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.

RWE chal­lenges for to­day's bio­phar­ma

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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As shares suf­fer from a lin­ger­ing slump, a bruised Alk­er­mes slash­es 160 jobs in R&D re­struc­tur­ing

With its share price in a deep slump after suffering through a regulatory debacle over their depression drug ALKS 5461, Alkermes CEO Richard Pops is taking the ax to its R&D organization in a restructuring aimed at cutting costs ahead of its next attempt at a rollout in a tough field.

Richard Pops, Endpoints via Youtube

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Acor­da's Ron Co­hen brings the ax back out as new drug sales on­ly trick­le in while cash cow is led to the slaugh­ter

With its new drug earning meager sums and its one-time cash cow reduced to a bony shadow of its former self, Acorda Therapeutics today is rolling out a new restructuring aimed at slashing the staff and cutting costs to get through the hard times ahead.

The biotech is chopping a quarter of its staff today, carving back R&D as well as SG&A expenses. And CEO Ron Cohen is cutting deep.

Under the new austerity budget, Acorda’s R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million. R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A
expenses for the full year 2020 are expected to be $160 – $165 million.

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RAPT Ther­a­peu­tics re­turns to Wall Street to re­vive IPO bid

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

EY vet set to re­place re­tir­ing Am­gen CFO Meline

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Eli Lil­ly’s USA, di­a­betes chief En­rique Con­ter­no is head­ing out af­ter 27 years, and he’s be­ing re­placed by a com­pa­ny in­sid­er

Close to 3 years after Eli Lilly CEO Dave Ricks added the title of president of the US operations to Enrique Conterno’s resume, which included his helmsmanship of the diabetes franchise, the Peruvian born exec is set to retire after a 27-year run at the pharma giant.

Lilly put out the news just as it was posting Q3 results, with a mix of upbeat and downbeat results in the latest set of numbers from Lilly.
Conterno — a grizzled, deeply experienced and sometimes gruff veteran of the pharma world — was a high-profile figure at Lilly, stepping up to expanded duties as the company was forced to deal with intense pricing pressure on the diabetes side of the business. He had replaced outgoing US president Alex Azar, who later popped up as head of Health and Human Services in the Trump administration.
As head of the diabetes unit, Conterno had to deal with an extraordinarily competitive field as payers demanded bigger discounts. Trulicity’s success helped generate new revenue for the company, but Q3’s miss on revenue had a lot to do with the need for discounting the drug ahead of Novo Nordisk’s rival therapy, Rybelsus, which was priced on the wholesale level at an almost identical rate.

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No­var­tis hands off $80M in cash to part­ner up with a top biotech play­er in the fi­bro­sis sec­tor

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.

Is there a recipe for M&A suc­cess? The best and worst buy­out deals in the past decade of­fer some keys to suc­cess — and fail­ure

It’s not easy achieving a solid win in M&A in this industry. But if you follow a few simple guidelines, you may be able to increase your odds of success.
Geoffrey Porges and the team at SVB Leerink went about the “notoriously difficult” task of scoring the biopharma buyout of 2009 to 2019. Sizing up current and expected revenue from the products that were gained, they came up with the 5 winners:
Merck/Schering Plough
Bristol/Medarex
Gilead/Pharmasset
Sanofi/Genzyme
AstraZeneca/Acerta
It says a lot about the field that it’s much easier sorting out the 5 worst deals, though there’s also a lot more competition for that title, notes Porges. As picked by the analysts:
J&J/Actelion
Merck/Cubist
Alexion/Synageva
AbbVie/Stemcentrx
Gilead/Kite

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