ver­sus 24.4 months). The haz­ard ra­tio for di­ag­no­sis was 0.41 (p=0.006).

Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.